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1377 Results

Pediatric Exclusivity-Associated Revenues and Labeling Changes, 2013-2023

Liu ITT, Raymakers AJN, Sarpatwari A, Kesselheim AS - Journal of Pediatrics

  • Research Publications
| September 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The Best Pharmaceuticals for Children Act (BPCA) provides a 6-month exclusivity for manufacturers that conduct additional pediatric studies of their products. 97% of pediatric exclusivities granted over a 10-year period resulted in new pediatric efficacy data added to the drug label. However, this data was often generated a decade after market entry, generating a median excess revenue of $134 million per exclusivity, suggesting optimization of BPCA requirements may be necessary to facilitate timely evidence generation.
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National Analysis of the Requirements and Implementation of State Prescription Drug Price Transparency Laws

Rahim H, Kesselheim AS - Milbank Quarterly

  • Research Publications
| September 2025
  • Price, Value, and Access
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In this review of the design and implementation of prescription drug price transparency laws across 21 US states, the authors find that while these laws have improved public access to pricing data and informed state-level policymaking, there is limited evidence that such policies have curbed drug prices or altered manufacturer behavior. Stronger enforcement mechanisms and data quality standards may enhance the impact of these transparency efforts.
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Fireproofing the FDA: Power, Politics, and Public Health

Daval CJR, Deshmukh A, Sinha M - University of Utah Quinney College of Law

  • Presentations & Panels
| September 2025
  • Regulation and Clinical Evidence
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Joseph Daval, Anjali Deshmukh, and Michael Sinha joined other FDA scholars and journalists for a University of Utah Quinney College of Law symposium on how to rebuild and improve the FDA amid a period of political interference and upheaval.
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The Advisory Committee on Immunization Practices-Legal Roles, Challenges, and Guardrails

Daval CJR, Kesselheim AS - JAMA

  • Commentary & Opinion
| August 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
The authors discuss the legal foundations of the CDC’s Advisory Committee on Immunization Practices (ACIP), a body that makes vaccine recommendations that have been statutorily embedded in numerous federal programs. They discuss the legality of disbanding ACIP, removing committee members, and the HHS Secretary to override ACIP decisions, highlighting the need for Congressional action to protect the committee’s independence and maintain a mechanism for evidence-based vaccine policy deliberation.
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Advance Market Commitments and Their Role in Public Innovation

Nagar SN, Cacodcar A, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Innovation Incentives and Competition
Talk bubble graphics representing commentary and opinion.
Advance market commitments (AMCs) have potential as an alternative funding mechanism for drug development, as seen with the Gavi pneumococcal vaccine initiative and Operation Warp Speed for COVID-19 vaccines. However, AMCs’ effectiveness is maximized when combined with robust public research funding and complementary policy tools.
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Health Professional Students’ Knowledge About FDA Drug and Medical Device Regulation

Sangha KK, Redberg RF, Kesselheim AS, Woloshin S, Dhruva SS - Academic Medicine

  • Research Publications
| August 2025
  • Regulation and Clinical Evidence
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A survey of final-year medical, pharmacy, and nurse practitioner students at the University of California, San Francisco identified significant gaps in students’ understanding of FDA drug and device regulation, as well as the quality of clinical trial evidence. Most students reported only basic or cursory teaching on these topics and struggled with key concepts, highlighting the need for stronger education on regulatory processes to better prepare future clinicians.
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Estimating Costs of Market Exclusivity Extensions for 4 Top-Selling Prescription Drugs in the US

Hong D, Tu SS, Beall RF, Russo M, Rome BN, Kesselheim AS, Sarpatwari A - JAMA Health Forum

  • Research Publications
| August 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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For four top-selling drugs with US market exclusivity beyond key patent expirations, this extended exclusivity was estimated to delay generic competition by 7 to 13 months, resulting in an estimated $3.5 billion in excess US spending on these products over two years in the absence of the exclusivity extension. These findings underscore how timely generic entry could yield substantial savings and the need for policy reforms to limit exclusivity extensions for top-selling products.
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Drug Versioning and Legal Accountability (Reply)

Tu SS, Bonis T - JAMA

  • Commentary & Opinion
| August 2025
  • Regulation and Clinical Evidence
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In response to comments on their prior analysis, the authors clarify that legal accountability for delayed drug releases should apply only when companies knowingly withhold safer alternatives, arguing that such liability is necessary to protect public health and prevent such practices from becoming industry standard.
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Benefits and Limitations of Direct-To-Consumer Pharmacies in the US

Lalani HS, Kesselheim AS, Rome BN - JAMA Network Open

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Commenting on an analysis of the prices of generic drugs for neurologic conditions available via a direct-to-consumer pharmacy, the authors emphasize that while direct-to-consumer pharmacies can offer lower prices for some generic drugs compared to traditional insurance, broader reforms are needed to improve drug affordability and access in the US.
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Combating Pharmaceutical Patent Thickets in the Trump Administration

Tu SS, Kesselheim AS, Feldman WB - Health Affairs Forefront

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
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The authors provide an overview of how pharmaceutical companies use patent thickets to delay generic and biosimilar competition and highlight bipartisan federal legislation like the ETHIC Act that could achieving meaningful patent reform.
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The Limits of Physician-Level Interventions for Cost-Related Nonadherence: A Case for Structural Reform

Eisenkraft Klein D, Kesselheim AS, Rand LZG - American Journal of Bioethics

  • Commentary & Opinion
| August 2025
  • Price, Value, and Access
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Commenting on an article discussing clinicians’ responsibility to mitigate cost-related nonadherence, the authors contend that while physicians do have a role in helping patients navigate high drug costs, lasting solutions require systemic reforms in pharmaceutical pricing, regulation, and patent law, rather than relying solely on individual clinicians.
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The Puzzle of Biologics Manufacturing Platform Patents (Reply)

Jefferson OA, Price WN, Tu SS, Vishnubhakat S, Rai AK - Nature Biotechnology

  • Commentary & Opinion
| August 2025
  • Innovation Incentives and Competition
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The authors respond to Amgen’s critique of the authors’ prior analysis of biologic platforms patents, reemphasizing how post-approval manufacturing patents and opaque litigation practices can delay biosimilar entry even after regulatory approval.
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Health Technology Assessment, Again: A Transparent, Evidence-Based Approach for CMS Drug Price Negotiations

Ollendorf DA, Rand LZG, Blonda A, Mooney H, Phillips M, Basu A - Health Affairs

  • Research Publications
| August 2025
  • Price, Value, and Access
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CMS should adopt a formal health technology assessment (HTA) framework for Medicare drug price negotiations that integrates comparative value assessment, multicriteria decision analysis, and independent advisory committee deliberation to ensure fair pricing and public trust in the negotiation process.
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Expansion of 340B Disproportionate Share Hospitals in the United States From 2010 to 2022

Liu BY, Russo M, Kesselheim AS, Knox RP, Sarpatwari A, Feldman WB - Health Services Research

  • Research Publications
| August 2025
  • Price, Value, and Access
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Between 2010 and 2022, new child sites and contract pharmacies associated with 340B disproportionate share hospitals were increasingly established in wealthier areas, with the Social Deprivation Index (SDI) for these sites decreasing from a median of 59 and 72, respectively, in 2010 to 53 and 53 by 2022. This shift towards serving more affluent communities raises questions about the alignment of the 340B program’s expansion with its original mission.
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Differences Between Trial Populations and Approved Label Populations of New Drugs in the United States and Europe (2012 to 2023): A Cross-Sectional Study

Vokinger KN, Serra-Burriel M, Glaus CEG, Welti L, Ross JS, Kesselheim AS - Annals of Internal Medicine

  • Research Publications
| August 2025
  • Regulation and Clinical Evidence
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In a review of 263 drugs approved in the US, EU, and Switzerland from 2012 to 2023, regulatory agencies were found to often approve drugs for use in populations broader than those studied in pivotal trials, particularly in the US. Labels were most commonly broadened by “patient fitness” (e.g., health status criteria like comorbidities, lab tests, and performance scores), meaning that drugs were often approved for patients who would not have qualified for the trials that supported their approval. These findings underscore the need for post-approval studies to ensure safety and efficacy in patient populations excluded from trials.
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Community Pharmacy Turnover and Context of Openings and Closings By Ownership Type

Mattingly II TJ, Sahu M, Anderson KE - JAMA Health Forum

  • Research Publications
| August 2025
  • Price, Value, and Access
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In this analyis of the rate of community pharmacy openings and closures in the US, market turnover was 86.8% between 2010 and 2023, corresponding to an annual turnover rate of 6.2%. Independent pharmacies opened and closed more frequently than chain pharmacies. These market shifts reflect the further need for research into the patient impact of pharmacy closures in the US.
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Insurer Exits After the Inflation Reduction Act Part D Redesign

Cai CL, Kesselheim AS, Bhaskar A, Rome BN - JAMA

  • Research Publications
| July 2025
  • Price, Value, and Access
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Following changes in Medicare Part D plan design under the Inflation Reduction Act, 7.5% of beneficiaries (2.9 million people) lost their Part D insurer in 2024, a sharp rise from prior years, with higher insurer exit rates among standalone and enhanced plans. These findings suggest a need for additional policies to ensure adequate competition and affordability in the Part D marketplace.
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Prescription Drug Method-Of-Use Patent Protection, 1991-2018

Bloomfield D, Teng TW, Kesselheim AS, Tu SS - Journal of General Internal Medicine

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Method-of-use patents have increasingly extended brand-name drugs’ market exclusivity in the US, with the median additional protection rising from 3.2 years in 1991 to 5.4 years in 2018. This shift has resulted in a total median patent protection of nearly 15 years after FDA approval for many products, emphasizing the need for the preservation of the “skinny-labeling” pathway and other strategies to enable timely generic competition.
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Share of Sales Subject to Medicare Inflation Rebates and Price Increases of Top-Selling Drugs

Egilman AC, Kesselheim AS, Rome BN - Health Services Research

  • Research Publications
| July 2025
  • Price, Value, and Access
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Top-selling drugs that were the most affected by Medicare inflationary rebates in 2023 due to a higher share of Medicare sales did not have larger year-over-year price increases than drugs less affected by the rebates. This suggests that although the rebates will save money for Medicare, there may not be spillover effects of lower prices for other payers; additional measures, such as extending inflation rebates to commercial insurance, may be needed to prevent manufacturers from raising prices on existing brand-name drugs.
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Revenue, Patents, and New Generic Competition for Prescription Drugs in the USA

Tu SS, Kesselheim AS, Ross JS, Gupta R - Journal of General Internal Medicine

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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Among 178 brand-name drugs approved in the US between 2000 and 2012, those with higher revenue were more likely to face generic competition, while those with more patents had fewer generic competitors. Specifically, every $100 million increase in average brand-name revenue was associated with an 8% increase in the likelihood of generic approval, while each additional patent reduced that likelihood by 7%.
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Impostor Syndrome: Fraudulent Participants in Qualitative Research Can Skew Results

Rand LZG, McGraw SA, Wang J, Darrow JJ, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Regulation and Clinical Evidence
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The authors discuss how fraudulent participants in qualitative health research can meaningfully distort study findings, emphasizing the need for robust screening and reporting strategies to protect data validity.
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PORTAL Post | July 2025

  • PORTAL Post
| July 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The July 2025 edition of the PORTAL Post monthly newsletter.
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Trump’s Ongoing Tariff Chaos Will Make Medications Less Affordable and Harder to Find

Kesselheim AS, Avorn J - Health Affairs Forefront

  • Commentary & Opinion
| July 2025
  • Price, Value, and Access
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Proposed tariffs on imported pharmaceuticals will likely raise medication costs and worsen drug shortages, while ignoring the root causes of high drug prices in the US and risking harms to patient access.
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The Strength and Importance of Government-Funded Patents for Approved Drugs

Gabriele SME, Martin MJ, Kesselheim AS, Tu SS - Nature Biotechnology

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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In this examination of the role of US federal funding in late-stage drug development, 2.5% of small molecule drug patents listed in the FDA Orange Book were found to cite government funding. However, these government-funded patents were more likely to be primary patents and to receive patent term extensions, indicating their strength and value. Recognizing the public’s investment in drug discovery should both inform efforts to address high drug prices and reemphasize the importance of robust federal funding for biomedical research.
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Frequency and Nature of Generic “Design Around” of Brand‐name Patents in the United States

Freilich J, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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In 153 patent litigation cases regarding generic manufactuers’ efforts to “design around” brand-name drug patents, 82% of cases involved changes to the physical product or packaging, with 48% altering the drug’s formulation, 12% changing the active ingredient, and 10% modifying the manufacturing process. These design-around strategies can help lower drug prices and increase market competition.
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Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis

Hogervorst MA, Vreman RA, Oduol TA, Mantel‐Teeuwisse AK, Goettsch WG, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| July 2025
  • Regulation and Clinical Evidence
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Across five countries they find that approved patient populations for oncology drugs are adjusted an average of 10 times throughout a drug’s market lifecycle. Drug developers made the most adjustments, often expanding the patient population, while regulators and HTA organizations often imposed restrictions. How patient populations were described was also inconsistent, highlighting the need for a standardized patient population reporting framework.
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Design Patents: A Potential Threat to Drug Competition

Freilich J, Kesselheim AS - Applied Health Economics and Health Policy

  • Research Publications
| July 2025
  • Innovation Incentives and Competition
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Drug design patents, which protect the ornamental appearance of drugs and delivery devices, are increasingly prevalent, are often easier and quicker to obtain than utility patents, and may complicate generic and biosimilar competition. To mitigate these impacts, USPTO should avoid granting design patents for drugs and the FDA should prohibit listing such patents in the Orange Book.
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Data Manipulation Within the US Federal Government

Freilich J, Kesselheim AS - Lancet

  • Research Publications
| July 2025
  • Regulation and Clinical Evidence
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The authors document the widespread, unlogged changes to federal health datasets, most notably the replacement of “gender” with “sex,” and raise concerns about data integrity, transparency, and the reliability of the research and policy decision-making that depend on these resources.
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Examining Pricing and Availability for Neglected Tropical Disease Therapies in the United States

Goh M, Outterson K, Kesselheim AS - Open Forum Infectious Diseases

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Many essential therapies for neglected tropical diseases (NTDs) remain expensive and vulnerable to shortage in the US, in part due to few manufacturers and supply chain fragility. Policy interventions such as public manufacturing and supply chain resilience efforts may help improve access to affordable, reliable treatment for these diseases
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Congress Should Remove the Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It

Mooney H, Kesselheim AS, Rome BN - Health Affairs Forefront

  • Commentary & Opinion
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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An expansion of the orphan drug exemption in Medicare drug price negotiation would shield high-revenue drugs from negotiation, reduce savings, and create new perverse incentives for drug manufacturers to avoid negotiation eligibility. The rare disease exemption should instead be removed to enable greater savings and improve affordability of rare disease drugs for Medicare patients.
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Disappearing Data at the U.S. Federal Government

Freilich J, Price WN, Kesselheim AS - New England Journal of Medicine

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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The authors discuss the alarming trend of disappearing health datasets from U.S. federal health agencies. Between January 21 and February 11, 2025, the CDC alone removed 203 datasets, undermining scientific research, public health efforts, and policy-making. As government datasets disappear, stakeholders are forced to turn to private sources, increasing costs and potentially reducing research quality. Action is needed to ensure the government continues to collect and publish high-quality data.
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Comments on Initial Guidance for the Medicare Drug Price Negotiation Program, IPAY 2028

Kim J, Kesselheim AS, Rome BN - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| June 2025
  • Price, Value, and Access
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The commenters support CMS’s proposed clarification that fixed-combination drugs containing therapeutically inactive ingredients (such as hyaluronidase used for subcutaneous delivery) should be aggregated with the original formulations for price negotiation purposes, preventing manufacturers from evading negotiation through strategic reformulations. They also recommend that CMS close a related loophole by aggregating spending only across formulations without generic or biosimilar competition when selecting drugs for negotiation.
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What Can We Learn From Patient Input at FDA Advisory Committee Meetings?

Rand LZG, Eisenkraft Klein D - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Summer 2025
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
Commenting on an analysis of FDA advisory committee decisions, the authors explore the complex role of patient testimony in regulatory deliberations. While patient input can offer valuable insights into lived experience and unmet needs, tensions exist between experiential narratives and evidence-based decision-making, especially in data-limited contexts. Clearer frameworks are needed to ensure that patient engagement enhances transparency and legitimacy without compromising scientific rigor.
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PORTAL Post | June 2025

  • PORTAL Post
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
A graphic icon of a megaphone as a symbol for the PORTAL Post email newsletter.
The June 2025 edition of the PORTAL Post monthly newsletter.
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Medicare Spending and Use of Subcutaneous Biologic Formulations with Hyaluronidase

Kim J, Kesselheim AS, Cliff ERS, Rome BN - The Oncologist

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Medicare spending on biologic drugs reformulated with hyaluronidase for subcutaneous administration totaled $10.3 billion in 2022. While these formulations may offer clinical advantages—such as fewer infusion reactions and reduced delivery time for patients—the timing of their introduction raises concerns about “hyaluronidase hopping” as a strategy to delay biosimilar competition and Medicare drug price negotiation. Policy safeguards are needed to ensure that such reformulations can still provide clinical value without undermining cost-containment efforts.
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Use and Cost of First-Line Biologic Medications to Treat Plaque Psoriasis in the US

Rome BN, Han J, Mooney H, Kesselheim AS - JAMA Dermatology

  • Research Publications
| June 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The average annual treatment cost for first-line biologic treatments for plaque psoriasis in the US more than doubled from 2007 to 2021. Costs in 2021 would have been 44% lower if patients had initiated the lowest-cost medication within each class, highlighting the potential savings from using lower-cost psoriasis therapies and better aligning prices with a drug’s clinical benefits.
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Predictors of Withdrawal of Anticancer Drug Indications Granted Accelerated Approval: a Retrospective Cohort Study

Tibau Martorell A, Cliff ERS, Romano A, Borrell M, Molto C, Kesselheim AS - eClinicalMedicine

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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Among 167 cancer indications granted FDA accelerated approval from 1992 to 2022, 19% were withdrawn and 61% were converted to regular approval, with drugs demonstrating low clinical benefit as measured using the ESMO-MCBS scale more likely to be withdrawn. Drugs with breakthrough therapy designations or genome-targeted mechanisms had lower withdrawal rates. These findings underscore the value of benefit assessment frameworks to inform regulatory and clinical decision-making.
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Changes in Oncology Medication Use After Withdrawal of Accelerated Approval

Hwang CS, Kesselheim AS, Kelkar AH, Cliff ERS, Rome BN - JAMA Oncology

  • Research Publications
| June 2025
  • Regulation and Clinical Evidence
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For four oncology drugs granted accelerated approval, the release of negative confirmatory trial results prompted a decrease in use, and few patients were initiating treatment by the time of the drug’s indication was withdrawn. However, the time between trial results publication and regulatory withdrawal should be shortened to minimize patient exposure to ineffective treatments.
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Association Between Industry Payments and Prescription of Inhaled Medications

Nadeem H, Donovan LM, Feemster LC, Au DH, Feldman WB, Duan KI - American Journal of Respiratory and Critical Care Medicine

  • Research Publications
| June 2025
  • Price, Value, and Access
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Inhaler prescribers who received payments from inhaler manufacturers in 2018 were found to be more likely to prescribe inhalers from the same manufacturer in 2019, with larger payments associated with increased prescribing.
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Op-Ed: The Trouble with ‘Do Your Own Research’ for Drugs

Avorn J - MedPage Today

  • Commentary & Opinion
| May 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The author critiques the notion that individual patients or physicians can independently evaluate drug efficacy, highlighting the complexity of clinical trial data and a lack of public access to the full evidence manufacturers submit for regulatory review. Bypassing FDA oversight risks exposing patients to ineffective or harmful treatments, while raising questions about who would be expected to pay for such therapies.
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Brief of 30 Scholars of Law, Economics, and Medicine in Hikma v. Amarin (Cert Petition)

Feldman WB, Kesselheim AS, Rome BN, Tu SS - US Supreme Court

  • Policy Interactions
| May 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Filed in support of Hikma’s cert petition, the amici claim that the Federal Circuit erred in holding that a generic manufacturer could face induced infringement liability based on truthful product equivalence statements, contending that this decision conflicts with the common law foundations of inducement doctrine and threatens to extend patent liability indefinitely through successive new-use patents. The ruling’s reach extends well beyond pharmaceuticals to any industry relying on equivalence or compatibility claims, and impeding such statements would stifle free-market competition and harm consumers by sowing confusion about equivalent products.
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The Comparative Effectiveness and Safety of Fluticasone-Salmeterol Via Metered-Dose Versus Dry Powder Inhalers for COPD: A New User Cohort Study

Demkowicz BJ, Rader K, Wang SV, Kesselheim AS, Feldman WB - PLOS Medicine

  • Research Publications
| May 2025
  • Regulation and Clinical Evidence
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The authors compared the effectiveness and safety of fluticasone-salmeterol delivered via metered-dose versus dry powder inhalers among patients with COPD and found similar outcomes in the two treatment groups. Given that metered-dose inhalers are associated with 20x the greenhosue gas emissions of dry powder versions, these findings highlight the value of efforts to transition to dry powder inhalers.
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Testimony: Improving Residency Selection and Addressing Threats to Science and the US Health Care System

Feldman WB - 119th Congress, House Committee on the Judiciary, Subcommittee on the Administrative State, Regulatory Reform, and Antitrust

  • Policy Interactions
| May 2025
  • Innovation Incentives and Competition
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Feldman argues that eliminating the residency match’s antitrust exemption would not solve physician shortages or low resident wages, and instead recommends increasing CMS funding for residency slots, streamlining licensing for international medical graduates, and supporting resident unionization. More broadly, he discusses threats to US medicine from the Trump administration’s proposed 37% NIH cut and 50% NSF cut, FDA layoffs that have already delayed drug reviews, and Medicaid cuts that would leave over 8 million uninsured. Watch his testimony.
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PORTAL Post | May 2025

  • PORTAL Post
| May 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
A graphic icon of a megaphone as a symbol for the PORTAL Post email newsletter.
The May 2025 edition of the PORTAL Post monthly newsletter.
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Costs of Extending the Small Molecule Exemption Period in Medicare Drug Price Negotiation

Cai CL, Kesselheim AS, Rome BN - Health Affairs Forefront

  • Research Publications
| May 2025
  • Price, Value, and Access
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The authors evaluate the impact of extending the waiting period for Medicare drug price negotiation from 9 to 13 years for small molecule drugs, finding that delaying negotiation eligibility could increase Medicare spending by billions of dollars annually, undermining cost-saving goals and delaying access to lower prices for key medications.
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Drug Versioning and Legal Accountability for Preventable Product Harms

Tu SS, Bonis T - JAMA

  • Commentary & Opinion
| May 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
The authors highlight a new form of “product hopping” called “drug versioning,” where pharmaceutical companies delay releasing improved drug versions to maximize profits on existing ones, using the alleged postponed launch of tenofovir alafenamide fumarate for HIV as a case study. This legal challenge highlights debates over corporate responsibility, and holding companies accountable for such behavior could deter unethical practices and ensure timely access to safer medications.
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Strategies to Advance Drug Repurposing for Rare Diseases

Hong D, Kesselheim AS - JAMA Network Open

  • Commentary & Opinion
| May 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
Talk bubble graphics representing commentary and opinion.
The authors comment on a discussion of the role of rare disease nonprofit organizations in drug repurposing. Policy reforms—such as increased public funding for repurposing studies, expanded FDA authority to update drug labels based on nonprofit-submitted data, and better use of real-world evidence—are essential to ensure that repurposed rare disease therapies are effective and affordable.
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Brief of Economists and Scholars of Health Policy in Teva v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US District Court for the District of Columbia

  • Policy Interactions
| May 2025
  • Price, Value, and Access
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The amici express support the Medicare drug price negotiation program and specifically defend CMS’s practice of aggregating products sharing the same active ingredient (including Austedo and Austedo XR) as a statutorily mandated response to “product hopping.” They argue that Teva’s proposed interpretation would create a systemic loophole enabling manufacturers to endlessly reset the negotiation clock through minor reformulation. They also reiterate that the negotiation program restores bargaining equity in Medicare, mirrors other federal drug pricing mechanisms, and poses minimal risk to genuine innovation given that targeted drugs have already generated billions in surplus revenue.
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Sotorasib’s Accelerated Approval: Wrong Dose and Indication

Ratain MJ, Kesselheim AS - JAMA Oncology

  • Commentary & Opinion
| May 2025
  • Regulation and Clinical Evidence
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The atuhors critique the FDA’s accelerated approval of sotorasib for non-small cell lung cancer, highlighting issues with the approved dose and indication. Confirmatory trials revealed no clear benefit of the higher dose over a lower dose, and reforms should be made to ensure drug labels reflect accurate dosing information. CMS should also consider covering lower doses if clinical trials show them to be equally effective.
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Opioid Settlements in Canada: Avoiding U.S. Pitfalls, Embracing Best Practices

Eisenkraft Klein D, Minhee C - International Journal on Drug Policy

  • Commentary & Opinion
| May 2025
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
Drawing on the early US opioid settlement experience, the authors argue that Canada’s opioid settlement funds risk dilution without binding safeguards. They recommend mandating dedicated remediation spending, public reporting with outcome metrics, investment in a broad range of harm‑reduction services, and community‑led allocation to ensure settlements meaningfully reduce overdose harms rather than replicate US missteps.
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Certification Marks for Supporting Generic Drug Quality

Lindgren L, Kesselheim AS, Tu SS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Spring 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Certification marks, similar to USDA Organic or Energy Star labels, have the potential to improve the perception and oversight of generic drug quality. Independent certification marks could be a cost-effective way to effectively communicate drug quality information to purchasers, incentivize manufacturers to exceed baseline quality standards, and improve consumer confidence in the generic drug supply.
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Brief of Economists and Scholars of Health Policy in National Infusion Center Association v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US District Court for the Western District of Texas

  • Policy Interactions
| April 2025
  • Price, Value, and Access
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The amici express support for the Medicare drug price negotiation program and rebut assertions that the program will harm biopharmaceutical innovation. They argue that patents, exclusivity periods, and insurance coverage distort the drug market far from free-market conditions, that continued strong R&D investment and oncology trial activity since the Act’s passage undermine industry claims of innovation harm, and that the program’s structure mirrors fair negotiation processes long used by other federal purchasers.
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Op-Ed: It’s Not Hard to See Through RFK Jr.’s ‘Radical Transparency’ Pledge

Lurie P, Kesselheim AS, Sharfstein JM - Washington Post

  • Commentary & Opinion
| April 2025
  • Regulation and Clinical Evidence
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The authors discuss Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr.’s claim that he would bring “radical transparency” to the Department, and how recent actions at the FDA, NIH, CDC, and other HHS agencies—including major staff reductions, communications restrictions, limiting public comment opportunities, and disbanding advisory committees—suggest a much more agenda-driven reality.
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Testimony: NIH Funding, Drug Price Negotiation, and Biosimilars: Three Factors Essential for Patients and Pharmaceutical Innovation

Kesselheim AS - 119th Congress, House Committee on Ways and Means, Subcommittee on Health

  • Policy Interactions
| April 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Kesselheim identifies three pillars essential to pharmaceutical innovation and patient access: restoring NIH research funding paused by the Trump administration, preserving and extending Medicare drug price negotiation, and fostering timely biosimilar competition for high-cost biologics. He rebuts constitutional and innovation-based challenges to the Inflation Reduct Act, noting that the first 10 negotiated drugs saw 38-79% price reductions while still generating substantial manufacturer revenue. Watch his testimony.
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Patent Thickets and Product Hops: Challenges and Opportunities for Legislative Reform

Feldman WB - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Spring 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The author provides an overview of how brand-name drug manufacturers use patent thickets and product hops to delay generic competition, and discusses policy reforms that may mitigate these tactics and improve competition.
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Engaging Pharmacists in Strategies for Navigating Prescription Drug Costs (Reply)

Lalani HS, Hwang CS, Rome BN - JAMA

  • Commentary & Opinion
| April 2025
  • Price, Value, and Access
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The authors discuss the root causes of high drug prices and high out-of-pocket costs for patients and emphasize how patients and clinicians can use the seven strategies outlined in their prior research to navigate high prescription drug costs.
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Therapeutic Benefit of Top-Selling Oncology Drugs in Medicare

Wang S, Egilman AC, Hahn G, Kesselheim AS - JAMA Network Open

  • Research Publications
| April 2025
  • Price, Value, and Access
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Of the 50 top-selling oncology drugs in Medicare in 2022, four-fifths were determined by French and German health technology assessment (HTA) bodies to provide high added benefit over alternatives. However, drugs found to provide low or no added benefit still accounted for $6.7 billion in post-rebate Medicare spending. These findings emphasize the role of value assessment in Medicare drug price negotiations and underscore the need for a national US HTA agency to facilitate cost-effective health care spending.
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PORTAL Post | April 2025

  • PORTAL Post
| April 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The April 2025 edition of the PORTAL Post monthly newsletter.
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Will I Feel Better? Raising the Bar for Quality of Life in Oncology

Tibau Martorell A, Romano A, Kesselheim AS - Nature Reviews Clinical Oncology

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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Applying the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) quality-of-life (QoL) checklist to oncology indications approved by the FDA and EMA between 2020 and 2023, nearly a quarter of drugs showed some QoL improvement, but only 13% met checklist requirements, highlighting gaps in QoL outcomes in pivotal trials.
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Glucose-Lowering Medications and Risk of Chronic Obstructive Pulmonary Disease Exacerbations in Patients with Type 2 Diabetes

Ray A, Paik JM, Wexler DJ, Sreedhara SK, Bykov K, Feldman WB, Patorno E - JAMA Internal Medicine

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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In a comparative effectiveness analysis of diabetes medications among nearly 400,000 patients with COPD, GLP-1 receptor agonists and SGLT-2 inhibitors were both associated with lower risks of moderate or severe COPD exacerbations compared to DPP-4 inhibitors.
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Frequency of First Generic Drugs Approved Through “Skinny Labeling,” 2021 to 2023

Ziaks TJ, Akanegbu CM, Egilman AC, Kesselheim AS - Journal of Managed Care & Specialty Pharmacy

  • Research Publications
| April 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The proportion of first generic drugs approved through “skinny labeling” decreased annually from 56% in 2021 to 20% in 2023, potentially due to increased legal risks following the 2021 decision in GSK v. Teva. Higher-selling brand-name drugs more frequently faced competition from skinny label generics, emphasizing the pathway’s importance in promoting cost-reducing generic competition for patients and payors.
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Comparative Bleeding Risk in Older Patients with HIV and Atrial Fibrillation Receiving Oral Anticoagulants

Quinlan CM, Avorn J, Kesselheim AS, Singer DE, Zhang Y, Cervone A, Lin KJ - JAMA Internal Medicine

  • Research Publications
| April 2025
  • Regulation and Clinical Evidence
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Among older adults with HIV and atrial fibrillation, warfarin and rivaroxaban were associated with higher rates of major bleeding versus apixaban, particularly among patients treated with antiretroviral therapy. These findings suggest apixaban may have a more favorable safety profile for this high-risk population.
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Factors in Time to Full Approval or Withdrawal for Anticancer Medicines Granted Accelerated Approval By the FDA

Tibau Martorell A, Hwang TJ, Romano A, Borrell M, Gich I, Molto C, Kesselheim AS - JAMA Network Open

  • Research Publications
| March 2025
  • Regulation and Clinical Evidence
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Priority review designation, absence of boxed warnings, initiation of confirmatory trials before approval, and ratings of intermediate or high clinical benefit were associated with shorter conversion times from FDA accelerated approval to full approval among 102 cancer drug indications. These findings emphasize the importance of rigorous efficacy and safety evaluations and timely trial initiation to support the benefits of accelerated approval drugs.
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Brief of 12 Narcolepsy Patients, Public Interest Organizations, Medical Professionals, and Professors of Law and Medicine in Jazz v. Avadel

Kesselheim AS, Tu SS - US Court of Appeals for the District of Columbia

  • Policy Interactions
| March 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The amici argue that Avadel’s once-nightly sodium oxybate product Lumryz offers substantial real-world benefits over Jazz’s twice-nightly Xyrem and Xywav products. Additionally, they contend that the Orphan Drug Act’s “same drug” phrase should be interpreted, consistent with FDA’s clinical superiority exception, to exclude clinically superior products from an incumbent’s exclusivity, because overly broad exclusivity scope harms competition and ultimately deprives patients of innovative treatments.
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Trends in Fills, Spending, and Prices of Doxepin for Insomnia

Cai CL, Kesselheim AS, Rome BN - JAMA

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Doxepin was originally developed in the 1960s as an antidepressant and has been off patent for decades, but low doses of doxepin have been found to be effective as a first line treatment for insomnia. The authors detail how this repurposing has sustained high prices despite the availability of generics. Liquid doxepin should be considered as a potentially cheaper alternative, alongside broader reforms to address market failures in generic drug repurposing.
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Active Postmarketing Device Surveillance as a Legislative Priority

Feldman WB, Kesselheim AS - BMJ

  • Commentary & Opinion
| March 2025
  • Regulation and Clinical Evidence
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Passive safety surveillance of medical devices, relying on stakeholder reporting of problems, is fraught with limitations, whereas active safety surveillance, in which the FDA conducts routine post-marketing studies using real-world evidence, can provide a more complete picture of potential safety concerns. The authors make a case for better tracking of devices in medical claims and more funding for the FDA to conduct postmarketing surveillance studies.
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Comments on Draft Guidance, “Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial Is Underway”

Tibau Martorell A, Martin MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| March 2025
  • Regulation and Clinical Evidence
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The commenters support the FDA’s efforts to prevent delays in confirmatory trials for drugs granted accelerated approval and recommend that the agency require expedited timelines for drugs with serious safety signals, strengthen transparency around trial progress, and apply lessons from research showing that boxed warnings and early trial initiation are key factors in timely conversion to full approval.
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PORTAL Post | March 2025

  • PORTAL Post
| March 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The March 2025 edition of the PORTAL Post monthly newsletter.
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Real-World Evidence Is a Vital Tool for Informing Treatment Strategies in Chronic Obstructive Pulmonary Disease

Feldman WB, Wang SV, Kesselheim AS - BMJ

  • Commentary & Opinion
| March 2025
  • Regulation and Clinical Evidence
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Real-world evidence, including longitudinal healthcare database studies, can provide valuable insights on clinical questions in COPD, including through the investigation of intraclass differences between COPD therapies and other subtle clinical differences that may impact routine clinical care.
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The Puzzle of Biologics Manufacturing Platform Patents

Jefferson OA, Price WN, Tu SS, Vishnubhakat S, Rai AK - Nature Biotechnology

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
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Biologic originator compaies strategically use manufacturing platform patents to delay or block biosimilar competition. Many such patents are filed long after FDA approval and may be invalid or unused, yet are still widely asserted in litigation, often without scrutiny of prior use by the USPTO. Improved FDA-USPTO coordination and caps on post-approval patent assertions can promote fairer competition and transparency.
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Research Submission to Inform Medicare Drug Price Negotiation, IPAY 2027

Martin MJ, Kesselheim AS, Rome BN - Centers for Medicare and Medicaid Services (CMS)

  • Policy Interactions
| March 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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PORTAL submits summaries of peer-reviewed research to inform CMS’s evidence-based assessment of drugs selected for Medicare price negotiation in Initial Price Applicability Year (IPAY) 2027, including Trelegy Ellipta, Breo Ellipta, Ozempic/Rybelsus/Wegovy, Pomalyst, Xtandi, and Calquence.
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Primary Medication Adherence in Medicare Beneficiaries Prescribed Sacubitril-Valsartan or Renin-Angiotensin System Blockers for Heart Failure with Reduced Ejection Fraction

Hwang CS, Desai RJ, Kesselheim AS, Levin R, Rome BN - American Heart Journal

  • Research Publications
| March 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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No difference in medication adherence was observed among patients newly prescribed a costly brand-name heart failure medication, sacubitril-valsartan (Entresto), compared to those prescribed an inexpensive angiotensin-converting enzyme inhibitor (ACE-I) or angiotensin-II-receptor blocker (ARB). Adherence was high among patients switching from an ACE-I or ARB to sacubitril-valsartan, suggesting that this switch was not associated with interruptions in renin-angiotensin blockade.
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Out-Of-Pocket Savings Under the Inhaler Price Cap

Mein SA, Kludze AK, Feldman WB, Tale AP, Stevens JP, Wadhera RK - JAMA Internal Medicine

  • Research Publications
| March 2025
  • Price, Value, and Access
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Three of the four major inhaler manufacturers in the US announced voluntary $35 out-of-pocket price caps for inhalers in 2024. Using data from the Medical Expenditure Survey Panel approximately 40% of patients receiving an inhaler for COPD or asthma during the study period would have benefited from the price cap, with median annual out-of-pocket savings of $76.
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National Survey of Factors Associated with Physician Antibiotic Prescribing Preferences

Hong D, Kesselheim AS, Morlock R, Metlay JP, Powers JH, Feldman WB - Journal of General Internal Medicine

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Among a group of 130 surveyed physicians, the majority reported prioritizing an antiobiotic’s clinical outcomes, including treatment efficacy and risk of side effects, over economic or public health-related factors like patient out-of-pocket costs or antibiotic resistance risk when making prescribing decisions. Understanding this prioritization can aid the development of policy interventions to incentivize antibiotic development.
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First-In-Class Drugs Experienced Different Regulatory Treatment in the US and Europe

Han J, Kesselheim AS - Health Affairs

  • Research Publications
| March 2025
  • Regulation and Clinical Evidence
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In this review of FDA and EMA approval data for nearly 200 first-in-class drugs between 2013 and 2023, the FDA was found to offer significantly greater regulatory flexibility, with 50% of first-in-class drugs approved based on pivotal trials lacking clinical endpoints and 30% lacking blinding. The FDA also grants more expedited review designations than the EMA. These findings reveal the challenge for regulators in balancing accelerated access to novel drugs with thorough regulatory scrutiny.
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Changes in Biologic Drug Revenues After Administrative Patent Challenges

Raymakers AJN, Van de Wiele VL, Kesselheim AS, Tu SS - Health Affairs

  • Research Publications
| March 2025
  • Innovation Incentives and Competition
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Since 2012, 70-74% of biologic patents challenged via inter partes review (IPR) by biosimilar or alternative product manufacturers were invalidated, and these challenges were associated with reduced revenue for reference biologics. Policymakers should not restrict access to IPR, as it is a valuable pathway to facilitate biosimilar entry.
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An Empirical Investigation Into Concerns Over Quality-Adjusted Life-Years: A Review of Cost-Effectiveness Analyses in Oncology

Raymakers AJN, Rand LZG, Feldman WB, Kesselheim AS - Journal of Cancer Policy

  • Research Publications
| March 2025
  • Price, Value, and Access
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A common argument put forth by those who wish to restrict the use of quality-adjusted life-years (QALYs) is that cost-effectiveness analyses (CEAs) using QALYs identify sub-groups of patients for whom a new treatment is not considered cost-effective, thereby limiting access. In this analysis, the authors identified all US-based cancer CEAs and found no substantive evidence that this analytical framework was used in the way suggested. These findings are further evidence that bans on QALY use lack empirical justification.
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Using Trademarks to Ensure Completion of Postapproval Trials

Hwang CS, Tu SS - JAMA Health Forum

  • Commentary & Opinion
| February 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The FDA should use trademarks to ensure the timely completion of postapproval confirmatory trials for drugs granted accelerated approval . Manufacturer use of a drug’s trademarked brand or generic name should be restricted until trials confirm clinical benefit, instead requiring investigational names in promotional materials to communicate the drug’s clinical uncertainty.
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The Moral Injury of Inhaler Prescribing

Feldman WB, Furie GL - New England Journal of Medicine

  • Commentary & Opinion
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors discuss the dilemmas in treating patients with chronic lung disease, including the tension between the environmental impacts of existing metered-dose inhalers, limited access to dry powder inhalers, and the affordability challenges patients may face from the high cost of new, low-carbon alternatives. They call for structural reform to address this tension to ensure patients can access, effective, affordable, and environmentally stable therapies.
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Brief of Law Scholars in Novartis v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Third Circuit

  • Policy Interactions
| February 2025
  • Price, Value, and Access
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The amici argue that the Medicare drug price negotiation program does not effectuate an unconstitutional taking under the Fifth Amendment, because the federal government routinely negotiates prices as a market participant and because the pharmaceutical industry is precisely the kind of highly regulated, government-privileged sector in which price regulation has long been held constitutional. They further warn that accepting Novartis’s per se takings theory would destabilize the negotiated discount structures underlying Medicare, Medicaid, the 340B program, and the Veterans Health Administration, and could even invite takings challenges to obligations like EMTALA’s emergency care mandate.
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Brief of 14 Professors of Medicine and Law in Teva v. Amneal (Rehearing En Banc)

Feldman WB, Kesselheim AS, Tu SS, Avorn J, Rome BN - US Court of Appeals for the Federal Circuit

  • Policy Interactions
| February 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Filed in opposition to Teva’s petition for rehearing en banc, the amici argues that Teva’s predictions that the panel’s decision will dramatically shrink the Orange Book and unleash a flood of antitrust litigation, are unfounded. Device-only patents represent only about 5% of the roughly 10,000 patents listed in the Orange Book over the past 40 years, and prior challenges to improperly listed patents have been limited. The authors contend that the panel’s ruling aligns with the Hatch-Waxman Act’s goals and FDA’s own understanding of listable patents, and that delisting improperly listed device-only patents will benefit patients by removing barriers to lower-cost generic competition for important therapies like inhalers.
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Out of Pocket Getting Out of Hand—Reducing the Financial Toxicity of Rapidly Approved Drugs

Ubel PA, Grouls A, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Patients increasingly face financial toxicity from the high cost of drugs with uncertain clinical benefits. Various strategies can protect patients from financial harm while these drugs undergo further review, including enhancing drug labeling for accelerated approval products, establishing a drug price review board to recommend value-based prices, and capping out-of-pocket costs until post-approval studies are completed.
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Health Care Spending After Initiating Sacubitril-Valsartan vs Renin-Angiotensin System Blockers for Heart Failure Treatment

Hwang CS, Desai RJ, Kesselheim AS, Levin R, K Sreedhara S, Rome BN - JAMA Health Forum

  • Research Publications
| February 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Among Medicare patients initiating sacubitril-valsartan (Entresto) versus ACE-I/ARBs for heart failure with reduced ejection fraction, those initiating sacubitril-valsartan had similar total health care spending compared to those starting on ACE-I/ARBs, with sacubitril-valsartan’s high cost offset by lower inpatient and outpatient spending. However, initiating sacubitril-valsartan was associated with higher out-of-pocket costs, which may negatively impact patient adherence and access.
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Physician Experiences with and Perspectives on Clozapine Prescribing

Sarpatwari A, Lu Z, Russo M, Zakoul H, Lee SB, Toyserkani GA, Zhou EH, LaCivita C, Hyatt Hawkins Shaw K, Dal Pan GJ, Kesselheim AS - JAMA Network Open

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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Clozapine, an effective therapy for treatment-resistant schizophrenia, had a risk evaluation and mitigation strategy (REMS) program in place to manage the risk of agranulocytosis. In a survey of nearly 200 physicians, two-thirds viewed the REMS program positively, though some physicians called for more educational materials and raised concerns about delays in patient access due to required laboratory testing.
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Predicting Patent Challenges for Small-Molecule Drugs: A Cross-Sectional Study

Memedovich A, Steele B, Orr T, Chaudhry S, Tadrous M, Kesselheim AS, Hollis A, Beall RF - PLOS Medicine

  • Research Publications
| February 2025
  • Innovation Incentives and Competition
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The authors used supervised machine learning techniques to predict Paragraph IV patent challenges within a branded drug’s first year of eligibility, finding that drugs with large market sizes are more likely to face patent challenges in the first year, while anti-infective drugs and those with fast track designations were less likely to be challenged, demonstrating inconsistencies in generic entry across the prescription drug market.
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PORTAL Post | February 2025

  • PORTAL Post
| February 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
A graphic icon of a megaphone as a symbol for the PORTAL Post email newsletter.
The February 2025 edition of the PORTAL Post monthly newsletter.
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Regulatory Flexibilities Balancing Unmet Needs, Benefits and Risks in the Approvals of Imported Cancer Drugs in China: a Cohort Study From 2012 to 2021

Mao X, Xu J, Liu X, Kong S, Li Y, Bai X, Yang J, Kesselheim AS, Li G - Lancet Regional Health - Western Pacific

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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Among new oncology drugs approved in China between 2012 and 2021, most pivotal trials supporting approval were early phase, single-arm studies with response rate as the primary endpoint and few Chinese trial participants. This reliance on limited clinical evidence reveals a balance between addressing unmet medical needs and maintaining regulatory standards.
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Physician-Patient Communication About Novel Drugs and High-Risk Medical Devices

Dhruva SS, Kesselheim AS, Woloshin S, Ji RZ, Lu Z, Darrow JJ, Redberg RF - Medical Decision Making

  • Research Publications
| February 2025
  • Regulation and Clinical Evidence
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In a survey of 500+ physicians, half reported that they “usually” tell patients when a drug or device was approved based on a surrogate measure or when the FDA required a post-approval trial to further evaluate a product’s safety or effectiveness. Physician communication with patients about the quality of evidence supporting the new drugs and devices they prescribe is critical important.
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Government Funding for the Development of Enzalutamide

Gyawali B, Jung EH, Mooney H, Avorn J, Kesselheim AS - JAMA Oncology

  • Research Publications
| February 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The US government contributed at least $74 million to the development of prostate cancer drug enzalutamide (Xtandi), most of which was invested in the early research stages when there is often the greatest risk. Given the treatment’s high cost, the government should act to ensure US taxpayers can access Xtandi and other drugs with substantial government investment at a fair price.
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Financial Challenges of Being on Long-Term, High-Cost Medications

Elshiekh C, Rudà R, Cliff ERS, Gany F, Budhu JA - Neuro-Oncology Practice

  • Research Publications
| February 2025
  • Price, Value, and Access
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Long‑term use of high‑cost cancer therapies imposes substantial financial toxicity on patients, particularly in health systems without price negotiation or cost‑effectiveness review. Expanding health technology assessment, price negotiation, and patient access protections are essential as indefinite targeted therapies become more common.
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AI Devices in Neurology-Moving From Diagnosis to Prognosis

Hillis JM, Cliff ERS, Vokinger KN - JAMA Neurology

  • Commentary & Opinion
| February 2025
  • Regulation and Clinical Evidence
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The FDA’s first authorization of an AI prognostic device for dementia progression marks a shift toward lifecycle regulation with heavy reliance on post‑market evidence. The authors emphasize the need for rigorous enforcement of post‑approval studies and caution clinicians against over‑interpreting probabilistic predictions in clinical decision‑making.
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Op-Ed: How to Protect HHS, FDA, NIH, and Other Health Agencies From Political Interference

Bhaskar A, Kesselheim AS, Carpenter D - STAT

  • Commentary & Opinion
| January 2025
  • Regulation and Clinical Evidence
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Political interference in the FDA and other health agencies during the 2020 election and COVID-19 pandemic severely damaged public trust in public health institutions. Amid new risks to scientific independence, Congress should act to ensure that public health decisions at HHS, FDA, NIH, and CDC are guided by evidence rather than politics.
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A Regulatory Roadmap for Repurposing: Comparing Pathways for Making Repurposed Drugs Available in the EU, UK, and US

Scholte M, Bendicksen L, Grimm SE, Abu-Zahra T, Pauly B, Joore MA, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Research Publications
| Winter 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The authors outline existing pathways in the US, EU, and UK for repurposed drugs to receive regulatory approval for new uses. A repurposed drug’s status as a new drug, off-patent drug, or shelved drug (drugs abandoned during the research phase) alter the regulatory standards required to obtain approval for new uses, alongside other barriers academic or nonprofit institutions may face in trying to repurpose drugs.
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Comments on Draft Guidance, “Expedited Program for Serious Conditions-Accelerated Approval of Drugs and Biologics”

Tibau Martorell A, Martin MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| January 2025
  • Regulation and Clinical Evidence
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The commenters offer recommendations to strengthen the accelerated approval pathway, including reforms to address delays in confirmatory trial completion, overreliance on unvalidated surrogate measures, and extended timelines for withdrawal of indications when clinical benefit is not confirmed.
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Brief of Law Scholars in Boehringer Ingelheim v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Second Circuit

  • Policy Interactions
| January 2025
  • Price, Value, and Access
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The amici support affirmance of the district court decisions in the challenges to the Medicare drug price negotiation program brought by Boehringer Ingelheim. They argue that the government routinely negotiates contract prices for goods including drugs without implicating the Takings Clause, that Congress has broad authority to directly regulate prices in the heavily-subsidized and highly-regulated pharmaceutical industry, and that ruling for Boehringer would destabilize Medicare, Medicaid, and Veterans Administration drug programs that depend on negotiated discounts.
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Characteristics of Trials Preceding FDA Approval of Novel Psychiatric Drugs

Ahn-Horst RY, Turner EH, Kesselheim AS - JAMA Network Open

  • Research Publications
| January 2025
  • Regulation and Clinical Evidence
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Among 16 novel psychiatric drugs approved by the FDA between 2013 and 2024, most products targeted common mechanisms of action and were approved based on multiple positive phase 3 trials. However, three drugs were found to have been approved despite having fewer than half of the supporting trials being positive, underscoring inconsistencies in FDA approval standards that suggest the need for clearer regulatory guidelines for psychiatric drug approvals.
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The Role of Importation in Remediating U.S. Generic Drug Shortages

Bollyky TJ, Nagar SN, Searchinger C, Kesselheim AS - New England Journal of Medicine

  • Commentary & Opinion
| January 2025
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Regulatory delays and manufacturing challenges have contributed to persistent shortages of generic drugs, particularly injectable products, and the FDA should expedite temporary importation of drugs in shortage from well-regulated foreign markets to enhance supply chain resilience to mitigate drug shortages.
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Comments on Draft Guidance, “Expedited Program for Serious Condions-Accelerated Approval of Drugs and Biologics”

Ratain MJ, Kesselheim AS - Food and Drug Administration (FDA)

  • Policy Interactions
| January 2025
  • Regulation and Clinical Evidence
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The FDA should clarify how it will evaluate confirmatory trials conducted in populations different from the original accelerated approval population. The commenters also urge the agency to require confirmation of appropriate dosing as part of the accelerated approval process to ensure accurate benefit-risk assessments.
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PORTAL Post | January 2025

  • PORTAL Post
| January 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
A graphic icon of a megaphone as a symbol for the PORTAL Post email newsletter.
The January 2025 edition of the PORTAL Post monthly newsletter.
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Academic Detailing Interventions and Evidence-Based Prescribing: A Systematic Review

Rome BN, Dancel E, Chaitoff A, Trombetta D, Roy S, Fanikos P, Germain J, Avorn J - JAMA Network Open

  • Research Publications
| January 2025
  • Regulation and Clinical Evidence
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In this systemic review of 118 studies, academic detailing (interactive educational outreach to clinicians) was shown to increase evidence-based prescribing in a variety of clinical settings. Among 36 studies deemed to have the lowest risk of bias, 25 (69%) showed significant improvements in at least one prescribing behavior, and the median proportion of patients who used the targeted medications changing by 4.0% in the intended direction.
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Accelerating Biosimilar Market Access: The Case for Allowing Earlier Standing

Tu SS, Goode R, Turner M, Van de Wiele VL - Journal of Law and the Biosciences

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
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Biosimilar manufacturers should be permitted to initiate patent litigation at the start of Phase 3 clinical trials rather than after FDA approval. This reform could expedite market access, reduce costs, and align biosimilar patent processes with those of small-molecule generics.
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Leveraging Old Hepatitis C Therapies

Tu SS, Kottilil S, Mattingly II TJ - New England Journal of Medicine

  • Commentary & Opinion
| January 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The authors highlight strategies to improve access to high-cost hepatitis C treatments in the US, including leveraging older direct-acting antivirals (DAAs), acquiring patents for generic production, or negotiating better prices for current therapies.
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Use of Means-Plus-Function Claiming to Evade the Enablement Requirement

Bonis T, Tu SS - Texas Intellectual Property Law Journal

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
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The authors argue that “means-plus-function” type patent claims should not be allowed to bypass the enablement requirement.  Courts should simply apply the Amgen v. Sanofi standard to all claims, including means-plus-function claims, to prevent overly broad patents.
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Revising EU Pharmaceutical Legislation: Will It Foster Drug Repurposing?

Scholte M, Grimm SE, Pauly B, Verbeeck F, Pasmooij AMG, Bouma B, Van Duijn-Wiersma J, Guney E, Kesselheim AS, Schmidt HHHW, Joore MA - Drug Discovery Today

  • Commentary & Opinion
| January 2025
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Drug repurposing provisions included in EU pharmaceutical legislation have the potential to improve access to affordable treatments, but additional regulatory support, fair data protection periods, and improved awareness strategies are needed tomake repurposed drugs more accessible, affordable, and sustainable.
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Pediatric Exclusivity Revenues for Cancer Drugs

Sarpatwari A, Bendicksen L, Hawkins DS, Gore L, Bourgeois FT - JAMA Pediatrics

  • Research Publications
| January 2025
  • Innovation Incentives and Competition
  • Price, Value, and Access
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When comparing the revenues generated by 6-month pediatric exclusivities to the cost of clinical trials required to obtain these exclusivities for a set of four oncology drugs, the average manufacturer revenue generated by the added exclusivity ($309 million) exceeded the average investment cost required to conduct the underlying trials ($156 million).
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Comparative Effectiveness and Safety of Single Inhaler Triple Therapies for Chronic Obstructive Pulmonary Disease: New User Cohort Study

Feldman WB, Suissa S, Kesselheim AS, Avorn J, Russo M, Schneeweiss SG, Wang SV - BMJ

  • Research Publications
| December 2024
  • Regulation and Clinical Evidence
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Two single-inhaler triple therapies for chronic obstructive pulmonary disease (COPD) are available in the US: Breztri Aerosphere, a twice daily metered-dose inhaler, and Trelegy Ellipta, a once daily dry powder inhaler. Breztri Aerosphere was associated with a 9% higher risk of moderate or severe COPD exacerbations and an identical risk of pneumonia hospitalizations compared to Trelegy Ellipta among patients with COPD treated in routine clinical practice.
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Differential Legal Protections for Biologics vs Small-Molecule Drugs in the US

Wouters OJ, Vogel M, Feldman WB, Beall RF, Kesselheim AS, Tu SS - JAMA

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors explore whether there is empirical justification for the greater legal protections given to biologics over small-molecule drugs in the US. Across development times, clinical trial success rates, research and development costs, patent protection, market exclusivity periods, revenues, and treatment costs, no no evidence supporting differential treatment was found, suggesting that US law overly rewards the development of biologics relative to small-molecule drugs.
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Op-Ed: Lessons from Humira on How to Tackle Unjust Extensions of Drug Monopolies with Policy

Bonis T, Tu SS - BioSpace

  • Commentary & Opinion
| December 2024
  • Innovation Incentives and Competition
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Using Humira as a case study, the authors highlight how drugmakers extend monopolies by building patent thickets of obvious, low‑value patents with terminal disclaimers that delay generic and biosimilar competition and keep prices high. Congress should curb these practices by limiting enforcement of patents tied by terminal disclaimers, promoting competition without undermining genuine pharmaceutical innovation.
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Characteristics of Academic Inventors on Government-Linked US Drug Patents

Martin MJ, Gabriele SME, Kesselheim AS, Tu SS - JAMA

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
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Among academic inventors listed on small molecule drug patents tied to US federal funding, most were senior researchers at major research institutions, many of whom had robust grant portfolios and prior industry experience. By contrast, early- and mid-career researchers were less represented, presenting an opportunity for NIH to expand its role in technology transfer by offering patent-related training and financing to these investigators.
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Use of Public Research and Manufacturing Enterprises to Lower Prescription Drug Prices and Increase Innovation

Moss A, Brown D, Tu SS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Winter 2024
  • Price, Value, and Access
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The authors discuss the opportunities and challenges associated with establishing public research and manufacturing enterprises (PRME) to expand the government’s role in the development, manufacturing, and distribution of new drugs. Moving beyond investments in early-stage research could improve access to and affordability of novel products while maximizing public welfare, though the financial and political capital required to launch such a venture could be substantial.
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Will the New Administration’s FDA Be a Threat or an Opportunity for the Public’s Health?

Kesselheim AS, Sharfstein JM - Health Affairs Forefront

  • Commentary & Opinion
| December 2024
  • Regulation and Clinical Evidence
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The authors outline the threat some proposed changes to the FDA pose to public health while outlining alternative reforms that would benefit the public interest by decreasing industry influence at the FDA, improving transparency, addressing prescription drug affordability, and strengthening the agency’s scientific integrity.
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Use, Spending, and Prices of Adalimumab Following Biosimilar Competition

Rome BN, Bhaskar A, Kesselheim AS - JAMA Health Forum

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Following the introduction of the first adalimumab (Humira) biosimilar to the US market, biosimilars made up 40%, the biologic’s list price increased by 8%. This indicates that Humira’s manufacturer likely offered sizeable rebates to health plans and PBMs to retain formulary placement.
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Prescription Drug Price Measures to Inform State Upper Payment Limits

Martin MJ, Mooney H, Kesselheim AS, Rome BN - National Academy for State Health Policy (NASHP)

  • Policy Interactions
| December 2024
  • Price, Value, and Access
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This memo provides an overview of the many prescription drug price measures that state PDABs may encounter when setting upper payment limits, organized by supply chain transactions, reimbursement rates, and purchaser-specific prices. It also compares these measures across federal programs and presents a price comparison for nine drugs selected in the first round of Medicare price negotiation.
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Brief of Law Scholars in Novo Nordisk v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Third Circuit

  • Policy Interactions
| December 2024
  • Price, Value, and Access
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The amici argue that the Medicare drug price negotiation program is constitutional because the federal government has long-settled authority to negotiate prices for goods it purchases and to regulate prices within industries, particularly those (like pharmaceuticals) that benefit from extensive government-granted privileges such as patents, regulatory exclusivities, and research subsidies. They warn that accepting Novo Nordisk’s Fifth Amendment takings theory would destabilize core federal health programs, including Medicaid, the 340B program, the Veterans Health Administration, and EMTALA, all of which rely on similar statutory pricing and discount mechanisms to function.
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Clearing Dense Drug-Patent Thickets

Chao B, Whalen R, Kesselheim AS, Tu SS - New England Journal of Medicine

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
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Two proposed patent reforms, the USPTO terminal disclaimer rule and a bipartisan bill to address patent thickets, could significantly reduce patent thickets on biologic and small molecule products. For two products, adalimumab (Humira), and lenalidomide (Revlimid), the USPTO rule would reduce each product’s thicket by 43% and 70%, respectively, while the legislationw would making only 25 of 105 Humira patents and 12 of 30 Revlimid patents enforceable.
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The Long CON: an Empirical Analysis of Pharmaceutical Patent Thickets

Tu SS - University of Pittsburgh Law Review

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
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“Secondary” patents that do not cover a drug’s active ingredient can contribute to patent thickets that can delay or deter competition. These patents often use a continuation application (CON) that allows brand manufacturers to obtain new patents based on a previous patent without disclosing any new information, with firms sometimes filing CONs more than five years after the original patent (“long CONs”).The pharmaceutical industry disproportionately files and litigates long CON patents relative to other industries, with long CONs representing 33% of all small molecule drug patents and 36% of all litigated drug patents between 2000 and 2022.
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PORTAL Post | December 2024

  • PORTAL Post
| December 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The December 2024 edition of the PORTAL Post monthly newsletter.
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Multiple Myeloma Clinical Trials Exclude Patients with the Highest-Risk Disease: A Systematic Review of Trial Exclusion Criteria

Zhukovsky S, White J, Chakraborty R, Costa LJ, Van Oekelen O, Sborov DW, Cliff ERS, Mohyuddin GR - Leukemia & Lymphoma

  • Research Publications
| December 2024
  • Regulation and Clinical Evidence
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Reviewing 80 randomized multiple myeloma trials, the authors find persistent exclusion of patients with high‑risk disease features, limiting both access and generalizability of evidence. They conclude that eligibility criteria have not meaningfully improved over time and call for deliberate inclusion of high‑risk populations in trial design.
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Impact of Priority Review Voucher Eligibility on Research and Development of Medical Countermeasures

Wang S, Hahn G, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Following the creation of the priority review voucher (PRV) program for medical countermeasures, no statistically significant increase in the rate of new medical countermeasure entry into clinical trials was observed. The sunset of PRVs for medical countermeasures in 202 paves the way for more effective policies to incentivize drug research and development.
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Clinical Benefit and Revenues of Drugs Affected By Rare Pediatric Disease Priority Review Vouchers, 2017-2023

Liu ITT, Kesselheim AS - Journal of Pediatrics

  • Research Publications
| December 2024
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Between 2017 and 2023, drugs for rare pediatric diseases generated similar revenues to other brand-name drugs, suggesting that these the FDA’s pediatric rare disease priority review voucher (PRV) program may not be necessary to incentivize the development of treatments for pediatric rare diseases.
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Association Between Changes in Prices and Out‐of‐Pocket Costs for Brand‐Name Clinician‐Administered Drugs

Lalani HS, Russo M, Desai RJ, Kesselheim AS, Rome BN - Health Services Research

  • Research Publications
| December 2024
  • Price, Value, and Access
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For 52 clinician-administered drugs, no association was found between annual list price changes and changes in patients’ out-of-pocket costs between 2009 and 2018, though both increased during the study period. These findings may be explained by payer reimbursements that exceed drug list prices and complex insurance benefit designs.
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A Comparative Analysis of International Drug Price Negotiation Frameworks: an Interview Study of Key Stakeholders

Syversen ID, Schulman K, Kesselheim AS, Feldman WB - Milbank Quarterly

  • Research Publications
| December 2024
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The authors compare the processes that health systems in seven other countries (Belgium, Canada, France, Germany, Netherlands, Norway, UK) and the US Department of Veterans Affairs use to negotiate brand-name prescription drug prices. Health systems differ across several axes, including how added health benefit is assessed, whether explicit willingness-to-pay thresholds are employed, and how specific approaches for priority disease areas are taken, but they largely coalesce around a set of practices that are absent from Medicare drug price negotiation.
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Azithromycin to Reduce Mortality

Goh M, Kesselheim AS, Outterson K - New England Journal of Medicine

  • Commentary & Opinion
| November 2024
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The authors emphasize the lifesaving benefits of azithromycin demonstrated in the AVENIR trial, which showed a 14% reduction in child mortality in Niger. Concerns about antimicrobial resistance should not hinder access to this critical intervention, and stewardship programs should instead be implemented to monitor resistance without creating barriers to drug availability.
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Strategies to Help Patients Navigate High Prescription Drug Costs

Lalani HS, Hwang CS, Kesselheim AS, Rome BN - JAMA

  • Research Publications
| November 2024
  • Price, Value, and Access
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The authors provide an overview of the tools and resources health care providers can use to help patients afford their medications depending on insurance type, including the use of patient assistance programs, copay cards, and direct-to-consumer pharmacies.
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Medicare’s First Round of Drug-Price Negotiation—Measuring Success

Rome BN, Kesselheim AS, Feldman WB - New England Journal of Medicine

  • Commentary & Opinion
| November 2024
  • Price, Value, and Access
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The authors reflect on the results of the first round of Medicare drug price negotiation, measuring the success based on the potential savings to the federal government, how the negotiated prices compare to prices in other countries, and how the program will lead to lower out-of-pocket costs for patients with Medicare prescription drug coverage.
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How Agencies Should Decide Which Costly Drugs to Target with Government March-In Rights

Ravinthiran J, Kesselheim AS, Knievel S - Health Affairs Forefront

  • Commentary & Opinion
| November 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Federal agencies should consider seven key factors when deciding whether to use march-in rights under the Bayh-Dole Act to address the high cost of prescription drugs with ties to federal funding, including product revenues, excessive prices, remaining non-patent exclusivities, and a product’s broader patent portfolio.
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US Food and Drug Administration’s Directive to Deal with Delayed Confirmatory Trials: Lessons From Pralatrexate and Belinostat for T-Cell Lymphoma

Cliff ERS, Russler-Germain DA, Daval CJR, Kesselheim AS - Journal of Clinical Oncology

  • Research Publications
| November 2024
  • Regulation and Clinical Evidence
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Prolonged delays in confirmatory trials under the FDA’s accelerated approval program expose patients to costly therapies with unproven clinical benefit for many years. FDA should require confirmatory trials to be underway at approval, optimize trial designs to speed up evidence generation, and use stronger incentives and penalties to prevent these “dangling” accelerated approvals.
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Does Policy Uncertainty Boost Vaccine Hesitancy? Political Controversy, the FDA and COVID-19 Vaccine Hesitancy in Fall 2020

Carpenter D, Dardet ME, Bhaskar A, Rand LZG, Feldman WB, Kesselheim AS - Journal of Health Politics, Policy and Law

  • Research Publications
| November 2024
  • Regulation and Clinical Evidence
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Policy uncertainty during the evaluation and rollout of COVID-19 vaccines in the fall of 2020 was associated with a spike in vaccine hesitancy, with the greatest increase among Democrats, Independents, Asian Americans, and Black Americans. Vaccine hesitancy after this spike did not decline uniformly, with Black Americans and college-educated individuals having more persistent hesitancy. These findings highlight the influence political instability may have on public confidence in vaccine safety and trust in federal health agencies.
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Testing of New Drugs Approved From 2015 Through 2021 Under the US Pediatric Research Equity Act

Liu ITT, Hwang TJ, Kesselheim AS - JAMA

  • Research Publications
| November 2024
  • Regulation and Clinical Evidence
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The Pediatric Research Equity Act (PREA), which added pediatric testing requirements for new drugs indicated for adults, applied to only 30% of new drugs approved by the FDA between 2015 and 2021, in part due to exemptions for drugs indicated for rare diseases. Furthermore, more than half of post-marketing pediatric trials were incomplete five years after approval.
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Shortages of Essential Generic Drugs with Limited Competition

Martin MJ, Rome BN, Kesselheim AS, Lalani HS - Journal of General Internal Medicine

  • Research Publications
| November 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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20% of the 117 drugs in shortage in the US as of November 2023 were generic drugs considered essential medicines by the World Health Organization that were nonetheless produced by a small number of manufacturers (< 3). Policymakers and other stakeholders may want to prioritize these essential drugs facing limited competition when developing solutions to address US drug shortages.
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Estimating Costs in Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa (Reply)

Raymakers AJN, Kesselheim AS, Feldman WB - JAMA Dermatology

  • Commentary & Opinion
| November 2024
  • Price, Value, and Access
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The authors agree with comments that the high cost of beremagene geperavec (B-VEC) for dystrophic epidermolysis bullosa may be offset by reductions in other health care costs. However, these offsets are likely to vary by disease subtype. They also reinforce the value of conducting a QALY-based cost-effectiveness analysis of B-VEC to further inform reimbursement decisions.
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Estimated Savings Under the Medicare High-Value Drug List Model

Cai CL, Kesselheim AS, Rome BN - JAMA Internal Medicine

  • Research Publications
| November 2024
  • Price, Value, and Access
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Under the CMS High-Value Drug List Model, a voluntary model under which Medicare Part D plans cap patient out-of-pocket costs for 150 generic drugs at $2 per month, an estimated 4 in 10 Medicare beneficiaries would experience modest savings under this model, with an annual savings of $11 per person.
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Comparing Supplemental Indications for Cancer Drugs Approved in the US and EU

Stoelinga J, Bloem LT, Russo M, Kesselheim AS, Feldman WB - European Journal of Cancer

  • Research Publications
| November 2024
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Manufacturers obtained supplemental indications for cancer drugs at a substantially higher rate in the US than in the EU, despite drugs often starting with the same number of indications in both jurisdictions. Differences in regulatory processes and incentives, like accelerated approval and rare disease drug designations, may contribute to this variation.
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Patent Claim Scope and Biosimilar Competition in the US and EU

Bloomfield D, Kesselheim AS - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Fall 2024
  • Innovation Incentives and Competition
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In this commentary on an analysis of the patent thicket surrounding adalimumab (Humira), the authors discuss how the findings open interesting avenues for further research. Such analyses, they argue, reinforce the need for policy changes at the USPTO to improve patent quality so as to not hinder biosimilar market entry.
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Invited Commentary on Interventional Pharmacoepidemiology: Origins, Current Status, and Future Possibilities

Avorn J - American Journal of Epidemiology

  • Commentary & Opinion
| October 2024
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The author explores the historical development and future potential of interventional pharmacoepidemiology—using claims data and other patient information to understand and develop interventions to address suboptimal drug use. Use of such methods can improve the quality and cost-effectiveness of medication use.
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Health Technology Assessment in Prescription Drug Coverage and Pricing Decisions: Examples From Other Countries

Rand LZG, Raymakers AJN, Martin MJ, Egilman AC, Kesselheim AS, Rome BN - National Academy for State Health Policy (NASHP)

  • Policy Interactions
| October 2024
  • Price, Value, and Access
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This memo summarizes how five high-income countries—England, Canada, Australia, France, and Germany—use health technology assessment (HTA) to inform drug coverage and price negotiations, including each country’s use of QALYs or alternative outcome measures. It also provides guidance on accessing publicly available HTA and pricing information and includes price comparisons between the US and these countries for ten top-spending drugs.
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The Patent Trial and Appeals Board: A Target for Prescription Drug Patent Reform?

Freilich J, Kesselheim AS - JAMA

  • Commentary & Opinion
| October 2024
  • Innovation Incentives and Competition
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Efforts to reform the US Patent Trial and Appeals Board (PTAB) via the PREVAIL Act threatens to worsen drug patent thickets by impeding efforts to challenge patents as improperly granted. Instead, allowing patent challenges at the PTAB to trigger the 180-day generic exclusivity period and for successful challenges to end the 30-month stay on generic entry would be better solutions to strengthen the PTAB and promote generic competition.
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Confirmatory Trials of Accelerated Approval Drugs—Will Imposing Fines Reduce Delays?

Daval CJR, Kesselheim AS, Cliff ERS - New England Journal of Medicine

  • Commentary & Opinion
| October 2024
  • Regulation and Clinical Evidence
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The authors discuss the need for stronger FDA action to address delays in confirmatory trials of accelerated approval drugs. The agency already has the authority to fine sponsors for failure to complete trials, yet the FDA has chosen not to do so, and in the absence of other reforms to the accelerated approval proces fines could be a worthwhile tool for FDA to ensure timely evidence generation.
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Vaccine Messaging By the FDA: A National Randomized Survey Study

Feldman WB, Rand LZG, Carpenter D, Campbell EG, Darrow JJ, Kesselheim AS - Journal of General Internal Medicine

  • Research Publications
| October 2024
  • Regulation and Clinical Evidence
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In a national survey of public attitudes toward vaccination, respondents were more likely to get a well-tested vaccine with demonstrated safety and efficacy over a vaccine that had been quickly tested, irrespective of whether vaccine development was announced by the FDA or the President. This suggests that political interference may need to be more explicit before it alters public trust in the FDA’s vaccine approval process.
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Use of Omalizumab for Pediatric Asthma After US Food and Drug Administration Expanded Indications

Deshmukh AD, Kesselheim AS, Tsacogianis T, Rome BN - Pharmacoepidemiology and Drug Safety

  • Research Publications
| October 2024
  • Regulation and Clinical Evidence
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An FDA advisory committee’s recommendation against pediatric label expansion for omalizumab (Xolair), a biologic treatment for asthma, did not affect off-label pediatric use, while use increased when the label was evenually expanded in 2016 based on new clinical evidence. These findings emphasize the need for timely testing of drugs in pediatric populations to inform evidence-based prescribing.
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The RACE Act and Pediatric Trials of Adult Cancer Drugs

Liu ITT, Kesselheim AS - Pediatrics

  • Research Publications
| October 2024
  • Regulation and Clinical Evidence
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Expanded pediatric testing requirements for adult cancer drugs under the Research to Accelerate Cures and Equity (RACE) for Children Act. was shown to increase pediatric post-approval testing requirements, yet early pediatric trial rates appear unchanged. As more evidence emerges, policymakers may consider using the RACE for Children Act as a model to ensure the timely completion of pediatric studies.
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Seeking a Better Paper Trail of Federal Research Funding

Bendicksen L, Kesselheim AS, Feldman WB - JAMA

  • Commentary & Opinion
| October 2024
  • Innovation Incentives and Competition
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Stronger reporting and compliance mechanisms are needed to track federal investments in pharmaceutical research and development, as current disclosure of federal funding in drug patents and government databases is inadequate. Developing a more robust system to monitor the government’s pharmaceutical investments would facilitate efforts to quantify federal contributions to drug development—data that would be informative to both federal and state prescription drug programs.
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Sales Revenues for New Therapeutic Agents Approved by the United States Food and Drug Administration from 1995 to 2014

Wouters OJ, Kesselheim AS, Kuha J, Luyten J - Value in Health

  • Research Publications
| October 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Global revenues on 361 new FDA-approved drugs averaged $15 billion per product from 1995 to 2014, with the 25 best-selling products accounting for 38% of total sales. When including production, marketing, and other costs, 2 in 10 drugs recovered R&D and other costs within 5 years of FDA approval, and 1 in 2 within 10 years.
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Patient Assistance Programs-A New Era of Law and Policy

Daval CJR, Kesselheim AS - JAMA

  • Commentary & Opinion
| October 2024
  • Price, Value, and Access
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The authors summarize the legal and policy landscape surrounding the use of patient assistance programs. They discuss the effectiveness of the federal government’s authority under the Anti-Kickback Statute to scrutinize these programs in Medicare and the limitations of this approach in the private market. To address the back-and-forth between manufacturers and private payers, government intervention is necessary to ensure patients are not caught in the middle.
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Estimating New Eligibility and Maximum Costs of Expanded Medicare Coverage of Semaglutide for Cardiovascular Risk Prevention

Chaitoff A, Bendicksen L, Feldman WB, Zheutlin AR, Lalani HS - Annals of Internal Medicine

  • Research Publications
| October 2024
  • Price, Value, and Access
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Under the proposed expansion of Medicare coverage for semaglutide (Wegovy) for patients with elevated body mass index and established cardiovascular disease, an estimated 3.6 million Medicare beneficiaries would likely be newly eligible, with a maximum annual cost to Medicare of $34.4 billion. However, if “established cardiovascular disease” was broadly defined, 15.2 million beneficiaries could be eligible, adding up to $145 billion in annual costs.
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Price and Variability in Hospital Charges for Generic Indomethacin Suppositories

Feder NM, Hernandez I, Feldman WB, Murali M, Kesselheim AS, Elmunzer BJ, Gellad WF - JAMA Network Open

  • Research Publications
| September 2024
  • Price, Value, and Access
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New clinical uses for generic indomethacin suppositories in 2012 fueled a price increase by the drug’s manufacturer from $13 in 2008 to over $720 in 2023, with a median hospital gross charge price over $1800. Medicare patients may be particularly vulnerable to this price increase, as the drug is not covered under Medicare Part B or Part D, suggesting that policy action may be needed.
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Brief of Economists and Scholars of Health Policy in AstraZeneca v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US Court of Appeals for the Third Circuit

  • Policy Interactions
| September 2024
  • Price, Value, and Access
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The amici argue that the prescription drug market does not function as a free market because patents and exclusivity periods grant manufacturers temporary monopolies that allow them to set prices far above production costs, and that AstraZeneca’s claim that the Medicare drug price negotiation program caps prices below “actual market value” misrepresents how drug pricing actually works. The brief further contends that the programs mirrors fair negotiation processes already used by federal agencies like the VA and DoD, and that its safeguards—including eligibility limits and category exemptions—protect innovation while restoring bargaining equity for Medicare.
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Letter on S.2140, The 2024 Patent Eligibility Restoration Act (PERA)

Bock JW, Carrier MA, Contreras JL, Duan C, Ford RA, Ghosh S, Kesselheim AS, Lemley MA, Liebesman YJ, Malone P, Morten CJ, Sandeen SK, Sarnoff JD, Sinha MS, Strandburg KJ, Tu SS, Vertinsky L - 118th Congress, Senate Committee on the Judiciary

  • Policy Interactions
| September 2024
  • Innovation Incentives and Competition
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The commenters urge the Committee to oppose the Patent Eligibility Restoration Act (PERA), arguing it would overturn centuries of jurisprudence preventing patents on scientific discoveries, laws of nature, and abstract ideas that belong in the public domain.They contend PERA would not solve alleged uncertainty problems in patent eligibility doctrine and would instead generate wasteful litigation and harm scientific discovery and health care.
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Brief of Law Scholars in BMS v. Kennedy and Janssen v. Kennedy

Kesselheim AS, Sarpatwari A, Tu SS - US Court of Appeals for the Third Circuit

  • Policy Interactions
| September 2024
  • Price, Value, and Access
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The amici support affirmance of the district court decisions in the challenges to the Medicare drug price negotiation program brought by Bristol Myers Squibb and Janssen. They argue that that the government routinely negotiates contract prices for goods including drugs without implicating the Takings Clause, that Congress has broad authority to directly regulate prices in the heavily-subsidized and highly-regulated pharmaceutical industry, and that ruling for the manufacturers would destabilize Medicare, Medicaid, and Veterans Administration drug programs that depend on negotiated discounts.
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Brief of Economists and Scholars of Health Policy in BMS v. Kennedy and Janssen v. Kennedy

Frank RG, Scott Morton FM, Kesselheim AS, Anderson GF, Conti RM, Cutler DM, Hoadley J - US Court of Appeals for the Third Circuit

  • Policy Interactions
| September 2024
  • Price, Value, and Access
An icon of a bill representing government policy.
The amici argue that the prescription drug market does not function as a free market because patents and exclusivity periods grant manufacturers temporary monopolies that allow them to set prices far above production costs, forcing payors like Medicare to pay exorbitant prices for brand-name drugs without generic alternatives. Thus, they contend that the Medicare drug price negotiation program restores bargaining equity through a fair process that mirrors existing federal drug purchasing programs (such as those used by the VA and DoD), while safeguarding innovation by targeting only drugs that have already recouped their R&D costs many times over.
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Extent of Drug Patents with Terminal Disclaimers and Obviousness-Type Double Patenting Rejections

Tu SS, Kesselheim AS, Chao B - JAMA

  • Research Publications
| September 2024
  • Innovation Incentives and Competition
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The authors investigated the use of terminal disclaimers in drug patents, particularly focusing on the practice of filing secondary patents to extend market exclusivity. 45% of small molecule patents and 33% of biologic patents had obviousness-type double patenting rejections followed by terminal disclaimers, contributing to large patent thickets that can delay generic and biosimilar entry.
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Brief of 14 Professors of Medicine and Law in Teva v. Amneal

Feldman WB, Kesselheim AS, Tu SS, Avorn J, Rome BN - US Court of Appeals for the Federal Circuit

  • Policy Interactions
| September 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Teva’s improper listing of device-only patents in the Orange Book for ProAir HFA is part of a broader pattern of strategies, including dense patent thickets, post-approval patent filings, and “device hops”, that inhaler manufacturers have used for decades to delay generic competition and maintain high prices. The amici contend that because device patents extended market protection on inhalers by a median of 5.5 years and because drug-device combinations represent a large share of top-selling drugs, the Federal Circuit’s decision in Teva v. Amneal could have far-reaching implications for generic competition and patient access across many therapeutic classes.
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The Double-Edged Sword of Extremely High Prices for Gene Therapies in Sickle Cell Disease

Cliff ERS, Tessema FA - JAMA

  • Commentary & Opinion
| September 2024
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
Health equity considerations should factor into the pricing of multi-million-dollar gene therapies approved for use in sickle cell disease, as these therapies are a “double-edged sword”—while high prices incentivize therapeutic development for neglected diseases, these prices conversely limit access to treatments for the underserved populations they are intended to benefit. The authors suggest creating federally funded grants targeted at drug development for historically underinvested diseases.
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Trends in Use and Evidence of Adherence to Risk Evaluation and Mitigation Strategy Pregnancy Testing Requirements for Thalidomide, Lenalidomide, and Pomalidomide in the USA, 2000-2020

Mahesri M, Sarpatwari A, Huybrechts KF, Lii J, Lee SB, Toyserkani GA, LaCivita C, Zhou EH, Dal Pan GJ, Kesselheim AS, Bykov K - Drug Safety

  • Research Publications
| September 2024
  • Regulation and Clinical Evidence
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The authors assessed patient use of and adherence to three teratogenic drugs subject to FDA-imposed risk evaluation and mitigation strategies (REMS). Patients who could become pregnant and initiated lenalidomide, thalidomide, and pomalidomide compromised 3% of all patients initiating these drugs. There was no evidence of adherence to pregnancy testing requirements in most patients of childbearing potential, pointing to the need for further research on how often patients do not adhere to REMS pregnancy testing requirements.
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Regulating Laboratory Tests: What Framework Would Best Support Safety and Validity?

Horrow C, Kesselheim AS - Milbank Quarterly

  • Commentary & Opinion
| September 2024
  • Regulation and Clinical Evidence
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The authors discuss the need for a balanced regulatory framework for laboratory-developed tests (LDTs) that protects patients without stifling laboratory innovation. They also discuss the impact and rationale behind proposals like the VALID Act and the FDA’s LDT final rule.
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Patient Perspectives on Evidence Supporting Drug Safety and Effectiveness: “What Does It Mean for Me?”

Rand LZG, McGraw SA, Wang J, Woloshin S, Wang SV, Darrow JJ, Kesselheim AS - Journal of the American Geriatrics Society

  • Research Publications
| September 2024
  • Regulation and Clinical Evidence
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When new drugs are approved, there is often limited evidence from older patient populations, and data from routine clinical care could provide patient population-relevant information. Focus groups with patients 65 years and older who take an anticoagulant were conducted to understand patient perspectives on the evidence of safety and effectiveness from clinical trials or routine clinical use data for similar patients.
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Letter to the Editor Re: “Life Years Gained From the FDA Accelerated Approval Program in Oncology: A Portfolio Model”

Liu ITT, Kesselheim AS - Journal of the National Comprehensive Cancer Network

  • Commentary & Opinion
| September 2024
  • Regulation and Clinical Evidence
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The authors comment on an analysis quantifying the life years gained from the accelerated approval program in oncology. Instead of using 37 months after accelerated approval as the counterfactual date in the authors’ analysis, they recommend using a shorter period between 7 and 19 months to reflect prior research and more accurately assess the benefits of accelerated approval.
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Drug Repurposing Via the Best Pharmaceuticals for Children Act

Liu ITT, Kesselheim AS - JAMA Pediatrics

  • Commentary & Opinion
| September 2024
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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Policymakers should consider creating an NIH-sponsored federal program to repurpose off-patent drugs for new indications. An existing piece of legislation, section 409(i) of the Best Pharmaceuticals for Children Act, which is intended to run pediatric clinical trials for off-patent drugs, could act as a model for a federal drug repurposing program.
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Commercial Markups on Pediatric Oncology Drugs at 340B Pediatric Hospitals

Liu ITT, Wang J, Sarpatwari A, Kesselheim AS, Feldman WB - Pediatric Blood & Cancer

  • Research Publications
| September 2024
  • Price, Value, and Access
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Negotiated prices for oncology drugs among pediatric hospitals participating in the 340B program varied across and within hospitals, with median prices ranging between 102% and 630% of the drug’s average sales price.
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Availability and Cost of Expensive and Common Generic Prescription Drugs: A Cross-Sectional Analysis of Direct-To-Consumer Pharmacies

Lalani HS, Tessema FA, Kesselheim AS, Rome BN - Journal of General Internal Medicine

  • Research Publications
| September 2024
  • Price, Value, and Access
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Among five national direct-to-consumer pharmacies, many expensive generic drugs were not available at one or more of these pharmacies, while less expensive commonly-used generics were widely available. Prices varied but were frequently much lower than at traditional retail pharmacies. Patients might need to comparison shop to identify the lowest price for each drug, which could be difficult for many patients.
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Emulating Randomized Trials By Observational Database Studies: The RCT-DUPLICATE Initiative in COPD and Asthma

Suissa S, Schneeweiss SG, Feldman WB, Tesfaye H, Wang SV - American Journal of Epidemiology

  • Research Publications
| August 2024
  • Regulation and Clinical Evidence
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In five observational studies designed to emulate randomized clinical trials (RCTs) of asthma and COPD drugs, the authors were unable to align the study results with their respective RCTs due to the trial requirmeent to discontinue treatment before randomization, which could not be replicated observationally.This outcome highlights the challenge of trial emulation in asthma and COPD and suggests that regulators should take caution in comparing RCT and observational study results in these therapeutic areas until adequately addressed.
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The Origins of “Confidential Commercial Information” at the FDA

Daval CJR, Kesselheim AS - JAMA

  • Commentary & Opinion
| August 2024
  • Regulation and Clinical Evidence
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The authors discuss the FDA’s handling of “confidential commercial information” submitted by drug manufacturers during the drug approval process and outline steps the agency can take to allow greater flexibility to disclose information of public interest.
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Clinical Value of Guideline Recommended Molecular Targets and Genome Targeted Cancer Therapies: Cross Sectional Study

Tibau Martorell A, Hwang TJ, Avorn J, Kesselheim AS - BMJ

  • Research Publications
| August 2024
  • Regulation and Clinical Evidence
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Using the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS) and the Scale for Clinical Actionability of Molecular Targets (ESCAT), only about one-eighth of genome-targeted cancer drugs recommended by the National Comprehensive Cancer Network (NCCN) were likely to provide high clinical benefit, while approximately one-third demonstrated promising but unproven efficacy.
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Revisiting the Potential of “Money Blinds”: Lessons From Lykos’s MDMA Research

Eisenkraft Klein D, Kesselheim AS - Health Affairs Forefront

  • Commentary & Opinion
| August 2024
  • Regulation and Clinical Evidence
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The authors discuss the FDA advisory committee’s decision to recommend against approving Lykos Therapeutics’ MDMA therapy for PTSD, arguing that the case highlights broader issues with companies funding and running their own trials. They propose the implementation of “money blinds,” where an independent intermediary would oversee the funding and selection of investigators, to reduce the impact of sponsors’ financial and ideological biases on trial outcomes.
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Merging Medical and Drug Benefits: Seeing the Whole Picture

Gondi S, Rome BN, Shrank WH - Health Affairs Forefront

  • Commentary & Opinion
| August 2024
  • Price, Value, and Access
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The authors caution against excessive restrictions on insurer-PBM integration, outlining how this integration can facilitate efforts to better manage care and comprehensively view benefit design. Rather, they recommend interventions to ensure the PBM market operates fairly, including greater scrutiny of vertical integration between PBMs and pharmacies, which may have a greater anticompetitive effect.
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Trust in the Food and Drug Administration: A National Survey Study

Feldman WB, Rand LZG, Carpenter D, Russo M, Bhaskar A, Lu Z, Campbell EG, Darrow JJ, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| August 2024
  • Regulation and Clinical Evidence
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In a survey of more than 2,000 participants, a lack of trust in the FDA was associated with various characteristics, including female sex, rural communities, conservative political views, worse self-reported health, lower health care satisfaction, paying less attention to health and science news, and not having children under 18. These findings underscore the challenges in convincing a heterogeneous American public to trust the FDA.
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Biosimilar Uptake in the US: Patient and Prescriber Factors

Hong D, Kesselheim AS, Sarpatwari A, Rome BN - Health Affairs

  • Research Publications
| August 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Between 2013 and 2022, the proportion of patients who initiated a biosimilar in the US increased from 1% to 34%. Patients were less likely to initiate biosimilars if they were younger than age eighteen or the drug was prescribed by a specialist or administered in a hospital outpatient facility. These findings highlight the need for targeted policy intervention to increase biosimilar use.
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Misaligned Pharmacy Incentives in Value-Based Care

Jain N, Rome BN, Mendu ML - JAMA

  • Commentary & Opinion
| July 2024
  • Price, Value, and Access
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The authors discuss why pharmacy spending poses particular challenges for value-based care contracts between insurers and accountable care organizations, including the lack of transparency around rebates and the high cost of new, clinically effective therapies. Incorporating rebates and new drug approvals into pharmacy spending benchmarks, moving toward value-based formularies, and other strategies can help realign incentives.
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Op-Ed: This Gene Therapy May Not Work. So Why Did the FDA Fully Approve It?

Bendicksen L, Cliff ERS, Kesselheim AS - Washington Post

  • Commentary & Opinion
| July 2024
  • Regulation and Clinical Evidence
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The authors discuss the FDA’s full approval of Elevidys for Duchenne muscular dystrophy, which failed to meet its primary endpoint in a confirmatory trial. They highlight the risks of granting full approval to treatments that have failed to demonstrate significant benefit in clinical trials and caution against the approval of unproven therapies.
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Comments on Draft Policy, “NIH Intramural Research Program: Promoting Equity Through Access Planning”

Gabriele SME, Martin MJ, Kesselheim AS - National Institutes of Health (NIH)

  • Policy Interactions
| July 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The commenters support finalizing NIH’s proposed policy requiring licensees of NIH-developed technologies to submit access plans, recommending the inclusion of reasonable pricing commitments in such access planning. They also urge the agency to require public disclosure of access plans, consider patent type and license exclusivity when evaluating plans, and implement systematic annual reporting to monitor licensee compliance.
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Use of Track One Prioritized Examination for Pharmaceutical Patents

Tu SS, Feldman WB - JAMA Health Forum

  • Research Publications
| July 2024
  • Innovation Incentives and Competition
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In this review of the USPTO “Track One” program which allows companies to pay a fee for prioritized patent examination, brand-name drug manufacturers were found to disproportionately rely on the Track One program to quickly build their patent portfolios, often obtaining patents just as market exclusivity periods expire.
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State Spending Targets for Prescription Drugs

Hwang CS, Lalani HS, Rome BN - JAMA Health Forum

  • Commentary & Opinion
| July 2024
  • Price, Value, and Access
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The authors discuss recent state efforts to set prescription drug spending targets for state-sponsored health insurance plans. They outline the limitations of using this policy to address high drug spending absent a more robust approach to improve affordability.
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Removing the FDA’s Boxed Hepatotoxicity Warning and Liver Function Testing Requirement for Ambrisentan

Feldman WB, Mahesri M, Sarpatwari A, Huybrechts KF, Zhu Y, Hwang CS, Lii J, Lee SB, K Sreedhara S, Toyserkani GA, Zhou EH, Zendel L, LaCivita C, Manzo C, Dal Pan GJ, Kesselheim AS, Bykov K - JAMA Network Open

  • Research Publications
| July 2024
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Two FDA-approved treatments for pulmonary arterial hypertension, bosentan and ambrisentan, initially carried boxed warnings for hepatotoxicity and required monthly liver function tests (LFTs). The FDA later removed the warning and LFT requirement for ambrisentan in 2011, but not for bosentan. This study found an immediate increase in the use of ambrisentan but no change for bosentan after the labelling changes. LFT monitoring decreased for ambrisentan while remaining stable for bosentan.
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Comments on Proposed Rule, “Terminal Disclaimer Practice to Obviate Nonstatutory Double Patenting”

Tu SS, Carrier MA, Chao B, Cook-Deegan RM, Feldman WB, Heled Y, Kesselheim AS, Robertson CT, Sinha MS - US Patent and Trademark Office (USPTO)

  • Policy Interactions
| July 2024
  • Innovation Incentives and Competition
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The commenters support the USPTO’s proposed rule to reform terminal disclaimer practice, presenting evidence that the current system enables brand-name drug manufacturers to build thickets of indistinct continuation patents that delay generic and biosimilar competition and contribute to high drug prices.
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Biosimilar Approval Pathways: Comparing the Roles of Five Medicines Regulators

Knox RP, Desai V, Sarpatwari A - Journal of Law and the Biosciences

  • Research Publications
| July 2024
  • Innovation Incentives and Competition
  • Regulation and Clinical Evidence
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The authors compare the regulatory approval process for biosimilars in five jurisdictions (Australia, Canada, EU, UK, and US), noting differences in biosimilar testing requirements and policies regarding indication extrapolation, exclusivities, and biosimilar substitution. Understanding the impact of these policies on biosimilar approval and uptake is important to strengthen the global biosimilar market.
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The Cost of Drug Patent Expiration Date Errors

Tu SS, Cheian D, Gabriele SME, Rome BN, Kesselheim AS - Nature Biotechnology

  • Research Publications
| July 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The USPTO uses an automated program to calculate patent expiration dates which is subject to errors. Among 200 key drug patents, the program had a 4% error rate that erroneously extended the patent term, potentially resulting in hundreds of millions of dollars in added drug spending.
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Patent Portfolios Protecting 10 Top-Selling Prescription Drugs

Horrow C, Gabriele SME, Tu SS, Sarpatwari A, Kesselheim AS - JAMA Internal Medicine

  • Research Publications
| July 2024
  • Innovation Incentives and Competition
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Nearly 75% of the US patents and patent applications protecting the 10 highest-revenue brand-name drugs in 2021 were filed after FDA approval. These post-approval patents, particularly those on parts of the drug other than the active ingredient, can contribute to dense patent thickets that warrant closer scrutiny by the USPTO to limit delays to generic and biosimilar competition.
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Measuring and Understanding Market Exclusivity Length for New Prescription Drugs in France, Australia, and the USA

Van de Wiele VL, Kesselheim AS, Gleeson D, Lu Z, Tu SS, Rome BN - Pharmaceutical Medicine

  • Research Publications
| July 2024
  • Innovation Incentives and Competition
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The length of market exclusivity obtained by brand-name drug manufacturers was found to be similar in the US, France, and Australia, in part due to various policy differences in the three markets that affect the duration of market exclusivity. Alignment of market exclusivity lengths internationally could better enable timely generic entry to address high prescription drug spending in these countries.
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Quantifying Research and Development Expenditures in the Drug Industry

Wouters OJ, Kesselheim AS - JAMA Network Open

  • Commentary & Opinion
| June 2024
  • Innovation Incentives and Competition
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In this comment on an a new bottom-up estimate of the costs of drug development, the authors highlight the value of these cost estimates to ongoing policy discussions on prescription drug pricing and innovation while also recognizing the methodological limitations of each estimate.
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Federal Trade Commission Actions on Prescription Drugs, 2000-2022

Daval CJR, Egilman AC, Sarpatwari A, Kesselheim AS - JAMA

  • Research Publications
| June 2024
  • Innovation Incentives and Competition
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From 2000-2022, the Federal Trade Commission (FTC) pursued 22 enforcement actions against pharmaceutical firms for alleged anticompetitive practices and challenged 62 mergers, of which 61 were allowed to continue, 58 after divesting drugs to third-party competitors. Congressional action may be necessary to strengthen the FTC’s authority and enforcement capacity related to pharmaceuticals.
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Patient-Level Savings on Generic Drugs Through the Mark Cuban Cost Plus Drug Company

Kouzy R, El Alam MB, Corrigan KL, Lalani HS, Ludmir EB - JAMA Health Forum

  • Research Publications
| June 2024
  • Price, Value, and Access
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In this economic evaluation, patients could have saved on 12% of all prescriptions filled across 124 generic drugs in 2019, with a median savings of $5 per fill, if the drugs were purchased through the Mark Cuban Cost Plus Drug Company’s (MCCDPC) direct-to-consumer pharmacy. Uninsured patients would have benefitted the most from using MCCDPC.
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Comments for Listening Session, “Optimizing FDA’s Use of and Processes for Advisory Committees”

Rand LZG - Food and Drug Administration (FDA)

  • Policy Interactions
| June 2024
  • Regulation and Clinical Evidence
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The FDA should convene advisory committee meetings more frequently with formal votes to promote public trust, provide explanations when agency decisions differ from committee recommendations, and develop guidance on how patient and caregiver testimony should be incorporated into advisory committee decision-making.
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Medicare Negotiation’s Drug Reformulation Problem

Vogel M, Rome BN, Kesselheim AS, Feldman WB - Annals of Internal Medicine

  • Commentary & Opinion
| June 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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The authors shed light on a loophole in the Medicare Drug Price Negotiation program: once a generic version of any formulation of a drug is marketed, all formulations of that drug are exempted from negotiation. They detail how this loophole could enable costly gamesmanship, listing expensive drugs that could be excluded through this technicality. Instead, negotiation eligibility should be determined by aggregating Medicare spending on all formulations that do not face bona fide competition.
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How Health Technology Assessment Can Help to Address Challenges in Drug Repurposing: a Conceptual Framework

Abu-Zahra T, Grimm SE, Scholte M, Raymakers AJN, Kesselheim AS, Joore MA - Drug Discovery Today

  • Research Publications
| June 2024
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Drug repurposing can be an efficient way to use existing drugs to treat new indications but is not without challenges, particularly in the early stages of development. This study identifies 20 challenges with drug repurposing, grouped into six categories (data access, research and development, collaboration, business case, regulatory and legal challenges), and outlines a framework for incorporating health technology assessment (HTA) methods to address these challenges.
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How Do Risk Evaluation and Mitigation Strategies Impact Clinical Practice? A National Survey of Physicians

Sarpatwari A, Lu Z, Russo M, Zakoul H, Lee SB, Toyserkani GA, Zhou EH, LaCivita C, Hyatt Hawkins Shaw K, Zendel L, Dal Pan GJ, Kesselheim AS - Clinical Pharmacology & Therapeutics

  • Research Publications
| June 2024
  • Regulation and Clinical Evidence
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Over two-thirds of surveyed physicians thought that the FDA-mandated risk evaluation and mitigation strategies (REMS) certification process for certain drugs provided useful context and information, though roughly the same proportion wanted REMS materials to include additional data on drugs’ benefits. Physicians also indicated that they wanted more information concerning non-REMS-related safety risks and that REMS paperwork should be better integrated into clinical workflows.
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Global Cooperation and Early Access-Clinical Outcomes Matter

Tibau Martorell A, Kesselheim AS - Lancet Oncology

  • Commentary & Opinion
| June 2024
  • Regulation and Clinical Evidence
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In this commentary on two analyses related to cancer drug approvals, the authors highlight that although using surrogate measures may reduce costs and expedite patient access to new treatments, these measures might not accurately predict overall survival benefits. Thus, rigorous post-approval testing and transparent data reporting are critical to ensure patients receive the most effective treatments.
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Estimated Medicare Part D Savings From Generic Drugs with a Skinny Label

Egilman AC, Kesselheim AS, Sarpatwari A, Rome BN - Annals of Internal Medicine

  • Research Publications
| June 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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For 15 brand-name drugs that faced competition from “skinny-label” generics, these generics entered the market a median of 2.5 years earlier than generic competition might otherwise be expected. This earlier competition aved Medicare Part D an estimated $14.6 billion from 2015 to 2021. To reinforce these savings, a safe harbor should be created to protect manufacturers engaged in skinny labeling from induced patent infringement lawsuits.
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Use of Artificial Intelligence in Drug Development

Druedahl LC, Price WN, Minssen T, Sarpatwari A - JAMA Network Open

  • Research Publications
| May 2024
  • Regulation and Clinical Evidence
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Out of more than 100,000 investigational and approved drugs listed in the Pharmaprojects database, developers reported using artificial intelligence (AI) to aid in the development of 164 investigational drugs and 1 approved drug. Thus, the use of AI in drug development has been modest and mostly limited to early-stage applications, such as molecule discovery and screening.
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Medicare Should Cover Weight Loss Drugs as Long as the Prices Are Affordable

Hwang CS, Kesselheim AS, Rome BN - Journal of Law, Medicine & Ethics

  • Commentary & Opinion
| Spring 2024
  • Price, Value, and Access
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Glucagon-like peptide-1 (GLP-1) receptor agonists are effective for treating obesity, but the high cost of these medications endangers the financial viability of our health care system. To ensure that these drugs are available to Medicare beneficiaries, pharmaceutical manufacturers must lower their prices.
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Testimony: Patent Thickets and Product Hops: How Congress Could Reward Legitimate Innovation While Facilitating More Timely Generic Competition

Feldman WB - 118th Congress, Senate Committee on the Judiciary

  • Policy Interactions
| May 2024
  • Innovation Incentives and Competition
  • Price, Value, and Access
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Feldman outlines how brand-name manufacturers delay generic competition through large patent thickets and product hops. He uses inhalers and GLP-1 receptor agonists as primary case studies, noting that more than half of patents on these drug-device combinations cover devices rather than active ingredients. He endorses four bipartisan patent reform bills currently under consideration and proposes additional reforms, including FDA authority to review Orange Book listings, mandatory USPTO re-examination, limits on patents asserted in infringement litigation, and adjustments to the 180-day generic exclusivity and 30-month stay periods. Watch his testimony.
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Clinical Benefit and Regulatory Outcomes of Cancer Drugs Receiving Accelerated Approval

Liu ITT, Kesselheim AS, Cliff ERS - JAMA

  • Research Publications
| May 2024
  • Regulation and Clinical Evidence
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Among a 5-year cohort of cancer drugs approved by the FDA under accelerated approval, only half of approvals demonstrated improvements in overall survival or quality of life in confirmatory trials, with 60% of conversions to full approval relying on clinical endpoints.
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The Value of the Quality-Adjusted Life Years

Willke RJ, Pizzi LT, Rand LZG, Neumann P - Value in Health

  • Commentary & Opinion
| May 2024
  • Price, Value, and Access
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Recent concerns about the quality-adjusted life year (QALY) being discriminatory against selected patient groups have resulted in US legislation and regulation that would ban the use of the QALY in government programs. The authors identify scientific support for the QALY and its usefulness as an input to healthcare decision-making, acknowledge its limitations, and argue that, when appropriately constructed and used, it will not be discriminatory against these patient groups.
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Clinical Value of Molecular Targets and FDA-Approved Genome-Targeted Cancer Therapies

Tibau Martorell A, Hwang TJ, Molto C, Avorn J, Kesselheim AS - JAMA Oncology

  • Research Publications
| May 2024
  • Regulation and Clinical Evidence
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Fewer than a third of genome-targeted oncology drugs approved in the US between 2015 and 2022 were shown to have substantial clinical benefit at the time of approval. Benefit frameworks like the ESMO Magnitude of Clinical Benefit Scale (MCBS) can help identify genome-targeted therapies with demonstrated clinical potential.
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From “Price Controls” to Robust Public Conversation: How CMS Can Move Drug Price Negotiation Into the Light

Ollendorf DA, Rand LZG, Mooney H, Phillips M, Basu A - Health Affairs Forefront

  • Commentary & Opinion
| April 2024
  • Price, Value, and Access
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The authors propose a framework for the Medicare Drug Price Negotiation Program that aims to promote an evidence-based, robust process that includes stakeholders and draws on both cost-effectiveness and open deliberation to reach maximum fair prices.
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The FDA in the Crosshairs-Science, Politics, and Abortion

Fernandez Lynch H, Kesselheim AS - JAMA

  • Commentary & Opinion
| April 2024
  • Regulation and Clinical Evidence
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The authors discuss the implications of the Supreme Court’s decision in FDA v. Alliance for Hippocratic Medicine for abortion access, FDA regulatory discretion, and the extent to which the FDA’s actions preempt conflicting state laws. They urge the Supreme Court to endorse the importance of the FDA’s public health mission and the agency’s authority to take regulatory action under a broad Congressional mandate.
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State Coverage and Reimbursement of Antiobesity Medications in Medicaid

Liu BY, Rome BN - JAMA

  • Research Publications
| April 2024
  • Price, Value, and Access
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In early 2023, 21% of state Medicaid programs covered at least one anti-obesity medication. From 2011 to 2022, the number of prescriptions for anti-obesity medications reimbursed by Medicaid increased by 1300%, driven by use of the glucagon-like peptide-1 (GLP-1) receptor agonists. Considering the cost burden of novel anti-obesity medications and their therapeutic potential, Medicaid beneficiaries’ access to these therapies will depend on negotiating lower prices.
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Changes in Use of Hepatitis C Direct-Acting Antivirals After Access Restrictions Were Eased By State Medicaid Programs

Davey S, Costello K, Russo M, Davies S, Lalani HS, Kesselheim AS, Rome BN - JAMA Health Forum

  • Research Publications
| April 2024
  • Price, Value, and Access
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In a difference-in-differences analysis using Medicaid data from 2015-2019, the use of highly effective direct-acting hepatitis C antivirals increased substantially after states eliminated coverage restrictions based on the severity of liver disease, active substance use, or prescriber specialty. These findings suggest that such restrictions have prevented access to an important and cost-effective public health intervention.
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Spending on Targeted Therapies for Duchenne Muscular Dystrophy

Bendicksen L, Kesselheim AS, Rome BN - JAMA

  • Research Publications
| April 2024
  • Price, Value, and Access
  • Regulation and Clinical Evidence
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The US spent an estimated $3 billion on targeted therapies for Duchenne muscular dystrophy from 2016-2023, despite limited evidence of effectiveness and delayed confirmatory trials. More than $1 billion was paid by Medicaid and Medicare. Policymakers should compel the timely completion of confirmatory trials for these therapies and implement alternative payment models to minimize financial risk in case novel interventions prove ineffective.
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We Need to Talk About Values: a Proposed Framework for the Articulation of Normative Reasoning in Health Technology Assessment

Charlton V, DiStefano MJ, Mitchell P, Morrell L, Rand LZG, Badano G, Baker R, Calnan M, Chalkidou K, Culyer A, Howdon D, Hughes D, Lomas J, Max C, McCabe C, O'Mahony JF, Paulden M, Pemberton-Whiteley Z, Rid A, Scuffham P, Sculpher M, Shah K, Weale A, Wester G - Health Economics, Policy and Law

  • Commentary & Opinion
| April 2024
  • Price, Value, and Access
Talk bubble graphics representing commentary and opinion.
Health technology assessment (HTA) is an inherently value-based activity, yet variation exists in the description of value-based judgements across HTA organizations and literature. The authors propose a framework that distinguishes between the considerations, claims, and beliefs used in HTA, providing a tool to support decision-makers and improve transparency.
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Use of Efficiency Frontiers to Align Prices and Clinical Benefits of Biologic Therapies for Plaque Psoriasis

Egilman AC, Kesselheim AS, Avorn J, Raymakers AJN, Rome BN - JAMA Dermatology

  • Research Publications
| April 2024
  • Price, Value, and Access
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Using an efficiency frontier approach, the prices for 13 biologic drugs used to treat plaque psoriasis would need to be reduced by a median of 71% in the US to align with prices for the most cost-effective therapeutic alternatives on the frontier. This approach offers policymakers a useful alternative to conventional cost-effectiveness analysis that could be useful for therapeutic classes with multiple drugs.
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