Existing FDA Pathways Have Potential to Ensure Early Access To, and Appropriate Use Of, Specialty Drugs

The FDA has multiple regulatory tools to balance timely patient access to specialty drugs, which offer substantial clinical benefits but carry significant risks and high costs, including expanded access programs and expedited approval pathways to accelerate availability, as well as post-approval mechanisms like narrow indications, companion diagnostics, and Risk Evaluation and Mitigation Strategies (REMS). The authors argue that effective implementation of these existing approaches can promote access for patients most likely to benefit while preventing expensive and potentially harmful overuse.