Therapeutic Value and Regulatory Characteristics of Breakthrough Therapy Drugs in the USA and China (2021-2023): A Retrospective Cohort Study

In this comparison of drugs granted breakthrough therapy designation (BTD) by the US FDA and China’s NMPA, BTD drugs were found in both markets to more often receive accelerated or conditional approval based on limited evidence. Many of these products were also classified as offering only minor or insufficient clinical benefit at market entry. Regulators in both countries should grant BTD more cautiously and emphasize robust, evidence-based evaluation of therapeutic value to ensure this expedited pathway delivers meaningful clinical benefits.