Surrogate Endpoints, Fewer Trials Continue to Back Novel Drug Approvals
The FDA’s novel drug approvals continued to be supported by fewer, less rigorous and more flexible pivotal trials as well as surrogate endpoints in 2020, a new analysis has found.
Of the 49 novel drug approvals and 75 pivotal trials supporting them in 2020, 28 drugs (57.1 percent) were approved based on a single pivotal trial, 17 (34.7 percent) were based on two trials and just four (8.2 percent) were backed by three or more, according to the analysis conducted by Mayookha Mitra-Majumdar, a research specialist at Brigham and Women’s Hospital, and colleagues.
The analysis, published in JAMA, also found that 34 (45.3 percent) of the pivotal trials used a surrogate measure for their primary endpoints, while 21 (28 percent) used a historical, external or other control, all of which were much more common in cancer trials.
Of these drugs, a majority (39) qualified for at least one of the FDA’s expedited approval pathways, while 25 of them were eligible for three or more. Specifically, 30 were given Orphan Drug status, 28 were granted priority review and 12 earned accelerated approval. In total, 178 postmarket requirements and commitments (PMR/PRC) were established for the drugs, with an average of 3.6 per drug and with 40 of the drugs having one or more. Only nine of the drugs had no PMR/PRC.
The varying amounts and quality of data that backed 2020’s novel drug approvals can in part be chalked up to an increase in drugs for rare diseases and other serious conditions that may require more flexibility, the researchers said.
Additionally, 19 of the 49 novel drug approvals were first-in-class, a classification that usually involves a new mechanism of action and can involve more flexible evaluations by the agency. And 30 of the drugs received Orphan Drug designation, for which the FDA may be willing to accept more uncertain evidence, the researchers said.
Read the full analysis findings here: https://bit.ly/3sJQANc.