Drugs

10% of FDA-approved Drugs Had Null Findings: JAMA

A new JAMA research article says that between 2018 and 2021, FDA approved 210 new drugs and 21 (10.0%) were approved based on pivotal studies with “null findings for one or more primary efficacy endpoints.” The researchers, from Yale Medical Scholl and Brigham and Women’s Hospital, say their interest in such data grew from the agency’s 2021 controversial accelerated approval of Biogen’s Alzheimer disease therapy aducanumab (see earlier story) after both pivotal trials of the drug were stopped for futility.

The researchers found that during 2018 to 2021, 21 drugs were approved for 21 unique clinical indications. “Of these drugs, 11 (52.4%) were first in class, 10 (47.6%) received orphan designation, and 13 (61.9%) received an expedited review designation,” they say. “Before approval, an advisory committee was convened for 3 (14.3%) of the drugs.”

Additionally, they note that the 21 drug approvals “were supported by 56 pivotal trials; five approvals (23.8%) were supported by a single pivotal trial. These 56 trials included 74 primary efficacy endpoints, 27 of which (36.5%) were null; among these 27 endpoints, 13 (48.1%) were clinical outcomes, seven (25.9%) were surrogate markers, four (14.8%) were clinical scales, and three (11.1%) were composite endpoints. Four drug approvals — naxitamab-gqgk for high-risk refractory or relapsed neuroblastoma, tazemetostat hydrobromide for epitheloid sarcoma, migalastat hydrochloride for adults with Fabry disease, and asparaginase erwinia chrysanthemi (recombinant) for acute lymphoblastic leukemia and lymphoblastic lymphoma — were based on a single pivotal study with null results for all pivotal primary endpoints.”

The rationale for approving the 21 drugs was favorable data in at least one other pivotal study (n = 13; 61.9%), positive findings from secondary or exploratory end points in the pivotal study (n = 10; 47.6%), and favorable post hoc analysis (n = 7; 33.3%), the research article says. FDA also “required or requested postmarketing studies for seven (33.3%) of the drugs to address the null endpoint or a related clinical endpoint,” they say.

Gupta Raises Concerns About Overton’s Independence, Ideology

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome