Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019
| Autor: | Nicholas S. Downing, Meera Dhodapkar, Nilay Shah, Jeremy Puthumana, Audrey D Zhang, Joseph S. Ross |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
medicine.medical_specialty
Randomization Prescription Drugs Placebo Design characteristics Food and drug administration Strength of evidence Interquartile range Internal medicine medicine Humans Drug Approval Original Investigation Biological Products End point business.industry United States Food and Drug Administration Research Health Policy Clinical Studies as Topic Drug Repositioning General Medicine United States Online Only Cross-Sectional Studies Research Design business |
| Zdroj: | JAMA Network Open |
| ISSN: | 2574-3805 |
| Popis: | Key Points Question What is the strength of evidence supporting supplemental new indication approvals for drugs and biologics and how does it compare with the evidence that supported their original approval? Findings In this cross-sectional study of 107 therapeutics approved for 146 supplemental indications by the US Food and Drug Administration between 2017 and 2019, supplemental approvals for oncology drugs were based on fewer pivotal efficacy trials with less rigorous designs than supplemental approvals for other therapeutic areas. Supplemental approvals were based on fewer pivotal trials than their original indication approvals, but their designs were similar. Meaning These findings suggest that there was little difference in the evidence supporting supplemental and original indication approvals, but the number and design of pivotal trials supporting supplemental indication approvals varied according to therapeutic area. This cross-sectional study seeks to characterize pivotal trials supporting recent supplemental new indication approvals of drugs and biologics by the US Food and Drug Administration (FDA) and compares them with pivotal trials that supported these therapeutics’ original indication approvals. Importance After US Food and Drug Administration (FDA) approval of a new drug, sponsors can submit additional clinical data to obtain supplemental approval for use for new indications. Objective To characterize pivotal trials supporting recent supplemental new indication approvals of drugs and biologics by the FDA and to compare them with pivotal trials that supported these therapeutics’ original indication approvals. Design, Setting, and Participants This is a cross-sectional study characterizing pivotal trials supporting supplemental indication approvals by the FDA between 2017 and 2019 and pivotal trials that supported these therapeutics’ original indication approvals. Data analysis was performed from August to October 2020. Main Outcomes and Measures Number and design of pivotal trials supporting both supplemental and original indication approvals. Results From 2017 to 2019, the FDA approved 146 supplemental indications for 107 therapeutics on the basis of 181 pivotal efficacy trials. The median (interquartile range) number of trials per supplemental indication was 1 (1-1). Most trials used either placebo (77 trials [42.5%; 95% CI, 35.6%-49.8%]) or active comparators (65 trials [35.9%; 95% CI, 29.3%-43.1%]), and most of these multigroup trials were randomized (141 trials [99.3%; 95% CI, 96.0%-100.0%]) and double-blinded (106 trials [74.5%; 95% CI, 66.6%-81.0%]); 80 trials (44.2%; 95 CI, 37.2%-51.5%) used clinical outcomes as the primary efficacy end point. There was no difference between oncology therapies and those approved for other therapeutic areas to have supplemental indication approvals be based on at least 2 pivotal trials (11.5% vs 20.6%; difference, 9.1%; 95% CI, 2.9%-21.0%; P = .10). Similarly, there was no difference in use of randomization (98.3% vs 100.0%; difference, 1.7%; 95% CI, 1.6%-5.0%; P = .43) among multigroup trials, although these trials were less likely to be double-blinded (50.8% vs 92.3%; difference, 41.5%; 95% CI, 27.4%-55.5%; P |
| Databáze: | OpenAIRE |
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