The use of real-world evidence to support FDA post-approval study requirements for oncology drugs

Abstract

ABSTRACT Background The 21st Century Cures Act of 2016 included provisions for the Food and Drug Administration (FDA) to evaluate the potential for real-world evidence (RWE) to support or fulfill post-approval study requirements. This study reviewed post-marketing requirement (PMR) and post-marketing commitment (PMC) obligations for oncology drugs approved by the FDA post-Cures Act to identify those with RWE components. Methods Approval letters issued by the FDA between 2017–2020 for oncology drugs were systematically analyzed for PMRs or PMCs with requests for RWE. For each PMR/PMC identified, the characteristics of the approvals, the PMRs/PMCs, and the RWE requested were reviewed. Results Of 189 oncology drug approvals with 456 associated PMRs/PMCs, a total of 15 PMRs/PMCs specified RWE. Compared with all oncology drug approvals, the 14 approvals with PMRs/PMCs requesting RWE were more frequently accelerated approvals, for new therapies, with orphan indications. All 15 PMRs/PMCs requested real-world safety data, with 3 also requesting real-world effectiveness data. RWE requested included post-marketing safety reports, prospective observational studies, expanded access study data, and registry data. Conclusion As a greater proportion of safety and efficacy data generation for oncology drugs shifts to the post-marketing setting, RWE has the potential to become an integral component of PMR/PMC fulfillment.