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

Background The 21(st) 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.