Pharmaceuticals Regulation

FDA’s use of real-world data

The 21st Century Cures Act required the Food and Drug Administration (FDA) establish a program for evaluating the use of real-world data (RWD) to support the approval of new indications for drugs. Real-world data is typically data from either health insurance claims, electronic health records (EHRs), patient registries, or mobile devices. But how has FDA used RWD in practice?

A paper by Feinberg et al. (2020) examines oncology drugs approved by the FDA between 2017 and 2019 to try to answer this question. In this time period, 40 new oncology drugs were approved.

Five of the 40 made reference to RWE submitted in support of the approval. During the same time period, 71 supplemental indication approvals were identified (for 38 oncology drugs); however, drug approval packages were only available for 13. Three of the 13 made reference to RWE submitted in support of the approval. All 8 of the approvals with submitted RWE involved indications with an unmet need for effective therapies. For 5 of the 8 approvals with submitted RWE, the data represented historical controls; in 2 cases the RWE was derived from expanded access studies, and in 1 case the RWE was collected from off-label use of an approved therapy in a new patient population. The submitted RWE was rejected by FDA in 3 of the 8 approvals.

Many of the drugs using RWD were indicated for rare diseases and a variety of different real-world data sources were used.

…we found that 4 of the 5 drugs reviewed had orphan drug designation, and the fifth was for a rare subset within a larger patient population (palbociclib for male patients with breast cancer). Three of the 5 drugs (avelumab, blinatumomab, and selinexor) received accelerated approval for the indications for which RWE was submitted; all 3 had PMRs [post-marketing requirements] for confirmatory clinical trial data, with avelumab and blinatumomab both requiring new clinical trials. The types of RWE used in the regulatory submissions included EHR data, claims data, postmarketing safety reports, retrospective medical record reviews, and expanded access study data.

Efficacy was most often justified using EHR data as a historical control or expanded access studies. Sometimes the EHR data was supplemented with claims data or diagnostic test results (e.g., next-generation sequencing data).


  • Feinberg BA, Gajra A, Zettler ME, Phillips TD, Phillips Jr EG, Kish JK. Use of Real-World Evidence to Support FDA Approval of Oncology Drugs. Value in Health. 2020 Sep 14.

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