Use of Real-World Evidence to Support FDA Approval of Oncology Drugs

Real-world evidence (RWE) has gained increased attention in recent years as a complement to traditional clinical trials. The use of RWE to establish the efficacy of oncology drugs for Food and Drug Administration (FDA) approval has not been described. In this paper, we review 5 recent examples where RWE was submitted in support of the FDA approvals of original or supplementary indications for oncology drugs. To identify cases where RWE was used, we reviewed drug approval packages available at Drugs@FDA for oncology drugs approved between 2017 and 2019. Five cases were selected to present a broad overview of different types of RWE, different circumstances under which RWE has been used for regulatory approvals, and how FDA evaluated the data in each case. The type of RWE submitted, the indication, limitations identified by FDA reviewers, and the outcome of the submission are discussed. RWE, particularly historical controls for rare or orphan indications, has been used to support both original and supplementary oncology drug approvals. Types of RWE included data from electronic health records, claims, post-marketing safety reports, retrospective medical record reviews, and expanded access studies. Small sample sizes, data quality, and methodological issues were among concerns cited by FDA reviewers. By bridging the gap between the constraints of the trial setting and the realities of clinical practice, RWE can add value to a regulatory submission. These early examples provide insight into how regulators evaluated RWE submitted as evidence of efficacy for oncology drugs. •In accordance with the 21st Century Cures Act directive, the Food and Drug Administration (FDA) is evaluating the potential for real-world evidence (RWE) to support the approval of supplementary indications or fulfill post-approval study requirements for drugs.•Our analysis found that significant precedent exists for the FDA’s acceptance of RWE (including data from electronic health records, claims, post-marketing safety reports, retrospective medical record reviews, and expanded access studies) to support oncology drug approvals, especially for rare or orphan indications.•The use of RWE to support drug approval is a viable strategy with the potential to provide clinically relevant information while reducing the time, cost, and patient burden associated with clinical trials.