Podcast
MSL Talk: Tom Caravela, Elizabeth Pash
Use of Real-World Evidence to Support FDA Approval of Oncology Drugs
Summary
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 December 2016, the 21st Century Cures Act (“Cures Act”) was signed into United States (US) law. The Cures Act, designed to improve the efficiency and speed of new medical product development and regulatory approval, mandated that the Food and Drug Administration (FDA) establish a program to evaluate the potential use of real-world evidence (RWE) to support the approval of new indications for drugs, and to satisfy post-approval study requirements. The Framework for the FDA’s Real-World Evidence Program (“Framework”) was published in December 2018.
In this document, the FDA defines real-world data (RWD) as data related to patient health status or the delivery of healthcare that is routinely collected from a variety of sources, such as electronic health records (EHRs), claims and billing reports, and registries, in addition to other sources such as mobile devices. RWE, derived from analysis of RWD, provides clinical insights with respect to the usage, benefits, or risks of a medical product...
Food and Drug Administration, oncology drug approval, real-world data, real-world evidence
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