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

Value in Health
October 2020 Volume 23, Issue 10, p1281-1402


Use of Real-World Evidence to Support FDA Approval of Oncology Drugs
Bruce A. Feinberg, Ajeet Gajra, Marjorie E. Zettler, Todd D. Phillips, Eli G. Phillips Jr., Jonathan K. Kish
Published online: September 14, 2020
:: 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.