FDA Acceptance of Surrogate End Points for Cancer Drug Approval: 1992-2019
- 194 approvals were analyzed, with 64 (32.9%) relying on surrogate endpoints for the first time in a specific cancer type.
- Surrogate endpoints often have weak or unknown correlations to overall survival (OS).
- Many approvals lack strong scientific or regulatory justification, indicating a higher tolerance for risk.
- The use of surrogate endpoints has increased over time but involves significant uncertainty regarding actual patient benefit.
- The importance of stringent postmarketing studies to confirm the clinical benefits of these drugs was emphasized by the researchers.
The US Food and Drug Administration (FDA) approves cancer drugs based on direct measures of patient benefit—such as overall survival (OS) or quality of life—or surrogate measures, such as change in biomarker level or tumor size on imaging studies. Surrogate end points often have weak or unknown correlations with OS, and postmarketing studies are limited. A surrogate end point can be used repeatedly in a particular cancer setting, such as response rate (tumor shrinkage) in mantle cell lymphoma in 2013 (with ibrutinib and lenalidomide) and 2017 (with acalabrutinib) after it was accepted for the first time in 2006 (with bortezomib).
One open question is how often does the FDA approve a drug based on a surrogate end point that has never been used before in treating that type of cancer? In this study, we assess the frequency of surrogate measures used for the first time vs subsequent times in a cancer setting and the surrogate’s strength of correlation with patient-centered outcomes...
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