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FDA Issues Guidance on Endpoints for Cancer Clinical Trials

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This week, the U.S. Food and Drug Administration (FDA) issued a guidance titled Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics. The guidance provides recommendations to applicants on endpoints for cancer clinical trials submitted to the FDA to support effectiveness claims in applications. This updated guidance will help advance the efficient development of cancer drugs and biologics. The guidance is a revision of the guidance of the same title published in May 2007, and replaces the prior document.

The new updates made to this guidance expand on the information regarding oncology endpoints and provide updated resources, references, and examples of regulatory approvals. The guidance clarifies how various oncology endpoints can serve different purposes (eg, clinical endpoint that represents clinical benefit for traditional approval, surrogate endpoint to support traditional approval, surrogate endpoint to support accelerated approval) and provides current thinking on the factors that are considered in making the determination. Other updates include the addition of examples of emerging oncology endpoints and the addition of intermediate clinical endpoints in the discussion of accelerated approval.

Although the general principles outlined in this guidance should help applicants select endpoints for marketing applications, the FDA recommends that companies meet with the agency before submitting clinical trial protocols intended to support new drug applications or biologics license applications. The FDA will ensure that these meetings include a multidisciplinary FDA team of oncologists, statisticians, clinical pharmacologists, and external expert consultants as needed. Ultimately, marketing approval depends not only on the design of clinical trials, but also clinical trial conduct and the findings from all studies in the drug marketing application.

Commentary From the Commissioner

“As part of [the] FDA’s strategy to continue to encourage the modernization of clinical trials, we’re providing new recommendations for drug developers regarding the most effective clinical trial endpoints to help advance the development of products to treat cancer. Over the past several decades, we’ve seen an evolution in cancer care in how treatment effect is measured, and which endpoints are successful measures of disease activity or clinical benefit to patients. As part of these advances, there’s been a robust debate about the use of surrogate endpoints to support both traditional and accelerated approvals. We’ve engaged patient and health-care professional communities to inform our regulatory decision-making around these issues, to ensure we are keeping pace with the science and continuing to encourage development of treatments that offer meaningful results for patients,” said FDA Commissioner Scott Gottlieb, MD. “Applying the most efficient clinical trial designs and using meaningful endpoints that measure benefits important to patients is key to our efforts to modernize clinical trial development programs.”

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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