Washington, DC - “As part of 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, M.D.
“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. It helps make research and development more effective and can lower the cost of bringing safe and effective treatments to patients. For example, we recently published on our website a list of the surrogate endpoints that were the primary basis of approval or licensure of a drug or biological product for both accelerated and traditional approvals. We also recently issued guidance on considerations for the kinds of evidence that can support qualifying biomarkers for use in drug and biologic development programs. Today, we’re revising guidance for the first time in more than a decade that outlines our thinking on oncology endpoints based on the specific context of use, as well as the advantages and disadvantages of these endpoints in clinical development programs. We continue to encourage companies to engage with FDA early in the drug development process so that we can work with them to apply modern and efficient trial designs, as well as explore novel endpoints, such as minimal residual disease, which recently formed the basis of approval for a new treatment for a type of leukemia.”
Today, the U.S. Food and Drug Administration issued guidance, 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 FDA to support effectiveness claims in applications. This updated guidance will help advance the efficient development of cancer drugs and biologics. Today’s guidance is a revision of the guidance of the same title published in May 2007 and replaces that 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 (e.g., 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 NDA or BLA marketing 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.