The U.S. Food and Drug Administration has updated its guidance to the pharmaceutical industry for cancer clinical trials, including new endpoints for proving a treatment's efficacy.
In a Dec. 19 update, the FDA expanded on its guidance for companies conducting clinical trials on drugs they ultimately want the regulator to approve. The guidance will help advance the efficient development of cancer drugs and biologics, the agency said. The update is a revision of the Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics guidance, which was issued in May 2007.
Changes include clarifications to how various cancer trial endpoints — the main goals of the studies — can serve different purposes, for example allowing "surrogate" endpoints to support a traditional or accelerated drug approval. Surrogate endpoints are defined by FDA regulation as a marker, such as a laboratory measurement, radiographic image or physical sign, that can be used to determine the clinical benefit of a drug.
The agency also provided its current thinking on factors that are considered in approving these clinical trials. Moreover, the guidance provides examples of newer cancer trial endpoints that have been used.
"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," said FDA Commissioner Scott Gottlieb in a Dec. 19 statement. "As part of these advances, there's been a robust debate about the use of surrogate endpoints to support both traditional and accelerated approvals."