FDA Updates Endpoints in Non-Small Cell Lung Cancer Clinical Trials

April 27, 2015

While overall survival remains the desired endpoint in treating non-small cell lung cancer, progression-free survival may suffice as the primary endpoint, the FDA says.

Sponsors should justify use of PFS as the primary efficacy endpoint and the magnitude of the PFS effect expected to predict overall survival or demonstrate clinical benefit versus the drug’s risk given the lung cancer stage and results of other therapies, the agency says in final guidance released today.

Typically, efficacy endpoints used in clinical trials of NSCLC drugs include overall survival, time to progression and progression-free survival, although patient-reported outcomes may provide evidence of treatment benefit if they are well-defined, reliable and evaluated in well-conducted, placebo-controlled or double-blinded, randomized trials, the FDA say.

Regardless of the efficacy endpoints used, NSCLC is a complex disease with varying response to treatment across different subgroups, the agency adds. Clinical trials should be prospectively designed to evaluate such differences in treatment effect.

The FDA encourages sponsors to meet with the agency before submitting protocols to support NDA or BLA marketing applications. Applicants also can request a special protocol assessment to confirm the appropriateness of their endpoint measures and protocol design for individual trials.

Lung cancer accounts for about 14 percent of all new cancers and is the leading cause of cancer deaths.

Read the guidance, Clinical Trial Endpoints for the Approval of Non-Small Cell Lung Cancer Drugs and Biologics, at www.fdanews.com/04-22-15-NSCLC.pdf. — Jessica Grinspan