Issue Date: July 17, 2025
Issued by: Oncology Center of Excellence
Topic(s): Clinical – Medical
Status: Draft
Development of Cancer Drugs for Use in Novel Combination – Determining the Contribution of the Individual Drugs’ Effects: Draft Guidance for Industry
This guidance provides recommendations for characterizing the safety and effectiveness of individual drugs used in novel combination regimens for treating cancer, with a focus on demonstrating the “contribution of effect” – how each drug contributes to the overall treatment benefit observed in patients. The guidance is intended for sponsors developing cancer drug combinations and addresses three specific scenarios: (1) Two (or more) investigational drugs that have not been previously approved by FDA for any indication, (2) An investigational drug with a drug(s) approved for a different indication, and (3) Two (or more) drugs approved for a different indication(s). The recommendations are designed to be consistent with existing FDA regulations for fixed-combination prescription drugs and expand upon the 2013 guidance for industry Codevelopment of Two or More New Investigational Drugs for Use in Combination. The guidance does not cover situations where an investigational drug is being developed in combination with a drug approved for the same indication for purposes of comparing the approved drug to the combination (i.e., “add-on” trials to standard of care), fixed combinations of previously approved drugs for the approved indications, safety or dosing considerations when designing combination trials, or the evaluation of safety data to support the benefit-risk assessment of combination therapies.
Download the Guidance Document
