FDA releases draft guidance for combination treatments

FDA released draft guidance for the co-development of two or more investigational drugs for use in combination. FDA said co-development of investigational compounds should be reserved for instances in which a compelling biological rationale for use of the combination exists; an established biomarker suggests the combination has substantial activity and provides greater additive activity or a more durable response than the individual agents alone; the agents cannot be developed individually; and the combination seeks to treat a serious disease or condition.

The draft noted that Phase I testing should characterize the safety of each individual drug in the same manner as would be done for development of a single drug, in addition to characterizing the safety of the combination. The agency said Phase II trials should demonstrate the contribution of each component of the combination by comparing the combination to individual components and placebo or standard of care. If findings from in vivo or in vitro models and/or Phase II trials adequately demonstrate the contribution of each component to the combination, the agency said Phase III trials comparing the combination to standard of care or placebo generally will be sufficient to establish effectiveness. ...