Stakeholders Share Thoughts on Draft Guidance on Targeted Therapies

PhRMA, Merck and Illumina offered feedback on the FDA’s draft guidance on the development of treatments that target diseases caused by molecular alterations, requesting the agency provide more details.

PhRMA noted the guidance focuses on grouping multiple molecular alterations within the same clinical disease, but urged the agency to also consider situations where one or more molecular alterations are found across multiple “diseases” — for example, a single genetic mutation that is found in tumors at different tissue sites.

The draft guidance, published last December, examines the appropriateness of pursuing an indication in a disease’s molecular subset, depending on whether evidence suggests patients with the genetic alteration are more likely to respond to the targeted therapy than those without.