Gottlieb’s FDA to rethink targeted therapy regulation to speed development, drive down healthcare costs

The FDA is working on a medical innovation development plan designed to lower healthcare costs by streamlining the path to market for targeted therapies and other novel drugs. Commissioner Scott Gottlieb, M.D., pitched the plan as a broad drive to ensure the FDA has the tools and policies to encourage and evaluate innovative therapies.   
Gottlieb’s discussion of the plan at a Senate subcommittee represented the deepest dive yet into his plans for regulation of innovative medicine R&D. The vision centers on equipping the agency to facilitate the speedy development of next-generation therapies, particularly targeted drugs that treat patients with rare diseases or molecularly-defined cancers. 
Such drugs will likely command big prices. But Gottlieb, drawing a distinction between price and value, still managed to frame the policy as part of his vision for how the FDA can help manage the cost of healthcare. 
“The most tangible way we’re going to reduce healthcare costs is by finding better treatments for a lot of costly diseases,” Gottlieb said. 
Gottlieb provided nuggets of information about what this will mean in practice. 
Targeted drugs designed to treat rare diseases and other unmet needs are one area of focus. The FDA plans to facilitate the efficient development of such drugs by updating guidance documents.  
“This includes guidance on clinical trial enrichment strategies to improve efficiency and adaptive trial designs so that we can modernize the statistical tools we use to evaluate safety and effectiveness,” Gottlieb said.
The FDA is also working on new guidance documents. Notably, Gottlieb is prioritizing the creation of guidance on the clinical evaluation of targeted therapies for rare disease subsets. The document will seek to simplify development of drugs aimed at rare genetic disorders and diseases that share a similar genetic fingerprint, even if they manifest in slightly different clinical expression. 
“One example is a cancer where a drug targets a particular molecular subset, regardless of where the tumor arises. We'll clarify when we can give broad approval to a drug in multiple different kinds of molecularly-similar cancers which are not particular to the tumor being in one specific tissue or organ,” Gottlieb said.
Gottlieb also wants to encourage clinical development programs that use lab tests to group rare subsets of patients into single trials. 
The commissioner presented the plan alongside internal changes at the FDA designed to speed the development of rare disease drugs. Gottlieb inherited a backlog of orphan drug applications and has made permanently fixing the situation a priority. The FDA has convened a team to clear the backlog within 90 days. Beyond that, Gottlieb wants to put processes in place that ensure the FDA always responds to applicants within 90 days.