Gottlieb Pledges Revamp on Targeted Drug Development/Reviews
FDA commissioner Scott Gottlieb has pledged to revamp the new drug development process for targeted drugs. Testifying before a Senate Appropriations subcommittee hearing 6/20, Gottlieb acknowledged that the “most tangible way we are going to reduce healthcare costs is by finding better treatments for a lot of costly diseases. Towards these ends, we will be announcing soon a medical innovation development plan that will include a broad range of steps that we will take to make sure our regulatory tools and policies are modern and risk-based and designed to facilitate the development of potentially breakthrough new treatments.”
One top focus area of this new plan is going to be on targeted drugs, especially those that affect rare diseases or diseases which there is no effective therapy, Gottlieb told the hearing. “Among other things, FDA will be updating various guidance documents on the kinds of drug development techniques that help facilitate the discovery and development of targeted therapies,” he said. “This includes guidance on clinical trial enrichment strategies to improve efficiency, and adaptive trial designs that will modernize statistical tools we use to evaluate safety and effectiveness. We will also be taking a fresh look at policies that support innovation to allow drugs to be targeted only to those patients who are most likely to benefit from the medicine.”
Among these new actions, Gottlieb said the agency will be issuing a new guidance document within the next six months on the clinical evaluation of targeted therapies for rare disease subsets. “This new policy will address targeted drugs and how we can simplify the development of drugs targeted to rare disorders that are driven by genetic variations and where diseases all have a similar genetic fingerprint, even if they have a slightly different clinical expression,” he said. “One example is a cancer where a drug targets a particular molecular subset of cancer regardless where the tumor arises. We will clarify when we can give broad approval to a drug in multiple different kinds of molecularly similar cancers, which are not particular to a tumor being in one specific tissue or organ. In another case, rare subsets may be grouped by lab testing so they can be studied in a single clinical trial. This sort of genetically driven medicine is more common as we understand the genetic basis of disease. Now, our new policy will describe when we will approach drug review less by how a disease is expressed and more by how it is driven by a common set of genetically driven factors.”
Many of these targeted drugs will be aimed at rare and orphan diseases, but Gottlieb said FDA currently has a backlog of about 200 orphan drug designation requests that require reviews and responses. “As part of our new plan, we are committing today that in 90 days we will completely eliminate this backlog of requests and provide an answer back to the sponsors. To help eliminate the backlog, we have created a special orphan designation ‘SWAT’ team. Moreover, we will never again develop a backlog. Going forward we are committing today that every orphan drug application will receive a response from FDA within 90 days of the request. To enable more efficient reviews and timely responses to sponsors, we are also implementing a new streamlined orphan designation review template.”
During the hearing, subcommittee chair John Hoeven (R-ND) said he was concerned that FDA’s current budget request relies on a doubling of user fees for industry. Hoeven said the fee increase is not feasible and unlikely to gain approval, indicating that Congress instead will more likely pass the previously negotiated user fee reauthorization bill.
Hoeven asked Gottlieb whether he was confident that FDA can meet its program needs based on FY 2017 appropriated levels. “I am confident that we have been able to be efficient in everything we do,” Gottlieb answered. “I think there are still places that we can look within the agency to try to improve our operational efficiency.” Asked about 1,000 vacancies currently at FDA, Gottlieb said the agency is starting to move forward with filling these now that the hiring freeze has been lifted.