On June 20, 2017, Food and Drug Administration (FDA) Commissioner Scott Gottlieb, M.D., testified
before a Senate Appropriations Subcommittee and discussed some of the programs he is implementing to streamline the FDA review process – some of which will have a significant impact on companies developing orphan drugs for rare diseases.
Gottlieb hopes to make orphan drugs easier to be reviewed by putting a stop to the requirement of companies to undertake new clinical trials for different disease subsets with the same drug. Gottlieb instead hopes to create a review process in which the mechanism of action for a particular drug will dictate the population in the clinical trial, rather than the particular disease.
How the medical, regulatory, payor, and pharmaceutical communities respond to this concept is yet to be seen. While the idea may help streamline the review process, it will require a completely foreign way of looking at diseases from all parties involved. Further, it may make some drugs ineligible to be labeled as “orphan” if it is targeting a particular cellular entity present in many cancers or diseases.
One initiative with which Gottlieb plans to impact the rare disease community has already been implemented by the FDA; there is a backlog of 200 applications for orphan drug designations needing review at the FDA and Gottlieb has created a strengths, weaknesses, opportunities, and threats (SWOT) team to have each of them addressed in the next 90 days. He also plans to permanently remove the current procedures in place that created that backlog to begin with.
In addition to the 2 initiatives, Gottlieb has continued to argue
that the FDA’s slow and outdated drug approval process needs to be updated, and that the current focus of the agency is hindering the development of novel treatments - specifically rare diseases where an over reliance on statistical analysis rather than common sense can make it almost impossible to conduct a trial in a timely manner.
For updates on Dr Scott Gottliebs proposals and initiatives as FDA Commissioner, follow Rare Disease Report