The FDA will launch a rare disease center to unify its policies and find new ways to evaluate drugs that can pose a regulatory dilemma, it announced Wednesday.
The new Rare Disease Innovation Hub comes after the agency has struggled to reach a consensus on what types of evidence and trial designs are needed to evaluate therapies for rare conditions, particularly when there are very small numbers of patients who could participate in a test. Companies like Stealth BioTherapeutics say that they have received conflicting advice from different FDA divisions on how to run studies, imperiling their therapies.
The new hub looks to connect various parts of the agency and will be led by CBER Director Peter Marks and CDER Director Patrizia Cavazzoni.
“We see huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance our intercenter connectivity to spur the development of treatments for rare diseases,” they wrote in the news release.
The hub will promote consistency across scientific, clinical and policy issues, while seeking to advance novel endpoints, biomarker tests, innovative trial designs and statistical methods, the officials said. A newly-created senior leadership position, director of strategic coalitions for the hub, will play a critical role in the work that spans therapeutics, medical devices and diagnostic tests.
Word of the hub emerged last month during a panel that included Marks, Endpoints News reported at the time. Marks has looked to speed up rare disease medicines, including through a pilot modeled after Operation Warp Speed that produced the Covid-19 vaccines.