The FDA’s Center for Drug Evaluation and Research (CDER) https://www.fda.gov is trying to develop therapies for the more than 30 million people living with a rare disease in the U.S.
So far in 2022, FDA has approved four new drugs for people with rare diseases in oncology and hematology. Despite this progress, the vast majority of rare diseases still do not have approved therapies.
CDER has multiple efforts underway to support rare disease drug development and believes that data sharing is critical to advancing drug development, particularly for rare diseases.
FDA is developing a Rare Disease Cures Accelerator-Data and Analytics Platform. The platform provides a database hub designed to promote the secure sharing of existing patient level data and encourages the standardization of new data collection.
The aim will be to receive and protect data from a variety of sources that can inform rare disease characterization, clinical trial design, and other critical questions in rare disease drug development.
The platform enables authorized users to access patient-level clinical data to better understand disease progression and disease heterogeneity (or differences among people with the same disease) across rare disease patient populations.
This information can inform trial design, selection of endpoints, and other important considerations pertaining to drug development. The hope is to avoid mistakes that can cause delays or failures in the development of new drugs for rare diseases.
Also, as part of the Standard Core Clinical Outcome Assessment Grant Program, CDER is working with grantees to develop publicly available core sets of clinical outcome assessments that may be used as endpoints in clinical trials.
Some of the assessments developed through this project may include physical function, pain in infants and young children, and communication in children with neurodevelopmental disorders. FDA is also working with investigators from the University of Michigan to develop innovative trial designs for rare diseases with small populations.
The Rare Disease Cures Accelerator-Data Analytics Platform is being led by the Critical path Institute through a cooperative grant agreement from FDA. Go to https://drugs/regulatory-science-research-and-education/rare-disease-cures-accelerator for more information on the Rare Disease Cures Accelerator-Data and Analytics Platform.