FDA Launches New Rare Disease Program

The FDA has launched a new program that is aimed to increase the development of drugs for people with rare diseases. Called Accelerating Rare Disease Cures (ARC), the new program is managed by Center for Drug Evaluation and Research’s (CDER) Rare Diseases Team and will provide coordination across the agency’s efforts in this area.

This new effort continues the regulator agencies effort to provide support for the development of rare diseases. One effort, developed in collaboration with the Critical Path Institute, is the Rare Disease Cures Accelerator-Data and Analytics Platform. The platform provides a database designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection.

There are about 7,000 rare diseases and conditions, which affect about 30 million people. Developing new therapies for rare diseases can be complex. Established trial designs may not work with these potential treatments, and endpoint selection can be challenging if there is a limited understanding of the natural history of the disease. Additionally, because of small patient populations, there may not be enough people available to participate in rare disease clinical trials.

Progress is being made for some rare diseases. In 2021, 26 of the 50 novel drugs approved by the FDA were for orphan diseases. And in the first few months of 2022, the FDA approved four new drugs for people with rare diseases in the areas of oncology and hematology. These drugs:

• The first therapy for uveal melanoma (a rare cancer that develops in a part of the eye called the uvea) that has spread to other parts of the body or cannot be surgically removed.
• The first therapy to decrease the need for red blood cell transfusion due to hemolysis (red blood cell destruction) in adults with cold agglutinin disease, a rare type of anemia.
• The first therapy to treat anemia in adults with pyruvate kinase deficiency, an inherited disorder that causes premature red blood cell destruction.
• A drug for adults with intermediate or high-risk primary or secondary myelofibrosis, a rare bone marrow disorder, who have low platelet (blood clotting cells) levels.

Kerry Jo Lee, M.D,

“However, despite an increase in the approval of novel drugs to treat rare diseases and conditions, there is still a tremendous unmet need for FDA approved therapies,” Kerry Jo Lee, M.D., associate director for rare diseases, Office of Rare Diseases at the FDA, said in a video introducing the new program.

“Through scientific and regulatory innovations and engagement, we believe the ARC program will help accelerate the availability of treatments for patients with rare diseases,” she said.

More than 700 drugs are in development for rare diseases, according to the Pharmaceutical Research and Manufacturers of America (PhRMA), which represents biopharmaceutical companies.