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FDA offers new hope for families facing rare disease

New hope for families facing rare disease

FDA’s Center for Drug Evaluation and Research (CDER) is pleased to announce the launch of the new Accelerating Rare disease Cures (ARC) Program. The CDER ARC Program is intended to speed up and increase the development of effective and safe treatment options for patients and their families facing rare diseases.

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Drug development in rare disease states

Drug development for the approximately 7,000 rare diseases and conditions can be complex for many reasons. There can be challenges with using well-established clinical trial designs, and endpoint selection can be complex if there is a limited understanding of the natural history of the disease. Small patient populations can also make it difficult to perform and interpret rare disease clinical trials.

Despite an increase in approvals for novel drugs to treat rare diseases and conditions, there is still a tremendous unmet need for FDA-approved treatments. The new CDER ARC Program will help support the development and approval of safe and effective treatment options for patients through scientific and regulatory innovation and engagement.

 

Future of rare

CDER is optimistic about the future of rare disease drug development and is looking forward to continuing this important work under the new CDER ARC Program — together with patients, advocacy groups, academics, industry, and other partners — to address the significant unmet needs of patients and families living with rare diseases.

 

 

For more information about the CDER ARC Program, please visit https://www.fda.gov/drugs/CDERARC.

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