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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Jessica K. Smith Founder and CEO; Executive Director

Jessica Keenan Smith is a patient advocate and epilepsy community leader with more than 15 years of experience. As Founder and CEO of Living Well With Epilepsy and Executive Director of ASENT she bridges the gap between the scientific and patient communities, with a particular focus on the needs of the epilepsies. Jessica Keenan Smith is Founder and CEO of Living Well With Epilepsy, an award winning online resource for people affected by epilepsy to share stories and access in-depth information on the disease. Since 2009, Living Well With Epilepsy (https://livingwellwithepilepsy.com) has been featured in Forbes, Wired, NBC, NPR and the cover story of Epilepsy Advocate Magazine and has partnered with companies such as UCB, Lundbeck, Sunovion, Eisai, and more. Ms. Smith is also the Executive Director of the American Society for Experimental Neurotherapeutics (ASENT) (https://asent.org), an organization that brings together leaders from industry, academia, government and advocacy who are engaged in bringing drugs and devices to market across all neurologic disease states. In this role she is responsible for leading a successful scientific journal and annual scientific conference with speakers from all over the world.

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