Drug Repurposing Could Bring Safe and Effective New Therapies to Rare Disease Patients

As part of MedChemNet's February focus on rare disease research, Emil D. Kakkis, President and Founder of the EveryLife Foundation for Rare Diseases, describes the challenges and opportunities of the field,

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Currently, only a few small percentage of the 7,000 known rare diseases have a treatment approved by the U.S. Food and Drug Administration. Although millions of rare disease patients in the U.S. have no approved treatment options, some of them may benefit from off-label use of medicines approved for other conditions.

Despite these challenges, I remain optimistic. History has shown that smart and targeted public policy can make a significant impact by helping to spur innovation for rare disease patients. The incentives created by the Orphan Drug Act of 1983, for example, are credited with dramatically increasing the number of therapies available to patients, from under a dozen available in 1983 to more than 400 therapies that are on the market today, with hundreds more in development.

While this progress is noteworthy, we have so much farther to go. One proposal, known as the Orphan Product Extensions Now, Accelerating Cures & Treatments (OPEN ACT; H.R. 971/S. 1421), passed the House of Representatives last summer as part of the 21st Century Cures Act with broad bipartisan support and is under review in the Senate. The OPEN ACT would encourage companies to repurpose therapies approved by the FDA to treat common diseases for rare disease indications by offering a 6-month extension of marketing exclusivity. This incentive would go a long way in ensuring we have the clinical data to support the often anecdotal use of off-label medications for rare diseases. The bill would also allow lower cost options for rare disease patients than drugs specifically developed only for rare disease patients given the pricing would be based on the larger market indication.

Repurposing approved drugs is the fastest and most-cost efficient way to get safe and effective therapy options to rare disease patients who are running out of time.

Emil D. Kakkis MD, PhD
President and Founder
EveryLife Foundation for Rare Diseases


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EveryLife Foundation

Senior Director, EveryLife Foundation for Rare Diseases

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases. Currently, there are fewer than 400 approved treatments for 7000 rare diseases affecting more than 30 million Americans. The science exists for many of these diseases to be treated; however, treatments may never be developed because of roadblocks in the development process, such as a lack of investment and a challenging regulatory environment. The Foundation works with Patient Organizations, Industry, Academic Scientists, the Food and Drug Administration (FDA), and National Institutes of Health (NIH) to improve the clinical development process through our CureTheProcess Campaign, Rare Disease Workshop Series, and legislation.

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