Reauthorize the Rare Pediatric Disease PRV Program as part of 21st Century Cures
Dear Majority Leader McConnell, Speaker Ryan, and Minority Leaders Reid and Pelosi,

On behalf of the 30 million men, women and children in the U.S. living with a rare disease, the undersigned organizations urge you to pass the 21st Century Cures Act (H.R.6) and include a long-term, optimally permanent, reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program.

There are an estimated 7,000 rare diseases, which are defined as a disease affecting fewer than 200,000 people. Of the nearly one in ten Americans with a rare disease, over half are children. Despite significant unmet medical need (the approximately 550 approved orphan products treat only about 375 rare diseases), there are significant obstacles that can hinder the pursuit of rare disease therapies. Pediatric development often proves additionally difficult due to issues associated with conducting clinical trials.

The 21st Century Cures Initiative includes various provisions that are widely supported by the rare disease patient community, including additional resources for the NIH, the expansion of the FDA Patient-Focused Drug Development Initiative (S.1597), provisions to streamline FDA review of targeted orphan therapies (S.2030), and additional incentives for adding rare disease indications onto labels (OPEN Act, S.1421).

The 21st Century Cures Act and the Senate Innovation for Healthier Americans Initiative both included reauthorizations of the Rare Pediatric Disease PRV Program. This program has enjoyed bipartisan and bicameral support since its inception, and it was recently unanimously extended in September through the end of this year.

Currently, upon FDA approval of a novel rare pediatric disease treatment, the Rare Pediatric Disease PRV Program provides a biopharmaceutical manufacturer the opportunity to receive a voucher guaranteeing a priority review of a new drug or biologic for another product. Unfortunately, this program is again set to expire at the end of this year, eliminating a clear pathway that encourages innovators to pursue treatments in a difficult disease space. Still in its infancy, this program has not been allowed to show its true effectiveness as short-term reauthorizations do not maximize the potential benefits of this powerful incentive.

Through 21st Century Cures, Congress has bipartisanly recognized that more needs to be done for the rare disease patient community. The Rare Pediatric Disease PRV Program is just one of many important provisions, but it uniquely requires immediate Congressional attention due to its approaching expiration date. Continued inaction or short-term reauthorizations will only work to discourage industry from investing in rare pediatric diseases.

Thousands of patients and advocates have asked Congress to act. It is imperative that Congress passes the 21st Century Cures Act and includes a long-term, optimally permanent, reauthorization of the Rare Pediatric Disease PRV Program.

Sincerely,

The National Organization for Rare Disorders

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To view the 2015 letter, please go here: http://rarediseases.org/assets/files/policy-statements/2015-04-13.renew-prv-senate.pdf

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