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21st Century Cures Brings Hope for Rare Disease Research


However, not everyone supports the bill's provisions that promote the development of pediatric rare disease drugs through the "voucher" incentive.

The 21st Century Cures bill, which was unanimously approved by a House panel just a few weeks back, brings promise of faster drug development, and also offers hope to the rare disease advocacy groups. The bill includes provision to extend the "voucher" program for companies developing treatments for pediatric rare diseases.

Approximately 15 million children are diagnosed with rare diseases, which can result in nearly 35% of deaths in the first year of life.

Pharmaceutical companies are often cautious about venturing into the world of rare diseases for several reasons: fear of potential negative outcomes, limited returns, increased stringency with clinical trials in children, etc. Therefore, to motivate the pharmaceutical industry to invest efforts in developing theses drugs, the Congress initiated a voucher program in 2011 which allows the company to have another one of their drugs approved quicker than usual. The company can also sell this voucher if they so desire.

The Cures bill is seeking an extension on this voucher program, which is scheduled to expire next year.

However, not everyone is happy with this program. Read more at Kaiser Health News: http://bit.ly/1KXbSvK

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