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When Rare Is Well Done: Priority Review Vouchers

Amanda Stevenson-Grund - Friday, October 23, 2015

Perhaps the most notable aspect of the vouchers so far is the cost they have been able to command. BioMarin's voucher, the first ever to be sold, was purchased for $67 million. Several months later, Gilead Sciences purchased Knight Therapeutics' voucher for $125 million.
 –
Gaffney. Regulatory Affairs Professional Society. 2015

A voucher that looks like a gift card with a bow represents the priority review voucher program.

When drug developers cannot serve certain patient populations for financial reasons, the FDA creates monetary incentives. This is seen in the priority review voucher programs. There are two types of priority review vouchers, tropical disease vouchers and rare pediatric disease vouchers. The biggest differences between the two programs are the diseases treated and program longevity.

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A Tender Age: Pediatric Patients with Rare Diseases

Amanda Stevenson-Grund - Friday, October 09, 2015

Approximately 50% of the people affected by rare diseases are children. 30% of children with rare disease will not live to see their 5th birthday.”
 – Global Genes. RARE Diseases: Facts and Statistics

 A zebra-print ribbon represents pediatric rare diseases

Many people hope to grow older and wiser. Some just hope to grow older. Pediatric patients, especially those with rare diseases, face life-threatening challenges. Three problems pediatric patients with rare diseases face are late diagnosis, few approved treatment options and lack of age-appropriate formats.

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