Will a $67.5 Million Priority Review Voucher Stimulate Pediatric Drug Development?

James Radke

September is National Childhood Cancer Awareness Month and to help commemorate that, Kids v Cancer is raising awareness of the programs available to increase clinical trials specific for children with cancer.

And one of the most powerful programs may be the priority review voucher program that is part of the FDA Safety & Innovation Act (S. 3187), or FDASIA signed into law in 2012.  

Section 908 of FDASIA states  the FDA will give priority review (≤  6 months) to medication/devices designed to treat  rare disease that mostly afflict children (≤ 18 years of age).  If the applicants provide proper evidence, not only will they get a priority review, the applicant will also receive a ‘priority review voucher.’ 

The priority review voucher entitles the sponsor of a rare pediatric disease product application to apply a priority review for another drug application. In other words, the company can get their next drug reviewed by the FDA in 6 month instead of the standard 10 months, regardless of whether it is a pediatric indication or not. 

The company can also sell the voucher to another company. Which is what Biomarin did for $67.5 million.

In July of this year, BioMarin sold its priority review voucher for $67.5 million to Regeneron and Sanofi. Biomarin obtained its voucher following the approval of the Vimizim for children with Morquio A syndrome.

For the past two years, many in the industry have been aware of the voucher program but until now, nobody knew what a voucher was worth.  We now know its worth– at least $67.5 million.

More importantly, the selling of that voucher may be the catalyst needed to inspire others to study pediatric indications.  According to Nancy Goodman, Founder and Executive Director of Kids v Cancer, “We have now reached a tipping point in pediatric drug development. " Ms Goodman added,  "Where it was once financially unfeasible, businesses now have a reason to develop drugs for kids with cancer and other rare diseases."

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