“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
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.
The tropical disease priority review voucher program is part of the Food and Drug Administration Amendments Act of 2007. Through the program, the FDA can grant vouchers to companies that are developing drugs for “neglected” tropical diseases. The companies can then use those vouchers for shortened review process and a faster time to market. Neglected tropical diseases are diseases that affect poor populations who can’t afford expensive treatment, so there is little financial incentive for drug developers. Such diseases include malaria, leprosy and tuberculosis. An additional act in 2014 added filoviruses, such as ebola, to the list of eligible diseases.
The rare pediatric disease voucher program is part of the Food and Drug Administration Safety and Innovation Act. This program allows the FDA to grant vouchers to companies that are developing drugs for rare diseases in pediatric patients. Eligible diseases are those that affect 200,000 people younger than age 18 in the United States or diseases that affect more than 200,000 children but for which no treatments are available because drug developers could not earn a profit after the costs of drug development. This program will end in spring of 2016 unless it is renewed before then.
Vouchers for both programs can be used after 90 days of notice. Both types of vouchers can be sold to other companies. There is no limit on the number of times a voucher can be sold. There is great financial benefit in selling such a voucher. For example, Sanofi paid $245 million to Retrophin in May of 2015.
The tropical disease and rare pediatric disease priority review voucher programs function similarly. However, they differ in the diseases their drugs treat and in the longevity of the programs. Orbis Biosciences has experience developing drugs for pediatric patients. Our Precision Particle FabricationTM technology allows us to create format-flexible and taste-masked medications. If incentives, such as those through the rare pediatric disease priority review voucher program, continue, innovation and experience in pediatric drugs will grow. Companies like Orbis will be able to prove that treating these underserved populations is a priority.