FDA's Priority Review Voucher Programs

A voucher is a bond of the redeemable transaction type which is worth a certain monetary value and which may be spent only for specific reasons or on specific goods. Examples include (but are not limited to) housing, travel, and food vouchers.

You may find it surprising, the voucher has been used by FDA as a tool to encourage the drug development in certain areas.

In 2008, FDA issued its 1st voucher guidance titled “Tropical Disease Priority Review Vouchers”. Last month, FDA has published its second guidance related to voucher. The draft guidance is called “Rare Pediatric Disease Priority Review Vouchers, Guidance for Industry”

How does it work?

• Sponsors must first have an NDA/BLA approved for an indication in designated tropical disease area or in qualified rare pediatric disease area. 
• Sponsor will then submit the application for priority review voucher
• Sponsor may need to pay additional application fee for voucher
• Once the priority review voucher is approved, the voucher can be sold and transferred to other sponsors
• Voucher can be redeemed for priority review for any NDA/BLA submission 

Both voucher programs are designed to provide incentives for drug developers to invest in the neglected disease area or in the disease area that return on investment (ROI) is very low.
According to FDA's MAPP "Review Designation Policy: Priority (P) and Standard (S)", applications or supplements submitted with a priority review voucher will automatically receive a priority review designation.

The tropical disease priority review voucher was issued in 2008 and it was not used often by the sponsors. However, the tropical disease priority review voucher may find new popularity thanks to the global fight against Ebola. Ironically, at the time when the Tropical Disease Priority Review Voucher guidance was issued, the deadly Ebola disease was not on the list of tropical disease areas.

Product applications for the prevention or treatment of the following tropical diseases may qualify:

• Tuberculosis
• Malaria
• Blinding trachoma
• Buruli Ulcer
• Cholera
• Dengue/Dengue haemorrhagic fever
• Dracunculiasis (guinea-worm disease)
• Fascioliasis
• Human African trypanosomiasis
• Leishmaniasis
• Leprosy
• Lymphatic filariasis
• Onchocerciasis
• Schistosomiasis
• Soil transmitted helminthiasis
• Yaws
• Any other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations, designated by regulation by the Secretary (section 524(a)(3))

One may argue that the Ebola can be included in the last item “any other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations, designated by regulation by the Secretary”.

To ensure that Ebola is included in the Tropical Disease Priority Review Voucher program and to remove the obstacles for voucher program to become popular, a Senate bill has been proposed. The bill passed the senate in November.

The newly issued guidance on Rare Pediatric Disease Priority Review Voucher program seems to be better designed and hopefully it will gain more popularity than the Tropical Disease Priority Review Voucher program. To avoid incentivizing sponsors to exclude adults affected by the rare pediatric disease from clinical trials, FDA expects adult patients to play a prominent role in process. Sponsors remain eligible for a voucher if they use adult patients in clinical trials or seek an adult indication in addition to the primary pediatric indication. The qualified rare pediatric disease will most likely also qualify for the orphan disease category. A sponsor may obtain the Orphan Drug Designation Status to avoid paying the application fee for voucher application.

How much is a priority review voucher worth?

The value of a priority review voucher is not entirely clear. There are very few transactions of a priority review voucher sold from one sponsor to another.

• On 30 July 2014, BioMarin announced that it had sold its voucher to Sanofi and Regeneron for $67.5 million.
• The Canadian pharmaceutical company Knight Therapeutics has reportedly sold its Neglected Tropical Disease Priority Review Voucher to Gilead Sciences for $125 million
• On May 27, 2015, Retrophin sold their priority review voucher to Sanofi for $245 million

Reference: 

                  raps.org: Everything you need to know about priority review voucher