Bill Would Make Permanent FDA's Rare Pediatric Voucher Program

Regulatory NewsRegulatory News | 24 March 2015 |  By 

New legislation introduced this week would reauthorize the US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program, which is currently set to end after triggering a sunset clause in its authorizing statute.


Priority review vouchers are incentives meant to catalyze the development of new therapies for historically under-served disease areas, such as rare pediatric diseases affecting fewer than 200,000 children in the US.

Due to the difficulty of developing and marketing drugs for diseases affecting only a small number of patients, in 2012 legislators created a new incentive intended to attract new pharmaceutical development.

That incentive is a special voucher which allows its owner to have any experimental drug the company owns reviewed by FDA under its "priority review" pathway. The priority review pathway allows for the review of a drug in just six months instead of the standard 10 months—a valuable incentive for many, if not most, companies.

The voucher may also be sold to other companies. To date, two companies have sold priority review vouchers: one (a rare pediatric disease voucher) for $67.5 million and another (a neglected tropical disease voucher) for $125 million.

Read our extensive Regulatory Explainer on the Priority Review Voucher program here.

A Sunset Clause

On 17 March 2015, FDA issued its sixth-ever priority review voucher, and its third rare pediatric disease priority review voucher.

Priority Review Vouchers Awarded to Date
Date Voucher AwardedVoucher TypeCompany Voucher Awarded to:
2009Tropical DiseaseNovartis
2012Tropical DiseaseJanssen
2014Rare Pediatric DiseaseBioMarin
2014Tropical DiseaseKnight Therapeutics
2015Rare Pediatric DiseaseUnited Therapeutics
2015Rare Pediatric DiseaseAsklepion Pharmaceuticals

As reported first by Regulatory Focus, the award of the third pediatric voucher triggered a little-known sunset clause contained in the 2012 law which authorized the voucher program, the Food and Drug Administration Safety and Innovation Act(FDASIA).

The sunset clause in FDASIA reads:

The Secretary may not award any priority review vouchers under paragraph (1) after the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section.

In plain terms: After FDA awards its third rare pediatric disease priority review voucher, a one-year countdown clock begins. After the year is up, FDA may no longer award any additional rare pediatric disease priority review vouchers. The vouchers themselves may still be redeemed.

According to statute, FDA's rare pediatric disease priority review voucher program is now slated to end after 17 March 2016.

A New Legislative Fix

But the program will continue if new legislation introduced on 23 March 2015 is signed into law.

The new bill, the Advancing Hope Act of 2015, would make permanent the rare pediatric disease priority review voucher program in the hopes of maintaining an incentive for pediatric drug development.

In addition, the bill would also modify the definition of "rare pediatric disease"—now any disease primarily affecting fewer than 200,000 children—to specifically include "any form of sickle cell disease" and "any pediatric cancers."

Tropical Changes

Other changes within the bill would also modify FDA's other voucher system, its neglected tropical disease priority review voucher program. The tropical voucher system works much like the rare pediatric voucher program, but is instead intended to promote the development of treatments for a select list of "neglected" tropical diseases.

The legislative language addresses a criticism of the tropical voucher program, which was that it allowed companies to submit drugs already approved in foreign markets for approval in the US, thereby allowing them to obtain a voucher.

The bill's new language prohibits companies from obtaining a voucher if their drug has "been approved for commercial marketing for any tropical disease indication by a government authority outside of the US" for longer than 24 months.

The bill also prevents companies from obtaining two vouchers for the same drug product (i.e. a drug which treats a rare, pediatric subset of a neglected tropical disease).


Advancing Hope Act of 2015

Press Statement


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