The Advancing Hope Act of 2015 – A First Shot at Reauthorizing the Rare Pediatric Disease Priority Review Voucher Program

By Alexander J. Varond –

On March 23, Representative G.K. Butterfield (D-NC) introduced H.R. 1537, the “Advancing Hope Act of 2015.”  The Advancing Hope Act would reauthorize the rare pediatric disease priority review voucher (“Pediatric Voucher”) program, which is slated to sunset in March 2015.  Rep. Butterfield’s press release can be found here.  We discussed the Pediatric Voucher program and its sunset clause here.  The bill would also amend the tropical disease priority review voucher (“Tropical Disease Voucher”) program.

The Advancing Hope Act would modify the definition of “rare pediatric disease” to specifically include “any form of sickle cell disease” and “any pediatric cancers.”  This appears to be in response to various requests for the inclusion of sickle cell disease submitted to FDA’s docket for its Draft Guidance on the Pediatric Voucher program and efforts by Kids v. Cancer.  We discussed the Draft Guidance here.  The Advancing Hope Act would also eliminate the possibility of receiving a Pediatric Voucher after a Tropical Disease Voucher was issued for the same drug or biological product.

The Advancing Hope Act also addresses the Tropical Disease Voucher program and would exclude drugs that have been “approved for commercial marketing for any tropical disease indication by a government authority outside of the United States for more than 24 months before the application is submitted.”  Such a limitation, however, could prove problematic for a number of reasons.  We discuss two of these reasons below.

First, the World Health Organization’s Prequalification Programme relies on regulatory findings from a select number of regulatory agencies (e.g., FDA, EMA, Health Canada).  WHO’s website explains:

The Prequalification Programme, set up in 2001, is a service provided by the World Health Organization (WHO) to facilitate access to medicines that meet unified standards of quality, safety and efficacy for HIV/AIDS, malaria and tuberculosis.  From the outset, the Programme was supported by UNAIDS, UNICEF, UNFPA and the World Bank as a concrete contribution to the United Nations priority goal of addressing widespread diseases in countries with limited access to quality medicines.

Prequalification was originally intended to give United Nations procurement agencies, such as UNICEF the choice of a range of quality medicines. With time, the growing list of products (i.e. medicines) that have been found to meet the set requirements has come to be seen as a useful tool for anyone bulk purchasing medicines, including countries themselves and other organizations.  For instance, the Global Fund to Fight AIDS, Tuberculosis and Malaria disburses money for medicines that have been prequalified by the WHO process.

Eliminating incentives for sponsors to seek FDA approval would likely slow WHO’s efforts to prequalify medicinal products and, therefore, could have a significant impact on global health.

Second, adding this exclusion to the Tropical Disease Voucher program would remove incentives for sponsors to further develop, manufacture, and seek U.S. approval of drugs that, at some point, were approved outside of the United States but are no longer manufactured.  FDA’s Draft Guidance on the Tropical Disease Voucher program addresses a variety of reasons the program was enacted.  Among these is the concern that “[a]lthough these tropical diseases are rare in the United States, intercontinental jet transport, immigration, tourism, and military operations are increasing the direct impact these diseases have on the health of Americans.”  Without these incentives, it is unlikely sponsors would seek approval in the United States.  If confronted with a health emergency for a tropical disease in the United States, stocks of a drug approved outside the United States might be unavailable.  Even if they were available outside the United States, they would not have been determined to be safe or effective in the United States.