News flash: the priority review voucher (PRV) may be getting easier to use, and expansions are on the docket. On Wednesday, Senator Brownback (R-KS) introduced a bill that includes some important amendments to the FDA’s PRV program. The bill was co-sponsored by Senator Sherrod Brown (D-OH) and Al Franken (D-MN), and has been named the “Creating Hope Act of 2010,” or more specifically, bill number S. 3697 to amend the Federal Food, Drug, and Cosmetic Act to improve the priority review voucher incentive program relating to tropical and rare pediatric diseases.

This new legislation includes some critical changes for the PRV program. The changes below have been on BIO Ventures for Global Health’s advocacy agenda, which is informed by our PRV Working Group, a multi-stakeholder group that includes the product development partnerships (PDPs) focused on neglected tropical diseases (NTDs), economists, and investors, as well as biotechnology and pharmaceutical companies. We believe that these changes will be instrumental in helping capable innovators create new medicines for NTDs and fulfill the program’s potential: 

• Adds Chagas disease to the list of eligible NTDs, to align with the World Health Organization’s list of NTDs
• Allows for multiple transfers/sales of the voucher, once it is awarded 
• Allows for the withdrawal of a PRV by the sponsor before full review so the sponsor retains the rights to the voucher. This helps reduce the risk for industry to use the voucher because of the inherent uncertainty of clinical trial results, thereby increasing the estimated value
• Includes the ability of the FDA to make an “early designation” that a product would qualify for a voucher, at the request of a sponsor
• Closes the window for products that are in widespread use but have not gone through the FDA review process to be eligible for a PRV. We support the concept of closing the window for products that are in widespread use abroad because the PRV is meant to encourage new R&D, therefore it is of great value to include specific language around defining an “innovative treatment.” Specifically, the bill defines an innovative treatment as “a drug that has not 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 tropical disease product application is submitted.” While we believe this language supports the motivation for the incentives, we also want to make it clear that the first PRV award to Coartem (an anti-malarial treatment that was already in widespread use abroad) offers an important early test case for the program. To learn more about our thoughts on this, read our blog post on the subject here.

We’re excited to see many of the amendments we’ve been working toward captured in this bill, including amendments to the notification requirements, timelines, and user fee procedures. Until now, companies and PDPs have had difficulty structuring deals and business strategies to account for a PRV because the ‘rules of engagement’ had not yet been defined. Outlining these specifications acknowledges the importance of giving sponsors the information they need to value the voucher. It also allows those leveraging the PRV to realistically calculate how the voucher can offset their investment in R&D for NTDs, so the incentive can work its magic. The bill also includes an interesting approach to ensuring products that are developed for NTDs make it to patients that are in need. The approach is to require sponsors to provide a “production report,” specifying manufacturing and marketing plans that indicate “a good faith effort to meet the global demand for the product that is the subject of such report during each of the first 4 years after approval of such product.” Upon failure to demonstrate good faith to get the product to market, this report can be published for all to see.

Among many important changes to the current PRV incentive program, the bill proposes expanding the PRV program to include rare pediatric diseases. This dramatically expands the initial scope of the PRV program, given that it was designed for neglected tropical diseases. We’re still weighing the implications of this expansion, and will have another blog post in the coming days about what this will mean for the program.

We are closely tracking the progress of this bill, and will have much more to say along the way. Stay tuned!

To read a technical summary of the amendment, read the FDA Law Blog.

Rianna Stefanakis is the Manager of Research and Policy at BIO Ventures for Global Health.