On July 11th Dr. Mathew Winton went to Capitol Hill with senators and staff from both parties to discuss collaboration with the FDA regarding the pending termination of the Priority Review Voucher (PVR) program in September.
The doctor can say with certainty that these meetings left him hopeful. Evidence of PVR’s success was presented. The discussions immediately led to FDA drug approvals. PVR’s future success lies in new groundbreaking treatments and accelerated drug approvals. Yet the requisite authorization is scheduled to be terminated in September of this year. There is, however, a slim chance of saving the program.
A History of the Priority Review Voucher Program
The program did have previous bipartisan support for reauthorization in two prior years. If the program expires in September, it will obviously leave only a few incentives to develop treatments for children. Investment in current programs will also be destabilized. The FDA will not be able to start the process enabling it to issue additional vouchers.
The PRV program dates back to 2006. The rare pediatric disease category was added in 2012 providing incentives to developers on behalf of children and families struggling against rare conditions. NORD has provided statistics showing that as of April 2024, 53 PRVs have been awarded for 39 rare pediatric disorders since the inception of the program. Prior to PRV, only three pediatric diseases had been approved and were available. Young lives were lost.
The goal of the PRV program is to incentivize drug development for rare pediatric diseases. When a sponsor is approved for a biological product or drug, it may qualify for one of the PRVs to be redeemed or to receive priority review for a different product. Note that the sponsor may transfer or sell the voucher as well.
Dr. Winton spoke of his commitment to helping the underserved rare disease community. In fact, his company Inozyme joined the Rare Disease Company Coalition recently with others hoping to initiate changes and advocate for PRV renewal.
A Proven Track Record
RDCC researchers recently assembled to discuss the challenges and drug development with policymakers and found the PRV program has addressed unmet needs of more than 200,000 patients and 47 rare pediatric conditions. To be more precise, 90% of PRVs were awarded for rare disease therapies that had no approved treatments.
Out of 10,000 rare diseases that have been described, about 95% lack an approved treatment. This lays a heavy burden on society. Around half of rare disease patients are children. Further, a third of them will not see their fifth birthday.
In addition to the priority reviews, another benefit of the program is that companies can sell their PRVs to other companies. This generates capital for research. Selling, or monetizing the voucher gives an incentive to investors as well. The company that purchases a PRV can use it for a priority review for one of its therapies. This has the dramatic effect of reducing the time it takes to get the drugs to market and to the patients who urgently need the drug.
Dr. Winton and others have expressed their opinion regarding the impact of collaborating with the FDA. Yet just as the program’s benefits are being acknowledged, the program faces termination. Vouchers can also be sold to other developers.
Inozyme is developing therapeutics that have an impact on soft tissues, skeleton, and blood vessels (vasculature). Two such diseases, ABCC6 and ENPP1 Deficiencies are devastating to infants and children. Statistics indicate that up to 50% of individuals who are born with one of these conditions will not survive more than six months.
Dr. Winton has taken a special interest in the development of new therapies after meeting families affected by these diseases. He notes that in so many cases their message is similar. It is hope for scientific advances and new treatments to save these children. The doctor’s message is clear, and it is urgent. Time is short. Congress must act now to renew the PRV program creating a future where no child’s potential is destroyed by a rare disease.
