Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program
In a political climate where discourse is often marked by division, there remain crucial issues that bridge partisan divides. The vital importance of treating children living with rare diseases is certainly one such cause. That is why I went to Capitol Hill on July 11 to discuss the impact of the Priority Review Voucher (PRV) program with several U.S. senators and their staff. Without reauthorization, the program will expire on Sept. 30 of this year.
Our bipartisan meetings on Capitol Hill left us hopeful and justified the need for continued and more education on the importance and impact of the PRV program. With the deadline of Sept. 30 quickly approaching, it is clear that work still needs to be done before then, making our visit timely and more appreciated. There is cautious optimism that the program will not lapse, whether through passage of the bill or short-term extension. Still, nothing is guaranteed, as the only thing solidified from our meetings was that we must continue to advocate for the PRV program’s reauthorization and how this impacts rare disease innovation.
The PRV program was reauthorized with bipartisan support in 2016 and 2020. However, it now faces a serious threat of not being approved for a third extension, this time through 2030. If the program is allowed to expire this fall, it will leave fewer incentives for developing new treatments for pediatric rare diseases and it will destabilize investment in ongoing programs. The FDA will no longer be allowed to initiate the process necessary to issue new rare pediatric disease PRVs.
Biotechnology companies can also sell vouchers to generate badly needed capital.
With more than 15 years of experience in the life sciences industry, my passion for rare diseases is unwavering. I am committed to bringing hope to unserved populations. As the senior vice president and chief operating officer at Inozyme Pharma, I am driven to do all I can for patients and support and work with the communities we serve. I believe that the power of many can help improve policy and regulation for rare diseases. My company, Inozyme Pharma, joined the Rare Disease Company Coalition (RDCC) this year to come together with likeminded biopharmaceutical companies to make multiple changes and that includes standing together to advocate for the renewal of PRV.
Lessons Learned From 12 Years of Priority Review
The first PRV program was launched in 2006 and later expanded to include rare pediatric diseases in 2012. This was a crucial step, offering incentives to develop therapies for children living with devastating rare conditions. According to NORD, as of April 2024, 53 PRVs have been awarded across 39 rare pediatric diseases since the program’s inception. Before the program, only three of these diseases had FDA-approved treatments. Many of these rare diseases lead to shortened lives for these children, often before they have the chance to reach adulthood. The impact of the PRV program is essential in providing these children with the opportunity to receive treatment and possibly extend their lives.
In this time, it has become clear that PRVs incentivize and accelerate development of pediatric rare disease drugs. Researchers at the RDCC assembled to communicate the challenges and promise of rare disease drug development to policymakers recently found that the program has helped address urgent unmet needs for 47 rare pediatric indications, benefiting over 200,000 patients so far. More than 90 percent of all PRVs have been awarded to therapies for rare diseases without approved treatments, according to the white paper.
Although termed “rare” due to the small number of individual patients per condition, rare diseases collectively affect 1 in 10 Americans. Of the 10,000 known rare diseases, about 95 percent have no approved treatment, placing a staggering burden on society. Tragically, half of the 30 million patients diagnosed with a rare disease are children, and one-third of these children—around 4.5 million—do not live to celebrate their fifth birthday. Priority review of new therapies is critical to addressing these not-so-rare patients.
But that’s not the only benefit of the PRV program. Biotechnology companies can also sell PRVs to other companies, using the often badly needed capital generated to advance research. The ability to monetize the voucher offers an incentive for investors to finance rare disease companies and provides a novel source of funds for such companies to advance their programs. Ultimately, the purchaser of a PRV can apply it to gain a priority FDA review of its own drug, which can dramatically reduce the time to market and further accelerate getting new drugs to patients in need.
The ability to monetize the voucher offers an incentive for investors to finance rare disease companies.
The need and impact of efficient collaboration with the FDA is clear, and yet, just as the PRV program’s transformative impact is being realized—evidenced by groundbreaking treatments and accelerated drug approvals—it faces imminent termination.
On Capitol Hill, we shared numerous examples of how the PRV program has directly affected our respective companies and directly benefited patients. These ranged from companies purchasing a PRV to bringing a drug to market faster to selling a PRV and reinvesting 100% of the proceeds into R&D, thereby funding additional trials and programs. We also discussed the crucial role of obtaining a PRV in investor discussions, highlighting its significance in shaping a company’s financial future and R&D indication prioritization.
Having witnessed firsthand the transformative impact of PRVs on drug development, I am inspired by the hope they bring to individuals in need of therapies. My involvement in the first drug approval designed to treat children and adults with spinal muscular atrophy (SMA) was a testament to this. SMA, a rare and often fatal genetic disease, affects muscular strength and movement in children and adults. Receiving this PRV increased investment in neuromuscular drug development and helped change the trajectory of the lives of children living with this disease.
My Front-Row View of the Urgent Need
Inozyme Pharma is developing novel therapeutics for rare diseases that impact the vasculature, soft tissues and skeleton. Two of the diseases we are targeting—ENPP1 Deficiency and ABCC6 Deficiency—cause devastating effects in infants and children. Tragically, up to 50 percent of babies born with these diseases do not survive past six months of age.
In my role, I have the privilege of meeting families impacted by these diseases. Despite living with the burdens and loss associated with having a rare disease, their consistent message is one of unyielding hope for advances in science and the development of new therapies. These interactions serve to drive our pursuit of new treatments and emphasize the critical nature of the PRV program.
I know firsthand how essential this work is, as do the leaders of many other biotechnology companies that joined me on the Hill, where we spent time discussing how innovation in the rare disease space comes from cutting-edge companies like those we represented. The PRV program equips our industry with an important tool for getting treatments to those who need them most. In contrast, the potential failure to reauthorize it adds uncertainty to the development pipelines for drugs treating rare pediatric diseases.
I cannot imagine not fighting for every possible program or opportunity that might bring these families closer to relief. I was proud to be a part of meeting with senators last week to encourage them to join us in advocating for the reauthorization of the PRV program. The interest and support of rare diseases we received on Capitol Hill was promising. Our work is not completed as we need to continue to educate congresspeople who are not supportive and continue to showcase the impact and success of this program. The patient advocacy groups’ involvement in telling their stories has been the most powerful. Our collective voice can ensure that innovation and hope continue to thrive for those who need it most.
Together we can convey a clear, urgent message: time is running out. Congress must act now to renew the PRV program, ensuring a future where no child’s potential is diminished by a rare disease.
