A Father's Perspective On The Rare Pediatric Disease PRV Program
By Stuart Siedman, VP Patient Advocacy, Chiesi Global Rare Diseases
The FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program — it’s a mouthful. And, if we don’t act now, it will sunset at the end of the year. I care. Why should you?
Rare diseases impact small populations with complex and variable symptoms. Half of people diagnosed with a rare disease are children and many do not survive beyond their early childhood. Less than 5% of the 10,000 rare diseases identified to date have any treatment available. Lack of treatment for a rare disease is associated with an increased cost burden on society — 21% increase in total costs per patient per year. The overall cost of rare diseases is about 10 times higher per patient per year than for patients with a high prevalence disease. These are costs that affected families and society must bear.
I know these challenges all too well. My middle son, Benjamin, had the rare disease Sanfilippo Syndrome and died at age 17. His diagnosis set our family on a 25+ year journey that started with the creation of a nonprofit focused on finding a treatment. We sponsored research and ultimately helped launch a company to enable two gene therapy clinical trials. This was not in time for Benjamin and there is still no treatment for our community, but one of those programs has been picked up by another company and is on the cusp (hopefully) of approval. That would not have been possible without the promise of the PRV.
PRVs Provide A Crucial Incentive
The PRV program provides incentives for innovation. When a new drug is approved for a rare pediatric disease, the sponsor qualifies for a voucher that can be used to speed the review for a different product or sold to another company enabling the seller to reinvest in other rare disease programs. These PRV options help sponsors lower the risk portion of the risk/benefit calculation of future development programs.
The PRV program is a success. Since 2012, it has led to new innovations that benefit over 200,000 rare disease patients and address unmet medical needs across 47 rare pediatric indications. Importantly, more than 90% of all vouchers were awarded to therapies for indications with no approved treatment available. We’re talking about “first-in-class” and “best-in-class” treatments for rare diseases that would not have been possible without this program. And more than half of the awarded vouchers were granted in just the last four years alone.
If the PRV program is eliminated, investment in rare disease research and development would fall precipitously, leading to higher healthcare costs and, most importantly, denying children with rare diseases who are still waiting for a treatment. Children do not have the time to wait. We must act now to reauthorize the rare pediatric disease PRV program.
About The Author:
Stuart Siedman is Vice President of Patient Advocacy at Chiesi Global Rare Diseases, where he is responsible for advocacy strategy and community engagement across the Chiesi rare disease portfolio. He is committed to empowering those impacted by rare diseases by building trusted partnerships with them and their communities. Stuart has over 20 years of global advocacy experience, including roles at Sanofi Genzyme and Bioverativ Therapeutics. He is the Cofounder and Director of the Sanfilippo Research Foundation, a nonprofit dedicated to research in Sanfilippo syndrome/MPS III, an ultra-rare inherited lysosomal storage disorder that affected his son Benjamin.