For some parents, a visit to a doctor is a day forever burned in their memory. Rare pediatric diseases, or those affecting less than 200,000 children, collectively affect 10 million children in the US with only five percent of rare diseases with any FDA-approved treatment. Our country’s collective conscience is at risk with the looming expiration of a key program supporting the search for cures.
Spinal Muscular Atrophy Type 1 (SMA) is one such single gene rare disease. Infants with SMA develop progressive weakness with muscle wasting, difficulty swallowing, and respiratory failure. Many die before their second birthday, leaving families emotionally and spiritually devastated. While newborn testing provides patients with rapid answers, it transforms a day of celebration into a day of sorrow and isolation.
The tide finally turned on this treacherous diagnosis in 2019 when the FDA approved Zolgensma, a novel gene therapy for the treatment of SMA Type 1, administered in a single treatment. This had been preceded by the development of Spinraza, the first treatment developed and approved for SMA Type 1 in 2016 followed by the first oral treatment in 2020. With fewer than 10,000 children living with this disease, both private investment incentives and public efforts devoted to funding research are limited, and finding a treatment is a miracle in itself.
Rare diseases lacking a sizeable population to shoulder development costs are unlikely targets for therapeutic development. Acknowledging this reality, in 2007 Democratic Senator Brown and Republican Senator Brownback introduced and spearheaded legislation to create a priority review voucher (PRV). Expanded to include rare pediatric diseases in 2012, innovators involved in the development of therapies for qualifying rare pediatric conditions are eligible to receive a PRV upon FDA approval.
The voucher reduces the FDA’s standard 10-month review timeline to six months, allowing earlier access to therapies for vulnerable patients, which is especially important with progressive diseases impacting infants and children. Those granted vouchers may hold them for use towards a future drug in development. Alternatively, vouchers can be sold to another company, with a recent sale yielding $110 million, providing innovators the financial resources to continue the costly work of drug development. The PRV program aligns incentives and protects the dignity and lives of very vulnerable children—literally bringing life to those who may lose it, including those with SMA Type 1.
Critics suggest that the evidence for pediatric PRVs resulting in more new drugs being brought into clinical testing is lacking, and therefore the program should be terminated. However, this ignores the findings of a recent Government Accountability Office study in which those drug developers interviewed cited the voucher program as a factor in pursuing a therapeutic development program. Further validation is found in the FDA’s own data collected over a decade of experience: the agency has granted 38 rare disease PRVs since 2014 for diseases ranging from SMA to Rett Syndrome to Duchenne Muscular Dystrophy.
It is important to recognize the fundamental importance of the pediatric PRV program: children are not little adults, nor mere members of a collective vulnerable group. A rare diagnosis and narrow window for treatment mean speed is just as critical as the number of new drugs developed.
Despite current misconceptions, roughly two-thirds of funding and the majority of cures emerge from the private sector. While investors seek a financial return on their investment, the scientists and entrepreneurs that they fund are motivated by both scientific curiosity and a sincere desire to relieve the burden of severely debilitating—and at times terminal—illnesses afflicting children. Rare pediatric diseases often result in exorbitant medical expenses and economic losses, along with extreme stress and a reduced quality of life for parents and families. Policymakers must recognize the urgency of the need and promote pragmatic policy.
The Creating Hope Reauthorization Act, a bipartisan bill to reauthorize the pediatric PRV program is advancing through Congress and we are glad to see this effort gaining the increased attention it deserves. With the program’s expiration set to occur this September, thousands of children face a darker future. Failure to renew the program would mark not only a policy failure but a moral one.
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