The rare pediatric disease voucher program creates new treatments. I have new data to prove it

The Food and Drug Administration’s rare pediatric disease priority review voucher program, which has been providing incentives for lifesaving innovations since 2012, is doomed to disappear unless Congress reauthorizes it before the end of September. Some lawmakers have downplayed its impressive track record based on unfounded allegations. I have data showing it works.

Tracy Dixon-Salazar — whose two-year-old daughter, Savannah, started having hundreds of seizures a day in 1995 before being diagnosed with Lennox-Gastaut syndrome at age 5 — has seen the impact of these vouchers. With no FDA-approved treatment effective for Savannah’s condition, the family tried nearly 30 off-label medications in their search for relief. None worked.

But everything changed in 2018 when the FDA approved a treatment for the condition. Like an answered prayer, the therapy gave Savannah precious relief and sent many others into remission. This therapy was incentivized by the rare pediatric disease voucher program.

More than 30 million Americans live with a rare disease; nearly half are children. By the end of September, Congress must vote to continue the program that gave new life to people like Savannah and new hope to the rare disease community. Otherwise, it will sunset.

The economics of developing therapies for rare diseases have always been complicated. Small patient populations make it harder for companies to conduct robust clinical trials. It is also difficult to recover investments because there are relatively few people who need the therapy. To encourage industry not to give up on children living with rare diseases, Congress established the rare pediatric disease priority review voucher program to incentivize investment in new medicines to treat these conditions.

Under the program, a company that develops a therapy for a rare disease primarily affecting children can be awarded a voucher that guarantees priority FDA review of any future drug application. The voucher gives the drug innovator an asset to help offset the significant expenses and challenges associated with developing a therapy for a rare pediatric disease.

These vouchers can be redeemed by the original manufacturer to fast-track FDA review of their next candidate. It can also be sold to another company and redeemed by the purchaser to receive a priority review for a medicine in its pipeline. Priority review means the FDA will take action on a new drug application sooner than it would with a regular application. Bumping up possible approval time by even a few months can mean millions of dollars in sales for a company.

As key congressional committees consider bills this month that could determine the fate of this program, the data about its benefits are compelling. A recent analysis by the National Organization for Rare Disorders (NORD), which I lead, shows that 53 vouchers have been awarded in the program’s first 12 years, resulting in 39 rare diseases gaining treatments to reduce children’s suffering and, in some instances, save their lives. Remarkably, there had been no FDA-approved treatments for 36 of these 39 rare diseases.

Instead of allowing the program to lapse, Congress should be acting swiftly to reauthorize it.

Yet, a handful of lawmakers continue to downplay this impressive track record and focus instead on the theoretical leg-up a pharmaceutical company could receive by selling a voucher or purchasing a voucher from a rare disease innovator.

Some voucher critics cite an outdated Government Accountability Office report that questioned the program’s effectiveness. But this analysis was based on 2019 data collected over the first seven years of the program, when it often takes 12 years or more for drugs in development to progress through the pipeline and earn FDA approval.

Other detractors claim that priority review vouchers risk clogging the FDA pipeline. NORD’s analysis over the program’s full 12-year history shows that only 23 of the 53 rare disease pediatric vouchers issued have been redeemed. That equates to an average of two FDA priority reviews a year, which is certainly within the agency’s capacity to absorb.

Critics also allege that the program provides a mechanism for larger pharmaceutical companies to unfairly jump the line to fast-track approval of blockbuster drugs at the expense of their competitors. Again, this concern doesn’t withstand factual scrutiny. Our analysis found that only three drugs ranked among the top 50 by Medicare drug spend in 2022 received an FDA priority review due to this program.

Another objection is that priority review vouchers are awarded to companies for drugs that would have been developed even if the program did not exist. The fact that 36 of the 39 rare disease patient communities had zero FDA-approved treatments before the voucher program incentives began clearly undercuts this claim. The GAO report itself cites that all seven drug sponsors interviewed stated that these vouchers were a factor in their drug development decisions.

NORD’s updated data should satisfy the holdouts’ concerns. However, a few powerful members of Congress remain unconvinced; their concerns about industry excesses are distorting their ability to recognize the enormous positive impact this program is having on some of the most critically ill children in the United States.

Rather than give up on rare pediatric disease vouchers due to fears of program abuses that have never come to fruition, I urge lawmakers to review the new data and listen to the more than 130 patient advocacy organizations that sent letters to Senate leaders urging swift reauthorization of this essential program.

The FDA needs congressional approval to operate the program beyond September. It would be a devastating gut punch to millions of American families affected by rare diseases if Congress lets this successful program lapse.

In an effort to stave off this possibility, Sens. Bob Casey (D-Penn.) and Markwayne Mullin (R-Okla.) and Reps. Mike McCaul (R-Texas) and Anna Eshoo (D-Cal.) have introduced the Creating Hope Reauthorization Act (H.R. 7384; S. 4583).

Whatever it takes, the successful rare pediatric disease priority review voucher must be extended to give hope — and hopefully effective treatments — to children living with rare diseases.

Pamela K. Gavin is the president and CEO of the National Organization for Rare Disorders.