Time is running out for Congress to vote to reauthorize a program that incentivizes pharmaceutical companies to develop and bring to market drugs and therapies for children with rare diseases.

In the U.S., about 30 million people—half of them children—have a rare disease, according to the U.S. Government Accountability Office.

“It is hard to develop drugs for pediatrics, certainly for people with rare diseases,” says Erica Barnes of the Minnesota Rare Disease Advisory Council.

Barnes has been involved in the rare disease field since 2010 when her two-year-old daughter Chloe Barnes was diagnosed with MLD, or metachromatic leukodystrophy, a rare genetic disorder that affects the body’s metabolism and can cause damage to the brain, nervous system, bones, and other tissues.

There were no viable treatments when she was diagnosed, and she died during a bone marrow transplant doctors hoped would at least help her quality of life.

“She was born happy, healthy, nothing detected in newborn screening,” Barnes told 5 EYEWITNESS NEWS. “No concerns from the medical community whatsoever. When we finally were able to get her to the Mayo Clinic, we were told that she had an aggressive neurodegenerative disease.”

Barnes is now helping raise awareness of the need for Congress to reauthorize the “Rare Pediatric Priority Review Voucher (PRV) Program” for five years. If it’s not reauthorized by Dec. 20, the program will expire, and drugs and therapies in the approval pipeline could be in jeopardy.

“The PRV program is essentially an incentive to drive interest and make it viable to bring to market new therapies for diseases that predominantly affect children,” says Jamie Sullivan, a vice president of the EveryLife Foundation for Rare Diseases in Washington, DC.

Sullivan says the PRV program doesn’t require taxpayer funding.

It does make pharmaceutical companies eligible for “vouchers” from the Food and Drug Administration (FDA) that can speed up the timeframe for developing drugs and making them available to patients. She says the PRV program has helped lead to new treatments for diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and progeria syndrome.

“I think that’s the message we want to get across,” Sullivan said in an interview from her Washington, D.C. office. “It is working. There have been over 50 therapies for rare diseases that affect kids approved as a result of the PRV program.”

Barnes knows how important new therapies can be for pediatric patients. Although there were no viable therapies for MLD when her daughter was diagnosed 14 years ago, the situation is changing.

“It’s hard even to imagine, but now there’s a gene therapy on the market for her disease,” Barnes says. In fact, she once met a young boy with MLD who is now thriving as a teenager. “He’s a 16-year-old. He plays soccer. He’s in school. So much of that has come from all of the ways we’ve been supporting the development of these pediatric treatments.”

The U.S. House already approved the reauthorization of the Rare Pediatric Priority Review Voucher Program in September, but the U.S. Senate has until Dec. 20 to do the same. The program for pediatric diseases has been around since 2012 and was last reauthorized for a four-year period in 2020.

A fact sheet from the Every Life Foundation for Rare Diseases can be found below.