Capito, Reed Introduce Bipartisan Plan to Accelerate Pediatric Rare Disease Research and Treatment Advances

CHARLESTON, W.Va. - U.S. Senators Shelley Moore Capito (R-W.Va.), Ranking Member of the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies (Labor-HHS), and Jack Reed (D-R.I.) recently introduced the Innovation in Pediatric Drugs Act, legislation to accelerate research and advance treatments for rare diseases that affect children.

"As I meet with doctors, researchers, and parents, I am often reminded that while children sadly can get adult diseases, they are not in fact small adults. That's why it is essential medicines be studied specifically for children's use, especially for rare diseases," Ranking Member Capito said. "This legislation will help ensure pediatric studies are actually being done on both new and innovative drugs, as well as those older drugs now off-patent."

"Congress must work together to help address the unmet needs of those affected by rare diseases, particularly children. The Innovation in Pediatric Drugs Act would include children in the drug development process to expand access to safe and effective treatments and therapies for children with rare ailments and appropriately meet their needs. Our bipartisan bill would provide new paths for pediatric rare disease research and development and ensure patients with rare diseases aren't left behind," Senator Reed said.

BACKGROUND:

The bipartisan Innovation in Pediatric Drugs Act would ensure drugs for rare diseases are studied in children and drug companies are accountable for completing pediatric study requirements. It would close research gaps created by the growth in so-called "orphan drug" approvals by the U.S. Food and Drug Administration (FDA).

Children are not just small adults who can take smaller doses of adult medication. They metabolize drugs differently and in order for drugs to be safe and effective for kids, they must be studied specifically for children's use. Yet too often, drug development still leaves children behind. The Innovation in Pediatric Drugs Act would help speed therapies to children who need them, making needed changes to the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA), two laws that encourage and require the study of drugs in children.

The Innovation in Pediatric Drugs Act would help ensure children can benefit from advancements in new treatments and therapies by:

Ensuring Drugs for Rare Diseases are Studied in Children:

There are close to 7,000 rare diseases without appropriate treatments, and the vast majority of these diseases affect children. Unfortunately, in most cases, drugs for rare diseases, also known as orphan drugs, are not required to be studied in children. Yet the majority of new drugs approved are orphan drugs, meaning that the majority of newly approved drugs are exempt from pediatric study requirements, leaving doctors, parents, and sick kids in the dark about possible treatments.

The Innovation in Pediatric Drugs Act would ensure that children with rare diseases can benefit from new and innovative treatments, lifting the orphan drug exemption in PREA.

Providing Equal Accountability for Pediatric Study Requirements:

Due dates for studies required by PREA are typically deferred by FDA until after the approval of the drug for adults. Unfortunately, FDA has no effective enforcement tools to ensure that these studies are completed on time- or at all. Congress tried to solve this problem in 2012. It allowed FDA to send "non-compliance letters" to companies that failed to complete their pediatric studies. Disappointingly, this did not fix the problem. According to an analysis conducted by the American Academy of Pediatrics, as of early 2021, 123 PREA non-compliance letters had been issued, yet only one third had been resolved. On average, studies were 4.4 years late, with some more than 15 years late.

The Innovation in Pediatric Drugs Act would give FDA the authority it needs to ensure that legally required pediatric studies are completed in a timely manner.

Investing in Pediatric Studies of Older Off-Patent Drugs:

The FDA incentives and requirements under BPCA and PREA work for many newer drugs, but unfortunately cannot help encourage studies of older drugs. For this reason, in 2002, Congress authorized a program which funds the National Institutes of Health to conduct studies of off-patent drugs used in children that would never be completed otherwise. Drug studies are expensive and costs have only increased since then, but the program has been flat-funded at $25 million since it was created more than 20 years ago.

The Innovation in Pediatric Drugs Act would increase funding for the BPCA NIH program to ensure we have better data about older drugs to treat diseases in children.

Senators Capito and Reed previously teamed up to pass and fund the landmark Childhood Cancer STAR Act.

Companion legislation to the Capito-Reeds Innovation in Pediatric Drugs Act has been introduced in the U.S. House of Representatives by U.S. Reps. Anna G. Eshoo (D-Calif.) and Michael McCaul (R-Texas).

# # #

Public link

Please use the above public link if you want to share this noodl on another website.