WASHINGTON, DC – In an effort to accelerate research and treatment advances for rare diseases that affect children, U.S. Senators Jack Reed (D-RI) and Shelley Moore Capito (R-WV) teamed up this week to introduce the Innovation in Pediatric Drugs Act (S.4905).  This bipartisan bill 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 of 2024 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.

“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,” said Senator Reed.

“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,” Senator 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.”

According to the National Institutes of Health, a “rare disease” is any disease affecting fewer than 200,000 people in the United States (60 cases per 100,000 individuals).

There are more than 7,000 known rare diseases that affect about 30 million people in the U.S., according to the Orphan Drug Act.  These rare diseases can vary widely, from genetic conditions to certain types of cancers to cases that are the result of an infection or allergy or unknown causes.

More than 90 percent of rare diseases lack a treatment approved by the FDA.  And according to rarediseases.org: Approximately two-thirds of Americans with rare diseases are children.

“Children are not just little adults. It is critical that we understand how drugs work differently in children and prioritize their needs in the drug development process so they do not miss out on new therapies and treatments. The Innovation in Pediatric Drugs Act of 2024 would make needed changes to pediatric drug laws to ensure children can benefit from new advancements in medicine – including children with rare diseases. Pediatricians applaud Senators Jack Reed (D-RI) and Shelley Moore Capito (R-WV) for their leadership on this issue and call on Congress to swiftly pass this bipartisan legislation into law,” said American Academy of Pediatrics President Ben Hoffman, MD, FAAP.

“The biology of cancer children is different from cancer adults. Drugs to treat children must be developed that are tailored for children. The Innovation In Pediatric Drugs Act of 2023 builds upon the promise of the Research To Accelerate Cures and Equity (RACE) Act For Children to increase pediatric studies of novel therapeutics for pediatric cancers. The  bill is an important step to ensure that required studies are completed for children as they are currently done for adults,” said Steve Wosahla, Chief Executive Officer of Children’s Cancer Cause.

“Kids are full of potential and promise. So are the new therapies that we’re currently testing to find the next generation of pediatric treatments. The Innovation in Pediatric Drugs Act would hold trial sponsors accountable and open the door to exciting treatments – so that children can open the door to their futures” said Gwen Nichols, M.D., Chief Medical Officer at The Leukemia & Lymphoma Society.

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.

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

Companion legislation to the Reed-Capito Innovation in Pediatric Drugs Act has been offered in the U.S. House of Representatives by Representatives Anna G. Eshoo (D-CA) and Michael McCaul (R-TX).