[Congressional Record Volume 170, Number 125 (Wednesday, July 31, 2024)]
[Senate]
[Pages S5685-S5686]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Mr. REED (for himself and Mrs. Capito):
  S. 4905. A bill to amend the Federal Food, Drug, and Cosmetic Act 
with respect to molecularly targeted pediatric cancer investigations, 
and for other purposes; to the Committee on Health, Education, Labor, 
and Pensions.
  Mr. REED. Madam President, today, I am joining Senator Capito to 
introduce the Innovation in Pediatric Drugs Act of 2024 in order to 
improve access to needed therapies for children.
  Children are not just small adults. Drugs affect their developing 
bodies differently, so new treatments need to be studied carefully to 
ensure that they are appropriately prescribed and that dosages are 
properly adjusted. Additionally, drugs that are designed to treat a 
specific condition in adults may have enormous benefits in treating 
completely different illnesses in kids, but research is needed to 
unlock these potentially lifesaving possibilities.
  Unfortunately, drug development still leaves children behind. The 
legislation we are introducing today would help speed therapies to 
children who need them by 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.
  Data resulting from BPCA and PREA studies are added to drug labels to 
give parents and providers essential information on the safety and 
efficacy of drugs used in children. I was proud to have helped author 
these laws when I

[[Page S5686]]

was a member of the Health, Education, Labor, and Pensions Committee. 
While we have made tremendous progress in advancing treatments for 
children because of these laws, there are gaps. For example, there is a 
loophole in PREA that exempts drug companies from pediatric study 
requirements when the treatment would only be used for a rare pediatric 
condition.
  There are close to 7,000 rare diseases without appropriate 
treatments, and the vast majority of these diseases affect children as 
well as adults. But in developing new drugs also known as orphan drugs 
to treat rare diseases, pharmaceutical developers focus their research 
on adult patients only since they are not required study their impact 
on children.
  And since the majority of new drugs approved by the Food and Drug 
Administration, FDA are orphan drugs, this means that the majority of 
newly approved drugs have not been studied for their impacts on kids. 
This leaves doctors, parents, and sick kids in the dark about the best 
possible treatments. Our bill closes this loophole to require studies 
for children so that they, too, can benefit from new and innovative 
treatments for rare diseases.
  In addition to this change, the Innovation in Pediatric Drugs Act 
would invest 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. Our legislation would increase the authorization for 
the BPCA NIH program to ensure we have better data about older drugs to 
treat diseases in children.
  Lastly, 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. Due dates for studies required by 
PREA are typically deferred by FDA until after the approval of the drug 
for adults, but, FDA has no effective enforcement tools to ensure that 
these studies are completed on time--or at all.
  I am pleased to be working with my colleague Senator Capito on 
pediatric health issues. We have worked closely for many years on 
pediatric cancer, first authoring the Childhood Cancer Survivorship, 
Treatment, Access, and Research, STAR Act in 2015. That bill was signed 
into law in 2018, and we worked to fully fund the law every year since.
  I look forward to working with her as well as the sponsors of the 
House companion legislation, Representatives Anna Eshoo and Michael 
McCaul to move the Innovation in Pediatric Drugs Act forward, to give 
children and their families more options for treatments.

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