Podcast
MSL Talk: Tom Caravela, Sarah Snyder
Pediatric Trials for Cancer Therapies With Targets Potentially Relevant to Pediatric Cancers
Summary
- The Research to Accelerate Cures and Equity (RACE) for Children Act was enacted in 2017.
- The Act authorizes the US FDA to require pediatric studies for new cancer drugs with molecular targets relevant to pediatric cancers.
- An examination of 78 adult cancer drugs approved by the FDA from 2007 to 2017 showed that only 17 (21.8%) had any pediatric labeling information.
- Based on the FDA's Pediatric Molecular Target List, the RACE Act could have potentially increased the proportion of cancer drugs subject to pediatric study requirements from 0% to 78.2%.
- The actual effect of the legislation depends on the frequency of pediatric trial requirements and the timely completion of these trials.
In 2017, Congress enacted the Research to Accelerate Cures and Equity (RACE) for Children Act to stimulate the development of targeted therapies for pediatric cancers (1). The RACE Act closed a legislative gap that had exempted cancer drugs from mandatory pediatric studies.
Specifically, the RACE Act amended the Pediatric Research Equity Act (PREA), which ordinarily authorizes the US Food and Drug Administration (FDA) to require pediatric studies for new drugs, indications, dosage forms, and routes of administration in all relevant pediatric subpopulations (2–4). For sponsors that do not comply with PREA requirements, the FDA can determine that a drug is “misbranded” (5); the FDA can also publicly post noncompliance letters for overdue PREA studies...
antineoplastic agents, adult, child, pediatrics, continental population groups, united states food and drug administration, statutes and laws, cancer therapy, childhood cancer, molecular target
Related Topics
You May Also Like
MSL Talk: Tom Caravela, Sarah Snyder
Article
Rajat Thawani, Neha Agrawal, Nicholas F Taflin, Adel Kardosh, Emerson Y Chen
Application of Value Framework to Phase III Trials of Immune Checkpoint Inhibitors in Esophageal and Gastric Cancer
Article
Meena N Murugappan, PharmD, MPH, PhD(c), Bellinda L King-Kallimanis, PhD, Gregory H Reaman, MD, Vishal Bhatnagar, MD, Erica G Horodniceanu, MPH, Najat Bouchkouj, MD, Paul G Kluetz, MD
Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective
Article
S.F. Oosting, J. Barriuso, A. Bottomley, M. Galotti, B. Gyawali, B. Kiesewetter, N.J. Latino, F. Martinelli, M. Pe, G. Pentheroudakis, F. Roitberg, H. Vachon, E.G.E. de Vries, M. Piccart, N.I. Cherny
Methodological and reporting standards for quality-of-life data eligible for European Society for Medical Oncology-Magnitude of Clinical Benefit Scale
Article
Dylan V Neel, David S Shulman, Clement Ma, Florence Bourgeois, Steven G DuBois
Sponsorship of oncology clinical trials in the United States according to age of eligibility
Article
Michael Z. Liao, Dan Lu, Matts Kågedal, Dale Miles, Divya Samineni, Stephanie N. Liu, Chunze Li