Pediatric Trials for Cancer Therapies With Targets Potentially Relevant to Pediatric Cancers

Area of Science:

• Pediatric Oncology
• Drug Regulation
• Cancer Therapeutics

Background:

• The Research to Accelerate Cures and Equity (RACE) for Children Act was enacted in 2017.
• This act empowers the US Food and Drug Administration (FDA) to mandate pediatric studies for new cancer drugs with targets relevant to pediatric cancers.

Purpose of the Study:

• To evaluate the potential impact of the RACE Act on pediatric study requirements for adult cancer drugs.
• To assess the scope of pediatric labeling for cancer drugs approved between 2007 and 2017.

Main Methods:

• Analysis of 78 adult cancer drugs approved by the FDA from 2007 to 2017.
• Examination of pediatric labeling information for these drugs.
• Application of the FDA's Pediatric Molecular Target List to determine potential eligibility under the RACE Act.

Main Results:

• Only 17 (21.8%) of the examined adult cancer drugs had any pediatric labeling information.
• The RACE Act could potentially subject 78.2% of these drugs to pediatric study requirements, a significant increase from 0% previously.
• The actual proportion of drugs subject to requirements could increase substantially.

Conclusions:

• The RACE Act has the potential to substantially increase the number of cancer drugs requiring pediatric studies.
• The ultimate effectiveness of the legislation hinges on FDA's consistent enforcement of pediatric trial requirements.
• Timely completion of mandated pediatric trials is crucial for realizing the full benefits of the RACE Act.