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Pediatric oncology: regulatory initiatives
S Hirschfeld1, A Shapiro, R Dagher
1Division of Oncology Drug Products, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Maryland 20852, USA. hirschfelds@cder.fda.gov
Abstract:
The majority of children with cancer receive therapy as participants in clinical research protocols coordinated by national pediatric cooperative groups. One of the highest priorities of these groups is the development of novel therapies. Due to differences in the biology of pediatric and adult tumors and in physiology between adults and children, it is usually necessary to evaluate the safety and effectiveness of new drugs separately in adult and pediatric populations. To stimulate the development of new therapies for pediatric indications and encourage the submission of clinical data to support pediatric product labeling, the U.S. Food and Drug Administration has undertaken two initiatives. In 1998 the FDA issued a regulation (The 1998 Final Pediatric Rule) that mandated if a drug or biological is under review for a claim and the disease exists in both pediatric and adult populations, pediatric studies must be performed. Section 111 of the FDA Modernization Act of 1997 states that if a drug product has exclusivity based on a patent or marketing license, the exclusivity can be extended by six months for the submission of pediatric data to the FDA. The incentive applies to both approved drugs and those under development. These initiatives are intended to enhance access to new anticancer therapies and promote labeling for pediatric oncology indications.
Insights
The U.S. Food and Drug Administration (FDA) implemented rules to boost pediatric cancer drug development. These initiatives encourage new therapy research and ensure pediatric patients benefit from novel treatments.
Area of Science:
- Pediatric Oncology
- Drug Development
- Regulatory Science
Background:
- Most children with cancer participate in clinical trials coordinated by national pediatric cooperative groups.
- Developing novel therapies is a key priority for these groups.
- Pediatric and adult tumors have distinct biology, necessitating separate drug evaluations.
Purpose of the Study:
- To stimulate the development of new anticancer therapies for pediatric indications.
- To encourage the submission of clinical data supporting pediatric product labeling.
- To enhance access to novel anticancer treatments for children.
Main Methods:
- The U.S. Food and Drug Administration (FDA) issued the 1998 Final Pediatric Rule, mandating pediatric studies for drugs treating conditions in both populations.
- Section 111 of the FDA Modernization Act of 1997 offers a six-month exclusivity extension for submitting pediatric data.
- These initiatives apply to both approved and developing drugs.
Main Results:
- The FDA's initiatives aim to increase the availability of new anticancer drugs for children.
- These efforts are designed to promote the labeling of drugs for pediatric oncology indications.
- The regulations and incentives encourage research and data submission for pediatric cancer therapies.
Conclusions:
- Regulatory actions by the FDA are crucial for advancing pediatric cancer drug development.
- Incentives like extended exclusivity can drive pharmaceutical companies to conduct pediatric studies.
- These measures ultimately improve treatment options and outcomes for children with cancer.
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