News & Events

FDA In Brief: FDA recommends new, more efficient approach to drug development for rare pediatric diseases

For Immediate Release: Dec. 6, 2017

Media Inquiries

  Andrea Fischer

"The FDA is committed to helping develop drugs for rare diseases – especially rare pediatric diseases where unmet needs exist,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Working with our European regulatory colleagues at the EMA, the FDA has drafted an approach to pediatric rare disease drug development that could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful drug. This draft guidance aims to enhance the efficiency of drug development, while minimizing the number of patients required in the trials. Ultimately, we hope to promote new, creative approaches to drug development for Gaucher and other rare pediatric diseases."

As part of the FDA’s commitment to assisting sponsors and expediting drug development for rare pediatric diseases, today the FDA issued a draft guidance, Pediatric Rare Diseases: Collaborative Approach for Drug Development Using Gaucher Disease as Model. This draft guidance describes a possible new approach for companies to collaborate and test multiple drug products in the same clinical trials, thereby reducing the number of patients necessary to be treated with placebo. Through controlled, multi-arm, multi-company clinical trials, multiple products can be tested in a more time-efficient manner. Specifically, the guidance encourages the extrapolation of available clinical data through modeling and simulation to predict how a drug might work in children and adolescents based on studies conducted in adults and older pediatric populations. It also discusses the possibility of using a single control group as the basis for comparing the safety and effectiveness of more than one investigational drug.

The development of today’s draft guidance started in 2011, with the FDA working collaboratively with the European Medicines Agency (EMA) to gather input from the patient, scientific and drug development communities to develop “Gaucher disease: A strategic collaborative approach from EMA and FDA.” Today, the FDA is releasing this as an FDA guidance using Gaucher disease, a rare pediatric lysosomal storage disorder, as a disease model, but with expectation that the principles underlying this proposal may, in some instances, be appropriate to extended to other rare pediatric diseases.

The FDA welcomes comments on the draft guidance for 60 days and will continue to identify ways the agency can foster greater efficiency in drug development to bring innovative products to patients sooner.

For More Information:

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.


Page Last Updated: 12/06/2017
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
Language Assistance Available: Español | 繁體中文 | Tiếng Việt | 한국어 | Tagalog | Русский | العربية | Kreyòl Ayisyen | Français | Polski | Português | Italiano | Deutsch | 日本語 | فارسی | English