Rare Disease Report

FDA Recommends New Approach to Orphan Drug Development

DECEMBER 06, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has published its recommendation for a new, more efficient approach to drug development for rare pediatric diseases.

The draft guidance, Pediatric Rare Diseases: Collaborative Approach for Drug Development Using Gaucher Disease as Model was issued this morning to promote the regulatory group‘s commitment to assisting sponsors and expediting drug development for rare pediatric diseases. The proposed amendments walk companies through a new approach for partnering and evaluating multiple drug products in the same clinical trials, thus reducing the number of patients necessary to be treated with placebo. It is believed that through controlled, multi-arm, multi-company clinical trials, several products can be tested in a more time-efficient manner.

"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 in the official FDA press release.

“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 This draft guidance aims to enhance the efficiency of drug development, while minimizing the number of patients required in the trials.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."

To predict how a drug might work in children and adolescents based on studies conducted in adults and older pediatric populations, it is being encouraged that available clinical data be extrapolated through modeling and simulation. Gaucher disease is being used as the model for these changes to the paradigm, and while the rare pediatric lysosomal storage disorder is the focus of the draft guidance, the intent of it is to facilitate and promote exploration for efficient drug development in all uncommon conditions.

The release of this new publication comes on the heels of the House Republicans’ proposal to eliminate the Orphan Drug Tax Credit (ODTC), part of a basket of financial incentives for drug developers in the 1983 Orphan Drug Act. Results from a 2015 study concluded that one-third fewer orphan products would be developed if the tax credit did not exist.

The FDA is accepting comments on the draft guidance until February 5, 2018.

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