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More Efficient Rare Pediatric Drug Development: FDA Drafts Guidance

Posted 06 December 2017 | By Zachary Brennan 

More Efficient Rare Pediatric Drug Development: FDA Drafts Guidance

The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could help speed the development of treatments for rare pediatric disease drugs.

Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, said the draft guidance "could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would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."

The 14-page draft uses Gaucher disease as an example for building an approach with a controlled, multi-arm, multi-company clinical trial that minimizes the number of patients necessary to be treated with placebo.

According to Wednesday’s Federal Register notice, the draft guidance was originally developed as a strategic collaboration between FDA and the European Medicines Agency (EMA) to enhance the efficiency of Gaucher disease drug development, which was released in 2014 for public comment. "The draft guidance is an updated version of the document and has no fundamental changes to the original intent and content," the notice said.


Though the general principles in the draft "should be viewed as a proposal only," FDA says, "The principles underlying the proposal may be extended to other areas of drug development in rare diseases."

For instance, it may be necessary to develop, validate and employ age-specific endpoints, the draft notes. And because "the quality of available clinical outcome assessments (COAs) can vary, qualification and standardization is strongly recommended. Developers are encouraged to discuss the selected COA for the outcomes of interest with the FDA; involvement of relevant stakeholders, including patients is encouraged."

The guidance also addresses long-term follow-up, extrapolation from adult studies to demonstrate efficacy, modeling and simulation to optimize the design of studies and inform dosing rationales, and a strategy for designing a multi-arm, multi-company drug development program.

"It includes a description of the main inclusion criteria, relevant age groups, suggested efficacy endpoints, and study duration. Although such a program can be very challenging, the aim of the strategic plan is not only to facilitate agreement on individual applications, but also to address the feasibility of developing multiple drug products for a rare disease in a time-efficient manner," the draft says.

Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry


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