FDA Draft on Pediatric Rare Diseases

As reported before, last year the European Medicines Agency (EMA) published a joint proposal together with the U.S. Food and Drug Administration (FDA) entitled "Paediatric Gaucher disease - A strategic collaborative approch from EMA and FDA". The FDA now published this strategy paper in a different format. The title of the draft document is Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.

FDA says the "guidance has been prepared by the Division of Gastroenterology and Inborn Errors Products in the Center for Drug Evaluation and Research at the FDA. This guidance adapts with minor modifications the 2017 update of the 2014 FDA – EMA Collaborative Approach document titled “Gaucher Disease — A Strategic Collaborative Approach From EMA and FDA”.

Due to the limited number of patients worldwide with any given rare condition trials for multiple investigational drug products for the treatment of rare diseases can pose significant challenges to effective drug development. The purpose of this guidance is to facilitate drug development for pediatric rare diseases. In particular, it discusses a new possible approach to enhance the efficiency of drug development in pediatric rare diseases. This new approach consists of a controlled, multi-arm, multi-company clinical trial, which aims to facilitate the development of multiple drug products in a time efficient manner while minimizing the number of patients necessary to be treated with placebo.

The proposed document considers pediatric extrapolation of efficacy from adults to children. This extrapolation can be considered when the course of the disease and the expected response to a drug product would be sufficiently similar in the pediatric and reference population (i.e., adult or other pediatric age population). The use of extrapolation of efficacy can

  • avoid unnecessary studies,
  • increase efficiency,
  • reduce testing burden to patients, and
  • better allocate resources to address relevant questions.

An extrapolation plan could be established early during drug development, with the recognition that the plan may not address all aspects necessary in the development of emerging drug products across all ages of pediatric patients. Hence, additional clinical studies may be necessary for determination of efficacy across all age groups.

FDA states the proposal may be extended to other areas of drug development in rare diseases. Furthermore, modified approaches may be proposed, but the sponsor should justify the specific choice of each new strategy. Given that rare disease drug development tends to require global involvement, and the potential differences in requirements between the FDA and other regulatory agencies, sponsors should consult the appropriate regulatory agency prior to initiation of such trials.

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