Expert Insights

Expert Insights

Pediatric Rare Disease – A Collaborative Approach: FDA Draft Guidance

Pediatric Rare Disease – A Collaborative Approach: FDA Draft Guidance
16 April 2019

The emergence of concomitant trials for multiple investigational drug products for the treatment of rare diseases in pediatrics can pose significant challenges to effective drug development due to the limited number of patients worldwide with any given rare condition. The purpose of the guidance described in “Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry” is to facilitate drug development by discussing a new possible approach to enhance the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example. Gaucher disease is an inherited condition that can affect tissues and organs and commonly manifests in childhood.

This guidance represents the current thinking of the Food and Drug Administration and is a good resource for drug developers as they consider development of pediatric rare disease plans. It is estimated that there are over 7,000 rare diseases and that 30 million Americans are affected, with two-thirds of them being children.

The approach described in the guidance consists of a controlled, multi-arm, multi-company clinical trial that 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 general principles presented should be viewed as a proposal only, although the principles underlying the proposal may be extended to other areas of drug development in rare diseases.

Of note, the specific recommendations regarding drug development for Gaucher disease apply only to systemic (i.e., non-neurological) manifestations of Gaucher disease in treatment-naïve patients with Type I and Type III phenotypes, across all the pediatric ages (i.e., up to 18 years of age).

Modified approaches also 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 can be significant, sponsors are advised to consult the appropriate regulatory agencies prior to initiation of such trials.

Click here for the full draft guidance.

At Synteract, we have expertise in both rare and orphan, and pediatric clinical trials. We have conducted more than 130 trials in rare and orphan disease  across a wide range of therapeutic indications in the past five years alone and over 260 clinical studies in pediatrics over our history.

Contact us for more information on drug development in pediatric and rare diseases.

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