The US Food and Drug Administration (FDA) has released draft guidance, Pediatric Rare Diseases—A Collaborative Approach for Drug Development Using Gaucher Disease as a Model, to facilitate drug development in pediatric rare diseases.

The draft guidance adapts with minor modifications the 2017 update of the 2014 FDA–European Medicines Agency collaborative approach document, Gaucher Disease A Strategic Collaborative Approach From EMA and FDA. In particular, the draft guidance discusses a new possible approach to enhance the efficiency of drug development in pediatric rare diseases using Gaucher disease as an example.

The document says the general principles presented should be viewed as a proposal only, but that the principles underlying the proposal may be extended to other areas of drug development in rare diseases.

For drug-development programs in pediatric rare diseases, the guidance says it may be necessary to develop, validate, and employ age-specific endpoints. It also says that because the quality of available clinical outcome assessments (COAs) can vary, qualification and standardization is strongly recommended. The guidance continues to say developers are encouraged to discuss the selected COA for the outcomes of interest with the FDA; involvement of relevant stakeholders, including patients is encouraged, says the FDA.

Additionally, the guidance discusses: long-term follow-up to evaluate the long-term safety and efficacy of treatment; the use of pediatric extrapolation of efficacy from adults to children; and 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 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, according to the guidance.

Source: FDA