FDA releases draft guidance to facilitate drug development in paediatric rare diseases

Draft guidance has been released by the US Food and Drug Administration (FDA) which could help to accelerate the development of drugs for treating paediatric rare diseases. The draft guidance could eradicate the need for certain clinical studies and, when paediatric clinical studies are required, could decrease the total number of patients who would receive a placebo rather than a potentially beneficial drug.

Using Gaucher disease as an example, the guidance discusses a new possible approach to enhance the efficacy of drug development in paediatric rare diseases. The new approach consists of a controlled, multi-arm, multi-company clinical trial, which aims to promote the development of multiple drug products in a time-efficient manner.

The principles presented in the guidance should be viewed as a proposal only and it is possible that these may be drawn out to other areas of drug development in rare diseases.

The guidance states that it may be necessary to develop, validate and establish age-specific endpoints. The draft also notes that because “”the quality of available clinical outcome assessments (COAs) can vary, qualification and standardisation 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.”

Also addressed in the draft guidance is long-term follow-up, extrapolation from adult studies to demonstrate efficacy, modelling and simulation to optimise the design of studies and inform rationales, and a method for designing a multi-arm, multi-company drug development program.

To read the draft guidance for paediatric rare diseases, please click here.