The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Monday released a new strategy document to aid in the development of new treatments for Gaucher disease, a rare lysosomal storage disorder.

Given the limited number of patients worldwide with Gaucher disease, EMA and FDA put forth two possible approaches to better facilitate investigations of such products:

• Extrapolation across age groups within a single drug development program, including through modeling and simulation techniques, to better predict how a medicine may work in children and adolescents based on studies conducted in adults or other pediatric populations
• A multi-arm, multi-company clinical trial, which addresses the feasibility of developing multiple drug products in a time-efficient manner and would mean the same control arm is used to compare more than one medicine under evaluation thereby reducing the total number of children included in trials.

The principles underlying the strategy document, which the regulators say applies only to systemic (i.e., non-neurological) manifestations of Gaucher disease in treatment-naïve patients with Type I and III phenotypes, across all the pediatric ages, may be extended to other areas of drug development in rare diseases. The regulators say that drug developers hoping to apply these new approaches are advised to seek scientific advice and can approach EMA or FDA separately, or request parallel scientific advice.

"Due to differences in the regulatory requirements of both Europe and the United States, particularly regarding extrapolation of efficacy from adults to children, additional trials may be required to support an application for approval," the document says.

In addition, EMA said it is finalizing a reflection paper outlining a systematic approach to scientifically sound and reliable extrapolation of data to support medicine authorization. The paper is due to be published in the fourth quarter of 2017 and EMA said it will complement the approach published Monday.

Joint proposal to promote the use of innovative approaches in the development of medicines for Gaucher disease