As for the newly published strategic collaborative approach, the EMA and FDA discuss possible ways to enhance the efficiency of medicine development in Gaucher disease, a rare lysosomal storage disorder.

Gaucher disease is being used as a disease model, the document notes, clarifying that the principles in the document may be extended to other areas of drug development in rare diseases. In addition, different approaches may be proposed and the applicant should justify the specific choice of each new strategy, the document states.

“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,” it adds.

The document deals with general considerations for study population, and practicalities in the design and execution of pediatric trials of drugs for Gaucher disease, and covers the use of extrapolation of efficacy data for the disease.

It also proposes a multi-arm, multi-company trial for non-neurological manifestations of Gaucher disease. This proposal covers issues such as study design features; study population and subset definition; number of study participants by pediatric subset (e.g., age, sex, severity or stage); main inclusion criteria; main exclusion criteria; study duration for participants; dosage, treatment regimen, and route of administration; controls; endpoints with times of assessment; statistical plan (SAP) including study conduct and analysis; measures to minimize pain and distress; and external independent data safety monitoring boards.