News Feature | May 16, 2014

EMA And FDA Post Joint Proposal For Investigation Of New Gaucher Drugs

By Estel Grace Masangkay

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) have posted a draft joint proposal to facilitate clinical investigation of new therapeutics for the treatment of Gaucher disease in children.

Gaucher disease is a rare condition in which glucocerebrosides accumulate in certain cells throughout the body. The severity of Gaucher disease varies with a range of symptoms. According to the EMA, there is an urgent medical need for affected children with neurological symptoms, specifically for novel methods of administering treatment that could reduce the treatment burden.

The proposal aims to facilitate agreement of an EMA Pediatric Investigation Plan and FDA Pediatric Study Plan. It will also investigate the feasibility of developing a number of drugs for a rare disease in a shorter timeframe and with fewer patients.

Jordi Llinares Garcia, Head of the EMA’s Product Development Scientific Support Department, said, “The complementary approach proposed would not only facilitate the investigation of new medicines for rare diseases in children but also optimize the number of patients recruited, thereby reducing the burden on children and families.”

The joint proposal offers two complementary approaches. These are outlined as:

  • The extrapolation of efficacy from adults to children which would include the use of modelling and simulation approaches. The EMA and FDA aim to avoid unnecessary studies in children for ethical reasons, to increase efficiency, to reduce the burden on children, and to ensure that resources are assigned to the areas with the most need for studies;
  • The possibility to conduct multi-arm, multi-company clinical trials to determine the efficacy of several treatments at the same time. This approach would reduce the total number of children included in trials, compared to separate controlled trials, as the same control arm would serve more than one medicine under evaluation. A proposed design for such trials is included in the document.

The proposal is open for public consultation until the end of August this year. The two agencies are inviting sponsors to discuss possibility of using the new approach in their drug development plans.