EMA and FDA aim to alleviate burden of paediatric Gaucher trials

The challenges of designing and conducting clinical trials in children suffering from LSD’s, and the attendant burden on their families, are well known.

The EMA and the FDA have recently jointly published a strategy document on drug development research in paediatric Gaucher disease. The key recommendations are:-

  1. That specific paediatric trials no longer be carried out. Instead, data from adult trials and paediatric populations can be modelled to be applied to paediatric patients.
  2. Safety and efficacy be studied in the same trial.
  3. There may be a case for conducting multi-arm, multi-company trials.

These are important and welcome recommendations which, if formalised, will achieve two important outcomes:-

  1. Reducing the need for affected children to participate in clinical trials (and the attendant burden on their families).
  2. Considerably reducing the time and cost involved.

Although the recommendations apply to Gaucher disease, one can see no reason why it should not be possible to include other LSD’s in due course.

Here is the link to the EMA website from which the document can be downloaded.

Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration


Author: Ashok Vellodi

I have had a lifelong interest in lysosomal storage disorders. Having recently retired I now have time to pursue my passion for teaching and training of young doctors and scientists, and hopefully instil in them the passion for their patients that is so integral to working in this field.

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