Innovative strategy announced by EMA and FDA in the development of medicines for rare diseases in children

On 3rd July 2017 in a joint proposal, the European Medicines Agency (EMA) and US Food and Drugs Administration (FDA) launched a new strategy aimed at encouraging innovative approaches in the development of medicines for rare diseases in children.

In this innovative strategy, the EMA and FDA have discussed potential pathways that may enhance the efficiency of the drug development process. The published document discusses Gaucher disease as an example, which is a rare lysosomal storage disorder. It uses this disease to demonstrate how recent developments in data extrapolation can be used.

This approach can equally be applied more generally to the development of medicines for rare conditions in children. It has been developed with the extensive involvement of various groups of stakeholders, including patients and healthcare professionals.

The strategy document suggests that when developing paediatric medicines for rare diseases, companies should consider including:

  • Extrapolation of available clinical data, including using techniques that can simulate/predict how a medicine may work in children and adolescents by using data collected from adults or other paediatric populations
  • The potential to undertake multi-arm, multi-company clinical trials to ensure the safety and efficacy of medicines. This would enable a single control arm to be used to evaluate more than one medicine in a single trial – reducing the total number of children required for each trial.

This collaborative approach aims to reduce the patient numbers required for clinical trials, while ensuring that drug development maintains the highest possible standards.

Seeking scientific advice (from the EMA, FDA or both in parallel,) is recommended to any medicine developers considering using this innovative strategy.

A reflection paper is due to be published by the EMA later this year, to complement this document. The FDA is also expected to publish this strategy in another format in due course.

The full article can be found by clicking here.