Facilitating development of medicines for autism spectrum disorder

News 04/03/2016

First EMA guideline on evaluation of medicines for autism launched for public consultation

The European Medicines Agency (EMA) has released a PDF iconguideline on the clinical development of medicines for the treatment of autism spectrum disorder (ASD) for a 6-month public consultation.

This is the first guidance document issued by the Committee for Medicinal Products for Human Use (CHMP) for developers of medicines targeting autism. It is based on recent progress in the understanding of the pathological mecha­nisms behind ASD. The guideline also builds on EMA scientific advice for development plans for Autism Spectrum Disorder, as well as the qualification letters of support for the selection of patients in clinical trials.

The draft guideline provides advice on:

  • Diagnosis and inclusion criteria for the selection of patients
  • Methods for assessment of the efficacy of medicines
  • Design of clinical trials
  • Evaluation of clinical safety

Stakeholders are invited to send their comments by 31 August 2016 to cnswpsecretariat@ema.europa.eu using the template provided.

Comments received during the consultation will be taken into account during the finalisation of the guideline.


ASD is a group of disorders which affect the brain. Symptoms typically start to show before the age of 3 years and persist into adulthood. They are described as "spectrum disorders" because they affect each patient in a variety of different ways and can range from very mild to severe. The condition is characterised by difficulties with social interaction and restrictive, excessively repetitive behaviour patterns.

According to the World Health Organization at least 1 in 160 children has an ASD, however poor awareness of the condition and diagnostic methods can make it difficult to identify.

There is no authorised effective medical treatment for the core symptoms of autism. Non-specific treatments can be used to manage problematic behaviour. Management of ASD today relies mostly on behavioural therapies and social and educational programmes.

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