Medicines for children and rare diseases

The European Commission has launched an open public consultation on the revision of the legislation on Medicines for children and rare diseases.

This is an important step in the process of assessing the impact of possible amendments to EU rules for these medical areas. Recent evaluation has shown that the regulations have stimulated research and development of medicines to treat rare diseases and of medicines for children. However, while 95% of rare diseases still have no treatment option, the evaluation also revealed shortcomings in the current system concerning the development of medicines in areas of high unmet need for patients and their accessibility to all EU patients across the Member States.

Interested parties, in particular health professionals, patients, doctors, academia, researchers, the pharmaceutical industry and citizens are invited to share their views via a questionnaire, until 30 July, to help explore several options in view of the revision of the legislation. A link to take part in the consultation can be found here:

Please do let us know ( if you would like to be part of a combined response from the respiratory community.