EC launches public consultation on medicines for children and rare diseases

Last updated: 2 December 2022

The European Commission (EC) has launched a public consultation on the revision of the legislation on medicines for children and rare diseases.

What does the consultation hope to achieve?

The consultation is an important step in the process of assessing the impact of possible amendments to EU rules for these medical areas which builds on the recent evaluation published in summer 2020. The main report without the annexes is available here.

This initiative will explore several options to address the shortcomings identified in the evaluation of the Regulations on medicines for children and rare diseases, in view of a revision of the existing legislation.

What did the evaluation published in 2020 show?

The evaluation showed that the Orphan medicinal products Regulation (EC) No 141/2000 and the paediatric Regulation (EC) No 1901/2006 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 in particular the development of medicines in areas of high unmet need for patients and their accessibility to all EU patients across the Member States.

Who is the consultation aimed at?

All Interested parties, in particular health professionals, patients, doctors, academia, researchers, pharmaceutical industry and citizens are invited to share their views via a questionnaire to help explore several options in view of the revision of the legislation.

Where can you provide feedback on the consultation?

Feedback can be provided via a questionnaire available here.

What were the start and end dates of the consultation?

Start date: 7 May 2021

End date: 30 July 2021

Is there a summary report available from the consultation?

Yes there is. You can download it from this page.

Source: Europa website