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Public Health

Impact assessment – EU legislation on medicines for rare diseases and paediatric medicines


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Directorate-General for Health and Food Safety


Public health community

Zweck der Konsultation

Based on the outcome of the evaluation of the medicines for rare diseases and children legislation, the Commission is currently analysing the impacts of future policy options to update the legal framework on medicines for rare diseases and paediatric medicines.

The revision of the two legislations is also one of the actions stated in the EU Pharmaceutical Strategy.


The European Commission published a roadmap in November 2020 on the revision of the orphan and paediatric legislation. This roadmap presented the main problems referred in the evaluation for these medicines for special populations, including an outline of the policy options to be considered.

Stakeholders were invited to submit comments on the roadmap until 6 January 2021. In total, 112 replies were received: 24 from business associations, 30 from companies, 8 from public authorities, 29 from NGOs, 5 from academic/research institutions, 7 from EU citizens and 9 from non-EU citizens. Feedback received on the roadmap.

Consultation activity

Stakeholder consultation is one of the key sources of evidence used to support an impact assessment for revision. The aim is to collect views and opinions on the different policy options presented, to gather factual information on what works well and where there is still room for improvement. It also serves to gather data and knowledge about the impact of the options.

An online public consultation on the impact assessment of the orphan and paediatric legislation was launched on 7 May 2021 and ran until 30 July 2021. The online public consultation was conducted via a survey consisting of open and closed questions, which allowed citizens and stakeholders to give their views on the main obstacles they are facing concerning treatments for rare diseases and children. Citizens were also asked about possible ways to overcome these obstacles and on how to make the current legislation future-proof. A total of 304 responses was received. Outcome of the consultation.

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