About this consultation

Feedback period
12 October 2018 - 11 January 2019
Public health

Target audience

The consultation is addressed both to individual citizens, i.e. individuals
replying in their personal capacity, and healthcare professionals
treating patients with rare diseases (Including pharmacists and

Why we are consulting

The EU legal framework for medicines for human use is intended to
ensure a high level of public health protection and to promote the
functioning of the internal market, and includes measures that
encourage innovation. Medicines need an authorisation before they can
be marketed in the EU. This can be a national or an EU authorisation.
A rare or orphan disease has been defined in the EU as a disease that
affects no more than five in 10,000 people. A study to support the
evaluation shall also take into account the links between the areas of
orphan and paediatric medicines, as a considerable number of
paediatric diseases also qualify as a rare disease.
This consultation concerns both medicines for rare diseases and
paediatric diseases that qualify as rare. Other medicines treating
diseases that do not qualify as “rare” are out of the scope of this
With this open public consultation, the European Commission asks
private citizens and healthcare professionals to share their experiences
with and perspectives on access to orphan medicines in general, and
on the role the EU Orphan Regulation plays in the development of
orphan medicines.
More information on the legal framework for orphan medicines and on
the EU Orphan Regulation (No 141/2000) in particular can be found on https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A32000R0141
and an inventory of rare diseases can be found on https://www.orpha.net/consor4.01/www/cgi-bin/Disease_Search_List.php?lng=EN

Responding to the questionnaire