About this initiative


This joint evaluation covers the legislation on medicines for special purposes, in particular medicines for children and medicines to treat rare diseases. The evaluation will assess to which extent the EU legislation is efficient and effective and considers whether it is fit for purpose in the light of developments in the area of pharmaceuticals. It will look in particular in the impact of the incentives introduced for research, development and marketing, for these specific medicines.

Public health
Type of act
Staff working document


Feedback: closed

Evaluation and Fitness Check Roadmap
More about roadmaps.

Feedback period
11 December 2017 - 08 January 2018 (midnight Brussels time)
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Download (389.1 KB - PDF - 3 pages)

Feedback (23)

  • UNITE2CURE - CCI (France)
    8 January 2018 Non-governmental organisation (NGO)
    UNITE2CURE - CCI (France)

    Cancer is the first cause of death by disease for children over one year of age in Europe. Drug companies have little interest in paediatric cancers, which are perceived as an unattractive commercial market. As a consequence, more that 50% of drugs given to children have never been investigated in this population, but only in adults, and those drugs are administered “off-label”. Both the Paediatric Regulation and the Orphan Regulation have...

  • Ministry of Health, Welfare and Sport, the Netherlands (Netherlands)
    8 January 2018 Public authority
    Ministry of Health, Welfare and Sport, the Netherlands (Netherlands)

    Dutch government feedback on the roadmap for the evaluation of the legislation on medicines for children and rare diseases (medicines for special populations) 8 January 2018 The Ministry of Health, Welfare and Sport of the Netherlands is strongly in favor of a proper and thorough assessment of the regulatory framework to ensure that the EU offers effective incentives that encourage innovation, but also safeguard accessibility, availability and...

  • TEDDY - European Network of Excellence for Paediatric Clinical Research (Italy)
    8 January 2018 Academic/research institution
    TEDDY - European Network of Excellence for Paediatric Clinical Research (Italy)

    This initiative is strongly welcome. Some comments are following reported: Context -Evidence shows that the orphan reward is not as attractive as expected by the various legislations. In the same time, it must be acknowledged that the development of a medicine to treat a rare disease in a paediatric setting could be very difficult (e.g. inborn errors of metabolism affecting children from very early life). -With reference to the special funds...

All feedback (23) >

Public consultation

Feedback: closed
Feedback period
12 October 2018 - 11 January 2019 (midnight Brussels time)
Go to consultation >

Commission adoption


Staff working document
More about adopted acts

Planned for
Third quarter 2019

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