Health and Food Safety
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Commission publishes evaluation of the legislation on medicines for children and rare diseases

Today, the Commission published its report on the functioning of the EU Regulations on orphan and paediatric medicines (2000 - 2017). The evaluation found that both Regulations fostered the development and availability of medicines for patients with rare diseases and for children which have increased in recent years. They have also become available faster and have reached a higher number of patients in the Member States. Similarly, the evaluation indicates that the Regulation on medicines for children increased the number of clinical trials in children, which are essential to develop medicines for them. Nevertheless, both Regulations have not adequately managed to support development in all areas of rare and paediatric diseases where the need for medicines is greatest. The evaluation has also shown that authorised medicines for patients with rare diseases and for children are not equally accessible by patients across the EU. As a follow-up, and based on the outcome of the evaluation, the Commission will examine the impacts of possible future policy options to update the legal framework. The first step will be a preliminary impact assessment that the Commission will publish for public feedback this autumn. Any future solution will aim to address any identified shortcomings and foster patient-centred innovation as well as ensuring the availability of, and access to, medicines for patients with rare diseases and for children. These aspects are also key objectives of the Pharmaceutical Strategy for Europe.

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