The joint evaluation examines the strengths and weaknesses of the Regulation on medicinal products for pediatric use and the Regulation on orphan medicinal products. It follows an evidence-based analysis of the functioning of the two regulations and consultation carried out with all relevant interested parties through workshops, as well as public and targeted consultations.
- The evaluation observed some inefficiencies and consequences of the implementation of both regulations.
- The document notes that both regulations fostered the development and availability of medicines for rare diseases and for children; through the introduction of incentives, obligations and rewards, private and public investment was redirected towards previously neglected areas.
- Furthermore, the joint evaluation showed that both regulations have not managed to support development in areas where the need for medicines is greatest; medical products continue to be developed in more profitable therapeutic areas.
- In particular, the evaluation found that:
- medicines for rare diseases have reached a higher number of patients across the EU;
- the number of clinical trials in children increased;
- the ‘off-label’ use of medicines for adults in children was also reduced, which were not tested or adapted for use in children;
The results of the evaluation will guide reflections on any future changes to the current legislative framework and will inform the New Pharmaceutical Strategy.