On the 11th August 2020, the European Commission published its joint evaluation on the legislation on medicinal products for paediatric use and on orphan medicinal products. This is the first comprehensive evaluation of the two Regulations since their adoption in 2000 and 2006 respectively. They are evaluated together, given that the majority of rare diseases may appear already in children and many children’s diseases are also rare. The evaluation process consisted of several steps, including the publication of a Roadmap, various recent studies on paediatric medicines and on medicines for orphan diseases and extensive consultation of stakeholders. Moreover, in the context of the revision of the Paediatric and Orphan Regulations, the European Commission (EC) has published an inception impact assessment roadmap. This roadmap is open for feedback until 6 January 2021.
The main problems indicated in the evaluation of the legislation for medicines for rare diseases and children are:
- Insufficient development in areas of greatest unmet medical needs for patients
- Availability and accessibility varies considerably across Member States
- Scientific and technological developments cannot be fully exploited
- Certain procedures are inefficient and burdensome
In order to tackle these issues, the EC is proposing different options for new/different rewards and incentives, linked to unmet medical needs and availability of medicines.
Here it is possible to find more information on the Inception Impact Assessment and on how to provide any feedback.
EPTRI is participating in this consultation by promoting the inclusion of the themes of paediatric research in the EU Agenda.
More information is available at this link.