Orphan and paediatric medicines share very similar challenges; however, some overlaps currently exist between the Regulation for medicines for rare diseases and the Regulation for medicines for children.
EPTRI is participating in the EC Consultation on the revision of the Paediatric and Orphan Regulations (Ref. Inception Impact Assessment Ares (2020)7081640 – 25/11/2020) to promote more paediatric and orphan medicines and the inclusion of the themes of paediatric research in the EU Agenda. EPTRI supports the implementation of joint solutions to harmonise and optimise procedures, incentives and rewards addressed to paediatric and orphan medicinal products.
Here below are some of the main points.
- A public paediatric research plan should be developed as integral partof the ‘first Strategic Plan for Horizon Europe’, building on the most relevant EU funded paediatric and paediatric relevant research initiatives and leading to the set up of a European Paediatric Medicines Partnership.
- Paediatric research activities should be included in the EU Strategic Plan for Horizon Europe, by identifying paediatric topics to be funded and allocating dedicated funds for paediatric research within topics of general interest. Priority should include unmet paediatric needs and orphan paediatric indications.
- Research on ethical and legal issues and social impactof both paediatric and rare diseases also in a children/fundamental rights perspective should be promoted and funded to comply with the EU recognised principles and.
- Unmet therapeutic needs specific to children of different ages should be identified, by revising the “Therapeutic Needs” Lists prepared by the EMA-PDCO and engaging experts from paediatric clinical and nonclinical research initiatives (e.g. c4c, ERNs, EJP-RD, EnprEMA Members EPTRI, EUPFI, etc.) with the active participation of patients/children organisations.
- PRIME scheme adoption should be expanded once adapted to paediatric specificities.
- The current system of rewards and incentives should be implemented and harmonised in case of paediatric & orphan medicines.
- Technological and scientific advances should be embraced by adopting criteria based on mechanism of action instead of disease classification (also leading to revise waivers criteria).
- Effective and efficient proceduresshould be provided by accelerating and simplifying the PIP applications and by revising waivers and deferrals criteria.
- The implementation of regulatory-compliant innovative studies’ methodologies(Bayesan, adaptive design, RWD, etc) should be supported to develop paediatric and orphan medicines.