The European Multi-Stakeholder Group for Value Assessment and Funding Processes in Rare Diseases, has finalised its Principles on how to improve the consistency of OMP P&R assessments in Europe.
The paper addresses the difficulty of making P&R decisions in rare diseases considering that there has been little consensus between stakeholders including Member States on the most appropriate assessment criteria so far. Under consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients the Group has developed a set of recommendations to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. The agreed Principles are the following:
Principles on OMP decision criteria
- OMP assessment should consider all relevant elements of product value for OMPs in an appropriate multi-dimensional framework
- Pricing and reimbursement decisions should be founded on the assessment of OMP value for money and adjusted to reflect other considerations beyond product value
Principles on OMP decision process
- All official regulatory and health technology assessments of OMPs undertaken at the European level should be acknowledged by national health authorities
- The assessment and appraisal of OMPs in Europe should incorporate rare disease expertise including both the healthcare professionals’ and patients’ perspectives
- To accommodate uncertainty, value assessment and pricing and reimbursement decisions should be adaptive subject to the need and availability of information over time.
- All eligible patients within the authorised label of an OMP should be considered in the reimbursement appraisal although different decisions on access may apply to different sub-populations
Principles on OMP sustainable funding systems
- Funding should be provided at the national level to ensure patient access to OMPs
- Evidence-based funding mechanisms should be developed to guarantee long-term sustainability
Principles on OMP European co-ordination
- In the future there should be greater co-ordination of OMP value assessment processes at a European level