Assessment of pricing and reimbursement for orphan drugs in Europe is complicated due to the lack of “consensus on the most appropriate assessment criteria, perspective or appraisal process” and the fine line it has to walk between sustainability of healthcare system and augmenting innovation. To improve this process the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) deliberated on this topic and have provided a set of recommendations which is published in Orphanet Journal of Rare Diseases.
Comprising of rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives, the group also sought the opinion of the wider public through a public consultation, debated during the EURORDIS Multi-Stakeholder Symposium and finally discussed it with the MoCA group. This extensive process led to a set of 9 principles, which according to the authors is likely “to stimulate the current debate regarding cooperation between HTA agencies in Europe, and to inspire future research” .
These principles fall into four priority areas of the orphan drug pricing and reimbursement process in Europe which include OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. The final recommendations are summarised below:
- “OMP assessment should consider all relevant elements of product value for OMP’s in an appropriate multi-dimensional framework
- Pricing and reimbursement decisions should be founded on the assessment of OMP value and adjusted to reflect other considerations beyond product value
- Those making P&R decisions about OMPs at a national level should take account of all official regulatory and health technology assessments of OMPs undertaken at the European level
- The assessment and appraisal of OMPs to inform national P&R decisions should incorporate rare disease expertise including both the healthcare professionals’ (HCP) 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 national P&R decision although different decisions on access may apply to different sub-populations
- 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
- In the future there should be greater co-ordination of OMP value assessment processes at a European level 7”