Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

Abstract

Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles 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. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP’s is realised.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-017-0601-9) contains supplementary material, which is available to authorized users.

Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

“…However, separate routes, such as ear-marked budgets, heavily rely on political support and risk being revised or discontinued [10], particularly in a time when the proportion of pharmaceutical expenditure is likely to increase for RDTs and other high cost medicines [30].…”

“…These aspects have led to increasing recognition that it may be difficult to subject RDTs to conventional HTA processes, particularly in those diseases that require highly specialised care and that are very rare or for so-called "ultra-OMPs" [3,4,[9][10][11].…”

A proposal for value informed, affordable (“via”) prices for innovative medicines

“…This has also been confirmed in more recent work, such as in Shiroiwa et al 18 and Richardson et al 19 , where the latter suggest that higher willingness to pay is especially relevant for very severe conditions. Value should, therefore, be defined at least by both disease and treatment related characteristics, which should both be objectivized 20,21 .…”

The role of primary care in management of rare diseases in Ireland

“…Most RDs are associated with chronic physical, intellectual or neurological disabilities. Collectively, they pose a significant economic burden [1][2][3]. The greater than 6100 known RDs are estimated to affect 3.5-5.9% of the population (excluding rare cancers and infectious diseases) equating to 263-446 million persons globally [4] and approximately 300,000 Irish people.…”