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

International audienceAbstractRare 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

Volkontinu bij Shell-Pernis 1977

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Cultural Networks and Social Inclusion of Migrants in Europe: Heritage, ICT, and Participatory Governance

The “Digital Heritage: Spotlight on Europe” column examines technological advances internal and external to cultural institutions. The digital shift changed radically how cultural heritage is made, disseminated, distributed, accessed, consumed, and monetized. One of the most important revolutions is that the user's role changed dramatically, shifting from passive observers to active participants and content producers with many new and exciting opportunities for engagement, creative use, and access. The strength of the column is its broad, international focus, and contributors are encouraged to explore issues and recent advances in digital heritage theories, methodologies, standards relevant to the European region, as well as the larger, global audience. This study brings together and critically reflects on the social, policy, and technological contexts within which cultural heritage institutions are addressing current challenges of social inclusion in Europe. It explores the role of cultural networks in helping institutions and their host societies to manage the tensions and realise the opportunities arising from migration. The concept of “migrating heritage” is proposed to tackle this complex interdisciplinary research area