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

Additional file 1 of The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

Additional file 1. Agenda for workshop with PWD and FM

Additional file 2 of The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

Additional file 2. Participants in national working group meeting on diabetes outcomes constructs

Additional file 3 of The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

Additional file 3. The agenda for the national expert working group meeting on value based diabetes care and development of a core diabetes outcome construct set

Additional file 4 of The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

Additional file 4. Survey completed by PWD and FM prior to workshop

The participatory development of a national core set of person-centred diabetes outcome constructs for use in routine diabetes care across healthcare sectors

Abstract Background This study sought to utilise participatory research methods to identify the perspectives of people with diabetes regarding which diabetes outcomes were most important to them. These findings were then used to support an expert working group representing multiple health sectors and healthcare disciplines and people with diabetes to establish a core set of patient-important outcome constructs for use in routine diabetes care. Methods 26 people with diabetes and family members were recruited through purposive sampling to participate in interviews, focus groups, voting and plenary activities in order to be part of identifying outcome constructs. Content and qualitative analysis methods were used with literature reviews to inform a national multi-stakeholder consensus process for a core set of person-centred diabetes outcome constructs to be used in routine diabetes care across health care settings. Results 21 people with diabetes and 5 family members representing type 1 and 2 diabetes and a range of age groups, treatment regimens and disease burden identified the following patient-reported outcome constructs as an important supplement to clinical indicators for outcome assessment in routine diabetes care: self-rated health, psychological well-being, diabetes related emotional distress and quality of life, symptom distress, treatment burden, blood sugar regulation and hypoglycemia burden, confidence in self-management and confidence in access to person-centred care and support. Consensus was reached by a national multi-stakeholder expert group to adopt measures of these constructs as a national core diabetes outcome set for use in routine value-based diabetes care. Conclusions We found that patient-reported outcome (PRO) constructs and clinical indicators are needed in core diabetes outcome sets to evaluate outcomes of diabetes care which reflect key needs and priorities of people with diabetes. The incorporation of patient-reported outcome constructs should be considered complementary to clinical indicators in multi-stakeholder value-based health care strategies. We found participatory research methods were useful in facilitating the identification of a core prioritised set of diabetes outcome constructs for routine value-based diabetes care. The use of our method for involving patients may be useful for similar efforts in other disease areas aimed at defining suitable outcomes of person-centred value-based care. Future research should focus on developing acceptable and psychometrically valid measurement instruments to evaluate these outcome constructs as part of routine diabetes care