A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.

Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies and reimbursement of OMPs in a selection of 12 countries in the Western Eurasian region: Armenia, France, Germany, Kazakhstan, Latvia, The Netherlands, Poland, Romania, Russia, Turkey, Ukraine, and the United Kingdom. A systematic literature review was performed and an analysis of publicly available legislative and rare disease health policy data was undertaken in five focus areas: rare disease definition, newborn screening, registries, national plans, access to/reimbursement of OMPs.

[Policy developments of health technology assessment in the European Union].

The purpose of the note below is to familiarize readers with the current status and future initiatives of cooperation in the field of health technology assessment (HTA) in the European Union (EU). The European Commission is trying to create a framework for efficient collaboration between member states of EU in the field of HTA and is gradually working on strengthening it. The following initiatives, projects and programs have already been implemented: - Project on Coordination and Development of Health Care Technology Assessment in Europe - The European Collaboration for Assessment of Health Interventions and Technology - European Network for Health Technology Assessment - Beneluxa Initiative, The Valletta Declaration, FINOSE and Fair and Affordable Pricing - European Network for Health Technology Assessment Currently there is an ongoing proposal for an HTA Regulation of the European Parliament and the Council, which is the measurable result of this cooperation.

Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs-a Systematic Literature Review. Next Steps in Methodology Development?

Multi-criteria decision analysis (MCDA) is a decision-making tool that can take into account multidimensional factors and enables comparison of (medical) technologies by combining individual criteria into one overall appraisal. The MCDA approach has slowly gained traction within Health Technology Assessment (HTA) and its elements are gradually being incorporated into HTA across Europe. Several groups of scientists have proposed MCDA approaches targeted toward orphan drugs and rare diseases by including criteria specific to rare diseases.