Differences and Similarities in the Principles Involved in the HTA Appraisal of Orphan Drugs in CEE Countries
Author(s)
Djambazov S1, Filipov A1, Dacheva A1, Vutova Y1, Vekov T2
1HTA Ltd., Sofia, 23, Bulgaria, 2Medical University Pleven, Pleven, Bulgaria
OBJECTIVES: The objective of this study is to evaluate the principles involved in the health technology assessment (HTA) of orphan medical products (OMPs) in CEE countries METHODS: A comparative analysis of some of the various aspects of the reimbursement process for OMPs was conducted for all CEE countries (Bulgaria, the Czech Republic, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Slovenia, and Slovakia). The assessment was based on key features: (1) are rare diseases officially distinguished from other diseases, (2) do separate rare disease institutions responsible for the approval process exist, (3) does OMP reimbursement differ, and (4) do CEE countries rely on external referencing for OMP appraisal. The choice of countries is based on the similar economic history of the listed European Union states (former Eastern Bloc members). Bibliographic databases, online sources, and government/HTA organization websites were searched. The collected data was synthesized in Excel and used for qualitative comparative analyses. RESULTS: Half of CEE countries recognize rare diseases as a distinct category with Bulgaria, Romania, Estonia, Latvia, and Lithuania using EMA orphan designation. Slovakia utilizes a separate definition. Slovakia and Lithuania also recognize ultra-rare diseases. Latvia and Lithuania utilize specialized rare disease institutions in the appraisal process, which allows for case-based individual reimbursement. Slovakia also allows for individual reimbursement. Bulgaria, Romania, the Czech Republic, Poland, and Estonia apply the same conditions for OMP reimbursement as those for regular drug reimbursement (although the Czech Republic permits temporary reimbursement). Hungary, Latvia, and Estonia have special reimbursement processes for OMPs. External referencing is used in all countries apart from Hungary, Poland, and Estonia. CONCLUSIONS: Most CEE countries recognize officially rare diseases and allow for some leniency in the application process. However, few have dedicated institutions or offer alternative reimbursement processes.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PNS171
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Reimbursement & Access Policy, Systems & Structure, Thresholds & Opportunity Cost, Value of Information
Disease
No Specific Disease