Differences and Similarities in the LEVEL of Evidentiary Requirements Required for Orphan Drugs during HTA Appraisal between CEE Countries and the Top Three Leading EU Economies.
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 level of evidentiary requirements required for the health technology assessment (HTA) of orphan medical products (OMPs) in CEE countries and the top three leading economies in the European Union. This study highlights differences and similarities in the clinical aspects of HTA appraisals. METHODS: A comparative analysis of the published regulatory requirements regarding the level of evidence necessary for reimbursement approval was conducted for all CEE countries (Bulgaria, the Czech Republic, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Slovenia, and Slovakia) and Europe’s top three economies (Germany, France, and the UK). This selection is made to compare states with similar economic history (CEE countries, which were part of the former Eastern Bloc) with the leading economic powerhouses of Europe, which often set the example for HTA appraisal. 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: Germany, France, and the UK officially require the same level of evidence for OMPs as therapies for common diseases. However, the three leading economies consider the limited availability of data and data outside of randomized clinical trials (RCTs) may be accepted. More than half of CEE countries have official, more lenient requirements towards OMPs, and almost all accommodate for the data limitations of OMPs. Quality of life may be optional for some of the analyzed countries but is generally viewed favorably both in CEE countries and in Germany, France, and the UK. CONCLUSIONS: The HTA institutions in Europe usually take into consideration, either officially or unofficially, the specific limitations of rare disease data regardless of economic power. However, further formalization may be warranted.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PNS168
Topic
Epidemiology & Public Health, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Public Health, Public Spending & National Health Expenditures, Reimbursement & Access Policy, Systems & Structure
Disease
No Specific Disease