Comparative Analysis of Legislative Requirements About Patients' Access to Biotechnological Drugs for Rare Diseases in Central and Eastern European Countries

Abstract

Objectives: The aim of the study was to compare the access of patients with rare diseases (RDs) to biotechnological drugs in several Central and Eastern European countries (CEECs). We focused on the legislative pricing and reimbursement requirements, availability of biotechnological orphan medicinal products (BOMPs) for RDs, and reimbursement expenditures.Methods: A questionnaire-based survey was conducted among experts from 10 CEECs: Bulgaria, Croatia, Estonia, Greece, Hungary, Poland, Romania, Slovakia, Serbia, and Macedonia. The legal requirements for reimbursement and pricing of BOMPs were collected. All BOMPs and medicines without prior orphan designations were extracted from the European list of orphan medicinal products, 2017. The reimbursement status of these medicinal products in 2017 in the public coverage of the included CEECs as well as the share of their costs in relation to the total public pharmaceutical spending for the period from 2014 to 2016 were defined.Results: Our survey revealed that some differences in the legal requirements for pricing and reimbursement of BOMPs amongst the countries included in the study. All European Union countries have developed and implemented pharmacoeconomic guidelines with or without some specific reimbursement requirements for orphan medicinal products. Cost-effectiveness analysis, cost-utility analysis, Markov models, meta-analysis, and discount levels of costs and results were required only in Bulgaria, Poland and Hungary. The number of reimbursed BOMPs and biotechnological medicinal products for RDs without prior orphan designation was the highest in Hungary (17 and 40, respectively). Patient-based reimbursement schemes were available only in Hungary for 11 out of 17 BOMPs. Poland and Greece have the highest pharmaceutical expenditure of reimbursed BOMPs with are ~214 million and 180 million EUR, respectively in the observed period from 2014 to 2016. High proportion of the pharmaceutical expenditure on the reimbursed biotechnological medicinal products for RDs for the observed period 2014–2016 is presented in Bulgaria and Slovakia.Conclusions: The non-European Union CEECs face a significant delay in the legal implementation of pharmacoeconomic guideline for assessment of BOMPs. The access to BOMPs is similar among the observed CEECs and the countries with the best access are Hungary and Greece. The influence of BOMP expenditures on the budget in the individual countries is significant.

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

“…Armenia 4 , Kazakhstan 11 (43), Latvia and Romania 12 (44,45), only screen for phenylketonuria and congenital hypothyroidism. England, Scotland, and Wales screen for nine diseases, whereas Northern Ireland (part of the UK as well) screens only for 5 (29) 13,14 . In several countries the number of screened diseases is being expanded or planned to 10 https://dosyaism.saglik.gov.tr/Eklenti/11173,259822214447pdf.pdf?0 (accessed be expanded, notably in Turkey (going from 6 to 10 screened diseases) 11 and the Netherlands (from 20 to 32) (37), but without specific timelines.…”

“…Rare disease policies are a high focus area, given the medical need surrounding rare diseases and the relatively large impact these diseases and their treatment potentially have on healthcare budgets. The reimbursement status of orphan drugs in Eastern Europe has been described by several authors recently (11)(12)(13)(14)(15). There have been multiple publications describing OMP policies in Central and Eastern Europe in single countries (16)(17)(18) or covering a larger number of countries in Europe (5,(19)(20)(21)(22).…”

Reimbursement Legislations and Decision Making for Orphan Drugs in Central and Eastern European Countries

“…Kamusheva et al (2018) provided a comprehensive description of access of patients with rare diseases to biotechnological drugs in several CEE countries in 2018, showing that special legislation for orphan drugs was implemented in several CEE countries. The share of accessible orphan drugs as well as total expenditures varied across countries, being the highest in Greece and the lowest in Romania.…”

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries

“…Kamusheva et al (2018) compared the access of patients with rare diseases to biotechnological drugs between several CEE countries in 2018, reporting that all these countries implemented special legislation for orphan drugs. The share of accessible orphan drugs as well as total expenditures varied across countries, being the highest in Greece and the lowest in Romania.…”