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

Abstract

Background Reimbursement policies influence access of patients to orphan drugs in the European countries. Objectives To provide a comprehensive description of orphan drug reimbursement policies and to assess reimbursement decision-making process in the EU-CEE countries as well as the impact of the type of approval and disease on reimbursement decisions. Methods For each drug, the information regarding conditional approval or approval under exceptional circumstances was obtained from the EMA website. The reimbursement status for analyzed drugs was collected in a questionnaire survey performed in a group of experts in reimbursement policy. The agreement between countries was assessed using the κ coefficient, nominal variables tests were compared using the χ2 test or the Fisher exact test. The impact of the EMA’s conditional approval and approval under exceptional circumstances was assessed using logistic regression and presented as an odds ratio (OR). Results The analysis revealed that most orphan drugs were authorized for the treatment of oncological or metabolic diseases [36 drugs (38%) and 22 drugs (23%), respectively]. The shares of reimbursed orphan drugs varied significantly ( p = 0.0031) from 6.3% in Latvia to 27.4% in Poland. No correlation ( r = 0.02; p = 0.9583) with GDP per capita was observed. The highest agreement in reimbursement decisions was observed between Estonia and Lithuania, and the lowest – between Estonia and Latvia, with kappa of 0.69 and 0.11, respectively. Significant impact of the type of approval and reimbursement status was observed for Czechia, Lithuania and Slovakia where conditional approval and exceptional circumstances negatively influenced reimbursement decision. Type of disease has significant influence on reimbursement decision in 4 out of 10 analyzed countries with significant outweigh of positive decisions for oncological diseases. Conclusion In considered countries specific regulations on reimbursement of orphan drugs are valid but in Lithuania and Romania no formal HTA process was employed; in case of some countries higher ICER values for orphans are used. The share of reimbursed orphan drugs varied significantly across the countries, but it was not associated with GDP per capita.

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

“…(41), Turkey 6 (40, 42) 10 . 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 .…”

“…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.…”

“…Poland reimburses 48, the vast majority of which within so-called "Drug Programs" (DPs), introduced by the MoH in 2012 for expensive medical technologies (replacing previous "therapeutical programs") (11,(70)(71)(72). DPs are mainly designed to control consumption of the most expensive drugs 22 .…”

“…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).…”

Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe

“…Total expenditures on the reimbursement of analysed drugs were highly correlated with the total GDP in all . No correlation between GDP and the share of reimbursed orphan drugs was observed for analysed countries [12].…”