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

Abstract

Background The reimbursement of orphan drugs (OD) is an increasingly important for country policymakers, and still insufficiently understood, especially in Central and Eastern Europe. The aim of this research was to provide a comprehensive description of country-specific health technology assessment (HTA) policies as well as evaluate the percentage of HTA recommendations and reimbursement decisions for oncology OD. In addition, the study was designed to elucidate the impact of reimbursement of these drugs on the public budget and the agreement between HTA recommendations and reimbursement decisions in the analysed countries. A questionnaire survey was used to collect data on the reimbursement status, HTA recommendation, marketing authorisation, and public expenses on reimbursement in 2014, 2015, and 2016 for all oncology drugs with an orphan designation by the European Medicine Agency in 2017 in Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, and Slovakia. The agreement between the HTA recommendation and reimbursement status was assessed using the kappa coefficient. The Pearson’s correlation was used to analyse the relationship between gross domestic product (GDP) and GDP per capita and reimbursement expenses. Results A total of 36 drugs were analysed (25% conditionally approved; 5.56% approved under exceptional circumstances). The share of reimbursed drugs ranged from 11.11% in Latvia to 41.67% in Poland. The highest share of positive recommendations was observed for Bulgaria and Estonia (36.11%), and the lowest, for Latvia (11.11%). The agreement varied from 0.4 for Poland to 1 for Latvia, Hungary, and Slovakia. Expenses were correlated with GDP (0.95 [0.81–0.99]), and not with GDP per capita (0.54 [− 0.136 to 0.873]). Expenses per capita were not correlated with GDP per capita (0.52 [− 0.15 to 0.87]). Conclusions In Hungary, Latvia, and Slovakia, a positive recommendation was associated with a reimbursement, and a negative one, with the lack of reimbursement. The reimbursement of oncology OD is associated with a growing burden for public budget, and the expenses are correlated with the total GDP. The highest share of drugs with any recommendation was observed in Poland, and the lowest, in Latvia and Romania. The share of reimbursed drugs was the lowest in Latvia and the highest in Poland.

Enhancing Choices Regarding the Administration of Insulin Among Patients With Diabetes Requiring Insulin Across Countries and Implications for Future Care

“…Such activities and growth rates in prices and expenditures for new medicines for oncology and orphan diseases are difficult to sustain under opportunity cost considerations within universal healthcare systems (Barrett et al, 2006;Godman et al, 2021g), necessitating a closer look at the most effective use of current resources. This is imperative with publications advocating greater spending on new medicines for cancer and orphan diseases in CEE countries despite limited health gain for most new cancer medicines (Cohen, 2017;Tomić et al, 2018;Cufer et al, 2020;Malinowski et al, 2020). The situation is different among LMICs, including sub-Saharan Africa, where in the case of patients with diabetes, key initial considerations include early diagnosis as well as availability and access to standard insulins (Godman et al, 2021b).…”

Hybrid Closed-Loop Systems for the Treatment of Type 1 Diabetes: A Collaborative, Expert Group Position Statement for Clinical Use in Central and Eastern Europe

“…While lack of funding represents the major problem in the region, nascent HTA agencies are also a hindrance in some Central and Eastern European countries, such as Romania and Ukraine-these agencies have less health economic expertise to draw from than their Western European counterparts, which have long-running and experienced HTA bodies in place [such as the National Institute for Health and Care Excellence (NICE) in England, the Haute Autorite ´de sante ´(HAS) in France, and the Institut fu ¨r Qualita ¨t und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) in Germany] [ [69][70][71][72]. Outdated and non-evidence-based eligibility criteria for novel therapies can prevent PwD receiving new, efficacious technologies, particularly if funding is instead diverted to medications at high prices with limited health gains [73][74][75][76]. It has been suggested that HTA implementation in the Central and Eastern European region be particularly resource-conscious, with transferability of evidence (e.g., clinical evidence, HTA methodology, and policy) between countries in the region emphasized wherever possible [77].…”

How can health technology assessment be improved to optimise access to medicines? Results from a Delphi study in Europe

“…Even though better access to medicines might be a shared goal amongst healthcare stakeholders, its achievement has proven complicated. In Europe, a plethora of evidence showcases variability in access to medicines across countries [3][4][5][6][7][8][9][10][11]. These variations can be attributed to a variety of factors: some are associated with broaderlevel features such as (i) the general country characteristics, including gross domestic product (GDP) per capita and the epidemiological profile; and (ii) the country's healthcare system characteristics, including healthcare expenditure, organisation of the healthcare system and clinical practices.…”