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

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 similar to 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

Up-to-date monitoring of childhood cancer long-term survival in Europe: Leukaemias and lymphomas

Background: In recent decades, following the introduction of effective chemotherapy, the prognosis of children with leukaemia and lymphoma has dramatically improved, but data reflecting further possible improvement achieved in the 1990s are scarce. Methods: Using the Automated Childhood Cancer Information (ACCIS) database, we carried out a period analysis of 10-year survival for the 1995-99 period. Analyses were carried out by diagnostic groups, age-group at diagnosis, sex and four European regions. Results: Ten-year survival estimates for the 1995-99 period were 73% for any type of leukaemia, 78% for acute lymphoid leukaemia and 52% for acute non-lymphocytic leukaemia. The corresponding 10-year survival rates for all types of lymphomas, Hodgkin lymphoma, and non-Hodgkin lymphoma were 84, 91 and 79%, respectively. These figures are much higher than those obtained by traditional (cohort-based) methods of survival analysis. A large difference in prognosis is still observed between the East and other parts of Europe. Conclusion: Major improvement in prognosis for children with leukaemia or lymphoma has been ongoing in Europe during the 1990s, but further monitoring and investments are required to remove the large regional differences between European regions