Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications. Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs. Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary (p < 0.001). The odds that ODs were reimbursed were reduced in countries with a "strong" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision (p < 0.001) and mandatory (without exception) safety assessments (p=0.004). The probability that ODs were reimbursed was increased in countries with a "moderate" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation (p < 0.001), the presence of the "positive HTA recommendation guarantees reimbursement" policy (p < 0.001), higher GDP per inhabitant (p=0.003), and higher healthcare expenditure (p < 0.001). Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement.

• Keywords
• CEE, HTA, clinical, orphan drugs, policy, rare diseases, reimbursement,
• Publication type
• Journal Article MeSH

In both pediatric and adult populations with type 1 diabetes (T1D), technologies such as continuous subcutaneous insulin infusion (CSII), continuous glucose monitoring (CGM), or sensor-augmented pumps (SAP) can consistently improve glycemic control [measured as glycated hemoglobin (HbA1c) and time in range (TIR)] while reducing the risk of hypoglycemia. Use of technologies can thereby improve quality of life and reduce the burden of diabetes management compared with self-injection of multiple daily insulin doses (MDI). Novel hybrid closed-loop (HCL) systems represent the latest treatment modality for T1D, combining modern glucose sensors and insulin pumps with a linked control algorithm to offer automated insulin delivery in response to blood glucose levels and trends. HCL systems have been associated with increased TIR, improved HbA1c, and fewer hypoglycemic events compared with CSII, SAP, and MDI, thereby potentially improving quality of life for people with diabetes (PwD) while reducing the costs of treating short- and long-term diabetes-related complications. However, many barriers to their use and regional inequalities remain in Central and Eastern Europe (CEE). Published data suggest that access to diabetes technologies is hindered by lack of funding, underdeveloped health technology assessment (HTA) bodies and guidelines, unfamiliarity with novel therapies, and inadequacies in healthcare system capacities. To optimize the use of diabetes technologies in CEE, an international meeting comprising experts in the field of diabetes was held to map the current regional access, to present the current national reimbursement guidelines, and to recommend solutions to overcome uptake barriers. Recommendations included regional and national development of HTA bodies, efficient allocation of resources, and structured education programs for healthcare professionals and PwD. The responsibility of the healthcare community to ensure that all individuals with T1D gain access to modern technologies in a timely and economically responsible manner, thereby improving health outcomes, was emphasized, particularly for interventions that are cost-effective.

• Keywords
• Advanced hybrid closed-loop, Central and Eastern Europe, Hybrid closed-loop, Position statement, Type 1 diabetes,
• Publication type
• Journal Article MeSH