Background: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies and reimbursement of OMPs in a selection of 12 countries in the Western Eurasian region: Armenia,

Purpose To develop population norms for the EQ-5D-5L questionnaire based on a representative sample of Moscow citizens. Methods We used quota sampling accounting for sex, age group and administrative district of residence. Respondents in randomly selected outdoor and indoor locations were surveyed with the official Russian paper-and-pencil version of the EQ-5D-5L questionnaire and a set of socio-demographic questions. We estimated four types of EQ-5D results: the distribution of limitations according to EQ-5D-5L dimensions, the perception of the health-related quality of life (HRQoL) with a visual analogue scale (EQ VAS), the unweighted score for a respondent’s health state (Level Sum Score, LSS) and the Russian health preferences-based weighted score (EQ index). In order to estimate the EQ-5D-5L index, we used a newly developed Russian EQ-5D-3L value set, together with EuroQol Group cross-over methodology. Results A total of 1020 respondents (18–93 years old) from the general Moscow adult population completed the EQ-5D-5L questionnaire. HRQoL domains with the largest number of identified health limitations were pain/discomfort (48.6%) and anxiety/depression (44.1%). Two hundred seventy-nine respondents (27.0%) did not report any health restrictions. The mean EQ VAS and EQ-5D-5L index were 74.1 (SD 17.3) and 0.907 (0.106) respectively. Multivariate analysis showed that female sex, advanced age and lack of access to the Internet had a negative influence on HRQoL, whereas residence in certain districts had a positive impact. Conclusions The study provides population norms of health-related quality of life in Moscow, measured according to the EQ-5D-5L questionnaire. These reference values can be used to optimise the effectiveness of resource allocation in healthcare.

Objectives: Methodological challenges in the evaluation of medical devices (MDs) may be different for early and late technology adopter countries, as well as the potential health technology assessment (HTA) solutions to tackle them. This study aims to provide guidance to Central and Eastern European (CEE) countries on how to address key challenges of HTA for MDs with special focus on the transferability of scientific evidence.Methods: As part of the COMED Horizon 2020 project, a comprehensive list of issues related to MD HTA were identified based on a targeted literature review. Health technology assessment issues which pose a greater challenge or require different solutions in late technology adopter countries were selected. Draught recommendations to address these issues were developed and discussed in a focus group. The recommendations were then validated with a wider group of experts, including HTA and reimbursement decision makers from CEE countries in May and June 2020.Results: A consolidated list of 11 recommendations were developed in 3 major areas: (1) clinical value assessment, focusing on the use of joint EU work, relying on real-world evidence, use of coverage with evidence development schemes, transferring evidence from foreign countries and addressing the challenges of learning curve and centre effect; (2) economic value assessment, covering cost calculation of complex medical devices and transferability of economic evaluations of MDs; (3) HTA processes, related to the frequent product modifications and various indications of MDs.Conclusions: Central and Eastern European countries with limited resources for conducting HTA, can benefit from HTA methods and evidence generated in early technology adopter countries. Considering the appropriate reuse of international HTA materials, late technology adopter countries can still implement HTA, even for MDs, which have a more limited evidence base compared with pharmaceuticals.

Objectives: We performed a systematic review of health state utility values (HSUVs) obtained using the EQ-5D questionnaire for patients with hematologic malignancies. Methods:The following databases were searched up to September 2018: MEDLINE, EMBASE, The Cochrane Library, and the EQ-5D publications database on the EuroQol website. Additional references were extracted from reviewed articles. Only studies presenting EQ-Index results were incorporated. In view of the heterogeneity across the included publications, we limited ourselves to a narrative synthesis of original HSUVs found.Results: Fifty-nine studies (described in 63 articles) met the inclusion criteria. Data from 21 635 respondents provided 796 HSUV estimates for hematologic malignancy patients. EQ-Index scores ranged from -0.025 to 0.980. The most represented area was multiple myeloma (4 studies, 11 112 patients, and 249 HSUVs). In clinical areas such as chronic myeloid leukemia, acute myeloid leukemia, chronic lymphocytic leukemia, non-Hodgkin lymphoma, and mantle cell lymphoma, we described over 50 health utilities in each. In contrast, we identified only 13 HSUVs (based on 4 studies and the data of 166 patients) for Hodgkin lymphoma. Areas without EQ-5D-based health utilities comprised: polycythemia vera, primary myelofibrosis, essential thrombocythemia, mastocytosis, myeloid sarcoma, chronic myelomonocytic, eosinophilic leukemia, and neutrophilic leukemia. Conclusions:There is a wide range of HSUVs available for hematologic cancer patients with different indications. The review provides a catalog of utility values for use in cost-effectiveness models for hematologic malignancies.

To describe characteristics of the drug policy in Russia in terms of health technology assessment (HTA), registries of patients, pricing of drugs, cost-containment methods, and reimbursement of drugs. Methods: The legal and regulatory frameworks and the literature were reviewed to analyze several aspects of Russian healthcare: the scheme of cooperation between its structures, its levels, drug provision, issues related to HTA, the reimbursement system, pricing of medicines, and cost-containment methods. Results: The Russian drug policy has improved over the last few years: HTA has been developed, rules for the pricing of drugs and cost-containment methods have been established, and registries of patients have been created. The reimbursement system in Russia is different from the ones in Western Europe and consists of a few programs: reimbursement for specific categories of citizens, vital and essential drug list, list of 24 orphan diseases, list of 7 nosologies, and other programs, depending on region. Financing for drug provision in Russia is divided into 2 levels: federal and regional. There is still a lack of transparency and equality in healthcare as well as huge differences in access to healthcare, depending on region. Conclusions: The healthcare system in Russia is complicated and needs improvement. Nowadays, changes are being made; for example, there are attempts to implement HTA at federal and regional levels.

Celem poniższej notki jest przybliżenie czytelnikom tematu współpracy w zakresie oceny technologii medycznych w krajach Unii Europejskiej (UE), obecnej sytuacji oraz przyszłych planowanych prac w tej dziedzinie. Komisja Europejska próbuje stworzyć ramy sprawnie działającej współpracy pomiędzy krajami członkowskimi Unii Europejskiej w zakresie oceny technologii medycznych i sukcesywnie pracuje nad jej wzmocnieniem. W jej ramach zrealizowano następujące inicjatywy, projekty oraz programy: - Projekt dotyczący koordynacji i rozwoju oceny technologii opieki zdrowotnej w Europie - Europejska współpraca na rzecz oceny interwencji zdrowotnych - Sieć ds. oceny technologii medycznych  - Mechanizmu skoordynowanego dostępu do sierocych produktów leczniczych - Inicjatywa Beneluxa, deklaracja z Valletty, Nordycka Współpraca, Inicjatywa sprawiedliwych i przystępnych cen produktów medycznych (zasięg regionalny). Obecnie trwa praca nad Rozporządzeniem Parlamentu Europejskiego i Rady w sprawie oceny technologii medycznych, które jest mierzalnym rezultatem tej współpracy. Kluczowym założeniem tego rozporządzenia jest scentralizowana wspólna ocena kliniczna efektywności produktu leczniczego oraz wyrobów medycznych na poziomie europejskim.

Objectives: Primary-Progredient Multiple Sclerosis (PPMS) is an indication in which, until recently, only the best supportive therapy (BSC) was used. Drugs used in highly active remitting multiple sclerosis (HARMS) are high-cost therapy and their effectiveness differ. Ocrelizumab is a new drug from the group of monoclonal antibodies to the CD20 B-lymphocyte receptor and its use can lead to changes in the budget for the treatment of PPMS and HARMS. The aim of the study was to determine the budget impact of the inclusion in the public funding of the use of ocrelizumab in both indications in Russia. Methods: The populations of PPMS and HARMS were determined on the basis of literature data (both is 10% of the total population of MS patients). The analysis point of view (federal and regional), the time horizon (1, 2, 5 years), and analysis scenarios were set basing on MS experts' opinion. For PPMS, it was assumed that patients would receive BSC (existing scenario) or ocrelizumab (new scenario). For HARMS, it was assumed that patients would receive alemtuzumab or natalizumab (existing scenarios 1 and 2) or ocrelizumab (new scenario). Results: The use of ocrelizumab instead of BSC in PPMS increases costs/year by 73 864 873.84 euro. The use of ocrelizumab instead of natalizumab and alemtuzumab in HARMS reduces costs in the first year by 35 134 031,85 and 174 442 131,17 euro, respectively. Conclusions: The use of ocrelizumab instead of BSC in PPMS causes additional budget costs, and in HARMS instead of natalizumab and alemtuzumab, with reduces them. The use of ocrelizumab in both indications could lead to providing PPMS patients with the first effective drug therapy in their indication and at the expense of financial savings associated with the use of ocrelizumab instead of alemtuzumab in HARMS and would not lead to additional costs of MS therapy.

CASP checklists. The success characteristics were extracted from the eligible articles. Results: The search returned with 1833 articles with 944 excluded for duplicates, non-relevance, study design and endpoints, and publication types after initial review. Preliminary result showed that diabetes, cardiology and obesity were the most common target disease areas for digital technology implementation. The most used digital technology were mobile applications, wearables, and web-based intervention, with artificial intelligence is increasingly studied. Publications on industry-sponsored trials, digital technology in clinical trials and as specific treatment companion are limited. The endpoints for outcome measurement and result of digital technology in improving patients' quality of care vary. The success factors for digital technology implementation are quality of care achievement (effective, efficient, accessible/ coverage, and standardized) and positive user experience (usability, acceptability, non-interference and reliability). Conclusions: Digital technology is increasingly used in healthcare settings and showed promising benefits to measure and improve patient outcome. Patients' insights, system standardization and validation are crucial for the success of digital solution. A standardized and robust study design is required to demonstrate the impact of digital technology on patients' quality of care.