Background: Multi-criteria decision analysis (MCDA) is a decision-making tool that can take into account multidimensional factors and enables comparison of (medical) technologies by combining individual criteria into one overall appraisal. The MCDA approach has slowly gained traction within Health Technology Assessment (HTA) and its elements are gradually being incorporated into HTA across Europe. Several groups of scientists have proposed MCDA approaches targeted toward orphan drugs and rare diseases by including criteria specific to rare diseases. The goal of this article is to provide an overview of the current state of knowledge and latest developments in the field of MCDA in HTA for orphan drugs, to review existing models, their design characteristics, as well as to identify opportunities for further model improvement. Methods: A systematic literature search was conducted in January 2018 using four databases: MEDLINE (Pubmed), EBSCO HOST, EMBASE, and Web of science to find publications related to use of MCDA in the rare disease field (keywords: MCDA/orphan drug/rare disease and synonyms). Identified MCDA models were analyzed, e.g., structure, criteria, scoring, and weighting methodology. Results: Two hundred and eleven publications were identified, of which 29 were included after removal of duplicates. 9 authors developed own MCDA models, 7 of which based on literature reviews intended to identify the most important and relevant decision criteria in the model. In 13 publications (8 models) weights were assigned to criteria based on stakeholder input. The most commonly chosen criteria for creation of the MCDA models were: comparative effectiveness/efficacy, the need for intervention, and disease severity. Some models have overlapping criteria, especially in the treatment cost and effectiveness areas. Conclusions: A range of MCDA models for HTA have been developed, each with a slightly different approach, focus, and complexity, including several that specifically target rare diseases and orphan drug appraisal. Models have slowly progressed over the years based on pilots, stakeholder input, sharing experiences and scientific publications. However, full consensus on model structure, criteria selection and weighting is still lacking. A simplification of the MCDA model approach may increase its acceptance. A multi-stakeholder discussion on fundamental design and implementation strategies for MCDA models would be beneficial to this end.

The phenomenon of population ageing observed over recent years involves growing healthcare needs and the limited staffing and financing of healthcare systems, and as such demands some functional changes in the healthcare model in many countries. This situation is particularly significant in the face of a pandemic, e.g., flu, and currently COVID-19.As well as social education, preventive vaccinations are the most effective method of fighting the infectious diseases posing a special threat to seniors. Despite this, the vaccination coverage level in most European countries is relatively low. This is largely due to patients having limited access to vaccinations. In some countries, implementing vaccinations in pharmacies and by authorized pharmacists has significantly improved vaccination coverage rates and herd immunity, while lowering the cost of treating infections and the resulting complications, as well as minimizing the phenomenon of inappropriate antibiotic therapies. This article presents the role of pharmacists in the prevention of infectious diseases, pointing out the measurable effects of engaging pharmacists in conducting preventive vaccinations, as well as analyzing the models of implementing and conducting vaccinations in pharmacies in selected countries, and depicting recommendations regarding vaccinations developed by international organizations. The presented data is used to suggest requirements for the implementation of preventive vaccinations in community pharmacies.

Heart failure (HF) is a chronic disease of great clinical and economic significance for both the healthcare system and patients themselves. To determine the consumption of medical resources for treatment and care of HF patients and to estimate the related costs. The study involved 400 primary care practices and 396 specialist outpatient clinics, as well as 259 hospitals at all reference levels. The sample was representative and supplemented with patient interview data. Based on the consumption of particular resources and the unit costs of services in 2011, costs of care for HF patients in Poland were estimated. Separate analyses were conducted depending on the stage of the disease (according to NYHA classification I-IV). The public payer's perspective and a one year time horizon were adopted. Direct annual costs of an HF patient's treatment in Poland may range between PLN 3,373.23 and 7,739.49 (2011), the main cost item being hospitalisation. The total costs for the healthcare system could be as high as PLN 1,703 million, which is 3.16% of the National Health Fund's budget (Ex. rate from 05.03.2012: 1 EUR = 4.14 PLN). The costs of treating heart failure in Poland are high; proper allocation of resources to diagnostic procedures and treatment may contribute to rationalisation of the relevant expenditure.

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.

Introduction: Mortality, whether in or out of hospital, increases with age. However, studies evaluating in-hospital mortality in large populations did not distinguish between surgical and non-surgical causes of death, either in young or in elderly patients. The aim of the study was to assess in-hospital non-surgical mortality in a large group of patients, with a special focus on the elderly.Material and methods: Data from the database of the Polish National Health Fund (NHF) regarding hospitalizations of adult (≥ 18 years) patients not related to surgical procedures in the years 2009–2013 were used to assess in-hospital mortality.Results: 15,345,025 hospitalizations were assessed. The mean in-hospital non-surgery-related mortality rate was 3.96 ±0.17%, and increased from 3.79% to 4.2% between 2009 and 2013. The mean odds ratio for in-hospital death increased with the age of patients, reaching a 229-fold higher rate in the ≥ 95 years age group as compared to the 18–24 age group. The highest mean mortality was associated with respiratory diseases (6.91 ±0.20%), followed by heart and vascular diseases, nervous system diseases, as well as combined gastrointestinal tract, liver, biliary tract, pancreas and spleen diseases (5.65 ±0.27%, 5.46 ±0.05% and 4.01 ±0.13%, respectively).Conclusions: The in-hospital non-surgery-related mortality rate was approximately 4%. It significantly increased with age and, regardless of age, was highest in patients suffering from respiratory diseases.