Health policy outcomes

Since the end of the 18th century, vaccines against several infectious diseases have been developed. Having gone through a long process of technology improvements, traditional vaccination represents one of the most significant and cost-effective public health measures nowadays. Although many infectious diseases are vaccine-preventable, vaccines are generally still under-valued and under-utilized, resulting in a high burden of infectious diseases worldwide. Vaccination coverage rates are often used as a quality indicator of healthcare systems and are an important measure for reducing child mortality as outlined in Millennium Development Goal (MDG) number 4. However, there are challenges to deploying vaccinations as preventive measure to their full extent. For manufacturers, a high level of know-how is required as well as high upfront investments and fixed costs, which leads to there being only relatively few manufacturers for vaccines. At individual and societal level, there are prob...

The performed analysis included 5 approved combination vaccines marketed in Europe (5-in-1 DTaP-IPV-Hib vaccines, and 6-in-1 DTaP-IPV-Hib--HepB vaccines). Although in the vast majority of countries the analysed vaccinations are not mandatory, the vaccination rate remains very high. Significant differences between the European countries can be seen in vaccination schedules or the types of available combination vaccines in the national childhood immunisation programmes. Despite the different rules and regulations regarding the funding of combination vaccinations from public resources, countries systematically seek to provide patients with the latest generation formulations.

Increasing antibiotic resistance is a global problem. Vaccinations is one of the best solution to reduce the burden of infectious diseases. Despite universal access to antibiotics, pneumococci still are one of the major causes of morbidity and mortality in both developing and developed countries. Criteria for making the decision on fi nancing immunisation are similar in many countries and include identifying the target groups based on epidemiological risk, the clinical effi cacy and safety of a vaccine, and the economic aspects of the implementation of vaccination. To reduce the risk of diseases caused by Diplococcus pneumoniae pneumococcal conjugate vaccine (PCV) is included in national immunisation programmes in most countries. The paper describes international differences of organisations and funding mechanisms for pneumococcal vaccination. The analysis covered 34 countries in Europe. The study analyzed availabili-ty and type of vaccines used in vaccination programmes, children in the target age group to vaccination, vaccination financing, level of co-payment to vaccination for children and mechanism of purchasing vaccines. In most European countries, PCV is included in national immunisation programmes. The diff erences regard the availability of two types of conjugate pneumococcal vaccines (10-valent and 13-valent). The method of recommending vaccines, mechanism of purchasing and funding in diff erent countries varies.

In the field of rare diseases, data on a significant number of patients can only be archived due to national and international collaboration. Data summarized from specialized centers are a powerful tool. Thanks to modern technology and networks, national and international databases can be constructed legally, with respect to ethical and safety principles. Currently registries for primary immunodeficiencies are established on different levels: single center, national collaboration involving specialized tertiary centers on the country level and on international level as for example ESID registry. Not all registries cover the same dataset, but generally allow to estimate prevalence of diseases, description of natural history, morbidity and mortality. Moreover registries can provide information of diagnostic criteria used, quality of care and management modalities. In the field of primary humoral immunodeficiencies is crucial to assess availability of immunoglobulin G substitution and to plan demand for this product. In other ultra-rare primary immunodeficiencies registries are the unique opportunity to compare different treatment interventions and strategies as hematopoietic stem cell transplantation. The review presents current published data from different registries of primary immunodeficiencies to underscore the need for founding polish registry.

Severe asthma is a heterogeneous disease, which, as it is now believed, requires a personalized therapy dependent on phenotype. Omalizumab, a humanized monoclonal antibody that specifically binds to free immunoglobulin E, proved to be particularly effective in the treatment of allergic asthma. Because this treatment is very expensive, in Poland it is reimbursed under the drug program: "Omalizumab treatment of severe, allergic, IgEdependent asthma". One of the main features of drug programs is precise defining the group of patients, which may be subject to treatment. The current experience shows that it significantly increase the efficacy of the therapy. On the other hand, however, may adversely affect the possibility of treating patients with difficult access to specialized centres.

Background: This study aimed at estimating the cost-effectiveness of the fixed-dose combination indacaterol/glycopyrronium (IND/GLY) 85/43μg versus salmeterol/ fluticasone 50/500μg (SFC) and tiotropium 18μg (TIO) in the management of patients with chronic obstructive pulmonary disease (COPD) in Greece. Methods: A microsimulation model was developed in MS Excel. Effectiveness and utility data were obtained from the international literature and mortality data from the WHO database. Distribution of patients by severity stage of airflow limitation, maintenance costs and costs associated with severe/ non-severe exacerbations were taken from published Greek studies. Unit costs were taken from officially published sources (Price Bulletin, reimbursement list, diagnosis-related groups). The study perspective was that of the Social Insurance Fund; costs and outcomes were discounted at 3.5%, and the outcomes are reported over time horizons of one, three, five and 10 years and over a lifetime. ...

Background: The objective of this review is to assess the clinical effectiveness and cost-utility of sunitinib and best supportive care (BSC) versus placebo and best supportive care in the treatment of patients with unresectable or metastatic well-differentiated pancreatic neuroendocrine tumors with disease progression. Methods: Assessment of the clinical effectiveness of the interventions was conducted in accordance with the principles of systematic review, based on the Cochrane Collaboration guidelines (Cochrane Reviewer's Handbook) and the guidelines of the Polish Agency for Health Technology Assessment (AOTM). The Markov model constructed in TreeAge Pro 2009 was used in the cost-utility analysis. The time horizon covered the period from the beginning of the treatment until the patient's death (lifetime horizon). Quality adjusted years (QALY) were used as the measure of effectiveness and the results were presented as incremental cost-utility. CUA was conducted from the perspective of the public payer for health services (Polish National Health Fund, PNHF) and from the patient's and PNHF's perspective. Results: As the result of the systematic search, one primary randomized clinical trial satisfying the inclusion criteria was found (Raymond 2011). The results of the present analysis clearly prove that sunitinib administered in a 37.5 mg dose is an effective and safe therapy in the treatment of patients with unresectable or metastatic well-differentiated pancreatic neuroendocrine tumors with disease progression. The cost of gaining an additional QALY by replacing place-bo+BSC with sunitinib+BSC is PLN 84,21 4 /PLN 84,296 (€20,441 /€20,461) from PNHF/PNHF+patient perspective. Conclusion: Sunitinib is a more costly and a more effective therapy than BSC.

Introduction: The purpose of this paper is to critically assess the Pharmaceutical reforms implemented in Greece before and during the economic crisis. The effects of the crisis are multiple in terms of GDP reduction, severe cuts of health and pharmaceutical expenditures and the deterioration of the health status of the population. However, the crisis has been a “window opportunity” to implement the long anticipated reforms in the health sector. Methods: The analysis is based on Time series data from the OECD Health data source covering the period 1970-2014. Double Logarithmic models are explored to estimate income elasticity for pharmaceutical expenditures. The value of the income elasticity in the Pre-crisis period is 1.72 the highest in Europe. The average annual growth of Pharmaceutical expenditure in the pre-crisis period was 12.3% i.e. the highest among the EU-28 countries and during the crisis this rate dropped to -8.5% the lowest among the EU-28. Results: The evolution of th...

Introduction: The German PID-NET registry was founded in 2009, serving as the first national registry of patients with primary immunodeficiencies (PID) in Germany. It is part of the European Society for Immunodeficiencies (ESID) registry. The primary purpose of the registry is to gather data on the epidemiology, diagnostic delay, diagnosis, and treatment of PIDs. Methods: Clinical and laboratory data was collected from 2,453 patients from 36 German PID centres in an online registry. Data was analysed with the software Stata ® and Excel. Results: The minimum prevalence of PID in Germany is 2.72 per 100,000 inhabitants. Among patients aged 1-25, there was a clear predominance of males. The median age of living patients ranged between 7 and 40 years, depending on the respective PID. Predominantly antibody disorders were the most prevalent group with 57% of all 2,453 PID patients (including 728 CVID patients). A gene defect was identified in 36% of Conclusion: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.

It has been proven that even small concentrations of certain drugs in the environment can be toxic to plants, but also fatal for animals and humans. The only proper way to prevent this phenomenon is to minimize the adverse effects due to improper waste management of medications. In developed countries, the Ministry of Health, other authorities or academic bodies set up guidelines or recommendations for the proper management of unused and/or outdated medications in the community pharmacy setting. Unfortunately, no such regulations (in the form of standard operational procedures) exist in Poland. This lack of adequate guidelines can lead to the improper and uncontrolled circulation of harmful substances, both at the level of the community pharmacy and other places disposing of medicines, as well as in the environment. The aim of this paper is to introduce the concept of medicine disposal around the world and to suggest recommendations for the proper removal of pharmaceuticals in Poland

Introduction: The German PID-NET registry was founded in 2009, serving as the first national registry of patients with primary immunodeficiencies (PID) in Germany. It is part of the European Society for Immunodeficiencies (ESID) registry. The primary purpose of the registry is to gather data on the epidemiology, diagnostic delay, diagnosis, and treatment of PIDs. Methods: Clinical and laboratory data was collected from 2,453 patients from 36 German PID centres in an online registry. Data was analysed with the software Stata ® and Excel. Results: The minimum prevalence of PID in Germany is 2.72 per 100,000 inhabitants. Among patients aged 1-25, there was a clear predominance of males. The median age of living patients ranged between 7 and 40 years, depending on the respective PID. Predominantly antibody disorders were the most prevalent group with 57% of all 2,453 PID patients (including 728 CVID patients). A gene defect was identified in 36% of Conclusion: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.

Background: The ways of gaining the information on healthcare should be verified as the discrepancies between different sources may lead to serious mistakes. Unfortunately, there are few reports on the methodology on healthcare needs assessment especially when mental and behavioural disorders are considered.

Background. Health technology assessment (HTA) introduction and implementation for development of the National List of Essential Medicines (NLEM) took place in Ukraine in 2016 in the context of National Drug Policy until 2025. The study aimed to outline the reimbursement of medicines for asthma treatment, market access for innovative medicines, main steps of HTA development in the decision making process in Ukraine in line with the international requirements and future directions. Methods. Literature review was conducted examining legislation database and scientific publications on the study issues in Ukraine: epidemiology of asthma, reimbursement programs, HTA development, affordability of innovative medicines for asthma treatment, market analysis. The study was performed to investigate the practices, processes and policies of value assessment and their impact in Ukraine. Results. In 2016 a legal framework was developed for the elaboration of NLEM and procedures for reference prici...

Introduction: The German PID-NET registry was founded in 2009, serving as the first national registry of patients with primary immunodeficiencies (PID) in Germany. It is part of the European Society for Immunodeficiencies (ESID) registry. The primary purpose of the registry is to gather data on the epidemiology, diagnostic delay, diagnosis, and treatment of PIDs. Methods: Clinical and laboratory data was collected from 2,453 patients from 36 German PID centres in an online registry. Data was analysed with the software Stata ® and Excel. Results: The minimum prevalence of PID in Germany is 2.72 per 100,000 inhabitants. Among patients aged 1-25, there was a clear predominance of males. The median age of living patients ranged between 7 and 40 years, depending on the respective PID. Predominantly antibody disorders were the most prevalent group with 57% of all 2,453 PID patients (including 728 CVID patients). A gene defect was identified in 36% of Conclusion: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.

Background: This study aimed at estimating the cost-effectiveness of the fixed-dose combination indacaterol/gly copyrronium (IND/GLY) 85/43μg versus salmeterol/ fluticasone 50/500μg (SFC) and tiotropium 18μg (TIO) in the management of patients with chronic obstructive pulmonary disease (COPD) in Greece. Methods: A microsimulation model

Effective pain management and pain prevention is one of the fundamental obligations of doctors. The need to improve the management of pain is widely recognized in public debate in Poland. Background: Identification and assessment of financial barriers in access to opioids in pain treatment of cancer patients in Poland. Material and Methods: The main area of investigations was availability of opioids and cost of treatment for patients. The principles, conditions and procedures for reimbursement of opioids were recognized and analyzed from cancer patients point of view. All reimbursement lists announced by MoH were taken into account as well as data concerning reimbursed drug consumption and NHF spending's. Results: Polish cancer patients have access to subsequent reimbursed opioids: morphine, fentanyl, oxycodone, methadone, buprenorphine, tramadol, dihydrocodeine. In the case of opioids limit groups are narrow and very precisely defined and very compartmentalized. In practical terms, almost all patients may have access to opioids with limit price without additional co-payment. Expenditures on opioids reimbursement between 2012 and 2014 increased from PLN 178M to 231M. Consumption of opioids increased faster than reimbursement expenditures between 2012 and 2014 because growth of consumption between 2012-2013 and 2013-2014 accounted for 17,7% and 23,4% respectively. Significant price decrease of opioids leaded to decrease of average patients co-payment for pack of opioids from PLN 9,63 to PLN 6,10. Conclusions: Financial barriers in access to cancer pain treatment with opioids in Poland seems to be very limited.

Introduction: The German PID-NET registry was founded in 2009, serving as the first national registry of patients with primary immunodeficiencies (PID) in Germany. It is part of the European Society for Immunodeficiencies (ESID) registry. The primary purpose of the registry is to gather data on the epidemiology, diagnostic delay, diagnosis, and treatment of PIDs. Methods: Clinical and laboratory data was collected from 2,453 patients from 36 German PID centres in an online registry. Data was analysed with the software Stata ® and Excel. Results: The minimum prevalence of PID in Germany is 2.72 per 100,000 inhabitants. Among patients aged 1-25, there was a clear predominance of males. The median age of living patients ranged between 7 and 40 years, depending on the respective PID. Predominantly antibody disorders were the most prevalent group with 57% of all 2,453 PID patients (including 728 CVID patients). A gene defect was identified in 36% of Conclusion: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.

Background: For cost-effectiveness analysis quality of life weights estimated by preference based utility measures are needed. In many studies however, quality of life is estimated by instruments that cannot provide utility measures. The aim of the study was to derive a function which can map the EORTC QLQ-BR23 questionnaire onto the EuroQol-5D (EQ-5D) questionnaire in breast cancer patients. Methods: A cross sectional study was performed in Hungary in 615 breast cancer patients with different states of the disease. Quality of life was measured by both EORTC QLQ-BR23 and EuroQol-5D. Ordinary stepwise backward least-squares regression was used to develop a mapping function. Adjusted R2, Akaike's Information Criterion (AIC) and root mean square error (RMSE) were used to assess model performance. The robustness of the models was tested by 10-fold cross-validation and bootstrapping. Results: The "best fitting" model contained 26 BR23 item levels as predictors selected in a stepwise backward procedure. However, this model showed considerable variability in the selection of predictors. A model, which performed only marginally worse than the "best fitting model" (adjusted R2 0.44, RMSE: 0.216, AIC:-85.8) and contained the BR23 items was much more stable, therefore we considered it as the best mapping function. Conclusions: The expected value of EQ-5D can be reasonably well predicted based on the results of EORTC QLQ-BR23 in patients with breast cancer. Its applicability, however, for prediction on the individual level is limited.

Objective: Our aim was to validate the TSIDAV vignette in thrombocytopenia patients. Methods: The vignettes validation was performed during a study on adults and children with thrombocytopenia. The data were collected among Lublin hospital patients within the period from1st March to 30th April 2016. Results: We have validated the vignette based on the 61 questionnaires collected among thrombocytopenia patients and children's caregivers. 31 vignettes were from female patients, 17 from male patients and 13 from children's caregivers. The most frequently mentioned symptom influencing daily activities in all groups was petechiae and easy bruising. Among men 29% declared it as significant, 68% of women also considered petechiae on their skin and mucosa as having significant impact on their daily activities and 65% declared a significant impact of easy bruising. Among parents and caregivers 62% of them also indicated petechiae as having significant impact on their daily lives and 54% also declared significant easy bruising. We have used a developed special scale for the numerical presentation of the obtained results allowing their interpretation. Conclusions: The properties of the vignette make it appropriate to assess the impact of the thrombocytopenia symptoms on patients daily activities. TSIDAV and its dual construction lets us collect the catalogue of different symptoms of thrombocytopenia and their impact of daily life, which could be very helpful in working with thrombocytopenia patients from the psychological and medical point of view.

Objective: Assessment of the impact of thrombocytopenia symptoms on patients’ daily activities using vignettes. Methods: Vignettes were used to collect information from adult patients and children’s caregivers in order to assess the impact of thrombocytopenia symptoms on patients’ daily activities. Each vignette consisting of two parts aimed at collecting qualitative information about major health problems related to thrombocytopenia symptoms and at measuring the intensity of the symptoms’ impact on daily activities in a quantitative way. Descriptive part collected patients’ qualitative information about symptoms inf luencing their daily functioning or those bothering them the most. Quantitative part using Likert scale assessed the impact of specific symptoms on patients’ lives. Results: We collected 61 questionnaires from 31 women, 17 men, 13 children’s caregivers. Among men the biggest impact on activities was observed in relation to petechiae and easy bruising (both symptoms were...

Introduction. The goal of the study is to assess the usefulness of Sankey model in the analysis of healthcare phenomena. Material and methods. Sequence analysis and Sankey model generation was based on the data provided to the National Health Fund in the years 20132015 for the following diseases defined by the following ICD-10 codes: C50.% – malignant neoplasm of breast; G35 – multiple sclerosis; B18.2, B17.1 – chronic hepatitis C. The reported data concerned all types of services provided by the public payer. Results. The analysis was conducted for: C50.% – 3.26 million data records, for 236.6 thousand patients; G35 – 110 thousand data records, for 23.9 thousand patients; B18.2, B17.1 – 1,1 million data records, for 100 thousand patients. Sequence analysis and the generated Sankey diagrams indicate that paths taken by patients in the health care system are varied and are strictly conditional on the medical problem. Conclusion. Sequence analysis, and in particular the diagram-based ...

Background: The objective of this review is to assess the clinical effectiveness and cost-utility of sunitinib and best supportive care (BSC) versus placebo and best supportive care in the treatment of patients with unresectable or metastatic well-differentiated pancreatic neuroendocrine tumors with disease progression. Methods: Assessment of the clinical effectiveness of the interventions was conducted in accordance with the principles of systematic review, based on the Cochrane Collaboration guidelines (Cochrane Reviewer's Handbook) and the guidelines of the Polish Agency for Health Technology Assessment (AOTM). The Markov model constructed in TreeAge Pro 2009 was used in the cost-utility analysis. The time horizon covered the period from the beginning of the treatment until the patient's death (lifetime horizon). Quality adjusted years (QALY) were used as the measure of effectiveness and the results were presented as incremental cost-utility. CUA was conducted from the perspective of the public payer for health services (Polish National Health Fund, PNHF) and from the patient's and PNHF's perspective. Results: As the result of the systematic search, one primary randomized clinical trial satisfying the inclusion criteria was found (Raymond 2011 ). The results of the present analysis clearly prove that sunitinib administered in a 37.5 mg dose is an effective and safe therapy in the treatment of patients with unresectable or metastatic well-differentiated pancreatic neuroendocrine tumors with disease progression. The cost of gaining an additional QALY by replacing place-bo+BSC with sunitinib+BSC is PLN 84,21 4 /PLN 84,296 (€20,441 /€20,461 ) from PNHF/PNHF+patient perspective. Conclusion: Sunitinib is a more costly and a more effective therapy than BSC.

In the field of rare diseases, data on a significant number of patients can only be archived due to national and international collaboration. Data summarized from specialized centers are a powerful tool. Thanks to modern technology and networks, national and international databases can be constructed legally, with respect to ethical and safety principles. Currently registries for primary immunodeficiencies are established on different levels: single center, national collaboration involving specialized tertiary centers on the country level and on international level as for example ESID registry. Not all registries cover the same dataset, but generally allow to estimate prevalence of diseases, description of natural history, morbidity and mortality. Moreover registries can provide information of diagnostic criteria used, quality of care and management modalities. In the field of primary humoral immunodeficiencies is crucial to assess availability of immunoglobulin G substitution and t...