Patient care efficiency in primary healthcare - pilot study in Medical and Diagnostical Centre.

In Poland, there is a niche that can be filled with original research in the area of performance indicators of individual health care professionals. AIM: The aim of the study was the empirical verification of original effectiveness indicators and the identification of the degree of patient care effectiveness of selected primary health care professionals with the application of the developed indicators. MATERIALS AND METHODS: The study population consisted of physicians employed in the primary care in Medical and Diagnostic Centre (MDC) in Siedlce, Poland. The final study sample consisted of 29 respondents. 14 original indicators in three areas: structure, process and effect were developed and verified. RESULTS: The distribution of indicator values for the physicians included in the study demonstrated a diverse level of their effectiveness. The factor analysis performed for the 14 original indicators demonstrated that a highly reliable scale can be created out of 5 of the original indicators. CONCLUSIONS: In our pilot study we assessed the reliability of the designed tools. However, unfortunately, our research did not offer the opportunity to identify any dependencies of variables. A study on a larger sample of physicians would therefore be needed.

Real-World Evidence of Patient Outcome Following Treatment of Advanced Gastrointestinal Stromal Tumor (GIST) with Imatinib, Sunitinib, and Sorafenib in Publicly Funded Health Care in Poland.

BACKGROUND This study aimed to undertake an analysis of ten years of real-world evidence (RWE) on overall survival (OS) following treatment of advanced gastrointestinal stromal tumor (GIST) with imatinib, sunitinib, and sorafenib using data from the Polish National Health Fund. MATERIAL AND METHODS Data from the Polish National Health Fund, the sole Polish public payer, identified 1,641 patients with advanced GIST who were treated with imatinib (n=1047), sunitinib (n=457), and sorafenib (n=137). The differences in overall survival (OS) were analyzed. RESULTS For patients with advanced GIST, the median follow-up time for patients treated with imatinib was 71 months (95% CI, 64.8-79.2), the median OS was 56.9 months (95% CI, 50.4-61.2), with survival at 12 months (89.5%), 24 months (77.9%), 36 months (66.9%), and 60 months (48.4%). The median follow-up time for patients treated with sunitinib was 41.4 months (95% CI, 34.6-49.3), the median OS was 22.8 months (95% CI, 19.2-26.8), with survival at 12 months (68.2%), 24 months (47.1%), and 36 months (31%). The median follow-up time for patients treated with sorafenib was 17.4 months (95% CI, 14.6-22.9), the median OS was 16.9 months (95% CI, 13.7-24.3), with survival at 12 months (61.9%), at 24 months (36.2%), and at 36 months (16.8%). CONCLUSIONS Real-world data collected in a ten-year period confirmed the effectiveness of the use of imatinib, sunitinib, or sorafenib for the treatment of advanced GIST and was comparable with the findings from clinical trials.

[Policy developments of health technology assessment in the European Union]. / Rozwój wspólpracy w zakresie oceny technologii medycznych w krajach Unii Europejskiej.

The purpose of the note below is to familiarize readers with the current status and future initiatives of cooperation in the field of health technology assessment (HTA) in the European Union (EU). The European Commission is trying to create a framework for efficient collaboration between member states of EU in the field of HTA and is gradually working on strengthening it. The following initiatives, projects and programs have already been implemented: - Project on Coordination and Development of Health Care Technology Assessment in Europe - The European Collaboration for Assessment of Health Interventions and Technology - European Network for Health Technology Assessment - Beneluxa Initiative, The Valletta Declaration, FINOSE and Fair and Affordable Pricing - European Network for Health Technology Assessment Currently there is an ongoing proposal for an HTA Regulation of the European Parliament and the Council, which is the measurable result of this cooperation. A key element of the proposed regulation is a centralized joint clinical assessment (for HTA purposes) of the efficacy of the medicinal product/medical device.

Treatment persistence in the use of basal insulins in Poland and Germany
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AIMS: To compare short-term basal insulin therapy persistence and its predictors in Poland and Germany. METHODS: Persistence was defined as proportions of patients remaining on the initial basal insulin (analogs: Poland: n = 6,889, Germany: n = 454,067; neutral protamine Hagedorn (NPH) insulins: Poland: n = 50,761, Germany: n = 226,064) over 2 years based on nationwide prescription databases (LRx; IMS Health) in Poland and Germany from 2013 to 2015. Persistence was evaluated by Kaplan-Meier curves (log-rank tests). Risk of discontinuation of initial basal insulin was investigated using Cox regression models adjusting for age, sex, comedication with other glucose-lowering agents and baseline or comedication with antihypertensives, lipid-lowering drugs, antidepressants, and antiepileptics. RESULTS: In Poland, 2-year persistence was 83.0% in analog insulin and 73.3% in NPH users (p < 0.001). In Germany, persistence was also higher in patients with analog insulins (92.6% vs. 79.0%; p < 0.001). Analog insulin users were less likely to discontinue basal insulin compared with NPH users (adjusted hazard ratio (95%CI): Poland: 0.73 (0.67 - 0.79); Germany: 0.27 (0.27 - 0.28)). Higher age (> 75 vs. ≤ 60 years: Poland: 1.24 (1.16 - 1.33), Germany: 1.09 (1.07 - 1.11)) and GLP-1 receptor agonist use (Poland: 2.76 (1.38 - 5.53), Germany: 1.21 (1.16 - 1.26)) were related to higher risk of discontinuation. Male sex, metformin, sulfonylurea, thiazolidinedione, and short-acting insulin prescriptions as well as antihypertensive, anti-epileptic, and lipid-lowering drug use were associated with lower risk of discontinuation in both countries (all p < 0.05). CONCLUSIONS: This real-world study shows that both in Poland and Germany treatment persistence of newly-prescribed basal insulin is influenced by type of insulin (analog vs. NPH) and by glucose-lowering and other comedications.
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Regional differences in insulin therapy regimens in five European countries
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AIMS: The purpose of this study was to investigate differences of insulin therapy regimens in five European countries. METHODS: Proportions of basal bolus therapy (intensified insulin therapy (ICT), basal insulin supported oral therapy (BOT), conventional therapy (CT), and short-acting prandial insulin (SIT) among insulin-treated diabetes patients in Germany (n = 64,055), the UK (n = 6,740), and France (n = 4,779) were estimated using representative general medicine practice databases (Disease Analyzer: 2014). Insulin regimens in Hungary (n = 40,769) and Poland (n = 68,136) were analyzed based on nationwide prescription databases (LRx: 2014). RESULTS: ICT was the most frequent insulin regimen (46 - 81%) in all countries except France (BOT > ICT). SIT showed the lowest use, ranging from 2.5% in the UK to 11.2% in Germany. BOT was more frequently used than CT in Germany and Hungary, which was just the opposite in the UK and Poland. The share of insulin analogs among all prescriptions was higher in Germany, the UK, and France (short-acting insulins: 59 - 98%; basal insulins: 70 - 93%) than in Hungary and Poland (short-acting insulins: 41 - 57%; basal insulins: 23 - 46%) (all p < 0.001). CONCLUSIONS: Despite national and international guidelines, insulin regimens differ substantially between European countries. Our results most likely reflect differences in regulations and reimbursement systems, national diabetes care systems as well as patient characteristics and expectations.
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In-Hospital Mortality in a 4-Year Cohort Study of 3,093,254 Operations in Seniors.

BACKGROUND: Surgery-related mortality depends on a number of factors including the type of surgical procedure, quality of healthcare, co-morbidities, and age of patient. The objective of the study was to assess the in-hospital mortality in the elderly undergoing surgical treatment. METHODS: This was a national data-based retrospective cohort study. Data were extracted from the National Health Fund, a public organization financing medical procedures in Poland. Adult citizens who underwent 9,344,384 surgical interventions (including 3,093,254 cases in seniors who were above 65 years old) between 2009 and 2012 were included in this study. Overall, surgery type-dependent, age-stratified in-hospital mortality related to surgery was assessed. RESULTS: Overall in-hospital surgery-related mortality rate in seniors was stable (approximately 2 % annually, P for trend = 0.104). It doubled with each successive decade of life (1.2, 2.3, 5.6, and 13 % in 65-74, 75-84, 85-94 and ≥ 95 years old groups, respectively, in 2012). In ≥ 75-year-old mortality exceeded 10 % only after neurological surgeries, in ≥ 85-year-old after neurological, vascular, gastrointestinal, and endocrinological surgeries, and in ≥ 95-year-old also after heart and circulation, bones and muscles, liver, pancreas, and spleen operations. However, even in the oldest individuals it was low after genitourinary, female genital tract, head and neck, and eye surgeries. CONCLUSIONS: The overall rate of in-hospital mortality after surgery, although increasing with age, is rather low up to the ninth decade of life. Whereas some surgeries pose a significant risk, others may be relatively safe even in the oldest subjects.

Drug-related risk of severe hypoglycaemia in observational studies: a systematic review and meta-analysis.

BACKGROUND: Diabetes mellitus (DM) leads to multiple complications, including severe hypoglycaemia events (SHEs). SHEs can impact a patient's quality of life and compliance and may directly result in additional costs to the health care system. The aim of this review was to evaluate the risk of severe hypoglycaemia in patients with type 1 (T1) and 2 (T2) DM as observed in everyday clinical practice for various drug regimens. METHODS: We conducted a systematic review of observational (retrospective or prospective) studies in the MEDLINE, Embase, and Cochrane Library databases that covered at least 100 children or adults with T1/T2 DM. In T1 DM, basal-bolus/pre-mix insulin (human or analogue) and insulin pump were reviewed, and in T2 DM, basal-bolus/pre-mix insulin (human or analogue), oral antidiabetic drugs supported with basal insulin (human or analogue), sulfonylureas in monotherapy, and combined oral treatment were reviewed. In order to estimate SHE rates, we extracted data on the time horizon of the study, number of patients, number of SHEs, and number of patients experiencing at least one SHE. We used a random effects model to estimate the annual SHE rate. We considered the risk for other antidiabetic medications in T2 DM to be negligible and the results of our main review yielded no observational data for premixes in T1 DM so they were assessed based on relative rates taken from additional systematic reviews. The study, being a desk research, did not involve any human subjects (including human material or human data) and no ethical committee approval was asked for. For the same reason there was no need to collect informed consent for participation in the study. RESULTS: We identified 76 observational studies encompassing 707,722.30 patient-years. The estimated annual SHE rate varied from 0.168 (95 % CI 0.123-0.237) for insulin pump up to 1.628 for biphasic human insulin in T1 DM patients, and from 0.0035 for oral antidiabetic drugs up to 0.554 (95 % CI 0.157-7.534) for basal-bolus with human insulin in T2 DM patients. CONCLUSIONS: Our review indicates that SHE rates differ between patients depending on treatment regimen. However, SHEs are also driven by other factors. Proper modelling techniques are needed to use various types of information in published studies.

Evaluation of economic effects of population ageing--methodology of estimating indirect costs.

Process of demographic ageing, especially in recent decades, is steadily growing in dynamics and importance due to increasing health-related needs and expectations with regard to a guarantee of social services. Elaboration of the most effective model of care, tailored to Polish conditions, requires an estimation of actual costs of this care, including indirect costs which are greatly related to informal care. The fact that the costs of informal care are omitted, results from a determined approach to analyses. It is discussed only from a perspective of budget for health and does not cover societal aspects. In such situation, however, the costs borne by a receiver of services are neglected. As a consequence, the costs of informal care are underestimated or often excluded from calculations, even if they include indirect costs. Comprehensive methodological approach for estimating the costs of informal care seems to be important for a properly conducted economic evaluation in health care sector.

Knowledge and Beliefs about Clinical Trials among Adults in Poland: A Cross-Sectional Study.

Clinical trials, by contributing to the development of diagnostics and to the search for modern, more effective, and safer therapies, have become one of the most important elements of the healthcare system. They enable the introduction of innovative drugs and treatments that can significantly improve patients' quality of life. Not only does this research help to understand disease mechanisms, but it also enables the personalization of therapy, which often increases the effectiveness of treatment. Public awareness of clinical trials helps build trust in science and medicine, which is fundamental to the effective functioning of the healthcare system. The aim of this study was to assess Poles' knowledge and beliefs about clinical trials. METHODS: The survey was conducted among Poles aged 18 and over with the help of an external company, Ariadna, which is an independent research panel. The questionnaire contained 22 questions, of which 13 questions concerned beliefs and attitudes towards clinical trials. RESULTS: One thousand and seventy-nine participants took part in the study (n = 1079). The mean age of respondents was 44.96 years (SD = 16.30). Slightly more women (n = 568, 52.6%) than men (n = 511, 47.4%) took part in the study. Among the respondents, 86.5% (n = 933) were aware of clinical trials. The main sources of information about clinical trials were the media (53.8%) including the Internet (n = 355, 32.9%), TV (n = 175, 16.2%), press (n = 30, 2.8%), and radio (n = 21, 1.9%). 43.2% (n = 466) of respondents reported little knowledge of clinical trials, while more than three quarters (n = 805, 75.2%) said they would like to learn more about clinical trials. Most respondents (n = 879, 81.4%) agreed with the statement that participation in a clinical trial is completely voluntary, and more than half (n = 580, 53.7%) agreed with the statement that hospitals participating in clinical trials provide better healthcare. The statement that the results of clinical trials are made available to the public was disagreed with by 37.2% (n = 402) of participants. Only 30.3% (n = 327) of participants agreed that clinical trials should be conducted with children. Most respondents (n = 638, 59.1%) agreed with the statement that a patient in a clinical trial is insured. 48.3% (n = 521) of participants are aware that a clinical trial can be withdrawn from at any time. CONCLUSIONS: Poles rate their knowledge of clinical trials as low and would like to learn more. Poles' knowledge of clinical trials is mainly based on commercial sources.

Facilitators and Barriers of Incremental Innovation by Fixed Dose Combinations in Cardiovascular Diseases.

Despite the availability of affordable pharmaceuticals treating cardiovascular diseases (CVDs), many of the risk factors remain poorly controlled. Fixed-dose combinations (FDCs), a form of incremental innovation, have already demonstrated improvements over combinations of single medicines in adherence and hard clinical endpoints. Nevertheless, there are many barriers related to the wider use of FDCs in CVDs. Our aim was to identify these barriers and explore system-level facilitators from a multi-stakeholder perspective. Identified barriers include (i) hurdles in evidence generation for manufacturers, (ii) limited acceptance of adherence as an endpoint by clinical guideline developers and policymakers, (iii) limited options for a price premium for incremental innovation for healthcare payers, (iv) limited availability of real-world evidence, and (v) methodological issues to measure improved adherence. Initiatives to standardize and link healthcare databases in European countries, movements towards improved patient centricity in healthcare, and extended value assessment provide opportunities to capture the benefits of FDCs. Still, there is an emerging need to facilitate the generalizability of sporadic clinical evidence across different FDCs and to improve adherence measures. Finally, healthcare payers need to be convinced to pay a fair premium price for the added value of FDCs to incentivize incremental innovation in CVD treatment.

Cost-effectiveness of nonavalent vs bivalent HPV vaccine in Polish setting.

OBJECTIVES: Human papillomavirus (HPV) is a prevalent sexually transmitted infection with significant implications for public health. In Poland, a nationwide vaccination program offers a choice between the 9-valent (9v) and 2-valent (2v) HPV vaccines. We aimed to assess the cost-effectiveness of the 9v vs 2v vaccine from the public payer perspective in Poland. MATERIAL AND METHODS: A cost-effectiveness analysis was conducted to compare the public health and economic benefits of using 9v vs 2v vaccine in Poland over 100-year horizon using a previously published deterministic dynamic transmission model. A target population of girls and boys aged 12-13 years was considered. The model was populated with local epidemiological inputs, utilities, and costs, including vaccine and administration costs, as well as costs related to medical procedures for HPV-related diseases. RESULTS: The 9v vaccine reduced the prevalence of HPV infections and HPV-related diseases substantially more than 2v vaccine when both are compared to no vaccination strategy. The total discounted cost savings of using the 9v vaccine instead of 2v, excluding the vaccine costs, amounted to EUR 66 million. The incremental cost-effectiveness ratio amounted to 8094 EUR per quality-adjusted life year, much below the official cost-effectiveness threshold in Poland set up at the three times the annual gross domestic product per capita. 9v cost-effectiveness ratio remained unchanged when shorter time-horizons of 20, 40, 60, or 80 years were considered. CONCLUSIONS: Using 9v HPV vaccine in Poland is highly cost-effective compared to the 2v vaccine.

Epidemiology of Respiratory Syncytial Virus Hospitalizations in Poland: An Analysis from 2015 to 2023 Covering the Entire Polish Population of Children Aged under Five Years.

BACKGROUND: Respiratory syncytial virus (RSV) is an important cause of childhood hospitalizations. The aim of the study was to estimate the rates of RSV-related hospitalizations in children aged less than 5 years in Poland. METHODS: This retrospective observational cohort study was based on data obtained from the National Health Fund in Poland regarding all acute respiratory tract infections and RSV-coded admissions of children (age < 5 years) to public hospitals between July 2015 and June 2023. Patients were stratified based on the following age groups: 0-1 month, 2-3 months, 4-6 months, 7-12 months, 13-24 months, and 25-60 months. RESULTS: The number of RSV-related hospitalizations increased every season, both before and through the ending phase of the coronavirus disease 2019 (COVID-19) pandemic. The COVID-19 pandemic was associated with a shift in the seasonality pattern of RSV infection. Hospitalization rates per 1000 inhabitants were the highest for children aged 0-12 months, reaching 47.3 in the 2022/23 season. Within this group, the highest hospitalization rate was observed for children aged 2-3 months-94.9 in the 2022/23 season. During the ending phase of the COVID-19 pandemic, the observed increase in admission rates was 2-, 4-, and 5-fold the pre-COVID rate for children aged <12 months, 12-24 months, and 25-60 months, respectively. CONCLUSIONS: In Poland, RSV infections cause a significant burden in hospitalized children aged less than 5 years. RSV-related hospitalizations were most frequent in children aged less than 1 year. The COVID-19 pandemic was associated with a shift in the seasonality pattern of RSV infections. After the pandemic, more RSV-related hospitalizations were observed in older children (aged 13 months and older) vs. the pre-pandemic phase.

Cost saving analysis of prediabetes intervention modalities in comparison with inaction using Markov state transition model-A multiregional case study.

BACKGROUND: Prediabetes management is a priority for policymakers globally, to avoid/delay type 2 diabetes (T2D) and reduce severe, costly health consequences. Countries moving from low to middle income are most at risk from the T2D "epidemic" and may find implementing preventative measures challenging; yet prevention has largely been evaluated in developed countries. METHODS: Markov cohort simulations explored costs and benefits of various prediabetes management approaches, expressed as "savings" to the public health care system, for three countries with high prediabetes prevalence and contrasting economic status (Poland, Saudi Arabia, Vietnam). Two scenarios were compared up to 15 y: "inaction" (no prediabetes intervention) and "intervention" with metformin extended release (ER), intensive lifestyle change (ILC), ILC with metformin (ER), or ILC with metformin (ER) "titration." RESULTS: T2D was the highest-cost health state at all time horizons due to resource use, and inaction produced the highest T2D costs, ranging from 9% to 34% of total health care resource costs. All interventions reduced T2D versus inaction, the most effective being ILC + metformin (ER) "titration" (39% reduction at 5 y). Metformin (ER) was the only strategy that produced net saving across the time horizon; however, relative total health care system costs of other interventions vs inaction declined over time up to 15 y. Viet Nam was most sensitive to cost and parameter changes via a one-way sensitivity analysis. CONCLUSIONS: Metformin (ER) and lifestyle interventions for prediabetes offer promise for reducing T2D incidence. Metformin (ER) could reduce T2D patient numbers and health care costs, given concerns regarding adherence in the context of funding/reimbursement challenges for lifestyle interventions.

Improving Vaccine Assessment Pathways and Decision Making in the Polish Immunization Program.

This study examines the vaccine market access pathway in Poland to evaluate its efficiency and propose recommendations for its improvement. The research spans a comprehensive analysis of the vaccine assessment process, ranging from pre-registration to sustainability, encompassing critical components such as national immunization technical advisory groups (NITAGs), health technology assessments, resource evaluations, and decision making. This investigation utilizes a multi-phase approach. Initial desk research aimed to collect accumulated evidence about each step of the vaccine access pathway. This constituted the background for an expert panel discussion (n = 13) and a final online questionnaire (n = 12), evaluating the timeframes, inclusiveness, transparency, and consistency of the elements of the process. Poland is a late adopter of new vaccines. The country faces budget constraints and lacks a formalized framework for the inclusion of vaccines into the national immunization program. Notably, NITAGs play a crucial role, yet their limited resources and dependence on public health stakeholders diminish their impact. A formal and well-supported advisory body may become a foundation for decision-making processes. The health technology assessment conducted by the national agency is recognized for its timeliness and transparency, though the absence of fiscal analyses in vaccine assessments is identified as a gap that limits the understanding of the value of vaccinations. Resources are key drivers of decision making, and recent changes in legislation offer increased flexibility in financing vaccines. Challenges in the procurement process include a limited consideration of non-acquisition costs and an increased absence of a documented general strategy for immunization program development in Poland, pointing to a need for strategic planning. In conclusion, this study recommends the establishment of a robust NITAG with enhanced resources, incorporating fiscal analyses, transparent resource allocation, and strategic planning for immunization program development. Addressing these recommendations is crucial for optimizing Poland's vaccine market access pathway, ensuring timely and efficient population-wide vaccine access.

Leveraging the holistic benefits of biosimilars in Europe - part 2: how payers can safeguard the future of a healthy biosimilar market environment.

INTRODUCTION: Biosimilars have improved access to biologic medicines; however, historical thinking may jeopardize the viability of future markets. AREAS COVERED: An expert panel of eight diverse European stakeholders provided insights about rethinking biosimilars and cost-savings, reducing patient access inequalities, increasing inter-market equity, and improving education. The insights reported here (Part 2) follow a study that provides perspectives on leveraging the holistic benefits of biosimilars for market sustainability based on independent survey results and telephone interviews of stakeholders from diverse biosimilar markets (Part 1). Directional recommendations are provided for payers. EXPERT OPINION: The panel's market maturity framework for biosimilars has three stages: 'Invest,' 'Expand' and 'Harvest.' Across market stages, re-thinking the benefits of biosimilars beyond cost-savings, considering earlier or expanded access/new indications, product innovations, and re-investment of biosimilar-generated cost-savings should be communicated to stakeholders to promote further engagement. During 'Expand' and 'Harvest' stages, development of efficient, forward-looking procurement systems and mechanisms that drive uptake and stabilize competition between manufacturers are key. Future biosimilars will target various therapy areas beyond those targeted by existing biosimilars. To ensure a healthy, accessible future market, stakeholders must align their objectives, communicate, collaborate, and coordinate via education, incentivization, and procurement, to maximize the totality of benefits.

Capturing the holistic value of biosimilars in Europe - part 1: a historical perspective.

INTRODUCTION: Approved biosimilars exhibit comparable efficacy, safety, and immunogenicity to reference products. This report provides perspectives on the societal value of biosimilars within Europe and potential factors that have influenced market dynamics. METHODS: An independent, self-administered survey or one-on-one in-depth interview was used to collect viewpoints about the impact of biosimilar medicines within European markets. Key insights were also sought from an expert panel of European stakeholders. RESULTS: Survey respondents were clinicians, pharmacists, and payers from Europe (N = 103). Perceived benefits of biosimilars included increased access to innovative medicines (73% of respondents) or biologic treatments (66%). Biosimilar competition was thought to expand access to biologics (~50% of respondents) or drug combinations (~36%) and reduce biologic access time (34%). Key drivers of biologic access after biosimilar competition included increased biologic awareness (51%) and changes to prescribing guidelines (37%) and/or treatment paradigms (28%). The expert panel developed a market maturity framework of biosimilar adoption/opportunities comprising three stages: 'Invest,' 'Expand,' and 'Harvest.' Findings were supported by published literature. CONCLUSIONS: In Europe, the perceptions of well-informed survey/interview respondents are that biosimilars have improved patient outcomes via increased access to biologics and innovative biologic products, contributing to earlier and longer treatment of a broader population.

The Impact of Hospital Pharmacy Operation on the Quality of Patient Care.

This paper presents the role, tasks, and place of a hospital pharmacy in the structure of the entire facility. The role of hospital drug management and pharmacy seems to be extremely important in providing patients with high-quality care. Particular emphasis was placed on the distribution systems of medicinal products and medical devices in the hospital. The advantages and disadvantages of the classical distribution system and modern systems such as unit-dose and multi-dose-and the most important differences between them-are presented. Difficulties related to implementing modern distribution systems in hospitals were also discussed. The information provided is presented in the context of the legal regulations in Poland.

Awareness, Attitudes and Willingness to Donate Biological Samples to a Biobank: A Survey of a Representative Sample of Polish Citizens.

Biotechnology is developing at an ever-increasing pace, and the progressive computerization of health care and research is making it increasingly easy to share data. One of the fastest growing areas is biobanking. However, even with the best equipment and the best trained staff, a biobank will be useless without donors. For this reason, we have decided to gauge Polish citizens' awareness and attitudes towards biobanking and their willingness to donate biological samples. For this purpose the survey was conducted among a nationwide group of 1052 Poles aged 18 and over where the totals for gender, age and place of residence were selected according to their representation in the total population of adult Poles. The survey was conducted using the Computer Assisted Web Interview (CAWI) technique. Approximately two thirds of respondents N = 701 (66.6%) indicated that they had heard of scientific studies in which samples of biological material such as blood, saliva or urine are collected. More than half of respondents (N = 613, 58.3%) had a positive opinion regarding scientific research in which samples of biological material are taken. Only N = 220 (20.9%) of respondents had previously encountered the term biobanking. More than a half N = 687 (65.3%) of respondents would participate in a scientific study that biobanked biological material and health information. Almost half of the respondents (48.0%) would like specific consent to be used in biobanking. In our study we observed a negligible correlation between socio-demographic factors and a willingness to donate biological material to a biobank. Considering the results presented above, the level of knowledge and awareness of biobanks, and their role in scientific research and the health care system, among Polish citizens is low and requires education and information activities.

Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland.

BACKGROUND: Spinal muscular atrophy (SMA) is a debilitating neuromuscular disease resulting in children's mortality and disability. Nusinersen is available to all SMA patients in Poland since 2019. AIM: To compare mortality or disease progression to mechanical ventilation in two patient cohorts before and after the program's introduction. Additionally, to describe the patient population treated with nusinersen and costs incurred by the public payer. METHODS: We used the National Health Fund (NHF) database to identify patients born in either 2014 or 2019, who received at least two health services with an ICD10 G12 diagnosis. Outcomes were time to event: death or first mechanical ventilation. We identified all benefits received by nusinersen-treated patients, between 1 January 2019 and 31 May 2022. RESULTS: Children with SMA born in 2019 had significantly lower mortality in the first years of their lives than children born in 2014. Approximately 875 patients (all age groups) were treated with nusinersen in the analysis period. The cost of causal drugs in this period amounted to €51.4 million. The cost of healthcare benefits amounted to €14.9 million. CONCLUSIONS: The drug program to treat SMA improved patient care in Poland. The NHF database was a reliable source to monitor resource-intensive therapies' costs, demography, and selected patient outcomes.

Guidance on using real-world evidence from Western Europe in Central and Eastern European health policy decision making.

Aim: Real-world data and real-world evidence (RWE) are becoming more important for healthcare decision making and health technology assessment. We aimed to propose solutions to overcome barriers preventing Central and Eastern European (CEE) countries from using RWE generated in Western Europe. Materials & methods: To achieve this, following a scoping review and a webinar, the most important barriers were selected through a survey. A workshop was held with CEE experts to discuss proposed solutions. Results: Based on survey results, we selected the nine most important barriers. Multiple solutions were proposed, for example, the need for a European consensus, and building trust in using RWE. Conclusion: Through collaboration with regional stakeholders, we proposed a list of solutions to overcome barriers on transferring RWE from Western Europe to CEE countries.