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.

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.

Respiratory Syncytial Virus Infections in Polish Pediatric Patients from an Expert Perspective.

Respiratory syncytial virus (RSV) is the most common pathogen causing respiratory tract infections in infants, affecting over 90% of children within the first two years of life. It may cause lower respiratory tract infections, which constitute a significant healthcare burden both in the primary and secondary care settings. Meanwhile, the data regarding RSV disease in Poland is scarce, and published data significantly differs from the numbers reported for other countries with longstanding surveillance and reporting systems. A literature review and an expert panel were conducted to (1) understand the healthcare burden of RSV infections in Poland; (2) collect data on infection seasonality, patient pathway, and management patterns; and (3) evaluate RSV infection surveillance in Poland. According to the literature, RSV is the major agent responsible for non-influenza respiratory diseases in Poland. The reported rates of hospitalization for RSV infections are 267.5/100,000 for children under 5 years of age and 1132.1/100,000 for those under 1 year of age. Comparisons with data from other countries suggest that these values may be underestimated, possibly due to insufficient access to microbiological testing and a low awareness of RSV. Infections occur mainly between December and April, however, this pattern has changed following the implementation of preventive measures for coronavirus disease 2019 in the past few years. According to available reports, bronchodilators, antibiotics, corticosteroids, and X-ray imaging have been frequently used. The surveillance system in Poland has limitations, but these may be overcome due to recent changes in healthcare law as well as the availability and reimbursement of diagnostic tests.

Recommendations for patient involvement in health technology assessment in Central and Eastern European countries.

Introduction: Meaningful patient involvement in health technology assessment (HTA) is essential in ensuring that the interests of the affected patient population, their families, and the general public are accurately reflected in coverage and reimbursement decisions. Central and Eastern European (CEE) countries are generally at less advanced stages of implementing HTA, which is particularly true for patient involvement activities. As part of the Horizon2020 HTx project, this research aimed to form recommendations for critical barriers to patient involvement in HTA in CEE countries. Methods: Built on previous research findings on potential barriers, a prioritisation survey was conducted online with CEE stakeholders. Recommendations for prioritised barriers were formed through a face-to-face workshop by CEE stakeholders and HTx experts. Results: A total of 105 stakeholders from 13 CEE countries completed the prioritisation survey and identified 12 of the 22 potential barriers as highly important. The workshop had 36 participants representing 9 CEE countries, and 5 Western European countries coming together to discuss solutions in order to form recommendations based on best practices, real-life experience, and transferability aspects. Stakeholder groups involved in both phases included HTA organisation representatives, payers, patients, caregivers, patient organisation representatives, patient experts, health care providers, academic and non-academic researchers, health care consultants and health technology manufacturers/providers. As a result, 12 recommendations were formed specified to the CEE region's context, but potentially useful for a broader geographic audience. Conclusion: In this paper, we present 12 recommendations for meaningful, systematic, and sustainable patient involvement in HTA in CEE countries. Our hope is that engaging more than a hundred CEE stakeholders in the study helped to spread awareness of the importance and potential of patient involvement and that the resulting recommendations provide tangible steps for the way forward. Future studies shall focus on country-specific case studies of the implemented recommendations.

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.

Recommendations to overcome barriers to the use of artificial intelligence-driven evidence in health technology assessment.

Background: Artificial intelligence (AI) has attracted much attention because of its enormous potential in healthcare, but uptake has been slow. There are substantial barriers that challenge health technology assessment (HTA) professionals to use AI-generated evidence for decision-making from large real-world databases (e.g., based on claims data). As part of the European Commission-funded HTx H2020 (Next Generation Health Technology Assessment) project, we aimed to put forward recommendations to support healthcare decision-makers in integrating AI into the HTA processes. The barriers, addressed by the paper, are particularly focusing on Central and Eastern European (CEE) countries, where the implementation of HTA and access to health databases lag behind Western European countries. Methods: We constructed a survey to rank the barriers to using AI for HTA purposes, completed by respondents from CEE jurisdictions with expertise in HTA. Using the results, two members of the HTx consortium from CEE developed recommendations on the most critical barriers. Then these recommendations were discussed in a workshop by a wider group of experts, including HTA and reimbursement decision-makers from both CEE countries and Western European countries, and summarized in a consensus report. Results: Recommendations have been developed to address the top 15 barriers in areas of (1) human factor-related barriers, focusing on educating HTA doers and users, establishing collaborations and best practice sharing; (2) regulatory and policy-related barriers, proposing increasing awareness and political commitment and improving the management of sensitive information for AI use; (3) data-related barriers, suggesting enhancing standardization and collaboration with data networks, managing missing and unstructured data, using analytical and statistical approaches to address bias, using quality assessment tools and quality standards, improving reporting, and developing better conditions for the use of data; and (4) technological barriers, suggesting sustainable development of AI infrastructure. Conclusion: In the field of HTA, the great potential of AI to support evidence generation and evaluation has not yet been sufficiently explored and realized. Raising awareness of the intended and unintended consequences of AI-based methods and encouraging political commitment from policymakers is necessary to upgrade the regulatory and infrastructural environment and knowledge base required to integrate AI into HTA-based decision-making processes better.

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.

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.

Seasonally Dependent Change of the Number of Fractures after 50 Years of Age in Poland-Analysis of Combined Health Care and Climate Datasets.

Aim: The incidence of fractures correlates with many independent and interrelated factors. The aim of the study was to examine trends in fracture incidence and to find possible reasons for changes. Materials and methods: A complete dataset of Polish population aged above 50 from the National Heath Fund­which is a single, state-owned payer for the health service procedures in Poland­covering the years between 2010 and 2015 was analyzed along with climate dataset. Results: The analysis indicated that there was a substantial and statistically significant decrease in the incidence of forearm and hip fractures (p = 0.007 and 0.007, respectively). On the other side, there was a statistically significant increase in incidence of humerus and lumbar fractures (p = 0.002, p < 0.001, respectively). The observed changes (especially decrease in forearm and hip fracture incidence) happened mostly in the cold season and were correlated to mean-temperature changes during the assessed time period. Conclusion: In the analysis based on the dataset obtained from fracture-related database collected in Poland in the years 2010−2015 in the population of patients over 50 years of age, we observed that the changes of fracture incidence during the observation period are associated with and may be dependent on the season (warmer versus colder) and on mean temperature increase during the observation period.

Use of single pill combinations in the treatment of arterial hypertension in Poland: The current practice and guidelines, the impact on reimbursement spending and patient co-payment.

BACKGROUND: Clinical guidelines recommend using single pill combinations (SPC) when initiating and intensifying the treatment of arterial hypertension (AH), which is not reflected in the Summaries of Product Characteristics (SMPC) for individual preparations. The drug reimbursement system in Poland (with a few exceptions) does not provide for reimbursement outside the indications specified in the SMPC. Therefore, it excludes the use of SPC under reimbursement. In 2020 the share of SPC in the treatment of AH amounted to 12.8% of unit volume and was lower than the 80% based on the guidelines of the Polish Society of Hypertension. METHODS: Using the data from a sample of pharmacies in Poland over the period November-December 2020, the potential was assessed of switching from existing AH therapy with monocomponent drugs containing selected combinations of active ingredients to the equivalent SPC. RESULTS: The potential of switching from AH treatment in the analyzed period using monocomponent drugs with the equivalent SPC amounted to 19% of unit volume (a reduction of 212M units), with the highest switch potential (43.9%) for drugs containing amlodipine. The public payer's savings would be EUR 12.3 million and patient savings would amount to EUR 5.0 million. CONCLUSIONS: Enabling reimbursement of SPC in Poland in line with the clinical guidelines can significantly increase the share of SPC in the treatment of AH, which will result in better health outcomes and a significant reduction in the payer's drug reimbursement spending and will lower the financial barrier for patients to access this type of treatment.

Impact of COVID-19 Pandemic on the Use of Antidepressant and Antianxiety Pharmaceuticals as Well as Sick Leave in Poland.

The COVID-19 pandemic caused a major upheaval to the lives of people and placed a strain on societal mental health. The aim of this research is to estimate the impact of the pandemic on the mental condition of the Polish population measured through the consumption of relevant medication and medical leave of absence from the workplace. METHODS: We analyzed national-level data on the consumption of pharmaceuticals used in clinical practice in Poland in the treatment of depression and anxiety alongside medical absence in the workplace using the Interrupted Time Series model to estimate the significance of the pandemic. RESULTS: We found no significant change regarding the consumption of pharmaceuticals with the development of the pandemic. Conversely, medical leaves of absence for psychiatric reasons increased significantly with the onset of COVID-19. The influence was strongest in the diagnosis of anxiety or reaction to severe stress and weakest in recurrent depression. CONCLUSION: The pandemic had a significant influence on the ability to work for psychiatric patients in Poland but did not change pharmaceutical use. Physicians should consider the mental health of patients impacted by the anti-epidemic measures. Further study is needed to fully understand the long-term impact of the pandemic on mental health in Poland.

Overall survival of patients with EGFR mutation-positive non-small-cell lung cancer treated with erlotinib, gefitinib or afatinib under drug programmes in Poland - real-world data.

INTRODUCTION: The aim of the study was to estimate the overall survival of patients with EGFR mutation-positive non-small-cell lung cancer treated with erlotinib, gefitinib or afatinib. MATERIAL AND METHODS: Real-world patients who received afatinib, erlotinib or gefitinib between 1 July 2012 and 30 October 2017 were analysed in five subgroups. RESULTS: Among 267 patients treated with afatinib financed as the first line of treatment, 76 (28.46%) deaths occurred. Median observation time was 12.8 months (95% CI: 11.2-13.9). Median OS was 22.8 months (95% CI: 19.2-27.1). Among 83 patients who received erlotinib financed exclusively as the second line of treatment the number of deaths was 74 (89.16%). Median observation time was 64.3 months (95% CI: 60.4-64.6). Median OS was 16 months (95% CI: 13.2-22.9). Among 622 patients who received erlotinib financed both as first and second line treatment, there were 400 (64.3%) deaths. Median observation time was 33.3 months (95% CI: 31.2-37.6). Median OS was 17.8 months (95% CI: 16.4-19.7). Among 137 patients who received gefitinib financed only as the first line of treatment, there were 128 (93.4%) deaths. Median observation time was 58.3 months (95% CI: 49.4-62.5). Median OS was 16 months (95% CI: 13.8-19.7). Among 348 patients who received gefitinib financed both as the first and second line of treatment the number of deaths was 208 (59.8%). Median observation time was 23.7 months (95% CI: 20.7-28.7). Median OS was 15.5 months (95% CI: 12.9-17.5). CONCLUSIONS: Our real-world data regarding OS confirm the benefits found in clinical trials from the use of afatinib, erlotinib or gefitinib. However, the lower overall survival rate of Polish patients compared to similar studies from other research centres suggests the need for deeper investigation of this issue.

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.

Prevalence of Chronic Polypharmacy in Community-Dwelling Elderly People in Poland: Analysis of National Real-World Database Helps to Identify High Risk Group.

Introduction: Multimorbidity often comes with age, making elderly people particularly prone to polypharmacy. Polypharmacy, in turn, is a risk factor for adverse drug reactions, drug-drug interactions, non-adherence to medication, negative health outcomes, and increased healthcare services utilization. The longer the exposure to polypharmacy is, the higher the risk of these consequences is. Therefore, a detailed assessment of the prevalence and drivers of chronic polypharmacy in the elderly is particularly important. Aim of study: To find out the prevalence of chronic polypharmacy in the elderly population of Poland, and to characterize the subgroup with the highest risk of this problem, using real-world data. Methodology: A retrospective analysis of data on dispensation and healthcare services utilization held by the national payer organization for the year 2018. Chronic polypharmacy was defined as possession, as a result of dispensation, of five or more prescribed drugs within 80% of each of the consecutive 6 months. Results: Chronic polypharmacy was found in 554.1 thousand patients, i.e. in 19.1% of the national 65+ cohort. On average, those patients were 76 years old, and 49.3% of them were female. The vast majority (68.6%) continued their polypharmacy for the period of the whole year. There was a marked variation in geographical distribution of chronic polypharmacy with the highest value of 1.7 thousand per 100,000 inhabitants in the Lódz Voivodeship. Patients exposed to chronic polypharmacy filled prescriptions from 4.5±2.36 healthcare professionals. The average number of drugs they used was 8.3±3.84 DDD per patient per day. The most often prescribed drugs were Metformin, Atorvastatin and Pantoprazole. The average annual hospitalisation rate in those patients was 1.03±2.4. Conclusion: This study was the first of this kind involving a nationwide assessment of chronic polypharmacy in Polish elderly people. We found that this problem affected one fifth of Polish older adults and it remains stable due to its direct relation to chronic conditions. Thus, our results confirm that this phenomenon is highly important for the national health policy and requires relevant interventions. The planned introduction of pharmaceutical care in Poland is expected to help in solving the problem.

Do Advanced Therapies Have a Future in the Low- and Middle-Income Countries - The Case of Bulgaria, Romania, and Poland.

Introduction: The significant therapeutic potential of the advanced therapies (ATs) has predetermined the increased interests in their development mainly in the context of rare diseases most of which are genetically determined. However, there are still many challenges in front of the health insurance funds related to the cost-effectiveness and budget impact issues of these therapies. Our aim was to review and analyze the potential of low- and middle-income countries for health technology assessment (HTA) of advanced therapies focusing on Bulgaria, Romania and Poland as reference countries. A literature review of the existing good practices related to HTA of advanced therapies across the world and comparison with the national reality were performed. A list of challenges and issues from the point of view of the payer institution of all analyzed countries was performed. Pilot recommendations on how to overcome the barriers were created based on the existing practices and the potential of the national system. Discussion: 15 out of 80 articles identified in PubMed were found as applicable to the study scope as most of them were published in the period 2019-2021. Undoubtedly, the main challenges correspond to the high treatment costs, the uncertainty in clinical effectiveness, and poor HTA methodological approaches applicable for ATs worldwide. The issues identified for low and middle-income countries are similar having as well the lack of enough qualified health economists for the purposes of assessment and appraisal of HTA dossiers of the advanced therapies, lack of adequate existing separate financial programs for those therapies, and not preparedness of the health system and the society as a whole for such therapies. Conclusions: Despite the difficulties and challenges, the advanced therapies can be defined as a futuristic therapy for which great discoveries are yet to come. Therefore, each country should consider the implementation of reliable and nationally oriented programs for HTA and adequate financial coverage of these therapies.

The Potential to Reduce Patient Co-Payment and the Public Payer Spending in Poland through an Optimised Implementation of the Generic Substitution: The Win-Win Scenario Suggested by the Real-World Big Data Analysis.

High medication costs are one of the major barriers to patient adherence. Medication affordability might be improved by generic substitution. The aim of this study was to assess the effectiveness of the implementation of generic substitution mechanisms in Poland. This was a retrospective analysis of nationwide real-world big data corresponding to dispensation of metformin preparations in 2019 in Poland. Relevant prescription and dispensation data were compared to assess the prevalence of generic substitution and its economic consequences. Among the 1,135,863 e-prescriptions analysed, a generic substitution was found in only 4.81% of the packs dispensed, based on e-prescriptions issued for metformin under its originator version and 2.73% under generic drugs. It is estimated that if these values were applied to the total Polish drug market, patients could lose the opportunity to lower their co-payment by 15.91% and the national payer to reduce its reimbursement expenditures by 8.31%. Our results point at the suboptimal implementation of generic substitution in Poland. Therefore, relevant actions need to be taken in order to maximise the benefits provided by this mechanism. It could not only lead to the win-win scenario in which both patients and the national payer are secured substantial savings, but it could also have a positive impact on patient adherence.