Population-level norm values by EQ-5D-3L in Hungary - a comparison of survey results from 2022 with those from 2000.

PURPOSE: Although population norms of the EQ-5D-3L instrument had been available in Hungary since 2000, their evaluation was based on a United Kingdom (UK) value set. Our objective was to estimate the population norms for EQ-5D-3L by using the new Hungarian value set available since 2020, to extend the scope to adolescents, and to compare with norms from 2000. METHODS: A cross sectional EQ-5D-3L survey representative of the Hungarian population was conducted in 2022. The EQ-5D-3L dimensional responses were analyzed by age and sex and compared with the survey from 2000, by estimating population frequencies with their 95% confidence intervals; index values were evaluated by both value sets. RESULTS: Altogether, 11,910 respondents, aged 12 or more (578 between 12 and 17), completed the EQ-5D-3L. There was a notable improvement in reporting problems for both sexes (age 35-64) regarding the pain/discomfort and anxiety/depression compared to 2000. Below the age 44, both sexes had an EQ-5D-3L index plateau of 0.98, while above the age 55, men tended to have numerically higher index values compared to women, with the difference increasing with older age. Improvement in dimensional responses were also translated to numerically higher index values for both sexes between ages 18 and 74 compared to 2000. Multivariate regression analysis showed that higher educational attainment, lower age, larger household size, and active occupational status were associated with higher index values. CONCLUSION: Over the past 22 years, there was a large improvement in HRQoL of the middle-aged to elderly men and women in Hungary.

Health states can be described by a combination of statements of health-related quality of life measures. 'Value sets' are numerical expressions of how preferred a health state is. The provision of population-level health-related quality of life estimates (also known as 'population norms') are expected to improve the precision of patient-level clinical decision making, and health economic and public health studies. However, preference towards these health states is influenced by culture, resulting in differences across populations. While responses for the EQ-5D-3L instrument for adults have been available in Hungary since 2000, the evaluation of these responses was based on a 'value set' from the United Kingdom, rather than a Hungarian one.This research, utilizing the newly introduced Hungarian 'value set' (available since 2020) for the EQ-5D-3L instrument, offers a larger sample size, inclusion of adolescents and potentially improved sampling compared to the prior research conducted in 2000. Comparison of the two surveys allows us to estimate changes in both dimensional responses and overall health-related quality of life of the population over a 20-year time horizon, while we also compare the impact of different 'value sets' on health-related quality of life assessment. A large EQ-5D-3L improvement was observed in middle-aged-to-elderly people.

Framework for developing cost-effectiveness analysis threshold: the case of Egypt.

BACKGROUND: Cost-effectiveness analyses rarely offer useful insights to policy decisions unless their results are compared against a benchmark threshold. The cost-effectiveness threshold (CET) represents the maximum acceptable monetary value for achieving a unit of health gain. This study aimed to identify CET values on a global scale, provide an overview of using multiple CETs, and propose a country-specific CET framework specifically tailored for Egypt. The proposed framework aims to consider the globally identified CETs, analyze global trends, and consider the local structure of Egypt's healthcare system. METHODS: We conducted a literature review to identify CET values, with a particular focus on understanding the basis of differentiation when multiple thresholds are present. CETs of different countries were reviewed from secondary sources. Additionally, we assembled an expert panel to develop a national CET framework in Egypt and propose an initial design. This was followed by a multistakeholder workshop, bringing together representatives of different governmental bodies to vote on the threshold value and finalize the recommended framework. RESULTS: The average CET, expressed as a percentage of the gross domestic product (GDP) per capita across all countries, was 135%, with a range of 21 to 300%. Interestingly, while the absolute value of CET increased with a country's income level, the average CET/GDP per capita showed an inverse relationship. Some countries applied multiple thresholds based on disease severity or rarity. In the case of Egypt, the consensus workshop recommended a threshold ranging from one to three times the GDP per capita, taking into account the incremental relative quality-adjusted life years (QALY) gain. For orphan medicines, a CET multiplier between 1.5 and 3.0, based on the disease rarity, was recommended. A two-times multiplier was proposed for the private reimbursement threshold compared to the public threshold. CONCLUSION: The CET values in most countries appear to be closely related to the GDP per capita. Higher-income countries tend to use a lower threshold as a percentage of their GDP per capita, contrasted with lower-income countries. In Egypt, experts opted for a multiple CET framework to assess the value of health technologies in terms of reimbursement and pricing.

Consensus-Based Recommendations for the Implementation of Health Technology Assessment in the United Arab Emirates.

OBJECTIVES: Healthcare research and development have accelerated at an unprecedented rate creating a challenge even for high-income countries to finance all new technologies. Health technology assessment (HTA) aims to maximize health gains out of available resources. Our study aimed to provide tailor-made recommendations for HTA implementation in the United Arab Emirates (UAE). METHODS: We conducted a policy survey to explore the gap between the current and preferred future environment of HTA implementation in the UAE. The survey was distributed in 2 workshops discussing the importance of HTA implementation, and results were further aggregated to yield a list of draft recommendations. Recommendations were then assessed for their feasibility in a round table discussion with experts in the field to generate actions for HTA implementation. RESULTS: Survey results and round table discussion indicated the need to leverage HTA for value-based decision making. Experts confirmed the unmet need for broader coverage of graduate and postgraduate HTA training with the aim of specific PhD programs in the UAE. Public funding for HTA appraisals and expanding the scope of HTA to nonpharmaceuticals was recommended. Furthermore, experts recommended that several HTA bodies should be coordinated centrally and highlighted the importance of having an explicit soft cost-effectiveness threshold for common technologies and a higher threshold for orphan drugs. CONCLUSIONS: Although HTA in the UAE is still in its early stages, strong initiatives are being taken for its implementation. Our survey results served as a step in developing a detailed action plan for HTA implementation that will enhance the sustainability of the healthcare system.

Strategic Approaches to Reducing the Burden of Atopic Dermatitis in the Middle East and Africa Region.

OBJECTIVES: Atopic dermatitis (AD) creates a significant burden on patients and society. This study proposes a set of health policy interventions that can reduce the burden of AD in the Middle East and Africa. METHODS: We conducted a scoping review to find relevant actions that have been implemented or recommended to decrease AD burden globally. An expert panel was conducted to discuss the review findings, then experts were surveyed to suggest the most efficient actions. Finally, survey results and recommendations were formulated into key actions to reduce the burden in the Middle East and Africa region. RESULTS: Recommended actions were related to 5 domains; capacity building, guidelines, research, public awareness, and patient support and education. Several actions related to each domain can help reduce the burden. One of the most advocated recommendations was investing in patient education through trained healthcare professionals. Understanding the disease and learning how to control it is a key cornerstone to treatment optimization and reducing the burden. Multidisciplinary care, publishing defined therapeutic guidelines, and investing in research were the most recommended actions based on the experts' discussion and survey results. CONCLUSIONS: Although the burden of AD is the highest among dermatological diseases, a well-grounded action plan has the potential to reduce the disease burden. Decision makers may develop a national AD action plan by selecting the most relevant items of this study based on their potential impact, feasibility, timeliness, and affordability.

Development of the Emirates Multi-Criteria Decision Analysis Tool for Orphan Drugs.

Background The number of orphan drug approvals is currently increasing globally. This creates a significant burden on payers and healthcare systems. This study aimed to create a multi-criteria decision analysis (MCDA) tool for evaluating orphan drugs within the United Arab Emirates (UAE). The intended result of the tool is to provide evidence-based guidance to decision-makers in reimbursement and procurement decisions. Methods We conducted a literature search and local expert interviews to identify relevant preliminary criteria for the MCDA tool. Then we conducted a structured consensus-building session for healthcare experts and decision-makers in the UAE to develop the Emirati MCDA tool for orphan drugs. The experts voted for the criteria to be included in the tool and their ranking according to importance, as well as the weight of each criterion and its scoring function. To improve understanding and facilitate the voting process, experts were provided with a brief illustration of similar tools conducted in other countries before the voting sessions. Finally, the tool was developed in a Microsoft Excel sheet (Microsoft Corporation, Redmond, Washington, United States), and it was validated and tested based on real case studies, then it was fine-tuned accordingly based on the experts' discussions. The final tool was provided to the attendees to guide their decisions in the reimbursement and procurement of orphan drugs. Results The created tool provides a score for each analyzed orphan drug based on its value. Ten criteria were included in the final MCDA tool. These were cost-effectiveness (25.1% of the weight), magnitude of health gain (20.1%), availability of therapeutic alternative (14.3%), disease severity (11%), budget impact (7.9%), disease rarity (5.6%), strength of clinical evidence (5.6%), burden on households (4.5%), indication uniqueness (3.2%), and patients' age (2.6%). Conclusions Implementation of evidence-based healthcare necessitates assessing the fair value of each health technology. Addressing the high unmet medical needs and improving healthcare for patients with rare diseases are priorities within the UAE. The created Emirates MCDA tool for orphan drugs has the potential to help decision-makers implement value-based and evidence-based reimbursement decisions for orphan drugs.

Importance of aligning the implementation of new payment models for innovative pharmaceuticals in European countries.

INTRODUCTION: The uptake of complex technologies and platforms has resulted in several challenges in the pricing and reimbursement of innovative pharmaceuticals. To address these challenges, plenty of concepts have already been described in the scientific literature about innovative value judgment or payment models, which are either (1) remaining theoretical; or (2) applied only in pilots with limited impact on patient access; or (3) applied so heterogeneously in many different countries that it prevents the health care industry from meeting expectations of HTA bodies and health care payers in the evidence requirements or offerings in different jurisdictions. AREAS COVERED: This paper provides perspectives on how to reduce the heterogeneity of pharmaceutical payment models across European countries in five areas, including 1) extended evaluation frameworks, 2) performance-based risk-sharing agreements, 3) pooled procurement for low volume or urgent technologies, 4) alternative access schemes, and 5) delayed payment models for technologies with high upfront costs. EXPERT OPINION: Whilst pricing and reimbursement decisions will remain a competence of EU member states, there is a need for alignment of European pharmaceutical payment model components in critical areas with the ultimate objective of improving the equitable access of European patients to increasingly complex pharmaceutical technologies.

Multistakeholder Perceptions of Additional Value Elements for United States Value Assessment of Health Interventions.

OBJECTIVES: Limitations in conventional cost-effectiveness methods have led to calls for incorporation of additional value elements in assessments of health technologies. However, gaps remain in how additional value elements may inform decision making. This study aimed to prioritize additional value elements from the perspective of healthy individuals without a specific condition or indicated for a specific treatment in the United States among a multistakeholder panel and compare the importance of perspective-specific value elements. METHODS: Additional value elements were prioritized in 2 phases: (1) we identified and categorized additional value elements in a targeted literature review, and (2) we convened a multistakeholder group-based preference elicitation study (N = 28) to evaluate the description of each value element and rank and generate normalized weights of each value element for its significance in value assessment. The importance of additional value elements was also weighted relative to patient-centric value elements. RESULTS: The rank and weight of contextual value elements among 28 stakeholders were "severity of the disease" (26.2%), "disadvantaged and vulnerable target populations highly represented" (21.8%), "broader economic impact" (17.3%), "risk protection" (13.8%), "rarity of the disease" (11.3%), and "novel mechanism of action" (9.7%). Relative weight of the additional value elements versus patient-centric value elements was 52% and 48%, respectively. CONCLUSIONS: Study findings may inform priority setting for value frameworks and emerging US government assessments. The group-based elicitation method is repeatable and useful for structured deliberative processes in value assessment and may help improve the consistency and predictability of what is important to stakeholders.

Frailty prevalence in 42 European countries by age and gender: development of the SHARE Frailty Atlas for Europe.

Comparative frailty prevalence data across European countries is sparse due to heterogeneous measurement methods. The Survey of Health, Ageing and Retirement (SHARE) initiative conducted interviews with probability sampling of non-institutionalized elderly people in several European countries. Previous frailty analyses of SHARE datasets were limited to initial SHARE countries and did not provide age- and gender-stratified frailty prevalence. Our aim was to provide age- and gender-stratified frailty prevalence estimates in all European countries, with predictions where necessary. From 29 SHARE participating countries, 311,915 individual surveys were analyzed. Frailty prevalence was estimated by country and gender in 5-year age bands using the SHARE Frailty Instrument and a frailty index. Association of frailty prevalence with age, gender, and GDP per capita (country-specific economic indicator for predictions) was investigated in multivariate mixed logistic regression models with or without multiple imputation. Female gender and increasing age were significantly associated with higher frailty prevalence. Higher GDP per capita, with or without purchasing power parity adjustment, was significantly associated with lower frailty prevalence in the 65-79 age groups in all analyses. Observed and predicted data on frailty rates by country are provided in the interactive SHARE Frailty Atlas for Europe. Our study provides age- and gender-stratified frailty prevalence estimates for all European countries, revealing remarkable between-country heterogeneity. Higher frailty prevalence is strongly associated with lower GDP per capita, underlining the importance of investigating transferability of evidence across countries at different developmental levels and calling for improved policies to reduce inequity in risk of developing frailty across European countries.

Cost-Effectiveness of Bariatric Surgery in Patients Living with Obesity and Type 2 Diabetes.

Aims: The favourable effects of bariatric surgeries on body weight reduction and glucose control have been demonstrated in several studies. Additionally, the cost-effectiveness of bariatric surgeries has been confirmed in several analyses. The aim of the current analysis was to demonstrate the cost-effectiveness of bariatric surgeries in obese patients with type 2 diabetes in Hungary compared to conventional diabetes treatments based on economic modelling of published clinical trial results. Materials and Methods: Patients entered the simulation model at the age of 45 with body mass index (BMI) ≥ 30 kg/m2 and type 2 diabetes. The model was performed from the public payer's perspective, comparing sleeve gastrectomy (SG) and Roux-en-Y gastric bypass (RYGB) procedures to conventional care of diabetes. The results were provided separately for three BMI categories. Results: The base-case analysis demonstrated that both surgery types were dominant; i.e., they saved 17 064 to 24 384 Euro public payer expenditures and resulted in improved health outcomes (1.36 to 1.50 quality-adjusted life years gain (QALY)) in the three BMI categories. Bariatric surgeries extended the life expectancy and the disease-free survival times of all the investigated diabetes complications. All the scenario analyses confirmed the robustness of the base-case analysis, such that bariatric surgeries remained dominant compared to conventional diabetes treatments. Conclusion: The results of this cost-effectiveness analysis highlight the importance of bariatric surgeries as alternatives to conventional diabetes treatments in the obese population. Therefore, it is strongly recommended that a wider population has access to these surgeries in Hungary.

A concept for multi-winner tenders for medicinal products with balancing between efficient prices, long-term competition and sustainability of supply.

Achieving price efficiency via tenders, the sustainability of competition, and the prevention of shortages are hot topics in the debates about shaping the pharmaceutical markets. Single-winner tenders receive growing criticism for concentrating on achieving low prices at the expense of the long-term maintenance of a competitive pharmaceutical industry, the security of continuous supply, and disregarding the therapeutic needs of patient populations with specific conditions. This paper aims at drafting a concept to assist the design of multi-winner tenders for medicinal products with a focus on supply and sales guarantees, price efficiency, and equity in access. The concept shall be generally applicable to all kinds of medicinal products including generics, biosimilars, and on-patent products in the out- and in-patient sector. Principles for multi-winner tenders for medicinal products are set and a number of delimitations are made in order to get rid of factors that prevent clairvoyance amid the various pricing and reimbursement systems when designing a concept. The steps to plan and implement a multi-winner tendering procedure are drafted on the basis of the defined principles. The tender should consist of planning, bidding, preparation, sales, and evaluation phases. Pharmaceutical companies shall make bids with price and quantity pairs, which shall be ranked by prices and if applicable then taking into account other factors. The tenderer shall predefine market shares to the various places of the ranking. A double ceiling shall be applicable for the sales of the winners: their sales must not exceed their quantity offer and the predefined market share applicable to their place in the ranking. The implementation of the concept will require the careful adjustment of the tender conditions to the specificities of the pharmaceutical market concerned on the one hand and to the local pricing and reimbursement system on the other hand.

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.

Maximizing the benefits of using biosimilars in Egypt.

BACKGROUND: Biosimilars constitute a pathway for sustainable financing of healthcare systems in the era of expensive biologics. However, such a pathway is not free of challenges. Since the biosimilars market is expanding in Egypt, there is an urgent need for a policy framework to optimize their use and diffusion in the market. We aim to characterize a national framework based on the experiences of other countries and consultation with local experts. METHODS: A narrative literature review was conducted to identify biosimilars' policy elements worldwide. A workshop was organized with experts to discuss the narrative review findings and create consensus on recommendations. RESULTS: The narrative literature review highlighted the need for biosimilar policy actions in four areas: market authorization, pricing, reimbursement, and uptake. Eighteen experts representing the Egyptian healthcare authorities attended the workshop. The most significant conclusions from the workshop included setting the price of the biosimilar at 30-40% less than its originator's price and establishing financing protocols, in which the more expensive biologics with significant price premiums should be excluded from the formulary. CONCLUSIONS: A summarized national framework policy recommendation for biosimilars was created by local experts from the main public healthcare entities in Egypt. These recommendations coincide with the international policies adopted across different countries that aim to improve patient access while sustaining health expenditure.

Incremental cost of premature birth - a public health care payer perspective from Hungary.

BACKGROUND: Preterm birth remains a significant burden to families, health systems and societies. The aim was to quantify the incremental prematurity-related public health expenditure in Hungary and to estimate the potential impact of a decrease in the prevalence of prematurity on the public payer's spending. METHODS: Over a 6-year time horizon, public financing data of inpatient, outpatient and pharmaceutical care for children born at ≥ 25 weeks of gestation in 2009/2010 were retrieved from the Hungarian National Health Insurance Fund database. In descriptive analysis, the public payer's spending was given as cost/capita. The impact of a decrease in prematurity prevalence was specified as the total budget impact. An exchange rate of 294 Hungarian forint/Euro was applied. RESULTS: A total of 93,124 children (including 8.6% who were premature babies) were included in the analysis. A strong negative relationship was shown between gestational age and per capita cost. The 6-year cost of care for the cohort born at 26 weeks of gestation (28,470 Euro per capita) was 24 times higher than that for the cohort born at 40 weeks. First-year inpatient spending accounted for the largest proportion of total health care spending across all gestational ages. All investigated prematurity complications (retinopathy of prematurity, necrotizing enterocolitis, bronchopulmonary dysplasia, intraventricular cerebral bleeding and leukomalacia) resulted in additional significant incremental spending. If 70% of pregnancies ending with preterm birth could be prolonged by 1 week, the savings would be almost 7.0 million Euros in the first 6 years of life. CONCLUSION: This comprehensive analysis of prematurity-related health care spending confirmed that premature infants have much higher costs for care than those born at term in Hungary. These quantitative outcomes can provide essential inputs for the cost-effectiveness analysis of medical technologies and public health interventions that can decrease the prevalence of premature birth. TRIAL REGISTRATION: Not applicable.

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.

Health technology assessment of tests for SARS-CoV-2 and treatments for COVID-19: A proposed approach and best-practice recommendations.

OBJECTIVES: To develop best-practice guidance for health technology assessment (HTA) agencies when appraising diagnostic tests for SARS-CoV-2 and treatments for COVID-19. METHODS: We used a policy sandbox approach to develop best-practice guidance for HTA agencies to approach known challenges associated with assessing tests and treatments for COVID-19. The guidance was developed by a multi-stakeholder workshop of twenty-one participants representing HTA agencies, clinical and patient experts, academia, industry, and a payer, from across Europe and North America. The workshop was supported by extensive background work to identify the key challenges, including: targeted reviews of existing COVID-related methods guidance for assessing interventions and clinical guidelines, engagement with clinical experts, a survey and workshop of HTA agencies, a systematic review of published economic evaluations, and a workshop of health economic modelers. RESULTS: We suggest HTA agencies should consider using other types of evidence (e.g., real world) where high-quality randomized controlled trials may be lacking and healthcare systems would value timely HTA outputs. A "living" HTA approach may be useful, given the context of an evolving disease, scientific understanding and evidence base, allowing for decisions to be efficiently revisited in response to new information; particularly, if supported by a common "disease model" for COVID-19. Innovative ways of engaging with the public and clinicians, and early engagement with regulators and payers, are recommended. CONCLUSIONS: HTA agencies should consider the elements of this guidance that are most suited to their existing processes to enable them to assess the effectiveness and value of interventions for COVID-19.

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.

Systematic Literature Review of Economic Evaluations of Treatment Alternatives in Chronic Lymphocytic Leukemia.

BACKGROUND: Economic evaluations are widely used to predict the economic impact of new treatment alternatives. Comprehensive economic reviews in the field of chronic lymphocytic leukemia (CLL) are warranted to supplement the existing analyses focused on specific therapeutic areas. METHODS: A systematic literature review was conducted based on literature searches in Medline and EMBASE to summarize the published health economics models related to all types of CLL therapies. Narrative synthesis of relevant studies was performed focusing on compared treatments, patient populations, modelling approaches and key findings. RESULTS: We included 29 studies, the majority of which were published between 2016 and 2018, when data from large clinical trials in CLL became available. Treatment regimens were compared in 25 cases, while the remaining four studies considered treatment strategies with more complex patient pathways. Based on the review results, Markov modelling with a simple structure of three health states (progression-free, progressed, death) can be considered as the traditional basis to simulate cost effectiveness. However, more recent studies added further complexity, including additional health states for different therapies (e.g. best supportive care or stem cell transplantation), for progression-free state (e.g. by differentiating between with or without treatment), or for response status (i.e. partial response and complete response). CONCLUSIONS: As personalized medicine is increasingly gaining recognition, we expect that future economic evaluations will also incorporate new solutions, which are necessary to capture a larger number of genetic and molecular markers and more complex patient pathways with individual patient-level allocation of treatment options and thus economic assessments.

Patient and Caregiver Experience Decision Factors in Treatment Decision Making: Results of a Systematic Literature Review of Multiple Myeloma Decision Aids.

OBJECTIVES: Decision-aids (DAs) may facilitate shared decision-making for patients and caregivers, by providing evidence-based information to assist healthcare professionals, patients, and caregivers in making choices about aspects of care, and/or highlighting decision factors to discuss with the potential of altering the treatment decision. These decision factors may not be well integrated in DAs. METHODS: A systematic literature review was conducted in the field of multiple myeloma (MM) on peer-reviewed publications, extended with a gray literature search. Data on whether and how patient and caregiver experience elements, other than survival and physical quality of life, were mentioned as decision factors in the identified MM DAs were extracted and analyzed qualitatively. RESULTS: Seventy MM DAs were found and analyzed; 51% of DAs mentioned any patient non-routinely assessed experience decision factors and only 17% mentioned any caregiver-related information. One hundred and forty potential decision factors were extracted, deduplicated and categorized into the following categories: 1) financial, 2) mode of administration / transportation issues, 3) personal beliefs and values, 4) emotional and social quality of life, 5) other medical information, 6) availability of social support, 7) caregiver burden. None of the DAs presented a comprehensive framework on all seven categories of decision factors being consider when mapping patient and caregiver experience value elements in MM. CONCLUSIONS: Based on available DAs, we recommend a set of patient and caregiver experience decision factors that have the potential to affect treatment choices of patients with MM, which should be included in DAs, including MM clinical guidelines.

Humanistic and Economic Burden of Atopic Dermatitis for Adults and Adolescents in the Middle East and Africa Region.

INTRODUCTION: Atopic dermatitis (AD) is a chronic skin disease that poses a significant burden on both patients and the society. AD causes the highest loss in disability-adjusted life years compared with other skin diseases. This study aimed to estimate the economic and humanistic burden of AD in adults and adolescents in seven countries in the Middle East and Africa region (Egypt, Lebanon, Saudi Arabia, Kuwait, Algeria, South Africa, and United Arab Emirates). METHODS: We conducted a literature review to identify country-specific data on this disease. Subsequently, meetings were organized with experts from each country to complete the missing data. The data were aggregated and calculation models were created to estimate the value of the humanistic and economic burden of the disease in each country. Finally, we conducted meetings with local experts to validate the results, and the necessary adjustments were made. RESULTS: On average, a patient with AD loses 0.19 quality-adjusted life years (QALYs) annually owing to this disease. The average annual healthcare cost per patient is highest in the United Arab Emirates, with an estimated value of US $3569 and a population-level indirect cost of US $112.5 million. The included countries allocated a range of 0.20-0.77% of their healthcare expenditure to AD-related healthcare services and technologies. The indirect cost of AD represents approximately 67% of the total disease cost and, on average, approximately 0.043% (range 0.022-0.059%) of the gross domestic product (GDP) of each country. CONCLUSION: Although the humanistic and economic burdens differ from country to country, AD carries a significant socioeconomic burden in all countries. The quality of life is severely affected by the disease. If AD is controlled, the costs, especially indirect costs, could decrease and the disease burden could be alleviated significantly.

Atopic dermatitis is a chronic condition characterized by inflamed and itchy skin. The prevalence and symptoms of atopic dermatitis are observed to increase in dry weather. Owing to its high prevalence in children, the majority of studies on atopic dermatitis are in children. Although it is also prevalent in adults and adolescents, its burden on adults has not been sufficiently studied, especially in Africa and the Middle East. This study quantified the burden of atopic dermatitis in adults and adolescents in seven countries in the Middle East and Africa. We estimated the economic and humanistic burden of this disease. We conducted a literature review and expert interviews to determine the effects on patients and caregivers. We created mathematical models to calculate the disease burden in each country, and local experts in each country validated the data. The study results showed that atopic dermatitis significantly affects the quality of life of patients. The direct medical costs of treatment in each country were calculated. The management of atopic dermatitis consumes around 0.20­0.77% of the healthcare expenditure in a country. The indirect cost of atopic dermatitis represents 0.022­0.059% of the gross domestic product (GDP) of a country. The country-specific burden data are essential to guide decision-makers in arriving at evidence-based decisions and efficiently allocating available resources. This study focused on the significant indirect economic burden of the disease, which can sometimes be underestimated because the disease is not fatal.

Framework for Patient Experience Value Elements in Rare Disease: A Case Study Demonstrating the Applicability of Combined Qualitative and Quantitative Methods.

BACKGROUND AND OBJECTIVE: Several novel methods have been suggested to extend a conventional value assessment to capture a more comprehensive perspective of value from a patient perspective. The objective of this research was to demonstrate a framework for implementing a combined qualitative and quantitative method to elicit and prioritize patient experience value elements in rare diseases. Neuromyelitis optica spectrum disorder was used as a case study. METHODS: The method for eliciting and prioritizing patient experience value elements involved a three-step process: (1) collecting potential patient experience value elements from existing literature sources followed by deliberation by a multi-stakeholder research team; (2) a pre-workshop webinar and survey to identify additional patient-reported value elements; and (3) a workshop to discuss, prioritize the value elements using a swing weighting method. Outcomes were prioritized value elements with normalized weights for patients considering a treatment for neuromyelitis optica spectrum disorder. RESULTS: A literature review and deliberation resulted in the following initial value elements: ability to reach important personal milestones, patient's financial burden, value of hope/balance or timing of risks and benefits, Uncertainty about long-term benefits and safety of the treatment, Patient empowerment through therapeutic advancement and technology, Caregiver/family's financial burden, patient experience related to treatment regimen, Therapeutic options, and Caregiver/family's quality of life. Eight patients with neuromyelitis optica spectrum disorder participated in the case study. In the online survey, participants found the nine proposed patient experience value elements both understandable and important with no additions. During the workshop, 'Uncertainty about long-term benefits and safety,' 'Patient experience related to treatment regimen,' and 'Patient's financial burden' were found to be the most important patient experience value elements, with a respective weight of 25%, 19.2%, and 14.4% (out of total 100%). CONCLUSIONS: This case study provides a framework for eliciting and prioritizing patient experience value elements using direct patient input. Although elements/weights may differ by disease, and even in neuromyelitis optica spectrum disorder, additional research is needed, value frameworks, researchers, and manufacturers can use this practical method to generate patient experience value elements and evaluate their impact on treatment selection.