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Radeva JI, Reed SD, Kaló Z, Kauf TL, Cantu E III, Cretin N, Schulman KA. Economic evaluation of everolimus vs. azathioprine at one year afterde novoheart transplantation.Clin Transplant 2004 DOI: 10.1111/j.1399-0012.2004.00312.x© Blackwell Munksgaard, 2004Everolimus decreases acute rejection and cardiac allograft vasculopathy after heart transplantation. We compared within-trial costs and resource use over 1 yr of follow-up inde novoheart transplant patients randomized to everolimus 1.5 mg/d (n = 209), everolimus 3.0 mg/d (n = 211), or azathioprine (n = 214).Resource use data were collected prospectively for 634 patients from 14 countries. We used the nonparametric bootstrap method to test for differences in mean costs and to estimate confidence intervals for cost-effectiveness ratios.Everolimus patients had lower incidence of efficacy failure compared with azathioprine patients (41.6%, everolimus 1.5 mg; 32.2%, everolimus 3.0 mg; 52.8%, azathioprine). Compared with patients receiving azathioprine, everolimus patients spent more days in the hospital [36.3 d for everolimus 1.5 mg/d (p = 0.21); 38.4 d for everolimus 3.0 mg/d (p = 0.01); 32.2 d for azathioprine]. Mean total costs, excluding the study medications, were not significantly different among treatment groups ($72 065 for everolimus 1.5 mg;$72 631 for everolimus 3.0 mg;$70 815 for azathioprine).Over 1 yr of follow-up after heart transplantation, everolimus did not significantly increase treatment costs, excluding the costs of the study medications, while reducing efficacy failure. Longer follow-up and the cost of everolimus are required to fully evaluate the cost-effectiveness of everolimus vs. azathioprine in post-transplant maintenance. [ABSTRACT FROM AUTHOR]

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. 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. 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. 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. [ABSTRACT FROM AUTHOR]

Background: In Kuwait, the government is planning to improve the specifications for purchase of medicine and to improve the tendering system intending to slow the growth of the expenditure for medicine and to improve the sustainability of the healthcare system. Multiple Criteria Decision Analysis (MCDA) is a method which can help to assess multiple and sometimes conflicting criteria in the evaluation of the available alternatives. The objective of this initiative was to develop collaboratively a MCDA tool which is locally relevant, and which could be used to improve the use of performance indicators in the purchasing of off-patent pharmaceuticals. Methods: Nineteen leading experts representing a broad range of pharmaceutical policy stakeholders elaborated a locally adapted MCDA format by following a 7-step process for criteria selection, scoring, ranking and weighting. Results: The most important criterion was the price measured as savings versus the originator product with a weight of 35% in the final decision and a full score with a 60% price reduction. In addition, eight criteria were considered important for assessing the product performance in the Kuwaiti healthcare system: 'equivalence with the reference product' (weight of 16.2%), 'stability and drug formulation' (13.5%), 'quality assurance' (11.2%), 'reliability of drug supply' (8.8%), 'macroeconomic benefit' (5.5%), 'real world outcomes (clinical and economic)' (4.2%), 'pharmacovigilance' (3.3%), and 'added value services related to the product' (2.3%). Conclusions: A MCDA model was successfully adapted to the Kuwait decision context by a group of Kuwaiti pharmacists from a broad range of institutions. The participants agreed with the approach and considered it suitable to improve the transparency and consistency of decision making for off-patent pharmaceuticals in Kuwait. A pilot implementation project was proposed. [ABSTRACT FROM AUTHOR]

Background: OnkoNetwork is a recently established integrated care model with a personalized pathway system to manage patients with first suspect of a solid tumour in secondary care, that evolved as a regional initiative in Hungary. The primary aim of OnkoNetwork is the improvement of clinical outcomes via timely access to quality assured and defragmented healthcare services. The Horizon 2020 funded SELFIE project has selected OnkoNetwork for in-depth qualitative and quantitative evaluation. The aim of this study was to provide a qualitative evaluation of OnkoNetwork along the six components of the SELFIE conceptual framework: 1) service delivery, 2) leadership and governance, 3) workforce, 4) financing, 5) technologies and medical products, and 6) information and research.Methods: Analysis of published and grey programme documentation, followed by 20 semi-structured interviews with representatives of programme initiators, general and financial managers, involved physicians and non-physician professionals, patients and their informal caregivers. Transcripts of all interviews were analysed by Mayring's content analysis method by two independent researchers.Results: This study yielded the first comprehensive description of the programme. OnkoNetwork is a blue dahila in Central and Eastern Europe, providing timely and quality-assured healthcare services for the target patients by personalized patient path monitoring and management in a financially sustainable manner without macro-level financing of its operation. Innovative professional roles were implemented for non-physicians and physicians, and a supporting information technology application was developed.Conclusions: This paper provides a systematic description of OnkoNetwork on the six components of the SELFIE conceptual framework for integrated care in multimorbidity to understand how and why OnkoNetwork was implemented and cares (better) for its patients. Because integrated care models are designed and adjusted to their specific local needs and context, those few successful and sustainable models that were established in Central and Eastern European countries represent important benchmarks for other initiatives in this region. Experience with OnkoNetwork during its planning, implementation and operation including the description of key success factors and barriers as perceived by various stakeholder groups, may support the development of further integrated care models especially in countries with similar economic status and healthcare settings. [ABSTRACT FROM AUTHOR]

Background: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU).Objectives: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states.Methods: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries.Results: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures.Conclusions: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level. [ABSTRACT FROM AUTHOR]

Background: A Multi Criteria Decision Analysis (MCDA) technique was adopted to reveal the preferences of the Appraisal Body of the Polish HTA agency towards orphan drugs (OMPs).Results: There were 34 positive and 23 negative HTA recommendations out of 54 distinctive drug-indication pairs. The MCDA matrix consisted of 13 criteria, seven of which made the most impact on the HTA process. Appraisal of clinical evidence, cost of therapy, and safety considerations were the main contributors to the HTA guidance, whilst advancement of technology and manufacturing costs made the least impact.Conclusions: MCDA can be regarded as a valuable tool for revealing decision makers' preferences in the healthcare sector. Given that only roughly half of all criteria included in the MCDA matrix were deemed to make an impact on the HTA process, there is certainly some room for improvement with respect to the adaptation of a new approach towards the value assessment of OMPs in Poland. [ABSTRACT FROM AUTHOR]

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 b o d y m a s s i n d e x B M I ≥ 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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

Introduction: Although previously regarded as a children's disease, it is clear that atopic dermatitis (AD) is also highly prevalent in adults. Because AD is not associated with mortality, it is usually neglected compared with other, fatal diseases. However, several studies have highlighted that AD burden is significant due to its substantial humanistic burden and psychosocial effects. This study aims to summarize and quantify the clinical, economic, and humanistic burden of AD in adults and adolescents. Methods: A systematic literature search was performed in PubMed, Scopus, Cochrane, Centre for Reviews and Dissemination (CRD), EconPapers, The Professional Society for Health Economics and Outcomes Research (ISPOR), The National Institute for Health and Care Excellence (NICE), and The Canadian Agency for Drugs and Technologies in Health (CADTH). Studies were included if they reported clinical, economic, or humanistic effects of AD on adults or adolescents, from January 2011 to December 2020. The Grading of Recommendations Assessment tool was used to assess risk of bias for the included studies. Regression models were used to explain the correlation between factors such as disease severity and quality of life (QoL). Results: Among 3400 identified records, 233 studies were included. Itch, depression, sleep disturbance, and anxiety were the most frequently reported parameters related to the clinical and humanistic burden of AD. The average utility value in studies not stratifying patients by severity was 0.779. The average direct cost of AD was 4411 USD, while the average indirect cost was 9068 USD annually. Conclusions: The burden of AD is significant. The hidden disease burden is reflected in its high indirect costs and the psychological effect on QoL. The magnitude of the burden is affected by the severity level. The main limitation of this study is the heterogeneity of different studies in terms of data reporting, which led to the exclusion of potentially relevant data points from the summary statistics. Plain Language Summary: Atopic dermatitis is a very common skin disease among children and adults. The disease is nonfatal but may lead to patients and families having a low quality of life and decreased productivity, especially in its severe state. Because atopic dermatitis is more common in children than adults, most published research is directed to studying the effect of the disease on children. Atopic dermatitis affects patients' health, quality of life, financial state, and productivity. Therefore, our study aims to study and quantify the burden caused by the disease represented in the clinical burden, humanistic burden, and economic burden. We conducted a systematic literature review to determine all relevant studies providing specific values for the burden. The studies included are those providing information on the percentage of patients affected by specific symptoms, costs paid for treatment, number of days of productivity lost due to the disease, and quality-of-life questionnaire results for patients with atopic dermatitis or their caregivers. We analyzed the data from all relevant studies to calculate average values and quantify the burden. The results of our study should help healthcare sector decision-makers in understanding the real effect of the disease on adults and adolescents and rearrange their priorities for treating different diseases based on the specific burden of each disease. [ABSTRACT FROM AUTHOR]

OnkoNetwork is a patient navigation program established in the Moritz Kaposi General Hospital to improve the timeliness and completeness of cancer investigations and treatment. The H2020 SELFIE consortium selected OnkoNetwork as a promising integrated care initiative in Hungary and conducted a multicriteria decision analysis based on health, patient experience, and cost outcomes. In this paper, a more detailed analysis of clinical impacts is provided in the largest subgroup, non-small cell lung cancer (NSCLC) patients. A retrospective cohort study was conducted, enrolling new cancer suspect patients with subsequently confirmed NSCLC in two annual periods, before and after OnkoNetwork implementation (control and intervention cohorts, respectively). To control for selection bias and confounding, baseline balance was improved via propensity score weighting. Overall survival was analyzed in univariate and multivariate weighted Cox regression models and the effect was further characterized in a counterfactual analysis. Our analysis included 123 intervention and 173 control NSCLC patients from early to advanced stage, with significant between-cohort baseline differences. The propensity score-based weighting resulted in good baseline balance. A large survival benefit was observed in the intervention cohort, and intervention was an independent predictor of longer survival in a multivariate analysis when all baseline characteristics were included (HR = 0.63, p = 0.039). When post-baseline variables were included in the model, belonging to the intervention cohort was not an independent predictor of survival, but the survival benefit was explained by slightly better stage distribution and ECOG status at treatment initiation, together with trends for broader use of PET-CT and higher resectability rate. In conclusion, patient navigation is a valuable tool to improve cancer outcomes by facilitating more timely and complete cancer diagnostics. Contradictory evidence in the literature may be explained by common sources of bias, including the wait-time paradox and adjustment to intermediate outcomes. [ABSTRACT FROM AUTHOR]

Background: Multi- criteria decision analysis (MCDA) can assist policymakers in objectively choosing between alternative therapeutic options based on multiple value attributes. Our aim was to create an MCDA tool for the national tenders of off-patent oncology medicines in Egypt. Methods: An initial list of criteria was developed through a literature review complemented by local expert interviews. Price or cost-related criteria were excluded to abide by the national regulations of the tender process. Next, a workshop hosting diversified stakeholders representing different governmental bodies was held. Anonymous voting was used to rank and weigh the criteria as well as assigning scores. Price was added as a separate step to identify best option based on price per point. The tool was then tested on a national tender sample of off-patent oncology medicines to assess its performance, and it was readjusted accordingly in a second workshop. Results: Seven non-price criteria were selected, including use in reference countries (23.49% weight), equivalence with the reference product (18.79%), manufacturing quality (15.53%), provision of pharmacovigilance services (12.94%), supply reliability (10.78%), previous use in local settings (9.8%) and macroeconomic benefit (8.67%). A medicine receives a score ranging from 0 to 100% of each criterion's weight. The aggregated score is calculated on a hundred-point scale. Based on participants' consensus, an overall score of 65 was set as a cut-off for passing the technical eligibility phase of the tendering process. Any product receiving a lower score would be disqualified from the tender. For qualified products, the lower price per point represents preferential option for the national tender. Conclusions: The created MCDA tool is capable of objectively comparing similar off-patent oncology medicines by considering multiple value attributes and providing reliable scoring functions for each. [ABSTRACT FROM AUTHOR]

Background: The Palliative Care Consult Service (PCCS) programme was among the first initiations in Hungary to provide palliative care for patients admitted to hospital. The PCCS team provides palliative care for mainly cancer patients and their family members and manages the patient pathway after being discharged from the hospital. The service started in 2014 with 300–400 patient visits per year. The aim of this study is to give a comprehensive overview of the PCCS programme guided by a conceptual framework designed by SELFIE ("Sustainable intEgrated chronic care modeLs for multi-morbidity: delivery, FInancing, and performancE"), a Horizon2020 funded EU project and to identify the facilitators and barriers to its wider implementation. Methods: PCCS has been selected by the SELFIE consortium for in-depth evaluation as one of the Hungarian integrated care models for persons with multi-morbidity. The qualitative analysis of the PCCS programme was based on available documents of the care provider and interviews with different stakeholders related to the programme. Results: The integrated, multidisciplinary and patient-centred approach was well-received among the patients, family members and clinical departments, as verified by the increasing number of requests for consultations. As a result of the patient pathway management across providers (e.g. from inpatient care to homecare) a higher level of coordination could be achieved in the continuity of care for seriously-ill patients. The regulatory framework has only partially been established, policies to integrate care across organizations and sectors and adequate financial mechanism to support the enhancement and sustainability of the PCCS are still missing. Conclusions: The service integration of palliative care could be implemented successfully in an academic hospital in Hungary. However, the continuation and enhancement of the programme will require further evidence on the performance of the integrated model of palliative care and a more systematic approach particularly regarding the evaluation, financing and implementation process. [ABSTRACT FROM AUTHOR]

Background: Successful development of new treatments for rare diseases (RDs) and their sustainable patient access require overcoming a series of challenges related to research and health technology assessment (HTA). These impediments, which may be unique to RDs or also apply to common diseases but are particularly pertinent in RDs, are diverse and interrelated.Objective: To develop for the first time a catalog of primary impediments to RD research and HTA, and to describe the cause and effect of individual challenges.Methods: Challenges were identified by an international 22-person expert working group and qualitative outreach to colleagues with relevant expertise. A broad range of stakeholder perspectives is represented. Draft results were presented at annual European and North American International Society for Pharmacoeconomics and Outcomes Research (ISPOR) congresses, and written comments were received by the 385-strong ISPOR Rare Disease Review Group from two rounds of review. Findings were refined and confirmed via targeted literature search.Results: Research-related challenges linked to the low prevalence of RDs were categorized into those pertaining to disease recognition and diagnosis, evaluation of treatment effect, and patient recruitment for clinical research. HTA-related challenges were classified into issues relating to the lack of a tailored HTA method for RD treatments and uncertainty for HTA agencies and health care payers.Conclusions: Identifying and highlighting diverse, but interrelated, key challenges in RD research and HTA is an essential first step toward developing implementable and sustainable solutions. A collaborative multistakeholder effort is required to enable faster and less costly development of safe, efficacious, and appropriate new RD therapies that offer value for money. [ABSTRACT FROM AUTHOR]

Rationale, aims, and objectives To compare the effect of osteoporotic fractures and complications of diabetes mellitus on quality of life (QoL). Method A cross-sectional study was performed in 840 patients with osteoporosis and in 943 patients with diabetes in Hungary to estimate the effect of osteoporotic fractures and microvascular and macrovascular complications of diabetes on QoL using the EQ-5D questionnaire. Ordinary least-squares regression was performed for the analysis to control for age and gender. Results The effects of certain of osteoporotic fractures and diabetes complications were similar in size measured by the EQ-5D. Patients with hip fractures and compressions of the vertebrae suffered more than 0.2 drop in their QoL, which is comparable in size to the most severe complications of diabetes, such as vision loss and amputations. Conclusions The use of mortality and premature mortality as the traditional measures of disease burden in public health policy making means that diseases which strongly affect QoL but less survival might not get the necessary priority. This is especially the case in low-income and middle-income countries where studies on QoL are scarce. Our comparative analysis, which showed that osteoporotic fractures reduce QoL as much as major complications of diabetes, highlights the need for comprehensive disease burden assessment, including losses in functionality and QoL, to support decision making. [ABSTRACT FROM AUTHOR]

• Highly priced but uncertain pharmaceutical innovations challenge healthcare reimbursement systems. • CEE and ME payer representatives indicate they want to mitigate these challenges by using outcome-based reimbursement and delayed payment models more often. • Financial-based reimbursement and upfront payment models are currently most applied. • Many relevant barriers for implementation of outcome-based reimbursement and delayed payment models are listed. • The development of an implementation framework could aid the feasibility of a successful implementation of the preferred models. There is growing interest in innovative reimbursement and payment models in Central and Eastern European (CEE) and Middle Eastern (ME) countries. A questionnaire was sent to payers from CEE and ME countries regarding the current use of, future preferences for and perceived barriers with these models. Twenty-seven healthcare payers from 11 countries completed the survey. Results showed participants preferred using outcome-based reimbursement models and delayed payment models more often; however, currently they are rarely applied. Barriers hindering implementation were mostly related to IT and data infrastructure, measurement issues, transaction costs and the administrative burden. Given these barriers highlighted in our study, policymakers should focus on the development of an implementation framework with contract templates for the preferred reimbursement and payment schemes to aid the feasibility of a successful implementation. [ABSTRACT FROM AUTHOR]

Health care decisions are complex and involve confronting trade-offs between multiple, often conflicting objectives. Using structured, explicit approaches to decisions involving multiple criteria can improve the quality of decision making. A set of techniques, known under the collective heading, multiple criteria decision analysis (MCDA), are useful for this purpose. In 2014, ISPOR established an Emerging Good Practices Task Force. The task force's first report defined MCDA, provided examples of its use in health care, described the key steps, and provided an overview of the principal methods of MCDA. This second task force report provides emerging good-practice guidance on the implementation of MCDA to support health care decisions. The report includes: a checklist to support the design, implementation and review of an MCDA; guidance to support the implementation of the checklist; the order in which the steps should be implemented; illustrates how to incorporate budget constraints into an MCDA; provides an overview of the skills and resources, including available software, required to implement MCDA; and future research directions. [ABSTRACT FROM AUTHOR]

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.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.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%).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.Methods: 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.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.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%).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.Results: 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.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.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%).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.Conclusions: 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.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.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%).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. [ABSTRACT FROM AUTHOR]

Background: In the evolving healthcare landscape of the United Arab Emirates (UAE), establishing cost-effectiveness thresholds (CETs) is pivotal to informing decision-makers about the value of health technologies., Objective: This study aimed to establish CETs for the UAE that harmonise with international standards while reflecting the nation's unique healthcare needs and economic context., Setting: United Arab Emirates., Methods: A multitiered methodology was employed, involving a literature review, a panel of national experts and workshops with key stakeholders, including healthcare providers, government health departments and healthcare payers. The panel and workshops were integral in assessing global CET practices and their applicability to the UAE providing a preliminary framework for CET in the UAE. Structured voting sessions were then conducted allowing voting on crucial aspects of CET to determine the baseline threshold, multipliers for severity, rarity and health gain, and methodologies for quantifying disease severity., Results: CETs were linked to the economic status of the UAE, with a baseline threshold of 0.75 times the gross domestic product per capita for one quality-adjusted life year gained. A multiplier system was introduced to reflect societal views on disease severity, disease rarity and the relative health benefit of health technologies. Based on the voting results, disease rarity was deemed the most crucial factor, receiving a maximum multiplier of 3X, while severity and health gain were assigned a maximum of 2X. The multiplier values for both disease severity and relative health gain would be determined on a continuous scale. The proportional or relative shortfall method would be used to assess disease severity., Conclusions: The proposed CET framework for the UAE will be dependent on local generation of cost-effectiveness evidence. Periodic review of CETs based on initial experiences ensures the responsiveness of policymakers to the changing healthcare and economic environment., Competing Interests: Competing interests: Syreon Middle East was a contractual partner of Bayer. AF, ZK and SAb are shareholders in Syreon Middle East. NK is an employee in Syreon Middle East. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Introduction: This paper summarizes the results from a forum of healthcare experts, academia representatives, and public agency officials from emerging and established market countries on Value-Based Healthcare (VBHC) and Health Technology Assessment (HTA). Presentations from experts provided insights into current developments and challenges, followed by interactive roundtable discussions. Emerging markets have unique healthcare systems, patient populations, resource constraints and needs., Areas Covered: Each roundtable explored specific topics including the role of HTA and Real-world evidence (RWE) in healthcare decision-making, challenges in biosimilar value assessment and incorporating non-price criteria reflecting context-related specifications of emerging markets such as the multifaceted nature of value in healthcare decision-making, emphasizing stakeholder perspectives and system complexities., Expert Opinion: RWE emerged as important in understanding biosimilar value recognition and decision-making processes, with insights into its applications and challenges. Recommendations were provided for utilizing Multi-Criteria Decision Analysis (MCDA) in pharmaceutical procurement, particularly for off-patent medicines, underscoring the importance of comprehensive evaluation frameworks and adherence to value-based principles. Overall findings suggest avenues for collaboration between industry, academia, and public agencies to address implementation barriers and promote equitable, efficient, and high-quality healthcare systems in emerging markets through public-private partnerships, joint capacity building and training initiatives, and knowledge transfers.

Background: Atopic dermatitis (AD) imposes a hidden burden through its negative effects on quality of life and productivity. We aim to estimate this hidden burden in adults and adolescents in Central and Eastern European (CEE) countries., Methods: We created a burden of disease model to quantify AD's hidden burden. Humanistic burden was calculated by estimating the monetary value of quality-adjusted life years (QALYs) lost, using prevalence data from the Global Burden of Disease study and gross domestic product (GDP) per capita for each country. Indirect economic burden was estimated based on productivity loss from absenteeism and presenteeism, adjusted for labor force participation and unemployment rates. Total hidden burden was determined by combining productivity losses and QALYs lost., Results: QALY loss due to AD ranged from 1,832 to 58,596 annually in CEE countries, equating to 38 million to approximately 1 billion Euros per country. Productivity losses ranged from 3.6 to 148.9 million Euros annually. The total hidden burden of AD represents 0.11% to 0.43% of the GDP., Conclusions: Our estimates reflect significant differences in population size, prevalence, and economic strength among CEE countries. Adjusting findings to country-specific GDP provided insights into AD's true hidden burden, offering valuable information for decision-making.

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., (© 2024. The Author(s).)

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., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section. All conclusions expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations., (Copyright © 2024 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Background The scarcity of resources restricts healthcare financing decisions, affecting the population's health. Health technology assessment (HTA) balances restricted budgets with the best possible health outcomes. We aim to characterize the current status of HTA in Algeria and describe the future directions for HTA implementation according to the priorities set by local stakeholders. Methods Stakeholders from the public and private sectors responded to a policy survey about the current and preferred future status of HTA implementation in Algeria. The survey was administered during an online workshop and used a widely accepted international scorecard covering eight domains: capacity building, HTA financing, process and organizational structure, scope of HTA implementation, decision criteria, standardization of methodology, use of local data, and international collaboration. After that, one-on-one interviews with another local expert were conducted to validate and modify the draft recommendations. The interviewees were representatives from government agencies, hospitals, and pharmaceutical companies. Results Thirty-one experts filled out the HTA scorecard survey; most of them were from the public sector (74%). They highlighted that project-based HTA workshops or short courses were the most common form of HTA education in Algeria and recommended the establishment of postgraduate HTA training programs in the future to build sustainable capacities. They reported a lack of funding for HTA research and critical appraisal and recommended an increased public budget for HTA and the introduction of submission fees by manufacturers. There was consensus about the need for local HTA evidence generation in the future. Most of the experts advocated an explicit soft decision threshold. The interviewees further recommended using multi-criteria decision analysis in the short term. The application of quality indicators was believed to improve the reliability of the HTA process. Conclusion The results of our policy research delineate the gap between the current and preferred future status of HTA in Algeria based on insights from multiple stakeholders. The need to improve the educational HTA programs in Algeria, use local data in policy decisions, and increase funding for HTA were the most advocated recommendations., Competing Interests: Human subjects: Consent was obtained or waived by all participants in this study. Animal subjects: All authors have confirmed that this study did not involve animal subjects or tissue. Conflicts of interest: In compliance with the ICMJE uniform disclosure form, all authors declare the following: Payment/services info: Abbvie funded this study and contracted Syreon to conduct the study. However, Abbvie had no role in the design, analysis, or interpretation of the results. Financial relationships: Zoltán Kaló, Sherif Abaza, Ahmad Nader Fasseeh, Kareem A. El-Fass declare(s) employment and stock/stock options from Syreon. Syreon Middle East was a contractual partner of Abbvie. ANF, SA and ZK are shareholders in Syreon Middle East. KF is an employee at Syreon Middle East. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as potential conflicts of interest. Other relationships: All authors have declared that there are no other relationships or activities that could appear to have influenced the submitted work., (Copyright © 2024, Hedibel et al.)

Purpose: Health technologies are advancing rapidly and becoming more expensive, posing a challenge for financing healthcare systems. Health technology assessment (HTA) improves the efficiency of resource allocation by facilitating evidence-informed decisions on the value of health technologies. Our study aims to create a customized HTA roadmap for Oman based on a gap analysis between the current and future status of HTA implementation., Design/methodology/approach: We surveyed participants of an advanced HTA training program to assess the current state of HTA implementation in Oman and explore long-term goals. A list of draft recommendations was developed in areas with room for improvement. The list was then validated for its feasibility in a round table discussion with senior health policy experts to conclude on specific actions for HTA implementation., Findings: Survey results aligned well with expert discussions. The round table discussion concluded with a phasic action plan for HTA implementation. In the short term (1-2 years), efforts will focus on building capacity through training programs. For medium-term actions (3-5 years), plans include expanding the HTA unit and introducing multiple cost-effectiveness thresholds while from 6-10 years, publishing of HTA recommendations, critical appraisal reports, and timelines is recommended., Originality/value: Although the HTA system in Oman is still in its early stages, strong initiatives are being taken for its advancement. This structured approach ensures a comprehensive integration of HTA into the healthcare system, enhancing decision-making and promoting a sustainable, evidence-based system addressing the population's needs., (© Ibrahim Al Rashdi, Sara Al Balushi, Alia Al Shuaili, Said Al Rashdi, Nadiya Ibrahim Al Bulushi, Asiya Ibrahim Al Kindi, Qasem Al Salmi, Hilal Al Sabti, Nada Korra, Sherif Abaza, Ahmad Nader Fasseeh and Zoltán Kaló.)

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., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section., (Copyright © 2024 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

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., Competing Interests: Syreon Research Institute received grants from Egis Pharmaceuticals PLC for pharmaceutical policy research related to off-patent pharmaceuticals on various topics, including generic, biosimilar, and value-added medicines. A.I., Z.K., Z.P., K.G. and T.Á. are employees of Syreon Research Institute. The funder was not involved in the study design, collection, analysis, interpretation of data, the writing of this article or the decision to submit it for publication.

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., (© 2024. The Author(s).)

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., (© 2023. The Author(s).)

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., Competing Interests: The authors have declared financial relationships, which are detailed in the next section., (Copyright © 2024, Alnaqbi et al.)

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.

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., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Németh, Mágó, Kaló, Lám, Balogh and Brodszky.)

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., Competing Interests: Patient involvement should be open to all and non-discriminative on the grounds of previous experience and presumed support time needed. There are multiple initiatives locally and internationally aiming to ease patient recruitment with education, coordination and/or databases. We recommend organisations leading these initiatives to come together and join forces on the base of commonly agreed principles. We also recommend local HTA/payer organisations to set up their open call for local patients, carers, patient advocates, patient experts and patient organisation representatives to be able to express interest. We suggest to actively promote this opportunity to harder to reach patient communities and a periodical revision of the registry. Regarding ethical and compliance issues, a clear policy on financial and other conflicts of interests (how interests are declared, assessed and addressed) should be in place. Those registered should complete a declaration of interest form both on personal and organisational level conflict of interests and update it periodically. It should be clarified what kind of involvement with industry (e.g., attending a single advisory board meeting with a company versus only in case of direct conflict of interest) would impose restrictions on how a person can be involved in the HTA and decision-making process. We argue that in situations where patient experts and/or patient organisation representatives are difficult to identify, a softer approach should be taken and special measures could be proposed, equivalent to the “expert witness” status at the European Medicines Agency (EMA). Expert witnesses can be heard or participate in the deliberations but are not allowed to take part in the vote. However, the consequences of not being transparent with potential conflict of interests should be serious and communicated clearly from the beginning.KrT, RH, ZP, BN, and ZK were employed by Syreon Research Institute. At the time of the study IJ was the President of the European Patients’ Forum Youth Group, a Board of Trustees member at the EUPATI Foundation and employed by Syreon Research Institute. MN is the founder and co-owner of HealthQuest, a health technology assessment and market access consulting company. DaD is a Trustee of Thrombosis UK. TB was employed by Patvocates GmbH. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Jakab, Dimitrova, Houÿez, Bereczky, Fövényes, Maravic, Belina, Andriciuc, Tóth, Piniazhko, Hren, Gutierrez-Ibarluzea, Czech, Tesar, Niewada, Lorenzovici, Kamusheva, Manova, Savova, Mitkova, Tachkov, Németh, Petykó, Dawoud, Delnoij, Knies, Goettsch and Kaló.)

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., (© 2023. The Author(s).)

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., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2023 Zemplényi, Tachkov, Balkanyi, Németh, Petykó, Petrova, Czech, Dawoud, Goettsch, Gutierrez Ibarluzea, Hren, Knies, Lorenzovici, Maravic, Piniazhko, Savova, Manova, Tesar, Zerovnik and Kaló.)

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.

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.

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., (© 2022. The Author(s).)

Aims: The goal of this study was to review the economic evaluations of health technologies in multiple myeloma (MM) and provide guidance and recommendations for future health economic analyses., Materials and Methods: A systemic literature review (SLR) was conducted on original economic assessment studies and structured review papers focusing on the studies in MM. The search was limited to English language papers published from 1 January 2000 onwards. Publications not applying any type of modelling methodology to describe disease progression and patient pathways over a specific time horizon were excluded., Results: A total of 2,643 publications were initially identified, of which 148 were eligible to be included in the full-text review phase. From these, 49 publications were included in the final analysis. Most published health economic analyses supported by models came from high-income countries. Evaluations from middle-income countries were rarely published. Diagnostic technologies were rarely modelled and integrated care had not been modelled. Very few models investigated MM treatments from a societal perspective and there was a relative lack of evaluations regarding minimal residual disease (MRD)., Limitations: Limitations of the publications included differences between trial populations and modelled populations, justification of methods, lack of confounder analyses, and small trial populations. Limitations of our study included the infeasibility of comparing MM economic evaluations due to the significant variance in modelled therapeutic lines and indications, and the relative scarcity of published economic evaluations from non-high-income countries., Conclusions: As published economic models lacked many of the elements of the complex and heterogeneous patient pathways in MM and they focused on single decision problems, a thorough, open-source economic whole disease modelling framework is needed to assess the economic value of a wide range of technologies across countries with various income levels with a more detailed view on MM, by including patient-centric and societal aspects.