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This study assessed direct-acting oral anticoagulant (DOAC) switching/discontinuation patterns in patients with non-valvular atrial fibrillation (NVAF) in 2019, by quarter (Q1–Q4), and associated socioeconomic risk factors. Adults with NVAF initiating stable DOAC treatment (July 2018–December 2018) were selected from Symphony Health Solutions’ Patient Transactional Datasets (April 2017–January 2021). Switching/discontinuation rates were reported in 2019 Q1–Q4, separately. Non-medical switching/discontinuation (NMSD) was defined as the difference between switching/discontinuation rates in Q1 and mean rates across Q2–Q4. The associations of socioeconomic factors with switching/discontinuation were assessed. Of 46,793 patients (78.7% ≥ 65 years; 52.6% male; 7.7% Black), 18.0% switched/discontinued their initial DOAC in Q1 vs. 8.8% on average in Q2–Q4, corresponding to an NMSD of 9.2%. During the quarter following the switch/discontinuation, more patients who switched/discontinued in Q1 remained untreated (Q1: 77.0%; Q2: 74.3%; Q3: 71.2%) and fewer reinitiated initial DOAC (Q1: 17.6%; Q2: 20.8%; Q3: 24.0%). Factors associated with the risk of switching/discontinuation in Q1 were race, age, gender, insurance type, and household income (all p < 0.05). More patients with NVAF switched/discontinued DOACs in Q1 vs. Q2–Q4, and more of them tended to remain untreated relative to those who switched/discontinued later in the year, suggesting a potential long-term impact of NMSD. Findings on factors associated with switching/discontinuation highlight potential socioeconomic discrepancies in treatment continuity.

Healthcare is a huge business sector in many countries, focusing on the social function of delivering quality health when people develop illness. The system is essentially financed by public funds based on the solidarity principle. With a large financial outlay, the sector must use economic evaluation methods to achieve better efficiency. The objective of our study was to evaluate and to understand how health economics is used today, taking Belgium as an example of a high-income country. The evaluation started with a historical view of healthcare development and ended with potential projections for its future. A literature review focused on country-specific evaluation reports to identify the health economic methods used, with a search for potential gaps. The first results indicated that Belgium in 2021 devoted 11% of its GDP, 17% of its total tax revenue, and 30% of the national Social Security Fund to health-related activities, totalizing EUR 55.5 billion spending. The main health economic method used was a cost-effectiveness analysis linked to budget impact, assigning reimbursable monetary values to new products becoming available. However, these evaluation methods only impacted at most 20% of the money circulating in healthcare. The remaining 80% was subject to financial regulations (70%) and budgeting (10%), which could use many other techniques of an economic analysis. The evaluation indicated two potentially important changes in health economic use in Belgium. One was an increased focus on budgeting with plans, time frames, and quantified treatment objectives on specific disease problems. Economic models with simulations are very supportive in those settings. The other was the application of constrained optimization methods, which may become the new standard of practice when switching from fee-for-service to pay-per-performance as promoted by value-based healthcare and value-based health management. This economic refocusing to a more constrained approach may help to keep the healthcare system sustainable and affordable in the face of the many future challenges including ageing, climate change, migration, pandemics, logistical limitations, and financial instability.

Background: The purpose of this paper is to address how to handle uncertainty when performing an economic valuation of a medical innovation R&D project in orphan diseases from the perspective of the investor. Methods: We describe the specific uncertainty related to cash flows and the cost of capital for innovation in orphan diseases. The uncertainty in cash flows relates to sales, manufacturing and R&D costs, and probabilities of failure for each phase in the clinical trial program. We consider different net present values (NPVs) and higher standard deviations for orphan drugs compared to non-orphan drugs. Results: Numerical case base examples showed the differences in trade-off by an investor for R&D projects with differences in NPV and level of uncertainty. The investor will transfer the additional uncertainty in cash flows in a higher cost of capital. An alternative approach is the application of an “acceptability curve” based on a probabilistic sensitivity analysis, which displays the cumulative probabilities at a range of different values for the NPV. Finally, we consider uncertainty in the cost of capital itself by applying the Capital Asset Pricing Model (CAPM). Conclusions: In this paper, we described various types of uncertainty and explored various approaches to how to handle uncertainty in the economic valuation of medical innovation in orphan diseases. The bridging of health economics with economic valuation theory in the healthcare market is to our knowledge a novel approach for the valuation of medical innovation by investors.

Economic evaluations are used to compare the costs and consequences of healthcare interventions, including those for musculoskeletal (MSK) disorders, which are very common and a major source of morbidity and absence from work. Reimbursement decisions for interventions for MSK disorders by decision-makers rely on the findings of economic evaluations, the design and results of which depend largely on the perspective adopted. Despite methodological advancements in economic evaluations, there are no clear guidelines on the perspective to adopt. This paper explores the adoption of a societal perspective in economic evaluations of MSK disorders. Within health economics evaluations, the most commonly used perspectives include the payer perspective, the healthcare perspective, and the societal perspective. To facilitate optimal resource allocation decisions in order to reduce the significant economic burden of MSK disorders and improve the health outcomes of individuals with these disorders, all costs and benefits associated with interventions for them should be included. Thus, the societal perspective is arguably a preferable option to the others for economic evaluations of interventions for MSK disorders.

Background: Considering regulatory, supervision, and control health policy, an innovative knowledge management model is proposed for the Colombian health system, which is recognized as a complex system. Methods: A model is constructed through a comparative analysis of various theoretical and conceptual frameworks, and an original methodology is proposed based on an analysis of the macroprocesses of the Supervision and Control System (SSC) of the Colombian General Social Security System in Health (SGSSS). After formulating hypotheses and conceptual references, information errors are determined within the different macroprocesses of the SGSSS, including those of governance and the SSC. Results: The risks of generating duplicate, wrong, hidden, or non-existent information arise when the associated regulations need more specificity to be applied in all cases, thus leading to the risk of different interpretations by some actors. In this way, it is possible to hinder the generation of unified information, as there is no clarity as to who is responsible for the generation or creation of certain data. Conclusions: The proposed model is characterized by its flexibility and adaptability, integrating several processes that can be executed simultaneously or cyclically (depending on the system’s needs) and allowing for the generation and feedback of knowledge at different stages, with some processes simultaneously executed to complement each other.

Patient experience and satisfaction are the keystones in evaluating the effectiveness of clinical care in musculoskeletal medicine. Although all orthopedic settings work on the same principles of providing safe and quality health care, community hospitals represent a unique environment. There may be key differences with regard to patient experience between these settings. Accessibility to care, choices of provider, personalized care, availability of and access to resources, cultural and social variances, and waiting times are a few of the many elements that may impact patient experience and satisfaction. This narrative review aims to explore the core differences in these settings and how they can reflect on patient experience and satisfaction.

National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings. Therefore, this research aims to (i) identify the opportunities and challenges faced by Beneluxa, (ii) gather insights from stakeholders, namely (possible) applicants and policymakers, within and beyond Beneluxa on the initiative and broader cross-country collaboration principles, and (iii) transfer these insights into learnings and recommendations in anticipation of the full implementation of the new HTA Regulation. Fifteen semi-structured interviews were conducted with industry and European HTA/policy stakeholders. The principal challenges discussed by stakeholders encompass hesitancy from the industry toward Beneluxa assessments, which were attributed to procedural and timeline uncertainties, legislative framework ambiguity, and challenges in terms of industry’s internal organization. Another challenge highlighted is the resource-intensive nature of the procedure due to diverse approaches among member states. In addition, industry stakeholders mentioned limited communication and procedural complexity. Despite challenges, both stakeholder groups recognized important opportunities for cross-country collaboration. Transferable insights for future cross-country collaboration include transparent communication, clear legislative embedding, internal industry restructuring to facilitate joint HTAs, and member state support for conducting collaborative assessments. The study underscores diverging views among stakeholders on cross-country collaboration’s potential to support HTA and the market access of complex health technologies. While acknowledging benefits, there still are challenges, including industry hesitancy, emphasizing the need for transparent communication and clear guidance in the evolving EU HTA landscape.

Alopecia is an undesirable side effect of cancer chemotherapy. The mitigation of alopecia is a desirable adjunct treatment for patients with cancer. FDA-cleared scalp cooling (SC) devices have been successfully used to prevent or reduce chemotherapy-induced alopecia (CIA). This paper provides an understanding of the implementation and value of the new Insurance-Based Billing Model used in the USA for SC and its benefits compared with the original self-pay model. This improved compensation change will result in all patients in need, including underserved and disadvantaged populations, receiving equitable healthcare by allowing access to this valuable supportive care technology.

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country.

This study aimed to close an evidence gap concerning the relative efficacy of finerenone versus SGLT2is in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D). Canagliflozin was selected as a proxy for the SGLT2i class. Patient-level data of two randomized controlled trials (RCTs) of finerenone (FIDELIO-DKD and FIGARO-DKD) were used alongside aggregated data from CREDENCE, an RCT of canagliflozin. To account for meaningful between-study heterogeneity between each finerenone trial and CREDENCE, a matching-adjusted indirect comparison of a range of efficacy outcomes was undertaken for each finerenone study versus CREDENCE. These results were meta-analyzed, enabling the estimation of the relative effects of finerenone against canagliflozin. For the cardiorenal composite endpoint, the hazard ratio (HR) comparing finerenone to canagliflozin was 1.07 (95% CI: 0.83 to 1.36). The corresponding HRs for all-cause mortality, end-stage kidney disease and cardiovascular death were 0.99 (95% CI: 0.73 to 1.34), 1.03 (95% CI: 0.68 to 1.55) and 0.94 (95% CI: 0.64 to 1.37), respectively. The absence of statistically significant differences was consistent throughout the main analysis and a range of sensitivity analyses. Based on this study, using a large sample of data and adjusted for meaningful differences between the baseline characteristics of the included RCTs, there was no statistically significant evidence indicating a difference in the efficacy of finerenone compared to canagliflozin in the treatment of CKD in patients with T2D.

Background: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies’ role; (ii) role of clinical guidelines; (iii) interface with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS); and (iv) approaching ‘best-available evidence’ (BAE). A descriptive analysis of questionnaire outcomes was conducted to inform the European Access Academy (EAA) Fall Convention 2023. Within the working groups (WGs), action points were identified and prioritised. Results: A total of 57 experts from 15 countries responded to the survey. The WGs were attended by (i) 11, (ii) 10, (iii) 12, and (iv) 12 experts, respectively, representing a variety of national backgrounds and stakeholder profiles. The most relevant action points identified were as follows: (i) incorporation of clinical context into population, intervention, comparator, outcomes (PICO) schemes, (ii) timely provision of up-to-date therapeutic guidelines, (iii) ensuring the inclusion of MCBS insights into the EU HTA process, and (iv) considering randomized controlled trials (RCTs) as the gold standard and leveraging regulatory insights if development programs only include single-arm trials. Conclusions: The involvement of medical societies is a critical success factor for the EU HTA. The identified key action points foster the involvement of patient associations and medical societies.

Background: A decrease in populations could affect healthcare access and systems, particularly in medically underserved areas (MUAs) where depopulation is becoming more prevalent. This study aimed to simulate the future population and land areas of MUAs in Japan. Methods: This study covered 380,948 1 km meshes, 87,942 clinics, and 8354 hospitals throughout Japan as of 2020. The areas outside a 4 km radius of medical institutions were considered as MUAs, based on the measure of areas in the current Japanese Medical Care Act. Based on the population estimate for a 1 km mesh, the population of mesh numbers of MUAs was predicted for every 10 years from 2020 to 2050 using geographic information system analysis. If the population within a 4 km radius from a medical institution fell below 1000, the institution was operationally assumed to be closed. Results: The number of MUAs was predicted to decrease from 964,310 (0.77% of the total Japanese population) in 2020 to 763,410 (0.75%) by 2050. By 2050, 48,105 meshes (13% of the total meshes in Japan) were predicted to be new MUAs, indicating a 31% increase in MUAs from 2020 to 2050. By 2050, 1601 medical institutions were tentatively estimated to be in close proximity. Conclusions: In Japan, the population of MUAs will decrease, while the land area of MUAs will increase. Such changes may reform rural healthcare policy and systems.

Background: Understanding the concept and dynamic process of the evolution of professional identity and roles of market access (MA) in the pharmaceutical industry (pharma) is critical to personal, interpersonal, and professional levels of development and impact. Objective: The aim was to carry out a scoping review of the conceptualisation of MA within pharma. Data Sources: BioMed Central, WorldCat.org, and Directory of Open Access Journals were searched from 2003 to 2023. Study Selection: All articles on concepts or definitions and other surrogate terms on MA in pharma were selected. Data Extraction: Keywords generated from an initial cursory literature search on MA in pharma were used in conjunction with AND/OR as search terms. Using the data charting method, key findings were mapped and summarised descriptively. inductive analysis was performed, allowing codes/themes that are relevant to the concept to emerge. Data Synthesis: Arskey and O’Malley’s six-stage framework and the PRISMA extension for scoping reviews extension checklist were used as the review and reporting templates. The databases search yielded 222 results. Following title and abstract screening, a total of 146 papers were screened, and 127 of them were excluded. Full-text review was conducted for 19 papers that were deemed by two reviewers to meet the eligibility criteria. One of the authors arbitrated on disputed papers for inclusion. Only 14 of the included papers were found to meet the criteria for the final analysis. Five conceptual dimensions of MA in pharma were identified as “right products”, “right patient”, “right price”, “right point” (time), and “right place” (setting). Conclusions: Market access in pharma is a process that commences with the development and availability of the right products that are proven to be efficacious and disease/condition-specific (including medications, medical devices, and vaccines); specifically produced for the right patients or end users who will maximise best clinical outcomes and economic value; delivered at the right point in a timely, sustained, and efficient manner, given at the right price (commercially viable or reimbursed price that represents good value); and conducted within the economic, policy, societal, and technological contexts, with the overarching goal of achieving the best patient outcomes and ensuring product profitability.

Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.

The vision of a unified European HTA is by no means a new endeavor. At its core are the publicly declared ambitions to harmonize assessments of clinical data within the EU and avoid the duplication of efforts. Not surprisingly, these ambitions are publicly announced to be motivating the new 2022 EU HTA regulation. However, industry experts typically see more of a risk for additional bureaucracy resulting in delays, further scrutiny, and one additional EU (clinical) dossier to submit on top of all national HTA dossiers, which could be considered a duplication of effort and therefore counterproductive. Regardless of how the details of the process will be defined and how the entire process will work in practice, we can be sure that EU officials will refer to the EU HTA and Joint Clinical Assessment (JCA) in particular as a learning system. The purpose of this article is to take a closer look at the new EU HTA regulation and analyze threats and opportunities for manufacturers and what the resulting opportunities and threats will be at the affiliate level throughout the EU.

Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases, not possible. Numerous ITC techniques are described in the literature. A systematic literature review (SLR) was conducted to identify all the relevant literature on existing ITC techniques, provide a comprehensive description of each technique and evaluate their strengths and limitations from an HTA perspective in order to develop guidance on the most appropriate method to use in different scenarios. Methods: Electronic database searches of Embase and PubMed, as well as grey literature searches, were conducted on 15 November 2021. Eligible articles were peer-reviewed papers that specifically described the methods used for different ITC techniques and were written in English. The review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Results: A total of 73 articles were included in the SLR, reporting on seven different ITC techniques. All reported techniques were forms of adjusted ITC. Network meta-analysis (NMA) was the most frequently described technique (in 79.5% of the included articles), followed by matching-adjusted indirect comparison (MAIC) (30.1%), network meta-regression (24.7%), the Bucher method (23.3%), simulated treatment comparison (STC) (21.9%), propensity score matching (4.1%) and inverse probability of treatment weighting (4.1%). The appropriate choice of ITC technique is critical and should be based on the feasibility of a connected network, the evidence of heterogeneity between and within studies, the overall number of relevant studies and the availability of individual patient-level data (IPD). MAIC and STC were found to be common techniques in the case of single-arm studies, which are increasingly being conducted in oncology and rare diseases, whilst the Bucher method and NMA provide suitable options where no IPD is available. Conclusion: ITCs can provide alternative evidence where direct comparative evidence may be missing. ITCs are currently considered by HTA agencies on a case-by-case basis; however, their acceptability remains low. Clearer international consensus and guidance on the methods to use for different ITC techniques is needed to improve the quality of ITCs submitted to HTA agencies. ITC techniques continue to evolve quickly, and more efficient techniques may become available in the future.

Objective: Currently there are no disease-specific approved therapies for non-alcoholic fatty liver (NAFL) and non-alcoholic steatohepatitis (NASH); however, several treatments are under development. This study aimed to estimate the cost-effectiveness of hypothetical innovative therapies compared with lifestyle intervention alone and combined with pioglitazone, and assess the health economic consequences of their future availability for patients. Methods: A Markov cohort model was developed, considering fourteen disease health states and one absorbing state representing death. Transition probabilities, costs, utilities, and treatment efficacy were based on published data and assumptions. Four treatment strategies were considered, including two existing therapies (lifestyle intervention, small molecule treatment) and two hypothetical interventions (biological and curative therapy). The analysis was performed from the US third-party payer perspective. Results: The curative treatment with the assumed efficacy of 70% of patients cured and assumed price of $500,000 was the only cost-effective option. Although it incurred higher costs (a difference of $188,771 vs. lifestyle intervention and $197,702 vs. small molecule), it generated more QALYs (a difference of 1.58 and 1.38 QALYs, respectively), resulting in an ICER below the willingness-to-pay threshold of $150,000 per QALY. The sensitivity analyses showed that the results were robust to variations in model parameters. Conclusions: This study highlighted the potential benefits of therapies aimed at curing a disease rather than stopping its progression. Nonetheless, each of the analyzed therapies could be cost-effective compared with lifestyle intervention at a relatively high price.

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy.

The aim of this study was to evaluate the comparative clinical effectiveness and cost-utility of the active transcutaneous Osia® System versus the passive transcutaneous Baha® Attract System for patients with conductive or mixed hearing loss or single-sided deafness in an Australian healthcare setting. In the absence of direct comparative evidence, an indirect treatment comparison (ITC) of the clinical effectiveness and utility gains was needed. The ITC was informed by three studies identified through a systematic literature review. A Markov model was developed to evaluate the cost-utility of the Osia System. The literature review identified three studies suitable to inform an ITC: Mylanus et al. 2020 and Briggs et al. 2022 (Osia System) and den Besten et al. 2019 (Baha Attract System). The Osia System was found to be clinically superior to the Baha Attract System, across objective audiological outcomes resulting in a clinically meaningful utility benefit of 0.03 measured by the Health Utility Index with at least equivalent safety. In conclusion, the Osia System is more effective than the Baha Attract System, providing better hearing and health-related quality of life outcomes. In an Australian healthcare setting, the Osia System is cost-effective as demonstrated in a cost-utility analysis versus the Baha Attract System.

The Journal of Market Access and Health Policy (JMAHP) is the first peer-reviewed open access journal that focuses on the concept of ‘market access’ from economic, technical, scientific, sociological, anthropological, psychological, and policy perspectives [...]

ABSTRACTAt least since the Age of Enlightenment, good health has been a tenet for society. Healthy societies could learn better, work harder, improve their wealth, and live longer. Today societies focus on life expectancy, as we value long and healthy lives. As illustrated by the provision of COVID-19 vaccines first for the elderly, societies value life-saving actions. Paradoxically, health economic assessments conventionally devalue long-lasting health through the practice of discounting health benefits along with costs. However, health, with its intrinsic and instrumental characteristics, is not synonymous with money cash, a tradeable asset that devalues with time. If improving healthy life expectancy is a societal ambition, it seems counter-intuitive to value future health less as a result of an artificial mathematical construct when evaluating economically new medical interventions. In this paper, we investigate the application of discounting health in healthcare and consider paradoxical findings, especially in relation to disease prevention with vaccination. We argue that there is no economically sustainable argument to discount health gains, except for the benefit of the payer with a goal of spending less on life-saving products. If that is the objective for discounting health, there are other means to achieve the same goal in a more transparent and simpler way. From the long-term perspective of healthcare development, not discounting health gains would encourage research that values long-term effects. This in turn has the potential to benefit the investor, the payer, and the patient/consumer, improving the situation from multiple perspectives.

ABSTRACTBackground: Data on the therapeutic management and healthcare cost of moderate to severe psoriasis in France are scarce.Objective: To assess the therapeutic management and economic burden of patients with moderate to severe psoriasis.Setting: This is a retrospective observational study on the Generalist Beneficiaries Sample of the National Health Data System.Patients and outcome measures:Adults with moderate to severe psoriasis (with a topical vitamin D derivative followed by systemic treatment or hospitalization for psoriasis) were included and followed-up from 1 January 2009 to 31 December 2018. Patients were matched to controls without psoriasis. Patients’ characteristics and healthcare cost from the National Health Insurance’s (NHI) perspective were described.Results: Overall, 1,848 and 5,544 adults were included in the psoriatic and control cohorts, respectively. The most frequent treatments were methotrexate (18.5% to 21.4% of patients by year), phototherapy (29.9% in 2010 down to 6.2% in 2018), and acitretin (25.9% in 2010 down to 8.6% in 2018). Overall, 19% of patients used biotherapies. The mean healthcare costs reimbursed by NHI was €5,365/psoriatic patient (including €2,685 potentially attributable to psoriasis), which was twice as high as in controls. In both cohorts, healthcare costs increased over time.Conclusion: Moderate to severe psoriasis healthcare costs are high. [Figure: see text]

ABSTRACTObjective Direct oral anticoagulants (DOACs) are first-line therapy for stroke prevention for 1.4 million atrial fibrillation (AF) patients in the UK. However, the rates of DOAC dosing below evidence-based recommendations are estimated between 9% and 22%. This study explores specific patient and physician factors associated with prescribing inappropriate DOAC underdoses.Methods DOAC-prescribing physicians within the UK completed both a clinical vignette survey, which contained 12 hypothetical patient profiles designed to replicate DOAC prescribing scenarios, and a physician survey to capture sociodemographic, clinical experience, and prescriber-related beliefs and motivations related to DOAC prescribing. Eight patient factors based on a literature search and an expert consultation process were varied within the vignettes. Associations between the prescribers’ dosing choices and patient factors were explored via multilevel logistic regression. The analysis is focused on the most frequently selected DOACs, apixaban and rivaroxaban, both of which have different dosing guidelines.Results In all, 336 prescribers (69% male; 233/336) completed the survey, mostly general physicians (GPs) (45%) or cardiology specialists (36%) with a mean of 17.9 years’ experience. Most prescribers (73%; 244/336) inappropriately underdosed at least once; rates between GPs and specialists were nearly identical. Patient factors most strongly associated with apixaban inappropriate underdosing included a history of major bleeding and falls. For rivaroxaban, these were major bleeding and severe frailty. Only 32% (106/335) of prescribers reported DOAC dosing guidelines as the sole influence on their prescribing behaviour. Among prescribers who did not inappropriately underdose, greater prescribing confidence was aligned to increased perception of inappropriate underdose risk.Conclusions Overall, patient factors such as major bleeding and severe frailty were found to be associated with inappropriate underdosing of apixaban and rivaroxaban. Furthermore, prescribers who were more confident in DOAC prescribing, and were more worried about the risk of stroke, were significantly less likely to inappropriately underdose. These findings suggest that all prescribers, regardless of speciality, may benefit from education and training to raise awareness of the risks associated with inappropriate DOAC underdosing.

ABSTRACTObjectives: To obtain real-world data on rational drug use, pharmacoeconomic regarding women’s and children’s health and the benefits of Hainan free trade port (HFTP) health policies, we retrospectively investigated drug indicators, prescribing trend, and economic data.Method: We retrospectively gathered the data from the database of the hospital information system and the quality indicators of pharmacy; we compared the monthly indicators from 2015 to 2019.Results: In 2017, the HFTP maternal mortality ratio (MMR) was 24.46. In 2019, the HFTP infant mortality rate (‰) was 4.15, and the under-five mortality rate (‰) was 6.19. A total of 1,922,798 prescriptions included in the analysis, the defined daily dose of 2015–2019 ranged from 46.59 to 32.34. In 2019, the proportions of antibiotics prescribed in outpatient, emergency, and inpatient care were 14.19%, 16.68%, and 46.26%, respectively. The injection prescription percentage ranged from 13.08% to 8.08%. The proportion of medicine income to total hospital income of 2015–2019 ranged from 26.66% to 25.31%.Conclusion: According to the analysis of women’s and children’s real-world drug data, economic investment and strict quality control of antimicrobial stewardship programs can lead to the rational use of drugs.

ABSTRACTBackground: Heart failure is a chronic disease linked with significant morbidity and mortality, and uncontrolled resting heart rate is a risk factor for adverse outcomes. This systematic literature review aimed to assess the efficacy, safety, and patient-reported outcomes (PROs) of ivabradine in patients with heart failure (HF) with reduced ejection fraction (HFrEF) in randomized controlled trials (RCTs) and observational studies.Methods: We searched electronic databases from their inception to July 2021 to include studies that reported on efficacy, safety, or PROs of ivabradine in patients with HFrEF.Results: Of 1947 records screened, 51 RCTs and 6 observational studies were identified. Ivabradine on top of background therapy demonstrated a significant reduction in composite outcomes including hospitalization for HF or cardiovascular death. In addition, observational studies suggested that ivabradine was associated with a significant reduction in mortality. Across all studies, ivabradine use on top of background therapy was associated with greater reductions in heart rate, improved EF, and improved health-related quality of life (QoL) and comparable risk of total adverse events compared to those treated with background therapy alone.Conclusions: Ivabradine on top of background therapy is beneficial for heart rate, hospitalization risk for HF, mortality, EF, and patients’ QoL. Moreover, these benefits were achieved with no significant increase in the overall risk of total adverse events.

ABSTRACTBackground: Copay cards are intended to mitigate patient out-of-pocket (OOP) expenses. This qualitative, exploratory focus group study aimed to capture patient perceptions of copay cards and copay adjustment programs (CAPs; insurers’ accumulator and maximizer policies), which redirect the copay card utilization benefits intended for patients’ OOP expenses.Methods: Patients with chronic conditions were recruited through Janssen’s Patient Engagement Research Council program. They completed a survey and attended a live virtual session to provide feedback on copay cards.Results: Among 33 participants (median age, 49 years [range, 24–78]), the most frequent conditions were cardiovascular-metabolic disease and inflammatory bowel disease. Patients associated copay cards with lessening financial burden, improving general and mental health, and enabling medication adherence. An impact on medication adherence was identified by 10 (63%) White and nine (100%) Black respondents. Some patients were unaware of CAPs despite having encountered them; they recommended greater copay card education and transparency about CAPs.Conclusion: Patients relied on copay cards to help afford their prescribed medication OOP expenses and maintain medication adherence. Use of CAPs may increase patient OOP expenses. Patients would benefit from awareness programs and industry – healthcare provider partnerships that facilitate and ensure access to copay cards.

ABSTRACTObjectives: To assess the comparative budget and health impact of lower-dose dabigatran versus reduced doses of apixaban and rivaroxaban in atrial fibrillation (AF) patients eligible for a lower-/reduced-dose due to individual patient characteristics in the Netherlands.Methods: A budget impact model was developed in accordance with ISPOR guidelines. A 3-year-time horizon was considered, and analyses were conducted from a Dutch healthcare payer’s perspective. The model applies published data to local AF-epidemiology, allowing calculations to estimate clinical events (strokes and haemorrhages) and costs. The analyses were based on real-world outcomes from patients with AF receiving a first direct oral anticoagulant (DOAC) prescription for low-dose dabigatran (110 mg) and a reduced dose of apixaban (2.5 mg) or rivaroxaban (15 mg). Two situations of switching treatments from one to another DOAC were modelled: switching from apixaban to dabigatran and from rivaroxaban to dabigatran. Base case results were given as savings per 100 patient-year, per total Dutch population, and events avoided. A univariate sensitivity analysis was conducted to explore the uncertainty around epidemiological and event costs input data. Scenario analyses were performed to estimate the effect of different market shares and potential price reductions due to future patent expiry for the total real-world population from the Netherlands.Results: The 3-years outcomes of switching patients eligible for a lower-/reduced-dose due to individual patient characteristics from apixaban or rivaroxaban to dabigatran resulted in cost savings estimated at €157 or €72 thousand per 100 patient-years, respectively, or €146 million per total Dutch population. Looking into the clinical events, dabigatran reflected the lowest number of mortalities, ischemic strokes, major bleeding, non-major bleeding, and haemorrhagic stroke compared to apixaban and rivaroxaban. The sensitivity analysis consistently reflected cost savings, with the ischeamic stroke events having the biggest impact. Accounting for the Dutch situation, both scenarios showed total savings ranging from €45 to €229 million over 3 years.Conclusions: Switching eligible AF-patients from reduced-dose apixaban or rivaroxaban to lower-dose dabigatran has the potential to reduce healthcare payer’s budget expenditures and provide health gains. Cost savings can potentially be further enhanced by market share adjustments and further price reductions.

ABSTRACTPurpose: Nowadays, systematic literature reviews (SLRs) and meta-analyses are often placed at the top of the study hierarchy of evidence. The main objective of this paper is to evaluate the trends in SLRs of randomized controlled trials (RCTs) throughout the years.Methods: Medline database was searched, using a highly focused search strategy. Each paper was coded according to a specific ICD-10 code; the number of RCTs included in each evaluated SLR was also retrieved. All SLRs analyzing RCTs were included. Protocols, commentaries, or errata were excluded. No restrictions were applied.Results: A total of 7,465 titles and abstracts were analyzed, from which 6,892 were included for further analyses. There was a gradual increase in the number of annual published SLRs, with a significant increase in published articles during the last several years. Overall, the most frequently analyzed areas were diseases of the circulatory system (n = 750) and endocrine, nutritional, and metabolic diseases (n = 734). The majority of SLRs included between 11 and 50 RCTs each.Conclusions: The recognition of SLRs’ usefulness is growing at an increasing speed, which is reflected by the growing number of published studies. The most frequently evaluated diseases are in alignment with leading causes of death and disability worldwide.

ABSTRACTObjectives In this paper, we outline and compare pharmaceutical pricing and reimbursement policies for in-patent prescription medicines in three Maghreb countries, Algeria, Morocco, and Tunisia, and explore possible improvements in their pricing and reimbursement systems.Methods The evidence informing this study comes from both an extensive literature review and a primary data collection from experts in the three studied countries.Key findings Twenty-six local experts where interviewed Intervieweesincluded ministry officials, representatives of national regulatory authorities, health insurance organizations, pharmaceutical procurement departments and agencies, academics, private pharmaceutical-sector actors, and associations. Results show that External Reference Pricing (ERP) is the dominant pricing method for in-patent medicines in the studied countries. Value-based pricing through Health Technology Assessment (HTA) is a new concept, recently used in Tunisia to help the reimbursement decision of some in-patent medicines but not yet used in the pricing of innovative medicines in the studied countries. Reimbursement decision is mainly based on negotiations set on Internal Reference Pricing (IRP).Conclusion Whereas each country has its specific regulations, there are many similarities in the pricing and reimbursement policies of in-patent medicines in Algeria, Morocco, and Tunisia. The ERP was found to be the dominant method to inform pricing and reimbursement decisions of in-patent medicines. Countries in the region can focus on the development of explicit value assessment systems and minimize their dependence on ERP over the longer-term. In this context, HTA will rely on local assessment of the evidence.

ABSTRACTBackground: The benefits of preventive interventions lack comprehensive evaluation in standard health technology assessments (HTA), particularly for rare and transmissible diseases.Objective: To identify possible considerations for future HTA using analogies between the treatment and prevention of rare diseases.Study design: An Expert panel meeting assessed whether one HTA assessment framework can be applied to assess both rare disease treatments and preventive interventions. Experts also evaluated the range of value elements currently included in HTAs and their applicability to rare, transmissible, and/or preventable diseases.Results: A broad range of value should be considered when assessing rare, transmissible disease prevention. Although standard HTA can be applied to transmissible diseases, the risk of local outbreaks and the need for large-scale prevention programs suggest a modified assessment framework, capable of incorporating prevention-specific value elements in HTAs. A ‘Rule of Prevention’ framework was proposed to allow broader value considerations anchored to severity, equity, and prevention benefits in decision-making for preventive interventions for rare transmissible diseases.Conclusion: The proposed prevention framework introduces an explicit initial approach to consistently assess rare transmissible diseases, and to incorporate the broader value of preventive interventions compared with treatment.

ABSTRACTIntroduction: New procedures and diagnostic tests in hematopoietic stem cell transplantation (HSCT) are associated with a significant increase in costs. The last cost estimate of allogeneic HSCT done in Tunisia was in 1996 and concerned only direct medical costs. Therefore, an updated cost analysis is needed.Objective: Analysis of direct costs during the first-year post-allogeneic HSCT in two groups of patients: Bone Marrow Transplant (Allo-BMT) and Peripheral Blood Stem Cell Transplant (Allo-PBSCT) and identification of factors leading to interindividual variations in costs in order to compare these costs with the budget allocated by the payer (CNAM).Methods: Pharmacoeconomic retrospective study, concerning patients who underwent allogeneic HSCT in 2013. Clinical and unit cost data were obtained from medical and administration records.Results:This study showed that the average direct cost of allogeneic HSCT in the population during the first year reached 56 638€. The average cost of Allo-BMT was 63 612€, and Allo-PBSCT was 45 966€ (p > 0.05). The initial hospitalization counted for 88% of total direct cost with an average cost of 41 441€ in Allo-BMT and 24 672€ in Allo-PBSCT (p

ABSTRACTIntroduction: A rapid literature review (RLR) is an alternative to systematic literature review (SLR) that can speed up the analysis of newly published data. The objective was to identify and summarize available information regarding different approaches to defining RLR and the methodology applied to the conduct of such reviews.Methods: The Medline and EMBASE databases, as well as the grey literature, were searched using the set of keywords and their combination related to the targeted and rapid review, as well as design, approach, and methodology. Of the 3,898 records retrieved, 12 articles were included.Results: Specific definition of RLRs has only been developed in 2021. In terms of methodology, the RLR should be completed within shorter timeframes using simplified procedures in comparison to SLRs, while maintaining a similar level of transparency and minimizing bias. Inherent components of the RLR process should be a clear research question, search protocol, simplified process of study selection, data extraction, and quality assurance.Conclusions: There is a lack of consensus on the formal definition of the RLR and the best approaches to perform it. The evidence-based supporting methods are evolving, and more work is needed to define the most robust approaches.

ABSTRACTPurpose: Remarkable progress in healthcare technology has recently been made alongside changes in concepts related to drugs and medical devices. It is speculated that this progress benefits not only patients but also healthcare professionals, such as medical doctors. We performed a systematic review of the characteristics of current data assessment tools to measure medical doctors’ work-related quality of life (QOL).Methods: A literature search was conducted through PubMed and Ichushi-Web in 2020. The related search terms used were ‘medical doctor,’ ‘quality of work life,’ and ‘questionnaire/interview.’ Two reviewers independently screened the studies, and the characteristics of the QOL assessment tools used in the identified studies were qualitatively reviewed and summarized.Results: In total, 5,443 and 760 articles were retrieved from PubMed and Ichushi-Web, respectively, of which 82 studies were included in this review. Sixty-five (79%) studies used structured questionnaires, and 17 (21%) studies used semistructured questionnaires. In terms of the study purpose, the identified studies mainly included four: mental health, the work or labor situation, satisfaction, and QOL. Components used to measure work-related QOL included satisfaction, burnout, QOL, the work environment, stress, mental health, work-life balance, and others. None of the studies used an originally developed QOL questionnaire to assess the work-related benefits of medical doctors.Conclusion: This systematic review found that there is a lack of studies directly assessing the work-related QOL of medical doctors and a lack of effective data collection tools to assess all work-related QOL components.

ABSTRACTBackground: Gene replacement therapy (GRT) is a treatment method used to combat or prevent various diseases. Its high one-off cost constitutes a major obstacle for successful market access. This paper aims to assess and discuss the applicability of amortization in models, such as cost-effectiveness models (CEMs) and budget impact models (BIMs) informing HTA recommendations and reimbursement decisions.Methods and findings: A hypothetical CEA and BIA were considered. The objective was to compare the GRT with and without amortization. A straight-line amortization model was used. The CEM and BIM were considered and assessed based on two set of scenarios: considering different amortization duration or different discounting rate. The impact of amortization against the total cost of gene therapy was assessed for all the scenarios.The cost difference between GRT with and without amortization in relation to its total cost was -$58,855, thus amortization does not have a significant impact on the results and conclusions of the cost-effectiveness analysis. For BIM in the base case, amortization had no impact on the results.Conclusion: Amortization has negligible impact on the results of CEM and total BIM and no impact on the conclusions from the model. One exception is the budget impact in case of an amortization period longer than the time horizon of BIM, where a half of the GRT price is moved beyond the model time horizon. Amortization has a distinguishing effect from an accounting perspective, but it does not have any implication for payers.

ABSTRACTIntroduction: Primary immune thrombocytopenia is a rare autoimmune disease characterised by a decreased platelet count resulting in an increased risk of bleeding events and even life-threatening haemorrhages. Thrombopoietin receptor agonists (TPO-RAs) are the standard of care second-line therapy for adult patients with chronic immune thrombocytopenia. The first TPO-RAs approved and reimbursed in Italy, eltrombopag and romiplostim, while effective, pose some issues in terms of safety (e.g., hepatotoxicity) or general management (e.g., dietary restrictions). Avatrombopag, an effective and well-tolerated TPO-RA, was recently granted reimbursement.Methods: A 3-year (2023–2025) budget impact analysis (BIA) was conducted to estimate its impact on the Italian National Health Service (NHS). Two scenarios were compared, of which one represents the current situation, without avatrombopag, and the other provides for an increasing market share of avatrombopag (up to 26.6%).Results: BIA shows that the increase in the use of avatrombopag correlates with savings for NHS: in the first year, saving would be €1,300,564, increasing to €2,774,210 in the third year, for a total of €6,083,231 over the 3-year period. The sensitivity analysis confirmed these savings in the scenario with avatrombopag.Conclusions: Based on this BIA, the introduction and reimbursement of avatrombopag is an efficient and advantageous choice for the Italian NHS.

ABSTRACTBackground and objectives: Multiple reforms aimed at improving the Chinese population’s health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends.Methods: Targeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs.Results: Drug reimbursement in China is becoming increasingly centralized due to the removal of provincial pathways, the establishment of the National Healthcare Security Administration and the implementation of the National Reimbursement Drug List (NRDL), which is now the main route for drug reimbursement in China. There is also an increasing number of other channels via which patients may access innovative treatments, including various types of commercial insurance and special access. Health technology assessment (HTA) and health economic evidence are becoming pivotal elements of the NRDL decision-making process. Alongside the optimization of HTA decision making, innovative risk-sharing agreements are anticipated to be increasingly leveraged in the future to optimize access to highly specialized technologies and encourage innovation while safeguarding limited healthcare funds.Conclusions: Drug public reimbursement in China continues to align more closely with approaches widely used in Europe in terms of HTA, health economics and pricing. Centralization of decision-making processes for public reimbursement of innovative drugs allows consistency in assessment and access, which optimizes the improvement of the Chinese population’s health.

ABSTRACTInvolvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process.A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of involvement, determine their suggested future role, identify challenges to contribution, and highlight efficient ways to fulfilling their role. The ‘key’ stakeholder groups identified and covered by this research included: patients‘, clinicians‘, regulatory, and Health Technology Developer representatives. The survey was circulated to a wide expert audience including all relevant stakeholder groups in order to determine self-perception by the ‘key’ stakeholders regarding involvement in the HTA process (self-rating), and in a second, slightly modified version of the questionnaire, to determine the perception of ‘key’ stakeholder involvement by HTA bodies, payers, and policymakers (external rating). Predefined analyses were conducted on the submitted responses.Fifty-four responses were received (patients 9; clinicians: 8; regulators: 4; HTDs 14; HTA bodies: 7; Payers: 5; policymakers 3; others 4). The mean self-perceived involvement score was consistently lower for each of the ‘key’ stakeholder groups than the respective external ratings. Based on the qualitative insights generated in the survey, a RACI Chart (Responsible/Accountable/Consulted/Informed) was developed for each of the stakeholder groups to determine their roles and involvement in the current EU HTA process.Our findings suggest extensive effort and a distinct research agenda are required to ensure adequate involvement of the key stakeholder groups in the evolving EU HTA process.

ABSTRACTBackground: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by DMD gene mutations. Early cost-effectiveness analyses are important to help contextualize the value of gene therapies for reimbursement decision making.Objective: To determine the potential value of delandistrogene moxeparvovec using a cost-effectiveness analysis.Study design: A simulation calculated lifetime costs and equal value of life years gained (evLYG). Inputs included extrapolated clinical trial results and published utilities/costs. As a market price for delandistrogene moxeparvovec has not been established, threshold analyses established maximum treatment costs as they align with value, including varying willingness-to-pay up to $500,000, accounting for severity/rarity.Setting: USA, healthcare system perspectivePatients: Boys with DMDIntervention: Delandistrogene moxeparvovec plus standard of care (SoC; corticosteroids) versus SoC aloneMain outcome measure: Maximum treatment costs at a given willingness-to-pay thresholdResults: Delandistrogene moxeparvovec added 10.30 discounted (26.40 undiscounted) evLYs. The maximum treatment cost was approximately $5 M, assuming $500,000/evLYG. Varying the benefit discount rate to account for the single administration increased the estimated value to >$5M, assuming $500,000/evLYG.Conclusion: In this early economic model, delandistrogene moxeparvovec increases evLYs versus SoC and begins to inform its potential value from a healthcare perspective.

ABSTRACTBackground: Like many developing countries, Colombia faces difficulties in financing health-care services as well as programs for health promotion and health education and there is evidence that its health-care system is underperforming.Objective: To provide evidence-based estimates of potential funding levels and assess the strengths, weaknesses, and viability of innovative funding mechanisms with a focus on treating rare diseases in Colombia.Methods: The strategy involved evidence-based projections of potential funding levels and a qualitative viability assessment using an expert panel.Results: Crowdfunding, corporate donation, and social impact bonds (SIBs) were deemed to be the most viable of numerous potential strategies. Expected funding levels over 10 years for rare diseases in Colombia from crowdfunding, corporate donations, and SIBs were roughly $7,200, $23,000, and $12,400, respectively.Conclusions: Based on the combination of projected funding potential along with expert consensus regarding viability and operability, crowdfunding, corporate donations, and SIBs, especially in combination, have the potential to substantially improve funding for vulnerable patient populations in Colombia.

ABSTRACTBackground: Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patient safety, effectiveness, and transparent AI solutions.Objectives: Identify and summarize FDA approved RPM devices to provide information for the US medical device industry based on previous approvals and the markets’ needs.Methods: We searched publicly available databases on FDA-approved RPM devices. Selection criteria were established to classify a solution as AI. Technical information was analyzed on pre-identified 16 parameters for the qualified solutions.Results: A total of 47 RPM devices were reviewed, among which 12.8% were classified as a De Novo product and the remaining devices fell under the 510(K) FDA category. The cardiovascular (74%) AI RPM solutions dominated the US market, followed by ECG-based arrhythmia detection algorithms (59.4%), and Hemodynamics and Vital Sign monitoring algorithms (21.9%). The trend observed in the FDA rejected devices was their inability to be classified into clinically relevant categories (Criteria 2 and 3).Conclusion: The market needs more innovative RPM solutions under the De Novo category, as there are very few. The transparency is low on the technical aspect of AI algorithms. The market needs AI algorithms that can effectively classify patients rather than merely improve device functionality.

ABSTRACTIntroduction The Indian healthcare system is evolving towards better healthcare implementation and coverage. However, even today, the health-care system faces several challenges, a few of which are yet to be addressed. The present review is aimed to delineate the past and present healthcare scenarios in India, health-care policies, and other initiatives for achieving universal health coverage (UHC).Methods A literature search was done on various government databases, websites, and PubMed for obtaining data and statistics on healthcare funding, health insurance schemes, healthcare budget allocations, categories of medical expenses, government policies, and health technology assessment (HTA) in India.Results The available data indicates 37.2% of the total population is covered by any health insurance of which 78% are covered by public insurance companies. Around 30% of the total health expenditure is borne by the public sector, and there is high out-of-pocket (OOP) expenditure on healthcare.Discussion Several new health policies and schemes, an increase in 2021 budget for healthcare by 137%, vaccination drives, augmenting manufacturing of medical devices, special training packages, Artificial Intelligence/Machine Learning (AI/ML)-based standard treatment workflow systems to ensure proper treatment and clinical decision-making have been initiated by the government for improving healthcare funding, equity, and access.

ABSTRACTIntroduction: Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disease that significantly reduces occupational productivity and quality-of-life in affected patients. Italy has an estimated FMF prevalence of 1 in 60,000 people. While colchicine is the primary treatment for FMF, biologics are administered to intolerant and non-responder patients. Anakinra and canakinumab are the only biologics approved and reimbursed for FMF in Italy. Both medicines have demonstrated efficacy in FMF patients yet differ in treatment costs. This study aimed to perform a budget impact analysis (BIA) following anakinra’s reimbursement for FMF treatment, considering pharmaceutical costs from the Italian National Healthcare Service (NHS) perspective. Methods: A ‘Reference scenario’ (all patients treated with canakinumab) was compared to an ‘Alternative scenario’, with increased anakinra market shares. The target population was estimated based on the Italian population, epidemiological and market research data. Drugs costs were estimated based on Summary of Product Characteristics and net ex-factory prices. Sensitivity analyses were implemented to test results’ robustness. Results:The base case analysis showed an overall cumulative expenditure of €30,586,628 for ‘Reference scenario’ and € 16,465,548 for ‘Alternative scenario’. A cumulative savings of €14,121,080 (46.2%) was calculated over 3 years as a result of the reimbursement and increasing uptake of anakinra. The sensitivity analyses, even considering a discount of 50% for canakinumab, confirmed the base case results. Conclusions: Anakinra’s introduction, in FMF treatment, provides a financially sustainable option for Italian patients, with savings increasing according to greater use of anakinra.

ABSTRACTIntroduction: To ensure the sustainability of the AT access improvement, it is important that health system stakeholders have timely, analyzed information accessible for reference and decision-making support. In this study, we projected the direct costs required as well as the expected direct medical cost-offset and productivity benefits resulting from improving the disease control. Methods: We implemented a deterministic, prevalence-based mathematical model to project the annual cost of rheumatoid arthritis (RA) management within the public healthcare system in Malaysia. We also calculated the annual productivity loss due to uncontrolled RA in monetary value. Using the projection model, we compared the projected costs of the status quo scenario vs. several scenarios of improved advanced therapy (AT) access over a 5-year period. Results: We projected that between 10,765 and 11,024 RA patients in Malaysia over the period of 2020–2024 will need access to AT due to treatment failure with conventional synthetic disease modifying antirheumatic drugs (DMARDs). The projected net total medical cost under the status quo scenario were 163.5 million annually on average (approximately MYR 15,000 per patient per year). Cost related to health service utilization represented the heaviest component, amounting to 71.8% followed by drug cost (24.7%). Under the access improvement scenarios, drug cost constituted a higher proportion of the total medical, ranging from 25.6% to 30.4%. In contrast, the cost of health service utilization shown a reverse pattern (reducing to between 66.3% and 70.1%). Productivity costs were also expected to reduce as AT access improved leading to better outcomes. Treatment shifts to targeted synthetic DMARDs in anticipation of price adjustment appeared to have a cost saving advantage to the health system if all other parameters remain unchanged. Discussion: Improving AT access for RA patients towards the aspirational target appeared to be feasible given the current health budget in Malaysia. Broader socio-economic consequences of productivity and income loss should be included as an important part of the policy consideration. The financial implication of different AT utilization mixes and the anticipated price adjustment will likely result in some cost saving to the health system.

ABSTRACTBackground The evidence base of tisagenlecleucel is uncertain.Objective To evaluate the cost-effectiveness of tisagenlecleucel. To conduct expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.Study Design A three-state partitioned survival model. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 5 years; general population mortality with a standardised mortality ratio was then applied. EVPI and EVPPI were scaled up to population according to the incidence of the decision.Setting Irish healthcare payer.Participants Patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL).Interventions Tisagenlecleucel versus Salvage Chemotherapy (with or without haematopoietic stem cell transplant).Main Outcome Measure Incremental cost-effectiveness ratio (ICER). Population EVPI and EVPPI.Results At list prices, the ICER was €119,509 per quality-adjusted life year (QALY) (incremental costs €218,092; incremental QALYs 1.82). Probability of cost-effectiveness, at a €45,000 per QALY threshold, was 0%. Population EVPI was €0.00. Population EVPI, at the price of tisagenlecleucel that reduced the ICER to €45,000 per QALY, was €3,989,438. Here, survival analysis had the highest population EVPPI (€1,128,053).Conclusion Tisagenlecleucel is not cost-effective, versus salvage chemotherapy (with or without haematopoietic stem cell transplant), for R/R DLBCL in Ireland. At list prices, further research to decrease decision uncertainty may not be of value.

ABSTRACTBackground: The increasing prevalence of obesity imposes a significant cost burden on individuals and societies worldwide.Objective: In this nationally representative study, the association between body mass index (BMI) groups and the number of metabolic comorbidities (MetC) with total direct costs was investigated in the Finnish population.Study design, setting, and participants: The study cohort included 5,587 adults with BMI ≥18.5 kg/m2 who participated in the cross-sectional FinHealth 2017 health examination survey conducted by the Finnish Institute for Health and Welfare. Data on healthcare resource utilization (HCRU) and drug purchases were collected from national healthcare and drug registers.Main outcome measure: The primary outcome was total direct costs (costs of primary and secondary HCRU and prescription medications).Results: Class I (BMI 30.0–34.9 kg/m2) and class II – III (BMI ≥35.0 kg/m2) obesity were associated with 43% and 40% higher age- and sex-adjusted direct costs, respectively, compared with normal weight, mainly driven by a steeply increased comorbidity in the higher BMI groups. In all BMI groups combined, individuals with ≥2 MetCs comprised 39% of the total study population and 60% of the total costs.Conclusion: To manage the cost burden of obesity, treatment should be given equal consideration as other chronic diseases, and BMIs ≥30.0 kg/m2 should be considered in treatment decisions.

ABSTRACTBackground Perianal fistula (PF), a complication of Crohn’s disease (CD), affects health-related quality of life (QOL).Objective To elucidate QOL of health states corresponding to different stages of PF associated with CD in Japan.Method This cross-sectional, observational, web-based questionnaire survey assessed eight different health states in patients with CD and PF and individuals without CD (non-patients) from the Medilead Healthcare Panel (MHP) and determined the utility values (QOL scores) in each health state by the time trade-off method. In patients, we determined also the utility value of the current health state associated with CD and the PF. The analysis excluded respondents with logical inconsistencies.Results The analysis included 82 patients and 576 non-patients with the same sex and age distribution as the Japanese population. In both groups, mean utility values were higher in remission (patients, 0.78; non-patients, 0.51) than in non-remission states, with lowest values for poor prognosis after proctectomy (patients, 0.13; non-patients, −0.10) and highest values for the state with mild symptoms (patients, 0.60; non-patients, 0.30). In patients, the mean utility value of the current health state was 0.71.Conclusion QOL decreases with increasing severity of PF and is lower for good prognosis after proctostomy than for remission.

ABSTRACTBackground The economic consequences of the recent COVID-19 pandemic were substantial. However, direct medical costs in France have not been determined.Objective To describe patient characteristics, intensity of care, mortality, and direct medical costs in patients hospitalised for COVID-19 infections in France.Study design A retrospective study of the French national hospital claims database for 2020.Setting Hospital care.Patients or other participants All patients hospitalised for COVID-19 in 2020 were included and classified by hospitalisation duration into acute phase and prolonged COVID-19.Intervention Stratification by intensity of care (Level 1: no or low-flow oxygen support; Level 2: non-invasive ventilation; Level 3: mechanical ventilation).Main outcome measure Cost of hospital care in 2020 Euros from a payer perspective.Results 199,455 patients were hospitalised for COVID-19 in France in 2020. 17,824 patients (8.9%) received mechanical ventilation and 32,602 patients (16.3%) died. Mean per patient cost was €5,510 ± 7,142. This cost was highest in patients receiving Level 3 care, patients aged >80 years and in those with prolonged COVID.Conclusion The economic burden of hospitalisations for COVID-19 infections in France during 2020 was substantial. The study provides robust baseline data to benchmark advances in the standard of care and to nurture epidemiological models.

ABSTRACTBackground Clinician’s choice of hypoallergenic formulas in the first-line management of cow’s milk protein allergy (CMPA) should be informed by evidence on clinical efficacy and cost-effectiveness.Objective We compare the cost-effectiveness of amino acid-based formula (AAF), extensively hydrolyzed casein formula with Lactobacillus rhamnosus Gorbach Goldin (EHCF+LGG), extensively hydrolyzed whey formula (EHWF), and rice hydrolyzed formula (RHF) in non-breastfed children in France.Methods Immunotolerance and atopic manifestations’ prevalence were based on a prospective non-randomized study with a 36-month follow-up. Resource utilization was sourced from a survey of French clinicians, and unit costs were based on national data. Costs and health consequences were discounted at 2.5% annually. Results were reported using the Collective and French National Health Insurance perspectives.Results Children receiving EHCF+LGG were predicted to require less healthcare resources, given their reduced prevalence of CMPA symptoms at 3 years. In the base case, EHCF+LGG led to savings of at least €674 per child compared to AAF, EHWF, and RHF at 3 years, from both perspectives. Nutrition had the highest economic burden in CMPA, driven by hypoallergenic formulas and dietetic replacements costs. Results were robust to one-way and probabilistic sensitivity analyses.Conclusions EHCF+LGG was associated with more symptom-free time, higher immune tolerance, and lower costs.