Methodological tutorials support the application of statistical methods in health technology assessment. Using an R tutorial for trial-based economic evaluation as a case study, we explored how applied researchers engaged with the tutorial and identified factors influencing its usability and perceived value. We conducted a three-step test interview study with applied researchers without experience with the tutorial (n = 17) and semi-structured interviews with applied researchers who had previously used it in their own work (n = 3). Participants worked through the tutorial whilst thinking aloud, followed by focussed and reflective interviews. Qualitative data were analysed using reflexive thematic analysis. Three themes were generated: (1) ‘getting in and getting it to run’, describing technical barriers; (2) ‘understanding and making sense of what tutorials teach’, reflecting challenges in applying methods and interpreting outputs; and (3) ‘adapting and applying tutorials in practice’, highlighting difficulties modifying code for real-world datasets. Barriers stemmed not only from methodological complexity but also from software setup, unclear terminology, limited coding skills and lack of support. Despite these, participants described tutorials in general as efficient learning tools when clearly structured, transparent and adaptable. We derived practical considerations that may help improve the accessibility, clarity and adaptability of future methodological tutorials. Methodological tutorials can support the learning and application of statistical methods, but their effective use depends on technical accessibility, user capability and access to support. These findings may inform future tutorial design and the development and validation of more structured guidance, such as a checklist for tutorial development.
Huntington’s disease (HD) is a rare neurodegenerative condition caused by mutations in the huntingtin gene. Emerging therapies such as Tominersen and AMT-130 have potential to treat HD, highlighting the importance of evaluating their cost-effectiveness. This study evaluates the cost-effectiveness of Tominersen and AMT-130 versus standard of care (SoC) for early stage HD in the US. A Markov model with four health states (early, middle, late, and death) was developed to estimate lifetime costs and benefits from a modified societal perspective, with death as an absorbing state. The model used 1-year cycle length with half-cycle correction, and an annual discount rate of 3
The aim of this study was to evaluate the budget impact of introducing the subcutaneous (SC) form of ocrelizumab for patients with relapsing forms of multiple sclerosis (RMS) with high disease activity (HA) despite previous treatment or with rapidly evolving severe (RES) disease in Italy from the hospital perspective. A 3-year dynamic budget impact model with a Markov structure was developed de novo to simulate patient transitions across Expanded Disability Status Scale–defined health states. The current scenario (without ocrelizumab SC) was compared with an alternative scenario reflecting expected ocrelizumab SC uptake. Number of eligible patients reflected Italian population and projected market shares. Efficacy inputs were obtained from published literature, with adherence and persistence sourced from Italian real-world data. Unit costs—including drug administration, monitoring, adverse events, disease management, and relapse—were collected from Italian sources. Drug acquisition costs were considered in a scenario analysis. Parameter uncertainty was explored through deterministic sensitivity and scenario analyses. Over 3 years, 22,128 patients were eligible, of whom 5389 were assumed to receive ocrelizumab SC. Ocrelizumab SC introduction would generate cumulative savings of about €2.0 million, mainly from reduced administration and monitoring costs, with additional savings from relapse and disease management expenditures. When drug acquisition costs were included, total savings rose to roughly €7.1 million. Sensitivity analyses confirmed the robustness of these findings. Ocrelizumab SC may be a cost-saving and resource-efficient option for HA/RES RMS management in Italy. By reducing administration time while maintaining the established clinical profile of the intravenous (IV) formulation of ocrelizumab, its adoption can enhance hospital efficiency and deliver meaningful budgetary savings.
Severe uncontrolled asthma (SUA) represents a complex and heterogeneous form of asthma that persists despite treatment. Allergic immunoglobulin E (IgE)-mediated SUA can be treated with tezepelumab or omalizumab. The aim was to estimate the cost-effectiveness of tezepelumab compared to omalizumab in the treatment of patients with allergic SUA, from the perspective of the Spanish National Health System (NHS). A Markov model was developed with a time horizon of 60 years, 28-day cycles, and five health states: controlled asthma; uncontrolled asthma; controlled asthma with exacerbation; uncontrolled asthma with exacerbation; and death. The efficacy parameters of the model were based on the NAVIGATOR and SOURCE clinical trials for tezepelumab and standard therapy, and on a network meta-analysis for omalizumab. Utilities and disutilities were extracted from NAVIGATOR and SOURCE and from the literature. The model considered direct costs (€, 2025): pharmacological, administration, exacerbations, disease management, and adverse events arising from oral corticosteroid use, obtained from Spanish data sources. Incremental costs per quality-adjusted life-year (QALY) gained were estimated for tezepelumab compared to the 106 omalizumab dosing profiles defined by weight and IgE. The results were contextualized to the Spanish setting using weight and IgE data obtained from the Primary Care Clinical Database and the literature. Deterministic sensitivity analysis (DSA) and probabilistic sensitivity analysis (PSA) were performed. Tezepelumab was cost-effective compared with omalizumab (450 mg/4 weeks; incremental cost-effectiveness ratio €17,213.44/QALY), considering a willingness-to-pay threshold of €30,000/QALY, and was dominant (more effective and less costly) at higher doses. This represents 69.81
Background/AimImmune checkpoint inhibitors (ICIs) have revolutionized cancer therapy, yet their uptake in low- and middle-income areas is hindered by high prices and uneven distribution. Anhui Province, China-characterized by marked urban-rural economic contrasts-offers a critical setting to examine real-world access to these agents. The objective of this study was to assess the availability, price levels, and affordability of ICIs across Anhui Province and to identify policy levers that could narrow observed access gaps.Materials and MethodsWe applied the World Health Organization/Health Action International (WHO/HAI) standardized survey methodology to evaluate the availability, price differentials, and economic burden of immune checkpoint inhibitors (ICIs) marketed in Anhui Province, China. The survey was conducted in 2025 across 199 public hospitals. Affordability was further assessed using a four-tier framework comprising the WHO/HAI standard indicator (defined as the number of days' wages required to afford 30 days of treatment), adjusted using local urban and rural per capita disposable income to account for China's socioeconomic heterogeneity, and incorporating insurance reimbursement scenarios on the basis of Anhui provincial policy (85% for urban employee insurance and 70% for urban-rural resident insurance) to estimate out-of-pocket expenditure. In addition, catastrophic health expenditure (CHE), defined as household out-of-pocket health spending exceeding 40% of non-food expenditure, was used to capture household-level financial risk beyond individual income-based measures. Availability was defined as the proportion of facilities stocking a medicine on the survey day. Prices were collected at unit level and summarized as medians across facilities, with affordability assessed using the median price ratio (MPR), calculated as median local unit price relative to the international reference price (IRP) from the MSH International Drug Price Indicator Guide, in line with WHO/HAI methodology.ResultsSurveying 199 hospitals, we found that domestic PD-1 inhibitors-sintilimab, camrelizumab, and tislelizumab-were stocked in roughly 4 out of 5 facilities, whereas 11 of the 15 mainly imported ICIs appeared in fewer than 16% of hospitals; although some imported agents had acceptable median-price ratios (0-2), their absolute prices remained several-fold higher than domestic alternatives; thus even after insurance (85% urban, 70% rural), a year's treatment with cadonilimab or durvalumab still exceeded the catastrophic-expenditure threshold by up to 40 times for rural households, while domestic sintilimab or camrelizumab stayed well below that line.ConclusionsDomestic ICIs are broadly available and relatively affordable, whereas imported brands remain scarce and financially out of reach; boosting reimbursement limits, expanding centralized procurement, and favoring cost-effective domestic options may help reduce Anhui's access gap, although their implementation should take budget constraints and opportunity costs into account.
Varicella vaccination is not currently included in Portugal’s childhood national immunization program. We modeled the clinical and economic impact of a two-dose universal varicella vaccination (UVV) program in Portugal. A dynamic transmission model was adapted to Portugal to assess the impact of UVV over a 50-year time horizon. Two two-dose UVV strategies (first dose at 12 months, second dose at 5 years) were compared to no vaccination. Clinical outcomes included varicella and herpes zoster incidence rates, cases, deaths, quality-adjusted life years (QALYs) lost, and varicella-related health care resource use. Costs were assessed from the payer perspective (direct costs of treatment) and societal perspective (direct costs plus indirect costs of lost workdays). Both UVV strategies reduced total varicella cases by 79.4–85.3
Universal health coverage (UHC) is central to the global health agenda. South Africa is in the process of developing a single financing system for UHC through a National Health Insurance (NHI). The NHI Act legislates the establishment of a health technology assessment (HTA) body to develop and, over time, add to an explicit package of health services funded by the NHI. This study aims to identify data that is publicly available for conducting HTA using the South African Values and Ethics for Universal Health Coverage (SAVE-UHC) framework. A targeted literature review was conducted to identify data sources for conducting HTA using the SAVE-UHC framework. The SAVE-UHC lists 12 domains that can be used to conduct HTA. We searched for published literature on PubMed using the following search terms: ‘South Africa’, ‘healthcare’, ‘data sources’, ‘databases’ and ‘evidence’ in the Title/Abstract fields. We also used our experience in the field to search for existing data from local and international organisations. We then mapped the data sources to the SAVE-UHC domains. South Africa has some data to conduct HTA using the SAVE-UHC domains. Data for six of the domains (burden of disease, harms and benefits, cost effectiveness, budget impact, personal financial impact and equity) is available from local and international databases. Some data for the domain ‘system factors and constraints’ is available from the Office of Health Standards Compliance and the Human Resource Strategy for Health. Data on the five remaining domains (respect and dignity, solidarity and social cohesion, impact on personal relationships, impact on safety and security and ease of suffering) is limited. There are a number of data sources available to inform health technology assessment in South Africa. However, the data sources available only measure some of the value elements in the SAVE-UHC framework. Thus, South Africa needs to invest in healthcare data collection and management under the NHI to ensure healthcare decisions are made using relevant and recent evidence.
BackgroundThe Norwegian public-private partnership, CONNECT, identified the reimbursement submission case of selpercatinib-a first-line targeted therapy for RET fusion-positive non-small cell lung cancer (NSCLC)-as a unique opportunity to assess the role of structured expert elicitation (SEE) in conjunction with phase II evidence.ObjectiveThis study aims to compare the potential of using SEE and other sources of external evidence with phase III clinical trial results to explore the possibility of facilitating earlier (conditional) reimbursement decisions.MethodsWe conducted a series of cost-effectiveness analyses, grouping different sources of evidence (i.e., published clinical evidence and SEE) to estimate the long-term health and economic consequences of selpercatinib versus pembrolizumab plus pemetrexed and platinum-based chemotherapy for patients with advanced RET fusion-positive NSCLC in Norway. We developed a probabilistic partitioned survival model to estimate and compare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) associated with evidence potentially available early in the product lifecycle, including SEE, with those using phase III evidence.ResultsICERs consistently ranged between $8257 and $19,819 per QALY gained when we used SEE, either alone or in combination with additional external evidence, or when we used the phase III randomized controlled trial (RCT) evidence.ConclusionsThe consistent cost-effectiveness results between pre-submission and post-submission phases support the potential of SEE to complement health technology assessment (HTA) and potentially inform earlier (conditional) reimbursement decisions for this single case study. However, future research should focus on refining SEE protocols for continued methodological development and validation to improve generalizability and applicability.
Health utility values (HUVs) are fundamental parameters in economic evaluations used to assess the value of healthcare interventions. However, patients living with breast cancer in low- and middle-income countries (LMICs) face distinct burdens compared with those in high-income countries (HICs), driven by profound disparities in resources, infrastructure, and cultural context. High reliance on HUVs from HICs risks misrepresenting the specific disease burden in LMICs, introducing uncertainty in local healthcare resource allocation. This protocol outlines the methods for a planned systematic review and meta-analysis to synthesize available evidence from LMICs to generate pooled EuroQol-5 Dimensions HUV estimates that are contextually appropriate for local decision making. By following the Preferred Reporting Items for Systematic Review and Meta-Analysis (PRISMA) guidelines, this protocol is registered in PROSPERO (CRD420251208371). The initial literature search will be conducted in Cochrane CENTRAL, PubMed, Web of Science core collection and Scopus using keywords such as ‘health-related quality of life’, ‘EuroQol-5 Dimensions’, ‘breast cancer’ and ‘low-middle-income countries’. Risk-of-bias assessment will be conducted using the Cochrane Risk-of-Bias Tool (RoB 2) and Newcastle-Ottawa Scale (NOS) depending on study designs. R packages (metaprop) will be used to pool the mean HUVs of people living with breast cancer using fixed-effect (inverse variance method) and random-effect models (Der Simonian-Laird method). Subgroup analysis will be conducted based on different categories of interest such as EQ-5D versions, country income level and clinical stagings. This protocol provides a systematic guide to synthesize EQ-5D utility values specific to breast cancer in LMICs. The anticipated findings will establish a critical evidence base for decision making in resource-constrained settings. By generating contextually relevant estimates, this will minimize parameter uncertainty and support more equitable resource allocation. Hence, it will contribute to health systems that are responsive not only to the clinical burden of the disease but also to the multidimensional well-being of the patient and families it affects.
B3 breast lesions, of uncertain malignant potential, pose diagnostic challenges owing to their intermediate malignancy risk. While surgical excision (SE) is traditionally used for definitive diagnosis, vacuum-assisted excision (VAE) offers a less invasive alternative. As diagnostic practices evolve, this study assesses the financial impact of adopting VAE for B3 lesion management from the hospital perspective within the Italian healthcare system. A budget impact analysis (BIA) was conducted from the hospital perspective over a 5-year time horizon. Clinical complication rates for SE and VAE were derived through a systematic literature review and meta-analysis. A micro-costing approach was used to estimate direct medical costs on the basis of detailed resource utilization data collected via structured questionnaires administered across five Italian hospitals. One-way sensitivity analyses and scenario analyses were conducted to test the robustness of the results. VAE was associated with significantly lower costs than SE, mainly owing to reduced hospitalization, operating room use, and personnel time. The average per-patient cost was €820 for VAE versus €1663 for SE, yielding savings of €843. Over 5 years, with VAE adoption rising from 20
BackgroundCommunity-based human papillomavirus (HPV) self-sampling can expand cervical cancer screening coverage in rural settings, but local programmatic cost evidence is limited.MethodsWe conducted an activity-based micro-costing study from the healthcare system perspective to estimate start-up (January 2022-February 2023) and implementation (March-December 2023) costs of a community-based HPV self-sampling screen-triage approach in Siem Reap Province, Cambodia. Resource use and unit costs were measured using direct observation, semi-structured interviews with program staff, and review of budgets and financial reports. All costs were converted to 2024 US dollars.ResultsBetween January 2022 and December 2023, 7524 women were screened; 359 (4.77%) tested HPV-positive and 312 of 359 (86.91%) completed triage during the study period. Total programmatic cost was US$204,507.14, yielding an average cost of US$27.18 per woman screened and US$569.66 per HPV-positive case identified. Laboratory testing was the largest cost component (55.01% of total programmatic cost), followed by the community-based self-sampling activity (31.12%). Estimated incremental unit costs for triage options were US$3.69 for visual inspection of cervix with acetic acid (VIA), US$16.42 for partial HPV genotyping (types 16 and 18), and US$32.92 for colposcopy per woman receiving the respective triage test.ConclusionsCommunity-based HPV self-sampling can be delivered at moderate programmatic cost in rural Cambodia, with overall costs driven primarily by laboratory testing and community-based screening delivery. These findings provide inputs for budgeting and planning scale-up of HPV screening from a healthcare system perspective in Cambodia.
Survival data from pivotal clinical trials are critical for estimating quality-adjusted life years (QALYs). However, immature survival data require extrapolation beyond observed follow-up to project outcomes, introducing potential prediction error into QALY estimates used in economic evaluations. Immune checkpoint inhibitors (ICIs) present unique extrapolation challenges due to delayed responses and extended survival benefits. We therefore quantify the QALY prediction error of early extrapolations by benchmarking them against updated follow-up at a common time horizon. Using reconstructed individual patient data derived from published Kaplan–Meier curves of pivotal trials, this study assessed the accuracy of early survival extrapolations for ICIs approved in China by comparing early projections with QALYs obtained from updated data at the same horizon. A partitioned-survival framework using overall survival (OS) and progression-free survival (PFS) informed state occupancy, and QALYs were obtained via restricted mean survival time (RMST) integration of health-state utilities at the target horizon. Statistical analyses evaluated bias, precision, and agreement between extrapolated and updated QALY estimates. Linear regression and sensitivity analyses assessed the impact of target extrapolation horizon (T) on prediction error. In total, 14 randomized controlled trials (4839 patients) were included for analysis. The mean deviation between extrapolated and observed QALYs was − 0.01 (95
Background Economic models support healthcare decision makers to efficiently assess value and allocate resources; formal validation is critical to ensure confidence in model outputs. The Obesity Lifecycle Model is a patient-level simulation model capturing natural history, clinical complications, quality-of-life and economic outcomes associated with obesity; however, it has yet to undergo formal external and cross-validation. Objective The aim of this study was to validate the Obesity Lifecyle Model as per best practice guidelines from the Professional Society for Health Economics and Outcomes Research (ISPOR) and the Society for Medical Decision Making (SMDM) Task Force. Methods Relevant data sources and outcomes were identified for all validations. Selected validation studies informed the model to simulate study outcomes. The model was populated with study characteristics to reproduce studies used in model development (dependent validation), studies not used in model development (independent validation), or other published models (cross-validation). Accuracy between predicted and observed outcomes was assessed using standard statistical methods and mean error calculations. Results The model demonstrated overall concordance with observed outcomes, supported by coefficient of determination (R-2) and ordinary least squares linear regression line (OLS LRL) estimates generally close to 1.0. Independent validation showed an underprediction for cardiovascular disease (CVD) and mortality with an OLS LRL of 0.9309 and an R-2 of 0.8984 across all populations (normoglycaemic/prediabetic populations: OLS LRL = 0.9485, R-2 = 0.8854; T2DM populations: OLS LRL = 0.9208, R-2 = 0.9068). Conclusions The Obesity Lifecycle Model demonstrates favourable concordance with observed clinical and quality-of-life outcomes, supporting its use for evaluating obesity-related complications and informing healthcare decision making.
An adverse drug reaction (ADR) is a harmful, unwanted patient response to a drug that happens at doses normally used for prevention, diagnosis, or treatment. Despite limited and inconsistent primary evidence on the cost of ADRs in Africa, this systematic review aims to synthesize the magnitude of ADR-related costs and assess the reporting quality of existing studies. The authors followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist. Three databases (PubMed, Scopus, and ScienceDirect) and a hand search were employed. All empirical African cost-of-illness studies on ADRs were included regardless of publication year, disease type, or population, while letters to the editor, case reports, conference proceedings, and abstracts were excluded. Two independent authors conducted screening, data extraction, and quality assessment. A consensus-based cost-of-illness checklist was used to assess reporting quality. The primary outcome was the cost of adverse drug reactions, reported in 2024 international dollars (I); the secondary outcome was the reporting quality of studies. A total of 324 studies were identified, of which eight were included in the final analysis. Nearly all studies reported only direct medical costs, excluding direct non-medical and indirect costs. ADR-related hospitalization costs per patient ranged from I54.75 in Nigeria to I7438.47 in South Africa. Costs were mainly driven by hospitalization, medications, and investigations, and influenced by patient, disease, and health system–related factors. While all studies described their population, objectives, time horizon, and data collection methods, many failed to report important methodological details. Adverse drug reactions impose a substantial economic burden through direct medical costs influenced by multiple factors; however, many studies lacked key reported details. Future research should adopt recommended methodologies and comprehensive cost reporting to improve the reliability of cost estimates.
The objective was to evaluate the overall economic burden of spinal muscular atrophy (SMA) in Singapore. A retrospective cohort study of electronic medical records and billing data was used to obtain medical costs from public healthcare consumption. Private medical costs, nonmedical costs from transportation and hired domestic helpers, and indirect costs due to caregiver productivity loss were estimated with a cross-sectional caregiver survey. The survey also examined intangible negative and positive effects on these caregivers. All costs were adjusted to 2024 Singapore dollar (SGD). A total of 61 patients were identified in the electronic medical records, and 15 caregivers were interviewed. One caregiver who took care of two patients with SMA was excluded from the analysis. The gross cost of inpatient admission is SGD 1663.81 (standard deviation [SD] = 986.03) per day with mean length of stay of 6.22 days (SD = 10.89) per year, while annual mean outpatient cost is SGD 940.31 (SD = 1071.28). In addition to public healthcare services, private services incur an additional SGD 5992.35 (SD = 7557.33) per year, with fixed costs for respiratory devices and mobility aids at SGD 5677.06 (SD = 4519.57) and SGD 12,412.70 (SD = 8307.35), respectively. Nonmedical costs from hiring a domestic helper, medical-related transport, and nonmedical transport amount to SGD 7002.22 (SD = 6516.36), SGD 387.86 (SD = 196.36), and SGD 840.95 (SD = 1067.81) per annum, respectively. Paid work productivity loss is estimated at SGD 42,932.88 per family per year. Average overall economic burden is estimated at SGD 62,004.03, SGD 10,840.13, and SGD 59,629.64 per patient per year from the societal perspective, healthcare system perspective, and patient perspective, respectively. SMA poses a significant economic burden on patients, families, and the healthcare system. This study informs policy discussions for SMA management, advocating for a comprehensive, compassionate approach and societal perspective in economic evaluations to capture all costs and benefits.
Lung cancer screening (LCS) with low-dose computed tomography (LDCT) reduces mortality. Ireland will likely adopt a national screening programme in the coming years, and evidence on the size of the eligible population is required to inform planning. The objective was to estimate the number and characteristics of individuals in Ireland eligible for LCS under different international criteria and to project future uptake. We combined population data from the 2022 Census and subsequent projections with smoking history from the 2017 Eurobarometer survey. Eligibility was assessed under four criteria: Irish pilot (55–74 years, ≥ 20 pack-years, quit < 15 years), US Preventive Services Task Force (USPSTF) 2013, USPSTF 2021, and NELSON. Projections to 2045 accounted for demographic change and smoking trends. Expected uptake was estimated using participation rates from SUMMIT, a large UK LCS implementation trial. In 2022, 245,105 individuals met the Irish pilot criteria, and 345,328 met USPSTF 2021. Eligibility remains broadly stable until the mid-2030s, then declines across all criteria. Relative differences between criteria persist over time, with USPSTF 2021 consistently identifying the largest group. Applying SUMMIT uptake rates, around 36,000 LDCT scans annually would be required at steady state. Approximately a quarter of a million people in Ireland would currently be eligible for LCS, rising to more than 340,000 under broader criteria. Numbers are expected to remain high for the next decade before gradually declining. These findings provide key evidence for service capacity planning and highlight the importance of flexible programme design to meet future demand.
Productivity losses from illness, treatment, and informal caregiving impose a substantial economic burden on patients, caregivers, employers, and society in the United States (US), but measurement remains inconsistent and often omits key domains, such as presenteeism and unpaid non-market work productivity. These gaps limit the integration of productivity into economic evaluations and policy and may lead to undervaluation of interventions that improve health and reduce disease burden. The aim was to identify evidence priorities and methodological improvements for measuring and valuing productivity in US economic evaluations. A targeted literature review (TLR) of US-based studies published from 2020 to 2024 was conducted to evaluate measurement of absenteeism, presenteeism, or unpaid non-market work productivity. Definitions, instruments, valuation methods, and reporting practices were extracted. Findings informed a virtual workshop with eight stakeholders, who completed prioritization exercises and participated in facilitated discussions to refine evidence priorities and methodological recommendations. Thirty studies met the TLR inclusion criteria. Absenteeism was assessed in 77
Background/ObjectivesPostmenopausal osteoporosis (PMO) is a major public health and economic burden in the USA. The objective of this study was to evaluate the cost-effectiveness of biosimilar denosumab compared with alendronate, risedronate, ibandronate, and zoledronic acid in treating women with PMO at high risk of fracture from a US payer perspective.MethodsA cost-effectiveness analysis was conducted using a Markov cohort model and a lifetime horizon was applied to capture long-term costs and effects. Treatment effects were based on published network meta-analyses. The cost of biosimilar denosumab was estimated on the basis of assumptions; other costs were obtained from publicly available sources. Main outcomes included quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratios (ICERs).ResultsBiosimilar denosumab was associated with greater efficacy and higher costs versus all comparators. Over a lifetime horizon, the ICER was $101,017, $93,544, $100,515, and $144,995 per QALY gained for biosimilar denosumab compared with alendronate, risedronate, ibandronate, and zoledronic acid, respectively. Cost-effectiveness was most sensitive to starting age, discount rate, treatment duration, and biosimilar price. At a willingness-to-pay (WTP) threshold of $150,000 per QALY gained, biosimilar denosumab was likely to be cost-effective compared with alendronate, risedronate, ibandronate, and zoledronic acid in the treatment of PMO. At a WTP threshold of $100,000 per QALY gained, biosimilar denosumab was likely to be cost-effective relative to risedronate, while ICERs relative to alendronate and ibandronate only marginally exceeded this WTP threshold.ConclusionsBiosimilar denosumab was estimated to be cost-effective compared with alendronate, risedronate, ibandronate, and zoledronic acid at a WTP threshold of $150,000/QALY gained and compared with risedronate at a threshold of $100,000/QALY gained. As a lower-cost alternative to reference denosumab, biosimilar denosumab may enhance patient access to effective PMO treatment in the USA.
BACKGROUND:Multi-attribute utility instruments (MAUIs) are commonly used in health economics to measure health-related quality of life (HRQoL), yet their sensitivity to different health domains varies. This study examines the sensitivity of six widely used MAUIs-EQ-5D, SF-6D, HUI3, 15D, AQoL-4D, and AQoL-8D-to the eight dimensions of the SF-36 survey. METHODS:We analyzed the associations between SF-36 dimensions and utility scores generated by each MAUI using regression models, focusing on the eight SF-36 domains as predictors. Our analyses used data from the Multi-Instrument Comparison (MIC) project, a cross-national project comprising 8022 respondents from Australia, Canada, Germany, Norway, the United Kingdom, and the United States, including both general population participants and individuals with a range of chronic health conditions. The sensitivity of each instrument was further evaluated through simulations of health interventions targeting mental health, pain relief, stress management, and post-surgical recovery. RESULTS:The analysis revealed distinct sensitivity patterns across instruments. The SF-6D and 15D were particularly sensitive to social and role-oriented domains, while EQ-5D and HUI3 demonstrated greater sensitivity to physical health dimensions, especially bodily pain and physical functioning. AQoL-4D and AQoL-8D showed strong sensitivity to mental health, indicating their suitability for mental health-focused interventions. Regression models identified mental health, bodily pain, and physical functioning as the primary predictors of utility scores across MAUIs. Simulation results further highlighted that mental health interventions were best captured by AQoL-8D and HUI3, while EQ-5D and HUI3 were most sensitive to pain-focused interventions. CONCLUSIONS:The choice of MAUI significantly impacts observed HRQoL outcomes depending on the health domains most relevant to the intervention. This study underscores the need for careful instrument selection in HRQoL assessments to ensure alignment with specific health contexts. Future research should validate these sensitivity patterns across diverse populations and clinical applications to optimize MAUI selection in health economic evaluations.
