PharmacoEconomics最新文献

Background: The choice of perspective in valuation tasks is likely to affect the scale of EQ-5D-Y-3L value sets, but less is known about how it affects the relative importance of different dimensions.

Objectives: The aim of this study was to examine how preferences for EQ-5D-Y-3L health states differ according to different perspectives utilising two methods: the Online elicitation of Personal Utility Functions (OPUF) tool and a discrete choice experiment (DCE).

Methods: An online survey was designed containing the OPUF tool and a DCE. Adult respondents from the United Kingdom were randomised to one of five different perspective arms: (1) 4-year-old child, (2) 10-year-old child, (3) a child of unspecified age, (4) another adult, and (5) own health. The resulting OPUF value sets (social utility functions), and relative importance scores for the five dimensions from both methods, were compared across perspectives.

Results: Results differed by perspective in both valuation tasks. In both tasks, 'looking after myself' was less important and 'pain or discomfort' was more important in the child perspectives than in the adult perspectives. Furthermore, the scale of the value sets produced by the OPUF tool differed by perspective, with the value of the worst health state being significantly lower in the adult perspectives than in the child perspectives.

Conclusion: Our results suggest that the valuation of the EQ-5D-Y-3L is affected by the perspective that adult respondents are asked to take. Researchers should be aware of the potential impact and ensure that relevant stakeholders understand this when designing valuation studies.

Background: Recently, the gene therapy eladocagene exuparvovec received accelerated approval from the US Food and Drug Administration (as eladocagene exuparvovec-tneq) for treatment of aromatic L-amino acid decarboxylase deficiency (AADCd), a rare, infantile-onset disorder characterized by developmental delays.

Objectives: To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).

Methods: Multistate survival modeling was implemented tracking disease progression from a "no motor function" health state to achievement of motor-function improvements, measured by: (1) multiples of the meaningful score difference (MSD) of the Peabody Developmental Motor Scales-Second Edition (PDMS-2) total score and (2) motor milestones. Eladocagene exuparvovec trials informed clinical inputs. Health-state utilities were from a US time-trade-off study that valued AADCd quality-of-life impacts. Outcomes were discounted (3%); costs were reported in 2024 US dollars. Scenario analyses, characterizing alternative approaches of the multistate survival model analyses and probabilistic sensitivity analysis to assess the impact of parameter uncertainty, were conducted.

Results: Discounted incremental quality-adjusted life-years (QALYs) for eladocagene exuparvovec were 20.83 (multiples of the MSD of total PDMS-2) and 18.44 (motor milestones). Incremental cost per QALY ranged from $199,007-$224,104. The scenario and sensitivity analyses results supported the validity of the base case analysis.

Conclusions: Eladocagene exuparvovec is associated with considerable QALY gains compared with BSC. Within the context of other ultra-rare and/or one-time treatments, eladocagene exuparvovec provides substantial clinical improvements at lower cost than many other rare-disease treatments. Findings from this study highlight that eladocagene exuparvovec is an important treatment option for patients with AADCd.

Globally, advances in methods to examine equity alongside economic evaluations are being considered for application in health technology assessment (HTA) processes which inform decisions to publicly fund pharmaceuticals and medical services. In this practical application, we focus on the Australian context and explore how several of these methods might be used to incorporate equity more explicitly in HTA decisions. Specifically, we describe distributional cost-effectiveness analysis, extended cost-effectiveness analysis, equity weighting, multi-criteria decision analysis, mathematical programming and other quantitative approaches. We consider the feasibility and suitability of each method considering contextual factors, highlight challenges, describe current use in Australia and make recommendations for their application. We argue that there are opportunities to use aspects of several different methods in the Australian context which would illuminate how costs (including out of pocket costs) and benefits are distributed across the population, how normative concerns for equity influence the calculated social value of a new technology, how equity criteria complement other measures of value and how the technology impacts non-health outcomes. However, some of the stated requirements for the methods, such as using the same social groupings for all analyses, applying cost-effectiveness thresholds and the application of algorithms may restrict the usefulness of the evidence generated in a context where there are many priority populations of interest, several aspects of equity are considered important and there is a well-established deliberative approach to decision-making. We recommend that the equity implications of new health technologies should be universally evaluated. However, a tailored and transparent approach should be taken whereby specific equity analyses consider the context of the technology and the chosen methods are well justified.

Background and objective: Although heterogeneous treatment effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) have been revealed, the heterogeneous economic value of SGLT2is in real-world type 2 diabetes (T2D) populations with diverse clinical characteristics remains unclear. We conducted subgroup cost-effectiveness analyses of SGLT2is versus dipeptidyl peptidase 4 inhibitors (DPP4is) among patients with T2D.

Methods: A multi-state transition model was used to estimate the incremental cost-effectiveness ratios (ICERs, in US$ per quality-adjusted life-years [QALYs] gained) and value-based pricing (VBP) among patients with T2D stratified by age, estimated glomerular filtration rate (eGFR), glycated hemoglobin (HbA1c), and body mass index (BMI) over 5 years and a Lifetime horizon from a healthcare sector perspective, with both costs and quality-adjusted Life years discounted at 3% annually. Model inputs included treatment effects derived from analysis of individual-level data in Taiwan, and health utilities and costs sourced from published Literature of Taiwanese populations. Deterministic and probabilistic sensitivity analyses across subgroups were performed. All costs were standardized to 2022.

Results: Over 5 years, the ICERs of SGLT2is versus DPP4is were as follows: age subgroups (< 65 versus ≥ 65 years: $26,520 versus $2298/QALY-gained), eGFR subgroups (60 ~ < 90 versus ≥ 90 ml/min/1.73 m²: $7700 versus $12,884/QALY-gained), HbA1c subgroups (< 8.5 versus ≥ 8.5%: $7001 versus $9488/QALY-gained), and BMI subgroups (< 30 versus ≥ 30 kg/m²: $7266 versus $9714/QALY-gained). Over a lifetime, the ICERs became lower, ranging from $2369/QALY-gained for those aged ≥ 65 years to $4239/QALY-gained for those aged < 65 years. Over 5 years, the annual VBP of SGLT2is ranged from $310 for those aged < 65 years to $1267 for those aged ≥ 65 years.

Conclusions: Our analysis suggests that adopting SGLT2is over DPP4is for T2D is highly cost-effective across patient subgroups, particularly for the elderly and patients with mild renal impairment.