Value in Health最新文献

Objectives: A number of HTA bodies, including NICE, have introduced 'severity modifiers' that assign greater value to health gains to patients with greater health shortfalls. Such modifiers are consistent with qualitative societal values, but their alignment with quantitative strength of preference among the public is not clear. We sought to understand quantitative preferences over severity in England and Wales, relative to NICE's severity modifier.

Methods: We elicited Person Trade-Off (PTO) weights, severity cutoffs, and attitudinal questions to understand the range of public concern for severity and the additional weight (if any) given to health gains for patients with greater health shortfalls. The questionnaire was developed via qualitative interviews and administered to a representative sample of the public in England and Wales. Severity was defined in terms of proportional (PS) and absolute (AS) health shortfall.

Results: Based on 990 complete-case responses, we find the median value of health gains to more-severe patients increases over the lower range of health shortfall and plateaus beyond 65% PS. The PTO value function and stated severity cutoffs suggest public concern for severity extends beyond NICE's current range. Our AS results are not directly comparable to NICE's discounted cutoffs.

Conclusions: Societal concern for severity appears to begin at substantially lower shortfalls, and the relative value of health gains is greater at almost every level of severity, than NICE's current modifier. Further research will be required to confirm this interpretation, especially in understanding preferences for older or younger reference ages and incorporating NICE's discounting of shortfall.

Objectives: People with serious illness approaching end of life often end up in emergency and hospital care, frequently against expressed preferences. Consequently, oncology trials record care days as a measure of treatment burden called "time toxicity." However, this measure ignores the diminishing marginal utility of time: that the value of a day is higher when fewer remain. We aimed to incorporate this relationship into time toxicity.

Methods: We derived a preference-independent time-toxicity metric sensitive to time remaining, end-weighted time toxicity. It has units of toxiles and is based on the fraction of the patient's remaining time taken up by a care visit. We trialed this metric in a cohort of Swedish cancer patients referred to palliative care.

Results: There were 192 patients in the cohort; 146 (76%) eventually enrolled in palliative care. In the cohort, mean (SD, skewness) and median (IQR) end-weighted time toxicities were 0.63 (0.89, 1.77) and 0.23 (0.03-0.81) toxiles. End-weighted time toxicity was significantly less for the group eventually enrolled in palliative care (0.52 [0.75] and 0.22 [0.04-0.63] toxiles) than for the never enrolled group (0.97 [1.18] and 0.27 [0.27-1.76] toxiles) (P = .024). A time-series analysis showed that total toxicity in never- or not-yet-enrolled patients was 120.2 toxiles, whereas total toxicity in already-enrolled patients was 1.0 toxiles.

Conclusions: A healthcare metric that incorporates diminishing marginal utility of time can highlight the effect of palliative care and could improve resource allocation and patient satisfaction with care near end of life. Further research should explore stakeholder perspectives and practical applications.

Objectives: We conducted a systematic review and bibliometric analysis to explore the economic evaluation methods, outcomes, trends, and geographical distribution of orphan drugs for rare kidney diseases (RKDs) in low- and middle-income countries over the past decade.

Methods: A systematic review was conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Studies published from 2014 to 2024 were identified in PubMed and Scopus using keywords related to orphan drugs and RKDs. Eligible studies included full economic evaluations in low- and middle-income countries, reporting outcomes, such as incremental cost-effectiveness ratio per quality-adjusted life-years gained, life-years gained, and other clinical outcomes. Study quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. Bibliometric analysis was performed with VOSviewer and Tableau.

Results: Among 406 identified studies, 16 met inclusion criteria. Cost-utility analysis was most common (88%), followed by cost-effectiveness (6%) and cost-minimization (6%). Most studies adopted a healthcare system (56%) or societal (19%) perspective. The majority used life-years and quality-adjusted life-years as outcome measures and considered direct medical costs, with 63% applying both probabilistic and deterministic sensitivity analyses. Reporting quality was high (87.5% rated as good), although gaps remained in heterogeneity, distributional effects, and funding disclosure. Overall, 63% found orphan drugs for RKDs cost-effective. Bibliometric analysis highlighted themes such as "cost-effectiveness," "advanced renal cell carcinoma," and "nivolumab." Geographically, 67% of studies were conducted in China.

Conclusions: This review unexpectedly found that many studies identified orphan drugs for RKDs as cost-effective, despite their traditionally high costs. Future evaluations should refine methods to better capture long-term value and affordability in low- and middle-income countries.

Objectives: To compare composite time trade-off (cTTO) values derived from recent and early EQ-5D-5L value set studies.

Methods: We analyzed cTTO data from early (2012 in China; 2014-2015 in Singapore) and recent (2023 in China; 2023-2024 in Singapore) EQ-5D-5L value set studies. We compared value distributions, mean values for each health state, and resultant value sets derived from these studies within each country.

Results: In China, the recent study showed a higher proportion of the highest cTTO value (ie, 1.000). Model-predicted values were higher for mild states and lower for severe states, with a wider value range (-0.541, 0.967) compared with the early study (-0.339, 0.881). In Singapore, the recent study showed a higher proportion of 1.000 value and a lower proportion of the lowest cTTO value (ie, -1.000). Model-predicted values were higher for mild and moderate states, with a wider value range (-0.653, 0.960) compared with the early study (-0.569, 0.823). In both countries, the pain/discomfort dimension showed the highest model-predicted disutility in recent studies, whereas in the early studies, mobility problems and anxiety/depression had the highest disutility in China and Singapore, respectively.

Conclusions: In both China and Singapore, observed and model-predicted cTTO values from recent EQ-5D-5L valuation studies differed from earlier ones. Although the differences may be partially attributed to advances in valuation technology and changes in population composition, this study suggests that public health preferences evolve and underscores the potential need to periodically update value sets of preference-weighted health-related quality-of-life measures.

Objectives: Tuberculosis (TB) remains a major global health challenge. Understanding the economic burden of TB in adolescents is crucial for policy making, particularly in regions with limited healthcare resources.

Methods: This study utilized disability-adjusted life-years from the Global Burden of Disease 2021 to estimate the economic impact of TB among adolescents (under 20 years of age) globally and regionally. Disability-adjusted life-years were monetized into Value of a Statistical Life-Year using the Value of a Statistical Life-Year framework, and macroeconomic data, including Gross Domestic Product (GDP) per capita (Purchasing Power Parity. Constant 2021 international dollars) from the World Bank, were incorporated. The primary outcome was the total economic burden of TB, expressed as the Value of Lost Welfare (VLW).

Results: In 2021, the global economic burden of TB among adolescents was estimated at $262.98 billion (95% uncertainty interval, UI: 198.14, 350.17), or 0.17% of global GDP (95% UI: 0.13, 0.23). The burden was high in low- and lower-middle-income regions, with VLW in low-middle sociodemographic index regions reaching $153.92 billion (2.22% of GDP). In sub-Saharan Africa, VLW was $114.40 billion (2.37% of GDP). In contrast, high-income regions, such as North America and Western Europe, had much low VLWs (less than 0.01% of GDP). Thirteen countries had VLWs exceeding $5.00 billion, with India having the highest at $82.49 billion.

Conclusions: The study highlights significant regional disparities, with particularly high burdens in low- and middle-income regions. It emphasizes the need for targeted investments in TB control in these areas.

Objectives: The increasing use of digital health technologies (DHTs) in Europe presents opportunities and challenges. Although DHTs could enhance care delivery and health outcomes, existing health technology assessment (HTA) methods often lack flexibility to address their diverse, fast-evolving nature. This article explores the perspectives of 5 stakeholder groups-policy makers, HTA agencies, technology developers, healthcare providers, and patients-regarding the development of a harmonized HTA framework for DHTs within the Horizon Europe funded European Digital Health Technology Assessment (EDiHTA) project.

Methods: Findings are drawn from mixed-method research, including stakeholder surveys, interviews and focus groups with 97 stakeholders across European countries. The analysis of the data was performed per each stakeholder group through thematic analysis to identify points of consensus before identifying needs and requirements across stakeholders, relevant to the development of EDiHTA, using the Innovation Health Technology Assessment Methods framework.

Results: Results from 8 focus groups and more than 45 interviews are presented concentrating on 5 main themes. The emerging themes focus on the need for harmonization of HTA methods related to DHTs, the current characteristics of DHTs that are most highly valued, the importance of multistakeholder collaboration, the principles of a concept design for EDiHTA framework, and the discussion on the most relevant domains and criteria to be considered.

Conclusions: Stakeholders agreed that a harmonized HTA framework is needed; however, differences persist regarding timing and flexibility of evaluation, evidence types, and stakeholder involvement. The EDiHTA project will address differences through framework piloting, supported by multistakeholder workshops and expert advisory groups.