Applied Health Economics and Health Policy最新文献

Background: Despite the high prevalence of mental disorders in China, most patients do not utilize mental health services. Digital mental health services offer a potential solution, but their full potential remains untapped. Understanding consumer preferences is key to tailoring these services and boosting acceptance and adoption.

Objective: This study aims to elicit preferences for digital mental health services in urban China.

Methods: A discrete choice experiment was conducted with 867 participants, including both current and potential consumers of mental health services, recruited from urban areas in China. Under the guidance of the Theory of Planned Behavior, attributes relevant to digital mental health services were identified through a multi-stage process including a literature review and in-depth semi-structured interviews with 30 consumers and potential consumers of mental health services, and five professionals from Internet-based healthcare and online psychological counseling fields. Six attributes were identified: (1) professional engagement; (2) intervention type; (3) endorser/recommender; (4) mode of content delivery; (5) monthly costs; and (6) privacy policy. Recruited via a market research company, participants completed a discrete choice experiment survey with 11 option sets online. Participants' choices were analyzed with a mixed logit model and further stratified by experience with mental health services, sex, education, income, age, and risk for mental health conditions. The relative importance score was calculated to interpret the results of the discrete choice experiment. Additionally, the willingness-to-pay approach was used to estimate the monetary value of each attribute level.

Results: The most important influence on the choice of digital mental health services was the level of professional engagement (β = 2.470, β = 1.011), followed by the customized and adaptive intervention (β = 0.508) and data usage solely used for iterative product upgrades (β = 0.492). The relative importance score also revealed the most important attributes for consumers were professional engagement, intervention type, and privacy policy. Considering the professional engagement attribute, consumers were willing to pay an additional ¥3916.5 per month for services provided by mental health professionals throughout. Preferences diverged based on participant characteristics including experience with mental health services, sex, and education.

Conclusions: Our results indicated a consistent preference for services provided by mental health professionals across demographics and regardless of previous mental health service experience. We also found a preference for personalized services, highlighting a shift towards customization in healthcare and emphasizing the need to customize digital mental health services.

As newborn screening (NBS) programs consider adding conditions, policymakers would benefit from evidence showing whether early screening offers greater economic and health benefits than standard clinical diagnosis and treatment. However, economic evaluations have not been consistently used to guide decisions on expansion of NBS programs globally. This narrative review discusses data-related and methodological challenges in conducting cost-effectiveness analyses (CEAs) of NBS programs including (1) difficulties in identifying appropriate comparators and related data, (2) gaps in understanding and limited data on health outcomes and costs of care among children with newborn-screened conditions who received effective treatment after clinical diagnosis without screening, (3) lack of data on short- and longer-term costs and outcomes for affected individuals identified through screening, (4) challenges in distinguishing the effects of screening from improvements in treatment over time, and (5) limited data on costs outside of the healthcare system, limiting the ability to conduct evaluations from the societal perspective. Advancements in genomic technologies, such as genomic sequencing and gene-targeted therapies, are expanding the number of conditions that are potential targets for NBS. Genomic NBS poses additional challenges for CEAs that stem from the ability to simultaneously identify hundreds or even thousands of conditions and limitations of knowledge of genotype-phenotype associations. The data-related challenges discussed herein may encourage cross-disciplinary collaboration to improve data collection. Addressing these methodological issues is imperative for generating reliable evidence on the costs and benefits of NBS program expansion, thereby supporting informed policymaking on adding new conditions to NBS programs.

Background and objective: European guidelines recommend transcatheter aortic valve implantation (TAVI; class IA) for symptomatic severe aortic stenosis (sSAS) in patients aged ≥ 75 years, if a transfemoral approach is possible. Recent cost-utility analyses based on the PARTNER 3 trial have suggested that TAVI with the SAPIEN 3 device is cost effective versus surgical aortic valve replacement (SAVR) in patients at low risk of surgical mortality in several European countries. This cost-utility analysis compares TAVI with SAPIEN 3 versus SAVR in patients with sSAS at low risk of surgical mortality from the UK healthcare system perspective, using 5-year PARTNER 3 trial data.

Methods: A two-stage, Markov-based, cost-utility analysis was performed using published and validated methodology to estimate changes in both direct healthcare costs and health-related quality of life for TAVI with SAPIEN 3 versus SAVR in patients with sSAS at low surgical risk from the perspective of the UK healthcare system. The model used a lifetime horizon with a 3.5% yearly discounting factor. Uncertainty was addressed using deterministic and probabilistic sensitivity analyses.

Results: Transcatheter aortic valve implantation with SAPIEN 3 generated an incremental health benefit of 0.47 (90% credible interval 0.35-0.60) quality-adjusted life-years per patient compared with SAVR, at an increased cost of £7999 (£852-£15,035 90% credible interval) per patient over a lifetime horizon (incremental cost-effectiveness ratio: £16,979 per quality-adjusted life-year gained). Transcatheter aortic valve implantation has a 63-90% probability of cost effectiveness based on a £20,000-£30,000 willingness-to-pay threshold. Transcatheter aortic valve implantation remained cost effective across most deterministic sensitivity analyses, confirming the relative robustness of the results.

Conclusions: Transcatheter aortic valve implantation with SAPIEN 3 has a 63-90% probability of being cost effective compared with SAVR for low-surgical-risk patients with sSAS. These findings may inform policy decision making in the management of this patient group.

Background: The inappropriate use of antibiotics is a key driver of antimicrobial resistance (AMR) and can lead to various adverse side effects. C-reactive protein (CRP) point-of-care testing (POCT) in primary care has shown potential as an effective strategy to reduce inappropriate antibiotic prescribing. This study evaluates the budget impact of introducing CRP POCT in Belgian primary care over a 5-year time horizon.

Methods: A decision tree model was developed to compare the budget impact of implementing CRP POCT in primary care versus usual care (without CRP POCT) based on 712,102 acute cough patients annually. Cost and probability parameters were derived through literature review and primary data collection. The robustness of the findings was assessed using univariate and probabilistic sensitivity analyses. Additionally, alternative scenarios were explored by modifying key assumptions in the base-case scenario to evaluate the effect on the results.

Results: Over a 5-year period, implementing CRP POCT in Belgian primary care for the management of 712,102 acute cough patients annually is estimated to result in an incremental cost of approximately €12.8 million (95% credible interval: 12,699,459 to 12,924,898), primarily driven by capital expenditures for analysers and CRP test expenses. Scenario analyses showed substantial variation in budget impact depending on implementation choices, ranging from €13.6 million to €39.6 million, with the highest costs when general practitioners' (GPs') compensation was included. Reduced inappropriate antibiotic use, adverse side effects, and AMR rates would decrease costs, with these savings expected to grow over time as CRP POCT becomes more widely adopted. However, the proportion of these savings relative to total expenditure is limited.

Conclusion: The implementation of CRP POCT entails a significant financial investment. Our scenario analyses indicate a substantial increase in budget impact when GPs' compensation was included. However, a well-designed implementation strategy with an appropriate GP compensation structure may be crucial to avoid inefficiencies from over- or underuse of CRP testing and achieve the anticipated health benefits.

Objectives: The integration of environmental impact into health technology assessment (HTA) is increasingly discussed, but its practical implementation remains uncertain. This study explores multi-stakeholder perspectives on the progress, challenges, and equity implications of incorporating environmental considerations into HTA.

Methods: As part of the Horizon Europe project HI-PRIX (ID: 101095593), 13 semi-structured interviews were conducted with experts across academia, HTA agencies, regulatory bodies, and industry in seven European countries. Interview transcripts were analyzed through a thematic analysis conducted manually, using a grounded-theory approach. From the interviews, 101 quotes corresponding to 37 codes were extracted and organized in eight analytical themes.

Results: Stakeholders agreed that environmental impact is relevant but under-addressed in HTA due to fragmented data, methodological uncertainty, and lack of regulatory guidance. Many emphasized that a health system perspective is too narrow, where methods conventionally used to assess health technologies are not necessarily appropriate to also assess environmental impact, calling for a societal approach that reflects lifecycle impacts. Equity consequences linked to the inclusion of environmental impact of health technologies in HTA were specifically recognized as both reasons for its incorporation and potential barriers. At this stage, procurement was identified as a practical approach to account for the environmental dimension.

Conclusions: Integrating environmental impact into HTA will require shifting toward a societal perspective, stronger collaboration across stakeholders, and development of EU-level guidance, expert panels, and standardized methodologies. Methodologies incorporating equity into economic analyses should be explored as a potential direction for future research. Until frameworks mature, environmental impact is likely to remain informative but not yet decisive in HTA.

Objectives: Due to limited clinical data, high uncertainty, and outcome variability, assessing therapies for paediatric and rare disease populations poses specific challenges, often requiring adjustments to standard health technology assessment (HTA) frameworks. This study examines how national HTA guidelines and recommendations across Europe reflect these demands, identifying methodological adaptations and country-specific disparities.

Methods: HTA organisations across the 27 EU Member States and the UK were identified via INAHTA, EUnetHTA, and ISPOR reference listings. Publicly available documents were screened, and 29 relevant national guidelines were selected. A structured document analysis was performed using a predefined coding framework. Key terms were systematically searched, and content was categorised into thematic domains.

Results: Among the 29 guidelines, 16 included references to rare disease populations, and 12 to paediatric populations. For paediatric populations, most references focused on quality-of-life measurement and proxy assessments. Adaptations for rare diseases recognised flexible cost-effectiveness thresholds (e.g. adjusted incremental cost-effectiveness ratios [ICERs] or gross domestic product [GDP]-based modifiers), tailored economic modelling, and acceptance of alternative data sources. However, significant variability was observed across countries, with no consistent pattern.

Conclusion: While several HTA bodies have introduced adjustments for paediatric and rare disease populations, guidance often remains limited and heterogeneous. The findings indicate that HTA guidelines and recommendations require further collaboration to properly define and account for the specific needs of these patients. Methodological shortcomings are mainly due to the nature of these diseases, where the limited data available are primarily from clinical practice and often lack comparative effectiveness evidence.

The United States Inflation Reduction Act of 2022 established the Medicare Drug Price Negotiation Program, directing the Centers for Medicare and Medicaid Services (CMS) to establish "maximum fair prices" for select drugs. In arriving at maximum fair prices, CMS is required to consider several criteria, including the extent to which a selected drug addresses unmet medical need (UMN). Through a targeted literature review of 48 original research studies, we identified and categorized 40 potential elements of UMN as they relate to pharmaceuticals, as well as treatment-related barriers to addressing this UMN, from the patient, caregiver, and societal perspectives. We synthesized these elements of UMN into seven domains: (1) traditional clinical effectiveness, (2) impacts on patient, caregiver, or family quality of life, (3) economic burden on the patient, caregiver, or family, (4) economic burden on society, (5) societal perspective elements, (6) elements of treatment administration, and (7) availability of other treatments. Comparing these elements with the United States Food and Drug Administration guidance regarding UMN reveals potential key gaps in the current CMS approach to assessing UMN in the Medicare Drug Price Negotiation Program, particularly regarding domains 2 through 6, including quality of life, economic burdens, societal elements, and treatment administration.

Background: Health care spending continues to rise, and recent policies have made prices more visible.

Purpose: To assess how cost effectiveness analyses obtain price information for common cancer screenings and account for price variation.

Methods: A systematic search of PUBMED was conducted, extracting studies from 2021 to 2024 in English and evaluating health care services in the USA. Cost-effectiveness analyses were included for four common cancer screenings: prostate cancer, breast cancer, colon cancer, and lung cancer. A single investigator extracted data and assessed quality, reviewed by a second investigator.

Results: A total of 16 articles met inclusion criteria. Nearly all (94%) cited the Medicare Fee Schedule as the data source for pricing information. About half (44%) of analyses included a degree of price variation. Only three articles (19%) performed a cost-effectiveness analysis with a wide degree of price variation that accurately reflected the true degree of price variation observed in empirical data.

Limitations: The sample size of included studies was modest, and generalizability is limited beyond these four common cancer screenings.

Conclusions: Cost-effectiveness analyses in the USA need to reflect the wide price variation that exists in health care, and publicly available price transparency data should guide future work.