Applied Health Economics and Health Policy最新文献

System-level risks generate volatility that can threaten the stability of public health systems and jeopardise population health. In financial terms, these risks may be systematic, arising from macroeconomic shocks, or systemic, arising from cascading failures due to interconnectedness. Interventions such as disease elimination and eradication (DEE) play a crucial role in mitigating the aggregate impact of such risks. However, conventional health economic evaluations often overlook these broader benefits realised during periods of instability, focusing instead on programme-specific risks under steady-state assumptions. Insights from financial economics can help capture this value and inform a more robust economic appraisal. This paper explores how concepts from financial economics can strengthen conventional health economic methods for evaluating programmes that reduce the aggregate impact of such system-level risks, using DEE as a primary example. It draws on asset pricing theory, macroeconomic models of rare disasters, real options analysis, and discounting practices. Key recommendations include recognising the added value of programmes that perform better during downturns due to the protection they offer against macroeconomic shocks or catastrophic events; using 'real options' thinking to manage uncertainty and preserve flexibility in long-term, high sunk cost projects; and accounting for equity considerations when setting discount rates for programmes with significant intergenerational impacts. The financial economics tools highlighted here could serve as key components of a broader analytical framework, supporting investment decisions that recognise and more accurately capture the value of investments that reduce the aggregate impact of system-level risks.

Aims: Spinal muscular atrophy (SMA) was recently included in the French national newborn screening (NBS) program. The aim of this study was to assess the public health impact and cost-utility of SMA NBS in the French setting.

Materials and methods: A cost-utility model combining a decision tree with a lifetime state transition model (six health states) was adapted from a previous model. The model simulated a cohort of 700,000 newborns annually, aligned with French birth statistics. Clinical inputs were derived from SMA trials. Cost and utility estimates reflected French healthcare system data and societal burden studies. Analyses were conducted from a societal perspective, with key outcomes including the incremental cost-utility ratio (ICUR), quality-adjusted life years (QALYs), and incremental net monetary benefit (INMB). Deterministic, probabilistic, and scenario analyses were performed.

Results: With NBS, 66.5 patients are expected to be treated presymptomatically each year. At age 15 years, mortality is reduced to 2% in the NBS arm compared with 26% without screening; 94% of patients will retain the ability to sit or walk versus 38% without NBS. NBS is a dominant strategy versus no NBS, with lower costs (- 233.9 million [M] euros) and higher QALYs (+ 1586). The INMB was 265.6M € at a 20,000 €/QALY threshold. Results were robust across all sensitivity and scenario analyses.

Limitations: Key limitations include uncertainty in long-term treatment effects, exclusion of SMA type 0 and 4 from the model, and limited data on long-term outcomes of presymptomatically treated patients.

Conclusions: NBS for SMA in France offers significant clinical and economic value, supporting its national implementation.

Background: The Italian assessment system for innovative medicines represents a distinctive European regulatory approach. Integrated with the Italian Medicines Agency (AIFA), since 2017 it has provided a formal evaluation based on three predefined criteria, Therapeutic Need (TN), Added Therapeutic Value (ATV) and Quality of Evidence (QoE), granting access to dedicated funds and simplified procedures.

Objective: This study investigated the evolution of the Italian system from 2017 to the 2025 reform, focusing on outcomes, decision criteria, timelines, and financial implications.

Methods: Publicly available AIFA data on 266 assessments were analysed and stratified by outcome, therapeutic area, orphan status and Advanced Therapy Medicinal Product (ATMP) designation. Logistic regression models were used to assess the internal consistency of appraisal criteria and to explore whether regulatory attributes modified their association with innovativeness outcomes. Time to recognition (TIR) and Decision-to-Implementation Interval (DII) were calculated for "Full Innovative" medicines. Annual spending was compared to fund availability to assess the financial impact of innovative medicines.

Results: Among the 266 evaluations, approximately 29% were classified as "Full Innovative", 29% "Conditionally Innovative" and 42% "Not Innovative". Added Therapeutic Value and QoE showed the strongest associations with positive designations, while orphan and ATMP status showed no independent association. Access timelines were comparable across therapeutic areas, with slightly longer delays for ATMPs. Spending exceeded fund limits several times but fell below budget after fund unification in 2022.

Conclusions: The Italian system has been applied consistently over time, with appraisal outcomes largely reflecting the formal decision criteria rather than regulatory status. The 2025 reform may represent a turning point, potentially changing designation patterns and their determinants. Ongoing monitoring is needed to ensure access does not compromise evidence standards or sustainability, offering lessons that may be relevant to other health systems considering similar mechanisms.

Background: China's hierarchical medical system seeks to route first-contact care for mild, nonurgent conditions to primary healthcare (PHC), yet hospital-centrism persists. Evidence is needed on how financing and visible access/quality signals jointly shape initial provider choice, and which groups respond most. This study quantifies those trade-offs and simulates policy impacts in Shanghai.

Methodology: A discrete choice experiment (12 tasks; two labeled provider alternatives plus opt-out) was administered to adult residents and long-term migrants in Shanghai (N = 441). Attributes included reimbursement rate, deductible, travel time, provider title (ref. General physician), hospital tier (ref. PHC), and online booking. A respondent-panel mixed logit with random coefficients for reimbursement, deductible, and travel time captured unobserved heterogeneity; effects were summarized as marginal rates of substitution (MRS) in reimbursement percentage points.

Results: Higher reimbursement pulled patients toward PHC. Beyond price, visible access and quality signals exerted strong influence on first-contact choice. Price responsiveness varied meaningfully across people, with those who better understand insurance reacting more strongly to reimbursement. Policy simulations raised PHC's first-contact share from 23.4% (UEBMI baseline) to 29.7% with a PHC-tilted reimbursement schedule, and to 33.3% when pairing the same schedule with assured booking and senior staffing at PHC.

Conclusions: Price gradients are necessary but insufficient; coupling PHC-tilted financing with visible access/quality signals delivers the largest shift of mild first-contact care away from tertiary hospitals.

Background: Health interventions, particularly those targeted at health promotion and disease prevention, often have a range of impacts that span beyond the healthcare sector. Making the case for investment in these interventions may require an inventory of costs and outcomes across multiple sectors beyond the health sector.

Objectives: To perform a scoping review of economic evaluations that used existing approaches for cross-sectoral evaluation of healthcare interventions and provide an understanding of how these approaches have been applied in empirical studies.

Methods: Scoping reviews, a type of evidence synthesis, follow a systematic approach to map evidence on a topic and identify main concepts, theories, sources, and knowledge gaps. We used the PRISMA extension for scoping reviews and a pearl-growing search approach. A forward citation searching in Google Scholar and Web of Science of an initial set of selected papers that recommend cross-sectoral evaluations of health interventions was performed, complemented by free-word search in Google and Google Scholar. Cross-sectoral evaluations of health interventions that consider costs and outcomes beyond healthcare were included.

Results: From the 204 identified cross-sectoral evaluation studies of health interventions, the vast majority (85%) were cost-effectiveness and cost-utility analyses taking the societal costing perspective. Other approaches included social return on investment (6%), cost-benefit analysis (4%), cost study (3%), and combined approaches (2%). Two-thirds of the studies evaluated a treatment-based intervention while the remainder evaluated preventive interventions. In addition to healthcare, studies evaluated mostly costs related to productivity and non-direct medical costs, e.g., transport costs. Outcomes were focused on clinical results and patient-reported health and well-being.

Conclusions: There is a limited number of published cross-sectoral evaluations of health interventions despite the need of public and private investors for global value assessment. Issuing guidance on performing cross-sectoral evaluations and highlighting their need by health technology assessment agencies may improve existing evidence and therefore novel forms of investment in population health interventions.