Applied Health Economics and Health Policy最新文献

Objectives

Health regulators have progressively increased their attention and focus on patient-reported outcomes (PROs), driven by the diffusion of a patient-centred approach to the drug development process. This study investigates the consideration of PROs and their measures (PROMs) in the authorisation of medicines in Europe.

Methods

All medicines for human use authorised or refused by the European Medicines Agency (EMA) in the period 2017–2022 were identified, and corresponding European Public Assessment Reports (EPARs) were downloaded for review. Medicine and PROs/PROM characteristics were systematically recorded. A multivariate logistic regression was performed to identify variables associated with the use of patient-reported evidence in EPARs.

Results

Overall, 497 EPARs of authorised medicines and 19 EPARs of refused medicines were analysed; of these, 240 (48.3%) and 10 (52.6%), respectively, reported any use of PROs/PROMs (p = 0.710). For authorised medicines, the likelihood of using PROs/PROMs was negatively affected by generic (OR = 0.01, p < 0.001) and biosimilar status (OR = 0.46, p = 0.013) and positively affected by orphan status (OR = 1.41, p = 0.177). The use of PROMs (50.6% in 2017 vs 47.9% in 2022) did not show a clear pattern over the 6-year period considered (p = 0.758) and was particularly uncommon in some therapeutic areas (e.g., 15.2% in infectious diseases). A total of 816 dyads of PROs/PROMs were identified. On average each EPAR considered 1.6 (range: 0–14) instruments. Patient-reported outcomes were typically secondary (53.3%) and exploratory endpoints (18.8%); in one-third of cases (32.5%), they assessed generic quality of life. Among the PROMs, 227 (27.8%) targeted general population; EQ-5D (11.0%), SF-36/SF-12 (5.9%) and EORTC QLQ-C30 (5.6%) were the instruments most frequently used.

Conclusions

This study suggests PROs/PROMs are considered in less than half of total medicine assessments and even more rarely in some disease areas. The adoption of PROs is key in EMA strategy to 2025 and would be facilitated by consensus development on their measures and optimisation of data collection.

Objective

This systematic review aimed to summarise the outcomes of economic evaluations that evaluated sodium-glucose cotransporter-2 inhibitors (SGLT2i) in combination with standard of care compared to standard of care alone for patients with chronic heart failure.

Methods

This systematic review searched MEDLINE, CINAHL+, Econlit, Scopus, the Cochrane Library, the National Health Service Economic Evaluation Database and the Cost-Effectiveness Analysis Registry from inception to 31 December, 2022, for relevant economic evaluations, which were critically appraised using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and Bias in Economic Evaluation (ECOBIAS) criteria. The costs, quality-adjusted life-years, incremental cost-effectiveness ratios and cost-effectiveness thresholds were qualitatively analysed. Net monetary benefits at different decision thresholds were also computed. Subgroup analyses addressing the heterogeneity of economic outcomes were conducted. All costs were adjusted to 2023 international dollar (US$) values using the CCEMG-EPPI-Centre cost converter.

Results

Thirty-nine economic evaluations that evaluated dapagliflozin and empagliflozin in patients with heart failure were found: 32 for the left ventricular ejection fraction (LVEF) ≤ 40% and seven for LVEF > 40%. Sodium-glucose cotransporter-2 inhibitors were cost-effective in all but two economic evaluations for LVEF > 40%. Economic outcomes varied widely, but favoured SGLT2i use in LVEF ≤ 40% over LVEF > 40% and upper-middle income over high-income countries. At a threshold of US$30,000/quality-adjusted life-year, ~ 90% of high to upper-middle income countries would consider SGLT2i cost-effective for heart failure treatment. The generalisability of study findings to low- and low-middle income countries is limited because of insufficient evidence.

Conclusions

Using SGLT2i to treat heart failure is cost-effective, with more certainty in LVEF ≤ 40% compared to LVEF > 40%. Policymakers in jurisdictions where economic evaluations are not available could potentially use this study’s findings to make informed decisions about treatment adoption.

Systematic Review Protocol Registration

This study protocol was registered with the International Prospective Register of Systematic Reviews (PROSPERO; CRD42023388701).

Aim

To estimate the potential cost of lost labour productivity due to cancer-related premature mortality in Europe (EU-27 plus Norway, Switzerland, Iceland and United Kingdom) from 2018 to 2040.

Methods

Deaths and years of potential productive life lost due to 23 types of cancer were estimated for 2018–2040, for 31 European countries. The data were analysed by age groups, by sex and by year. Projected productivity costs were estimated by calculating gross earnings by country, gender and age group using the Human Capital Approach, adjusting for projected labour force participation and unemployment rates. Various data sources were used. Sensitivity analyses were conducted.

Results

Between 2018 and 2040, cancer is expected to cause around eight million premature deaths (58% male). The cumulative projected productivity costs in this respect are €1.3 trillion, representing an annual average of €58.7 billion, or 0.43% of the EU-27 gross domestic product. Labour productivity costs are projected to decrease by 6% from 2018 to 2040. The highest cost region is Western Europe, where Germany and France will experience the highest cumulative losses (€288 and €192 billion, respectively). The most costly cancers, in terms of total costs related to productivity losses, are of the lung and colorectum (€264.4 and €116.3 billion, respectively). In terms of average productivity cost per death, the most costly forms of cancer are Hodgkin lymphoma (€301,157) and melanoma (€260,522).

Conclusion

The novel information presented could help national policymakers anticipate possible areas for cost savings. Action should be taken on disease prevention, on reducing mortality and on delaying the age of death due to Hodgkin lymphoma, brain cancer, leukaemia and melanoma. Furthermore, the study findings enhance our understanding of macroeconomic variables and could be useful in determining a re-allocation of health expenditures.

Background and Objectives

India has one of the world’s highest proportions of out-of-pocket expenditure (OOPE) payments. The low share of public health expenditure coupled with the double burden of disease (communicable and non-communicable) has a direct financial impact on individual OOPE and an indirect impact in the form of decreasing life expectancy, reduced productivity, and hence a negative impact on economic growth. This systematic review aims to compare and assess the estimated economic cost of non-communicable diseases (NCDs) in India and ascertain the methods used to derive these estimates.

Methods

This paper reviews the past 12-year (2010–22) literature on the economic impact of health shocks due to NCDs. Three databases were searched for the literature: PubMed, Scopus, and Google Scholar. Thematic analysis has been performed to analyse the findings of the study.

Results

The OOPE was very high for NCDs. The increasing cost was high and unaffordable, pushing many people into financial distress measured by catastrophic payments and rising impoverishment.

Conclusion

The results indicate both the direct and indirect impact of NCDs, but the indirect burden of loss of employment and productivity, despite its relevance, has been less studied in the literature. A robust economic analysis will allow an evidence-based policy decision perspective to reduce the rising burden of NCDs.

Drug repurposing is the process of identifying a new use for an existing drug or active substance in an indication outside the scope of the original indication. Drug repurposing has important advantages including reduced development time and costs, and potentially large societal healthcare cost savings. However, current generic drug repurposing research faces a number of challenges in obtaining research funds. Furthermore, regardless of the success of a repurposing trial, commercial parties often lack interest in pursuing marketing authorisation for financial reasons, and academic researchers lack the knowledge, time and funding. Therefore, the new indication of a repurposed drug often does not make it ‘on label’. We propose a large increase in public funding for generic drug repurposing research, including funds for the marketing authorisation process when a trial is successful, and a reduction in the regulatory burden of the marketing authorisation process for repurposed generic drugs.

Background

Although safe and effective anti-retrovirals (ARVs) are readily available, non-adherence to ARVs is highly prevalent among people living with human immunodeficiency virus/acquired immunodeficiency syndrome (PLWHA). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. This systematic review aimed to review and appraise the decision analytical economic models developed to assess ARV adherence-improvement interventions.

Methods

The review protocol was registered on PROSPERO (CRD42022270039), and reporting followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) checklist. Relevant studies were identified through searches in six generic and specialized bibliographic databases, i.e. PubMed, Embase, NHS Economic Evaluation Database, PsycINFO, Health Economic Evaluations Database, tufts CEA registry and EconLit, from their inception to 23 October 2022. The cost-effectiveness of adherence interventions is represented by the incremental cost-effectiveness ratio (ICER). The quality of studies was assessed using the quality of the health economics studies (QHES) instrument. Data were narratively synthesized in the form of tables and texts. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis.

Results

Fifteen studies, mostly conducted in North America (8/15 studies), were included in the review. The time horizon ranged from a year to a lifetime. Ten out of 15 studies used a micro-simulation, 4/15 studies employed Markov and 1/15 employed a dynamic model. The most commonly used interventions reported include technology based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15) and others that involved multi-component interventions (5/15). In 1/15 studies, interventions gained higher quality-adjusted life years (QALYs) with cost savings. The interventions in 14/15 studies were more effective but at a higher cost, and the overall ICER was well below the acceptable threshold mentioned in each study, indicating the interventions could potentially be implemented after careful interpretation. The studies were graded as high quality (13/15) or fair quality (2/15), with some methodological inconsistencies reported.

Conclusion

Counselling and smartphone-based interventions are cost-effective, and they have the potential to reduce the chronic adherence problem significantly. The quality of decision models can be improved by addressing inconsistencies in model selection, data inputs incorporated into models and uncertainty assessment methods.

Objective

To assess the range of strategies analysed in European cost-effectiveness analyses (CEAs) of colorectal cancer (CRC) screening with respect to the screening intervals, age ranges and test cut-offs used to define positivity, to examine how this might influence what strategies are found to be optimal, and compare them with the current screening policies with a focus on the screening interval.

Methods

We searched PubMed, Web of Science and Scopus for peer-reviewed, model-based CEAs of CRC screening. We included studies on average-risk European populations using the guaiac faecal occult blood test (gFOBT) or faecal immunochemical test (FIT). We adapted Drummond’s ten-point checklist to appraise study quality.

Results

We included 39 studies that met the inclusion criteria. Biennial screening was the most frequently used interval which was analysed in 37 studies. Annual screening was assessed in 13 studies, all of which found it optimally cost-effective. Despite this, 25 of 26 European stool-based programmes use biennial screening. Many CEAs did not vary the age range, but the 14 that did generally found broader ranges optimal. Only 11 studies considered alternative FIT cut-offs, 9 of which found lower cut-offs superior. Conflicts between current policy and CEA evidence are less clear regarding age ranges and cut-offs.

Conclusions

The existing CEA evidence indicates that the widely adopted biennial frequency of stool-based testing in Europe is suboptimal. It is likely that many more lives could be saved throughout Europe if programmes could be offered with more intensive annual screening.