Journal of Medical Economics最新文献

Background: In Egypt, there were 150,578 new cancer cases and 95,275 cancer deaths in 2022, indicating a substantial burden on patients and the healthcare system. The analysis aims to support decision-making related to investments in cancer prevention and new treatments, by highlighting the economic burden associated with five types of cancer.

Methods: The human capital approach was used to estimate productivity losses from premature mortality due to liver, lung, breast, bladder, and cervical cancer in Egypt in 2019 by calculating years of life lost (YLL), years of productive life lost (YPLL), and present value of future lost productivity (PVFLP). Mortality data were sourced from the World Health Organization (WHO), while life expectancy, retirement age, gross domestic product (GDP) per capita, and labor force participation rates were obtained from the World Bank. Income data, such as annual earnings and minimum wage were sourced from the Wage Indicator database. Deterministic sensitivity analysis (DSA) assessed the sensitivity of results to input variations.

Results: In 2019, Egypt had a total of 45,114 deaths, from liver, lung, breast, cervical, and bladder cancers, resulting in a productivity loss of $430,086,636. Liver cancer led to the most male deaths (17,745) and breast cancer to the most female deaths (6,754), with PVFLP of $232,663,468 and $130,745,592, respectively. The five cancers resulted in 551,336 YLL and 235,415 YPLL in Egypt. The total PVFLP was estimated at $217,224,178 for females and $212,862,458 for males, with a total PVFLP/death of $9,533. The DSA showed that the PVFLP was most sensitive to changes in the retirement age.

Conclusion: In conclusion, there is a substantial economic burden relating to premature cancer mortality in Egypt, highlighting that policies and treatment advances to decrease cancer are working, however, there is need for continuous prioritization of awareness programs, cancer screening and treatment advancements.

Aims: Empagliflozin confers cardioprotective benefits among patients with heart failure, across the range of ejection fraction (EF), regardless of type 2 diabetes status. The long-term cost-effectiveness of empagliflozin for the treatment of heart failure (HF) in the Philippines remains unclear. This study aims to determine the economic benefit of adding empagliflozin to the standard of care (SoC) vs the SoC alone for HF in the Philippines.

Methods: Using a Markov model, we predicted lifetime costs and clinical outcomes associated with treating HF in the Philippine setting. We used estimates of treatment efficacy, event probabilities, and derivations of utilities from the EMPEROR trials. Costs were derived from hospital tariffs and expert consensus. Separate analyses were performed for patients with left ventricular EF > 40%, categorized under mid-range ejection fraction or preserved ejection fraction (HFmrEF/HFpEF), and patients with left EF ≤ 40%, categorized under HF with reduced ejection fraction (HFrEF).

Results: Our model predicted an average of 0.09 quality-adjusted life year (QALY) gains among HFmrEF/HFpEF patients and HFrEF patients when empagliflozin was compared to SoC. The addition of empagliflozin in the treatment results in a discounted incremental lifetime cost of PHP 62,692 (USD 1,129.99) and PHP 17,215 (USD 308.67) for HFmrEF/HFpEF and HFrEF, respectively. The incremental cost-effectiveness ratio (ICER) of empagliflozin is PHP 198,270 (USD 3,570.72)/QALY and PHP 742,604 (USD 13,385.08)/QALY for HFrEF and HFmrEF/HFpEF, respectively.

Limitations: This study employed parameters derived from short-term clinical trial data, alongside metrics representative of Asian populations, which are not specific to the Philippine cohort.

Conclusions: Adding empagliflozin to the SoC in comparison to the SoC is associated with improved clinical outcomes and quality-of-life, at additional costs for both HFrEF and HFmrEF/HFpEF.

Aims: To compare all-cause and Alzheimer's disease (AD)-related healthcare resource utilization (HCRU) by cognitive stage.

Methods and materials: This retrospective study analyzed insurance claims data linked to electronic health records (01/01/2015-12/31/2021). Patients with ≥1 cognitive assessment (Mini-Mental State Examination or Montreal Cognitive Assessment) and ≥1 medical or pharmacy claim for an AD diagnosis or AD medications were included. Inverse probability of treatment weighting (IPTW) was used to address potential confounding. All-cause and AD-related HCRU were summarized per patient per year (PPPY) and compared between early AD and advanced AD cohorts (defined according to cognitive scores) using generalized linear regression models; adjusted incidence rate ratios (IRRs), and 95% confidence intervals (CI) were reported.

Results: A total of 193 patients were included (median age: 82 years; 63.2% female), 108 with early AD and 85 with advanced AD, with similar mean follow up. All-cause HCRU, on average, was similar between early AD and advanced AD cohorts (37.4 PPPY and 38.9 encounters PPPY, respectively). For AD-related HCRU, patients with early AD had fewer encounters PPPY, on average, than patients with advanced AD (1.26 and 3.88 encounters, respectively). Following IPTW adjustment, the advanced AD cohort had significantly higher overall AD-related HCRU (IRR: 3.64 [95% CI: 1.96-6.75], p < 0.001) and outpatient visits (IRR: 2.76 [95% CI: 1.68-4.54], p < 0.001) compared to the early AD cohort.

Limitations: The relatively small sample size of patients with linked claims and cognitive score data limited the ability to assess contribution of all encounter types to HCRU trends, as well as generalizability to the broader AD population.

Conclusions: Although all-cause HCRU was similar, patients with advanced AD incurred higher AD-related HCRU compared to patients living with early AD. Further research is needed to determine whether interventions earlier in disease progression can mitigate the AD-related healthcare burden for patients with advanced AD.

Objective/aim: In 2009, dronedarone was approved by the United States Food and Drug Administration based on results from the ATHENA trial (NCT00174785), which showed significant reduction of cardiovascular (CV) hospitalization and death in patients with atrial fibrillation (AF) randomized to dronedarone versus placebo. In 2020, a retrospective study by Goehring et al. showed CV hospitalizations and deaths were lower in clinical practice following initiation of dronedarone compared to other antiarrhythmic drugs (AADs) in patients with AF and atrial flutter. However, the economic impact associated with dronedarone use has not been fully assessed. The objective of this study was to estimate the cost associated with CV outcomes reported by Goehring et al. (2020).

Methods: National average Medicare payments in the Centers for Medicare and Medicaid Services (CMS) database (www.data.CMS.gov) were used to assign cost estimates to CV outcomes evaluated in Goehring et al. (2020) by diagnosis-related grouping. When costs were unavailable in the CMS database, a literature search was performed to identify publications reporting hospitalization costs.

Results: The weighted average cost for CV hospitalization was calculated to be $20,508. When multiplied by the event rate reported in Goehring et al. (2020), cost per person year for CV hospitalization was 14% lower with dronedarone versus other AADs ($3,679 vs $4,272, respectively). For hospitalizations due to heart failure, cost was 31% lower with dronedarone compared with other AADs ($324 vs $472, respectively).

Limitations: Costs have been calculated based on national averages reported by CMS (Medicare perspective) and are estimates. Regional differences may be present.

Conclusions: Patients with AF taking dronedarone had lower costs associated with CV hospitalization compared with patients taking other AADs.

Aims: To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines.

Materials and methods: This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine.

Results: Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18-64 with ≥1 risk factor over the standalone influenza vaccine.

Limitations: Results are subject to potential hypothetical, responder, selection, and information biases.

Conclusions: Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

Aim: To compare all-cause and mental health (MH)-related short-term and long-term disability leaves and associated costs among patients in the United States with bipolar disorder (BP), major depressive disorder (MDD), or schizophrenia spectrum disorders (SCZ) before versus after cariprazine initiation.

Methods: Merative MarketScan Commercial and Health and Productivity Management (HPM) databases (January 2016 to December 2021) were utilized to identify adults diagnosed with BP, MDD, or SCZ with ≥2 pharmacy cariprazine claims (first claim = index), ≥3 months of cariprazine use (adjunctively for MDD), and continuous commercial insurance coverage and HPM eligibility during baseline (12 months pre-index) and ≥3 months post-index. Observation continued until cariprazine discontinuation, insurance or HPM eligibility end, 1 year post-index, or HPM data availability end. All-cause and MH-related disability claims, days, and costs were evaluated. Baseline versus post-index rates of disability claims (events) and days were compared using rate ratios (RR); costs were compared using mean cost differences. Comparisons were calculated from generalized estimating equation models. Analyses were replicated separately across indications.

Results: There were 489 patients overall (BP = 238, MDD = 233, SCZ = 18; mean age = 43.3 years; 60.7% female; mean follow-up = 7.6 months). All-cause rates of disability events and days following cariprazine initiation were 29% (RR = 0.71 [95% CI = 0.57, 0.86]) and 28% (0.72 [0.53, 0.94]) lower than baseline, respectively (both p < .05). MH-related rates of disability events and days were 40% (0.60 [0.43, 0.80]) and 43% (0.57 [0.34, 0.84]) lower, respectively (both p < .01). All-cause disability costs were $2,917 lower and MH-related disability costs were $2,482 lower than baseline (40% and 51% decrease, respectively; both p < .01). Results were similar for indication-specific analyses.

Limitations: Limited generalizability to patients who are unemployed, uninsured, or have public insurance.

Conclusions: Rates of disability events, days, and mean costs were significantly lower after versus before cariprazine initiation. These results can help contextualize cariprazine's role in managing disability for these patients.

Objective: Chronic kidney disease (CKD) is the leading cause of kidney failure, end-stage kidney disease (ESKD), and cardiovascular (CV) events in patients with type 2 diabetes (T2D). The FIDELIO-DKD trial demonstrated that finerenone lowered the risk of renal and CV events in patients with CKD and T2D, regardless of cardiovascular disease history. This study evaluated the cost-effectiveness of finerenone added to background treatment (finerenone + BT) versus background treatment (BT) alone in patients with CKD and T2D from the perspective of the National Health Service in England and Wales.

Methods: A lifetime Markov model assessed the indicated usage of finerenone for the treatment of stage 3 or 4 CKD with albuminuria associated with T2D in adults, as per the relevant marketing authorization. The model structure considered kidney disease progression and CV risk, with health states encompassing patients' kidney disease stage and CV event profiles, using patient-level data from the FIDELIO-DKD trial. Model outcomes were life years, quality-adjusted life years (QALYs), per-patient costs, incremental costs, and incremental cost-effectiveness ratio (ICER). Sensitivity and scenario analysis were performed, including an analysis exploring the impact of real-world data which suggests more frequent sodium-glucose co-transporter-2 (SGLT2) inhibitor use in the United Kingdom since FIDELIO-DKD.

Results: Patients receiving finerenone experienced kidney and CV benefits, including reduced rates of nonfatal CV events and CV deaths, translating to improvements in survival and quality-adjusted life years (QALYs) of 6.11 and 5.97 per patient for finerenone + BT versus BT, respectively. Total discounted per-patient costs were £48,940 for finerenone + BT and £47,716 for BT alone, resulting in an incremental cost-effectiveness ratio of £8,808 per QALY gained for finerenone + BT versus BT.

Conclusion: Sensitivity and scenario analyses, including more frequent SGLT2 inhibitor use consistent with real-world data, indicate a robust ICER that remains within the bounds of what is typically considered cost-effective.

Aims: Nirmatrelvir/ritonavir (NMV/r) is an orally administered antiviral indicated for the outpatient treatment of adult patients with mild-to-moderate COVID-19 at high risk for disease progression to severe illness. We estimated the cost-effectiveness of NMV/r versus best supportive care for 54 patient cohorts, specified according to age, vaccination status and comorbidity burden.

Materials and methods: A previously published and validated cost-effectiveness model was utilized and adapted to the Swedish setting. The model used a short-term decision-tree (1 year) followed by a lifetime 2-state Markov model. The short-term decision-tree captured costs and outcomes associated with the primary infection. Post-acute COVID-19 syndrome was only considered in terms of quality-of-life decrements for one year. Baseline hospitalization and mortality risks were taken from a Swedish, nationwide, uniquely granular, Omicron-era, real-world study. NMV/r effectiveness were taken from an Omicron-era US real-world study. Remaining inputs were informed by previous COVID-19 studies and publicly available Swedish sources.

Results: The incremental cost-effectiveness ratios (ICERs) showed a large variation ranging from almost nine million SEK for some of the youngest cohorts to being dominant (i.e. cost-saving with higher gains in quality-of-life vs standard of care) for twelve elderly cohorts. In general, higher age in combination with non-recent (>180 days) or no vaccination led to lower ICERs. Specifically, NMV/r was cost-effective for all but one patient cohorts at least 70 years old, and for most patient cohorts 60-69 years old.

Limitations: As the COVID-19 landscape changes, symptom burden and baseline risks constantly change. Thus, the cost-effectiveness of NMV/r will change with time. However, the future risks could be related to the risks in the current study, and thus remain useful for decision makers.

Conclusions: This study shows that NMV/r is a cost-effective or even cost-saving treatment option for many patient cohorts, including most elderly and not-recently vaccinated patients with at least some comorbidity burden.

Aims: Haemophilia is a rare genetic disease that hinders blood clotting. We aimed to review model-based cost-effectiveness analyses (CEAs) of haemophilia treatments, describe the sources of clinical evidence used by these CEAs, summarize the reported cost-effectiveness of different treatment strategies, and assess the quality and risk of bias.

Methods: We conducted a systematic literature review of model-based CEAs of haemophilia treatments by searching databases, the Tufts Medical Center CEA registry, and grey literature. We summarized and qualitatively synthesized the approaches and results of the included CEAs, without a meta-analysis due the diversity of the studies.

Results: 32 eligible studies were performed in 12 countries and reported 53 pairwise comparisons. Most studies analysed patients with haemophilia A rather than haemophilia B. Comparisons of prophylactic versus on-demand treatment indicated that prophylaxis may not be cost-effective, but there was no clear consensus. Emicizumab was generally cost-effective compared with clotting factor treatments and was always dominant for patients with inhibitors. Immune tolerance induction following a Malmö protocol was found to be cost-effective compared to bypassing agents, while there was no consensus for the other protocols. Gene therapies as well as treatment with extended half-life coagulation factors were always cost-effective over their comparators. Studies were highly heterogenous regarding their time horizons, model structures, the inclusion of bleeding-related mortality and quality-of-life impacts. This heterogeneity limited the comparability of the studies. 19 of the 32 included studies received industry funding, which may have biased their results.

Limitations: It was not possible to perform a quantitative synthesis of the results due to the heterogeneity of the underlying studies.

Conclusion: Differences in results between previous CEAs may have been driven by heterogeneity in modelling approaches, clinical input data, and potential funding biases. A more consistent evidence base and modelling approach would enhance the comparability between CEAs.