PharmacoEconomics最新文献

Background and objective: In Germany, all new drugs undergo an early benefit assessment (EBA) by the decision-making body (G-BA). Due to limited access to clinical data in pediatric healthcare since 2017, evidence transfer has allowed for data from adult studies to be used in the EBA of pediatric drugs. This study examines the acceptance of evidence transfer, aiming to understand its correlation with granted added benefit.

Methods: By searching the G-BA database, relevant EBAs were identified. In addition to descriptive statistics, agreement statistics regarding binary and ordinal extent of added benefit and binary logistic regression with and without intercept were performed to investigate acceptance of evidence transfer, juxtaposing it with manufacturers' claims, and to evaluate the impact of identified factors on evidence transfer.

Results: In 14 of 36 identified EBAs, the evidence transfer was accepted by the G-BA. They referred to four therapeutic areas, received a non-quantifiable added benefit and were subject to a pediatric investigation program. Non-quantifiable added benefit implies an added value in itself which can range from minor to major added benefit and is considering the genuine uncertainty mainly induced in theese EBAs due to evidence transfer, which is not allowing a quantification of an added benefit. The binary agreement between manufacturers' claims and G-BA's appraisals was less than by chance [kappa - 0.054 (- 0.158 to 0.050)] whereas the ordinal agreement became fair [kappa 0.333 (0.261-0.406)]. Congruence of the mechanism of action, alignment of disease pattern, transferability of efficacy and safety, and same comparator were fundamental for evidence transfer. Additionally, supportive evidence, therapeutic breakthroughs, and small-scale approval enhanced the acceptance of evidence transfer. The regression models yielded similar results showing different model fit and explained variance.

Conclusions: Evidence transfer hinges upon fulfilling various minimum criteria and additional supportive evidence. Availability of study data from adult or older patients and the pediatric group under evaluation is crucial.

Background: The Prostatype score (P-score) is a prognostic biomarker that integrates a three-gene (IGFBP3, F3, and VGLL3) signature derived from prostate biopsy samples, with key clinical parameters, including prostate-specific antigen (PSA) levels, Gleason grade, and tumor stage at diagnosis. The test has demonstrated superior predictive accuracy for prostate cancer outcomes compared with traditional risk categorization systems such as D'Amico. Notably, it reclassifies a higher proportion of patients into the low-risk category, making them eligible for active surveillance. This study assessed the cost-effectiveness of the P-score in comparison with D'Amico and the Swedish National Prostate Cancer Register (NPCR) risk categorization systems.

Methods: A two-step decision analytic model was developed. The model consisted of a decision tree-informed Markov structure estimating the lifetime outcomes of 60-year-old men with diagnosed prostate cancer. Prostate cancer was classified as low-risk, intermediate-risk, or high-risk using either the P-score or D'Amico. Initial therapy was based on observed treatment patterns from the Swedish NPCR. Costs (SEK, year 2022) and quality-adjusted life years (QALYs) were estimated from a healthcare perspective and discounted at 3% per year; incremental cost-effectiveness ratio (ICER) was the primary outcome.

Results: The P-score led to cost savings and generated an additional 0.19 QALYs compared with D'Amico. The added costs of the genetic test and higher costs of active surveillance and radiotherapy were counterbalanced by savings from reduced costs of surgery, treatment-related side-effects, and metastatic disease. The gain in QALYs was primarily due to the avoidance of metastatic disease and a reduction in treatment-related side-effects.

Conclusions: The results of this study suggest that the P-score is likely to be a cost-effective alternative to D'Amico for prognostic evaluation of newly diagnosed prostate cancer in Sweden and compared with NPCR when health-related quality of life was included.

Background: Testing high-risk populations for non-visible haematuria may enable earlier detection of bladder cancer, potentially decreasing mortality. This research aimed to assess the cost-effectiveness of urine dipstick screening for bladder cancer in high-risk populations in England.

Methods:  A microsimulation model developed in R software was calibrated to national incidence data by age, sex and stage, and validated against mortality data. Individual risk factors included age, sex, smoking status and factory employment. We evaluated three one-time screening scenarios: (1) current and former smokers of different ages within the 55-70 years range, (2) a mixed-age cohort of smokers aged 55-80 years and (3) individuals aged 65-79 years from high-risk regions. Probabilistic and scenario analyses evaluated uncertainty. The incremental cost-effectiveness ratio (ICER) was calculated and compared with the standard £20,000/quality-adjusted life year (QALY) threshold using payer's perspective and 2022 year of evaluation with 3.5% discounting for both costs and effects.

Results:  Screening all current and former smokers (scenario 1) and both mixed-age cohorts (scenarios 2 and 3) was not cost-effective at the threshold of £20,000/QALY. Screening at age 58 years had a 33% probability of being cost-effective at £20,000/QALY threshold and a 64% probability at £30,000/QALY threshold. Screening current and former smoking men aged 58 and 60 years was cost-effective, with ICERs of £18,181 and £18,425 per QALY, respectively. Scenario results demonstrated the high impact of assumptions on lead time, diagnostic pathway, and screening efficacy on predictions.

Conclusions:  Screening smoking men aged 58 or 60 years for bladder cancer using urine dipstick tests may be cost-effective.

Background and objective: Approximately half of lung adenocarcinomas in East Asia harbor epidermal growth factor receptor (EGFR) mutations. EGFR testing followed by tissue-based next-generation sequencing (NGS), upfront tissue-based NGS, and complementary NGS approaches have emerged on the front line to guide personalized therapy. We study the cost effectiveness of exclusionary EGFR testing for Taiwanese patients newly diagnosed with advanced lung adenocarcinoma.

Methods: This economic evaluation was conducted from the perspective of the healthcare sector with a lifetime horizon. Simulated patients were entered into a joint model combining decision trees and partitioned survival models upon diagnosis of advanced lung adenocarcinoma. We compared exclusionary EGFR testing with upfront tissue-based NGS and complementary NGS approaches. The model inputs were derived from regional estimates (prevalence of targetable gene alterations), trials (testing accuracy, survival outcomes, and adverse events), ACT Genomics (testing costs), National Health Insurance payments, retail prices (drug costs), and hospital cohorts (utility values). All costs were made equivalent to 2023 US dollars. An annual discount rate of 3% was applied. We adopted a willingness-to-pay threshold of US$70,000 per quality-adjusted life-year. One-way deterministic and probabilistic analyses were performed.

Results: The incremental cost-effectiveness ratio of exclusionary EGFR testing versus upfront tissue-based NGS was US$15,521 per quality-adjusted life-year, whereas the incremental net monetary benefit was US$2530. The costs of osimertinib and pembrolizumab were the major determinants. The incremental net monetary benefit of exclusionary EGFR testing versus complementary NGS approach was US$2174, and its major determinants included the true-negative rate of EGFR testing and the prevalence rate of an EGFR mutation. Given the willingness-to-pay thresholds of US$35,000, US$70,000, and US$105,000 (1, 2, and 3 per capita gross domestic product) per quality-adjusted life-year, the probabilities that exclusionary EGFR testing would be cost effective were 79.1%, 95.6%, and 91.2%, respectively.

Conclusions: Our analysis suggests that exclusionary EGFR testing is a cost-effective strategy for Taiwanese patients newly diagnosed with advanced lung adenocarcinoma.

Background: Knowing the health opportunity costs of funding decisions is crucial to assess whether the health gains associated with new interventions are larger than the health losses imposed by the displacement of resources. Empirical estimates based on the effect of health spending on health outcomes have been proposed in several countries, including Spain, as a proxy to capture these opportunity costs. However, there is a need to regularly update existing health opportunity cost estimates and to explore the role of omitted variable bias in these estimations.

Objective: The aim of this paper is to provide an updated and refined estimate of the causal impact of health spending on health in Spain that can be translated into an estimate of the incremental cost per quality-adjusted life-year produced by the Spanish national health system.

Methods: We applied fixed-effect models using data for 17 Spanish regions from 2002 until 2022 to estimate the impact of public health spending on health outcomes and explored the extent of omitted variable bias. Changes in these estimates over time were assessed and alternative specifications were tested.

Results: Based on fixed-effect models with control variables, the estimated spending elasticity was 0.061, which translated into an incremental cost per quality-adjusted life-year of approximately €34,000. The bias-corrected elasticity was 0.075, with a corresponding incremental cost per quality-adjusted life-year of €27,000. We found that the estimated impact of spending on health decreases when recent years of data are added, and that the extent of omitted variable bias appears to increase, particularly when adding the COVID-19 pandemic period.

Conclusions: This study provides an updated estimation of the incremental cost per quality-adjusted life-year produced by the Spanish national health system. The estimates provided can be easily updatable as new data become accessible, and the methods applied might be transferable to other settings with similar available data.

Background: Detailed information on the efficiency of health services targeting opioid use disorder (OUD) and treatment with opioid agonist treatment (OAT) is sparse. Many countries, including Norway, are still falling short of universal health coverage (UHC) of OAT. This study aims to evaluate the incremental lifetime costs and effects of treating OUD with OAT as compared to no OAT in Norway and scaling up the treatment to a universal coverage level using equity-adjusted health economic evaluations.

Methods: We conducted cost-utility and budget impact analyses and constructed a two-state Markov model to compare the lifetime costs and outcomes among patients with OUD with and without OAT. Model inputs were derived from routine health information systems and the literature, with costs reported in 2023 Norwegian Kroner (NOK). The analyses were conducted from a Norwegian extended health-service and societal perspectives, with a lifetime time horizon. Quality-adjusted life years (QALYs) was the metric of health benefits. Outcomes were reported as incremental cost-effectiveness ratios (ICERs). The willingness-to-pay (WTP) threshold was equity-adjusted according to the future prognostic healthy life year loss method in Norway (severity of disease criterion), which is sensitive to the size of future undiscounted healthy life year loss due to the affected conditions. The WTP threshold is NOK 825,000 per QALY gained in Norwegian policy for conditions with undiscounted future QALY loss > 20. Uncertainty in the parameters and robustness of the results were assessed with one-way and probabilistic sensitivity analyses and scenario analyses.

Findings: The mean results from probabilistic sensitivity analysis estimated that OAT was associated with 3.03 additional discounted QALYs gain and incremental lifetime discounted cost of NOK 1.45 million, leading to an ICER of NOK 479,099 per QALY gained when compared with not providing OAT, with the extended health-service perspective. From a societal perspective, OAT was cost-saving, i.e. OAT produced greater health benefits while resulting in lower overall societal costs compared to no OAT. The mean undiscounted future health loss was estimated to be 21.34 QALYs for the Norwegian patient group with OUD. A total 5-year budget increase of NOK 1.208 billion was estimated if OAT was going to be scaled up from the current coverage level of 70% to UHC. Compared with the current coverage, 100% coverage of OAT was associated with an additional lifetime cost of NOK 4.332 billion but also an additional 6760 QALYs gained.

Conclusion: Our analysis suggests that OAT is cost-effective in Norway and has the potential to be cost-saving from a societal perspective. Therefore, Norwegian policy should consider scaling up treatment to extend the coverage of OAT.

Background and objective: In a context of growing clinical and financial uncertainty, pricing and payment schemes can act as possible solutions to the problems of affordability and access to health technologies. However, a comprehensive categorization of the available schemes to help decision makers tackle these challenges is lacking. This work aims at mapping existing types of pricing and payment schemes, and proposes a new approach for their classification, in order to help decision makers and other stakeholders select the best type of scheme to meet their needs.

Methods: A Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR)-compliant scoping literature review was performed between 2010 and 2023 in three databases (PubMed, Web of Science, Scopus). The search strategy was developed around two groups of keywords, "pricing/payment schemes" and "scheme innovativeness". Eligible studies were those illustrating the unique design and features of each scheme type, which were extracted by two independent reviewers, and synthesized using a narrative format, including a detailed tabular description of each type of scheme.

Results: A total of 70 unique types of pricing and payment schemes were identified. Around one third (33%) was only specified in principle, while two thirds (67%) had been implemented in practice. About half of the scheme types were proposed for drugs (34/70, 49%), and the vast majority were not designed for a specific therapeutic area (55/70, 79%). Each scheme type was categorized based on distinctive characteristics: the objectives, the outcome component, the timing/modalities of payments, and the evidence collection requirements.

Conclusions: Instead of trying to fit the retrieved schemes into a rigid taxonomy, we propose a new approach that suggests a flexible need-driven use of the available scheme types, driven primarily by the specific objective that one might have, and allows leveraging of the other key characteristics of each type of scheme.

Background: Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevated pressure in the pulmonary arteries, commonly resulting in right heart failure. PAH is associated with a high economic burden throughout the duration of the disease.

Methods: This retrospective cohort study of the Milliman Contributor Health Source Data, the Medicare 100% Research Identifiable Files, and the Merative Marketscan^® Commercial dataset between 2018 and 2020 identified adult patients with prevalent PAH based on the earliest qualifying diagnosis date or medication date ('index date') between January 1, 2019 and November 30, 2020. Outcomes were assessed using patient data from index date through the earliest of end of enrollment, end of data, or death (Medicare fee-for-service [FFS] only). All-cause and PAH-related medical and pharmacy costs per-patient per-month (PPPM) and healthcare resource utilization per 1000 patients were summarized.

Results: The study included 11,670 Medicare FFS, 1021 Medicare Advantage, 274 Medicaid, and 1174 commercially insured patients in the US. The annual national burden to payers was estimated to be US$3.1 billion. The PPPM payer costs ranged from US$6500 to US$14,742; out-of-pocket (OOP) costs ranged from US$341 to US$907 PPPM. Inpatient utilization rate ranged from 435 to 770 per 1000 patients for all-cause admissions and from 15 to 58 per 1000 patients for PAH-related admissions.

Conclusions: This study demonstrates that PAH continues to be associated with a high economic burden and healthcare resource utilization across all payer types within the US healthcare system.