PharmacoEconomics最新文献

This paper investigates the current use of real-world data (RWD) for estimating relative treatment effects in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions. This review included 64 HTA submissions, which accounted for approximately 11% of the total NICE HTA submissions between January 2016 and December 2023. The main sources of RWD considered in the submissions were disease registries and electronic health records. RWD were primarily used to provide an external control arm to enable comparisons within single-arm trials and to inform long-term treatment effects when extrapolating survival data beyond the trial follow-up. Adjustments for potential systematic differences between treatment groups have improved over time; however, approximately one-third of the submissions still relied on unadjusted treatment comparisons. We found that approximately 10% of NICE HTA submissions used RWD to directly inform treatment effects estimation. Over one-third of the submissions relied on naïve and/or unadjusted treatment comparisons, which may have introduced biases. To ensure that RWD provide sound evidence for HTA, submissions should follow published guidelines, including the NICE real-world evidence (RWE) framework.

Objective: This study aimed to assess and compare the measurement properties of EQ-5D-Y-3L utilities derived from available countries' value sets (Chinese, Japanese, Slovenian, German, Spanish, Hungarian, Netherlandish, Belgian, and Indonesian), among Chinese adolescents.

Methods: From July to September 2021, a large-scale cross-sectional survey was administered across 16 cities in Shandong, China, with the objective of assessing the health status of junior high school students aged 10-18 years. Supported by the educational authorities, quick response (QR) codes and questionnaire links were disseminated to schools. A total of 97,413 junior high school students completed the questionnaire. Agreement, convergent validity, and known-group validity were determined in the nine country-specific value sets.

Results: The Indonesian value set demonstrated the highest mean health utility score (0.970), followed by the Japanese (0.961), Chinese (0.960), Netherlandish (0.948), Hungarian (0.942), German (0.938), Belgian (0.932), Slovenian (0.926), and Spanish (0.926) value sets, respectively. The utility scores derived from Asian value sets were higher than those from Europe. Good or excellent agreements (intraclass correlation coefficients > 0.7) were found between each paired value set. In Bland-Altman plots, the 95% limits of agreement for any two value sets were 0.046-0.348. A strong relationship (Spearman's correlation coefficients > 0.99) between any two value sets was found. The EQ-5D-Y-3L utility scores discriminated equally well for the nine value sets across three known groups. The effect size and the relative efficiency statistics showed the Chinese value sets were more sensitive in general. Referring to the Chinese value set, all the relative efficiency values in each value set were similar across three known groups, ranging from 0.9 to 1.0.

Conclusions: A total of nine country-specific EQ-5D-Y-3L value sets showed an overall high level of agreement, strong correlation, and good known-group validity. However, the utility scores derived from nine EQ-5D-Y-3L value sets were different and the country-specific value sets were not interchangeable.

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.