PharmacoEconomics最新文献

Background and objectives: Disorders of gut-brain interaction are highly prevalent and burdensome conditions for both patients and healthcare systems. Given the limited effectiveness of pharmacotherapy in treating disorders of gut-brain interaction, non-pharmacological interventions are increasingly used; however, the value for money of non-pharmacological treatments is uncertain. This is the first review to assess the economic evaluation evidence of non-pharmacological interventions for disorders of gut-brain interaction.

Methods: A scoping review was conducted in line with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews (PRISMA-ScR) guidelines. Reporting adhered to ISPOR's good practices for systematic reviews with cost and cost-effectiveness outcomes. Comprehensive searches were performed on 24 October, 2023, and an updated search was run on 18 May, 2024 in PubMed/MEDLINE, Embase, Web of Science, Scopus and the International HTA database, with two reviewers screening studies in parallel. The novel Criteria for Health Economic Quality Evaluation (CHEQUE) framework was used to assess methodological and reporting quality. Reporting quality was further assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022.

Results: Fifteen studies were included. Most studies examined treatments for irritable bowel syndrome. Cognitive behavioural therapy, dietary interventions and sacral neuromodulation were cost effective. Acupuncture and physiotherapy were not. CHEQUE assessment showed 12 studies met at least 70% of the methodological criteria, and 14 studies achieved 70% or more for reporting quality.

Conclusions: This review highlights gaps in the current evidence base, particularly in the robustness and generalisability of results due to methodological inconsistencies. Future research should incorporate longer follow-ups, comprehensive cost assessments, subgroup analyses, equity considerations and clearer justifications for modelling assumptions.

Objective: Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations.

Methods: A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation.

Results: The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations.

Conclusions: This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.

Background: Treating unresectable hepatocellular carcinoma (uHCC) is challenging. Clinical trials have shown that Single Tremelimumab Regular Interval Durvalumab (STRIDE) offers clinical benefits as a first-line treatment for uHCC, but its cost effectiveness remains unknown in the USA.

Objective: We aimed to assess the cost effectiveness of STRIDE (tremelimumab plus durvalumab) versus sorafenib and durvalumab monotherapy as the first-line treatment for uHCC in the USA.

Methods: A partitioned survival model was constructed to assess the cost effectiveness of STRIDE compared to sorafenib and durvalumab monotherapy as the first-line treatment for uHCC from the US societal perspective. The time horizon was 48 months with 1-month cycles. Seven parametric survival functions replicated survival curves from clinical trials, with the best-fitting model used to calculate survival probabilities. Costs, health utilities, and adverse events were included, with quality-adjusted life-years (QALYs) as the primary effectiveness measure. Both costs and effectiveness were discounted at 3%. In the base-case analysis, the incremental cost-effectiveness ratio was compared to a willingness-to-pay threshold of $150,000 per QALY gained. Deterministic and probabilistic sensitivity analyses were conducted to examine the uncertainty of the model.

Results: In the base-case analysis, STRIDE was cost effective compared to sorafenib, with an incremental cost-effectiveness ratio of $97,995.51 per QALY gained, based on a willingness-to-pay threshold of $150,000 per QALY gained. However, STRIDE was not cost effective compared to durvalumab monotherapy at the same threshold, with an incremental cost-effectiveness ratio of $754,408.92 per QALY gained. Deterministic sensitivity analyses were consistent with the base-case analysis. A probabilistic sensitivity analysis indicated that STRIDE was more likely to be cost effective than sorafenib and durvalumab monotherapy when the willingness-to-pay exceeded $101,000 and $713,000, respectively.

Conclusions: The STRIDE regimen appears to be cost effective compared to sorafenib but not compared to durvalumab for first-line uHCC treatment in the USA. However, durvalumab has not yet been approved for uHCC in the USA. Future research should focus on long-term data and economic evaluations of other recommended biologics.

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.

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.

Aim: This study sought to quantify the healthcare costs of multivessel disease (MVD) and determine the prevalence and incidence of recurrent major adverse cardiovascular events (MACE) in high-risk patients diagnosed with MVD following an acute myocardial infarction (MI).

Methods: This retrospective study utilized German claims data (AOK PLUS), between 01/01/2010 and 31/12/2020. Patients were included if they (1) had an inpatient diagnosis of an MI between 01/01/2012 and 31/12/2019 (index date), (2) were ≥ 18 years of age at date of MI diagnosis, and (3) had diabetes or met two of the following criteria: ≥ 65 years old, prior MI, peripheral arterial disease. MACE was defined as (1) MI, (2) stroke, or (3) death with a cardiovascular diagnosis within 30 days prior. To measure the burden of MVD, patients were identified during the index hospitalization by presence of MVD. Healthcare resource use and costs were compared after adjustment based on propensity score matching (PSM).

Results: A total of 5158 patients with evidence for MVD were included in the main analysis. 31.17% experienced a MACE within 365 days following the incident MI. After PSM adjustment, 33.22% of the MVD cohort experienced a MACE versus 36.48% of non-MVD patients. MVD patients had a higher rate of recurrent MI (14.22% vs. 9.81%). Additionally, public healthcare costs were about €4 million higher in the total MVD cohort than in the non-MVD cohort in the first year after an MI (€47,896,012.32 vs. €43,718,713.75, respectively), reflecting the MVD cohort's higher use of the public healthcare system. More MVD patients were prescribed guideline-recommended medication (61.4% vs. 46.0%).

Conclusion: This study found that presence of MVD contributed to higher rates of recurrent MI. Patients with MVD experienced higher rates of recurrent MI despite a higher proportion of patients receiving guideline-directed medication therapy compared to non-MVD patients. Conversely, there was a higher mortality rate observed in the non-MVD cohort.

Background: A key challenge in value assessment is how to summarize effectiveness, particularly the impact of interventions on patient health-related quality of life (HRQoL). One approach is to quantify the gains in HRQoL and life expectancy together as quality-adjusted life years (QALYs); however, this approach has faced various criticisms regarding its potential discriminatory aspects toward persons with disabilities, older adults, and the most vulnerable individuals in society.

Methods: Instead of QALYs, we provide an alternative approach that summarizes HRQoL gains from the perspective of its stakeholders (e.g., patients, parents, and caregivers) using an "experience" scale. On an experience scale, a positive value signifies an experience better than having no experience at all, while a negative value indicates an experience worse than having no experience. To illustrate the merits of this approach, we examine US preferences on the relief of child health problems, namely a discrete choice experiment (DCE) with kaizen tasks and alternatives described using the EQ-5D-Y-3L.

Results: Using this approach, we demonstrate the differences in perspectives between parents (N = 179), mothers (N = 99), and fathers (N = 80) of children younger than 18 years of age, as well as the feasibility of this patient-centered approach using a brief DCE survey of less than 100 respondents each (and without QALYs). Specifically, we found that mothers place a higher value on the child's feelings than fathers. The results also suggest other differences between the perspectives of mothers and fathers, but these differences were not statistically significant (p-values < .05).

Conclusions: We put forth that future value assessments may summarize gains in HRQoL on a patient experience scale (i.e., experience scale from the patient perspective) to inform decision-making.