Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: We propose a framework to assess the value of pharmaceutical innovations, with explicit clinical and methodological parameters, based on the therapeutic value and health needs.

Research design and methods: The study was based on the adaptation of health technology assessment methods documented in the literature, which was applied to a sample of oncological drugs. Difficulties and issues during the application of those tools were identified and addressed to develop a new framework with new and revised domains and clear classification criterion for each domain. Scores were assigned to each level and domain according to their relevance to generate the final score of innovativeness.

Results: The Pharmaceutical Innovation Index (PII) includes four domains, two related to clinical and social dimensions - Therapeutic Need and Added Therapeutic Value - and other two about methodological features - Study Design and Quality (risk of bias). The scores combined after assigned to each domain results Index of the Innovativeness of the medicines represents the degree of pharmaceutical innovation.

Conclusion: This work proposes a transparent methodology with well-defined criteria and script; the algorithm developed with authors' weightings and criteria may be switched to best adjust to other applications, perspective or clinical indications, while keeping the transparency and objectiveness.

Objectives: The objective of this study is to elicit health care preferences of people with diabetes and identify classes of people with different preferences.

Methods: A discrete choice experiment was conducted among people with diabetes in Germany comprising attributes of role division in daily diabetes care planning, type of lifestyle education, support for correct medication intake, consultation frequency, emotional support, and time spent on self-management. A conditional logit model and a latent class model were used to elicit preferences toward diabetes care and analyze preference heterogeneity.

Results: A total of 76 people with diabetes, recruited in two specialized diabetes care centers in Germany (mean age 51.9 years, 37.3% women, 49.1% type 2 diabetes mellitus, 50.9% type 1 diabetes mellitus), completed the discrete choice experiment. The most important attributes were consultation frequency, division in daily diabetes care planning, and correct medication intake. The latent class model detected preference heterogeneity by identifying two latent classes which differ mainly with respect to lifestyle education and medication intake.

Conclusion: While the majority of people with diabetes showed preferences in line with current health care provision in Germany, a relevant subgroup wished to strengthen lifestyle education and medication intake support with an aid or website.

Introduction: This systematic review study investigated the cost-effectiveness of stereotactic radiotherapy (SRT) and stereotactic radiosurgery (SRS) for treatment of various types of cancers.

Methods: PubMed, Scopus, and Web of Science were searched from 30 December 1990 to 1 January 2023. The entered studies were screened in accordance with the inclusion criteria. The inclusion criteria encompassed all types of economic evaluation studies that investigated SRT/SRS technologies in the treatment of various cancers.

Results: A total of 47 articles were included in the review. The findings suggest that the use of Linear accelerator technology for the treatment of lung cancer (8 out of 12 studies) and prostate cancer (4 out of 5 studies) was a cost-effective strategy. Linear accelerator was found to be cost-effective in the treatment of liver metastases and liver cancer (2 out of 5 studies). All of the included studies that used Gamma Knife technology in brain metastases reported Gamma-Knife was a cost-effective treatment. Furthermore, in the treatment of prostate and liver cancer, proton therapy was identified as a cost-effective option than other treatments.

Conclusions: This study confirms that SRT/SRS is a cost-effective procedure for the treatment of various types of cancers. Therefore, it is recommended to use SRT/SRS technology for optimal use of resources.

Introduction: Health economic evaluation (HEE) provides guidance for decision-making in the face of scarcity but ignores ecological scarcities as long as they involve external costs only. Following the imperative to account for planetary health, this study explores how this blind spot can be addressed.

Areas covered: The study is based on a critical review of relevant work, particularly in the fields of HEE and life cycle assessment (LCA). LCA can provide information on a technology's environmental impacts which can be accounted for on both the effect and cost sides of HEE. Cost-benefit analyses can incorporate environmental impacts in case vignettes used for eliciting consumers' willingness to pay. Existing LCA impact models can be used to estimate human health risks associated with environmental impacts and add them to the health benefits in cost-utility analyses. Many jurisdictions offer lists of shadow prices that can be used to incorporate environmental impacts on the cost side of HEE. Also, environmental impacts can be reported in a disaggregated manner.

Expert opinion: Accounting for planetary boundaries is likely to become a key field of methodological innovation in HEE. Decision relevance is likely to be highest for technologies with similar cost-effectiveness but different ecological impacts.

Background: Baloxavir marboxil is an oral, single-dose, cap-dependent endonuclease inhibitor that reduces the duration of influenza symptoms and rapidly stops viral shedding. We developed a susceptible, exposed, infected, recovered (SEIR) model to inform a cost-effectiveness model (CEM) of baloxavir versus oseltamivir or no antiviral treatment in the UK.

Research design and methods: The SEIR model estimated the attack rates among otherwise healthy and high-risk individuals in seasonal and pandemic settings. The CEM assumed that a proportion of infected patients would receive antiviral treatment. Results were reported at the population level (per 10,000 at risk of infection).

Results: The SEIR model estimated greater reductions in infections with baloxavir. In a seasonal setting, baloxavir provided incremental cost-effectiveness ratios (ICERs) of £1884 per quality-adjusted life-year (QALY) gained versus oseltamivir and a dominant cost-effectiveness position versus no antiviral treatment in the total population; ICERs of £2574/QALY versus oseltamivir and £128/QALY versus no antiviral treatment were seen in the high-risk population. Baloxavir was also cost-effective versus oseltamivir or no antiviral treatment and reduced population-level health system occupancy concerns during a pandemic.

Conclusion: Baloxavir treatment resulted in the fewest influenza cases and was cost-effective versus oseltamivir or no antiviral treatment from a UK National Health Service perspective.

Background: This study aims to assess the lifetime cost-effectiveness of a multi-component adherence intervention (MCAI), including a patient decision aid and motivational interviewing, compared to usual care in patients with a recent fracture attending fracture liaison services (FLS) and eligible for anti-osteoporosis medication (AOM).

Research design and methods: Data on AOM initiation and one-year persistence were collected from a quasi-experimental study conducted between 2019 and 2023 in two Dutch FLS centers. An individual level, state-transition Markov model was used to simulate lifetime costs and quality-adjusted life years (QALYs) with a societal perspective of MCAI vs usual care. One-way and probabilistic sensitivity analyses were conducted including variation in additional FLS and MCAI costs (no MCAI cost in baseline).

Results: MCAI was associated with gain in QALYs (0.0012) and reduction in costs (-€16) and is therefore dominant. At the Dutch willingness-to-pay threshold of €50,000/QALY, MCAI remained cost-effective when increasing costs of the FLS visit or the yearly maintenance cost for MCAI up to +€60. Probabilistic sensitivity analysis demonstrated MCAI to be dominant in 54% of the simulations and cost-effective in 87% with a threshold of €50,000/QALY.

Conclusions: A MCAI implemented in FLS centers may lead to cost-effective allocation of resources in FLS care, depending on extra costs.

Background: This study compares first-line pembrolizumab plus chemotherapy with chemotherapy alone for patients with HER2-negative advanced gastric cancer (GC) and gastroesophageal junction cancer (GEJC) in China.

Methods: A Markov state-transition model was developed based on the phase 3 randomized KEYNOTE-859 clinical trial data. The health state utility values and direct medical costs were derived from the KEYNOTE-859 clinical trial, the relevant literature, and local charges. The measured outcomes included quality-adjusted life-years (QALYs) and the incremental cost-effectiveness ratio (ICER). Probabilistic and one-way sensitivity analyses (OWSA) were performed to assess the uncertainty of the model.

Results: In the base analysis, the incremental effectiveness and cost of pembrolizumab plus chemotherapy versus chemotherapy alone were 0.22 QALYs and $16,627.31, respectively, resulting in an ICER of $76,936.60/QALY, which is higher than the willingness-to-pay threshold in China ($35,864.61/QALY). Subgroup analyses revealed that the ICERs of pembrolizumab plus chemotherapy versus chemotherapy alone were $72,762.68 and $34,813.70 in the populations with PD-L1 CPS of 1 or higher (CPS ≥ 1) and PD-L1 CPS ≥ 10 (CPS ≥ 10), respectively.

Conclusions: As first-line therapy for patients with locally advanced or metastatic HER2-negative GC/GEJC in China, pembrolizumab plus chemotherapy is less cost-effective than chemotherapy alone, however, in the CPS ≥ 10 subgroup is more.

Objectives: In 2013, South Korea introduced risk-sharing agreements (RSAs) as a new reimbursement mechanism to enhance access to new medicines and to manage pharmaceutical expenditures. This study evaluates RSAs in South Korea from the viewpoints of key stakeholders.

Methods: In 2022, a survey and semi-structured interviews were conducted. Study participants were recruited from academia (n = 3), domestic (n = 4) and foreign (n = 6) manufacturers, and government agencies (n = 6) using a purposive sampling method.

Results: Key stakeholders perceived the objective of RSAs to be 'access to medicines' and understood RSAs to manage uncertainty about 'expenditures.' They responded that financial- and performance-based RSAs address uncertainty about 'expenditures' and 'clinical effectiveness,' respectively. All stakeholders agreed that RSAs have increased the likelihood that new medicines will be listed and have reduced out-of-pocket expenditures for patients. However, foreign manufacturers insisted that the benefits of RSAs are marginal, while the administrative burden on manufacturers is high.

Conclusion: The gaps in perception between stakeholders could be narrowed by conducting a comprehensive evaluation. Financial- and performance-based RSAs need to be clearly distinguished and aligned to address the uncertainties of a new medicine in health systems.

Background: To evaluate the cost-effectiveness of first-line sintilimab plus chemotherapy versus chemotherapy for advanced esophageal squamous cell carcinoma (ESCC) from the perspective of the Chinese health service system.

Methods: A partitioned survival model was constructed to simulate quality-adjusted life years and incremental cost-effectiveness ratios over a patient's lifetime based on a phase III clinical trial.

Results: Sintilimab plus chemotherapy increased by 0.316 QALY and 0.285 QALY with the additional cost of $5692 and $5269, which led to the ICER of $18000/QALY and $18519/QALY gained in the overall population and the patients with CPS ≥ 10, respectively.

Conclusions: Compared with chemotherapy alone, sintilimab may be a cost-effective first-line treatment choice for locally advanced or metastatic ESCC.

Objectives: Adherence to the American Diabetes Association (ADA) Standards of Medical Care is low. This study aimed to assist pharmacists in identifying patients for diabetes control interventions using unsupervised machine learning.

Methods: This study analyzed the 2021 Medical Expenditure Panel Survey and used a k-mode cluster analysis. Patient features analyzed were adherence to a select set of preventive measures from the ADA Standards of Medical Care (HbA1c test, foot examination, blood cholesterol test, dilated eye examination, and influenza vaccination) and some patient characteristics (age, gender, health insurance, insulin use, and diabetes-related complications).

Results: The study included 1,219 patients with self-reported diabetes, and the adherence rate to the ADA standards was 33.72%. Five distinct clusters emerged: (A) moderate-complexity, privately insured male; (B) moderate-complexity, publicly insured female; (C) low-complexity, privately insured female; (D) high-complexity, publicly insured female; (E) moderate-complexity, publicly insured male. Groups B, C, and E exhibited nonadherence.

Conclusions: Pharmacists can target publicly insured elderly (Groups B and E) and privately insured middle-aged females (Group C) for interventions. For instance, pharmacists may help patients in Groups B and E locate existing resources in their insurance program and remind those in Group C of the importance of adequate diabetes care.