The Acceptance of Overall Survival Extrapolation Methods in Solid Tumor Treatments by Health Technology Assessment Agencies in England, France, and Australia between 2017 and 2022.

BackgroundSurvival extrapolation is used to predict patients' overall survival beyond clinical trial follow-up. It can significantly affect the results of a cost-effectiveness analysis and subsequent pricing and reimbursement decisions. However, selecting an appropriate model involves subjectivity, leading to discrepancies between methods submitted by manufacturers and those accepted by health technology assessment (HTA) agencies.

Methods for Indirect Treatment Comparison: Results from a Systematic Literature Review.

Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases, not possible. Numerous ITC techniques are described in the literature.

The Acceptance of Indirect Treatment Comparison Methods in Oncology by Health Technology Assessment Agencies in England, France, Germany, Italy, and Spain.

Introduction: Randomized controlled trials (RCTs) are the gold standard when comparing treatment effectiveness, and Health Technology Assessment (HTA) agencies state a clear preference for such direct comparisons. When these are not available, an indirect treatment comparison (ITC) is an alternative option. The objective of this study was to assess the acceptance of ITC methods by HTA agencies across England, France, Germany, Italy, and Spain, using oncology cases for a homogeneous sample of HTA evaluations.

Comparative Assessment of Reimbursement Recommendations by NICE and HAS for Oncology New Medicines Indicated for the Treatment of Solid Tumors from 2015 to 2021.

Objectives: This study aimed to compare reimbursement recommendations by the British National Institute for Health and Care Excellence (NICE) and the French National Authority for Health (Haute Autorité de Santé; HAS) for oncology new medicines indicated for the treatment of solid tumors.

Methods: Public assessment reports published on NICE and HAS Web sites between January 1, 2015, and December 31, 2021, describing appraisals for public reimbursement for oncology new medicines indicated for the treatment of solid tumors were searched and systematically reviewed. Biosimilars and generic drugs were excluded from the analysis.

Cost-effectiveness of encorafenib with binimetinib in unresectable or metastatic BRAF-mutant melanoma.

Objective: The objective of this study was to determine the cost-effectiveness of encorafenib with binimetinib (EncoBini) as compared to other targeted double combination therapies, namely dabrafenib with trametinib (DabraTrame) and vemurafenib with cobimetinib (VemuCobi), for the treatment of BRAF V600-mutant unresectable or metastatic melanoma (MM) from the French payer perspective.

Methods: A partitioned survival model was developed considering a lifetime horizon. The model structure simulated the clinical pathway of patients with BRAF V600-mutant MM.

Access to innovative drugs and the National Reimbursement Drug List in China: Changing dynamics and future trends in pricing and reimbursement.

Background And Objectives: Multiple reforms aimed at improving the Chinese population's health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends.

Methods: Targeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs.

Economic evaluation of encorafenib with cetuximab in patients with BRAF V600E-mutant metastatic colorectal cancer in France: a cost-effectiveness analysis using data from the BEACON CRC randomised controlled trial.

Objective: The BEACON CRC randomised controlled trial (NCT02928224) in BRAF-mutant metastatic colorectal cancer (mCRC) patients showed improved overall survival for the combination treatment of encorafenib (BRAF inhibitor) with cetuximab (EGFR inhibitor) compared with cetuximab with chemotherapy (FOLFIRI (folinic acid, fluorouracil and irinotecan) or irinotecan). We aimed to evaluate the cost-effectiveness of encorafenib with cetuximab in adult patients with BRAF-mutant mCRC after prior systemic therapy, from the perspective of the French healthcare system.

Design: A partitioned survival analysis model was developed to assess the cost-effectiveness of encorafenib with cetuximab using data from BEACON CRC (encorafenib with cetuximab and cetuximab with FOLFIRI or irinotecan).

Comparative efficacy and safety of targeted therapies for BRAF-mutant unresectable or metastatic melanoma: Results from a systematic literature review and a network meta-analysis.

Background: The objective of this study was to estimate the relative efficacy and safety of targeted therapies for the treatment of metastatic melanoma using a network meta-analysis (NMA).

Methods: A systematic literature review (SLR) identified studies in Medline, Embase and Cochrane published until November 2020. Screening used prespecified eligibility criteria.

Potential risks in using midodrine for persistent hypotension after cardiac surgery: a comparative cohort study.

Background: Persistent hypotension is a frequent complication after cardiac surgery with cardiopulmonary bypass (CPB). Midodrine, an orally administered alpha agonist, could potentially reduce intravenous vasopressor use and accelerate ICU discharge of otherwise stable patients. The main objective of this study was to explore the clinical impacts of administering midodrine in patients with persistent hypotension after CPB.

Cost-Effectiveness of Nalmefene Added to Psychosocial Support for the Reduction of Alcohol Consumption in Alcohol-Dependent Patients With High/Very High Drinking Risk Levels: A Microsimulation Model.

Objective: A microsimulation model was adapted to evaluate the cost-effectiveness of nalmefene combined with psychosocial support (NMF + PS) versus psychosocial support alone (PS). The economic impact of alcohol reduction using nalmefene treatment was not evaluated.

Method: The model simulates patient-level alcohol consumption over a 5-year time horizon across different treatment cohorts.

Health-related quality of life in alcohol dependence: Similar cross-cultural impact beyond specific drinking habits.

This study explores sociocultural differences in alcohol-related impact on quality of life between France and United Kingdom. We included 38 alcohol-dependent patients in France and United Kingdom in 10 focus groups. We used a text-mining approach.

A Comparison of Markov and Discrete-Time Microsimulation Approaches: Simulating the Avoidance of Alcohol-Attributable Harmful Events from Reduction of Alcohol Consumption Through Treatment of Alcohol Dependence.

Background And Objective: When modelling the pathophysiology of a disease, it is important to select a modelling approach that can adequately replicate its course. The objective of this paper was to compare the outcomes obtained by the Markov and discrete-time microsimulation modelling approaches using nalmefene clinical trial data.

Methods: Markov and microsimulation modelling approaches assessing alcohol dependence treatment with psychosocial support with or without nalmefene were compared in terms of the modelled evolution of patients' alcohol consumption and the resulting occurrence of alcohol-attributable harmful events over 1 year.

Economic burden associated with alcohol dependence in a German primary care sample: a bottom-up study.

Background: A considerable economic burden has been repeatedly associated with alcohol dependence (AD) - mostly calculated using aggregate data and alcohol-attributable fractions (top-down approach). However, this approach is limited by a number of assumptions, which are hard to test. Thus, cost estimates should ideally be validated with studies using individual data to estimate the same costs (bottom-up approach).

A Trial-Based Predictive Microsimulation Assessing the Public Health Benefits of Nalmefene and Psychosocial Support for the Reduction of Alcohol Consumption in Alcohol Dependence.

Background: Alcohol dependence causes considerable harm to patients. Treatment with nalmefene, aiming to reduce consumption rather than maintain complete abstinence, has been licensed based on trials demonstrating a reduction in total alcohol consumption and heavy drinking days. Relating these trial outcomes to harmful events avoided is important to demonstrate the clinical relevance of nalmefene treatment.

The Cost Effectiveness of Nalmefene for Reduction of Alcohol Consumption in Alcohol-Dependent Patients with High or Very High Drinking-Risk Levels from a UK Societal Perspective.

Aim: To evaluate costs and health outcomes of nalmefene plus psychosocial support, compared with psychosocial intervention alone, for reducing alcohol consumption in alcohol-dependent patients, specifically focusing on societal costs related to productivity losses and crime.

Methods: A Markov model was constructed to model costs and health outcomes of the treatments over 5 years. Analyses were conducted for nalmefene's licensed population: adults with both alcohol dependence and high or very high drinking-risk levels (DRLs) who do not require immediate detoxification and who have high or very high DRLs after initial assessment.

The Cost-Effectiveness of the Integration of Nalmefene within the UK Healthcare System Treatment Pathway for Alcohol Dependence.

Aims: To assess the cost-effectiveness of integrating nalmefene within the treatment pathway for alcohol dependence recommended by the National Institute for Health and Care Excellence in the UK.

Methods: A Markov model, taking a UK NHS perspective, followed a cohort with alcohol dependence and high/very high drinking risk levels (HVHDRLs), who do not require immediate detoxification and who continue at HVHDRLs after initial assessment, for 5 years. Costs and quality-adjusted life years (QALYs) from treatment with nalmefene plus psychosocial support versus psychosocial support alone were modelled.

Modelling the consequences of a reduction in alcohol consumption among patients with alcohol dependence based on real-life observational data.

Background: Most available pharmacotherapies for alcohol-dependent patients target abstinence; however, reduced alcohol consumption may be a more realistic goal. Using randomized clinical trial (RCT) data, a previous microsimulation model evaluated the clinical relevance of reduced consumption in terms of avoided alcohol-attributable events. Using real-life observational data, the current analysis aimed to adapt the model and confirm previous findings about the clinical relevance of reduced alcohol consumption.

Risk of All-Cause Mortality in Alcohol-Dependent Individuals: A Systematic Literature Review and Meta-Analysis.

Background: Alcohol dependence (AD) carries a high mortality burden, which may be mitigated by reduced alcohol consumption. We conducted a systematic literature review and meta-analysis investigating the risk of all-cause mortality in alcohol-dependent subjects.

Methods: MEDLINE, MEDLINE In-Process, Embase and PsycINFO were searched from database conception through 26th June 2014.

Clinical relevance of nalmefene versus placebo in alcohol treatment: reduction in mortality risk.

Reduction of long-term mortality risk, an important clinical outcome for people in alcohol dependence treatment, can rarely be established in randomized controlled trials (RCTs). We calculated the reduction in all-cause mortality risk using data from short-term (6 and 12 months) double-blind RCTs comparing as-needed nalmefene treatment to placebo, and mortality risks from meta-analyses on all-cause-mortality risk by reduction of drinking in people with alcohol dependence. A reduction in drinking in the RCTs was defined by shifts in drinking risk levels established by the European Medicines Agency.

A predictive microsimulation model to estimate the clinical relevance of reducing alcohol consumption in alcohol dependence.

Background: Alcohol consumption is one of the most important factors for disease and disability in Europe. In clinical trials, nalmefene has resulted in a significant reduction in the number of heavy-drinking days (HDDs) per month and total alcohol consumption (TAC) among alcohol-dependent patients versus placebo.

Methods: A microsimulation model was developed to estimate alcohol-attributable diseases and injuries in patients with alcohol dependence and to explore the clinical relevance of reducing alcohol consumption.

The cost-effectiveness and public health benefit of nalmefene added to psychosocial support for the reduction of alcohol consumption in alcohol-dependent patients with high/very high drinking risk levels: a Markov model.

Objectives: To determine whether nalmefene combined with psychosocial support is cost-effective compared with psychosocial support alone for reducing alcohol consumption in alcohol-dependent patients with high/very high drinking risk levels (DRLs) as defined by the WHO, and to evaluate the public health benefit of reducing harmful alcohol-attributable diseases, injuries and deaths.

Design: Decision modelling using Markov chains compared costs and effects over 5 years.

Setting: The analysis was from the perspective of the National Health Service (NHS) in England and Wales.

Trial-based cost-effectiveness analysis comparing surgical and endoscopic drainage in patients with obstructive chronic pancreatitis.

Objective: Published evidence indicates that surgical drainage of the pancreatic duct was more effective than endoscopic drainage for patients with chronic pancreatitis. This analysis assessed the cost-effectiveness of surgical versus endoscopic drainage in obstructive chronic pancreatitis.

Design: This trial-based cost-utility analysis (ISRCTN04572410) was conducted from a UK National Health Service (NHS) perspective and during a 79-month time horizon.