PharmacoEconomics

OBJECTIVE: To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions. METHODS: A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population...

OBJECTIVES: This study was designed to test hypotheses regarding the path dependence of health-outcome values in the form of linear additivity of health-state utilities and diminishing marginal utility of health outcomes. METHODS: We employed a discrete-choice experiment to quantify patient treatment preferences for major depressive disorder. In a series of choice questions, participants evaluated seven symptom-improvement sequences and out-of-pocket costs over 6-week durations...

Care as usual has failed to stem the tide of mental health challenges in children and young people. Transformed models of care and prevention are required, including targeting the social determinants of mental health. Robust economic evidence is crucial to guide investment towards prioritised interventions that are effective and cost-effective to optimise health outcomes and ensure value for money. Mental healthcare and prevention exhibit the characteristics of complex dynamic systems, yet dynamic simulation modelling has to date only rarely been used to conduct economic evaluation in this area...

BACKGROUND: Cost-utility analyses (CUAs) increasingly use models to predict long-term outcomes and translate trial data to real-world settings. Model structure uncertainty affects these predictions. This study compares pharmacometric against traditional pharmacoeconomic model evaluations for CUAs of sunitinib in gastrointestinal stromal tumors (GIST). METHODS: A two-arm trial comparing sunitinib 37.5 mg daily with no treatment was simulated using a pharmacometric-based pharmacoeconomic model framework...

OBJECTIVES: The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties. METHODS: A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital...

BACKGROUND AND OBJECTIVE: Accurately extrapolating survival beyond trial follow-up is essential in a health technology assessment where model choice often substantially impacts estimates of clinical and cost effectiveness. Evidence suggests standard parametric models often provide poor fits to long-term data from immuno-oncology trials. Palmer et al. developed an algorithm to aid the selection of more flexible survival models for these interventions. We assess the usability of the algorithm, identify areas for improvement and evaluate whether it effectively identifies models capable of accurate extrapolation...

BACKGROUND: The National Institute for Health and Care Excellence (NICE) may approve health technologies on condition of more evidence generated only in research (OiR) or only with research (OwR). NICE specifies the information needed to comply with its request, although it may not necessarily guarantee good quality and timely evidence for re-appraisal, before reaching a final decision. AIM: This study aimed to critically appraise the methods, quality and risk of bias of evidence generated in response to NICE OiR and OwR technology appraisal (TA) and highly specialised technologies (HSTs) recommendations...

OBJECTIVE: Chimeric antigen-receptor T-cell therapy (CAR-T) is characterised by early phase data at the time of registration, high upfront cost and a complex manufacturing and administration process compared with standard therapies. Our objective was to compare the performance of different models to assess the cost effectiveness of CAR-T using a state-transition model (STM), partitioned survival model (PSM) and discrete event simulation (DES). METHODS: Individual data for tisagenlecleucel for the treatment of young patients with acute lymphoblastic leukaemia (ALL) were used to populate the models...

BACKGROUND: Reporting standards of discrete choice experiments (DCEs) in health have not kept pace with the growth of this method, with multiple reviews calling for better reporting to improve transparency, assessment of validity and translation. A key missing piece has been the absence of a reporting checklist that details minimum standards of what should be reported, as exists for many other methods used in health economics. METHODS: This paper reports the development of a reporting checklist for DCEs in health, which involved a scoping review to identify potential items and a Delphi consensus study among 45 DCE experts internationally to select items and guide the wording and structure of the checklist...

BACKGROUND AND OBJECTIVE: China has the highest number of hepatitis C virus (HCV) infections in the world. However, it is unclear what levels of screening and treatment are needed to achieve the WHO 2030 hepatitis C elimination targets. We aimed to evaluate the impact of scaling up interventions on the hepatitis C epidemic and determine how and at what cost these elimination targets could be achieved for the whole population in China. METHODS: We developed a compartmental model incorporating HCV transmission, disease progression, and care cascade for the whole population in China, calibrated with data on demographics, injecting drug use, HCV prevalence, and treatments...

Survival extrapolation often plays an important role in health technology assessment (HTA), and there are a range of different approaches available. Approaches that can leverage external evidence (i.e. data or information collected outside the main data source of interest) may be helpful, given the extent of uncertainty often present when determining a suitable survival extrapolation. One of these methods is the multi-parameter evidence synthesis (MPES) approach, first proposed for use in HTA by Guyot et al...

BACKGROUND: Psoriatic arthritis (PsA) is an inflammatory disease characterised by a variety of clinical manifestations. Considering the economic burden posed by PsA and the increasing number of treatment options, economic evaluations are required to better allocate available resources. This work aims to update a previous published literature review on PsA cost-of-illness and cost-effectiveness analysis. METHODS: A search was performed of English-language literature between January 2017 and March 20, 2024 in Medline/PubMed, Embase and Cochrane library using the terms 'psoriatic arthritis', 'cost of illness' and 'cost effectiveness'...

OBJECTIVES: Published literature has levied criticism against the cost-minimisation analysis (CMA) approach to economic evaluation over the past two decades, with multiple papers declaring its 'death'. However, since introducing the requirements for economic evaluations as part of health technology (HTA) decision-making in 1992, the cost-minimisation analysis (CMA) approach has been widely used to inform recommendations about the public subsidy of medicines in Australia. This research aimed to highlight the breadth of use of CMA in Australia and assess the influence of preconditions for the approach on subsidy recommendations METHODS: Relevant information was extracted from Public Summary Documents of Pharmaceutical Benefits Advisory Committee (PBAC) meetings in Australia considering submissions for the subsidy of medicines that included a CMA and were assessed between July 2005 and December 2022...

Treatment effect waning (TEW) refers to the attenuation of treatment effects over time. Assumptions of a sustained immuno-oncologic treatment effect have been a source of contention in health technology assessment (HTA). We review how TEW has been addressed in HTA and in the wider scientific literature. We analysed company submissions to English language HTA agencies and summarised methods and assumptions used. We subsequently reviewed TEW-related work in the ISPOR Scientific Presentations Database and conducted a targeted literature review (TLR) for evidence of the maintenance of immuno-oncology (IO) treatment effects post-treatment discontinuation...

IMPORTANCE: The exceedingly high US spending per capita on prescription medications is mediated, at least in part, by the inefficiencies of existing generic pharmaceutical distribution and reimbursement systems; yet, the extent of potential savings and areas for targeted interventions for generic drug prescribers remains underexplored. OBJECTIVE: We aimed to analyze 2021 Medicare Part D spending on generic drugs in comparison with pricing of a low-cost generic drug program, the Mark Cuban Cost Plus Drug Company (MCCPDC), to gauge the extent of achievable potential savings...

BACKGROUND: People's time is a finite resource and a valuable input that ought to be considered in economic evaluations taking a broad, societal perspective. Yet, evaluations of interventions focusing on children and young people (CYP) rarely account for the opportunity cost of time in this population. As a key reason for this, health economists have pointed to uncertainty around when it is appropriate to include CYP time-related costs in an economic evaluation and highlighted the lack of clear guidance on the topic...

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The coronavirus disease 2019 (COVID-19) pandemic has increased public awareness of the influence of epidemiological and economic decision models on public policy decisions. Alongside this is an increased scrutiny on the development, analysis, reporting and utilisation of decision models for public policy making. Therefore, it is important that model developers can clearly explain and justify to all stakeholders what is included and excluded from a model developed to support decision-making, to both improve transparency and trust in decision-making...

BACKGROUND: The disability-adjusted life year (DALY), a key metric for health resource allocation, encompasses morbidity through disability weights. Widely used in tuberculosis cost-effectiveness analysis (CEAs), DALYs play a significant role in informing intervention adopt/reject decisions. This study reviews the values and consistency of disability weights applied in tuberculosis-related CEAs. METHODS: We conducted a systematic review using the Tufts CEA database, updated to July 2023 with searches in Embase, Scopus and PubMed...

OBJECTIVES: The objective of this study is to compare different information retrieval methods that can be used to identify utility inputs for health economic models. METHODS: The usual practice of using systematic review methods was compared with two alternatives (iterative searching and rapid review), using a health technology assessment (HTA) case study in ulcerative colitis (UC). We analysed whether there were differences in the utility values identified when using the alternative search methods...