A Framework for Using Cost-effectiveness Analysis to Support Pricing and Reimbursement Decisions for New Pharmaceuticals in a Context of Evolving Treatments, Prices, and Evidence.

Current approaches to the pricing and funding of new pharmaceuticals often focus on a one-time decision about a product for each clinical indication. This can result in multiple options being available to health systems without a clear signal about how to prioritise between them. This runs the risk that, as available treatments, evidence, and drug prices evolve, clinical and patient choices may not be aligned with the objective of allocating resources to promote population health.

Assessing the Value of New Antimicrobials: Evaluations of Cefiderocol and Ceftazidime-Avibactam to Inform Delinked Payments by the NHS in England.

Objectives: The UK has recently established subscription-payment agreements for two antimicrobials: cefiderocol and ceftazidime-avibactam. This article summarises the novel value assessments that informed this process and lessons learned for future pricing and funding decisions.

Methods: The evaluations used decision modelling to predict population incremental net health effects (INHEs), informed by systematic reviews, evidence syntheses, national surveillance data and structured expert elicitation.

Ceftazidime with avibactam for treating severe aerobic Gram-negative bacterial infections: technology evaluation to inform a novel subscription-style payment model.

Background: To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of ceftazidime-avibactam in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England.

Using economic analysis to inform health resource allocation: lessons from Malawi.

Despite making remarkable strides in improving health outcomes, Malawi faces concerns about sustaining the progress achieved due to limited fiscal space and donor dependency. The imperative for efficient health spending becomes evident, necessitating strategic allocation of resources to areas with the greatest impact on mortality and morbidity. Health benefits packages hold promise in supporting efficient resource allocation.

Cefiderocol for treating severe aerobic Gram-negative bacterial infections: technology evaluation to inform a novel subscription-style payment model.

Background: To limit the use of antimicrobials without disincentivising the development of novel antimicrobials, there is interest in establishing innovative models that fund antimicrobials based on an evaluation of their value as opposed to the volumes used. The aim of this project was to evaluate the population-level health benefit of cefiderocol in the NHS in England, for the treatment of severe aerobic Gram-negative bacterial infections when used within its licensed indications. The results were used to inform the National Institute for Health and Care Excellence guidance in support of commercial discussions regarding contract value between the manufacturer and NHS England.

Achieving dynamic efficiency in pharmaceutical innovation: Identifying the optimal share of value and payments required.

It has been argued that cost-effectiveness analysis of branded pharmaceuticals only considers static efficiency, neglects dynamic effects and undermines incentives for socially valuable innovation. We present a framework for designing pharmaceutical pricing policy to achieve dynamic efficiency. We develop a coherent framework that identifies the long-term static and dynamic benefits and costs of offering manufacturers different levels of reward.

Percutaneous Revascularization for Ischemic Left Ventricular Dysfunction: Cost-Effectiveness Analysis of the REVIVED-BCIS2 Trial.

Background: Percutaneous coronary intervention (PCI) is frequently undertaken in patients with ischemic left ventricular systolic dysfunction. The REVIVED (Revascularization for Ischemic Ventricular Dysfunction)-BCIS2 (British Cardiovascular Society-2) trial concluded that PCI did not reduce the incidence of all-cause death or heart failure hospitalization; however, patients assigned to PCI reported better initial health-related quality of life than those assigned to optimal medical therapy (OMT) alone. The aim of this study was to assess the cost-effectiveness of PCI+OMT compared with OMT alone.

Using Age-Specific Values for Pediatric HRQoL in Cost-Effectiveness Analysis: Is There a Problem to Be Solved? If So, How?

Value sets for the EQ-5D-Y-3L published to date appear to have distinctive characteristics compared with value sets for corresponding adult instruments: in many cases, the value for the worst health state is higher and there are fewer values < 0. The aim of this paper is to consider how and why values for child and adult health differ; and what the implications of that are for the use of EQ-5D-Y-3L values in economic evaluations to inform healthcare resource allocation decisions. We posit four potential explanations for the differences in values: (a) The wording of severity labels may mean the worst problems on the EQ-5D-Y-3L are descriptively less severe than those on the EQ-5D-5L; (b) Adults may genuinely consider that children are less badly affected than adults by descriptively similar health issues.

Randomised trial of stable chest pain investigation: 3-year clinical and quality of life results from CE-MARC 2.

Aims: Guidelines for suspected cardiac chest pain have used historical risk stratification tools, advocating invasive coronary angiography (ICA) first-line in those at highest risk. We aimed to determine whether different strategies to manage suspected stable angina affected medium-term cardiovascular event rates and patient-reported quality of life (QoL) measures.

Methods: CE-MARC 2, a three-arm parallel group trial, randomised patients with suspected stable cardiac chest pain and a Duke Clinical pretest likelihood of coronary artery disease between 10% and 90%.

Developing a Modeling Framework for Quantifying the Health and Cost Implications of Antibiotic Resistance for Surgical Procedures.

Unlabelled: Antimicrobial resistance (AMR) is a global public health threat. The wider implications of AMR, such as the impact of antibiotic resistance (ABR) on surgical procedures, are yet to be quantified. The objective of this study was to produce a conceptual modeling framework to provide a basis for estimating the current and potential future consequences of ABR for surgical procedures in England.

Assessing the potential of HTA to inform resource allocation decisions in low-income settings: The case of Malawi.

Health technology assessment (HTA) offers a set of analytical tools to support health systems' decisions about resource allocation. Although there is increasing interest in these tools across the world, including in some middle-income countries, they remain rarely used in low-income countries (LICs). In general, the focus of HTA is narrow, mostly limited to assessments of efficacy and cost-effectiveness.

Estimating the global demand curve for a leishmaniasis vaccine: A generalisable approach based on global burden of disease estimates.

Background: A pressing need exists to develop vaccines for neglected diseases, including leishmaniasis. However, the development of new vaccines is dependent on their value to two key players-vaccine developers and manufacturers who need to have confidence in the global demand in order to commit to research and production; and governments (or other international funders) who need to signal demand based on the potential public health benefits of the vaccine in their local context, as well as its affordability. A detailed global epidemiological analysis is rarely available before a vaccine enters a market due to lack of resources as well as insufficient global data necessary for such an analysis.

Using Economic Evaluation to Inform Responses to the Opioid Epidemic in the United States: Challenges and Suggestions for Future Research.

Several aspects of the opioid epidemic and of public health care organization in the United States (US) make the conduct of economic evaluation and the design of policies to respond to this crisis particularly challenging. This commentary offers suggestions for how economic evaluation may address and overcome four key features of the opioid epidemic: 1) its magnitude and geographical distribution, 2) its intersection with multiple epidemics, 3) its rapidly changing dynamics, 4) its multi-sectoral causes and consequences. We first offer pragmatic suggestions to address the difficulties in delivering a coordinated response given the fragmented nature of health care in the US.

Developing the EQ-5D-5L Value Set for Uganda Using the 'Lite' Protocol.

Objective: A 'lite' version of the EQ-5D-5L valuation protocol, which requires a smaller sample by collecting more data from each participant, was proposed and used to develop an EQ-5D-5L value set for Uganda.

Methods: Adult respondents from the general Ugandan population were quota sampled based on age and sex. Eligible participants were asked to complete 20 composite time trade-off tasks in the tablet-assisted personal interviews using the offline EuroQol Portable Valuation Technology software under routine quality control.

Antimicrobial Resistance: Is Health Technology Assessment Part of the Solution or Part of the Problem?

Antimicrobial resistance is a serious challenge to the success and sustainability of our healthcare systems. There has been increasing policy attention given to antimicrobial resistance in the last few years, and increased amounts of funding have been channeled into funding for research and development of antimicrobial agents. Nevertheless, manufacturers doubt whether there will be a market for new antimicrobial technologies sufficient to enable them to recoup their investment.

Estimating the shares of the value of branded pharmaceuticals accruing to manufacturers and to patients served by health systems.

Previous studies have estimated that patients served by health systems accrue 59-98% of the value generated by new pharmaceuticals. This has led to questions about whether sufficient returns accrue to manufacturers to incentivize socially optimal levels of R&D. These studies have not, however, fully reflected the health opportunity costs imposed by payments for branded pharmaceuticals.

Jointly Modelling Economics and Epidemiology to Support Public Policy Decisions for the COVID-19 Response: A Review of UK Studies.

COVID-19 in the UK has had a profound impact on population health and other socially important outcomes, including on education and the economy. Although a range of evidence has guided policy, epidemiological models have been central. It is less clear whether models to support decision making have sought to integrate COVID-19 epidemiology with a consideration of broader health, wellbeing and economic implications.

Program Evaluation of Population- and System-Level Policies: Evidence for Decision Making.

Background: Policy evaluations often focus on ex post estimation of causal effects on short-term surrogate outcomes. The value of such information is limited for decision making, as the failure to reflect policy-relevant outcomes and disregard for opportunity costs prohibits the assessment of value for money. Further, these evaluations do not always consider all relevant evidence, other courses of action, or decision uncertainty.

Methodological issues in economic evaluations of emergency transport systems in low-income and middle-income countries.

A recent systematic review identified few papers on the economic evaluation of systems for emergency transport of acutely ill or injured patients. In addition, we found no articles dealing with the methodological challenges posed by such studies in low-income or middle-income countries. We therefore carried out an analysis of issues that are of particular salience to this important topic.

Ideas About Resourcing Health Care in the United States: Can Economic Evaluation Achieve Meaningful Use?

The United States is one of the few high-income countries not to apply economic evaluation routinely to health care decision making on a national level, yet it excels at spending least efficiently on health care. In the interest of continuing to develop new solutions to curb spending on health care and reduce waste in the United States, perhaps now is an important moment to reconsider the benefits of economic evaluation and the barriers that must be overcome to have it emerge as a solution for health care institutions and the patients they serve. This article offers several distinct considerations to make economic evaluation methods (such as cost-effectiveness analysis) an effective component of value-based decision making in the United States.