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Michael F Drummond,1 Elisabeth Sophia Hartgers-Gubbels,2 Michael Chambers3 1Centre for Health Economics, University of York, York, UK; 2Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany; 3Healthcare Evaluation, London, UKCorrespondence: Elisabeth Sophia Hartgers-Gubbels, Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany, Email lisa.hartgers-gubbels@boehringer-ingelheim.comAbstract: The importance of payers and HTA: How did we end up here? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Mike Drummond about Health Technology Assessment (HTA) and how value is defined. Prof. Drummond is professor of Health Economics of York University, former president of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and author of two major textbooks in the field, as well as over 700 publications, and has acted as consultant to the WHO as well as European Union with regards to value assessment. Starting from the very beginning, Prof. Drummond explains in a simple yet engaging way why demonstrating value of new interventions has become so important.Keywords: market access, healthcare reimbursement, health technology assessment, HTA, value demonstration, health economics and outcomes research, payer

Benedikte R Lensberg,1 Michael F Drummond,2 Natalya Danchenko,3 Nicolas Despiégel,4 Clément François3 1OptumInsight, Uxbridge, 2Centre for Health Economics, University of York, York, UK; 3Lundbeck SAS, Issy-les-Moulineaux, 4OptumInsight, Nanterre, France Abstract: Lost productivity is often excluded from economic evaluations, which may lead to an underestimation of the societal benefits of treatment. However, there are multiple challenges in reliably estimating and reporting productivity losses. This article explores the main challenges, ie, selecting an appropriate valuation method (ie, human capital, friction cost, or multiplier), avoiding double counting, and accounting for equity. It also discusses the use of presenteeism instruments and their application in clinical trials, with a specific focus on their relevance in individuals with mood disorders. Further research and discussion is required on the development of reliable techniques for measuring and valuing productivity changes due to presenteeism. Keywords: mood disorders, cost-benefit analysis, technology assessment, biomedical, presenteeism, absenteeism, productivity loss

Daryl S Spinner,1 Julie Birt,2 Jeffrey W Walter,1 Lee Bowman,2 Josephine Mauskopf,1 Michael F Drummond,3 Catherine Copley-Merriman11RTI Health Solutions, Research Triangle Park, NC, USA; 2Eli Lilly and Company, Indianapolis, IN, United States; 3University of York, York, UKBackground: Health-technology assessment (HTA) plays an important role in informing drug-reimbursement decision-making in many countries. HTA processes for the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia, the Common Drug Review (CDR) in Canada, and the National Institute for Health and Clinical Excellence (NICE) in England and Wales are among the most established in the world. In this study, we performed nine in-depth case studies to assess whether different clinical evidence bases may have influenced listing recommendations made by PBAC, CDR, and NICE.Methods: Nine drugs were selected for which the three agencies had provided listing recommendations for the same indication between 2007 and 2010. We reviewed the evidence considered for each listing recommendation, identified the similarities and differences among the clinical evidence bases considered, and evaluated the extent to which different clinical evidence bases could have contributed to different decisions based on HTA body comments and public assessment of the evidence.Results: HTA agencies reached the same recommendation for reimbursement (recommended for listing) for four drugs and different recommendations for five drugs. In all cases, each agency used different evidence bases in their recommendations. The agencies considered overlapping sets of clinical comparators and trials when evaluating the same drug. While PBAC and NICE considered indirect and/or mixed-treatment comparisons, CDR did not. In some cases, CDR and/or NICE excluded trials from review if the drug and/or the comparator were not administered according to the relevant marketing authorization.Conclusions: In the listing recommendations reviewed, considerable variability exists in the clinical evidence considered by PBAC, CDR, and NICE for drug-listing recommendations. Differences in evidence resulted from differences in the consideration of indirect and mixed-treatment comparison data and differences in medical practice in each jurisdiction.Keywords: health-technology assessment, reimbursement decisions, evidence

Study Objective: To evaluate costs, clinical consequences, and cost-effectiveness from a German and French health-care system perspective of sequential i.v./po moxifloxacin monotherapy compared to co-amoxiclav with or without clarithromycin (AMC +/- CLA) in patients with community-acquired pneumonia (CAP) who required parenteral treatment.Methods: Costs and consequences over 21 days were evaluated based on clinical cure rates 5 to 7 days after treatment and health resource use reported for the TARGET multinational, prospective, randomized, open-label trial. This trial compared sequential i.v./po monotherapy with moxifloxacin (400 mg qd) to i.v./po co-amoxiclav (1.2 g i.v./625 mg po tid) with or without clarithromycin (500 mg bid) for 7 to 14 days in hospitalized patients with CAP. Since no country-by-treatment interaction was found in spite of some country differences for length of hospital stays, resource data (antimicrobial treatment, hospitalization, and out-of-hospital care) from all centers were pooled and valued using German and French unit prices to estimate CAP-related cost to the German Sickness Funds and French public health-care sector, respectively.Results: Compared to AMC +/- CLA, treatment with moxifloxacin resulted in 5.3% more patients achieving clinical cure 5 to 7 days after therapy (95% confidence interval [CI], 1.2 to 11.8%), increased speed of response (1 day sooner for median time to first return to apyrexia, p = 0.008), and a reduction in hospital stay by 0.81 days (95% CI, - 0.01 to 1.63) within the 21-day time frame. Treatment with moxifloxacin resulted in savings of 266 euro and 381 euro for Germany and France respectively, primarily due to the shorter length of hospital stay. Cost-effectiveness acceptability curves show moxifloxacin has a > or = 95% chance of being cost saving from French and German health-care perspectives, and higher probability of being cost-effective at acceptability thresholds up to 2,000 euro per additional patient cured.Conclusion: i.v./po monotherapy with moxifloxacin shows clinical benefits including increased speed of response and is cost-effective compared to i.v./po AMC +/- CLA in the treatment of CAP. [ABSTRACT FROM AUTHOR]

PURPOSE: The methods currently used by English National Health Service (NHS) hospitals to manage the use of medicines were studied. METHODS: A questionnaire was mailed to directors of pharmacy at all English NHS hospitals in May 2001. RESULTS: The response rate was 57% (157/ 275). Sixty-six percent of the responding hospitals provided general acute care services, and 34% provided mental health services. About 1 responder in 5 (19%) was a specialty hospital functioning either as a freestanding institution or alongside other hospitals. The average total expenditure by the hospital trusts was pound 94 million (dollar 175 million), of which drugs accounted for pound 3.5 million (dollar 6.5 million). Many hospitals either had formularies or were constructing them (86%), and most hospitals used a process to manage the introduction of new medicines. About three fourths of the hospitals had < or = 20 pharmacist full-time equivalents. The implementation of national guidelines was variable, although some of this variation may have been due to differences in service provision. Few hospitals were actively monitoring compliance with guidelines (31%), but audits of current care were common (72%). CONCLUSION: A survey of English NHS hospitals provided information on pharmacy staffing, drug expenditures, and measures taken to ensure rational medication use. [ABSTRACT FROM AUTHOR]

Although there is no debate around the effectiveness of colorectal cancer screening in reducing disease burden, there remains a question regarding the most effective and cost-effective screening modality. Current United States guidelines present a panel of options that include the 2 most commonly used modalities, colonoscopy and stool testing with the fecal immunochemical test (FIT). Large-scale comparative effectiveness trials comparing colonoscopy and FIT for colorectal cancer outcomes are underway, but results are not yet available. This review will separately state the "best case" for FIT and colonoscopy as the screening tool of first choice. In addition, the review will examine these modalities from a health economics perspective to provide the reader further context about the relative advantages of these commonly used tests., (Published by Elsevier Inc.)

Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncertainties about clinical evidence and budget impact at the time of market entry. The use of real-world evidence (RWE), including early coverage with evidence development, has been suggested as a means to support HTA decisions for rare disease treatments. However, the collection and use of RWE poses substantial challenges. These challenges are compounded when considered in the context of treatments for rare diseases. In this paper, we describe the methodological challenges to developing and using prospective and retrospective RWE for HTA decisions, for rare diseases in particular. We focus attention on key elements of study design and analyses, including patient selection and recruitment, appropriate adjustment for confounding and other sources of bias, outcome selection, and data quality monitoring. We conclude by offering suggestions to help address some of the most vexing challenges. The role of RWE in coverage and pricing determination will grow. It is, therefore, necessary for researchers, manufacturers, HTA agencies, and payers to ensure that rigorous and appropriate scientific principles are followed when using RWE as part of decision-making., (© 2024. The Author(s).)

Objectives: To investigate the extent to which articles of economic evaluations of healthcare interventions indexed in MEDLINE incorporate research practices that promote transparency, openness, and reproducibility., Study Design and Setting: We evaluated a random sample of health economic evaluations indexed in MEDLINE during 2019. We included articles written in English reporting an incremental cost-effectiveness ratio in terms of costs per life years gained, quality-adjusted life years, and/or disability-adjusted life years. Reproducible research practices, openness, and transparency in each article were extracted in duplicate. We explored whether reproducible research practices were associated with self-report use of a guideline., Results: We included 200 studies published in 147 journals. Almost half were published as open access articles (n = 93; 47%). Most studies (n = 150; 75%) were model-based economic evaluations. In 109 (55%) studies, authors self-reported use a guideline (e.g., for study conduct or reporting). Few studies (n = 31; 16%) reported working from a protocol. In 112 (56%) studies, authors reported the data needed to recreate the incremental cost-effectiveness ratio for the base case analysis. This percentage was higher in studies using a guideline than studies not using a guideline (72/109 [66%] with guideline vs. 40/91 [44%] without guideline; risk ratio 1.50, 95% confidence interval 1.15-1.97). Only 10 (5%) studies mentioned access to raw data and analytic code for reanalyses., Conclusion: Transparency, openness, and reproducible research practices are frequently underused in health economic evaluations. This study provides baseline data to compare future progress in the field., Competing Interests: Declaration of competing interest L.C., D.H., M.F.D., and D.M. are authors of the CHEERS statement. All other authors declare that they have no competing interests to declare., (Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)

Objectives: Social preference values of health states are a fundamental input for the preparation of studies in health economics. Several countries have undertaken studies to obtain these values. Our objective was to conduct a structured and systematic literature review of articles that calculates this set of representative values at the national level in low- and middle-income countries (LMICs)., Methods: In this systematic review, we searched the Embase, MEDLINE, Ovid, SciELO, and LILACS databases, among others, for studies published up to June 2022 that estimated nationally representative health states preferences values for LMICs. We summarized the information qualitatively and assessed the risk of bias in each article using the consensus-based standards for selecting health measurement instruments checklist tool., Results: Of the 23 663 articles identified, 35 studies were eligible for inclusion. The studies were from 19 countries in Latin-American, Europe, Africa, and Asia. No studies were found for low-income countries. The most commonly applied generic instrument for measuring health-related quality of life was the 5-level version of EQ-5D and 3-level version of EQ-5D. Preference was given to face-to-face administration of these instruments. The sociodemographic variables with the most significant negative correlation versus utility were older adults, marital status (widowed or divorced), and low educational level and income., Conclusions: Worldwide, there have been few studies that have estimated, in a nationally representative manner, the social values of health states preferences in LMICs. We consider the local estimate of this set of societal values relevant for any society to improve decision making in allocating resources in health budgets., (Copyright © 2023 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Background: This is the 23rd in a series of articles describing the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach to grading the certainty of evidence and strength of recommendations for systematic reviews, health technology assessments, and clinical guideline development., Objectives: We outline how resource utilization and cost-effectiveness analyses are integrated into health-related recommendations, using the GRADE Evidence to Decision (EtD) frameworks., Study Design and Setting: Through iterative discussions and refinement, in-person, and online meetings, and through e-mail communication, we developed draft guidance to incorporate economic evidence in the formulation of health-related recommendations. We developed scenarios to operationalize the guidance. We presented a summary of the results to members of the GRADE Economic Evaluation Project Group., Results: We describe how to estimate the cost of preventing (or achieving) an event to inform assessments of cost-effectiveness of alternative treatments, when there are no published economic evaluations. Evidence profiles and Summary of Findings tables based on systematic reviews of cost-effectiveness analyses can be created to provide top-level summaries of results and quality of multiple published economic evaluations. We also describe how this information could be integrated in GRADE's EtD frameworks to inform health-related recommendations. Three scenarios representing various levels of available cost-effectiveness evidence were used to illustrate the integration process., Conclusion: This GRADE guidance provides practical information for presenting cost-effectiveness data and its integration in the development of health-related recommendations, using the EtD frameworks., Competing Interests: Declaration of competing interest The GRADE system has been developed by the GRADE Working Group. The named authors drafted and revised this article. F.X. is a member of the EuroQol Group and elected deputy chair of its Executive Committee. Seven authors of this article (I.S., L.V., F.R., M.F.D., J.L., K.H., and M.X.R.) are all convenors or members of the Campbell and Cochrane Economics Methods Group, a Cochrane Methods Group. F.X., I.S., L.V., Y.Z., M.X.R., C.C.A., P.A.C., and H.J.S. are members of the GRADE working group. The views expressed in this article are those of the authors and not necessarily those of any organization., (Copyright © 2023 Elsevier Inc. All rights reserved.)