Health technology assessment (HTA) grew from a need recognized by researchers concerned about the effectiveness of health services. They were later joined by policy makers in search of valid information about the costs and benefits of investments in health care. The history of HTA shows that this need appeared in many countries at nearly the same time.

Objectives: In a practice setting where casting is considered the standard of care, the aim of this study was to assess the cost-effectiveness of wrist splints compared with routine casting in children with acceptably angulated distal radius greenstick or transverse fractures.

Methods: A cost-effectiveness analysis was conducted alongside a randomized controlled trial (RCT). One hundred children with acceptably angulated distal radius greenstick or transverse fractures received either a wrist splint or cast. Information on health care provider and patient and family resource use as well as productivity cost was collected. Resource use was costed using unit costs from local administrative data sources and expense diaries. Effectiveness was assessed at 6 weeks using the performance version of the Activities Scale for Kids (ASKp) questionnaire. Cost-effectiveness analysis related differential costs to differential ASKp scores.

Results: Mean total cost was $877.58 in the splint group and $950.35 in the cast group, with a mean difference of $−72.76 (standard error [SE] 45.88). Mean total healthcare cost was $670.66 in the splint group and $768.22 in the cast group, with a mean difference of $−97.56 (SE 9.24). Mean (SE) ASKp was 92.8 in the splint group and 91.4 in the cast group, with a mean difference of 1.439 (SE 1.585). Therefore, splint management was more effective and cheaper. After accounting for uncertainty, the probability of splint being cost-effective compared with cast was 94 percent for a willingness-to-pay threshold value of $0 for one-unit gain in ASKp score and exceeded 82 percent for all threshold values.

Conclusions: In this RCT, splint management was cost-effective compared with casting in children with acceptably angulated distal radius greenstick or transverse fractures. This study challenges the existing standard of care for children with this type of fracture and provides justification on clinical and economic grounds for a change in routine practice.

Objectives: Adapting health technology assessment (HTA) reports for different contexts could reduce the need for multiple reports on the same health technology with resultant saving of time and resources. This article describes an instrument, the adaptation toolkit, which has been developed to aid in the process of adaptation of HTA reports.

Methods: The toolkit was developed by a partnership of HTA agencies and networks from across Europe. The role of the toolkit is to guide the user through the process of selecting possible relevant material from these report(s), assessing the relevance, reliability, and transferability of the material, and adapting it for the desired context.

Results: The adaptation toolkit has been developed, it comprises a collection of resources that help the user assess whether data and information in existing HTA reports should and could be adapted for their own setting. The toolkit contains two sections: a preliminary speedy sifting section and the main toolkit. The main toolkit includes five domains: (i) technology use and development, (ii) safety, (iii) effectiveness (including efficacy), (iv) economic evaluation, and (v) organizational aspects. Legal, ethical, and social aspects are beyond the scope of the toolkit. The toolkit is designed for the adaptation of evidence synthesis rather than primary research.

Conclusions: The completed current version of the toolkit contains checklists and resources to aid in the adaptation of HTA reports. This collection of resources is available for use by all HTA agencies and can be accessed at: http://www.eunethta.net/upload/WP5/EUnetHTA_HTA_Adaptation_Toolkit_October08.pdf..

Objectives: The objective of this study was to estimate the cost-effectiveness of infliximab use in patients with rheumatoid arthritis (RA) in Swedish clinical practice, based on patient-level data from the Stockholm TNF-alpha follow-up registry (STURE).

Methods: Real-world patient-level data on infliximab use from the STURE registry were implemented in a Markov cohort model, in which health states of functional status were classified according to the Health Assessment Questionnaire Disability Index (HAQ—five categories) and twenty-eight joint count Disease Activity Score (DAS28). The transition probabilities between HAQ and DAS28 states during treatment, as well as discontinuation rates were modeled based on data from the registry for patients using infliximab as their first-line biological treatment. The transition probabilities in the comparator arm, that is, disease progression without biologic treatment, as well as mortality rates, costs, and utilities were based on published literature. The analysis had a societal cost perspective.

Results: Infliximab was associated with an incremental gain in quality-adjusted life-years of 1.02 and an incremental cost of €23,264 per patient compared with progression without biologic treatment, producing an incremental cost-effectiveness ratio (ICER) of €22,830 (SEK211,136 or US$31,230). Sensitivity analyses of input parameters and model assumptions produced ICERs in the range from €18,000 to €47,000.

Conclusions: Results from base-case and sensitivity analyses fell well below established benchmarks for cost-effectiveness in Sweden. The results, therefore, indicated that infliximab treatment for RA has provided good societal value for money in Swedish clinical practice, compared with a scenario of no biological treatment.

A decision analysis was performed to assess the risks, costs, and effects of no prophylaxis, oral penicillin, and cephalexin regimens currently being debated for dental patients at risk for late prosthetic joint infection (LPJI). The analysis suggests that there is a very small risk of LPJI (29.3 cases per 106 dental visits), which is outweighed by a greater risk of death with an oral penicillin strategy than with a “no prophylaxis” strategy (2.31:1.93). An oral cephalosporin appears to spare life and limb but does so at an extremely high cost. Over $500,000 must be spent to spare one year of life, while $480,000 needs to be spent to prevent one case of LPJI. Some individual dental patients may still be at a much greater risk for LPJI than others. However, from the evidence to date, routine predental antibiotic prophylaxis for all prosthetic joint patients is a very expensive preventive strategy and is not cost-effective. However, clinical experience suggests that antibiotic prophylaxis may be appropriate in some situations.

Though technology assessment and quality assessment have much in common, they differ significantly in emphasis. Technology assessment judges technology itself; quality assessment examines the extent to which a technology is used well in its various settings. This article explores the nature of quality assessment–including the performance of individual practitioners as they care for individual patients and the care received by communities–and examines its interrelationship with technology assessment.

Background: In the debate on improving the quality and efficiency of the United States healthcare system, comparative effectiveness research is increasingly seen as a tool for reducing costs without compromising outcomes. Furthermore, the recent American Recovery and Reinvestment Act explicitly describes a prioritization function for establishing a comparative effectiveness research agenda. However, how such a function, in terms of methods and process, would go about identifying the most important priorities warranting further research has received little attention.

Objectives: This study describes an Agency for Healthcare Research and Quality-funded pilot project to translate one current comparative effectiveness review into a prioritized list of evidence gaps and research questions reflecting the views of the healthcare decision makers involved in the pilot.

Methods: To create a prioritized research agenda, we developed an interactive nominal group process that relied on a multistakeholder workgroup scoring a list of research questions on the management of coronary artery disease.

Results: According to the group, the areas of greatest uncertainty regarding the management of coronary artery disease are the comparative effectiveness of medical therapy versus percutaneous coronary interventions versus coronary artery bypass grafting for different patient subgroups; the impact of diagnostic testing; and the most effective method of developing performance measures for providers.

Conclusions: By applying our nominal group process, we were able to create a list of research priorities for healthcare decision makers. Future research should focus on refining this process because determining research priorities is essential to the success of developing an infrastructure for comparative effectiveness research.

Abstract

The rapid spread of the current COVID-19 pandemic has affected societies worldwide, leading to excess mortality, long-lasting health consequences, strained healthcare systems, and additional strains and spillover effects on other sectors outside health (i.e., intersectoral costs and benefits). In this perspective piece, we demonstrate the broader societal impacts of COVID-19 on other sectors outside the health sector and the growing importance of capturing these in health economic analyses. These broader impacts include, for instance, the effects on the labor market and productivity, education, criminal justice, housing, consumption, and environment. The current pandemic highlights the importance of adopting a societal perspective to consider these broader impacts of public health issues and interventions and only omit these where it can be clearly justified as appropriate to do so. Furthermore, we explain how the COVID-19 pandemic exposed and exacerbated existing deep-rooted structural inequalities that contribute to the wider societal impacts of the pandemic.

Practice guidelines are rapidly becoming preferred decision-making resources in medicine, as advances in technology and pharmaceutics continue to expand. An evidence-based approach to the development of practice guidelines serves to anchor healthcare policy to scientific documentation, and in conjunction with practitioner opinion can provide a powerful and practical clinical tool. Three sources of information are essential to an evidence-based approach: a) an exhaustive literature synthesis; b) meta-analysis; and c) consensus opinion. The systematic merging of evidence from these sources offers healthcare providers a scientifically supportable document that is flexible enough to deal with clinically complex problems. Evidence-based practice guidelines, in conjunction with practice standards and practice advisories, are invaluable resources for clinical decision making. The judicious use of these documents by practitioners will serve to improve the efficiency and safety of health care well.

Clnical trial randomization schemes to control patient characteristics that can influence clinical response are not sufficient to control for their effects on operational efficiency and cost within the provider institution. This variation can result in biased economic findings when patients are allowed to select a provider themselves.

Of the four randomized controlled trials of mass mammography completed to date, the New York HIP trial used obsolete technology and was not designed to assess mammography in the absence of physical examination; the Swedish Two-County trial had serious methodological flaws; the U.K. trial was incompletely documented but showed no significant benefit; and the Malmö trial, which was perfect in design and execution, showed no benefit.

Paradoxicially, with each successive trial and an increasing sophistication of mammography, the benefit of screening was becoming not greater but less. This may be summarized by comparing the number of women who would have to be offered mammography for one breast cancer death prevented or postponed in the respective trials: 5,000 in the HIP trial, 13,000 in the Two-County trial, 18,000 in the U.K. trial, and 68,000 in the Malmä trial.

The enthusiasts in favor of mass mammography have created an atmosphere of false optimism. They have not informed the public about the very small potential benefit nor about the risks associated with screening, especially the risks of false positive diagnoses and of unnecessary surgical operations. This is an unacceptable situation, and the ethical vacuum in which population screening takes place needs to be filled with guidelines to protect women who are “targeted” for screening.

A decision analytic model often comprises a significant part of a health technology assessment. As health technology assessment in the hospital setting evolves, there is an increased need for modeling methods that account for patient care pathways and interactions between patients and their environment. For example, an evaluation of a computed tomography (CT) scanner for a new indication would need to consider the current and increased demand of the machine and how that may affect service in other areas of the hospital. This problem solving approach views “problems” through a systems perspective.

Objectives: The purpose of the study is to reassess the cost-effectiveness of hormone replacement therapy (HRT) based on new medical evidence found in the Women's Health Initiative (WHI). Within a model framework using an individual state transition model, the cost-effectiveness of 50- to 60-year-old women with menopausal symptoms is assessed based on a societal perspective in Sweden.

Methods: The model has a 50-year time horizon divided into a cycle length of 1 year. The model consists of the following disease states: coronary heart disease, stroke, venous thromboembolic events, breast cancer, colorectal cancer, hip fracture, vertebral fracture, and wrist fracture. An intervention is modeled by its impact on the disease risks during and after the cessation of therapy. The model calculates costs and quality-adjusted life years (QALYs) with and without intervention. The resulting cost per QALY gained is compared with the value of a QALY gained, which is set to SEK 600,000. The model requires data on clinical effects, risks, mortality rates, quality of life weights, and costs valid for Sweden.

Results: The cost-effectiveness ratios are estimated at approximately SEK 10,000, which is below the threshold value of cost-effectiveness. On the condition that HRT increases the quality of life weight more than 0.013 units, the therapy is cost-effective.

Conclusions: In conclusion, given the new evidence in WHI, there is still a high probability that HRT is a cost-effective strategy for women with menopausal symptoms.

Objectives: Health Technology Assessment (HTA) needs to address the challenges posed by high cost, effective technologies, expedited regulatory approaches, and the opportunities provided by collaborative real-world evaluation of technologies. The Health Technology Assessment International (HTAi) Policy Forum met to consider these issues and the implications for evidence production to inform HTA. This paper shares their discussion to stimulate further debate.

Methods: A background paper, presentations, group discussions, and stakeholder role play at the 2015 HTAi Policy Forum meeting informed this paper.

Results: HTA has an important role to play in helping improve evidence production and ensuring that the health service is ready to adopt effective technologies. It needs to move from simply informing health system decisions to also working actively to align stakeholder expectations about realistic evidence requirements. Processes to support dialogue over the health technology life cycle need to be developed that are mindful of limited resources, operate across jurisdictions and learn from past processes. Collaborations between health technology developers and health systems in different countries should be encouraged to develop evidence that will inform decision making. New analytical techniques emerging for real-world data should be harnessed to support modeling for HTA.

Conclusions: A paradigm shift (to “Health Innovation System 2.0”) is suggested where HTA adopts a more central, proactive role to support alignment within and amongst stakeholders over the whole life cycle of the technology. This could help ensure that evidence production is better aligned with patient and health system needs and so is more effective and efficient.

Objectives: For an efficient and fair allocation of medical resources, one must know which patients benefit more from medical care. The objective of this study is to assess the differential survival benefits of a general intensive care unit (ICU) by acute diagnoses and by Acute Physiological and Chronic Health Evaluation (APACHE II) scores.

Methods: The sample included all patients triaged for admission to the Hadassah-Hebrew University Medical Center ICU during a 7-month period (n = 381). The potential effect of ICU on in-hospital survival was estimated by a bivariate (admission–survival) probit model, using crowding in the unit as the identifying variable, controlling for observable patients characteristics: age, sex, acute diagnoses, and APACHE II score. Using the estimates, the differential predicted survival benefits of ICU were calculated for selected general acute diagnoses and for different APACHE II scores.

Results: Adjusting for age, sex, and general acute diagnoses, the average potential survival benefit of ICU is 17 percentage points (pts). The benefit of ICU for patients with central nervous system problems, with sepsis, or with respiratory failure are higher than average (23 pts). Adjusting for APACHE II scores as well increases the estimated average potential benefit to 21 pts. Over the range of APACHE II scores, the highest benefit (38 pts of potential benefit) is attained for patients with scores around 22.

Conclusions: Survival benefits differ across diagnoses and APACHE II scores. Facing limited resources, admission policies should distinguish between survival probabilities (and survival maximization) and survival benefits (and maximization of ICU benefits). Actual referral and admission policies to the present ICU do not maximize the potential survival benefits of ICU resources.

An economic evaluation of energy-absorbing flooring designed to prevent hip fractures revealed a payback period of 10½ years if only direct costs avoided were evaluated and just over 11 months when direct and indirect costs were included. Cost-effectiveness ratios of less than $0 per hip fracture prevented and life year saved were also estimated.

The use of economic evaluation in decision making appears to have increased over the past few years and economic evaluation is looked upon as another measure to help contain costs and improve efficiency in an evidence-based decision-making environment. Following the examples of Australia and the Canadian Province of Ontario, four European Union (EU) countries (Finland, the Netherlands, Portugal, and the United Kingdom) have recently introduced economic evaluation guidelines. In addition to the Australian and Canadian guidelines, which constitute a hurdle to reimbursement, the paradigm that seems to be evolving in the four EU countries follows a similar route. Finland and the Netherlands seem to be moving toward the notion of a fourth hurdle to reimbursement, whereas the National Institute for Clinical Excellence in England and Wales was in principle meant to influence practice, although in reality this essentially acts as a hurdle to reimbursement, requiring a different data set to that used by regulatory authorities. Whereas the Portuguese guidelines were developed to assist in preparing economic submissions to support reimbursement decisions, they are unclear about when such evidence will be required and also discuss the dissemination of economic evidence to broader audiences. The introduction of these guidelines poses a number of challenges to policy makers, the implications of which are analyzed in the paper: a) to ensure that economic evaluations are carried out scientifically without industrial or political bias; b) to define an acceptable methodology that would increase their credibility; and c) to address certain practical issues ranging from deciding how to use economic evaluations in policy making to setting up new institutions or improving the coordination and dissemination of evidence. The variation in the use of economic evaluation guidelines in the four EU countries highlights the differences in national pharmaceutical policies and is in line with policy makers' continuous attempts to contain costs. While the paper critically discusses the guidelines, it also points out that a series of methodologic issues need to be addressed if economic criteria are to be introduced in policy making with the aim to improve resource allocation. The paper concludes that economic evaluation as a discipline is beginning to impact on policy, whereas the consistent use of economic evaluation results is, in principle, being adopted by policy makers but needs to go a step further to reach practitioners.

Objectives: To develop and evaluate strategies for estimating hospitalization costs in multinational clinical trials.

Methods: Hospital cost estimates for eleven diagnoses were collected from twelve countries participating in a trial of therapies for congestive heart failure. Estimates were combined with U.S.-based diagnosis-related group weights to compute country-specific unit cost estimates for all reasons for hospitalization. Variations of hospital costing methods were developed. The unit cost method assigns a country-specific unit cost estimate to each hospitalization. The other methods adjust for length of stay using a daily cost (DC) estimate for each diagnosis, based on either the mean length of stay (DC-mean method) or the median length of stay (DC-median method) for each diagnosis in each country. Additional modifications were explored through adjustment of the distribution of daily costs incurred during a hospital stay.

Results: The mean cost for all hospitalizations was $10,242 (SD, 10,042) using the unit cost method, $10,242 (SD, 12,760) using the standard DC-mean method, and $13,967 (SD, 18,762) using the standard DC-median method. In comparisons of costs for all 5,486 hospitalizations incurred by a subset of 2,352 patients in the trial, the unit cost method provided 92% power to detect a $1,000 cost difference. The standard DC-mean method provided 76% power, and the standard DC-median method provided 44% power.

Conclusions: Hospital costing methods that adjust for differences in length of stay require a significantly larger sample to attain comparable statistical power as methods that assign unadjusted unit cost estimates to hospitalization events.

Objectives: The aim of this study was to explore stakeholders’ points of views regarding the applicability and relevance of a framework for user involvement in health technology assessment (HTA) at the local level. We tested this framework in the context of the assessment of alternative measures to restraint and seclusion among hospitalized adults and those living in long-term-care facilities.

Methods: Twenty stakeholders (health managers, user representatives, and clinicians) from seven regions of Quebec participated in a semi-structured interview. A thematic analysis of the transcribed interviews was performed.

Results: The findings highlighted the relevance and applicability of the framework to this specific HTA. According to interviewees, direct participation of users in the HTA process allows them to be part of the decision-making process. User consultation makes it possible to consider the views of a wide variety of people, such as marginalized and vulnerable groups, who do not necessarily meet the requirements for participating in HTA committees. However, some user representatives emphasized that user consultation should be integrated into a more holistic and participatory perspective. The most frequent barrier associated with user involvement in HTA was the top-down health system, which takes little account of the user's perspective.

Conclusions: The proposed framework was seen as a reference tool for making practitioners and health managers aware of the different mechanisms of user involvement in HTA and providing a structured way to classify and describe strategies. However, there is a need for more concrete instruments to guide practice and support decision making on specific strategies for user involvement in HTA at the local level.

Objectives: Immune thrombocytopenia (ITP) causes increased platelet destruction and suboptimal platelet production, increasing risk of bleeding. This analysis uses a Bayesian metaregression model to indirectly compare effectiveness of the thrombopoietin mimetics romiplostim and eltrombopag for increasing platelet counts, and contrasts the results with those of non-Bayesian approaches.

Methods: Ten databases were searched during 2010. Placebo-controlled trials of 24 weeks’ duration were included. An indirect comparison was undertaken using Bayesian metaregression, which includes all trials in a single model. This was compared with previous analyses in which data for each intervention were combined using simple pooling, logistic regression or meta-analysis, followed by indirect comparison of pooled values using the Bucher method.

Results: Two trials of romiplostim and one of eltrombopag were included. The indirect evidence suggests romiplostim significantly improves overall platelet response compared with eltrombopag. Bayesian metaregression gave an odds ratio (OR) for eltrombopag versus romiplostim of 0.11 (95 percent credible interval 0.02–0.66); p values and Bayesian posterior probabilities ranged from 0.01 to 0.05 for all analyses. There was no significant difference in durable platelet response in any of the analyses, although the direction of effect favored romiplostim (OR = 0.15; 95 percent credible interval, 0.01–1.88); p values and Bayesian posterior probabilities ranged from 0.08 to 0.40 across analyses. Results were relatively consistent between analyses.

Conclusions: Bayesian metaregression generated similar results to other indirect comparison methods, and may be considered the most robust as it incorporates all data in a single model and accounts appropriately for parameter uncertainty.