Fostering innovation for societal gain has been a core attribute of technology assessment (TA) since its inception. Although many observers were concerned that TA would be a means by which government would impede the development and use of technology, this was not the intent of policy makers or the agencies that conducted the original TAs.

Guidelines for reporting economic evaluations in health care are very welcome. A task force of the International Society for Pharmacoeconomics and Outcomes Research has organized a two-round, modified Delphi Panel process to compile a set of recommendations for reporting Consolidated Health Economic Evaluation Reporting Standards. The result is a 24-item checklist with the friendly acronym CHEERS (1), published in this issue of the International Journal of Technology Assessment in Health Care.

Objectives: The aim of this study was to assess whether a multicomponent cognitive behavioral group intervention is preferable to usual care in terms of (healthcare) costs and effects on fear of falling and activity avoidance.

Methods: This economic evaluation was embedded in a randomized controlled trial among 540 community-living adults in the Netherlands, aged 70 years and older who reported fear of falling and fear-induced activity avoidance. The participants allocated to the intervention group received a multicomponent cognitive behavioral group intervention consisting of eight weekly sessions and a booster session. The sessions were aimed at instilling realistic views of falls, reducing fall risk, and increasing activity and safe behavior. Participants in the control group received usual care. Cost outcome measures were healthcare costs, and patient and family costs. Clinical outcomes were fear of falling and activity avoidance. All outcomes were assessed at baseline and at 2, 8, and 14 months by means of registration forms, self-administered questionnaires, and interviews by telephone.

Results: Participants were randomly allocated to intervention (n = 280) and control groups (n = 260). Costs for the intervention program were on average €276 per person. Total costs per person were comparable (€4,925 in intervention group and €4,828 in control group). Furthermore, favorable effects of the program were observed for fear of falling and activity avoidance.

Conclusions: This study showed that the intervention program is preferable to usual care in terms of costs and effects. The program had comparable costs and significantly reduced fear of falling and associated activity avoidance among older community-living persons.

Objectives: The objective of this systematic review was to evaluate the cost-effectiveness of interventions aiming to increase the adherence to highly active antiretroviral therapy (HAART) in HIV-infected patients in developed countries (WHO stratum A).

Methods: A systematic search for comparative health economic studies was conducted in the following databases: EMBASE, MEDLINE, NHS Economic Evaluation Database, CINAHL, HEED, and EconLit. The identified publications were selected by two reviewers independently according to predefined inclusion and exclusion criteria. Furthermore, these were evaluated according to a standardized checklist and finally extracted, analyzed, and summarized.

Results: After reviewing the abstracts and full texts four relevant studies were identified. Different educational programs were compared as well as the Directly Observed Therapy (DOT). A critical aspect to be considered in particular was the poor transparency of the cost data. In three cost-utility analyses the costs per quality-adjusted life-year (QALY) in the baseline scenario were each under USD 15,000. The sensitivity analyses with a presumed maximum threshold of USD 50,000/QALY showed a predominantly cost-effective result. In one study that examined DOT the costs add up to over USD 150,000/QALY.

Conclusions: It seems that adherence interventions for HAART in HIV-infected patients can be cost-effective. Nevertheless, the quality of the included studies is deficient and only a few of the possible adherence interventions are taken into consideration. A final assessment of the cost-effectiveness of adherence interventions in general is, therefore, not possible.

Economic evaluations of health interventions pose a particular challenge for reporting. There is also a need to consolidate and update existing guidelines and promote their use in a user friendly manner. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement is an attempt to consolidate and update previous health economic evaluation guidelines efforts into one current, useful reporting guidance. The primary audiences for the CHEERS statement are researchers reporting economic evaluations and the editors and peer reviewers assessing them for publication.

The need for new reporting guidance was identified by a survey of medical editors. A list of possible items based on a systematic review was created. A two round, modified Delphi panel consisting of representatives from academia, clinical practice, industry, government, and the editorial community was conducted. Out of 44 candidate items, 24 items and accompanying recommendations were developed. The recommendations are contained in a user friendly, 24 item checklist. A copy of the statement, accompanying checklist, and this report can be found on the ISPOR Health Economic Evaluations Publication Guidelines Task Force website (www.ispor.org/TaskForces/EconomicPubGuidelines.asp).

We hope CHEERS will lead to better reporting, and ultimately, better health decisions. To facilitate dissemination and uptake, the CHEERS statement is being co-published across 10 health economics and medical journals. We encourage other journals and groups, to endorse CHEERS. The author team plans to review the checklist for an update in five years.

Background: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value.

Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers.

Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward “progressive health system decision making,” an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.

Objectives: A recent randomized, controlled trial in chronic heart failure patients showed that NT-proBNP-guided, intensive patient management (BMC) on top of multidisciplinary care reduced all-cause mortality and heart failure hospitalizations compared with multidisciplinary care (MC) or usual care (UC). We now performed a cost-utility analysis of these interventions from a payer's perspective.

Methods: Costs related to hospitalizations, ambulatory physician and nurse visits, and NT-proBNP testing for the three management strategies were acquired for both Austria (€) and Canada ($) and combined with the survival and quality of life data from the clinical trial for cost-effectiveness analysis. Data on long-term survival, costs, and quality-adjusted life-years (QALY) were extrapolated for a 20-year time horizon using a Markov model, which simulated the progression of disease through beta-blocker use, hospitalizations, and mortality.

Results: BMC was the most cost-effective strategy as it was dominant (cost-saving with improved health outcome) over both MC and UC based on both Austrian and Canadian costs. Incremental cost-effectiveness ratios for MC relative to UC were €3,746 and $5,554 per QALY gained for Austrian and Canadian costs, respectively. The probabilities for BMC being the most cost-effective strategy were 92 percent at a threshold value of Austrian €40,000 and 93 percent at a threshold value of Canadian $50,000.

Conclusions: NT-proBNP-guided, intensive HF patient management in addition to multidisciplinary care not only reduces death and hospitalization but also proves to be cost-effective.

Objectives: The aim of this study was to propose a statistical model that takes into account clinical data on earlier versions when evaluating the latest version of an implantable medical device (IMD).

Methods: We compared the performances of a Bayesian three-level hierarchical meta-analysis model with those of a Bayesian random-effects model through a simulation study. Posterior mean estimates of the success rate for each IMD version were computed as well as the probability that the latest version improved in effectiveness. Models were compared using the Deviance Information Criterion (DIC), the estimated bias and the standard deviation of the mean success rates. Sensitivity analyses to the choice of the priors were performed. These methods were applied to the evaluation of an intracranial stent used to treat wide-necked aneurysms.

Results: When IMD versions did not differ in effectiveness, the best-fitting model was the random-effects model. By contrast, when there was a version effect, the hierarchical model was selected in more than 95 percent of the cases. It provided precise estimations of success rates of each IMD version and allowed detecting an improvement in effectiveness of the latest version, with a low influence of the choice of the priors. No evidence of benefit from the latest version of the intracranial stent was found.

Conclusions: In the setting of IMD assessment, comparison of DIC between the two proposed models appeared useful for detecting version effects. In that case, Bayesian hierarchical meta-analysis model may help the decision maker by providing useful information on the latest version of IMD compared with the previous versions.

Background: Two general alternative approaches, cost-effectiveness analysis and the therapeutic added value approach, link the pricing and approval of drugs to value. Value as assessed by payers is a function of: benefit less cost, willingness to pay for benefit, and how they handle uncertainty.

Methods: This study uses international examples to explore the elements of value that can be included in the assessment of health technologies, approaches to scoring the elements of value and how they can be combined to make a decision.

Results: A range of value elements, measures, and approaches to aggregation are identified across different HTA systems. We show that seemingly arbitrary differences in measurement and aggregation can lead to significantly different outcomes, and argue that the choice of values, measures, and decision-making processes should be informed by the societal values that underpin a health system.

Conclusions: We identify three areas for further research to improve both health system and industry R&D decision making: (i) whether more consistency could be achieved across health systems on the elements of value that matter; (ii) the relative merits of discrete versus continuous measures of value; and (iii) how structured decision making (to aggregate the elements of value) could or should become.

Aortic stenosis (AS) is caused by age-related calcific degeneration of the aortic valve (1). Initially, cases are asymptomatic but, from the point that symptoms first develop, there is rapid progression and if left untreated survival estimates are low (2–3 years) (1). Therefore, managing AS effectively and efficiently is a priority for health systems with increasing healthcare costs and longer life expectancy.

Objectives: Due to a high risk of thromboembolism in patients undergoing major orthopedic surgery, it has become standard practice to give thromboprophylactic treatment. We assessed the relative efficacy and cost-effectiveness of two new oral anticoagulants, rivaroxaban and dabigatran, relative to subcutaneous enoxaparin for the prevention of thromboembolism after total hip replacement (THR) and total knee replacement surgery (TKR).

Methods: We conducted a systematic review of the literature to assess efficacy and safety, and evaluated quality of documentation using GRADE. Cost-effectiveness was assessed by developing a decision model. The model combined two modules; a decision tree for the short-term prophylaxis and a Markov model for the long-term complications and survival gain.

Results: For rivaroxaban compared with enoxaparin, we found statistically significant decreases in deep vein thrombosis, but also a trend toward increased risk of major bleeding. For mortality and pulmonary embolism there were no statistically significant differences between the treatments. We did not find statistically significant differences between dabigatran and enoxaparin for our efficacy and safety outcomes. Assuming a willingness to pay of EUR62,500 per QALY, rivaroxaban following THR had a probability of 38 percent, and enoxaparin following TKR had a probability of 34 percent of being cost-effective. Clinical efficacy had the greatest impact on decision uncertainty.

Conclusions: Dabigatran and rivaroxaban are comparable with enoxaparin following THR and TKR regarding the efficacy and safety outcomes. However, there is great uncertainty regarding which strategy is the most cost-effective. More research on clinical efficacy of rivaroxaban and dabigatran is likely to change our results.

Background: In recent years, there has been growing interest in evaluating the health and economic impact of medical devices. Payers increasingly rely on cost-effectiveness analyses in making their coverage decisions, and are adopting value-based purchasing initiatives. These analytic approaches, however, have been shaped heavily by their use in the pharmaceutical realm, and are ill-adapted to the medical device context.

Methods: This study focuses on the development and evaluation of left ventricular assist devices (LVADs) to highlight the unique challenges involved in the design and conduct of device trials compared with pharmaceuticals.

Results: Devices are moving targets characterized by a much higher degree of post-introduction innovation and “learning by using” than pharmaceuticals. The cost effectiveness ratio of left ventricular assist devices for destination therapy, for example, decreased from around $600,000 per life year saved based on results from the pivotal trial to around $100,000 within a relatively short time period.

Conclusions: These dynamics pose fundamental challenges to the evaluation enterprise as well as the policy-making world, which this paper addresses.

Objectives: The aim of this study was to develop a decision support tool to assess the potential benefits and costs of new healthcare interventions.

Methods: The Canadian Partnership Against Cancer (CPAC) commissioned the development of a Cancer Risk Management Model (CRMM)—a computer microsimulation model that simulates individual lives one at a time, from birth to death, taking account of Canadian demographic and labor force characteristics, risk factor exposures, and health histories. Information from all the simulated lives is combined to produce aggregate measures of health outcomes for the population or for particular subpopulations.

Results: The CRMM can project the population health and economic impacts of cancer control programs in Canada and the impacts of major risk factors, cancer prevention, and screening programs and new cancer treatments on population health and costs to the healthcare system. It estimates both the direct costs of medical care, as well as lost earnings and impacts on tax revenues. The lung and colorectal modules are available through the CPAC Web site (www.cancerview.ca/cancerrriskmanagement) to registered users where structured scenarios can be explored for their projected impacts. Advanced users will be able to specify new scenarios or change existing modules by varying input parameters or by accessing open source code. Model development is now being extended to cervical and breast cancers.

Objectives: This study aimed to investigate the technical documentation of manufacturers on issues of safe use of their device in a home setting.

Methods: Three categories of equipment were selected: infusion pumps, ventilators, and dialysis systems. Risk analyses, instructions for use, labels, and post market surveillance procedures were requested from manufacturers. Additionally, they were asked to fill out a questionnaire on collection of field experience, on incidents, and training activities.

Results: Specific risks of device operation by lay users in a home setting were incompletely addressed in the risk analyses. A substantial number of user manuals were designed for professionals, rather than for patients or lay carers. Risk analyses and user information often showed incomplete coherence. Post market surveillance was mainly based on passive collection of field experiences.

Conclusions: Manufacturers of infusion pumps, ventilators, and dialysis systems pay insufficient attention to the specific risks of use by lay persons in home settings. It is expected that this conclusion is also applicable for other medical equipment for treatment at home. Manufacturers of medical equipment for home use should pay more attention to use errors, lay use and home-specific risks in design, risk analysis, and user information. Field experiences should be collected more actively. Coherence between risk analysis and user information should be improved. Notified bodies should address these aspects in their assessment. User manuals issued by institutions supervising a specific home therapy should be drawn up in consultation with the manufacturer.

Objectives: The aim of this study was to systematically assess the long-term (≥ 6 months) benefits of epidural steroid injection therapies for patients with low back pain.

Methods: We identified randomized controlled trials by database searches up to October 2011 and by additional hand searches without language restrictions. Randomized controlled trials on the effects of epidurals for low back pain with follow-up for at least 6 months were included. Outcomes considered were pain relief, functional improvement in 6 to 12 months after epidural steroid injection treatment and the number of patients who underwent subsequent surgery. Meta-analysis was performed using a random-effects model.

Results: Twenty-nine articles were selected. The meta-analysis suggested that a significant treatment effect on pain was noted at 6 months of follow-up (weighted mean difference [WMD], −0.41; 95 percent confidence interval [CI], −0.66 to −0.16), but was no longer statistically significant after adjusting for the baseline pain score (WMD, −0.19; 95 percent CI, −0.61 to 0.24). Epidural steroid injection did not improve back-specific disability more than a placebo or other procedure. Epidural steroid injection did not significantly decrease the number of patients who underwent subsequent surgery compared with a placebo or other treatments (relative risk, 1.02; 95 percent CI, 0.83 to 1.24).

Conclusions: A long-term benefit of epidural steroid injections for low back pain was not suggested at 6 months or longer. Introduction of selection bias in the majority of injection studies seems apparent. Baseline adjustment is essential when we evaluate pain as a main outcome of injection therapy.

The notion of value, in the evaluation of the clinical and cost-effectiveness of therapeutic interventions, was the subject of discussion at the HTAi Policy Forum in February 2013. A summary of its discussions and conclusions is published in this issue of the journal. This commentary considers the implications of the proposal that health technology assessment (HTA) agencies should include, in the value proposition, wider societal costs and benefits as well as incorporating innovative promise.

Objectives: The aim of the study was to combine clinical results from the European Cohort of the REVERSE study and costs associated with the addition of cardiac resynchronization therapy (CRT) to optimal medical therapy (OMT) in patients with mild symptomatic (NYHA I-II) or asymptomatic left ventricular dysfunction and markers of cardiac dyssynchrony in Spain.

Methods: A Markov model was developed with CRT + OMT (CRT-ON) versus OMT only (CRT-OFF) based on a retrospective cost-effectiveness analysis. Raw data was derived from literature and expert opinion, reflecting clinical and economic consequences of patient's management in Spain. Time horizon was 10 years. Both costs (euro 2010) and effects were discounted at 3 percent per annum.

Results: CRT-ON showed higher total costs than CRT-OFF; however, CRT reduced the length of hospitalization in ICU by 94 percent (0.006 versus 0.091 days) and general ward in by 34 percent (0.705 versus 1.076 days). Surviving CRT-ON patients (88.2 percent versus 77.5 percent) remained in better functional class longer, and they achieved an improvement of 0.9 life years (LYGs) and 0.77 years quality-adjusted life years (QALYs). CRT-ON proved to be cost-effective after 6 years, except for the 7th year due to battery depletion. At 10 years, the results were €18,431 per LYG and €21,500 per QALY gained. Probabilistic sensitivity analysis showed CRT-ON was cost-effective in 75.4 percent of the cases at 10 years.

Conclusions: The use of CRT added to OMT represents an efficient use of resources in patients suffering from heart failure in NYHA functional classes I and II.

In many Western countries, long waiting times for elective surgery are a concern. Major joint replacement is an example of a type of surgery with a high volume of demand and relatively long waiting periods for patients. As populations get older, the prevalence of slowly progressive diseases, such as osteoarthritis (OA) in hip and knee joints, is increasing. Over three-quarters of a million total hip and knee replacement surgeries are done in the United States annually (1). Furthermore, according to March et al. (1997), the costs of OA have been estimated to account for up to 1–2.5 percent of the Gross National Product (GNP) in several developed countries (2). In Finland, a total of 11,104 total joint replacements (TJRs) were performed in 2004 (hip 6,600 and knee 5,905), with the median waiting time of 181 days for the surgery (hip 153 and knee 209 days). Until 2007, the number of TJRs was 17,334 (hip 7,698 and knee 9,636), with a median waiting time of 120 and 142 days, respectively (3;4). The mean waiting time for elective surgical procedures is approximately 3 months in several countries and the maximum waiting times can stretch into years.

Objectives: Mechanical circulatory support through left ventricular assist devices (LVADs) improves survival and quality of life for patients with end-stage heart failure who are ineligible for cardiac transplantation. Our aim was to calculate the cost-effectiveness of continuous-flow LVADs.

Methods: A cost-utility analysis from a societal perspective was performed. A lifetime Markov model was set up in which continuous-flow LVAD was compared with optimal medical therapy (OMT). The treatment effect was modeled indirectly combining the results of the REMATCH trial comparing OMT with a pulsatile-flow LVAD and the HeartMate II Destination Therapy Trial comparing a pulsatile-flow LVAD with a continuous-flow LVAD. Cost data were based on real-world financial data of sixty-nine patients with a HeartMate II implantation from the University Medical Centre Utrecht (the Netherlands). One-way and probabilistic sensitivity analyses were performed.

Results: Comparing the continuous-flow HeartMate II with OMT, 3.23 (95 percent confidence interval [CI], 2.18–4.49) life-years were gained (LYG) or 2.83 (95 percent CI, 1.91–3.90) quality-adjusted life-years (QALYs). The cost of an LVAD implant was approximately €126,000, of which the device itself represented the largest cost, being €70,000. Total incremental costs amounted to €299,100 (95 percent CI, 190,500–521,000). This resulted in an incremental cost-effectiveness ratio of €94,100 (95 percent CI, 59,100–160,100) per LYG or €107,600 (95 percent CI, 66,700–181,100) per QALY. Sensitivity analyses showed these results were robust.

Conclusions: Although LVAD destination therapy improves survival and quality of life, it remains a relatively expensive intervention which renders the reimbursement of this therapy questionable.