Healthcare technology assessment (HTA) aims to support decisions as to which technologies should be used in which situations to optimize value. Because such decisions will create winners and losers, they are bound to be controversial. HTA, then, faces a dilemma: should it stay away from such controversies, remaining a source of incomplete advice and risking an important kind of marginalization, or should it enter the controversy? The question is a challenging one, because we lack agreement on principles that are fine grained enough to tell us what choices we should make. In this study, we will argue that HTA should take a stand on ethical issues raised by the technology that is being investigated. To do so, we propose adding a form of procedural justice to HTA to arrive at decisions that the public can regard as legitimate and fair. A fair process involves deliberation about the reasons, evidence, and rationales that are considered relevant to meeting population-health needs fairly. One important way to make sure that there is real deliberation about relevant reasons is to include a range of stakeholders in the deliberative process. To illustrate how such deliberation might work, we use the case of cochlear implants for deaf children.

The practice of health care technology assessment involves ethical considerations in all of its varied functions and techniques. Ethical issues in technology assessment can be grouped into the broad categories of normative concepts, diagnosis, prevention and therapy, research and the advancement of knowledge, and allocation of resources. Moreover, the ethics of the assessment process itself must be evaluated in terms of the integrity of the project's goals, procedures, and effects, and evaluators’ open and self-critical acknowledgment of their purposes. As a relatively new field, technology assessment can benefit from using a variety of analytic approaches as it works to develop its own methods for evaluating ethical issues related to technology.

Objectives: To develop and validate a paper-based instrument that is simple to administer and produces a reliable estimate of patient standard gamble (SG) utilities for current health status.

Methods: A 1-page paper questionnaire instrument, paper standard gamble (PSG), was designed to estimate SG utilities. We performed two studies to assess the validity of PSG. First we compared PSG and SG utilities for current health in patients with prostate cancer. They randomly received either PSG followed by SG or vice versa, always with an intervening SF-12. In the second validity study, we assessed the test-retest reliability of PSG by administering it to prostate cancer patients twice, at least 2 weeks apart.

Results: In the first study, utilities were assessed in 64 men (32 per SG/PSG order group). A paired-comparison t test suggested no difference between SG and PSG (mean difference = −0.007; 95% confidence interval (CI), $-$0.022 to 0.008). The concordance correlation coefficient was 0.92 (95% CI, 0.79 to 0.99). In the second study, test and retest PSGs were available for 184 patients. The concordance correlation coefficient was 0.88 (95% CI, 0.73 to 0.94).

Conclusions: These data suggest that PSG may serve as a reliable substitute for SG when current health utility is of interest. PSG may have particular advantages for acquisition of health-related quality-of-life data in longitudinal studies.

Background: Although value issues are increasingly addressed in health technology assessment (HTA) reports, HTA is still seen as a scientific endeavor and sometimes contrasted with value judgments, which are considered arbitrary and unscientific. This article aims at illustrating how numerous value judgments are at play in the HTA process, and why it is important to acknowledge and address value judgments.

Methods: A panel of experts involved in HTA, including ethicists, scrutinized the HTA process with regard to implicit value judgments. It was analyzed whether these value judgments undermine the accountability of HTA results. The final results were obtained after several rounds of deliberation.

Results: Value judgments are identified before the assessment when identifying and selecting health technologies to assess, and as part of assessment. They are at play in the processes of deciding on how to select, frame, present, summarize or synthesize information in systematic reviews. Also, in economic analysis, value judgments are ubiquitous. Addressing the ethical, legal, and social issues of a given health technology involves moral, legal, and social value judgments by definition. So do the appraisal and the decision-making process.

Conclusions: HTA by and large is a process of value judgments. However, the preponderance of value judgments does not render HTA biased or flawed. On the contrary they are basic elements of the HTA process. Acknowledging and explicitly addressing value judgments may improve the accountability of HTA.

Economic evaluations of Pharmaceuticals are increasingly being conducted in conjunction with randomized phase III clinical trials to meet the demand for pharmacoeconomic data when new products are launched. While the need for such data is often global, the trials in which relevant information may be collected are often conducted in only one or a limited number of countries. A critical issue is how data from pivotal clinical trials in one setting can serve as the basis for pharmacoeconomic evaluations in others. We address this issue and report on four economic evaluations that we undertook in conjunction with a recent U.S. phase III clinical trial of recombinant human deoxyribonuclease (rhDNase), which is used to improve pulmonary function in patients with cystic fibrosis (CF). The objective of these evaluations was to estimate the potential impact of rhDNase therapy in France, Germany, Italy, and the United Kingdom on the direct costs of medical care for the treatment of respiratory tract infections (RTIs) in patients with CF. Analyses of economic impact were undertaken

Background: During 2001, over 32,000 patients in the United Kingdom received renal replacement therapy (RRT). Approximately half had a functioning transplant, with the remainder receiving dialysis therapy. The main form of dialysis is hemodialysis (HD), which is provided to 37.1 percent of the RRT population. HD is provided in three main settings: hospital (24.5 percent), satellite (10.9 percent), or home (1.7 percent). The objective of this study is to explore the cost-effectiveness of these different modalities.

Methods: By using clinical and cost data from a systematic review, a Markov model was developed to assess the costs and benefits of the three different modalities. The model included direct health service costs and quality-adjusted life years (QALYs). Sensitivity analyses were performed to assess the robustness of the results.

Results: Satellite HD has lower costs £46,000 and £62,050 at 5 and 10 years than home HD £47,660 and £63,540. The total effectiveness of home HD was slightly greater than for satellite HD, so the incremental cost per QALY of home versus satellite HD was modest at £6,665 at 5 years and £3,943 at 10 years. Both modalities dominated hospital HD.

Conclusions: Results from the study reveal that satellite HD was less costly than home HD, and home HD was less costly than hospital HD. The lack of robust data on the effectiveness and new dialysis equipment, which were not included in this review, throws some caution on these results. Nonetheless, the results are supportive of a shift from hospital HD to satellite and home HD.

Objectives: The aim of this study was to review studies reporting cost-effectiveness of exercise-based interventions in treatment of various diseases.

Methods: Systematic literature search using several databases. Abstracts initially screened independently by two authors, full-text articles again evaluated by two authors, who decided whether an article should be included. Included were scientifically valid articles describing controlled studies that included an exercise-based intervention in the treatment of an established medical condition, and also reported on the cost-effectiveness of the intervention, or its effect on the utilization of health services. Quality was assessed with an established approach.

Results: A total of 914 articles were identified, of them 151 were obtained for closer review. Sixty-five articles describing sixty-one studies were included. Most (82 percent) were randomized trials. Twenty-eight studies dealt with musculoskeletal disorders, fifteen with cardiology, four with rheumatic diseases, four with pulmonary diseases, three with urinary incontinence, and two with vascular disorders. There was one study each in the fields of oncology, chronic fatigue, endocrinology, psychiatry, and neurology. Exercise interventions in musculoskeletal disorders were deemed to be cost-effective in 54 percent, in cardiology in 60 percent, and in rheumatic diseases in 75 percent of the cases. There was some evidence that exercise might be cost-effective in intermittent claudication, breast cancer patients, diabetes, and schizophrenia.

Conclusions: The number of studies assessing cost-effectiveness of exercise interventions in various diseases is still limited. The results show large variation but suggest that some exercise interventions can be cost-effective. Most convincing evidence was found for rehabilitation of cardiac and back pain patients; however, even in these cases, the evidence was partly contradictory.

The Problem: There is a shortage and maldistribution of medically trained health professionals to deliver cost-effective maternal and child health (MCH) services. Hence, cost-effective MCH services are not available to over half the population of Uganda and progress toward the Millennium Development Goals for MCH is slow. Optimizing the roles of less specialized health workers (“task shifting”) is one strategy to address the shortage and maldistribution of more specialized health professionals.

Policy Options: (i) Lay health workers (community health workers) may reduce morbidity and mortality in children under five and neonates; and training for traditional birth attendants may improve perinatal outcomes and appropriate referrals. (ii) Nursing assistants in facilities might increase the time available from nurses, midwives, and doctors to provide care that requires more training. (iii) Nurses and midwives to deliver cost-effective MCH interventions in areas where there is a shortage of doctors. (iv) Drug dispensers to promote and deliver cost-effective MCH interventions and improve the quality of the services they provide. The costs and cost-effectiveness of all four options are uncertain. Given the limitations of the currently available evidence, rigorous evaluation and monitoring of resource use and activities is warranted for all four options.

Implementation Strategies: A clear policy on optimizing health worker roles. Community mobilization and reduction of out-of-pocket costs to improve mothers’ knowledge and care-seeking behaviors, continuing education, and incentives to ensure health workers are competent and motivated, and community referral and transport schemes for MCH care are needed.

Objectives: The aim of this study was to explore the use of surrogate outcomes—a substitute outcome that predicts final patient-related outcomes—in cost-effectiveness models (CEM) within health technology assessment (HTA) reports and provide guidance for their future use.

Methods: Our sampling frame was all UK HTA Program monograph series reports published in 2005 and 2006. Reports were included if they addressed a treatment effectiveness/efficacy question and included a CEM based on a surrogate outcome. The two authors independently applied inclusion and exclusion criteria, and the following data was extracted from included reports: source of surrogate outcome, level of evidence for validation of the surrogate outcomes, methods used in report to quantify link between surrogate outcome and final outcome, and consideration of the uncertainty associated with using surrogate outcomes in the results or conclusions of report.

Results: Of 100 HTA reports, 35 complied with the inclusion criteria. Of these, four (11 percent) reports included a CEM based on a surrogate outcome. All four reports sourced treatment-related changes in surrogate outcome through a systematic review of the literature. One provided Level 1 surrogate evidence (randomized controlled trial data showing a strong association between the change in surrogate outcome and change final outcome); two reported Level 2 evidence (observational study data); and one provided Level 3 evidence (disease natural/ history data). The transparency of quantification and exploration of uncertainty of the surrogate and final outcome relationship varied considerably across all four reports.

Conclusions: Recommendations are made for the use of surrogate outcomes in future HTA reports.

The costs, effects, and cost-effectiveness of the Dutch Lung Transplantation program were assessed. The results show that lung transplantation is a very costly intervention that improves survival and quality of life. Costs per life-year and per QALY gained were, respectively, US $90,000 and US $71,000.

Decisions relating to designing and implementing economic evaluations alongside clinical trials are dependent on a number of issues. Initial consideration should focus on whether an economic evaluation is appropriate, whether a randomized controlled trial (RCT) is a suitable vehicle, and whether the design of the specific RCT is appropriate for integration of economic evaluation. If an economic evaluation is appropriate, decisions are required on how economic data are to be collected and analyzed. This paper discusses these and other issues and, where appropriate, provides recommendations for future studies.

Objectives: Some countries make considerable effort to involve patients and patient groups in their health technology assessment (HTA) processes; others are only just considering or are yet to consider patient involvement in HTA.

Methods: This commentary offers four arguments why patient involvement should be prioritized by those HTA agencies that do not yet involve patients: (1) from a patients’ rights perspective, (2) based on patient and community values, (3) centering on evidentiary contributions, and (4) from a methodological perspective.

Results: The first argument builds on the Alma-Ata Declaration, which holds that patients have a right and duty to have a say in the planning and delivery of their health care, individually and collectively. Where HTA is used to determine access to technologies and services, we argue that patients have a right to be heard. The second argues that decisions about treatments and services need to be aligned with the core values and morals of the patients whom the health system serves. The third argues that patients have unique knowledge and insights about living with a health condition and their needs for services and treatments regarding that condition, which can add to the knowledge base and value of the HTA process. The fourth argues that involvement of patients can facilitate methodological advancement of HTA, in areas such as early scientific advice and managed entry with evidence development.

Conclusions: An HTA process that includes patient perspectives can, therefore, provide added value to patients, policy makers and healthcare professionals alike.

Objectives: Although there is increased awareness of patient and public involvement (PPI) among health technology assessment (HTA) organizations, evaluations of PPI initiatives are relatively scarce. Our objective as members of Health Technology Assessment International's (HTAi's) Patient and Citizen Involvement Group (PCIG) was to advance understanding of the range of evaluation strategies adopted by HTA organizations and their potential usefulness.

Methods: In March 2016, a survey was sent to fifty-four HTA organizations through the International Network of Agencies for Health Technology Assessment (INAHTA) and contacts of members of HTAi's PCIG. Respondents were asked about their organizational structure; how patients and members of the public are involved; whether and how PPI initiatives have been evaluated, and, if so, which facilitators and challenges to evaluation were found and how results were used and disseminated.

Results: Fifteen (n = 15) programs from twelve countries responded (response rate 27.8 percent) that involved patients (14/15) and members of the public (10/15) in HTA activities. Seven programs evaluated their PPI activities, including participant satisfaction (5/7), process (5/7) and impact evaluations (4/7). Evaluation results were used to improve PPI activities, identify education and training needs, and direct strategic priorities. Facilitators and challenges revolved around the need for stakeholder buy-in, sufficient resources, senior leadership, and including patients in evaluations.

Conclusions: A small but diverse set of HTA organizations evaluate their PPI activities using a range of strategies that reflect the range of rationales and approaches to PPI in HTA. It will be important for HTA organizations to draw on evaluation theories and methods.

Objectives: Chondral defects of the knee cartilage are prevalent. Autologous chondrocyte implantation (ACI) and mosaicplasty are increasingly used to treat symptomatic knee defects. This study assessed the costs and health status outcomes after ACI and mosaicplasty.

Methods: Patients were eligible to participate in this cross-sectional study if they received ACI or mosaicplasty at the Royal National Orthopaedic Hospital between 1997 and 2001 or were on a waiting list for ACI. Secondary-care resource use was collected to 2 years postoperatively using a resource collection proforma. Participants responded to postal questions about sociodemographic characteristics and knee-related (Modified Cincinnati Knee Rating System) and general health status (EQ-5D).

Results: Fifty-three ACI, twenty mosaicplasty, and twenty-two patients waiting for ACI participated. The average cost per patient was higher for ACI (£10,600: 95 percent confidence interval [CI], £10,036–£11,214) than mosaicplasty (£7,948: 95 percent CI, £6,957–£9,243). Postoperatively, ACI and mosaicplasty patients (combined) experienced better health status than those waiting for ACI. ACI patients tended to have better health status outcomes than mosaicplasty patients (not statistically significant). Estimated average EQ-5D social tariff improvements for quality-adjusted life year (QALY) calculations were 0.23 (ACI) and 0.06 (mosaicplasty). Average costs per QALY were £23,043 (ACI) and £66,233 (mosaicplasty). The incremental cost effectiveness ratio (ICER) for providing ACI over mosaicplasty was £16,349.

Conclusions: Average costs were higher for ACI than mosaicplasty. However, both the estimated cost per QALY and ICER for providing ACI over mosaicplasty fell beneath an implicit English funding threshold of £30,000 per QALY. Prospective studies should include measures of utility to confirm the estimated cost utility ratios of ACI and mosaicplasty.

Objective: Extracorporeal shock wave therapy (ESWT) as an emerging technology in orthopedics has been assessed in Austria with the objective to establish a scientific basis for pending and pressing health policy decisions. Despite encouraging results within some indications and the promising prospect of a noninvasive treatment for some orthopedic diseases, it seemed crucial to assess this new field of application in the light of evidence-based standards, without forgetting the reality of healthcare decisions. This article presents the results of the abovementioned assessment in the context of the policy-making process in Germany, Austria, and Switzerland.

Methods: The study is based on an overview of the already existing reviews on the effectiveness of ESWT as carried out by different actors (health insurances and orthopedists), and on an overview of additional literature covering all relevant aspects (mode of action, recommendations, economic estimates). Orthopedic societies, several institutions involved in health technology assessment, and health insurance agencies were contacted in search of further relevant literature, ongoing studies, and assessments.

Results: Until now, clear evidence has not been available on the effectiveness of ESWT in four currently recommended indications (tendinosis calcarea of the shoulder, epicondylopathia humeri radialis, calcaneal spur, and pseudarthrosis), although a substantial body of literature has been produced. Despite encouraging results in some indications, the lack of coherent therapy schemes and the poor quality in the design of clinical studies has been serious enough to hinder the drawing of reasonable conclusions.

Conclusions: Due to the rapid diffusion of ESWT and the pressure on reimbursement agencies to prove the ineffectiveness of ESWT, the policy option of a pragmatic approach is recommended to controll the diffusion of an uncertain health technology, alongside active research that would allow a more comprehensive appraisal of this potentially interesting treatment strategy in orthopedics.

Objectives: Differential pricing, based on countries’ purchasing power, is recommended by the World Health Organization to secure affordable medicines. However, in developing countries innovative drugs often have similar or even higher prices than in high-income countries. We evaluated the potential implications of trastuzumab global pricing policies in terms of cost-effectiveness (CE), coverage, and accessibility for patients with breast cancer in Latin America (LA).

Methods: A Markov model was designed to estimate life-years (LYs), quality-adjusted life-years (QALYs), and costs from a healthcare perspective. To better fit local cancer prognosis, a base case scenario using transition probabilities from clinical trials was complemented with two alternative scenarios with transition probabilities adjusted to reflect breast cancer epidemiology in each country.

Results: Incremental discounted benefits ranged from 0.87 to 1.00 LY and 0.51 to 0.60 QALY and incremental CE ratios from USD 42,104 to USD 110,283 per QALY (2012 U.S. dollars), equivalent to 3.6 gross domestic product per capita (GDPPC) per QALY in Uruguay and to 35.5 GDPPC in Bolivia. Probabilistic sensitivity analysis showed 0 percent probability that trastuzumab is CE if the willingness-to-pay threshold is one GDPPC per QALY, and remained so at three GDPPC threshold except for Chile and Uruguay (4.3 percent and 26.6 percent, respectively). Trastuzumab price would need to decrease between 69.6 percent to 94.9 percent to became CE in LA.

Conclusions: Although CE in other settings, trastuzumab was not CE in LA. The use of health technology assessment to prioritize resource allocation and support price negotiations is critical to making innovative drugs available and affordable in developing countries.

Objectives: A mapping exercise was undertaken to determine how HTA is being described and conducted across the International Network of Agencies for Health Technology Assessment (INAHTA), with the aim of harmonizing terminologies and approaches.

Methods: Three progressive surveys were undertaken. In 2010, INAHTA agencies were asked to provide details on all of their HTA products. In 2013, additional information was sought on key methodological characteristics of five of the most common HTA product types. Subsequently, final agreement was sought on three proposed product types.

Results: Forty-five HTA agencies responded to at least one of the surveys. In 2010, twenty-one agencies reported publishing over seventy named HTA products. Core domains associated with full HTA reports were reported by a third of agencies but were labeled differently, so products were classified according to product type (n = 17). Agencies producing short, tailored products increased between 2010 and 2013, with the publication of rapid reviews doubling from 33 percent to 66 percent. In 2013, half of the agencies adapted their common HTA products from documents produced by other agencies. A consensus (>70 percent) was achieved on definitions for HTA reports, mini-HTAs, and rapid reviews.

Conclusions: The product label for an HTA is not always indicative of its content. Terminology has, therefore, been agreed to make explicit the trade-off between rigor and timeliness in three common HTA product types. An INAHTA Product Type (IPT) Mark has been created to identify each of these. It is hoped this will further facilitate HTA adaptation between agencies and reduce duplication of effort.

Objectives: There is a growing need for efficient procedures for identification of emerging technologies by horizon scanning systems. We demonstrate the value of best-worst scaling (BWS) in exploring clinicians’ views on emerging technologies that will impact outcomes in hepatocellular carcinoma (HCC) in the next 5 to 10 years.

Methods: Clinicians in Asia, Europe, and the United States were surveyed and their views about eleven emerging technologies relevant to HCC were explored using BWS (case 1). This involved systematically presenting respondents with subsets of five technologies and asking them to identify those that will have the most and least impact on HCC within 5 to 10 years. Statistical analysis was based on sequential best-worst and analyzed using conditional logistic regression.

Results: A total of 120 clinicians uniformly distributed across ten countries completed the survey (37 percent response rate). Respondents were predominately hepatologist (41 percent) who focused on HCC (65 percent) and had national influence in this field (39 percent). Respondents viewed molecular targeted therapy (p < .001) and early detection of HCC (p < .001) as having most potential, while improved surgical techniques (p < .001) and biopsy free HCC diagnostics (p < .001) were viewed upon negatively.

Conclusions: We demonstrate that BWS could be an important research tool to facilitate horizon scanning and HTA more broadly. Our research demonstrates the value of including clinicians’ preferences as a source of data in horizon scanning, but such methods could be used to incorporate the opinions of a broad array of stakeholders, including those in advocacy and public policy.