Objectives: The aim of this study was to present the first four collaborative health technology assessment (HTA) processes on health technologies of different types and life cycles targeted toward diverse HTA users and facilitators, as well as the barriers of these collaborations.

Methods: Retrospective analysis, through four case studies, was performed on the first four collaboration experiences of agencies participating in the EUnetHTA Joint Action project (2010–12), comprising different types and life cycles of health technologies for a diverse target audience, and different types of collaboration. The methods used to initiate collaboration, partner contributions, the assessment methodology, report structure, time frame, and factors acting as possible barriers to and facilitators of this collaboration were described.

Results: Two ways were used to initiate collaboration in the first four collaborative HTA processes: active brokering of information, so-called “calls for collaboration,” and individual contact between agencies after identifying a topic common to two agencies in the Planned and Ongoing Projects database. Several success factors are recognized: predefined project management, high degree of commitment to the project; adherence to timelines; high relevance of technology; a common understanding of the methods applied and advanced experience in HTA; finally, acceptance of English-written reports by decision makers in non–English-speaking countries. Barriers like late identification of collaborative partners, nonacceptance of English language and different methodology of assessment should be overcome.

Conclusions: Timely and efficient, different collaborative HTA processes on relative efficacy/effectiveness and safety on different types and life cycles of health technologies, targeted toward diverse HTA users in Europe are possible. There are still barriers to overcome.

Objectives: Hospital-based health technology assessment (HB-HTA) is becoming increasingly relevant because of its role in managing the introduction and withdrawal of health technologies. The organizational arrangement in which HB-HTA activities are conducted depends on several contextual factors, although the dominant models have several similarities. The aims of this study were to explore, describe, interpret, and explain seven cases of the application of HB-HTA logic and to propose a classification for HB-HTA organizational models which may be beneficial for policy makers and HTA professionals.

Methods: The study was part of the AdHopHTA Project, granted under the European 7th Framework Research Programme. A case study methodology was applied to analyze seven HB-HTA initiatives in seven countries, with collection of qualitative and quantitative data. Cross-case analysis was performed within the framework of contingent organizational theory.

Results: Evidence showed that some organizational or “structural” variables, namely the level of procedure formalization/structuration and the level of integration with other HTA bodies at the national, regional, and provincial levels, predominantly shape the HB-HTA approach, determining a contingency model of HB-HTA. Crossing the two variables, four options have emerged: integrated specialized HTA unit, stand-alone HTA unit, integrated-essential HTA, independent group unit.

Conclusions: No one-best-way approach can be used for HTA at the hospital level. Rather, the characteristics of HTA models depend on many contextual factors. Such conceptualization may aid the diffusion of HB-HTA to inform managerial decision making and clinical practice.

Objectives: The aim of this study was to obtain information on methods used to measure health technology assessment (HTA) influence, decisions that were influenced, and outcomes linked to HTA.

Methods: Electronic databases were used to locate studies in which HTA influence had been demonstrated. Inclusion criteria were studies that reliably reported consideration by decision makers of HTA findings; comparative studies of technology use before and after HTA; and details of changes in policy, health outcomes, or research that could be credibly linked to an HTA.

Results: Fifty-one studies were selected for review. Settings were national (24), regional (12), both national and regional (3) hospitals (9), and multinational (3). The most common approach to appraisal of influence was review of policy or administrative decisions following HTA recommendations (51 percent). Eighteen studies (35 percent) reported interview or survey findings, thirteen (26 percent) reviewed administrative data, and six considered the influence of primary studies. Of 142 decisions informed by HTA, the most common types were on routine clinical practice (67 percent of studies), coverage (63 percent), and program operation (37 percent). The most frequent indications of HTA influence were on decisions related to resource allocation (59 percent), change in practice pattern (31 percent), and incorporation of HTA details in reference material (18 percent). Few publications assessed the contribution of HTA to changing patient outcomes.

Conclusions: The literature on HTA influence remains limited, with little on longer term effects on practice and outcomes. The reviewed publications indicated how HTA is being used in different settings and approaches to measuring its influence that might be more widely applied, such as surveys and monitoring administrative data.

Background: Adaptive approaches to the introduction of drugs and medical devices involve the use of an evolving evidence base rather than conventional single-point-in-time evaluations as a proposed means to promote patient access to innovation, reduce clinical uncertainty, ensure effectiveness, and improve the health technology development process.

Methods: This report summarizes a Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations from invited experts, discussions among attendees about real-world case examples, and background paper.

Results: For adaptive approaches to be understood, accepted, and implemented, the Forum identified several key issues that must be addressed. These include the need to define the goals of and to set priorities for adaptive approaches; to examine evidence collection approaches; to clarify the roles and responsibilities of stakeholders; to understand the implications of adaptive approaches on current legal and ethical standards; to determine costs of such approaches and how they will be met; and to identify differences in applying adaptive approaches to drugs versus medical devices. The Forum also explored the different implications of adaptive approaches for various stakeholders, including patients, regulators, HTA/coverage bodies, health systems, clinicians, and industry.

Conclusions: A key outcome of the meeting was a clearer understanding of the opportunities and challenges adaptive approaches present. Furthermore, the Forum brought to light the critical importance of recognizing and including a full range of stakeholders as contributors to a shared decision-making model implicit in adaptive pathways in future discussions on, and implementation of, adaptive approaches.

Objectives: To systematically assess the evidence for effectiveness of hyperbaric oxygen therapy in the treatment of conditions of significance in the West Midlands region and to determine whether there is a case for establishing a hyperbaric oxygen unit in the region.

Methods: Systematic review of the literature assessing randomized controlled trials from 1968 onward.

Results: A total of 154 full-text articles was obtained of which 13 relevant randomized control trials were identified. There was little consistency in the studies. Treatment protocols, study groups, time to treatment, and other characteristics and outcomes measured all varied considerably. No convincing evidence of effectiveness was identified.

Conclusions: Although hyperbaric oxygen therapy is clearly appropriate in the treatment of decompression sickness and air/gas embolism, there is no convincing evidence of effectiveness for the conditions reviewed, despite its widespread use. Although this review has found no evidence to support the establishment of a unit in the West Midlands, there is a physiological case for an effect in conditions involving hypoxia and, given the limited volume (and in some cases quality) of published research, a case for a national research program.I thank the staff of the West Midlands Development and Evaluation Service Systematic Reviews Course (Dr. Amanda Burls, Dr. Chris Hyde, and Ms. Jackie Young), who reviewed the report.

A pilot study was done to assess the feasibility of reducing the hospital stays of patients with total hip replacement (THR). The length of hospital stay for these patients depends largely on how rehabilitation, mostly physical therapy, is organized. This study shows that not more than a half hour per postoperative day was devoted to care services and rehabilitation activities. It is feasible and less expensive to reduce substantially hospital stay by planned physical therapy in the patient's home. These results have prompted a randomized controlled clinical trial to assess hospital versus home rehabilitation.

Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.

Objectives: The reduction in cognitive decline depends on timely diagnosis. The aim of this systematic review was to analyze the current available information and communication technologies-based instruments for cognitive decline early screening and detection in terms of usability, validity, and reliability.

Methods: Electronic searches identified 1,785 articles of which thirty-four met the inclusion criteria and were grouped according to their main purpose into test batteries, measures of isolated tasks, behavioral measures, and diagnostic tools.

Results: Thirty one instruments were analyzed. Fifty-two percent were personal computer based, 26 percent tablet, 13 percent laptop, and 1 was mobile phone based. The most common input method was touchscreen (48 percent). The instruments were validated with a total of 4,307 participants: 2,146 were healthy older adults (M = 73.59; SD = 5.12), 1,104 had dementia (M = 74.65; SD = 3.98) and 1,057 mild cognitive impairment (M = 74.84; SD = 4.46). Only 6 percent were administered at home, 19 percent reported outcomes about usability, and 22 percent about understandability. The methodological quality of the studies was good, the weakest methodological area being usability. Most of the instruments obtained acceptable values of specificity and sensitivity.

Conclusions: It is necessary to create home delivered instruments and to include usability studies in their design. Involvement of people with cognitive decline in all phases of the development process is of great importance to obtain valuable and user-friendly products. It would be advisable for researchers to make an effort to provide cutoff points for their instruments.

Objectives: To determine the cost-effectiveness (CE) and cost-utility (CU) of drug-eluting stents (DES) compared to bare metal stents (BMS) in Ontario using a large prospective “real-world” cohort study and determine the extent to which results vary by patient risk subgroups.

Methods: A field evaluation was conducted based on all stent procedures in the province of Ontario between December 1, 2003, and March 31, 2005, with a minimum subject follow-up of 1 year. Effectiveness data from the study using a propensity-score matched cohort were combined with resource utilization and cost data and quality of life (QOL) data from the published literature in a decision analytic modeling framework to determine 2-year cost-effectiveness (cost per revascularization avoided) and cost-utility (cost per quality-adjusted life-year ([QALY] gained). Stochastic model parameter uncertainty was expressed using probability distributions and analyzed using a probabilistic model. Modeling assumptions were assessed using traditional deterministic sensitivity analysis.

Results: Significant differences in revascularization rates were found for patients with two or more high risk factors. Despite these differences, the CE and CU of DES remained high (e.g., $419,000 per QALY gained in the most favorable patient risk subgroup). In sensitivity analysis, the difference in cost between DES and BMS had an impact on the CE and CU results. For example, at a price differential of $500, the CU of DES was $20,000/QALY for one patient subgroup and DES was dominant (i.e., less costly and more effective) in another.

Conclusions: At current prices, the CE/CU of DES compared with BMS is high even in patient high risk subgroups. As the relative price of DES decrease, the value for money attractiveness of DES increases, especially for selected high risk patients.

Objectives: The aim of this study was to examine the evidence payers cited in their coverage policies for multi-gene panels and sequencing tests (panels), and to compare these findings with the evidence payers cited in their coverage policies for other types of medical interventions.

Methods: We used the University of California at San Francisco TRANSPERS Payer Coverage Registry to identify coverage policies for panels issued by five of the largest US private payers. We reviewed each policy and categorized the evidence cited within as: clinical studies, systematic reviews, technology assessments, cost-effectiveness analyses (CEAs), budget impact studies, and clinical guidelines. We compared the evidence cited in these coverage policies for panels with the evidence cited in policies for other intervention types (pharmaceuticals, medical devices, diagnostic tests and imaging, and surgical interventions) as reported in a previous study.

Results: Fifty-five coverage policies for panels were included. On average, payers cited clinical guidelines in 84 percent of their coverage policies (range, 73–100 percent), clinical studies in 69 percent (50–87 percent), technology assessments 47 percent (33–86 percent), systematic reviews or meta-analyses 31 percent (7–71 percent), and CEAs 5 percent (0–7 percent). No payers cited budget impact studies in their policies. Payers less often cited clinical studies, systematic reviews, technology assessments, and CEAs in their coverage policies for panels than in their policies for other intervention types. Payers cited clinical guidelines in a comparable proportion of policies for panels and other technology types.

Conclusions: Payers in our sample less often cited clinical studies and other evidence types in their coverage policies for panels than they did in their coverage policies for other types of medical interventions.

Objectives: Little is known about the costs and the effects of cardiovascular prevention programs targeted at medical and behavioral risk factors. The aim was to evaluate the cost-utility of a cardiovascular prevention program in a general sample of highly educated adults after 1 year of intervention.

Methods: The participants were randomly assigned to intervention (n = 208) and usual care conditions (n = 106). The intervention consisted of medical interventions and optional behavior-change interventions (e.g., a tailored Web site). Cost data were registered from a healthcare perspective, and questionnaires were used to determine effectiveness (e.g., quality-adjusted life-years [QALYs]). A cost-utility analysis and sensitivity analyses using bootstrapping were performed on the intermediate results.

Results: When adjusting for baseline utility differences, the incremental cost was €433 and the incremental effectiveness was 0.016 QALYs. The incremental cost-effectiveness ratio was €26,910 per QALY.

Conclusions: The intervention was cost-effective compared with usual care in this sample of highly educated adults after 1 year of intervention. Increased participation would make this intervention highly cost-effective.

In our article “Romiplostim and eltrombopag for immune thrombocytopenia: methods for indirect comparison” published in the Journal (1), we presented an indirect comparison of the effectiveness of eltrombopag and romiplostim in raising platelet counts in patients with immune thrombocytopenia (ITP). Indirect comparison analyses are recommended by the National Institute for Health and Care Excellence (NICE) in cases where randomized head-to-head studies do not exist, and were used by NICE in their guidance for the eltrombopag Single Technology Appraisal submission (2).

Objectives: The aim of this study was to benchmark the proposal forms used by a sample of Italian hospitals to inform the budget process for the adoption of new technology to understand the relationship with the guidelines provided by the Health Technology Assessment (HTA) literature.

Methods: A literature review was first undertaken to identify the frameworks developed to support decision making regarding new technology at a hospital level. A checklist of criteria drawn up according to five main perspectives (technology, patient, organization, economics, and level of evidence) has been formalized to review and compare the collected proposal forms.

Results: The “technology” perspective appears to have been broadly covered. The “patient” perspective has focused to clinical issues and partially neglects other dimensions such as patient satisfaction and potential adverse events. The “organization” dimension has paid little attention to change management. The “economics” dimension has been broadly covered, even though a sensitivity analysis has not been considered. The “level of evidence” that is required for submitting the proposal form is little.

Conclusions: The proposal forms used to inform the budget process regarding the adoption of new technology are accountable for a limited set of dimensions from among those proposed in literature. Further research is required to understand how to render technology assessment multidimensional, multidisciplinary, evidence-based, and accountable at a hospital level.

Objectives: The use and implementation of health technologies do influence and are influenced by organizational structures and processes as well as patient's experiences and actions. The objective of this study is to discuss the content and managing of organizational and patient-related assessments in HTAs.

Methods: Quantitative and qualitative analysis were performed based on a review of organizational and patient-related assessments included in a random sample of fifty HTA reports. The reports were identified from INAHTA members’ Web sites where homepages of HTA agencies where searched for full HTA reports, published in English or Scandinavian languages.

Results: HTA reports including organizational assessments mainly comprised issues related to process and structure, while issues such as control and evaluation of the technology were included less often. Cultural and environmental issues were included in a little over half of the reports. Reports including patient-related assessments mainly comprised psychological issues. Patients’ perceptions of the technology's effect on their health, patient-information, and social and ethical issues were included less often. Few reports included implications for the patient's significant others. There was considerable variation in how comprehensive the issues were managed as there was in the methodological transparency of the HTA reports.

Conclusions: There is room for improvement in the assessments of organizational and patient-related issues. Absence of a description of the considerations made when determining the content and methods of the assessments limits the usefulness of an HTA. The reader is left uncertain of the relevance and validity of the organizational and patient-related assessments.

It has been suggested that preoperative total parentetal nutrition may be used to reduce the risk of nutrition-associated postoperative complications in high-risk patients. These patients can be identified based on their nutritional status. The efficiency of this intervention is assessed using the technique of cost-utility analysis. Data from multiple sources is integrated to perform the economic assessment.

The cost-utility ratios for treating several malnourished patients with localized upper gastrointestinal cancer are below $40,000. These cost-utility ratios compare favorably with published results of other programs. The ratios increase considerably if patients who are better nourished (at lower risk of postoperative complication) receive the intervention. The analysis is very sensitive to the efficacy of the intervention.

The consensus development program for health care is an American invention which dates back to 1977. It is derived out of concern for the appropriate use of medical technology and designed to formalize the process by which research pertinent to health care is disseminated and used.

Magnetic resonance (MR) imaging is an exciting new diagnostic modality that has created tremendous interest in the medical profession. Although not unparalleled, the excitement engendered by MR imaging conjures up memories of the “CAT fever” induced by introduction of X-ray computed tomography (CT) scanners in 1973 (19).

Physicians through the ages have practiced their trade with more or less regard to the effects of their treatment on their patients' sense of well-being, if not as much as on the disease itself.

Until recently, however, little attempt has been made to measure the effect of disease upon quality of life and how this quality is or is not improved by the treatment. Several means have now evolved and in areas of medicine where the treatment may profoundly affect the patient's sense of well-being, effort is being made to gauge these consequences.