Patient engagement in health technology assessment (HTA) has become increasingly important over the past 20 years. Academic and practitioner literature has produced numerous case studies and best practice accounts of patient involvement practices around the world. This text analyzes the experience of being involved in an Institute for Clinical and Economic Review (ICER) HTA review in the United States. The analysis comes from the joint perspective of three patient organizations: Lupus and Allied Diseases Association, Inc.; Lupus Foundation of America; and Black Women’s Health Imperative, as well as ICER. We suggest that meaningful, patient-centered engagement, where patient communities are systematically integrated throughout the review, can be a way of returning to the discipline’s roots focusing on technologies’ societal and ethical impact. It is a process that requires robust commitment from all involved but produces assessments relevant to those directly affected by them.

Objectives: The aim of this study was to predict the potential impact of the introduction of implantation of polyurethane scaffold for the treatment of partial meniscal lesions in the South Korean healthcare system.

Methods: The horizon scanning process was used to select a target technology and assess its potential impacts on patients and the Korean healthcare system. We identified and filtered research-phase health technologies that are not listed yet in Korean, but appear promising. After a process of prioritization, we chose the implantation of polyurethane scaffolds as a target technology. Then, through the procedures of assessment and peer review, we analyzed current evidence and its predicted potential impacts.

Results: There were eight studies included in the review: one prospective cohort and seven case-series studies. Six revealed significant improvements in function and pain relief. Of the six studies, which reported safety endpoints, four stated no major postoperative complications related to scaffold, and two reported adverse events and serious adverse events such as pain, joint swelling, et cetera. We also included the potential impact of this technology based on the experts’ consultation. They all agreed that it would satisfy the diverse needs of patients and fulfill clinical needs. However, the majority of related clinical studies were based on short-term follow-up observations without any validation process involving comparison with control groups.

Conclusions: Through a horizon scanning activity, we found that the implantation of polyurethane scaffolds is a promising technology to resolve articular cartilage defects; however, long-term evidence with comparison groups for safety and effectiveness is required.

Objectives: The aim of this study was to implement health technology assessment (HA) in the First General City Hospital in Astana, Kazakhstan.

Methods: We organized trainings to familiarize hospital staff with the purpose and details of HTA. An HTA committee was established, with representation from hospital physicians and managers, and criteria for prioritization of health technologies determined. Clinical departments of the hospital were asked to prepare applications for new technologies for their services.

Results: The HTA committee reviewed five applications and selected a technology from one of these, on single incision laparoscopic surgery (SILS), for assessment. A short HTA report on SILS was prepared, covering its safety, clinical effectiveness, and cost effectiveness. The report was used to support a request to the Department of Health for additional funding to implement this technology within the hospital. This funding was approved and SILS was established in several hospital departments.

Conclusions: This successful initial experience with HTA has paved the way for its routine use by the hospital for informing decisions on the procurement and use of new health technologies.

A National Patient Library, a public/private partnership, is proposed to identify and vet information for consumers. Library staff will help tailor general information to consumers' specific needs. The library includes an electronic network. The principal focus of the library's information is controversial and experimental procedures, Pharmaceuticals, and medical devices.

Objective: Our aim was to assess the impact of six recommendations regarding drug prescription on the clinical practices of French psychiatrists. The recommendations were part of the conclusions of a consensus conference entitled “Long-term therapy of schizophrenia” (Paris, January 1994).

Methods: The impact of the conference was assessed on the basis of awareness of the existence of the conference, knowledge of its conclusions, and actual changes in clinical practice. We performed: a) a survey of a representative sample of 396 psychiatrists 2 years after the conference; and b) an analysis of changes in drug prescriptions in a cohort of 2,407 patients with schizophrenia under treatment at the time of the conference.

Results: Overall, 78% of interviewed psychiatrists were aware of the existence of the conference and 70% of its conclusions. Declared prescription practices conformed with conference conclusions about 60% (10%–95%) of the time. No difference in practices was noted between psychiatrists who were aware of the recommendations and those who were not. Single neuroleptic prescriptions increased in the cohort study in line with the main conference recommendation. The increase was small, but significant from 51.1% to 56.4%, and mainly concerned patients recently put on treatment. Contrary to recommendations, prescriptions of anticholinergics plus neuroleptics inexplicably rose from 48.2% to 54.3%.

Conclusion: Small changes in prescription habits occurred in the wake of the consensus conference, but we cannot really ascribe them to a direct impact of the conference. Despite the great pains we took in disseminating the conclusions of the conference as widely as possible, it is clear that a more forceful action plan (e.g., including continuous medical education) is required.

Objectives: The purpose of the European network for Health Technology Assessment (EUnetHTA) is to make HTA agencies collaborate sharing methods and tools thus avoiding duplication of evaluative efforts and allowing resource savings. From 2010 to 2012, the activities of the network were carried out through EUnetHTA Joint Action 1 and Work Package 4 Strand B aimed at producing two Core HTAs with two main objectives: to test the Web based Core model and the collaborative working models. Our objective in this article is to give an historical record of the Work Package activities highlighting what worked and what did not in the collaboration of researchers’ groups coming from different agencies.

Methods: A retrospective description of all the steps for the joint production of the two Core HTAs is provided starting from the first step of selecting technologies of common interest. Primary researchers’ views on the whole process have been collected through a semi-structured telephonic interview supported by a questionnaire. Coordinators views were gathered during internal meetings and validated.

Results: Majority of respondents thought topic selection procedure was not clear and well managed. About collaborative models, small groups were seen to enable more exchange, whatever the model. According to coordinators, loss of expertise and experience during the production process, different languages, and novelty of the Online Tool were main barriers.

Conclusions: Lessons learned from this first experience in Joint Action 1 paved the path for the collaboration in Joint Action 2, as it allowed enhancements and changes in models of collaborations and coordination.

Objectives: The aim of this study was to determine the aspects of expert advice that decision makers find most useful in the development of evidence-based guidance and to identify the characteristics of experts providing the most useful advice.

Methods: First, semi-structured interviews were conducted with seventeen members of the Interventional Procedures Advisory Committee of the UK's National Institute of Health and Care Excellence. Interviews examined the usefulness of expert advice during guidance development. Transcripts were analyzed inductively to identify themes. Second, data were extracted from 211 experts’ questionnaires for forty-one consecutive procedures. Usefulness of advice was scored using an index developed through the qualitative work. Associations between usefulness score and characteristics of the expert advisor were investigated using univariate and multivariate analyses.

Results: Expert opinion was seen as a valued complement to empirical evidence, providing context and tacit knowledge unavailable in published literature, but helpful for interpreting it. Interviewees also valued advice on the training and experience required to perform a procedure, on patient selection criteria and the place of a procedure within a clinical management pathway. Limitations of bias in expert opinion were widely acknowledged and skepticism expressed regarding the anecdotal nature of advice on safety or efficacy outcomes. Quantitative analysis demonstrated that the most useful advice was given by clinical experts with direct personal experience of the procedure, particularly research experience.

Conclusions: Evidence-based guidance production is often characterized as a rational, pipeline process. This ignores the valuable role that expert opinion plays in guidance development, complementing and supporting the interpretation of empirical data.

In 1958 Joseph Murray and associates reported the first successful kidney transplantation between two identical twins at Peter Bent Brigham Hospital in Boston (14). Since then organ transplantation has advanced rapidly. Renal transplantation is now a standard treatment for irreversible renal failure, and liver transplantation is an accepted method for treating end-stage liver disease. A number of patients have survived more than 20 years after renal transplantation and more than 10 years after liver transplantation (20). Very few liver transplantations have been performed in Sweden but this method is expected to become more common in the near future.

The assessment of value within healthcare is undergoing a major transformation. Gone are the days when a new mechanism of action alone would be regarded as a high value innovation. Today, there is much more of an emphasis on what are the outcomes (i.e., mortality and morbidity benefits), how does this compare with the current standard of care and what is the impact to the usage of healthcare resources. This emphasis is quite understandable given the financial crisis we are experiencing with an ever growing and aging population that is placing considerable strain on the healthcare system.

The Austrian healthcare system relies mainly on physicians in private practice and on various services provided by hospitals. The social health insurance scheme is compulsory, covering 99% of the population. The system is very decentralized. While the federal state provides the framework, the nine autonomous provinces are responsible for administering health and social services. There is ongoing public discussion about centralizing the healthcare system to make it more efficient and to enforce structural reforms. Because of concerns about healthcare expenditures, in 1997 the Performance-Related Hospital Financing System (LKF), a system similar to the diagnosis-related group system, was introduced for hospitals, including a plan for large medical devices. It is too early to evaluate the success of this new system, although some effects of the LKF system that could have been anticipated, such as shortened lengths of stay and more hospitalizations, have been seen. Previously, health technologies have been almost uncontrolled in Austria. The evaluation of health technologies as an instrument to support or to control their dissemination and use or to help define policies is not institutionalized or systematically used. It seems clear that structural reforms of the Austrian healthcare system are needed. Health technology assessment should be part of such reforms.

Objectives: As health care budgets continue to face close scrutiny, any new acquisition must be evaluated for both costs and outcomes. This study was undertaken to demonstrate the application of an economic framework for the evaluation of a multileaf collimator as an example of a new technology that can have both quantifiable and nonquantifiable benefits for patients, staff, and cancer care institutions.

Methods: Using financial data from the Northeastern Ontario Regional Cancer Centre (NEORCC) and a recognized staffing model, a commercial spreadsheet, developed to economically characterize the principal radiotherapy processes has been used to determine the net incremental annual cost of a multileaf collimator (MLC).

Results: The incremental annual cost of purchasing an MLC is estimated at approximately $85,000 (1997 CDN $). Without increasing patient throughput, this increases the average cost of a course of radiotherapy by approximately CDN $200. Savings can be accrued by decreasing mold room activity, increasing the hourly patient capacity on each treatment machine, and decreasing sick time due to strain injuries.

Conclusions: Although the clinical outcome of techniques facilitated by MLCs, such as intensity-modulated radiation therapy, are unknown at this time, an economic context within which to objectively evaluate this technology is presented. The framework presented suggests a method of quantifying outcome-justified expenditures, such as improved patient outcome and greater treatment flexibility, which, when offset against the incremental annual equipment cost, may be used to help justify the acquisition of multileaf technology.

Target Product Profiles (TPPs) outline the characteristics that new health technologies require to address an unmet clinical need. To date, published TPPs for medical tests have focused on infectious diseases, mostly in the context of low- and middle-income countries. Recently, there have been calls for a broader use of TPPs as a mechanism to ensure that diagnostic innovation is aligned with clinical needs, yet the methodology underpinning TPP development remains suboptimal. Here, we propose that early economic evaluation (EEE) should be integrated within the TPP methodology to create a more rigorous framework for the development of “fit-for-purpose” tests. We discuss the potential benefits that EEE could bring to the core activities underpinning TPP development—scoping, drafting, consensus building, and updating—and argue that using EEE to help inform TPPs provides a more objective, evidence-based, and transparent approach to defining test specifications.

Biofeedback instrumentation has been a growing part of physical therapy practice for 20 years, and physical therapists have contributed to researching its efficacy in treating varying conditions. The application of biofeedback to the treatment of stroke was marked by some early difficulties with the quality of the electronics, but sophisticated contemporary equipment can do much more than was originally envisioned. The importance of relating quantified movement-based data to functional measures is projected as a high priority if appropriate reimbursement for physical therapy services with biofeedback is to continue in the future.

“Key principles for the improved conduct of health technology assessment for resource allocation decisions.” The title says it all. I am amazed that such a summary is even possible and that it defines this field so well. Perhaps this report will be cited for years to come as the best and central definition of HTA. A field like this can be defined in other ways.

Objectives: The object of this study was to determine, taking into account uncertainty on cost and outcome parameters, the cost-effectiveness of high-dose chemotherapy (HDC) compared with conventional chemotherapy for advanced breast cancer patients.

Methods: An analysis was conducted for 300 patients included in a randomized clinical trial designed to evaluate the benefits, in terms of disease-free survival and overall survival, of adding a single course of HDC to a four-cycle conventional-dose chemotherapy for breast cancer patients with axillary lymph node invasion. Costs were estimated from a detailed observation of physical quantities consumed, and the Kaplan–Meier method was used to evaluate mean survival times. Incremental cost-effectiveness ratios were evaluated successively considering disease-free survival and overall survival outcomes. Handling of uncertainty consisted in construction of confidence intervals for these ratios, using the truncated Fieller method.

Results: The cost per disease-free life year gained was evaluated at 13,074€, a value that seems to be acceptable to society. However, handling uncertainty shows that the upper bound of the confidence interval is around 38,000€, which is nearly three times higher. Moreover, as no difference was demonstrated in overall survival between treatments, cost-effectiveness analysis, that is a cost minimization, indicated that the intensive treatment is a dominated strategy involving an extra cost of 7,400€, for no added benefit.

Conclusions: Adding a single course of HDC led to a clinical benefit in terms of disease-free survival for an additional cost that seems to be acceptable, considering the point estimate of the ratio. However, handling uncertainty indicates a maximum ratio for which conclusions have to be discussed.

We studied the role of economic incentives for private providers in explaining Spain's disproportionately large number of extracorporeal shock wave lithotripters (ESWLs) and low number of linear accelerators (linacs) per million population (pmp) in comparison to other countries of the Organization for Economic Cooperation and Development (OECD). We found that the reimbursement policy for 1990 allowed an average profit per private ESWL of $732,000, but no profit for linacs. Regional per-capita income was positively correlated with ESWLs pmp in Spain (R2 = 0.49; p =.001), but not with linacs. Sixty-nine percent of ESWLs were privately held versus only 16% of linacs. To avoid these types of distortions, financial incentives should be based on a reasonable relationship between cost and charges and should be associated with the appropriateness of medical care.

Rationale: Bayesian methods provide an interesting approach to assessing an implantable medical device (IMD) that has evolved through successive versions because they allow for explicit incorporation of prior knowledge into the analysis. However, the literature is sparse on the feasibility and reliability of elicitation in cases where expert beliefs are used to form priors.

Objectives: To develop an Internet-based method for eliciting experts’ beliefs about the success rate of an intracranial stenting procedure and to assess their impact on the estimated benefit of the latest version.

Study Design and Setting: The elicitation questionnaire was administered to a group of nineteen experts. Elicited experts’ beliefs were used to inform the prior distributions of a Bayesian hierarchical meta-analysis model, allowing for the estimation of the success rate of each version. RESULTS: Experts believed that the success rate of the latest version was slightly higher than that of the previous one (median: 80.8 percent versus 75.9 percent). When using noninformative priors in the model, the latest version was found to have a lower success rate (median: 83.1 percent versus 86.0 percent), while no difference between the two versions was detected with informative priors (median: 85.3 percent versus 85.6 percent).

Conclusions: We proposed a practical method to elicit experts’ beliefs on the success rates of successive IMD versions and to explicitly combine all available evidence in the evaluation of the latest one. Our results suggest that the experts were overoptimistic about this last version. Nevertheless, the proposed method should be simplified and assessed in larger, representative samples.