Objectives: Within French university hospitals, some internal committees are in charge of conducting hospital-based health technology assessment (Hb-HTA) to support managerial decisions regarding the adoption of innovations. For manufacturers, hospitals are usually the entry point for new and innovative medical devices, which cannot be accessed without the Hb-HTA committees' approval. Thus, the main objective of this pilot survey was to explore manufacturers’ insights into Hb-HTA processes.

Methods: A two-step pilot survey was conducted in 2014. First, semi-structured phone interviews were carried out to capture manufacturers' feedback on the Hb-HTA procedure. Second, a prospective and iterative questionnaire designed to explore manufacturers’ market access strategies was administered.

Results: Eight manufacturers from the medical device industry completed the retrospective phone interviews, and five of them participated in the prospective survey. According to the overall feedback, the Hb-HTA process timeline and transparency are major issues, and the expectations of internal committees, especially in terms of clinical evidence, remain difficult to understand. However, despite this and due to the complexity of reimbursement processes at the national level, manufacturers are increasingly considering hospital adoption through Hb-HTA submission as a viable market access and coverage opportunity.

Conclusions: Our study reaffirms the primary role of hospitals in the diffusion of innovative medical devices. However, to ensure efficient and broad access to innovation, cooperation between local and national HTA bodies is critical and should be promoted.

Objectives: The aim of this study was to elucidate, whether shift from first generation antipsychotics (FGA) to olanzapine can affect health-related quality of life (HRQoL) of residually symptomatic schizophrenic outpatients.

Methods: Patients were randomized to either olanzapine or to continuation on their FGA. The 15D-measured HRQoL at baseline and end-point (after 12 weeks) was compared.

Results: Patients (n = 21) randomized to olanzapine achieved better HRQoL than those (n = 21) who continued on their FGA. This difference on the 15D (0.048 on a 0–1 scale; p = .037) was clinically important and comparable to that resulting from common surgical interventions, for example, hip or knee replacement.

Conclusions: HRQoL of stable outpatients with residual symptoms or adverse effects may improve substantially after shift from FGAs to olanzapine.

Objectives: The aim of this study was to describe 8-year results from post-implementation surveillance of neuroreflexotherapy (NRT), a health technology proven effective for treating neck and back pain.

Methods: Post-implementation surveillance included all patients undergoing NRT across five regions within the Spanish National Health Service (SNHS). Validated methods were used to assess pain, disability, adverse events, use of health resources, and patient satisfaction. Logistic regression models were developed to identify the variables associated with the risk of a pain episode requiring more than one NRT intervention. The number of relapses among discharged patients during the 8-year period was calculated.

Results: Between January 1, 2004, and June 30, 2012, 9,023 patients (median age: 53 years), presenting 11,384 subacute (25.2 percent) and chronic (74.8 percent), neck or back pain episodes, were discharged after receiving NRT. Spinal pain improved in 89 percent of cases, 83 percent abandoned drugs, and 0.02 percent required spine surgery. The only adverse event was skin discomfort (8.0 percent of patients). Number of patient complaints was 0, and answers to a standardized questionnaire reflected a high degree of satisfaction (response rate: 76.7 percent). Of the pain episodes, 18.9 percent required more than one NRT intervention; logistic regression models identified the variables associated with this. Over the 8-year period, the proportion of discharged patients referred for treatment due to relapse at the same level for neck, thoracic, and low back pain, was 16.4 percent, 6.5 percent, and 14.5 percent respectively.

Conclusions: Post-marketing surveillance for a non-pharmacological technology is feasible within the SNHS. These results support generalizing NRT across the entire SNHS under the current validated application conditions.

Objectives: The aim of this study was to describe the evolution of a cost-utility model used to inform the UK National Institute for Health and Clinical Excellence's (NICE) most recent decisions on the cost-utility of drug treatments for Alzheimer's disease (AD), and to explore the impact of structural assumptions on the cost-utility results.

Methods: Changes informed by noted limitations of the decision model used in NICE's previous decisions (in 2006) were made cumulatively to the original decision model for donepezil compared with best supportive care (for patients with mild to moderate AD). Deterministic and probabilistic analyses were undertaken for each cumulative change of the model. The expected value of perfect information (EVPI) of parameter estimates and structural assumptions was also calculated.

Results: Cumulative changes to the decision model highlighted how the results of the original model (incremental cost-effectiveness ratio of £81,000 per quality-adjusted life-year gained) related to those of the new model (where donepezil was estimated to be cost-saving), mainly due to uncertainty in the incremental cost of donepezil treatment over best supportive care (ranging from -£600 to £3,000 per patient). The partial EVPI analysis reflected this finding where further information on treatment discontinuations and cost parameter estimates were shown to be valuable in terms of reducing decision uncertainty.

Conclusions: Assessing the evolution of the cost-utility model helped to identify and explore structural differences between cohort-based models and is likely to be useful for decision models in other disease areas. This approach makes the structural uncertainty explicit, forcing decision makers to address structural uncertainty in addition to parameter uncertainty.

This paper examines the extent to which it is possible to measure the social importance, or macro benefits, of health care. In contrast to the many micro studies of the benefits of specific health care interventions, methodology relating to such macro benefits is at a very rudimentary stage. A theoretical model is presented that seeks to capture the social consequences of a health care system. In light of this model, three existing empirical approaches to answering the question are examined: the inventory approach, the avoidable mortality approach, and the production function approach. All three have severe limitations in terms of the underlying theoretical model, data availability, and analytic tools employed. A more fruitful approach may be to investigate the value of undertaking a direct survey of citizens' attitudes toward their health care system.

Objectives: The aim of this study was to assess whether publications of importance for improving the health system and its technologies are highly cited intrascientifically.

Methods: Bibliometric assessment of the 596 publications used as sources in the fifty SBU Alerts from 2001to 2004 from the Swedish Council on Technology Assessment in Health Care was carried out using the Thomson Scientific citation indexes. Normalized citation scores were calculated for all included studies. Additional factors such as the time and place of the research, subject categories, and journal source were analyzed.

Results: On average, the sources in SBU Alert have been cited eight times more than the world average consistently during the time period and across research areas. Articles used as its scientific basis are often published in a few, high impact, general medical journals. However, many of the articles are published in field-specific journals with low impact factors. Most articles used in SBU Alert are published by authors based in the United States or the United Kingdom. However, Swedish, Danish, and Dutch publications are overrepresented in its science base, whereas Japanese, Taiwanese, Indian, and Russian publications are underrepresented.

Conclusions: Publications used as sources in a Swedish system for identification and early assessment of new methods in health care are also highly cited within the scientific community. This finding increases the appropriateness of using bibliometric indicators in evaluations of clinical research and suggests that decision makers through SBU Alert are getting scientifically sound advice.

Objectives: Since 1997, members of the International Network of Agencies for Health Technology Assessment (INAHTA) have collaborated on a Joint Project to track the diffusion, evaluation, and clinical policy of positron emission tomography (PET). Part 2 of this updated Joint Project report summarizes HTA-based strategies for directing the clinical use of PET and a discussion on the value of HTA in managing the diffusion of high cost diagnostic technologies, which were presented at an INAHTA-sponsored workshop at the Health Technology Assessment International Annual Meeting in 2004 on strategies for managing high cost diagnostic technologies.

Methods: A summary of the workshop proceedings is presented.

Conclusions: Sharing assessment work, universal agreement in assessment conclusions, stakeholder input, and modeling techniques help manage the uncertainty in the evidence base while targeting clinical use of PET toward the most promising indications. Emphasis on HTA findings, linkage between financing of clinical PET and outcome evaluation, and targeted dissemination of scientific findings empower providers to reduce unnecessary utilization and contain costs within a quality improvement framework. Above all, a trustworthy source of HTA information and a process that is conducive to using scientific evidence as the basis for decision making are essential for managing the diffusion of complex and costly diagnostic technologies in patient care.

Objectives: The aim of this study was to describe an initial exploration by CADTH, Canada's pan-Canadian health technology assessment (HTA) agency, in using the INTEGRATE-HTA guidance in the production of an HTA that examined the use of both in-center and in-home dialysis modalities for the treatment of end-stage kidney disease in adults in Canada.

Methods and Results: We outline CADTH's standard HTA production process and context and then describe the experience of the assessment team in using the INTEGRATE-HTA guidance, specifically to help structure and guide the use of a logic model, the identification of implementation issues, and the identification and examination of ethical issues. For each of the aspects, we describe and reflect on how the assessment team used the guidance, challenges that were encountered in its use, and whether and how we might address these challenges when using the INTEGRATE-HTA guidance in the future.

Conclusions: INTEGRATE-HTA provided detailed and helpful guidance for truly integrating wide-ranging aspects of HTA. Our agency was challenged by a steep learning curve for assessment team members, tight project timelines, and a misalignment of current HTA processes with those required to implement the guidance. Nevertheless, using the guidance initiated a dialogue about what might be needed to assess complex interventions and the potential process changes that could facilitate conducting more integrated assessments.

Background: In April of 2014, the Turkish Ministry of Health held the First Annual Health Technology Assessment (HTA) Meeting in Antalya.

Objectives: The objectives were to understand the perceptions of stakeholders regarding the current status of HTA and document their recommendations and strategies for promoting systematic use of HTA in Turkey.

Methods: The study was conducted using a qualitative written survey assessing current compliance with the fifteen HTA principles suggested by Drummond et al. (Key principles for the improved conduct of health technology assessments for resource allocation decision. Int J Technol Assess Health Care. 2008;24:244–258) and a qualitative method referred to as the Collective Intelligence Platform®.

Results: A total of 216 stakeholders representing academic, public, and the private health sector attended the annual meeting; 178 completed the survey and 183 participated in the Platform. Quantitative Results: Survey participants reported that, although Turkey does not currently fully comply with any of the fifteen HTA principles, there is some compliance with all of them. The overall average score for all fifteen principles was 3.04. Quantitivate Results: Participants recommended a more transparent, independent, and evidence-based policy decision-making system through better coordination of HTA activities, data aggregation, capacity development, and a national HTA core model and framework.

Conclusions: Platform participants described the current HTA environment as disjointed and lacking in resources and support from policy-making leaders. Despite the persisting challenges, awareness of the strengths and weaknesses of the current system combined with increasing interaction among Turkish stakeholders and the international HTA community can meaningfully contribute to the continued development and promotion of HTA in Turkey.

Objectives: In the Czech Republic, the health technology assessment (HTA) approaches have been implemented in evaluation of medicinal products since 2008.

The aim of this study was to provide an overview of the implementation of HTA and different levels thereof in the evaluation process conducted by the State Institute for Drug Control (SUKL) and to describe the impact of HTA on the entrance of new medicinal entities into out-patient healthcare system including highly innovative and orphan drugs.

Methods: Materials supporting this overview were collected using the records in the database of administrative proceedings of SUKL, in-house standard operating procedures, and the legislation in force. Based on these sources as well as the hands-on knowledge of the current practice, a brief description of the general rules of administrative proceedings involving HTA of varying complexity was elaborated. Characteristic features of the individual types of proceedings, basic differences in the complexity of HTA employed, and its most important challenges were summarized.

Results: In Czech Republic, HTA in the formal administrative proceedings ensures a transparent process of introduction of new medicinal products into clinical practice and leaves space for restriction of reimbursement conditions to minimize budget impact.

Conclusions: As a robust as well as pragmatic HTA methodology has been implemented by SUKL, relevant stakeholders (marketing authorization holders, Health Care Funds, clinical expert groups) are now able to influence reimbursement of new technologies.

The objective of the study was to ascertain the extent of utilization of medical technologies (defined as equipment) used in various health care institutions in Nigeria. The study also investigated the factors accounting for use of and under- and nonutilization of this equipment. Sixteen major and minor health care institutions were surveyed. The outcomes of the study indicate that not less than a third of the equipment was out of order at the time of the study. A number of factors are responsible for nonutilization of this equipment. They include lack of infrastructural facilities such as piped water, storage, and power supply; poor handling by badly trained staff; and the low priority that is accorded to maintenance. The study leads to the important conclusion that the local culture is unable to sustain the technologies that are procured.

The U.S. Food and Drug Administration's (FDA) approval of the orphan biological product recombinant erythropoietin (rEPO) in June 1989 resulted both in a breakthrough treatment for the chronic anemia of people who suffer from chronic renal failure and a powerful argument for change in the legislation that spawned its development: the Orphan Drug Act of 1983. At a cost of over $6,000 per patient per year, Congress could not understand how a product that no manufacturer wanted to produce was suddenly costing the federal government hundreds of millions of dollars each year. Congress attempted to change the act in 1990 to preclude a manufacturer from using its provisions to secure lucrative monopolies in certain drug markets. In early 1991, the FDA finally issued regulations to implement the act that addressed some of the very concerns that were caused by rEPO.

The benefit for patients with operable breast cancer treated with adjuvant systemic therapy is small, if reduction of early mortality within the context of randomized control trials is used for treatment comparison. One might consider that the 75%–85% of patients who die despite treatment are overtreated, as are patients who remain alive even without therapy within a given time frame. Larger treatment benefits in terms of avoided or delayed breast cancer relapse have been demonstrated even at early phases of follow-up in the vast majority of adjuvant trials. Exposure of all patients to adjuvant therapy at a time at which no symptoms of disease are present is detrimental in terms of quality of life.

Based on our assumption that the quality of life of the patient is typically altered both by subjective toxic effects of adjuvant treatment and by the appearance of relapse, we developed a method of comparing treatment effects in terms of time without symptoms of disease and toxicity of treatment (TWiST). Because the impact of treatment on relapse rates appears earlier than survival effects in all adjuvant therapy trials, and because the value of time without relapse in terms of the quality of life of the patients is as yet poorly defined, we have generalized our method of comparing treatment attitudes to include individual qualitative judgment values. The experience gained from integrating quality-of-life issues into clinical trials for breast cancer might also be applied to other diseases characterized by a chronic course, toxic treatments, and gains in periods of relative or absolute freedom from toxic effects and progressive disease.

Outcomes measurement will be key to the appropriate use of Pharmaceuticals as health care costs continue to rise. The generation of data showing the clinical, economic, and humanistic outcomes of pharmaceutical care will be essential to avoid decision making based solely on cost and to ensure that cost-effective, high-quality health care remains the health care system's top priority.

An evaluation was conducted of the approaches, costs, and quality-of-life outcomes associated with communication aid assessment programs for the speech-impaired provided by specialist Communication Aids Centres (CACs) in the United Kingdom. The average costs of CAC assessment programs was £410 per client, which is not excessive. There was evidence of moderate quality-of-life benefits from clients' use of the recommended aids.

Bone marrow transplantation has become an accepted treatment for severe aplastic anemia, acute myelogenous leukemia, and acute lymphocytic leukemia (20;35). Less commonly thalassemia (23), multiple myeloma (25), severe combined immune deficiencies (10), and chronic granulocytic leukemia (14) are also treated with marrow transplantation. Most donors are siblings whose HLA phenotype is identical to the patient's. Such donors, however, are available to only one third of patients (6;16;27;28;35). In the absence of an HLA-identical sibling, partially matched, haplo-identical, related donors (differing by only one major histocom-patibility antigen) can be found for another 7% of patients (1;6;27;37). A variety of lower quality matches also can be found but have not been used extensively. Advances in tissue typing and the treatment of graft versus host disease now allow physicians to consider matched unrelated individuals as potential donors when a suitable related donor is not available (11;12;13;15;17;22).