The purpose of this paper is to outline some of the key methodologic issues in designing clinical trials to assess the cost-effectiveness of clinical magnetic resonance imaging (MRI). Because the possible objectives of such trials are so diverse, and the appropriate methodologies correspondingly numerous, no attempt is made to identify a single objective or methodology as appropriate. Rather, this paper should be read as an “options paper,” in which alternative formulations of objectives, outcome measures, study design, and protocol instrumentation are reviewed, with critical discussion of their theoretical strengths and weaknesses, as well as their practical implementability.

Objectives: This study examined users' perceptions of the role and value of the Scottish Health Purchasing Information Centre (SHPIC) from 1995 to 1998.

Methods: Questionnaires and interviews were used to gather data from Scottish GP fundholders and health board managers at two stages.

Results: Initially, purchasers sought help in identifying the most relevant information. By 1997, while some appreciated the clinical and cost-effectiveness information produced, others were critical of lack of timely production and apparent lack of cooperation between agencies.

Conclusions: New U.K. agencies can learn from SHPIC's problems, specifically in producing clear, coordinated, timely, independent, and well-marketed information with implementation strategies.

Policy Implications: It is important to evaluate the impact of agencies to produce and disseminate evidence-based information, even if this has to be done pragmatically rather than as an outcomes-based assessment. Evidence from evaluations can inform direction and strategy for existing and new agencies.

Background: Non small cell lung cancer (NSCLC) diagnosis and treatment is a highly complex process, requiring managerial skills merged with clinical knowledge and experience. Integrated care pathways (ICPs) might be a good strategy to overview and improve patient's management. The aim of this study was to review the ICPs of NSCLC patients in a University Hospital and to identify areas of quality improvement.

Materials and Methods: The electronic medical records of 169 NSCLC patients visited at the University Hospital were retrospectively reviewed. Quality of care (QoC) has been measured trough fifteen indicators, selected according main international Guidelines and approved by the multi-disciplinary team for thoracic malignancies. Results have been compared with those of a similar retrospective study conducted at the same hospital in 2008.

Results: A total of 146 patients were considered eligible. Eight of fifteen indicators were not in line with the benchmarks. We compared the results obtained in the two separate periods. Moreover, we process some proposal to be discussed with the general management of the hospital, aimed to redesign NSCLC care pathways.

Conclusions: ICPs confirm to be feasible and to be an effective tool in real life. The periodic measurement of QoC indicators is necessary to ensure clinical governance of patients pathways.

Some studies suggest that breast-conserving treatment increases the cost of breast cancer. Estimates from a social perspective show that incremental cost depends on the proportion of lumpectomies done in outpatient facilities and on the demand for reconstructive surgery. Conservative treatment reduces spending within a reasonable range of values.

This article seeks to comment on the approaches to health technology assessment (HTA) outlined in the four main country studies in this volume. It is written from the perspective of a general practitioner working in an inner city area in the United Kingdom and argues that, from the point of view of the clinician, HTA delivers considerably less than it promises. The problems center on the inevitability of judgment by both politicians and clinicians and the conflicting foundations of these judgments. Within political decision-making, the needs of the population inevitably outweigh the needs of the individual; within clinical decision making, the opposite is the case. Attempting a scientific rationality, HTA struggles with the impossibility of holding the balance between the two. These difficulties are further compounded by the implications of ever-increasing expectations of perfect health and the effects of multinational commercial pressures.

Adequate methods are urgently needed to guarantee the good practice of health technology assessment (HTA) for technologies with novel properties. The aim of the study was to construct a conceptual framework to help understand the innovation of HTA methods (IHTAM). The construction of the IHTAM framework was based on two scoping reviews, one on the current practice of innovating methods, that is existing HTA frameworks, and one on theoretical foundations for innovating methods outside the HTA discipline. Both aimed to identify and synthesize concepts of innovation (i.e., innovation processes and roles of stakeholders in innovation). Using these concepts, the framework was developed in iterative brainstorming sessions and subsequent discussions with representatives from various stakeholder groups. The framework was constructed based on twenty documents on innovating HTA frameworks and fourteen guidelines from three scientific disciplines. It includes a generic innovation process consisting of three phases (“Identification,” “Development,” and “Implementation”) and nine subphases. In the framework, three roles that HTA stakeholders can play in innovation (“Developers,” “Practitioners,” and “Beneficiaries”) are defined, and a process on how the stakeholders innovate HTA methods is included. The IHTAM framework visualizes systematically which elements and stakeholders are important to the development and implementation of novel HTA methods. The framework could be used by all stakeholders involved in HTA innovation to learn how to engage dynamically and collaborate effectively throughout the innovation process. HTA stakeholders in practice have welcomed the framework, though additional testing of its applicability and acceptance is essential.

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.

Objectives: The potential of routine data for health technology assessment (HTA) in the United Kingdom was assessed.

Methods: Compiled were a comprehensive list of routine databases, their classification according to data characteristics, literature review on their current use, and their comparison with key topics identified as priorities for HTA.

Results: Two hundred seventy health-care databases for England or the English regions were identified. Twenty-four included data on both health technology and patient health state. Eleven found some published use in effectiveness evaluation. Of 140 prioritized health technologies, only 22 could be identified in routine databases.

Conclusions: Routine data are plentiful but of limited use in HTA. The data sets usually do not include the effect of treatments. Coding is inadequate, and confidentiality regulations will make matters worse. Both need urgent attention.

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.

This paper indicates that certain economic evaluation methods (cost-effectiveness and cost-utility analyses) may yield inconsistent results. Along with the lack of formal grounding of these methods in economic “first principles,” this finding suggests the possible benefit of greater reliance on the more formally developed method of cost-benefit analysis.

The physical phenomenon of nuclear magnetic resonance (NMR) was first characterized almost forty years ago in 1946 by the simultaneous but independent experimental successes of American scientists Felix Bloch and Edward Purcell. Their discoveries prompted development of conventional NMR spectroscopy. a technique used to describe the molecular composition and behavior of chemical compounds. Twenty-five years later, in 1971, Damadian used NMR to demonstrate differences in the behavior of water in malignant and benign tissues, and he suggested that NMR possessed “many of the desirable features of an external probe for the detection of internal cancer” (7). In the same year, Lauterbur produced the first two-dimensional NMR image, a cross-sectional portrait of two tubes of water (25). The potential utility of this technique to medical imaging was obvious, and soon afterwards multiple researchers began development of clinical NMR imaging systems. The first human whole-body NMR scan was accomplished by 1977. Improvements in the scanning process and image quality continue with, as yet, no limits in sight. In this clinical context, NMR techniques have experienced a name change to the current prevailing appellation, magnetic resonance imaging (MRI).

This article argues that while health care and government authorities in the Netherlands in the last decades have attempted to rationalize the diffusion of medical technology, much work remains to be done. The authors contend that though a mix of direct government regulation and economic incentives will be needed, regulation by incentive is more effective than regulation by directive in achieving cost effective use of technology. Generally, bureaucratic structures provide only limited opportunities for tight control of the diffusion of medical technologies. The article offers possible future strategies for directing the adoption and use of medical technologies and stresses the importance of reliable infor mation culled from comprehensive technology assessments.

This review concludes that: a) the global burden of disease from non-insulin-dependent diabetes mellitus (NIDDM) cannot be completely estimated at present; b) evidence for the efficacy of key elements of a package of care is still needed; c) generalizing the results of evaluations of costs or effectiveness across different populations is not straightforward; and d) for this complex intervention, the costs and effectiveness of intervention may be highly dependent on methods of organizing care. Addressing this information deficit represents an important task for researchers and health decision makers.

Background: There is a need for innovative methodologies to identify and prioritize topics for health technology assessment (HTA). A pilot project to evaluate the methodology for specialty mapping was undertaken in the area of child and adolescent health. Two case studies are presented, in the area of sexually transmitted infections and acute pain.

Methods: The methodology comprised sequential stages, based on principles of systematic review. A “stakeholder model” encouraged wider participation. Key stages included identifying the topic area and setting the scope; developing a care pathway; searching for clinical guidelines/guidance, and evaluation literature; synthesis and mapping of literature to the “nodes” of the care pathway to highlight gaps; prioritizing the topics with stakeholders; and referring priorities to the appropriate agencies.

Results: A total of thirty guidelines/guidance documents and sixteen evaluation studies were mapped across the two case studies. In some nodes of the care pathway, more literature was mapped than others, suggesting important gaps in research and policy guidance. Sixty-two policy questions were identified and were rated by stakeholders in prioritization workshops. The highest priorities have been considered by senior committees for likely commissioning as research or guidelines/guidance.

Conclusions: This is one of the few published examples of innovative methodology to identify and prioritize topics for HTA. Specialty mapping can make a positive contribution to the policy agenda, with several research and policy gaps being fed into existing prioritization channels. Adequate time, resources, and capacity is required particularly in engaging stakeholders and developing a care pathway. Implementation of specialty mapping in other topic areas with on-going evaluation is recommended.

Objectives: Implantable cardioverter defibrillators (ICDs) are an effective but expensive treatment for the prevention of sudden cardiac deaths in patients with severe left-ventricular dysfunction. Recent studies suggest that microvolt T-wave alternans (MTWA) predicts mortality and severe arrhythmic events in this population. However, the impact of MTWA on ICD cost-effectiveness is unknown.

Methods: A Markov decision-analysis model evaluated three treatment strategies for primary prevention in patients with severe left-ventricular dysfunction: (i) medical therapy for all; (ii) ICD therapy for all; and (iii) selective ICD therapy based on non-negative (positive or indeterminate) MTWA test results. Incremental cost-effectiveness ratios (ICER) were calculated from the perspective of a third party payer using a 10-year time horizon. Sensitivity analyses examined the robustness of the estimates.

Results: A treatment strategy involving ICD therapy in all patients was associated with an ICER of $121,800/quality-adjusted life-year (QALY) compared with medical therapy, whereas a treatment strategy involving the selective use of ICDs based on MTWA test results was associated with an ICER of $108,900/QALY compared with medical therapy. Sensitivity analyses suggest that, under most scenarios, the selective use of ICDs based on MTWA results does not decrease the ICER to below $100,000/QALY.

Conclusion: MTWA only marginally improves the cost-effectiveness of ICDs for primary prevention in patients with severe left-ventricular dysfunction. There remains a need for improved means to effectively identify which patients will derive the greatest benefit from ICD implantation.