I have worked in health technology assessment (HTA) since 1975, beginning in the United States Congress Office of Technology Assessment (OTA), where we were charged with defining “medical technology assessment”. My main concern in HTA has always been efficacy of healthcare interventions. After years in OTA, I was invited to the Netherlands in 1985, where the Dutch government invited me to head a special commission concerning future healthcare technology and HTA. From there, I became involved in over forty countries, beginning in Europe and then throughout the world. My most intense involvements, outside the United States and Europe, have been in Brazil, China, and Malaysia. During these 40-plus years, I have seen HTA grow from its earliest beginnings to a worldwide force for better health care for everyone. I have also had some growing concerns, outlined in this Perspective article. Within HTA, I am most disappointed by a narrow perspective of cost-effective analysis, which tends to ignore considerations of culture, society, ethics, and organizational and legal issues. In the general environment affecting HTA and health care, I am most concerned about the need to protect the independence of HTA activities from influences of the healthcare industries.

Purpose: The aim of this study was to identify and analyze the characteristics of leadership and management associated with a successful Lean thinking adaptation in healthcare.

Design: A systematic literature review was undertaken using electronic databases: PubMed, PubMed Systematic Review, ABI/INFORM, Business Source Complete, Emerald, JBI, and Cinahl. Inclusion criteria were: (i) a description of Lean management or leadership in health care, (ii) a reference to Lean thinking, (iii) a peer-reviewed original research article or a literature review, and (iv) a full text article available in English. Among the 1,754 peer-reviewed articles identified, nine original articles and three systematic reviews met the inclusion criteria. Data on informants, methods, and settings were extracted and collated. Content analysis was used to conduct a review of the nine original studies describing and analyzing the success factors of Lean adaptation. The characteristics of leadership and management were analyzed by using the concept of a managerial windshield that divides leadership and management into four ontological dimensions: activities, style, focus, and purpose, each with typical developmental stages of skills and capabilities. The current study has some limitations: some papers from the journals not indexed in the searched databases may have been overlooked and the literature searches were carried out only for a 5-year period.

Findings: Considering the results using the windshield concept emphasizes the philosophy, principles, and tools of Lean thinking. Lean leadership and management factors in health care were mainly conceptualized as skills and capabilities such as problem solving, making changes occur, empowering, communicating, coaching, supporting, facilitating, being democratic, organizational learning, and organizational success, all of which represented middle-stage or advanced managerial skills and capabilities.

Practical Implications: A conceptual analysis of systematically reviewed studies of Lean leadership and management point to certain traits as being typical when adapting Lean thinking to health care. The concept of a managerial windshield is useful when categorizing and analyzing essential managerial skills and capabilities for Lean implementation. Findings are beneficial when learning and educating the skills required for Lean transformation in healthcare organizations.

Objectives: Earlier treatment of publicly funded patients may achieve health gains that justify the additional costs of reducing waiting times. This study reports on the cost-effectiveness of implementing a private contracting model to meet alternative maximum waiting time targets for publicly funded patients undergoing total knee replacement surgery in Australia.

Methods: A linked decision tree and cohort Markov model was developed and populated and validated using secondary data sources to represent the pathways, costs, and quality adjusted life-years (QALYs) gained of non-urgent patients with alternative waiting times for total knee replacement surgery to a maximum age of 100 years.

Results: Assuming public waiting times are reduced through the purchase of private services, additional QALYs are gained at an incremental cost of less than $40,000. Value could be increased if lower private prices could be negotiated. Results are also sensitive to the rate of deterioration in function while waiting for surgery and the impact of functional status at the time of surgery on postsurgery outcomes.

Conclusions: More evidence on the value of expanded capacity or new models of care may inform new funding models to support such investments and reduced prices for new technologies, leading to more efficient and sustainable publicly funded healthcare systems.

Objectives: The aims of this study are (i) to present the design of a tele-expertise system, based on the telephone and electronic patient record (EPR), which supports the counseling of the infectious diseases specialist (IDS) for appropriate antimicrobial use, in a French University hospital; and (ii) to assess the diffusion of the system, the users’ adherence, and their perceived utility.

Methods: A prospective observational study was conducted to measure (i) the number and patterns of telephone calls for tele-expertise council, the number of initial and secondary assessments from the IDS and multidisciplinary meetings; (ii) the clinicians’ adherence rate to therapeutic proposals by the IDS and the number of clinical situations for which the IDS decided to move to bedside; and (iii) the perceived utility of the system by the medical managers of the most demanding departments.

Results: The review of patients’ records for 1 year period indicates that 87 percent of the therapeutic recommendations were fully followed. The adherence was high, despite the IDS moving to the bedside only in 6 percent of cases. Medical managers of the most demanding departments considered the system to be useful. Moreover, 6,994 tele-expertise notifications have been recorded into the EPR for 48 months.

Conclusions: The tele-expertise system is an original way to design information technology supported antimicrobial stewardship intervention based on the remote access to relevant information by the IDS and on the traceability of the medical counseling for the clinicians.

Objectives: Value assessment frameworks have gained prominence recently in the context of U.S. healthcare. Such frameworks set out a series of factors that are considered in funding decisions. The UK's National Institute of Health and Care Excellence (NICE) is an established health technology assessment (HTA) agency. We present a novel application of text analysis that characterizes NICE's Technology Appraisals in the context of the newer assessment frameworks and present the results in a visual way.

Methods: A total of 243 documents of NICE's medicines guidance from 2007 to 2016 were analyzed. Text analysis was used to identify a hierarchical set of decision factors considered in the assessments. The frequency of decision factors stated in the documents was determined and their association with terms related to uncertainty. The results were incorporated into visual representations of hierarchical factors.

Results: We identified 125 decision factors, and hierarchically grouped these into eight domains: Clinical Effectiveness, Cost Effectiveness, Condition, Current Practice, Clinical Need, New Treatment, Studies, and Other Factors. Textual analysis showed all domains appeared consistently in the guidance documents. Many factors were commonly associated with terms relating to uncertainty. A series of visual representations was created.

Conclusions: This study reveals the complexity and consistency of NICE's decision-making processes and demonstrates that cost effectiveness is not the only decision-criteria. The study highlights the importance of processes and methodology that can take both quantitative and qualitative information into account. Visualizations can help effectively communicate this complex information during the decision-making process and subsequently to stakeholders.

Objectives: There are no current established pathognomonic diagnostic features for uterine leiomyosarcomas in the pre- or perioperative setting. Recent inadvertent upstaging of this rare malignancy during laparoscopic morcellation of a presumed fibroid has prompted widespread debate among clinicians regarding the safety of current surgical techniques for management of fibroids. This study aims to conduct a systematic review investigating significant diagnostic features in magnetic resonance imaging (MRI) of uterine leiomyosarcomas.

Methods: A comprehensive database search was conducted guided by PRISMA recommendations for peer-reviewed publications to November 2017. Parameters available in MRI were compared for reliability and accuracy of diagnosis of leiomyosarcomas. A decision tree algorithm classifier model was constructed to investigate whether T1 and T2 MRI signal intensities are useful indicators.

Results: Nine eligible studies were identified for analysis. There appears to be a significant relationship between histopathological type and T1 and T2 intensity signals (p < .05). A decision tree model analyzing T1 and T2 signal intensity readings supports this trend, with a diagnostic specificity of 77.78 percent for uterine leiomyosarcomas. The apparent diffusion coefficient (ADC) values were not observed to have a significant relationship with tumor pathology (p = .18).

Conclusions: Various studies have investigated pre- and perioperative techniques in differentiating uterine leiomyosarcoma from benign fibroids. Given the rarity of the malignancy and lack of pathognomonic diagnostic parameters, there is difficulty in establishing definitive criteria. A decision tree model is proposed to aid diagnosis based on MRI signal intensities.

Objectives: The aim of this study was to evaluate the direct costs of type 2 diabetes mellitus patients treated in a Brazilian public hospital.

Methods: This was an exploratory retrospective cost-of-illness study with quantitative approach, using medical records of patients treated in a public hospital (2012–14), with at least one consultation over a period of 12 months. Data on patient's profile, exams, number of consultations, medications, hospitalizations, and comorbidities were collected. The cost per patient per year (pppy) was calculated as well as the costs related to glycated hemoglobin (HbA1c) values, using thresholds of 7 and 8 percent.

Results: Data of 726 patients were collected with mean age of 62 ± 11 years (68.3 percent female). A total of 67.1 percent presented HbA1c > 7 percent and 44.9 percent > 8 percent. The median cost of diabetes was United States dollar (USD) 197 pppy. The median costs of medication were USD 152.49 pppy, while costs of exams and consultations were USD 40.57 pppy and 8.70 pppy, respectively. Thirty-eight patients (4 percent) were hospitalized and presented a median cost of 3,656 per patient per hospitalization with a cost equivalent to 53.1 percent of total expenses. Total costs of patients with HbA1c ≤ 7 percent were lower for this group and also costs of medications and consultations, whereas for patients with HbA1c ≤ 8 percent, only total costs and costs of medications were lower when compared with HbA1c > 8 percent patients.

Conclusions: Medications and hospitalizations were the major contributor of diabetes expenses. Preventing T2DM, or reducing its complications through adequate control, may help avoid the substantial costs related to this disease.

Objectives: This paper aims to describe the added value of combining cost-effectiveness and ethical evaluations when the preferences of the decision maker toward cost-effectiveness evaluation outcomes are not known, with the French national neonatal screening of cystic fibrosis (CF) as a case-study.

Methods: A cost-effectiveness analysis comparing four CF neonatal screening strategies, with or without DNA testing, was performed. Ethical positions toward their outcomes were described. In addition, a post-hoc analysis of the ethical issues being considered relevant from the decision-makers’ perspective was conducted.

Results: Two strategies were found equally cost-effective. Among them, choosing the non-DNA or a DNA-based strategy constrains the decision maker to render a judgement between different ethical issues or disagreements associated with the screening program.

Conclusions: The analysis supports the relevance of combining cost-effectiveness and ethics evaluation in developing health policy, as a way to reveal or clarify the motives associated with health. The choice of the decision maker to favor the DNA-based strategy, which was not originally recommended, creates the opportunity to make explicit the role played by ethical issues in the decision.

Objectives: Optional scientific advice (SA) for the early benefit assessment of pharmaceuticals is offered by the German decision maker, the Federal Joint Committee (FJC). The aim of this study was to elicit manufacturers’ experiences with the SA procedures offered by the FJC to date.

Methods: A preliminary survey on a small sample size was conducted. Subsequently, a questionnaire comprising eight items, which was developed on the basis of that survey, was used. Data were analyzed using qualitative and quantitative approaches.

Results: The elicitation, including a sample of 25 percent of the completed advice, highlighted the following, regarding the process as well as to the content shortcomings of the SA procedures from an industrial perspective: inconsistencies, FJC's lack of expertise in conducting clinical trials, partially incomplete answers. and a low willingness of the FJC to engage in dialogue with industry were criticized. On the other hand, the majority of respondents expressed a positive attitude concerning unambiguousness, completeness, traceability, discussion atmosphere, and the protocol of the advice. Early SA, before pivotal trials start, showed a significantly higher completeness compared with late SA with respect to endpoints and study duration. Within 4 years the quality of FJC's propositions on some topics improved significantly.

Conclusions: Only a few statistically significant differences were detectable between early versus late SA. A positive trend in industry's perception of the SA can be observed over time. A more active involvement of additional stakeholders and the incorporation of procedural elements from other healthcare systems could improve the quality of the SA offered by the FJC.

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: Health technology reassessment (HTR) is a policy process to manage health technologies throughout their lifecycle and ensure their ongoing optimal use. However, within an ever-evolving field, HTR is only one of many concepts associated with the optimization of health technologies. There is limited understanding of how other concepts and processes might differ and/or be interrelated. This study aims to describe the concepts underlying the various technology optimization processes and to reconcile their relationships within the HTR process.

Methods: A synthesis of the literature on approaches to HTR was completed. An inductive synthesis approach was completed to catalogue common concepts and themes. Expert stakeholders were consulted to develop a schematic to diagrammatically depict the relationships among concepts and frame them within the HTR process.

Results: A practical schematic was developed. Common concepts and themes were organized under six major domains that address the following discussion questions: (i) what is the value of the existing technology?; (ii) what is the current utilization gap?; (iii) what are the available tools and resources?; (iv) what are the levers for change?; (v) what is the desired outcome?; and (vi) who are the foundational actors?

Conclusions: Using these six questions to frame the issues faced by HTR will advance the common understanding of HTR, as well as improve implementation of HTR initiatives. These questions will clearly identify the process required to move forward within a complex healthcare system.

Objectives: The aim of this study was to explore the risk assessment tools and criteria used to assess the risk of medical devices in hospitals, and to explore the link between the risk of a medical device and how those risks impact or alter the training of staff.

Methods: Within a broader questionnaire on implementation of a national guideline, we collected quantitative data regarding the types of risk assessment tools used in hospitals and the training of healthcare staff.

Results: The response rate for the questionnaire was 81 percent; a total of sixty-five of eighty Dutch hospitals. All hospitals use a risk assessment tool and the biggest cluster (40 percent) use a tool developed internally. The criteria used to assess risk most often are: the function of the device (92 percent), the severity of adverse events (88 percent) and the frequency of use (77 percent). Forty-seven of fifty-six hospitals (84 percent) base their training on the risk associated with a medical device. For medium- and high-risk devices, the main method is practical training. As risk increases, the amount and type of training and examination increases.

Conclusions: Dutch hospitals use a wide range of tools to assess the risk of medical devices. These tools are often based on the same criteria: the function of the device, the potential severity of adverse events, and the frequency of use. Furthermore, these tools are used to determine the amount and type of training required for staff. If the risk of a device is higher, then the training and examination is more extensive.