The study by Hutton, Trueman, and Henshall provides a thoughtful and helpful set of observations about the potential benefits of linking reimbursement to requirements for further clinical research (coverage with evidence development—CED), as well as the likely challenges and obstacles to implementation. In this commentary, we will expand upon several of the key points made in their study and offer some additional suggestions for moving this policy discussion forward

The greatest problem concerning health care technology for developing countries is that they are dependent upon the industrialized world for technology. The only short-term solution to this problem is to improve the choices that are available to them. This goal will require changes in the structure and processes of policy making. A particular difficulty for these countries is the lack of trained personnel in fields related to technology assessment.

Objectives:To compare the six-year outcome of a multidisciplinary rehabilitation program with continued care within primary care in terms of health-related quality of life and cost-effectiveness. Furthermore, predictors of total costs to society were examined.

Methods:A prospective, matched, controlled, six-year follow-up was designed. The study included 236 patients (42 men, 194 women) nineteen to sixty-one years of age with prolonged musculoskeletal disorders. The intervention comprised a four-week multidisciplinary rehabilitation and an active one-year follow-up based on a bio-psycho-social approach. The control group received continued care within primary care. The main outcome measures were quality of life measured using the Nottingham Health Profile, motivation identified by an interview and patient-specific total costs to society. Differences in mean costs between groups and cost-effectiveness were evaluated by applying nonparametric bootstrapping techniques.

Results: Total costs per treated patient in the rehabilitation group and the control group were £43,464 (SD=31,093) and £44,123 (SD=33,333), respectively (p=.896). Multidisciplinary rehabilitation improved quality of life somewhat more cost-effectively. Motivation was revealed as a predictor of total costs.

Conclusion: In the long-run, the evaluated multidisciplinary rehabilitation improved the highly motivated patients' quality of life most cost-effectively. The latently motivated patients may require rehabilitation, which is less intensive and with a longer duration, to improve their health in a whole-person perspective. The burden of prolonged musculoskeletal disorders to society was reaffirmed. Motivation could be a predictor of total costs, a factor which has to be taken into account in the examination procedure.

Objectives: For medical devices, benefits other than direct clinical effects may have a large impact on the patients’ well-being, but a standardized method for measuring these benefits is unavailable. The objective was to explore potential patient benefits provided by medical devices, and to assess the relative preferences of these benefits in the general Swedish population.

Methods: To identify attributes of patient benefit, healthcare personnel within a wide range of disease areas were interviewed. The generalized attributes were then validated among healthcare personnel, patient organizations, and manufacturers; in two pilot studies in the general population; and in two rounds of cognitive interviews. The general population's preferences of the attributes were measured with a usability-tested questionnaire in a final responding sample of 3,802 individuals, representative of the Swedish population.

Results: Twenty attributes were identified, encompassing aspects of integrity, sense of security, social participation, and convenience. When measuring the relative preferences, the response rate was 37.0 percent, and the results showed that the attributes with the highest preferences concerned reliability, reduced need for assistance, and sense of control of the illness/disability.

Conclusions: A set of twenty attributes of patient benefit relevant to users of medical devices was identified and validated. A questionnaire for patient-reported assessment of the benefits provided by a medical device was developed, based on the attributes. The questionnaire, designated MedTech20, provides a generic measurement method for the evaluation of medical devices used in a wide range of diseases/disabilities.

Great variation exists in the health care delivery systems throughout Latin America and the Caribbean. In general, medical technology is concentrated in large cities at private hospitals that serve a small, elite segment of the population. What is missing in most of these countries is a clearly defined social policy guaranteeing distribution of medical technology to all segments of society. While data is scarce, it appears that political and economic concerns, rather than medical concerns, determine what and how much technology will be available to all segments of the society. Products are sometimes imported that compete with domestically produced ones. Moreover, transfers of technology often fail to include the necessary knowledge to make imported technology truly useful. However, current economic crises throughout the region are forcing changes in policies; as a result, there is a new emphasis on domestic production of technology over imports and the evaluation of medical technology for its appropriate use in Latin America and the Caribbean.

Objectives: Whether cost-effectiveness of secondary health care can be measured in a simple, yet commensurate way was studied.

Methods: Approximately 4,900 patients' health-related quality of life scores before and after treatment were measured. Used were a combination of quality of life data with diagnostic and financial indicators routinely collected in the hospital.

Results: Seventy percent of patients returned the first questionnaire and the informed written consent to participate. Of these patients, 80 percent also returned the second questionnaire sent out 3 to 12 months after treatment, depending on clinical specialty and diagnostic category. The routine of sending out questionnaires could be automated in such a way that data collection required only a limited amount of extra work. Patients were generally satisfied with the fact that the hospital was interested in their well-being also after treatment. No physician offered the chance to participate refused data collection in the patient group he or she was responsible for. The attitudes of the nursing staff were generally positive toward data collection, although it caused some extra work for some of them. The possibility of relating already routinely collected financial performance indicators with a relevant measure of treatment effectiveness, opened prospects for refined analysis of cost-effectiveness of secondary health care.

Conclusions: Routine collection of health-related quality of life data as an indicator of treatment effectiveness is feasible, requires only a small amount of extra work, and is potentially very useful when combined with existing measures of hospital performance.

Objectives: The history of health technology assessment (HTA) in Mexico is examined, starting with the efforts to incorporate this topic into the policy agenda and culminating with the recent creation of a specialized public agency.

Methods: Information was gathered through a bibliographic search and interviews with actors involved in HTA in Mexico.

Results: HTA efforts were developed in Mexico since the mid-1980s with the participation both of academics and of policy makers, a relationship that eventually led to the creation of the Center for Technological Excellence within the Ministry of Health.

Conclusions: Institutionalization of HTA in resource-constrained settings requires the development of a critical mass of researchers involved in this field, the implementation of information efforts, and the establishment of strong relationships between HTA experts and policy makers.

Chorionic villus sampling (CVS), a new technique for prenatally diagnosing chromosomal and genetic disorders, may soon replace amniocentesis. This procedure, performed by inserting a catheter through the pregnant woman's cervix or through the abdomen and removing cells from the placenta, has the advantages of being available earlier in the pregnancy than amniocentesis (at 9 to 11 weeks gestational age) and of yielding results in a shorter time. Although the medical aspects of the procedure are being investigated, its psychosocial and policy implications have not been studied systematically. These issues include the subjective assessment of risk that prompts women to choose or to reject CVS, the implications of earlier diagnosis for feelings about abortion, the potential of negative findings (i.e., the absence of the designated disorder) for well-being during the remainder of the pregnancy, the ramifications of first trimester identification of fetal sex, equity of access to prenatal diagnosis, and the possible over- utilization of these procedures. Drawing on previous research regarding amniocentesis and more recent research concerning CVS, this article discusses the potential implications of CVS.

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: The aim of this study was to evaluate the universal newborn hearing screening (UNHS) and intervention program in Shanghai, China.

Methods: This study included the quantitative analyses of the UNHS-Shanghai database in 2002–12 and qualitative assessment of the program. The Otoacoustic Emissions and the Automated Auditory Brainstem Evoked Responses tests were conducted in screening. The costs and benefits were calculated based on the number of participants in each stage. The short-term and long-term periods were defined as from birth to 15 years of age or to death (82-year-olds), respectively. Sensitivity analyses were conducted.

Results: A total 1,574,380 newborns were included, representing 93.6 percent of all eligible babies in Shanghai during the study period. The prevalence of newborn hearing loss was 1.66‰. The short-term/long-term program costs were ¥488.5 million (US$75.52 million)/¥1.08 billion (US$167.12 million), and the short-term/long-term program benefit was ¥980.1 million (US$151.53 million)/¥8.13 billion (US$1.26 billion). The program benefit was greater than its cost if the proportion of hearing-loss children enrolled in regular schools was no less than 41.4 percent of all hearing impaired children, as well as if the wage growth rate ranged from 3 percent to 8 percent. Qualitative results also suggested that stakeholders strongly supported this program.

Conclusions: The universal newborn hearing screening and intervention program in Shanghai is justified in terms of the resource input in the long run, although there is still room for further improvement with respect to educational rehabilitation and a better infrastructure system.

Objectives: The association between type and amount of clinical evidence and type of National Institute for Health and Clinical Excellence recommendations for interventional procedures was examined.

Methods: The evidence about 736 studies (including 183,729 patients) relating to 130 different interventional procedures and about relevant recommendations was analyzed. Associations were examined between type of recommendation (“normal arrangements” or “cautionary guidance”) and evidence type, total number of treated patients, and mean follow-up length. Evidence type was categorized as (a) randomized, (b) nonrandomized controlled, and (c) case series/reports. The main outcome measures were frequency of evidence type, total number of patients treated, and mean follow-up length, by type of recommendation.

Results: “Normal arrangements” recommendations were made for 70 (54 percent) procedures and “cautionary guidance” was issued for 60 (46 percent) procedures. Procedures supported by at least one randomized study (34 percent, n = 44) were more likely to receive a “normal arrangements” recommendation (relative risk 1.38, p = .063). Overall, there were 85 (12 percent), 135 (18 percent), and 516 (70 percent) studies in categories a–c, respectively. The number of treated patients was significantly larger among procedures with “normal arrangements” (median, 605; range, 26–6,842) than among those with “cautionary guidance” (median, 240; range, 1–3,261; p < .001) recommendation. Mean follow-up length was longer in studies relating to procedures with “normal arrangements” recommendation (median, 16.7; range, 0–84 months) compared with those with “cautionary guidance” (median, 14.6; range, 0–67 months; p = .160).

Conclusions: Procedures supported by randomized studies, and with larger numbers of patients and longer follow-up length, were more likely to receive positive guidance. Future research and development on interventional procedures should aim to produce better and more relevant evidence to optimize the possibility of such procedures being accepted by policy makers.

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: 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: To review the existing health economic literature on meningococcal disease vaccination.

Methods: A Medline search for economic evaluations of vaccination programs for meningococcal disease in developed countries was conducted. All identified studies were reviewed.

Results: Nine published studies were identified examining either mass vaccination during outbreaks or routine vaccination. Although net expenses were estimated in almost all studies, the resulting cost-effectiveness ratios varied widely. Vaccination of college-age students was found to be potentially cost-effective in Australia but not in the United States. With one exception, routine vaccination of children and adolescents in Europe was predicted to be cost-effective. Many simplifying assumptions were made, and important elements were often left out, in particular the potential for reduced transmission of disease.

Conclusions: The methods used and the vaccination strategies vary widely, and results do not provide strong grounds for making conclusions as to whether vaccination is cost-effective. Furthermore, in all instances, transmission of disease, changes in population carriage rates, and outbreaks are either ignored, dealt with using very broad simplifying assumptions, or are not necessarily generalizable to other settings. The analyses provide some insight into the potential cost-effectiveness of vaccination, but more importantly, they highlight areas requiring further study. Economic evaluations based on observed outcomes from recently implemented strategies would be helpful, as would more sophisticated health economic models. The choice of vaccination strategies cannot be based on the results of existing economic analyses.

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.

Background: Total thyroidectomy (TT) can be performed either with the traditional technique or using the ultrasound scalpel. Here, the use of the ultrasound scalpel is investigated in order to assess cost-effectiveness from an hospital, third party payer and societal perspective.

Methods: A randomized controlled trial was conducted at the University Hospital A. Gemelli in Rome. Data refer to 198 patients, randomized to either surgery with the ultrasound scalpel (n = 96) or traditional (n = 102) and followed for 3 months after hospital discharge. Operation time (OT) and resource consumption were recorded. Main clinical outcome investigated was quality of life (evaluated with EQ-5D).

Results: A shorter operation time (traditional: 76.36 vs ultrasound: 54.16 minutes, p < 0.001) was observed. 3 months after surgery, differences in QoL were significant (0.91 vs 0.84, p = 0.002). Concerning the hospital perspective, ultrasound scalpel allows savings of 119 EUR per patient. From a societal perspective, ultrasound scalpel is also related to lower medical resource consumption during a 3 month follow-up after discharge (traditional: 129.03 EUR vs ultrasound: 107.82 EUR) and lower non-medical resource utilization (transport/hotels costs traditional:535.51 EUR vs ultrasound: 342.77 EUR. No statistical difference was found in productivity losses up to 3 months (traditional: 377.71 EUR vs ultrasound: 385.51 EUR).

Conclusion: Allowing an overall saving of 325.36 EUR per patient, Ultrasound scalpel should be adopted for TT procedures in the “A.Gemelli” University hospital.

This paper considers the question of what ought to be valued in the context of measuring the outcomes of healthcare interventions. The answer is discrete health states in the case of the quality-adjusted life-year (QALY) model and an entire health profile in the case of the healthy-years equivalent (HYE) approach. How well the weighted average of values attached to the former approximates the overall value attached to the latter depends on the validity of the assumptions of the QALY model. The paper considers some of the empirical literature relating to them. One of the most important assumptions, which from the limited evidence available appears not to hold, is additive separability. However, it is argued that violation of this assumption does not in itself invalidate the QALY approach, since in some circumstances it might be more appropriate to elicit the value of a health state independently of the states that succeed it. Investigation into this issue is identified as one of the key areas where future research efforts should be directed.

Background: Most developing countries and resource-limited settings lack robust health technology assessment (HTA) systems. Because the development of locally relevant HTA is not immediately viable, and the extrapolation of external HTA is inappropriate, a new model for evaluating health technologies is required.

Objectives: The aim of this study was to describe the development and application of KNOW ESSENTIALS, a tool facilitating evidence-based decisions on health technologies by stakeholders in settings lacking formal HTA systems.

Methods: Current HTA methodology was examined through literature search. Additional issues relevant to resource-limited settings, but not adequately addressed in current methodology, were identified through further literature search, appraisal of contextually relevant issues, discussion with healthcare professionals familiar with the local context, and personal experience. A set of thirteen elements important for evidence-based decisions was identified, selected and combined into a tool with the mnemonic KNOW ESSENTIALS. Detailed definitions for each element, coding for the elements, and a system to evaluate a given health technology using the tool were developed.

Results and Conclusions: Developing countries and resource-limited settings face several challenges to informed decision making. Models that are relevant and applicable in high-income countries are unlikely in such settings. KNOW ESSENTIALS is an alternative that facilitates evidence-based decision making by stakeholders without formal expertise in HTA. The tool could be particularly useful, as an interim measure, in healthcare systems that are developing HTA capacity. It could also be useful anywhere when rapid evidence-based decisions on health technologies are required.