Battista has written a thoughtful and timely challenge to the field of health technology assessment (HTA). As the author points out, HTA is changing and evolving. One of the greatest challenges is that HTA now seems firmly established in a number of countries, and is being looked to more and more for the “answers.” We accept and endorse Battista's central thesis, and essentially all of his critical questions, including his ideas about future research topics. We hope to evoke further thoughts in the reader and perhaps stimulate other responses, either private and public.

Objectives: The aim of this study was to estimate the potential cost-effectiveness and expected value of perfect information of a recently derived clinical prediction rule for patients presenting to emergency departments with chest discomfort.

Methods: A decision analytic model was constructed to compare the Early Disposition Prediction Rule (EDPR) with the current standard of care. Results were used to calculate the potential cost-effectiveness of the EDPR, as well as the Value of Information in conducting further research. Study subjects were adults presenting with chest discomfort to two urban emergency departments in Vancouver, British Columbia, Canada. The clinical prediction rule identifies patients who are eligible for early discharge within 3 hours of presentation to the emergency department. The outcome measure used was inappropriate emergency department discharge of patients with acute coronary syndrome (ACS).

Results: The incremental cost-effectiveness ratio of the EDPR in comparison to usual care was (negative) $2,999 per inappropriate ACS discharge prevented, indicating a potential cost-savings in introducing the intervention. The expected value of perfect information was $16.3 million in the first year of implementation, suggesting a high benefit from conducting further research to validate the decision rule.

Conclusions: The EDPR is likely to be cost-effective; however, given the high degree of uncertainty in the estimates of costs and patient outcomes, further research is required to inform the decision to implement the intervention. The potential health and monetary benefits of this clinical prediction rule outweigh the costs of doing further research.

This paper examines the current status of in vitro fertilization and embryo transfer (IVF-ET) as a treatment for various types of infertility. We reviewed studies on the efficacy and safety of IVF-ET and intracytoplasmic sperm injection (ICSI) plus IVF-ET, compared with conventional treatment or no treatment for various infertility diagnoses. Material retrieved included English language publications between 1992 and January 1997 that reported the results of prospective controlled clinical trials, cohort studies, and retrospective comparative studies with large series, and reviews presenting risks, complications, and longer-term health consequences associated with IVF-ET and ICSI. No adequate prospective comparative studies of sufficient power on the use of IVF-ET for specific infertility diagnoses have been reported to date. Most of the published reports concerning results with IVF-ET as a treatment of infertility have been based upon small, uncontrolled studies, with various methodological weaknesses. Reported results are not directly comparable. There are few follow-up data on outcomes after pregnancy is established or on long-term health consequences of the use of IVF-ET on mothers and their babies. IVF-ET has diffused widely without comprehensive assessment of its efficacy and safety. The available evidence supports its use only for severe bilateral tubal occlusion. For other diagnoses of infertility the evidence is limited and does not establish whether IVF-ET is effective. Long-term, well-designed, prospective clinical trials are required to determine when and for what indications IVF-ET is effective and what its health effects are on both mothers and their babies.

Objectives: The aim of this study was to examine the use of implicit and explicit Bayesian methods in health technology assessments and to identify whether this has changed over time.

Methods: A review of all health technology assessment (HTA) reports of secondary research published by the UK National Institute of Health Research (NIHR) between 1997 and 2011. Data were extracted on the use and implementation of Bayesian methods, whether defined as such by the original authors (i.e., explicit) or not (i.e., implicit).

Results: A total of 155 of 375 (41 percent) NIHR HTA reports, identified as relevant to this review, contained a Bayesian analysis. Of these, 128 (83 percent) contained an implicit Bayesian analysis, 3 (2 percent) an explicit Bayesian analysis and 24 (15 percent) both implicit and explicit Bayesian analyses. Of the twenty-seven reports that explicitly used Bayes theorem, only six included prior information in the form of (informative) prior distributions. Over time, the percentage of HTA reports that used Bayesian (implicit and/or explicit) methods increased from 0 percent in 1997 to nearly 80 percent in 2011.

Conclusions: This review has shown that there has been an increase in the use of Bayesian methods in HTA, which is likely to be a result of the increase in freely available resources to implement the approach. Areas where Bayesian methods have the potential to advance healthcare evaluations in the future are considered in the discussion.

Objectives: The aim of this study was to present and illustrate an instrument to measure the level of innovation at country level.

Methods: The data used are the Organisation for Economic Co-operation and Development (OECD) health data 2009, in particular the information on use of medical technology. Two composite scales expressing a relative level of adoption of innovations in health care are regressed, using multilevel regression analysis, on country characteristics. The country characteristics are selected as proxies on availability or scarcity of resources in a country. We expect that scarcity will promote adoption of innovations that enhance efficiency, and that availability of resources will promote advanced, expensive innovations.

Results: Two scales were constructed. One scale indicates the use of efficiency-enhancing innovations (day case treatment), and the other scale indicates availability of advanced technical innovations. The application of day case treatment is significantly associated with education level (+), the ratio of people aged 15–64 versus younger and older people (+) and the number of hospital beds (−). Availability of advanced medical devices are associated with the expenditure on health (+), demographic dependency (−), number of hospital beds (+), and the annual reduction of hospital beds (−).

Conclusions: Diffusion of innovations is influenced by characteristics of the country and of the healthcare system; fewer resources encourage diffusion of innovations that enhance efficiency and more resources encourage diffusion of complex, expensive devices. This indicates that decisions by healthcare professionals on which innovation to adopt is embedded in a context that is influenced and shaped by the availability of resources on macro level.

Objectives: Shoulder complaints (SCs) constitute the second largest group of musculoskeletal disorders after low back pain. The economic burden in terms of costs of healthcare use and costs due to work absenteeism underlines the need for a cost-effectiveness analysis of the interventions involved. The education and activation program (EAP) is a newly developed early intervention to prevent the development of chronic SCs. A cost-effectiveness analysis should provide more information on the effect of an EAP on total costs related to SCs.

Methods: We conducted a cost-effectiveness analysis alongside a randomized clinical trial comparing the effectiveness of the EAP in addition to usual care (EAP group) with that of usual care alone (UC group) in terms of preventing chronicity in patients with acute SCs. The aim of the cost-effectiveness analysis was to compare the observed difference in costs with the clinical effectiveness (i.e., patient-perceived recovery after 26 weeks), using bootstraps.

Results: The comparison of total costs between treatment groups showed no significant (p = .077) difference after 26 weeks. The majority (82 percent) of the cost-effect pairs after bootstrap analysis were located in the northeast quadrant, suggesting more effect but at higher costs.

Conclusions: In view of the clinical relevance of the clinical outcomes and the considerable costs needed to achieve this, it can be concluded that the EAP is currently not cost-effective.

Objectives: We developed specific evaluation criteria to assess patient and public involvement in resource allocation decisions in health care.

Methods: We reviewed the literature from health and other sectors relevant to stakeholder involvement and conducted twenty-seven key informant interviews with stakeholders knowledgeable about patient and public involvement in Canadian drug resource allocation decisions. We used an inductive qualitative thematic approach to analyze the interviews with codes and categories developed directly from individuals’ interview transcripts.

Results: Integrating respondents’ comments and the literature review, we identified nine evaluation criteria of patient and the public involvement in healthcare resource allocation decision making: clarity regarding rationale and roles of patient and public members, sufficient support, adequate representation of relevant views, fair decision-making processes, legitimacy of committee processes, adequate opportunity for participation, meaningful degree of participation, noticeable effect on decisions, and considerations of the efficiency of patient and public involvement.

Conclusions: Our results will help to develop methods to evaluate patient and public involvement in healthcare decision making.

Objectives: The semiprivate health system in Iran has created an opportunity for unnecessary uses of advanced medical equipments including magnetic resonance imaging (MRI). This study aimed to evaluate the evidence for MRI overuse in private diagnostic imaging centers in Tehran, Iran. The objectives of this study were to determine the frequency of use of MRI scans for different complaints and to explore frequency of normal MRI findings as a function of unnecessary MRI use.

Methods: We conducted a survey among private MRI centers in Tehran, Iran, to study the proportion of MRI scans that may result in significant clinical finding. All MRI reports at a specific point in time at selected MRI centers were reviewed by a physician and the findings were recorded as normal, abnormal, or substantial changes.

Results: Of all the MRI reports, 17.2 percent had resulted in normal findings; 9.8 percent ordered for examination of headache, and 4.8 percent for lower back pain.

Conclusion: Unnecessary MRIs are most likely to result in normal finding; although not all the MRI with normal results could be identified as unnecessary. Negative findings from MRI scans may be reassuring to both clinicians and patients. The proportion of normal findings in MRI scans did not provide evidence of MRI overuse in Iran. The results of this study warrant formation of guidelines for the use of MRIs for headache and low back pain disorders.

The pulmonary artery catheter (PAC) is an invasive hemodynamic monitoring device that is used extensively in critical care units. This technological advance allows the critical care physician and nurse to closely monitor physiological functions at the bedside. There have been no formal evaluations of the impact of this device on patient care. Reviews and observational studies have yielded different conclusions regarding risks and benefits to patients. This has resulted in much editorial comment expressing divergent opinions on the value of the PAC, but there has been no scientific evidence to guide practice and no apparent effect on the use of these devices. The PAC and other medical monitoring devices must be evaluated with respect to their impact on patient care.

Objectives: The aim of this study was to estimate the cost-effectiveness of nebulized magnesium sulphate (MgSO4) in acute asthma in children from the perspective of the UK National Health Service and personal social services.

Methods: An economic evaluation was conducted based on evidence from a randomized placebo controlled multi-center trial of nebulized MgSO4 in severe acute asthma in children. Participants comprised 508 children aged 2–16 years presenting to an emergency department or a children's assessment unit with severe acute asthma across thirty hospitals in the United Kingdom. Children were randomly allocated to receive nebulized salbutamol and ipratropium bromide mixed with either 2.5 ml of isotonic MgSO4 or 2.5 ml of isotonic saline on three occasions at 20-min intervals. Cost-effectiveness outcomes were constructed around the Yung Asthma Severity Score (ASS) after 60 min of treatment; whilst cost-utility outcomes were constructed around the quality-adjusted life-year (QALY) metric. The nonparametric bootstrap method was used to present cost-effectiveness acceptability curves at alternative cost-effectiveness thresholds for either: (i) a unit reduction in ASS; or (ii) an additional QALY.

Results: MgSO4 had a 75.1 percent probability of being cost-effective at a GBP 1,000 (EUR 1,148) per unit decrement in ASS threshold, an 88.0 percent probability of being more effective (in terms of reducing the ASS) and a 36.6 percent probability of being less costly. MgSO4 also had a 67.6 percent probability of being cost-effective at a GBP 20,000 (EUR 22,957) per QALY gained threshold, an 8.5 percent probability of being more effective (in terms of generating increased QALYs) and a 69.1 percent probability of being less costly. Sensitivity analyses showed that the results of the economic evaluation were particularly sensitive to the methods used for QALY estimation.

Conclusions: The probability of cost-effectiveness of nebulized isotonic MgSO4, given as an adjuvant to standard treatment of severe acute asthma in children, is less than 70 percent across accepted cost-effectiveness thresholds for an additional QALY.

Background: The cost-effectiveness of triptans in the treatment of migraine has not been assessed since generic sumatriptan entered the Finnish market in 2008.

Methods: Using systematic review and mixed treatment comparison, the effectiveness of triptans was estimated with regard to 2-hour response, 2-hour pain-free, recurrence, and any adverse event, using published clinical data. Direct and indirect costs (2010 EUR, societal perspective) and quality-adjusted life-years (QALYs) were evaluated over one acute migraine attack using a decision-tree model.

Results: The meta-analysis combined data from fifty-six publications. The highest probability of achieving the primary outcome, “sustained pain-free, no adverse event” (SNAE), was estimated for eletriptan 40 mg (20.9 percent). Sumatriptan 100 mg was the treatment with lowest estimated costs (€20.86), and the incremental cost-effectiveness ratio of eletriptan 40 mg compared with sumatriptan 100 mg was €43.65 per SNAE gained (€19,659 per QALY gained).

Conclusion: Depending on the decision-maker's willingness-to-pay threshold, either sumatriptan 100 mg or eletriptan 40 mg is likely to be cost-effective.

Objectives: The aim of this study is to evaluate the cost-effectiveness of a patient-direct mailed advance notification letter on participants of a National Bowel Cancer Screening Program (NBCSP) in Australia, which was launched in August 2006 and offers free fecal occult blood testing to all Australians turning 50, 55, or 65 years of age in any given year.

Methods: This study followed a hypothetical cohort of 50-year-old, 55-year-old, and 65-year-old patients undergoing fecal occult blood test (FOBT) screening through a decision analytic Markov model. The intervention compared two strategies: (i) advance letter, NBCSP, and FOBT compared with (ii) NBCSP and FOBT. The main outcome measures were life-years gained (LYG), quality-adjusted life-years (QALYs) gained and incremental cost-effectiveness ratio.

Results: An advance notification screening letter would yield an additional 54 per 100,000 colorectal cancer deaths avoided compared with no letter. The estimated cost-effectiveness was $3,976 per LYG and $6,976 per QALY gained.

Conclusions: An advance notification letter in the NBCSP may have a significant impact on LYG and cancer deaths avoided. It is cost-effective and offers a feasible strategy that could be rolled out across other screening program at an acceptable cost.

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.

The Human Fertilisation and Embryology Act of the United Kingdom was passed in 1990, leading to the formation of the Human Fertilisation and Embryology Authority (HFEA), the first statutory body to regulate and control assisted conception anywhere in the world. The principal function of the HFEA is to license and monitor clinics that carry out in vitro fertilization (IVF), donor insemination (DI), and embryo research. Information on over 135,000 treatment cycles, 20,000 pregnancies, and 25,000 babies following IVF has now been collected as part of the regulatory process, and these data have permitted unbiased and accurate evaluation of treatment efficacy using pregnancy and live-birth rates. The treating clinics are required by law to provide information on the outcome of all births, including neonatal mortality and congenital malformations, but there is no systematic validation of these data using medical records or any follow-up of treated women, or babies, over time. In addition, the strict confidentiality of data supplied to the HFEA means that outside researchers have been unable to access the database for research projects. Thus, at the present time, it is not possible to evaluate the long-term safety of assisted conception procedures using HFEA data. There is reasonable scientific evidence to justify full investigation of the health of both treated women and resulting children. Particular health outcomes requiring evaluation include obstetric complications, preterm births, cerebral palsy, and cancer. The HFEA has recognized the need for follow-up studies and is currently investigating ways of enabling research projects using HFEA data to be undertaken.

Objectives: The aim of this article is to describe and analyze the stages toward recognition and implementation of health technology assessment (HTA).

Methods: System analysis of structures and institutions and their use of HTA.

Results: Austria is a latecomer in implementing evaluations/HTA as decision support. It can to a certain degree absorb the increasing international knowledge. Austria had a long time to observe the successes and failures of HTA in other countries and to learn from other countries. The implementation of HTA within the Austrian healthcare system ran through stages of uptake: starting 1989 with a systematic review on international activities, first international networking and collaboration since 1991, proposed assessments until the late 1990s, followed by reactive assessments on demand mostly on high volume and costly technologies since then. Since 2000, HTA is used on a regular basis for investment and reimbursement decisions by several players, namely the Ministry of Health, the Social Insurance and hospital cooperations. In 2006, the Austrian HTA-institute was founded.

Conclusions: It took approximately 15 years from first research activities in HTA to an institutionalization. HTA in Austria is not only product- but also process-oriented: The actual production of assessments for decision support is as important as the structuring and accompanying of the process of decisions making. In addition, shaping the public understanding of science (characterized by the intrinsic belief that all new medical interventions provide added value to the healthcare system) is part of Austrian HTA.