Objectives: To compare the cost-effectiveness of the treatment of ankylosing spondylitis (AS) with infliximab in the United Kingdom over lifetime estimated from two different clinical trials and adjusted for clinical practice guidelines.

Methods: A cost-effectiveness model was developed to incorporate clinical, epidemiological, and economic data and allow extrapolation of trial results and incorporation of long-term treatment. Assumptions regarding treatment beyond the trials were based on open extensions from the trials and treatment guidelines by the British Society for Rheumatology. Results are presented for both the societal perspective and the perspective of the National Health Service (UK £, discounted 3.5 percent).

Results: Under the assumption that disease activity would be controlled and functional capacity would remain stable while on drug, treatment with infliximab (5 mg/kg every 6 weeks) dominates standard treatment in the societal perspective. In the National Health Service perspective, the cost per quality-adjusted life-year (QALY) gained over lifetime was £28,300 and £26,800 for the two trials. If functional capacity were to deteriorate at half the rate of untreated patients, the cost per QALY gained would be £35,300 and £34,100, respectively. The results are sensitive to the dosing regimen adopted, the discontinuation rate, and assumptions concerning disease progression while on treatment.

Conclusions: The two clinical trials yield the same cost-effectiveness results and the cost per QALY gained with treatment was found to be in the acceptable range.

Objectives: The role and impact of health technology assessment (HTA) in health policy has been widely discussed. Researchers have started to analyze how decisions on coverage of new technologies are made. Although the involvement of HTA may be an indicator of a well established decision process, this hypothesis requires validation. Also, it is not known whether HTA involvement is associated with other characteristics of decision making like participation or transparency. The primary objective of this study was to develop and test statements on the association between the publication of an HTA and coverage decision making for newborn screening tests in European Union countries.

Methods: Five statements were defined on the relative role of HTA during the steps of decision processes: trigger, participation, publication, assessment, and appraisal. For this purpose, data on twenty-two decision processes in the area of newborn screening across Europe were analyzed, defined as a coverage decision for a given disorder in a specific country. Decision processes were compared by whether the decision was accompanied by the publication of an HTA report. To test differences, nonparametric statistical tests were used.

Results: The decision steps of trigger, participation and publication differed between the HTA and the non-HTA groups. No clear association between HTA and assessment methods in coverage decision making was identified.

Conclusions: It appeared that there is an association between HTA and coverage decision processes that are more explicit, inclusive, and transparent. It is unclear whether HTA is associated with formal evidence reviews and economic evaluations.

Objectives: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020.

Methods: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020. A semi-structured interview elicited payers' definitions of CER/RE and was structured around four hypothetical cases resembling drugs expected to be more common or poised to create policy challenges by 2020. Topics included acceptance of study designs and analytic methods associated with CER/RE. A systematic content review was done to extract relevant information.

Results: According to key informants, randomization will remain an essential component for assessing comparative or relative effectiveness. They anticipate greater use of policy levers such as conditional reimbursement or prior authorization to manage diffusion of new drugs. Case studies provided important insights into situations when certain types of CER evidence may be acceptable (e.g., observational data when differences between drugs are largely convenience).

Conclusions: Industry perceptions that CER/RE will change payers' evidentiary requirements in the future are consistent with our findings. Growing investment in payers' own data and increased reliance on policy tools to control diffusion of new drugs may also influence the type of evidence industry will be required to produce by 2020.

Objectives: The aim of this study was to conduct a review of health technology assessments (HTAs) in cervical cancer screening to highlight the most common metrics HTA agencies use to evaluate and recommend cervical cancer screening technologies.

Methods: The Center for Reviews and Dissemination (CRD), MedLine, and national HTA agency databases were searched using keywords (“cervical cancer screening” OR “cervical cancer” OR “cervical screening”) and “HTA” from January 2000 to October 2014. Non-English language reports without English summaries, non-HTA reports, HTAs unrelated to a screening intervention and HTAs without sufficient summaries available online were excluded. We used various National Institute for Health and Care Excellence (NICE) methods to extract key assessment criteria and to determine whether a change in screening practice was recommended.

Results: One hundred and ten unique HTA reports were identified; forty-four HTAs from seventeen countries met inclusion criteria. All reports evaluated technologies for use among women. Ten cervical screening technologies were identified either as an intervention or a comparator. The most common outcome metric evaluated was diagnostic accuracy, followed by economic effectiveness. Additional outcome metrics such as the use of adjunct testing, screening intervals, and age-specific testing were commonly evaluated. Nearly one-third (fifteen of forty-four) of HTAs recommended a change in practice.

Conclusions: This review highlights popular metrics used in HTAs for cervical cancer screening. Clinical and economic effectiveness metrics have been consistently assessed in HTAs, while the use of adjunct testing, screening intervals, and age-specific screening became increasingly prevalent from after 2007. Moreover, we observed an increase in optimized recommendations after 2007.

Objectives: Hospital Based Health Technology Assessment (HBHTA) practices, to inform decision making at the hospital level, emerged as urgent priority for policy makers, hospital managers, and professionals. The present study crystallized the results achieved by the testing of an original framework for HBHTA, developed within Lombardy Region: the IMPlementation of A Quick hospital-based HTA (IMPAQHTA). The study tested: (i) the HBHTA framework efficiency, (ii) feasibility, (iii) the tool utility and completeness, considering dimensions and sub-dimensions.

Methods: The IMPAQHTA framework deployed the Regional HTA program, activated in 2008 in Lombardy, at the hospital level. The relevance and feasibility of the framework were tested over a 3-year period through a large-scale empirical experiment, involving seventy-four healthcare professionals organized in different HBHTA teams for assessing thirty-two different technologies within twenty-two different hospitals. Semi-structured interviews and self-reported questionnaires were used to collect data regarding the relevance and feasibility of the IMPAQHTA framework.

Results: The proposed HBHTA framework proved to be suitable for application at the hospital level, in the Italian context, permitting a quick assessment (11 working days) and providing hospital decision makers with relevant and quantitative information. Performances in terms of feasibility, utility, completeness, and easiness proved to be satisfactory.

Conclusions: The IMPAQHTA was considered to be a complete and feasible HBHTA framework, as well as being replicable to different technologies within any hospital settings, thus demonstrating the capability of a hospital to develop a complete HTA, if supported by adequate and well defined tools and quantitative metrics.

Objectives: Surveillance programs are recommended to both families at high risk (Amsterdam-positive families with known- and unknown mutation) and moderate risk (families not fulfilling all Amsterdam criteria) of colorectal cancer (CRC). Cost-effectiveness has so far only been estimated for the group at high risk. The aim of the present study is to determine cost-effectiveness of surveillance programs where families at both high and moderate risk of HNPCC participate.

Methods: A decision analytic model (Markov model) is developed to assess surveillance programs where families at high and moderate risk of HNPCC are offered surveillance from age 25 and age 45, respectively. The model includes costs for all families referred to genetic counseling, including genetic risk assessment, mutation analysis, and surveillance in relevant families with or without known mutation, plus the costs related to any surgical treatment. The risk of metachronous CRC is also modeled.

Results: Incremental costs per life year gained are estimated to be €980 when families at both high and moderate risk of HNPCC undergo surveillance (€508 for high risk and €1600 for moderate risk) and €1947 when the moderate risk group is evaluated genetically but not offered surveillance. Sensitivity analysis showed these findings to be robust, although cost-effectiveness can be improved in cases of more conservative referrals to genetic counseling.

Conclusions: The result for high risk families confirms the findings in similar studies. Somewhat surprisingly, cost-effectiveness improves when surveillance of the moderate risk groups are included in the decision model.

Objectives: Several studies have sought to analyze the cost-effectiveness of advanced home care and home rehabilitation. However, the costs of informal care are rarely included in economic appraisals of home care. This study estimates the cost of informal care for patients treated in advanced home care and analyses some patient characteristics that influence informal care costs.

Methods: During one week in October 1995, data were collected on all 451 patients in advanced home care in the Swedish county of Östergötland. Costs were calculated by using two models: one including leisure time, and one excluding leisure time. Multiple regression analysis was used to analyze factors associated with costs of informal care.

Results: Seventy percent of the patients in the study had informal care around the clock during the week investigated. The patients had, on average, five formal care visits per week, each of which lasted for almost half an hour. Thus, the cost of informal care constituted a considerable part of the cost of advanced home care. When the cost of leisure time was included, the cost of informal care was estimated at SEK 5,880 per week per patient, or twice as high as total formal caregiver costs. When leisure time was excluded, the cost of informal care was estimated at SEK 3,410 per week per patient, which is still 1.2 times higher than formal caregiver costs (estimated at SEK 2,810 per week per patient). Informal care costs were higher among patients who were men, who were younger, who had their own housing, and who were diagnosed with cancer.

Conclusions: Studies of advanced home care that exclude the cost of informal care substantially underestimate the costs to society, regardless of whether or not the leisure time of the caregiver is included in the calculations.

Objectives: Health Technology Assessment (HTA) as a method for producing evidence in the health-care sector has been used for more than 25 years but has grown in extent during the past years. The objective of this study is to explore a possible evolution in these HTAs, in type of assessed technologies, in type of assessors, and in its methods.

Methods: A structured literature review was conducted of 433 HTA reports from the period 1989 to 2002 by eleven leading HTA institutions worldwide. The review focused on the methodology used, the assessors, and the assessed technologies and was designed to elucidate general time-trends in the practical application of HTA.

Results: The study shows that literature reviews are still the most often used method of assessment and accounts for a relatively stable fraction of assessments. The fraction of economic evaluations shows a slightly decreasing trend in contrast to randomized controlled trials and modeling/evaluations, which are applied more frequently. The data also demonstrate a more frequent use of external partners as assessors and a shift between devices and pharmaceuticals as assessed technologies.

Conclusions: The study shows an increase in the number of HTAs but no major developments in assessment methods used and, therefore, no widespread spillover from the development in research methods in general to the field of HTA methodology.

Abstract: The purpose of this study is to explain the rationale for the value of information approach to priority setting for research and to describe the methods intuitively for those familiar with basic decision analytical modeling. A policy-relevant case study is used to show the feasibility of the method and to illustrate the type of output that is generated and how these might be used to frame research recommendations. The case study relates to the use of glycoprotein IIb/IIIa antagonists for the treatment of patients with non-ST elevation acute coronary syndrome. This is an area that recently has been appraised by the National Institute for Health and Clinical Excellence.

Objectives: In 1998, a formal process using full health technology assessments (HTAs) was implemented to determine the suitability for public subsidy of new and emerging medical technologies in the Australian private healthcare sector. This process is overseen by the Medical Services Advisory Committee (MSAC). In 2004, horizon scanning was introduced in Australia with the stated objective of identifying new and emerging medical technologies into the public healthcare sector, with consideration to the publicly subsidized private healthcare sector. How well horizon scanning works in identifying new and emerging technologies suitable for government subsidized funding in the private healthcare sector is examined in this study.

Methods: A descriptive evaluation of the impact of horizon scanning as an early alert and awareness system identifying new and emerging technologies before these technologies are submitted to MSAC for a full HTA. All MSAC HTAs commenced after the introduction of horizon scanning in 2004 were cross-checked with the list of Prioritizing Summaries or Horizon Scanning Reports to determine whether a prior Prioritizing Summary or Horizon Scanning Report had been carried out.

Results: Of the forty-three technologies that were the subject of a full MSAC HTAs in the time period examined, only eleven had been the subject of either a Prioritizing Summary or Horizon Scanning Report. As a result of a full MSAC HTA, twelve of the technologies that were not the subject of a Prioritizing Summary or Horizon Scanning Report were given positive recommendations for public funding.

Conclusions: Horizon scanning was set up to scan the introduction of new and emerging medical technologies into the public healthcare sector, with consideration to the publicly subsidized private healthcare sector. Based on the number of new and emerging technologies that have been the subject of a full MSAC HTA without first being subjected to either a Prioritizing Summary or Horizon Scanning Report, horizon scanning in Australia does not function as an “early alert and awareness system” for funding in the publicly subsidized private healthcare sector in Australia.

Frequently, health care decisions must be made before compelling and unequivocal evidence is available about the benefits, risks, and costs of a proposed new health technology. It is common for decision makers to seek the advice and opinions of experts to supplement the available evidence. This article reviews some of the methods used or recommended for eliciting the opinions of experts about uncertain events and for combining these opinions with those of others and with available empirical evidence.

A cost-effectiveness (CE) analysis was performed of Bassini versus laparoscopic repair for primary inguinal hernia. Incremental costs per 1 -year recurrence-free patient were calculated for the societal and hospital perspective. From the hospital perspective, the incremental CE ratio of laparoscopic repair is 5.348 guilders. From the societal perspective, laparoscopic repair is both less costly and more effective than Bassini repair. Results were sensitive to assumptions about recurrence rates, laparoscopic operating time, and return to work. Laparoscopic repair should replace Bassini repair in order to benefit society. From the hospital perspective, the decision to accept laparoscopic repair depends on the willingness to pay.

Objectives: The aim of the study reported here was to investigate whether the use of magnetic resonance imaging (MRI) impacts on the clinical management of patients presenting with chronic knee problems, reduces costs, and improves patient outcome.

Methods: A single-center randomized controlled trial was conducted. Patients attending with knee problems in whom surgery was being considered were randomized either to investigation using an MRI scan or to investigation using arthroscopy. The study investigated benefits in terms of avoidance of surgery and patient health-related quality of life (using SF-36 and EQ-5D). Costs were assessed from the perspectives of the National Health Service and patients. All analyses were by intention to treat.

Results: The trial recruited 118 patients. No statistically significant differences were found between groups in terms of health outcome. However, the use of MRI was associated with a positive diagnostic/therapeutic impact: a significantly smaller proportion of patients in the MRI group underwent surgery (MRI=0.41, No-MRI=0.71; p value=.001). There was a similar mean overall cost for both groups.

Conclusions: The use of MRI in patients with chronic knee problems, in whom surgery was being considered, did not increase costs overall, was not associated with worse outcomes, and avoided surgery in a significant proportion of patients.

Objectives: The aim of this study was to describe generally the development and present situation with health technology assessment (HTA) in the United Kingdom.

Methods: The methods used are a review of important materials that have described the development process and present situation, supplemented by some personal experiences.

Results: The United Kingdom has been characterized historically as a country with a strong interest in evidence in health care, both clinical trials for efficacy and cost-effectiveness analyses. However, this evidence was not well-linked to the needs of the National Health Services (NHS) before formation of the NHS R&D Programme in 1991, The R&D Programme brought substantial resources into HTA and related activities, with the central aim of improving health care in Britain and increasing value for money. However, policy makers as well as staff of the R&D Programme were dissatisfied with the use of the HTA results in clinical and administrative practice. Therefore, the National Institute of Clinical Excellence (NICE) was formed in 1999. NICE issues guidance intended to influence practical decision making in health care at the national and local levels, based on efficacy information and, in some cases, economic analyses. NICE is now also seeking ways to maximize impacts on practice.

Conclusions: The UK experience shows that information on clinical and cost-effectiveness may not be enough to change practice, at least in the short-run. Still, one may conclude that the United Kingdom now has one of the few most important and influential HTA programs in the world.

Objectives: The clinical effectiveness and cost-effectiveness of implantable cardioverter defibrillators (ICD) for arrhythmias was assessed.

Methods: A systematic review of the literature of systematic reviews and randomized controlled trials that reported mortality outcomes associated with implantable cardioverter defibrillators compared with antiarrhythmic drug therapy in people at risk of sudden cardiac death due to arrhythmias was undertaken. Economic evaluations were also sought. Inclusion criteria, data extraction, and quality assessment were undertaken by standard methodology. A decision analytic model was constructed using best available evidence to determine cost-effectiveness in a UK setting.

Results: Eight randomized controlled trials, two systematic reviews, and a meta-analysis met the inclusion criteria and were of variable quality. Evidence suggests that ICDs reduce mortality in both secondary and primary prevention, although the magnitude of benefit depends on baseline risk for sudden cardiac death. Incremental cost per quality-adjusted life year ranged from £52,000 ($98,000) to over £200,000 ($379,000), depending on mortality risk and assumptions made.

Conclusions: Evidence suggests that ICDs reduce total mortality but may be cost-effective only in some subgroups of patients at high risk of ventricular arrhythmias. Further research is needed on risk stratification of patients in whom ICDs are most likely to be clinically and cost-effective.

Objectives: This study comprises a review of public involvement strategies across the five stages of research management in the UK's HTA program at the end of a 10-year period. These five stages are: identification of topics; prioritization of these topics as researchable questions; commissioning of research; monitoring of projects throughout their implementation; and publication and dissemination of findings.

Methods: Internal HTA documentation was analyzed alongside narrative analysis of semi-structured interviews of program staff, and a rapid review of published literature.

Results: Public involvement strategies have developed with the growth of the HTA program but were spread unevenly across the five stages of research management. Public involvement was present in identification, strongest in prioritization, present in commissioning but minimal in monitoring and absent in publication and dissemination.

Conclusions: The HTA program has developed public involvement strategies but mainly in prioritization. Further research is required to ascertain where public involvement can be most appropriately used and to evaluate its impact.

Although medical malpractice litigation in the United States has generated extensive professional and scholarly attention, few analyses of the issue have explored its underlying causes. This essay develops and employs an historical framework to explain the late 20th century phenomenon and concludes that widespread medical malpractice suits are the result of a combination of short-term topical causes and long-term cultural changes that are ignored or left untouched by most reform efforts. Most importantly, however, the development and proliferation of new and improved medical technologies has played a pivotal role throughout the entire history of the litigation, an effect that has become most prominent and important in the last third of the 20th century.

Automated ambulatory medical records systems (AAMRSs) have been operational for over 20 years but have not been adopted by more than a small fraction of their potential users. This paper presents a detailed analysis of the uses and benefits of the COSTAR-based AAMRS at the Harvard Community Health Plan and of the factors which have inhibited the dissemination of COSTAR. We conclude that AAMRSs have been an orphan technology and cite trends in health care that favor the future development of AAMRSs.

The authors provide a detailed overview of how the national and provincial health systems of Canada exercise control over the diffusion of medical technology. In particular, they examine the diffusion of CT scanning and the adoption of non-ionic radio contrast media. While the nature of the parliamentary system theoretically aflows the government, especially the executive, to exert more control over its policy agenda than in the United States, the authors believe that effective control is hampered by a lack of political will and insufficient “teeth” in the Ministry of Health' mandate and policy. The authors also conclude that the manipulation of reimbursement systems to encourage or discourage the diffusion of various medical technologies is not always effective, and that political clout often triumphs over rational decision making.