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: 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.

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.

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 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.

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: 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: 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.

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.

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.

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.

Objectives: Extended-release therapies avoid the need for children with attention-deficit/hyperactivity disorder (ADHD) to take medication at school. Recently a transdermal delivery system has been developed which can allow symptom control all day long but with greater dosing flexibility. This study explored the parents' preferences regarding oral and transdermal therapy.

Methods: A nonsystematic and qualitative literature review and in-depth interviews with parents and physicians helped identify salient treatment attributes for a discrete choice experiment. Treatment attributes included mode of administration (tablet or transdermal), speed of onset (30–90 min); duration (lasts until 3–9 pm) and ability to tailor the drug to different needs (no flexibility, limited flexibility, easy to adjust to different days). A convenience sample of parents of children treated for ADHD (n = 200) were recruited using a recruitment agency. Data were analyzed using generalized estimating equations (GEE).

Results: Parents' preferred once-a-day oral therapy (odds ratio [OR] = 1.76 [95 percent confidence interval {CI}, 1.43 – 2.18]); rapid speed of onset (OR = 1.22 [95 percent CI, 1.07 – 1.39]), and symptom control until 9 pm (OR = 3.79 [95 percent CI, 2.98 – 4.82]). Analyses identified that 30 percent of parents preferred transdermal treatment and this subgroup preferred treatments with a fast onset of action.

Conclusions: This survey demonstrates that parents of ADHD children have different preferences for the ADHD treatments prescribed for their children. A distinct subgroup of parents prefer the transdermal therapy. These parents were less likely to be working and so monitoring compliance and doing after school activities may have been easier.

This paper discusses the challenges facing health technology assessment (HTA) in Europe, based on an explicit analysis of the characteristics of an “optimal” HTA system. It has three objectives: a) to elaborate an explicit system of policy goals and the characteristics of an optimal HTA system that facilitates the achievement of these goals; b) to identify the general institutional incentive barriers (government and market failures) that prevent the attainment of an optimal HTA system in Europe; and c) to argue that evaluation of the implications of health technologies for equity and inequality in health is an essential part of this optimal system and a considerable challenge for HTA decision makers, especially as national governments realign policy toward equity goals.

Objectives: A patient reported outcome (PRO) is “any report of the status of a patient's health condition that comes directly from the patient without interpretation of the patient's response by a clinician or anyone else” (USFDA 2009). PROs are discussed widely, and many regard the patients’ perspective on treatment benefit as very valuable. Although many PROs have shown satisfactory measurement properties including reliability, validity, and responsiveness, there is great concern about risk of bias, that is, in clinical trials.

Methods: Differences in perspectives of PRO measurement in rare diseases are given arising from methodology, clinical, HTA, and patient advocacy views.

Results: PROs are playing an important role in dealing with treatment benefit especially in small sample size as occurring often in rare diseases. Challenges remain especially regarding lack of responsiveness of generic measures, limited capture of all patient relevant aspects, study design and high risk of bias.

Conclusions: PROs seem a valuable instrument to detect patient relevant aspects in rare diseases. They should be seen in addition to other approved assessment methods as randomized controlled trials but not as their substitute.

Objectives: Endobronchial ultrasound (EBUS), encompassing endobronchial ultrasound transbronchial needle aspiration (EBUS-TBNA) and Endobronchial ultrasound transbronchial lung biopsy (EBUS-TBLB) has been proven to be a useful modality in the staging and diagnosis of lung cancer. However, there are limited publications on the cost-effectiveness of EBUS and no economic evaluations relevant to the Singapore setting. An economic evaluation using our hospital's data was used to assess the cost implications of EBUS substituting where clinically appropriate: transthoracic needle aspiration; (TTNA), fluoroscopy-guided transbronchial lung biopsy (TBLB), and mediastinoscopy in the diagnosis and staging of lung cancer.

Methods: Relationship between the clinical and economic implications of alternative modalities was modeled using data inputs that were relevant to the Singapore setting. Two decision analytic models were constructed to evaluate the cost of EBUS compared with TTNA, TBLB, and staging mediastinoscopy. Only direct costs were imputed.

Results: In the base–case analysis, TTNA was the most economical strategy (SGD3,335 = US$2,403) where clinically suitable for the diagnosis of lung cancer as compared to the other options: TBLB (SGD4,499) and EBUS-TBLB (SGD4,857). On the other hand, EBUS-TBNA resulted in expected cost savings of SGD1,214 per positive staging of lung cancer as compared to mediastinoscopy.

Conclusions: The use of EBUS-TBNA could result in cost savings of SGD1,214 per positive staging of lung cancer as compared to mediastinoscopy. Whereas TTNA was the most economical intervention for the diagnosis of lung cancer as compared to the other options, its main limitation lies in its suitability only for peripheral lung lesions and high complication rate.

Objectives: The aim of this study was to obtain information on methods used to measure health technology assessment (HTA) influence, decisions that were influenced, and outcomes linked to HTA.

Methods: Electronic databases were used to locate studies in which HTA influence had been demonstrated. Inclusion criteria were studies that reliably reported consideration by decision makers of HTA findings; comparative studies of technology use before and after HTA; and details of changes in policy, health outcomes, or research that could be credibly linked to an HTA.

Results: Fifty-one studies were selected for review. Settings were national (24), regional (12), both national and regional (3) hospitals (9), and multinational (3). The most common approach to appraisal of influence was review of policy or administrative decisions following HTA recommendations (51 percent). Eighteen studies (35 percent) reported interview or survey findings, thirteen (26 percent) reviewed administrative data, and six considered the influence of primary studies. Of 142 decisions informed by HTA, the most common types were on routine clinical practice (67 percent of studies), coverage (63 percent), and program operation (37 percent). The most frequent indications of HTA influence were on decisions related to resource allocation (59 percent), change in practice pattern (31 percent), and incorporation of HTA details in reference material (18 percent). Few publications assessed the contribution of HTA to changing patient outcomes.

Conclusions: The literature on HTA influence remains limited, with little on longer term effects on practice and outcomes. The reviewed publications indicated how HTA is being used in different settings and approaches to measuring its influence that might be more widely applied, such as surveys and monitoring administrative data.