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: Untreated obstructive sleep apnea-hypopnea syndrome (OSAHS) is associated with excessive daytime sleepiness, increased risk of cardiovascular (CV) disease, and road traffic accidents (RTAs), which impact survival and health-related quality of life. This study, funded by the French National Authority for Health (HAS), aimed to assess the cost-effectiveness of different treatments (i.e., continuous positive airway pressure [CPAP], dental devices, lifestyle advice, and no treatment) in patients with mild-to-moderate OSAHS in France.

Methods: A Markov model was developed to simulate the progression of two cohorts, stratified by CV risk, over a lifetime horizon. Daytime sleepiness and RTAs were taken into account for all patients while CV events were only considered for patients with high CV risk.

Results: For patients with low CV risk, incremental cost-effectiveness ratio (ICER) of dental devices versus no treatment varied between 32,976 EUR (moderate OSAHS) and 45,579 EUR (mild OSAHS) per quality-adjusted life-year (QALY), and CPAP versus dental devices, above 256,000 EUR/QALY. For patients with high CV risk, CPAP was associated with a gain of 0.62 QALY compared with no treatment, resulting in an ICER of 10,128 EUR/QALY.

Conclusion: The analysis suggests that it is efficient to treat all OSAHS patients with high CV risk with CPAP and that dental devices are more efficient than CPAP for mild-to-moderate OSAHS with low CV risk. However, out-of-pocket costs are currently much higher for dental devices than for CPAP (i.e., 3,326 EUR versus 2,430 EUR) as orthodontic treatment is mainly non-refundable in France.

Objectives: Do utility scores based on patient preferences and scores based on community preferences agree? The purpose is to assess agreement between directly measured standard gamble (SG) utility scores and utility scores from the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3) systems.

Methods: Patients were assessed repeatedly throughout the process of waiting to see a surgeon, waiting for surgery, and recovery after total hip arthroplasty (THA). Group mean scores are compared using paired t-tests. Agreement is assessed using the intraclass correlation coefficient (ICC).

Results: The mean SG, HUI2, and HUI3 (SD) scores at assessment 1 are 0.62 (0.31), 0.62 (0.19), and 0.52 (0.21); n=103. At assessment 2, the means are 0.67 (0.30), 0.68 (0.30), and 0.58 (0.22); n=84. There are no statistically significant differences between group mean SG and HUI2 scores. Mean SG and HUI3 scores are significantly different. ICCs are low.

Conclusions: At the mean level for the group, SG and HUI2 scores match closely. At the individual level, agreement is poor. HUI2 scores were greater than HUI3 scores. HUI2 and HUI3 are appropriate for group level analyses relying on community preferences but are not a good substitute for directly measured utility scores at the individual leve.

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.

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: 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.

Objectives: Australia relies on managed entry agreements (MEAs) for many medicines added to the national Pharmaceutical Benefits Scheme (PBS). Previous studies of Australian MEAs examined public domain documents and were not able to provide a comprehensive assessment of the types and operation of MEAs. This study used government documents approved for release to examine the implementation and administration of MEAs implemented January 2012 to May 2016.

Methods: We accessed documents for medicines with MEAs on the PBS between January 2012 and May 2016. Data were extracted on Anatomical Therapeutic Classification (ATC), type of MEA (financial, financial with outcomes, outcomes, and subcategories within each group), implementation and administration methods, source of MEA recommendation, and type of economic analysis.

Results: Of all medication indication pairs (MIPs) recommended for listing, one-third had MEAs implemented. Our study of eighty-seven MIPs had 170 MEAs in place. The Government's expert health technology assessment (HTA) committee recommended MEAs for 90 percent of the eighty-seven MIPs. A total of 81 percent of MEAs were simple financial agreements: the majority either discounts (32 percent) or reimbursement caps (43 percent). Outcome-based MEAs were least common (5 percent). Ninety-two percent of MEAs were implemented and operated through legal agreements. Approximately half of the MIPs were listed on the basis of accepted claims of cost-minimization. Forty-nine percent of medicines were in ATC L group.

Conclusion: Advice from HTA evaluations strongly influences the implementation of ways to manage uncertainties while providing access to medicines. The government relied primarily on simple financial agreements for the managed entry of medicines for which there were perceived risks.

The papers on the 16 European countries presented in this issue show their great diversity of health systems. Health technology assessment (HTA) has been institutionalized in a number of members of the European Union and has a growing impact on health policy. A remaining challenge is to see HTA visible and useful at the European level, which requires more active coordination of national and regional activities. A network of HTA programs and researchers has been established. The HTA-Europe Steering Committee has suggested a number of actions by the European Commission to promote further coordination. The most important conclusion of the HTA-Europe report is that a permanent coordinating structure is needed at the European level.

Objectives: Magnetic resonance imaging (MRI) and computerized tomography (CT) scanners are high-tech equipment with the highest utilizations and expenditures in Taiwan. This study investigates the factors that affect MRI and CT utilization, such as whether supply-side factors have more impact than demand-side factors, and the effect of utilization control policies.

Methods: This study used the nationwide CT and MRI claim data for the years 1998 to 2001 from the Bureau of National Health Insurance (BNHI) in Taiwan. Multiple regression analysis was the statistical method used to determine the relative factors that influence CT and MRI utilization.

Results: CT : population ratio, hospital-based physician : population ratio, female ratio, pediatric (≤14 years) : population ratio, and family income significantly influenced CT utilization. MRI : population ratio and hospital-based physician : population ratio, female ratio, and family income significantly influenced MRI utilization. However, the proportion of the aged (≥65 years of age) did not significantly influence CT or MRI utilization after controlling for other factors. The rates of CT and MRI utilization for the outpatient and inpatient settings and the repeated uses of CT or MRI have decreased significantly after a utilization review was implemented by BNHI, which suggests that CT and MRI have been overused.

Conclusions: Increases in supply-side factors significantly increase CT/MRI utilization, and supply-side factors impact CT/MRI utilization more than demand-side factors. The results show that the utilization instant report policy effectively decreases repeated uses of CT/MRI.

Objectives: Several instruments for measuring health-related quality of life (HRQOL) have been developed, and others are under construction. The problem is whether the different HRQOL measures show comparable results. We first compared the functional relationship of the Nottingham Health Profile (NHP) and the 15-dimensional measure of HRQOL (15D) in hip and knee replacement patients. The hypothesis was that condition or intervention does not affect the functional relationship between NHP and 15D changes.

Methods: We assessed the agreement of the instruments by comparing observed changes in the 15D and its fitted values derived by regressing the 15D by the NHP dimensions. Patients (n = 452) were recruited consecutively from seven Finnish orthopedic departments during April 1991–May 1992. HRQOL was measured prior to surgery and 6, 12, and 24 months postoperatively.

Results: There was a different functional relationship between the HRQOL instruments in hip and knee patients; they agreed upon the direction of changes in HRQOL in 84% and showed opposite signs in 12%. The NHP showed significantly more improvement in quality of life than the 15D.

Conclusions: The two instruments were in slight but significant disagreement. Estimates of effectiveness can vary according to the instrument used. Comparisons of effectiveness in healthcare programs measured by differing instruments need information on the functional discrepancies between the instruments in the conditions and interventions in which they are applied.

Objectives: A recent study showed that estimates of cost-effectiveness submitted to National Institute for Health and Clinical Excellence (NICE) by manufacturers had significantly lower incremental cost-effectiveness ratios (ICERs) than those submitted by university-based Assessment Groups. This study extends that analysis.

Methods: Data were abstracted from relevant NICE documentation for thirty-two of eighty-two possible appraisals.

Results: The results from the analysis showed that sources of the difference in ICERs appear to be the effectiveness estimates relating to the comparator technology and the cost estimates relating to the technology under evaluation. That is, manufacturers estimated lower average benefits for the comparator technology and lower costs relating to the technology under evaluation compared with estimates submitted by the Assessment Groups.

Conclusions: These findings may be particularly important, given the introduction of the “Single Technology Appraisal.” Considerable difficulties were encountered when undertaking this study, highlighting, above all else, the complexity of explaining why results from economic evaluations purporting to answer the same question diverge.

Italy has a national health service (SSN) dating to 1978. Italy's system of government is characterized by a rather high degree of decentralization of power, and the health system is likewise decentralized. Most of the responsibilities for health care have been ceded to the regions. The state retains only limited coordinating and supervisory powers. The state has a financial responsibility for the national health service, but state contributions are limited and expenditures in excess of this made by the region must be financed from other sources. Health reforms of 1992–93 aimed at making the regions more sensitive to the need to control aggregate expenditure and to monitor measures to promote efficiency, quality, and citizen-patient satisfaction. The diffusion of individual health technologies has been relatively uncontrolled in many regions in Italy, although tight central constraints on capital spending have contained diffusion of new technology. Regulation of placement of services is a planning function and is the responsibility of both the Ministry of Health and the regions. Health technology assessment (HTA) activities have been expanding since the early 1990s, but these activities tend to be untargeted, uncoordinated, and without priorities. Nonetheless, the principal actors in the SSN at national, regional, and local levels are becoming more sensitive to the need to apply criteria of clinical and cost-effectiveness and to be more rigorous in deciding what services to guarantee. There are reasons to be guardedly optimistic about the future of HTA in Italy.

Objectives: The aim of this study was to assess empirically the social and ethical risks associated with implantable radio-frequency identification (RFID) devices.

Methods: Qualitative research included observational studies in twenty-three U.S. hospitals that have implemented new patient identification systems and eighty semi-structured interviews about the social and ethical implications of new patient identification systems, including RFID implants.

Results: The study identified three primary social and ethical risks associated with RFID implants: (i) unfair prioritization of patients based on their participation in the system, (ii) diminished trust of patients by care providers, and (iii) endangerment of patients who misunderstand the capabilities of the systems.

Conclusions: RFID implants may aggravate inequalities in access to care without any clear health benefits. This research underscores the importance of critically evaluating new healthcare technologies from the perspective of both normative ethics and empirical ethics.

Objectives: The objective of this study was to develop a model to assess the cost-effectiveness of a new treatment for patients with depression.

Methods: A Markov simulation model was constructed to evaluate standard care for depression as performed in clinical practice compared with a new treatment for depression. Costs and effects were estimated for time horizons of 6 months to 5 years. A naturalistic longitudinal observational study provided data on costs, quality of life, and transition probabilities. Data on long-term consequences of depression and mortality risks were collected from the literature. Cost-effectiveness was quantified as quality-adjusted life-years (QALYs) gained from the new treatment compared with standard care, and the societal perspective was taken. Probabilistic analyses were conducted to present the uncertainty in the results, and sensitivity analyses were conducted on key parameters used in the model.

Results: Compared with standard care, the new hypothetical therapy was predicted to substantially decrease costs and was also associated with gains in QALYs. With an improved treatment effect of 50 percent on achieving full remission, the net cost savings were 20,000 Swedish kronor over a 5-year follow-up time, given equal costs of treatments. Patients gained .073 QALYs over 5 years. The results are sensitive to changes in assigned treatment effects.

Conclusions: The present study provides a new model for assessing the cost-effectiveness of treatments for depression by incorporating full remission as the treatment goal and QALYs as the primary outcome measure. Moreover, we show the usefulness of naturalistic real-life data on costs and quality of life and transition probabilities when modeling the disease over time.

Objectives: To calculate the costs of blood collection, testing, storage, and transfusion in Greece.

Methods: Costing information was collected from two large public hospitals, in Athens and Crete, that also act as blood banks. Given that private health care accounts for 40 percent of total health spending, the same costs were also considered in a private setting by collecting key reagent cost data from a leading private hospital in Athens. Mainly direct costs were considered (advertising campaigns, personnel, storage and maintenance, reagent costs, transportation costs from blood bank to end-use hospitals, and cross-matching and transfusion costs in receiving hospitals) and some indirect costs (opportunity cost of blood donorship).

Results: Captive donorship accounts for over 50 percent of the national blood supply. A unit of blood transfused would cost between €294.83 and €339.83 in public hospitals and could reach €413.93 in a private facility. This figure may be an underestimate, as it excludes opportunity costs of blood transfusion for patients and the healthcare system.

Conclusions: Blood has a significant cost to the health system. Policy makers and practitioners should encourage its rational use, build on current policies to further improve collection and distribution, encourage further volunteer donorship in Greece, and also consider alternatives to blood where the possibility exists.

Objectives: Several studies have shown that a key determinant of successful health technology assessment (HTA) uptake is a clear, fair, and consistent decision-making process for the approval and introduction of health technologies. The aim of this study was to gauge healthcare providers' and managers' perceptions of local level decision making and determine whether these processes offer a conducive environment for HTA. An Area Health Service (AHS) aimed to use the results of this study to help design a new process of technology assessment and decision making.

Methods: An online survey was sent to all health service managers and healthcare providers working in one AHS in Sydney, Australia. Questions related to perceptions of current health technology decisions in participants' own institution/facility and opinions on key criteria for successful decision-making processes.

Results: Less than a third of participants agreed with the statements that local decision-making processes were appropriate, easy to understand, evidence-based, fair, or consistently applied. Decisions were reportedly largely influenced by total cost considerations as well as by the central state health departments and the Area executive.

Conclusions: Although there are renewed initiatives in HTA in Australia, there is a risk that such investments will not be productive unless policy makers also examine the decision-making contexts within which HTA can successfully be implemented. The results of this survey show that this is especially true at the local level and that any HTA initiative should be accompanied by efforts to improve decision-making processes.