Background: Timely provision of information on the cost-effectiveness of innovations in health care becomes more and more important, resulting in increasing pressure on researchers to provide proof of cost-effectiveness in a short time frame. However, most of these innovations require considerable time and effort to optimally implement leading to a biased “steady state” cost-effectiveness outcome. As decision makers in health care predominantly have a short-term focus, the discrepancy between short-term study outcomes and long-term cost-effectiveness may very well lead to misguided decisions about the adoption of innovations in health care.

Methods: Factors such as learning effects, capacity constraints, and delayed time to benefit are all related to a short-run timeframe and result in inefficiencies during the implementation of an innovation. These factors and the mechanisms by which they influence the cost-effectiveness outcome are explained for three different types of healthcare innovations.

Results: As standard cost-effectiveness analysis assumes costs and effects to behave constant and representative for an innovation's entire economic lifetime, resulting cost-effectiveness outcomes might give a biased, and often overly pessimistic, reflection of the actual cost-effectiveness of an innovation. This is further amplified by the fact that short-run inefficiencies are most prevalent and impactful during an innovation's earliest stage of operation.

Conclusions: This study advocates to carefully take into account the different factors contributing to lag-time bias in the design and analysis of cost-effectiveness studies, and to communicate potential biases due to short-run inefficiencies to all stakeholders involved in the decision making process.

Objectives: When incorporating treatment effect estimates derived from a random-effect meta-analysis it is tempting to use the confidence bounds to determine the potential range of treatment effect. However, prediction intervals reflect the potential effect of a technology rather than the more narrowly defined average treatment effect. Using a case study of robot-assisted radical prostatectomy, this study investigates the impact on a cost-utility analysis of using clinical effectiveness derived from random-effects meta-analyses presented as confidence bounds and prediction intervals, respectively.

Methods: To determine the cost-utility of robot-assisted prostatectomy, an economic model was developed. The clinical effectiveness of robot-assisted surgery compared with open and conventional laparoscopic surgery was estimated using meta-analysis of peer-reviewed publications. Assuming treatment effect would vary across studies due to both sampling variability and differences between surgical teams, random-effects meta-analysis was used to pool effect estimates.

Results: Using the confidence bounds approach the mean and median ICER was €24,193 and €26,731/QALY (95%CI: €13,752 to €68,861/QALY), respectively. The prediction interval approach produced an equivalent mean and median ICER of €26,920 and €26,643/QALY (95%CI: -€135,244 to €239,166/QALY), respectively. Using prediction intervals, there is a probability of 0.042 that robot-assisted surgery will result in a net reduction in QALYs.

Conclusions: Using prediction intervals rather than confidence bounds does not affect the point estimate of the treatment effect. In meta-analyses with significant heterogeneity, the use of prediction intervals will produce wider ranges of treatment effect, and hence result in greater uncertainty, but a better reflection of the effect of the technology.

Background: This article describes the lessons learned from an international pilot assessment using the first version of the HTA Core Model® and Guidelines for rapid Relative Effectiveness Assessment (REA) of pharmaceuticals based on input from three different perspectives: the assessors, the users (health technology assessment organisations) and the marketing authorisation holder.

Methods: A pilot assessment was performed of pazopanib for the treatment of advanced or metastatic renal cell carcinoma for which 54 individuals from 22 EUnetHTA member organisations from 16 European countries gave their contribution. The work was divided in eight domain teams. Subsequently, results of these domain teams were synthesised in one pilot report. Feedback on the outcomes of the pilot was gathered throughout the project and through structured surveys.

Results: The first version of the assessment was produced in six months and consisted of 55 question and answer pairs, 8 domain reports and a synthesis section that combined the results from the different domains. The organisation of the pilot required intense coordination. Main points of criticism on the assessment were the lengthiness of the document and overlap of information throughout the assessment.

Conclusions: A reduction in the number of authoring organisations and individuals participating is necessary to avoid information overlap and increase efficiency in undertaking the assessment. Involving several organisations (e.g. five) in an in-depth review could still ensure the benefit of broad participation from various countries. The focus of a rapid REA should be on the first four domains of the Model.

Objectives: Vaccinating healthy children is proposed as a strategy to produce a herd effect and protect vulnerable groups. The Hutterite Influenza Prevention Study investigated this strategy, comparing communities with or without childhood influenza immunization programs. There are costs associated with vaccination therefore there may be a trade-off between these costs and the benefits of avoiding influenza cases. This evaluation estimates the cost-effectiveness of immunizing only healthy children in preventing cases of influenza within entire communities.

Methods: Effect data and resource utilization were collected during the trial. Cost data were collected from payer, literature and Internet sources. A two-stage bootstrap (TSB) with shrinkage correction was used to estimate average costs and effects. The incremental cost effectiveness ratio (ICER) and sample uncertainty around this estimate were calculated from the TSB results.

Results: Mean costs per patient for the treatment and control arms were $69.07 and $32.66 (difference $36.41). Mean number of influenza cases for the treatment and control arms were 0.04 and 0.27 (difference 0.23). ICER was $164.12 ($28.38, $2767.75) per case of influenza averted.

Conclusions: Immunizing healthy children for influenza is more costly, yet more effective than no immunization in preventing cases in the sample. At a cost of $164.12 to prevent a case of influenza, immunizing healthy children to protect all community members may be considered costeffective. Estimated results are conservative as the influenza season was mild and the sample population was healthy. In a more severe season with a less healthy population the ICER is expected to decrease.

Background: The matching of favorable human leukocyte antigen (HLA) combinations is rarely performed in penetrating keratoplasty procedures for primary prophylaxis of immune reactions. However, clinical studies suggest that the incidence of graft rejection decreases substantially when patients receive favorably matched grafts.

Objective: The aim of this study was to assess the cost-effectiveness of HLA matching for patients undergoing penetrating keratoplasty in everyday clinical practice.

Methods: In the absence of a randomized controlled clinical trial, we used administrative data from the Freiburg University Eye Hospital in Germany. Our study population consisted of all patients who underwent their first keratoplasty between 11/2003 and 01/2010 and for whom information on HLA histocompatibility was available. We used propensity score matching to estimate a causal effect of favorable HLA matching, parametric survival regression techniques to predict graft survival and expert opinion to model incremental cost for HLA matching. Because the availability of favorable HLA histocompatibility ultimately depends on the patients’ HLA phenotype, we modeled the incremental cost-effectiveness ratio (ICER) as a function of the probability that a patient will receive a favorably matched HLA, and used expert opinion to set a point estimate.

Results: We predicted that corneal grafts with favorable HLA matching were associated with improved rejection-free graft survival time (more than 1,000 days). We estimated the incremental cost of HLA matching at EUR 1,200 and the ICER at EUR 4.62 per additional day of graft survival.

Conclusions: The ICER of HLA matching is acceptable, given the high cost of alternative treatment and the shortage of corneal donors in Germany.

Osteoradionecrosis (ORN) is a known complication to radiation therapy for head and neck cancer with a prevalence of 5–7% among radiated patients. Treatment might include dental surgery and reconstruction of the jawbone as well as hyperbaric oxygen treatment (HBOT). HBOT takes place in a closed compartment where patients are breathing 100% oxygen under pressure for 90 minutes once a day every weekday for 6 weeks. In Denmark, HBOT is available at two facilities with very different organizational set-ups.

Objectives: The study question was whether dual-energy X-ray absorptiometry (DXA) alone is more cost-effective for identifying postmenopausal women with osteoporosis than a two-step procedure with quantitative ultrasound sonography (QUS) plus DXA. To answer this question, a systematic review was performed.

Methods: Electronic databases (PubMed, INAHTA, Health Evidence Network, NIHR, the Health Technology Assessment program, the NHS Economic Evaluation Database, Research Papers in Economics, Web of Science, Scopus, and EconLit) were searched for cost-effectiveness publications. Two independent reviewers selected eligible publications based on the inclusion/exclusion criteria. Quality assessment of economic evaluations was undertaken using the Drummond checklist.

Results: Seven journal articles and four reports were reviewed. The cost per true positive case diagnosed by DXA was found to be higher than that for diagnosis by QUS+DXA in two articles. In one article it was found to be lower. In three studies, the results were not conclusive. These articles were characterized by the differences in the types of devices, parameters and thresholds on the QUS and DXA tests and the unit costs of the DXA and QUS tests as well as by variability in the sensitivity and specificity of the techniques and the prevalence of osteoporosis.

Conclusions: The publications reviewed did not provide clear-cut evidence for drawing conclusions about which screening test may be more cost-effective for identifying postmenopausal women with osteoporosis.

Objectives: The aim of this study was to examine the gaps between researchers and policy makers in perceptions and influencing factors of knowledge translation (KT) of health technology assessment (HTA) in China.

Methods: A sample of 382 HTA researchers and 112 policy makers in China were surveyed using structured questionnaires. The questionnaires contained two sections: perceptions of HTA research and assessments of six-stage KT activities. Wilcoxon rank sum test was applied to compare the differences in these two sections between HTA researchers and policy makers. Multivariate linear regression was performed to explore KT determinants of HTA for researchers and policy makers separately.

Results: Policy makers and researchers differed in their perceptions of HTA research in all items except collaboration in research development and presentation of evidence in easy-to-understand language. Significant differences in KT activities existed in all the six stages except academic translation. Regarding KT determinants, close contact between research unit and policy-making department, relevance of HTA to policy making, and importance of HTA on policy making were considered facilitators by both groups. For researchers, practicality of HTA report and presentation of evidence in easy-to-understand language can facilitate KT. Policy makers, on the other hand, considered an overly pedantic nature of HTA research as an obstacle to effective KT.

Conclusions: Substantial gaps existed between HTA researchers and policy makers regarding the perceptions of HTA research and KT activities. There are also some differences in KT determinants by these two groups. Enhancing collaboration, promoting practicality and policy relevance of HTA research, and making HTA findings easily understood are likely to further the KT of HTA evidence.

Objectives: Real-time virtual sonography (RVS) is a promising navigation technique for percutaneous radiofrequency ablation (RFA) treatment, especially in ablating nodules poorly visualized on conventional ultrasonography (US). However, its cost-effectiveness has not been established. The purpose of this study is to evaluate the cost-effectiveness of RVS navigated RFA (RVS-RFA) relative to US guided RFA (US-RFA) in patients with small hepatocellular carcinoma (HCC) in China, from the modified societal perspective.

Methods: A state-transition Markov model was created using TreeAge Pro™ 2012. The parameters used in the model, including natural history of HCC patients, procedure efficacy and related costs, were obtained from a systematic search of literature through PubMed, EMBASE, and Science Citation Index databases. The simulated cohort was patients with solitary, small HCC (<3 cm in diameter) and Child-Pugh class A or B, whose tumors are poorly visualized in B-mode US but clearly detectable by CT or MRI.

Results: In this cohort of difficult cases, RVS-RFA was a preferred strategy saving 2,467 CNY ($392) throughout the patient's life while gaining additional 1.4 QALYs compared with conventional US guidance. The results were sensitive to the efficacy of US-RFA and RVS-RFA including complete ablation rate and local recurrence rate, the median survival for patients with progressive HCC, the probability of performing RFA for recurrent HCC, and the cost of RVS navigation, disposable needle or hospitalization.

Conclusions: RVS-RFA is a dominant strategy for patients with small HCC unidentifiable in B-mode US, in terms of cost savings and QALYs gained, relative to the conventional US-guided method.

Objectives: This paper describes the development and findings for a policy brief on “Advancing the Integration of Palliative Care into the National Health System” and the subsequent use of this report.

Methods: Key stakeholders involved with palliative care helped identify the problem and potential policy solutions to scale up these services within the health system. A working group of national stakeholder representatives and external reviewers commented on and contributed to successive drafts of the report. Research describing the problem, policy options and implementation considerations was identified by reviewing government documents, routinely collected data, electronic literature searches, contact with key informants, and reviewing the reference lists of relevant documents that were retrieved.

Results: The palliative burden is not only high but increasing due to the rise in population and life expectancy. A few options for holistic, supportive care include: Home-based care increases chances of a peaceful death for the terminally ill surrounded by their loved ones; supporting informal caregivers improves their quality of life and discharge planning reduces unscheduled admissions and has the potential to free up capacity for acute care services. A combination of strategies is needed to effectively implement the proposed options as discussed further in this article.

Conclusions: The policy brief report was used as a background document for two stakeholder dialogues whose main outcome was that a comprehensive national palliative care policy should be instituted to include all the options, which need to be integrated within the public health system. A draft policy is now in process.

Objectives: The HTA Core Model is a framework for producing health technology assessments (HTAs) in a structured format. The Model splits the content of a HTA into assessment elements. The objective is to explore the adaptability of these assessment elements in national report production in a pilot case study comparing a national HTA report and the HTA Core Model.

Methods: An on-going Dutch HTA report on endovascular repair of abdominal aortic aneurysm (EVAR) was chosen as a typical representative of a national report on medical interventions. The author of the EVAR report assessed the relevance and comprehensiveness of the assessment elements of the HTA Core Model for her work. Another researcher annotated the Core Model specific content in the EVAR report. Matching and missing content, as well as the distribution of information in the EVAR report were tabulated and analysed in joint deliberations.

Results: Forty percent of the assessment elements of the Core Model were considered relevant for the EVAR report. Some issues relevant for EVAR but missing from the Core Model were identified: they were about re-interventions, secondary prevention, subpopulations that benefit most, and the length of the hospital stay. The distribution of information differed substantially between the Code Model and the national report.

Conclusions: The assessment elements of the HTA Core Model covered most relevant questions of the national report. In order to facilitate easy adaptation of information, the distribution of information should be more consistent in the national report and the Core model.

Objectives: The objectives of this systematic literature review were to identify all published literature on wearable defibrillators, assess the wearable defibrillator's efficacy and effectiveness in general and among specific patient groups, including post-myocardial infarction, post coronary artery bypass grafting or percutaneous coronary intervention, non-ischemic cardiomyopathy, and ischemic cardiomyopathy, and to evaluate the quality of evidence.

Methods: The search and synthesis was informed by the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement, and the quality of evidence was evaluated using the Grading of Recommendations Assessment, Development and Evaluation and the Newcastle Ottawa Scale.

Results: A total of thirty-six articles and conference abstracts from thirty-three studies were included in the review. It appears that wearable defibrillator use compared with no defibrillator use reduces the chance of ventricular tachycardia and ventricular fibrillation (VT/VF) associated deaths by an absolute risk reduction of approximately 1 percent, achieved by averting approximately 4/5th of all VT/VF associated deaths. The quality of evidence was low to very low quality, such that our confidence in the reported estimates is weak.

Conclusions: To validate beneficial results, further investigation using robust study designs conducted by independent researchers is warranted.

Objectives: The aim of this study was to evaluate the efficacy and safety of rituximab and tocilizumab compared with adalimumab, etanercept, and infliximab, in patients with rheumatoid arthritis not responding to first-line treatment, and to compare the efficacy and safety of rituximab versus tocilizumab in patients not responding to anti-tumor necrosis factor α (anti-TNF) therapy.

Methods: A literature search of randomized controlled trials, controlled clinical trials, and systematic reviews was performed to evaluate efficacy and safety of rituximab and tocilizumab.

Results: Twenty-four RCTs were included in this systematic review with 6,357 participants; 3,450 treated with biological DMARD and 2,907 with standard care. In patients not responding to first-line treatment, rituximab shows lower response rate in at least 50 percent improvement in the American College of Rheumatology criteria (ACR50) and ACR70 compared with etanercept, at 6 months of follow-up. Tocilizumab shows higher ACR70 response rate compared with infliximab, at the same follow-up time. Other results showed no significant differences. Indirect comparisons between rituximab and tocilizumab in patients not responding for at least one anti-TNF, shows higher ACR20 response rate for tocilizumab at 6 months of follow-up. Regarding safety, adalimumab and etanercept were associated with significant fewer withdrawals due to adverse events compared with infliximab.

Conclusions: Considering efficacy, safety, and the availability of 3 anti-TNFs in the National Medicines Formulary (adalimumab, etanercept, and infliximab), it seems appropriate to remove infliximab from the coverage, and introduce tocilizumab for patients not responding for at least one anti-TNF.

Objectives: Evidence exists that telehealth interventions (e.g., telemonitoring, telediagnostics, telephone care) in disease management for chronic heart failure patients can improve medical outcomes, and we aim to give an overview of the cost-effectiveness of these interventions.

Methods: Based on the literature search on “heart failure” in combination with “cost” and “telehealth” we selected 301 titles and abstracts. Titles and abstracts were screened for a set of inclusion criteria: telehealth intervention, heart failure as the main disease, economic analysis present and a primary study performed. In the end, thirty-two studies were included for full reading, data extraction, and critical appraisal of the economic evaluation.

Results: Most studies did not present a comprehensive economic evaluation, consisting of the comparison of both costs and effects between telehealth intervention and a comparator. Data on telehealth investment costs were lacking in many studies. The few studies that assessed costs and consequences comprehensively showed that telehealth interventions are cost saving with slight improvement in effectiveness, or comparably effective with similar cost to usual care. However, the methodological quality of the studies was in general considered to be low.

Conclusions: The cost-effectiveness of telehealth in chronic heart failure is hardly ascertained in peer reviewed literature, the quality of evidence is poor and there was a difficulty in capturing all of the consequences/effects of telehealth intervention. We believe that without full economic analyses the cost-effectiveness of telehealth interventions in chronic heart failure remains unknown.

Objectives: To date, IT strategic planning has been mostly theory-based with limited information on “best practices” in this area. This study presents the process and outcomes of IT strategic planning undertaken at a pediatric hospital (PH) in Canada.

Methods: A five-stage sequential and incremental process was adopted. Various tools / approaches were used including review of existing documentation, internal survey (n = 111), fifteen interviews, and twelve workshops.

Results: IT strategic planning was informed by 230 individuals (12 percent of hospital community) and revealed consistency in the themes and concerns raised by participants (e.g., slow IT projects delivery rate, lack of understanding of IT priorities, strained communication with IT staff). Mobile and remote access to patients’ information, and an integrated EMR were identified as top priorities. The methodology and used approach revealed effective, improved internal relationships, and ensured commitment to the final IT strategic plan. Several lessons were learned including: maintaining a dynamic approach capable of adapting to the fast technology evolution; involving stakeholders and ensuring continuous communication; using effective research tools to support strategic planning; and grounding the process and final product in existing models.

Conclusions: This study contributes to the development of “best practices” in IT strategic planning, and illustrates “how” to apply the theoretical principles in this area. This is especially important as IT leaders are encouraged to integrate evidence-based management into their decision making and practices. The methodology and lessons learned may inform practitioners in other hospitals planning to engage in IT strategic planning in the future.

Background: Contrast-induced acute kidney injury (CI-AKI) is defined as a deterioration in renal function after administration of radiologic iodinated contrast media (CM). Iodixanol, showed a lower CI-AKI incidence than low-osmolar contrast media (LOCM). A cost-effectiveness analysis was performed comparing iodixanol and LOCM in intravenous (IV) setting in Italy.

Methods: A Markov model was developed. Patients moved across four health states: CI-AKI free, CI-AKI, myocardial infarction, and death. The simulation horizon was lifetime with 1-month cycles. Costs and outcomes were discounted at 3.5 percent rate. CI-AKI incidence was considered from published literature across different definitions. Cost-effectiveness of iodixanol was assessed in terms of incremental cost per life-year gained. Net monetary benefit (NMB) was also calculated. Both deterministic and probabilistic sensitivity analyses were performed.

Results: Base-case results showed an average survival increase of 0.51 life-years and a savings of €7.25 for iodixanol versus LOCM. The cost-effectiveness of iodixanol was confirmed when other scenarios were explored, such as varying CI-AKI definition, sub-populations with specified risk factors, CM hospital bids prices, and inclusion of adverse drug reactions of allergic nature. An NMB ranging between €6,007.25 and €30,007.25 was calculated.

Conclusion: Base-case results show that IV iodixanol is cost-effective compared with LOCM in the Italian clinical setting of a hospital computed tomography radiology practice. However, some caution is due, mainly linked to inherent limitations of the modeling technique and to the lack of agreement on CI-AKI incidence data in the clinical literature.

Objectives: The purpose of the European network for Health Technology Assessment (EUnetHTA) is to make HTA agencies collaborate sharing methods and tools thus avoiding duplication of evaluative efforts and allowing resource savings. From 2010 to 2012, the activities of the network were carried out through EUnetHTA Joint Action 1 and Work Package 4 Strand B aimed at producing two Core HTAs with two main objectives: to test the Web based Core model and the collaborative working models. Our objective in this article is to give an historical record of the Work Package activities highlighting what worked and what did not in the collaboration of researchers’ groups coming from different agencies.

Methods: A retrospective description of all the steps for the joint production of the two Core HTAs is provided starting from the first step of selecting technologies of common interest. Primary researchers’ views on the whole process have been collected through a semi-structured telephonic interview supported by a questionnaire. Coordinators views were gathered during internal meetings and validated.

Results: Majority of respondents thought topic selection procedure was not clear and well managed. About collaborative models, small groups were seen to enable more exchange, whatever the model. According to coordinators, loss of expertise and experience during the production process, different languages, and novelty of the Online Tool were main barriers.

Conclusions: Lessons learned from this first experience in Joint Action 1 paved the path for the collaboration in Joint Action 2, as it allowed enhancements and changes in models of collaborations and coordination.

Objectives: Selecting technologies for formal assessment poses a great challenge to health technology assessment agencies. This study aims to contribute to the creation of a reference framework for the identification, filtering, and prioritization of new and emerging technologies which could be demanded in clinical practice within the next 1–2 years.

Methods: Technologies were identified using a prevalidated systematic Medline strategy. They were classified by medical specialty and then sent to selected professionals belonging to the medical units or areas responsible for their application, until there was a minimum of three participants per health care setting. A self-administered questionnaire was drawn up and health professionals were asked to: (1) assess the degree of innovation of the technologies, and (11) score their foreseeable clinical impact on the basis of predefined prioritization criteria (n = 4). Intra-rater reliability was analyzed using the intraclass correlation coefficient (ICC).

Results: The Medline search yielded 246 potentially relevant technologies. When analyzed by health care area or unit, sixty-eight were deemed to be high-impact innovative technologies (median score >6), with ICCs ranging from 0.03 to 0.83. The final list resulting from the aggregate analysis comprised fifty-one technologies.

Conclusions: This study constitutes an innovative contribution to horizon scanning, providing a systematic and reproducible basis for the identification and selection of relevant new and emerging technologies based on the views and values of health professionals involved in their use. In our opinion, the current proposal could be helpful and useful to many other organizations worldwide, serving to complement already existing strategies.

Objectives: Mobile systems are widely adopted in healthcare services. Mobile reporting systems have been recently introduced for social home care by municipalities in Sweden. The study aims to assess the benefits of using these systems.

Methods: We followed an expert survey approach. Data were collected by means of telephone interviews with the experts in charge of managing and implementing the systems at the municipalities. In addition, several workshops were organized for assessing the economic value of the systems at one municipality. We performed thematic analysis and cost-benefit analysis of the data.

Results: The thematic analysis showed the three main benefits of using the mobile reporting systems in social home care: municipal benefits, care providers’ benefits, and care recipients’ benefits. The cost-benefit analysis indicated that the systems could bring substantial long-term economic value for municipalities. The results also revealed the difficulties encountered at the early stage of the deployment and implementation of the systems.

Conclusions: The mobile systems yield benefits for all the actors, that is, municipalities, care recipients and care providers. These enhance the public-private coordination and cooperation in social home care in Sweden. The municipalities are called upon to address change management and technical challenges in the implementation.

Objectives: The aim of this study was to estimate direct and indirect excess costs attributable to stroke in Sweden in 2009 and to compare these with similar estimates from 1997.

Methods: Data on first-ever stoke admissions in the first half of 2009 from the Swedish national stroke register (RS) were used for cost calculations and compared with results from 1997 also using RS data. A societal perspective was taken including the acute and follow-up phase, rehabilitation, stroke re-admissions, drugs, home- and residential care services for activities of daily life (ADL) support, and indirect costs for premature death and productivity losses (2009 prices). Survival was extrapolated to estimate the lifetime present value cost of stroke.

Results: The societal lifetime present value cost for stroke in 2009 was €68,800 per patient (ADL support: 59 percent; productivity losses: 21 percent). Women had higher costs than men in all age groups as a result from greater need for ADL support. Patients treated at a stroke unit indicated low incremental cost per life-year gained compared with those who had not. The total lifetime cost increased between 1997 and 2009. Hospitalization costs per patient were stable, while long-term costs for home- and residential care services increased.

Conclusions: Changes in patient characteristics, longer expected survival, and possibly in the Swedish stroke care, have led to higher annual and lifetime costs per patient in 2009 compared with 1997. A comprehensive national stroke care performance register like RS may be suitable for health economic assessments.