To the Editor:

In a recent study in this Journal, Drummond and colleagues (5) attempt to address the question “Whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs” before proposing “a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.”

The different models of consensus development conferences (CDC5) are analyzed in relation to democratic technology assessment. In some countries CDCs are mainly concerned with influencing the quality of clinical practice and thus are dominated by medical experts. In other countries, CDCs are directed towards the public and the decision makers on the political and administrative level. The Danish experience demonstrates that CDCs may be a forceful social technology with a strong potential to influence decisions about medical as well as non-medical technology

This study describes decision making regarding the acquisition of technology in 12 major medical centers. The financial impact of a project was the most widely cited criterion of decision, but financial considerations were less important than either the impact of a technology on the quality of clinical care or its contribution to teaching and research. Rarely were criteria set out explicitly or in advance. Although exemplary models exist, the technology assessment process at most institutions is described as “political,” “informal,” or “ad hoc.”

Objectives: The aim of this study is to determine the relative value and contribution of searching different sources to identify adverse effects data.

Methods: The process of updating a systematic review and meta-analysis of thiazolidinedione-related fractures in patients with type 2 diabetes mellitus was used as a case study. For each source searched, a record was made for each relevant reference included in the review noting whether it was retrieved with the search strategy used and whether it was available but not retrieved. The sensitivity, precision, and number needed to read from searching each source and from different combinations of sources were also calculated.

Results: There were 58 relevant references which presented sufficient numerical data to be included in a meta-analysis of fractures and bone mineral density. The highest number of relevant references were retrieved from Science Citation Index (SCI) (35), followed by BIOSIS Previews (27) and EMBASE (24). The precision of the searches varied from 0.88% (Scirus) to 41.67% (CENTRAL). With the search strategies used, the minimum combination of sources required to retrieve all the relevant references was; the GlaxoSmithKline (GSK) website, Science Citation Index (SCI), EMBASE, BIOSIS Previews, British Library Direct, Medscape DrugInfo, handsearching and reference checking, AHFS First, and Thomson Reuters Integrity or Conference Papers Index (CPI).

Conclusions: In order to identify all the relevant references for this case study a number of different sources needed to be searched. The minimum combination of sources required to identify all the relevant references did not include MEDLINE

Objectives: This study reports the cost-effectiveness of interventions with nonsignificant differences in effect, and considers reporting of cost-effectiveness in situations where nonsignificant differences arise in some but not all end points.

Methods: Data on costs and effects associated with three end points (adequate assessment, risk factors, and life-years) were derived from a trial of methods to promote secondary prevention of coronary heart disease. Incremental cost per life-year gained figures were calculated, and the uncertainty around these was displayed on cost-effectiveness planes in the form of ellipses.

Results: There was a significant difference in one of the intermediate end points (adequate assessment) but nonsignificant differences in the other intermediate end point (risk factors) and the final end point (life-years). Estimation of cost per life-year figures revealed the cost-effectiveness of the interventions to be unfavorable.

Conclusions: Cost-effectiveness ratios based on final end points should be calculated even in situations where nonsignificant differences in life-years arise, to avoid publication bias and to provide decision makers with useful information. Uncertainty in the incremental cost-effectiveness ratios should be estimated and presented graphically.We thank Rebecca Turner for comments on an earlier draft; Professor Paul Fenn for permission to adapt an existing model; and Dr. Andy Briggs for advice on interpretation and presentation of results and comments on an earlier draft. In addition to the authors of the paper, the ASSIST trial collaborative group includes G. Fowler, E. Fullard, A. Fuller, M. Murphy, A. Neil, T. Schofield, R. Turner, S. Thompson, F. Wells, R. Wiles, and L. Youngman. This research was funded by a grant from the national research and development program grant of the NHS Executive.

Objectives: The aims of this study were to estimate preferences and willingness-to-pay (WTP) for genetic screening for CYP2D6 polymorphisms among a group of former and currently depressed patients.

Methods: A Web-based discrete choice questionnaire was sent to 89 respondents, age 18–65. Four attributes were included: (i) shifts in antidepressant medication before symptom relief, (ii) time with antidepressant medication without symptom relief, (iii) time with antidepressant medication without symptoms but with adverse side-effects, (iv) cost of genetic screening. We used a switching model with two scenarios, one representing patients’ own treatment history and the other a treatment scenario with genetic screening.

Results: In a main-effects model involving the four attributes all coefficients had the expected sign, indicating that as the number of shifts, price or time without symptom relief, and/or dosage-adjustments increased, the likelihood of choosing the screening test decreased. Price and number of shifts in medicine were significant. Marginal WTP for 5 percent probability of a reduction of one in antidepressant shifts was DKK2,599 (€350).

Conclusions: Patients value reductions in shifts in antidepressants and price when choosing between genetic screening and no screening. They do not focus on how the reductions are provided, nor do they value the genetic information the test provides irrespective of its effect on outcome. Given, that the test is able to provide a reduction of one shift in the number of antidepressant shifts with a probability of 5 percent, WTP for the test exceeds its cost.

Objectives: In this study, we explored crucial factors that explain a person's attitude toward and his or her assessment of telemedical systems. Special focus lies on the link between the perspective of physicians (telemedicine users) and technicians (telemedicine designers) to find potential barriers hindering the broad application of telemedical systems in hospitals and doctors’ offices.

Methods: A survey among medical professionals (n = 34), technical professionals (n = 39), and a control group (n = 44) was conducted. The collected data were assessed in terms of domain knowledge, attitudes toward telemedicine, and potential implementation barriers.

Results: Participants favored the conventional method over telemedical monitoring in regards to privacy, security, and time efficiency. In contrast, telemedicine was preferred with reference to efficiency of data analysis, long-term care, and emergency adequacy. Significant differences between the professional groups were found regarding perceived cost effectiveness, patients’ compliance, privacy protection, and false alarm sensitivity. Medical professionals exhibited the most reluctance toward using telemedical treatments.

Conclusions: The perceived drawbacks are attributed to a general uncertainty about the reliability of telemedical systems, in combination with concerns about personal data privacy, security, and loss of control. The reported fear of not being able to correctly use and handle the systems assumes a low usability of devices. To acquaint medical professionals with the benefits and limitations of telemedical systems, telemonitoring and tele-treatment should be included in the education of medical personnel at an early stage.

Objectives: To describe the development of the International Network of Agencies for Health Technology Assessment (INAHTA) and its activities.

Methods: Review of literature material and other documents produced by or relating to INAHTA.

Results: INAHTA includes organizations that provide health technology assessment (HTA) advice to governments and receive most of their funding from public sources. In early 2009, there were forty-six members from twenty-seven countries, including both national and regional agencies. Interaction with other organizations includes links to Health Technology Assessment International (HTAi), the European Union Network for Health Technology Assessment (EUnetHTA), PAHO (the Pan American Health Organization, and the World Health Organization (WHO). An important feature of INAHTA is the routine interaction of its members with public sector decision makers. Output from the network has included guidelines and frameworks on HTA, reports of surveys, and joint projects.

Conclusions: INAHTA has developed as a global point of contact and information for those with interests in HTA.

Objectives: There is evidence that breastmilk feeding reduces mortality and short and long-term morbidity among infants born too soon or too small. The aim of this study was to evaluate the cost-effectiveness of enhanced staff contact for mothers with infants in a neonatal unit with a birth weight of 500–2,500 g from the perspective of the UK National Health Service.

Methods: A decision-tree model linked clinical outcomes with long-term health outcomes. The study population was divided into three weight bands: 500–999 g, 1000–1,749 g, and 1,750–2,500 g. Clinical and resource use data were obtained from literature reviews. The measure of benefit was quality-adjusted life-years. Uncertainty was evaluated using cost-effectiveness acceptability curves and sensitivity analyses.

Results: The intervention was less costly and more effective than the comparator in the base–case analysis for each birth weight group. The results were quite robust to the sensitivity analyses performed.

Conclusions: This is the first economic evaluation in this complex field and offers a model to be developed in future research. The results provide preliminary indications that enhanced staff contact may be cost-effective. However, the limited evidence available, and the limited UK data in particular, suggest that further research is required to provide results with confidence.

Objectives: This study aims to compare the impact of two different sources of resource use, self-report versus care provider registrations, on cost and cost utility.

Methods: Data were gathered for a cost-effectiveness study performed alongside a 2-year randomized controlled trial evaluating the effect of an INTERdisciplinary COMmunity-based management program (INTERCOM) for patients with chronic obstructive pulmonary disease (COPD). The program was offered by physiotherapists, dieticians and respiratory nurses. During the 2-year period, patients reported all resource use in a cost booklet. In addition, data on hospital admissions and outpatient visits, visits to the physiotherapist, dietician or respiratory nurse, diet nutrition, and outpatient medication were obtained from administrative records. The cost per quality-adjusted life-year (QALY) was calculated in two ways, using data from the cost booklet or registrations.

Results: In total, 175 patients were included in the study. Agreement between self-report and registrations was almost perfect for hospitalizations (rho = 0.93) and physiotherapist visits (rho = 0.86), but above 0.55, moderate, for all other types of care. The total cost difference between the registrations and the cost booklet was 464 euros with the highest difference for hospitalizations 386 euro. Based on the cost booklet the cost difference between the treatment group and usual care was 2,444 euros (95 percent confidence interval [CI], −819 to 5,950), which resulted in a cost-utility of 29,100 euro/QALY. For the registrations, the results were 2,498 euros (95 percent CI, −88 to 6,084) and 29,390 euro/QALY, respectively.

Conclusions: This study showed that the use of self-reported data or data from registrations effected within-group costs, but not between-group costs or the cost utility.

Background: The concept of using public funds to pay for healthcare interventions only when provided in the context of ongoing research is receiving increasing attention worldwide. Nevertheless, these decisions are often controversial and implementation can be problematic.

Objectives: The aim of this study was to investigate the views of United Kingdom stakeholders on the current arrangements for implementing “only in research” (OIR) decisions and to investigate how improvements might be made.

Methods: After an internal review of previous OIR decisions issued by the National Institute for Health and Clinical Excellence (NICE), deliberations by NICE's Citizens Council, and an international workshop convened by NICE and the United States Agency for Healthcare Research and Quality, thirteen key stakeholders and experts from academia, industry, government, and the National Health Service (NHS) were interviewed using a semistructured interview guide. Interview transcripts were subjected to a framework-based analysis using computer-assisted qualitative data analysis software.

Results: All interviewees endorsed the use of the OIR option. There was a high degree of consensus for several suggestions regarding how the use of the OIR option might be improved. For example, there was universal agreement that a formal process should be established to prioritize research needs arising from OIR decisions and that funds for publicly funded research projects should be channeled in a manner that would better motivate healthcare providers to participate in OIR-related research.

Conclusions: The findings of this study suggest several potential modifications of the OIR pathway in the United Kingdom and may also be helpful to health technology assessment agencies in other countries that already use or are considering using an OIR-like option to reduce the uncertainty inherent in health technology assessment.

Objectives: The Weight Loss Maintenance Trial (WLM) was a multicenter, randomized trial comparing two weight loss maintenance interventions, a personal contact (PC) program with primarily telephone-based monthly contacts, and an Internet-based program (interactive technology, IT), to a self-directed control group, among overweight or obese individuals at high cardiovascular risk. This study describes implementation costs of both interventions as well as IT development costs.

Methods: Resources were micro-costed in 2006 dollars from the primary perspective of a sponsoring healthcare system considering adopting an extant intervention, rather than developing its own. Costs were discounted at 3 percent annually. Length of trial participation was 30 months (randomization during February–November 2004). IT development costs were assessed over 36 months. Univariate and multivariate, including probabilistic, sensitivity analyses were performed.

Results: Total discounted IT development costs over 36 months were $839,949 ($2,414 per IT participant). Discounted 30-month implementation costs for 342 PC participants were $537,242 ($1,571 per participant), and for 348 IT participants, were $214,879 ($617 per participant). Under all plausible scenarios, PC implementation costs exceeded IT implementation costs.

Conclusions: Costs of implementing and operating an Internet-based intervention for weight loss maintenance were substantially less than analogous costs of an intervention using standard phone and in-person contacts and are of a magnitude that would be attractive to many health systems, subject to demonstration of cost-effectiveness.

Objectives: The aim of this study was to systematically assess the long-term (≥ 6 months) benefits of epidural steroid injection therapies for patients with low back pain.

Methods: We identified randomized controlled trials by database searches up to October 2011 and by additional hand searches without language restrictions. Randomized controlled trials on the effects of epidurals for low back pain with follow-up for at least 6 months were included. Outcomes considered were pain relief, functional improvement in 6 to 12 months after epidural steroid injection treatment and the number of patients who underwent subsequent surgery. Meta-analysis was performed using a random-effects model.

Results: Twenty-nine articles were selected. The meta-analysis suggested that a significant treatment effect on pain was noted at 6 months of follow-up (weighted mean difference [WMD], −0.41; 95 percent confidence interval [CI], −0.66 to −0.16), but was no longer statistically significant after adjusting for the baseline pain score (WMD, −0.19; 95 percent CI, −0.61 to 0.24). Epidural steroid injection did not improve back-specific disability more than a placebo or other procedure. Epidural steroid injection did not significantly decrease the number of patients who underwent subsequent surgery compared with a placebo or other treatments (relative risk, 1.02; 95 percent CI, 0.83 to 1.24).

Conclusions: A long-term benefit of epidural steroid injections for low back pain was not suggested at 6 months or longer. Introduction of selection bias in the majority of injection studies seems apparent. Baseline adjustment is essential when we evaluate pain as a main outcome of injection therapy.

This article examines how large data sets can be used for evaluating the effects of health policy changes and for flagging providers with potential quality problems. An example is presented, illustrating how three risk-adjusted measures of hospital performance were developed using patient discharge abstracts. Advantages and disadvantage of this approach are discussed.

This review assesses the evidence regarding the effectiveness of bone density measurement (BDM) screening and related interventions—hormone replacement therapy (HRT) and intranasal salmon calcitonin (SCT[N])—in menopausal women to prevent fractures in later life. Data sources included systematic reviews of evidence and relevant primary studies identified through literature searches on MEDLINE and EMBASE. Study selection included trials of BDM screening programs, prospective studies exam-ining the predictive value of BDM, randomized controlled trials, cohort studies, and case-controlstudies of HRT and SCT(N). The evidence was evaluated using a classification system incorporatingstudy design and quality. Outcomes were measured in terms of relative risk of fracture for a 1 SDdecrease in bone mineral density below the age-adjusted mean, relative risks or odds ratios forfractures associated with treatments, and proportion of hip fractures potentially prevented by BDMscreening linked to treatments. Fair evidence from prospective cohort studies suggests that BDMcan predict the risk of fractures, but not with high accuracy. Fair evidence from low-quality randomizedcontrolled trials and observational studies suggests that HRT and SCT(N) are efficacious in preventingfractures. Good evidence supports the efficacy of these treatments in preserving bone mass, butthere is fair evidence that the effect wears off after cessation of therapy. There is little evidence onthe impact of screening menopausal women with BDM in association with HRT or SCT(N) treatment.Estimates based on combining existing evidence regarding the predictive value of BDM and efficacyof HRT suggests that 1–7% of hip fractures might be prevented.