were evaluated based on US and European PRO/HRQOL guidance criteria. RESULTS: The review identified 8 HRQOL measures used with EB patients: 3 generic (SF-36; EQ-5D, EQ-5DY); 4 dermatology-specific (DLQI, CDLQI, DQOLS, Skindex); and 1 EBspecific (QOLEB). Only the CDLQI and QOLEB were specifically designed for pediatric populations (ages 4-16 and Ն10 years, respectively). The QOLEB was the only instrument for which content was derived from EB patients; 26 patient interviews were conducted, of which 9 were with patients Ͻ18 years. Despite pediatric input during development, not all QOLEB content is relevant to children/adolescents; items assessing family and financial impact are not appropriate for these respondents. Psychometric properties have been established among pediatric EB patients only for the QOLEB (convergent validity [DLQI: R ϭ 0.774]; internal consistency [␣ ϭ 0.931]; test-retest reliability [ R ϭ 0.843]). CONCLUSIONS: Few published measures meet current regulatory standards for HRQOL evaluation in pediatric EB patients. Given the age range for EB, an HRQOL instrument that evaluates age-appropriate concepts was not identified. Further research is needed to document and assess HRQOL concepts in pediatric patients with EB. OBJECTIVES:To compare the rate and cost of traditional versus on line recruitment methods to medical surveys in 2 different patient populations. METHODS: We compared traditional to on line advertising in 2 separate projects (conducted in the UK) across the same time period using the same internet and social media recruitment methodologies allowing a comparison of the costs and effectiveness. By looking at 2 quite distinct populations -the first being cholesterol lowering agents, thus an older age with cardiovascular disease and the second women with problem periods, hence a younger and otherwise healthy group we could evaluate the recruitment methods. RESULTS: All costs are direct payments and exclude labour; Cholesterol lowering agents; Traditional methods -recruitment through chain of pharmacists using leaflets; 303 patients recruited over 9 months; cost/ survey £3.94. Internet/Social media -adverts placed on facebook.com, google and third party ad network; 3812 visitors to website; cost/ visitor £0.23; 572 survey completions over 11 weeks; cost/ survey £1.53. Problem Periods: Traditional methods -magazine adverts, exhibition stand, press releases; 232 Women recruited over 8 weeks; Cost/ survey £18; Internet/Social Media; Ͼ6000 visitors to website over 11 weeks; cost/ visitor £0.30; 1534 survey completions over 11 weeks; cost/ survey £1.51. CONCLUSIONS: Whilst traditional methods of recruiting participants were successful, they were generally slower and cost more per person recruited. Internet advertising and use of social media sites offers the potential of rapid, cost effective recruitment. This result however does not tell us if the traditional adverts were making it more likely people would search on line, thus driving the on line recruitment or if the statins population (our stat...

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OBJECTIVES: Decisions about the use of new medical technologies based on estimates of the average cost-effectiveness across a potentially heterogeneous population runs the risk of foregoing net health benefits(NHB) for sub-groups of the population. We propose a general framework within which to assess betweenpatient heterogeneity and its role in cost-effectiveness subgroup analysis(CESA), complementing this with a practical application. METHODS: We first describe how to extend methods for cost-effectiveness analysis (based on current information) to address issues such as estimation of NHB, sources of heterogeneity, definition and selection of subgroups. Next, we define the role of uncertainty in CESA, extending the concept of Value of Information(VoI) to include the notion of a static and dynamic Value of Heterogeneity(VoH). The application of the proposed theoretical framework is illustrated using a cost-effectiveness model developed for the analysis of a multicentre-trial(RITA-3), which assessed the efficacy of an early arteriography with revascularisation versus standard management in patients with acute coronary-syndrome. Using this model we conducted a re-analysis investigating alternative subgroup specifications, varying between one and five subgroups, with a view to produce an efficiency frontier for subgroup analysis relating to this decision problem. We assessed the static and dynamic VoH under each specification. RESULTS: The population expected NHB when considering five subgroups was 105,500 QALYs greater than decision based on estimates for the average population (static-VoH). Although, identifying 5 subgroups reduced in the Expected Value of Perfect Information(EVPI) (920 QALYs, at a threshold of £30,000/QALY), the potential NHB from resolving uncertainty was greater after heterogeneity has been identified (dynamic-VoH). CONCLUSIONS: Our initial findings support the argument that explicit consideration of heterogeneity in CEA leads to a positive static and dynamic VoH. In addition, heterogeneity not only may increase the EVPI but can also reduce its magnitude. The VoH framework offers a useful guidance for a more systematic CESA.

medical device, whilst crucial for late-stage reimbursement decisions, is often neglected by developers early on due to lack of data. The headroom method has been proposed as a way to integrate economic evaluation into early go/no-go decisions. By estimating the maximum reimbursable price (MRP) for a new device idea and comparing this with forecasted developer's costs, R&D resources can be channelled toward innovations for which future returns appear feasible. The aim of this study was to evaluate the method by applying it retrospectively to a large and diverse set of case studies, and comparing predicted 'headroom' with actual market success, within the UK setting. METHODS: The method was applied systematically to twenty devices/diagnostics invented in the past, retrieved from the UK national horizon scanning centre (NHSC)'s 2000-2009 database; literature searches were date restricted to mimic the information available at the 'concept stage' of development. Each case study was followed up to observe the product's actual market success or failure compared with the estimated 'headroom' and the development decision this would have engendered, in order to explore the method's predictive value. RESULTS: Headroom correlated with subsequent market success in 85% of cases. Headroom is most easily elicited where the change proposition of the innovation is straightforward (e.g. direct replacement technologies); diagnostic equipment posed particular difficulties. When the numerical headroom assessments were considered alongside unquantifiable factors identified relating to the clinical and market context, the method offered a good indication of future market potential. CONCLUSIONS: Despite the strong advocacy of early economic evaluation, the literature lacks a critical appraisal of any such method. This study for the first time explores the potential implications of basing development decisions on the headroom method, thereby assessing its potential value to stakeholders. OBJECTIVES:Clinical trials remain the gold standard for proving efficacy but restrictive enrolment criteria and overly controlled study environments limit generalizability. Increasingly, payers and health care providers are interested in real world evidence to support the case for pharmaceutical innovations. We review A465