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...

lenges and opportunities. Results: Only 22%-27% of payers and providers believe that personalized medicine is a "very important" strategic goal versus ~50% of large biopharma manufacturers. Although 47%-52% of all stakeholders have integrated staff with area expertise, fewer than 20% have developed a centralized focus on personalized medicine and only 8%-20% believe they have tools to evaluate its success. Decision maker perspectives ranged broadly on its key benefits but included improved and more predictable outcomes and cost efficiencies. Approximately 30% of all stakeholders believe that personalized medicines will continue to receive premium pricing to justify ROI for this business approach. ConClusions: Although most stakeholders see value in personalized medicines, they struggle with practical implementation and need actionable strategies to characterize the value and impact of these technologies. The survey suggests that limited emphasis on infrastructure development and methods, heterogeneous value assessment, and misalignment of incentives remain key challenges to enabling care and economic efficiencies promised by this evolving treatment paradigm. PHP145Predicting Price-to-cHarge ratios For community HosPitals in tHe united states objeCtives: Reliable price data could enable consumers to choose providers that offer better value than others, eventually leading to market-level gains in quality and efficiency. The objective of this study is to predict price-to-charge ratios (PCRs) for community hospital stays on the basis of charge data and information about individual stays, hospitals, market areas, and states. Methods: We used 2006 . We predicted PCRs for major payer categories for over 1,000 community hospitals as a function of state, market, hospital, and patient characteristics using exponential conditional mean models with a log link and gamma-distributed errors. The unit of analysis was the hospital. We used a two-stage iterative estimation in which equations are estimated individually and the errors saved. In the next step, each payer equation is estimated a second time with the first-stage errors of the other payer equations as new independent variables. We assessed goodness of fit through model characteristics and by assessing the match of actual and predicted PCRs. Results: Average demographic characteristics were significant predictors of PCRs for Medicare and Private insurance, but not for Medicaid or Self-Pay. Hospital characteristics were related to every PCR category. Critical-access hospitals and teaching hospitals were associated with significantly greater PCRs for Medicare, Medicaid, and Private insurance holders. Greater numbers of Medicare and Medicaid discharges were associated with significantly lower PCRs. Higher PCRs were most often associated with worse economic conditions and greater state generosity in Medicaid eligibility and spending. ConClusions: Inpatient encounter prices paid by Medicare, Medicaid, and private insurance can be estimated with acceptable accuracy for communi...

A491sure by demanding early decisions and rapid access. Decision makers may unduly delay potential benefits to patients by waiting for stronger evidence, or may endorse medicines that later turn out to have a less robust benefit-risk ratio, to be ineffective, cost-ineffective, or even harmful. Hence, many countries have developed mechanisms that allow temporary access to promising medicines while concurrently requesting the generation of additional evidence to reduce uncertainty. Their objective is an optimal trade-off between different stakeholder needs, flexibility, responsiveness, and rigor as well as the flexibility to revise decisions on access when new evidence becomes available. The ENCePP WG on HTA has the potential to become a capacity building tool for regulatory and HTA agencies to develop research structures aimed at complementing the evidence generation for MA and market access. Post-authorisation studies developed under the auspices of ENCePP could provide new safety and clinical effectiveness information of marketed medicines. ENCePP expertise, research experience and health care databases may also contribute to the coordination, methodological guidance and data sourcing for the enhancement of HTA processes.

lenges and opportunities. Results: Only 22%-27% of payers and providers believe that personalized medicine is a "very important" strategic goal versus ~50% of large biopharma manufacturers. Although 47%-52% of all stakeholders have integrated staff with area expertise, fewer than 20% have developed a centralized focus on personalized medicine and only 8%-20% believe they have tools to evaluate its success. Decision maker perspectives ranged broadly on its key benefits but included improved and more predictable outcomes and cost efficiencies. Approximately 30% of all stakeholders believe that personalized medicines will continue to receive premium pricing to justify ROI for this business approach. ConClusions: Although most stakeholders see value in personalized medicines, they struggle with practical implementation and need actionable strategies to characterize the value and impact of these technologies. The survey suggests that limited emphasis on infrastructure development and methods, heterogeneous value assessment, and misalignment of incentives remain key challenges to enabling care and economic efficiencies promised by this evolving treatment paradigm.