CLINICAL OUTCOMES RESEARCH
W1: META-ANALYSIS METHODS THAT ALLOW FOR COMPARISON OF INTERVENTIONS IN THE ABSENCE OF HEAD-TO-HEAD DATA
Discussion Leaders: Bruce Crawford MA, MPH, General Manager, Asia, Mapi Values, Tokyo, Meguro, Japan; Jeroen P Jansen PhD, Project Manager, Mapi Values, Houten, The Netherlands.

PURPOSE: Participants will be introduced to meta-analysis methods, focusing on the advantages of combining direct and indirect comparisons to benchmark and to support product value among alternatives. Focus is on the introduction of methods and how such analyses can provide value arguments to support product uptake.

ECONOMIC OUTCOMES RESEARCH
W2: USING LATENT CLASS ANALYSIS IN HEALTH OUTCOMES RESEARCH
Discussion Leaders: Jeonghoon Ahn PhD, Assistant Professor, University of Southern California, Los Angeles, CA, USA.

PURPOSE: To understand a new methodology which can be widely used in outcomes research. The participants of this workshop will gain 1) understanding of new methodology, latent class analysis (LCA), which can be used widely in outcomes research; and 2) knowledge on how to apply LCA in health care cost estimation. Also the participants will discuss applicable outcomes research areas of the LCA other than cost outcomes.

DESCRIPTION: The skewed nature of health care costs necessitates a special modeling technique, such as two part model (Newhouse et al. 1980 JE), expected conditional mean model (Mullahy 1998 JHE), and many more, to analyze cost outcomes. In this line, Latent Class Analysis (LCA) has been used as a cost regression framework (Deb and Trivedi 1997 JAE, 2002 JHE; Jimenez-Martin et al. 2002 HE; and many others). Recently, Ahn et al. (2008 VIH) applied LCA in cluster analysis framework to compare outcomes by different adherence patterns to antipsychotic drug therapy, which assumed as a latent index. This approach is convenient since many conventional statistical analyses, such as t-test or chi-square test, can be applied to the identified groups (patterns). In this workshop, LCA cluster analysis will be highlighted as a methodology applicable to other outcomes research including an example of cost estimation where health status can be assumed as a latent index.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W3: BURDEN OF ILLNESS STUDY FOR DEVELOPING HEALTH POLICY IN ASIA-PACIFIC Crane
Discussion Leaders: Bong-Min Yang PhD, Professor, School of Public Health, Seoul National University, Seoul, South Korea, South Korea; Q. Chau Tini Nguyen PharmD, AP Director, Sanofi-Aventis, Paris, France, France; Shu-Chuen Li Ph, D/MBA, Associate Professor, National University of Singapore, Pharmacy, Singapore; Shanlian Hu MD, MSc, Professor, Fudan University, School of Public Health, Shanghai, China
PURPOSE: To review current status of burden of illness (BoI) study in Asia-Pacific region and to discuss for the way to use it appropriately for healthcare policy setting.
DESCRIPTION: In BoI studies, health problems are usually weighed by expressing epidemiologic results (e.g. incidence, prevalence and mortality) and overall costs. Usually its aim is to inform choices in resource allocation by estimating resource consequences of health problems in relation to each other. BoI studies have been used to highlight and to weight different health problems for comparative purposes, both within a national context and internationally. In this workshop, audience will have introductory overview 1) BoI research trend at one targeted diseases area in AP with various methodology; 2) the criteria used to set up priority in public health in western countries and/or AP region (example from Korea, Singapore, Australia) under the common ground; 3) Example of priority setting in healthcare policy with BoI data. Furthermore, through discussions with the audience, the workshop intends to exchange opinions in searching for the way of prioritizing health resource use, based on real BoI data in the AP setting.

W4: FROM THEORY TO PRACTICE: EXPERIENCES OF SOUTH KOREA HEALTH INSURANCE REVIEW ASSESSMENT AND SERVICE (HIRA) (Speakers at this panel were invited)
Discussion Leaders: Sang Moo Lee MD PhD, Consultant EBH team, Health Insurance Review Assessment and Service, Seoul, Korea; So-Young Yi PhD, General Manager, Drug Listing Division, Health Insurance Review and Assessment Service, Seoul, Korea; SeungJin Bae ScD, Senior Researcher, Drug Policy Research Team, Health Insurance Review Assessment and Service, Seoul, Korea

PURPOSE: Authors will share with the audience how HIRA merged theory into practice.
DESCRIPTION: The Korean government announced in December 2006 that only medications with good value for money, based on cost-effectiveness analysis, will be reimbursed in the Korean National Health Insurance program (Positive List System, PLS). Under PLS, all newly developed chemical/biological compounds with Korean Food and Drug Administration (KFDA) approval are required to submit economic evaluation dossiers from 2008 and on, and optional for 2007, if they wish to be listed under the national health insurance system. HIRA’s Drug Listing Division is in charge of assessing the quality of the submitted dossiers, and Drug Policy Research team collaborates with DLD and mainly focuses on reviewing the submitted simulation models. HIRA’s Evidence-based Health care team (EBH team) has formulated the evidence-based review process, which enhanced the scientific basis of DLD’s review process. Drug Listing Division reports to the Drug Reimbursement Evaluation Committee, which decides the reimbursement status of the drugs. Both Drug Listing Division and Drug Policy Research team evaluates the quality of the submitted reports to help policy makers (Drug Reimbursement Evaluation Committee) make informed decisions.

ECONOMIC OUTCOMES RESEARCH
W6:  EXPERT OPINION AND COUNTRY SEPCIFIC MODEL ADAPTATIONS
Discussion Leaders: Bruce Crawford MA, MPH, General Manager, Asia, Mapi Values, Tokyo, Meguro, Japan; Chris Evans PhD, MPH, Director of Economics and Outcomes, Mapi Values, Boston, MA, USA.
PURPOSE: Often the results of pharmacoeconomic models are adapted to other countries through the use of expert opinion. It is therefore important for researchers to understand the relative strengths and shortcomings of data collection methods that rely on expert opinion and to critically evaluate studies that utilize expert opinion. This session is directed at individuals who design and conduct pharmacoeconomic evaluations as well as those in charge of interpreting pharmacoeconomic study results.
DESCRIPTION: Expert opinion is used frequently to elicit information on resource use, costs and probabilities in order to adapt decision analytic models from one country to another. Various techniques have been described and are available for researchers interested in obtaining information from a panel of experts. Round tables, nominal group techniques, modified Delphi and Delphi panels are the most common. Unfortunately there is little consistency in the way that expert opinion is obtained or used. This workshop explores the use of value panels, nominal group processes, modified Delphi panels and Delphi panels to derive estimates and distributions for pharmacoeconomic research. The workshop will focus on describing each technique and provide recommendations on appropriate use of each of them in pharmacoeconomic research. Discussion leaders and participants will also examine areas where mistakes commonly arise and ways to avoid deriving misleading information. The workshop will cover several key-areas: provision of baseline information, attrition of experts, criteria for selecting experts, definition of consensus, appropriate use of terms and overall validity of such data. In addition, for processes that aim to gain a consensus of opinion, the areas of stability and convergence will be reviewed and guidance will be given on reporting expert opinion in pharmacoeconomic studies.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH
W7: PHARMACOECONOMIC GUIDELINES IN CHINA AND THAILAND (Speakers at this workshop were invited)
Application of Pharmacoeconomics & Outcomes Research in China
Discussion Leader: Ming Tang PhD, Principal Director, Center of Pharmacoeconomic Evaluation, Chinese Medical Doctor Association, Beijing, China
PURPOSE: China as one of fast growing market of pharmacy consume is facing multiple problem in its own healthcare system. In this speech, speaker will introduce the current China healthcare system and the utilization of pharmacoeconomics in developing national formulary.
DESCRIPTION: In the year of 1989, China published a hand book listing basic medicine. It is a blue print of government reimbursement formulary. In the recent year, many new medications have been used in the treatment. Some local authority adjusted the local reimbursement list. Physicians and patients are severely confused by the inconsistence of federal and local reimbursement system. So there is urgency to develop a national formulary. Speaker will describe the procedure of developing national formulary and the application of outcomes research in the developing process.  Some other points will be covered: 1) The trend, the challenge and the barrier of conducting such study in China; 2) Briefing about the development procedure of the guideline of pharmacoeconomics; 3) Application of pharmacoeconmics in developing the national formulary.

Development of the National Guidelines for Conducting Pharmacoeconomic Evaluation in Thailand
Discussion Leader: Yot Teerawattananon PhD, Health Intervention and Technology Assessment Program, Ministry of Public Health, Thailand.
PURPOSE: This presentation provides rationales and principles supporting the development of the national guideline for conducting pharmacoeconomic evaluation in Thailand. It also describes the guideline development processes and future challenges if pharmacoeconomics can become a useful tool for decision making regarding health care resources.
DESCRIPTION: In Thailand, there is increasing impetus to use economic evidence to allow more explicit and transparent health care priority setting. However, the systematic review of literature relating to Thailand revealed a number of methodological flaws with prior economic evaluation publications. These problems could be tackled and reduced by setting up the standard guidelines. At the end of 2006, the Health Intervention and Technology Assessment Program consulted experts from both academic and research institutions across the country to make a concrete plan for the development of the first methodological guideline for conducting economic evaluation in Thailand. The development of this guideline began with a review of existing international guidelines including those developed by governments and standard health economic textbooks. Then the scope and major components of the Thai guidelines were defined. The invited experts reviewed each of the key components and drafted the guidelines. A series of consultation meetings were carried out to present the draft to various stakeholders including those from public and private health authorities. Suggestions from stakeholders were also included before the final publication. The guidelines were subsequently endorsed by the subcommittee for development of the National List of Essential Drug (NLED) as the guidelines can be a useful tool for future drug selection process. Although, at present it is difficult to predict the future usefulness of the guidelines, this presentation provides extensive discussion on the potential applications and contributions of pharmacoeconomic guidelines for policy decision making in Thailand.

W8:  HEALTH TECHNOLOGY ASSESSMENT IN ASIAN-PACIFIC COUNTRIES.
Discussion Leaders: Candace Gunnarsson EdD, President CEO, S2 Statistical Solutions, Inc, Cincinnati, OH, USA; Susan Ross MD, Consultant, SDRoss Consulting, Cohasset, MA, USA; Harry J. Kotlarz MBA, BS, Director, Health Economics and Reimbursement, DePuy Orthopaedics Inc, Warsaw, IN, USA.
PURPOSE: To teach new HTA developers and HTA consumers in Asian-Pacific countries how to produce their own state-of-the-art HTA reports, and/or how to critically appraise HTA reports from other sources, so as to enable better decision-making regarding health policy and coverage in their own locales.
DESCRIPTION: Using data from the INAHTA, we have charted an association between spending on healthcare, as a percentage of GDP, and the establishment of national health technology assessment (HTA) agencies. In developing and emerging economies, when spending on healthcare approaches 7% of GDP, HTA becomes more formalized and influential in determining how healthcare monies are spent. Many countries in the Asian-Pacific region, including China, Singapore, Malaysia, Korea, and India fall into this category. Problems arise, however, when the HTA groups that develop in these circumstances are inexperienced and have no locally-customized templates to follow to ensure their work is rigorous, reliable, and relevant. Furthermore, manufacturers seeking market entry in these economies suffer from the absence of clearly stated and transparently applied evidentiary requirements for HTA. The authors, including a clinical consultant in evidence-based medicine, educational methods and statistics expert, and an industry reimbursement professional, developed a customizable template approach to HTA for use in emerging economies. This template – in PowerPoint format - incorporates current best practices for systematic reviews and economic assessments, and provides structure and transparency to the development and presentation of best available evidence for policy-makers and payers that are interested in formalizing their HTA activities. We will walk through this slide template in an interactive session with attendees, to illustrate the structure and content of an ideal HTA report. In particular, we will review the formulation of policy questions, development of necessary background information, syntheses of research findings regarding safety, efficacy/effectiveness, psychological, social, & ethical considerations, organizational & professional implications, and economic issues, ending with policy conclusions & recommendations. We will give examples of how well-done HTAs can inform coverage decisions, formulary decisions, guidelines formation, medical practice, and future research. Lastly, we will provide a “yardstick” for manufacturers to measure the impact of the HTA reports.

PATIENT-REPORTED OUTCOMES
W9:  THE ROLE OF UTILITIES IN SUPPORTING MARKET ACCESS IN ASIA: LESSONS LEARNT FROM NICE & CDR
Discussion Leaders: Andrew Lloyd BSc, DrPH, Director, Oxford Outcomes, Oxford, United Kingdom; Nan Luo PhD, Research Fellow, National University of Singapore, Centre for Health Services Research, Singapore, Singapore; Paul Quarterman MSc, Director, Oxford Outcomes, Oxford, Oxon, UK.
PURPOSE: This workshop is designed to bring together lessons learnt from the use of utilities to support cost-effectiveness analyses (CEA) from the UK & Canada and to apply them to help demonstrate cost-effectiveness in Asian markets which are adopting CEA based reimbursement.
DESCRIPTION: Many countries have adopted centralized decision making processes for the approval of new health technologies. CEA is a very important input into this decision making alongside clinical effectiveness and clinical need and also resource implications or budget impact. Most countries have issued guidance for manufacturers regarding the analyses that they would like to see; in some countries this can be highly prescriptive - this is referred to as the ‘reference case' by NICE. A vital component of any cost effectiveness analysis is the source of quality of life weights (or utilities) which drive the estimation of QALYs. Expected standards regarding utility data are varied and in some places are becoming tougher (e.g. NICE). Some products have failed to gain reimbursement because of poor utility data and examples will be presented and discussed. This workshop will review the standards expected in different countries regarding the collection and use of utility data. We will then discuss how utility data can be collected or estimated including ways in which we can ensure that the full benefit or value of a new treatment can be captured. Finally we will present other means of capturing the value of a new treatment which are perhaps beyond the scope of the QALY. Attendees will participate in break out sessions to come up with innovative solutions to capture utility data for reimbursement submissions. Local operating companies often have data requirements for economic submissions despite the existence of clinical trial programmes. We hope this session will help to address this need.

USE OF REAL WORLD DATA

Tuesday, 9 September 2008
4:00PM-5:00PM

CLINICAL OUTCOMES RESEARCH
W11:  WEB-BASED SYSTEMATIC REVIEWS – A NEW TOOL FOR AN EVOLVING PROCESS
Discussion Leaders: Peter O'Blenis BCS, MBA, Vice-President, Product Development, TrialStat Corporation, Ottawa, ON, Canada.
PURPOSE: To examine paper-based processes and challenges in systematic reviews; to review web-based systematic reviews, their evolution and current state; to examine how research organizations are using web-based reviews to address quality, speed, capacity and transparency issues in their systematic reviews; to examine the metrics for measuring “improvement” in the systematic review process; to examine current shortfalls and challenges facing web-based reviews; and to examine the next significant challenges in the evolution of web-based systematic reviews.
DESCRIPTION: Systematic reviews represent a fast, safe and cost effective tool for developing and refining clinical evidence to inform medical practice, to improve and accelerate clinical research and to support product claims and regulatory submissions. Systematic reviews today are typically completed using paper-intensive processes and, as such, the practice of conducting systematic reviews is rapidly falling behind the clinical research industry's broader use of new information technologies. This, unfortunately, means longer than necessary times to study completion, more error prone data and ultimately higher costs for research. Web-based systematic reviews offer a new potential tool in the drive to produce better, faster and more accurate analysis of medical evidence. Organizations deploying IT-enabled systematic review tools are not only completing their research faster and more collaboratively but the quality and auditability of their results are also significantly improved. This presentation examines: 1) An overview of paper-based processes and challenges in systematic reviews;2)A review of web-based systematic reviews, their evolution and current state; 3) How research organizations are using web-based reviews to address quality, speed, capacity and transparency issues in their systematic reviews; 4) Metrics for measuring “improvement” in the systematic review process; 5)Current shortfalls and challenges facing web-based reviews; and 6) The next significant challenges in the evolution of web-based systematic reviews. The workshop will begin with an introductory overview of systematic reviews, followed by an in depth review of web-based systematic reviews as a new tool for an evolving process and will conclude with a live demo and question and answer period with the audience.

ECONOMIC OUTCOMES RESEARCH
W12:  IS ONE-PART MODEL ENOUGH FOR ANALYZING HEALTH CARE COST DATA?
Discussion Leaders: Xin (Sam) Ye MS, PhD, Associate Director¹; Henry Joe Henk PhD, Director¹.
¹i3 Innovus, An Ingenix Company, Eden Prairie, MN, USA.
PURPOSE: The purpose of this workshop is to demonstrate and discuss the use of one-part model versus two-part model in analyzing health care cost data.
DESCRIPTION: Health care cost outcomes(y) often have three fundamental statistical properties: (a) y>=0; and (b) the outcome y=0 is observed sufficiently frequently that the zeros cannot be ignored econometrically; and (c) y>0 is often highly skewed distribution resulting from few patients with high health care utilization. Given exogenous covariates x, the analysis of cost outcomes have often relied on the two-part model (2PM) when there are large number of zeros. The two-part model 2PM assumes that Pr( y>0| x) is governed by a parametric binary probability model like logit or probit (part one) , and either E(ln( y)| y>0;x)=xb (e.g., Ordinary Least Squares (OLS) model with log-transformed y) or E( y| y>0, x) = exp(xb) (e.g., Generalized Linear Model (GLM) with a log link and Gamma distribution) as part two. While the choice among competing 2PM estimation strategies has been addressed in the literature, the one-part model (1PM) has a certain attractiveness as it is fit to the data for all observations, despite whether they used any services. However, questions remain as to the performance of such a model when the proportion of observations with y=0 is large and data is highly skewed. Though a series of simulations based on observational data, this workshop will compare and discuss the performance of the 1PM and 2PM (both based on a GLM with a log link) across two dimensions: 1) proportion of y=0 and 2) distribution of y for y>0. Audience participation will be encouraged through the discussion of the application of these techniques to health care cost data analysis. This workshop will benefit researchers involved in analyzing and interpreting health care cost data.

PATIENT-REPORTED OUTCOMES
W14:  WILLINGNESS TO PAY FOR A QALY IN EAST ASIA
Discussion Leaders: Kiichiro Tsutani MD, PhD, Professor, The University of Tokyo, Graduate School of Pharmaceutical Sciences, Tokyo, Japan; Takashi Fukuda PhD, Associate Professor, The University of Tokyo, School of Public Health, Tokyo, Japan; Sang-Cheol Bae MD, PhD, MPH, Professor, Hanyang University College of Medicine, Seoul, South Korea; Hui-Chu Lang PhD, Associate Professor, National Yang-Ming University, Institute of Hospital and Health Care Administration, Taipei, Taiwan.
PURPOSE: In order to utilize pharmacoeconomic analyses for decision making, acceptable threshold for cost per QALY must be determined. Even though explicit threshold is not settled yet in Asian countries, this issue will be present in the near future. This workshop is based on the recent survey conducted in Japan, Republic of Korea, Taiwan, and some other countries about the willingness to pay for an additional QALY.
DESCRIPTION: We have conducted the survey of willingness to pay for an additional QALY in Japan, Republic of Korea, Taiwan, Australia and UK. After the introduction of the purpose and the structure of the workshop, current issues on cost per QALY and the method of deriving willingness to pay for a QALY will be presented. Discussion leaders from Japan, Republic of Korea and Taiwan will present the result of the survey and its implication to decision making in each country. We found some differences among the countries, which might be caused by cultural gap.

USE OF REAL WORLD DATA

PURPOSE: The participants in this workshop will learn about recent examples from Europe and North America of new models of paying pharmaceutical and diagnostics manufacturers for innovative products based on real-world performance measures, and will explore their implications for the Asia Pacific Region.
DESCRIPTION: Recently, there has been growing interest and discussion of new models for paying pharmaceutical and other medical technology manufacturers for innovative products. Payers would reward them with robust reimbursement for drugs or tests based on an acceptable threshold level of evidence of clinical utility in exchange for additional information and post-market tracking and analysis to determine whether the drug or test “performed” as expected. These types of arrangements—sometimes called “risk-sharing”-- have been a topic of discussion for many years. To date, the most prominent example has been a special reimbursement system for some multiple sclerosis drugs covered by the UK National Health Service. Other recent examples have drawn attention, such as measuring tumor response with Velcade in the UK, and a notable US reimbursement arrangement between United Healthcare and Genomic Health for its Oncotype Dx test, designed to predict the risk of recurrence among women who have had surgery for breast cancer. Measuring real-world performance is a costly scientific and operational challenge. The limited number of examples is testament to these challenges, as well to the understandable reluctance of technology manufacturers to take full responsibility for actual performance in a real-world setting. These new models raise a host of important economic questions about incentives and rewards for innovation. The discussion leaders in this workshop are co-investigators in a study to examine these new reimbursement models, analyze the associated incentives, and assess their feasibility and generalizability. These recent examples will be discussed, and their implications for practice and policy will be explored specifically for the Asia Pacific region.