W9: JOURNEY TOWARDS A TRUE EFFECT IN COHORT STUDIES: OBSTACLES, STATISTICAL TOOLS, AND “DIFFERENCE IN DIFFERENCE” ESTIMATES
Lee WC1, Pashos CL2
1Abt Associates Inc, Bethesda, MD, USA; 2Abt Associates Inc, Cambridge, MA, USA

Purpose: The goal of this workshop is to provide participants with 1) a basic understanding of what the difficulties and obstacles are in estimating a meaningful, unbiased effect in cohort studies; 2) statistical tools and study designs that can be used to address these challenges; 3) an approach known as the “difference in difference” econometric method; and 4) examples using the Medicare Public Use Files (PUF), that track the care and costs of a 5% random sample of Medicare beneficiaries.
Who Would Benefit: Researchers interested in designing a rigorous cohort study enhanced by econometric tools.
Workshop Description: Retrospective cohort studies are fraught with numerous potential sources of bias that make it difficult to ascertain the differential impact of a medical intervention on patient outcomes. These potential pitfalls include differing length of survival time between groups, censored data, ignored reverse causality, selection bias, improper selection of the control group and model misspecifications. This workshop will provide stepwise approaches to deal with these potential pitfalls. Relevant statistical strategies such as stratification, matching, and various econometric tools (e.g., logistic regression and Cox proportional hazards model) will be discussed. Throughout the workshop, participants will be given opportunities to study actual examples, to identify problems, and to offer and review suggestions for statistical tools to remedy or reduce these problems. Particular attention will be brought to the “difference in difference” method, a tool more commonly used in the field of health economics but which has been underutilized in epidemiological cohort outcome studies. In this regard, emphasis will be placed on understanding 1) how a cohort study design can be translated into a “difference in difference” econometric equation to adjust for imbalances that may remain even after matching; and 2) how the “difference in difference” estimates will complement or enhance a cohort study design.

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W17: STATISTICAL ANALYSIS OF PATIENT REGISTRIES: METHODS FOR THE MOST EFFECTIVE USE OF OBSERVATIONAL DATA
Pasta DJ, Miller DP
Ovation Research Group, Highland Park, IL, USA

Purpose: Observational outcomes studies, or registries, present a novel way of collecting naturalistic outcomes data associated with a particular disease or product. This workshop reviews the primary types of data collected in registries and the procedural and methodological challenges faced when analyzing those data.
Who Would Benefit: This workshop is intended for individuals who want to learn more about the methods of analyzing registry data. Individuals responsible for design and implementation of registries would also benefit from this workshop.
Workshop Description: Procedural Challenges: Analysis of observational studies has different challenges than analysis of randomized clinical trials. Obtaining accurate data on practice patterns, resource utilization, and patient-reported outcomes requires a naturalistic study design. As a result, treatment groups will not be randomized, sites who do not regularly perform certain diagnostic tests may have a great deal of missing data, and unexpected practice patterns are almost guaranteed. From a procedural standpoint, this means that a certain amount of directed exploratory analysis is usually necessary before a comprehensive analysis plan can be developed. The workshop provides concrete steps for meeting this procedural challenge through a process of successive refinement of the analytical approach. Methodological Challenges: The fact that the analysis of observational data tends to be less driven by a priori hypotheses leads to some specific methodological challenges. The absence of pre-specified hypotheses does not mean that the analysis is strictly descriptive; comparisons can still be made among groups even though those groups probably have not been pre-defined or randomized. However, there are special statistical techniques that need to be used to account for the likely differences between groups. Patients undergoing the various treatments or no treatment are likely to differ in material ways from each other. The workshop compares several statistical approaches to overcoming this problem, including matching, using propensity scores, and adjusting using covariates.

W25: HOW CAN HEALTH-RISK APPRAISAL DATA CONTRIBUTE TO CARDIOVASCULAR OUTCOMES RESEARCH?
Menzin J1, Brown J1, Manocchia M2
1Boston Health Economics, Inc, Waltham, MA, USA; 2BLUE CROSS AND BLUE SHIELD OF RHODE ISLAND, PROVIDENCE, RI, USA

Purpose: The objective of this workshop is to review the use of health-risk appraisals (HRAs) in health promotion, and describe how these data may supplement administrative claims in conducting outcomes research for cardiovascular disease.
Who Would Benefit: Persons involved in research using administrative claims data or cardiovascular outcomes research would benefit from this workshop.
Workshop Description: Cardiovascular disease is an active area for outcomes research. Administrative claims data, sometimes supplemented by clinical laboratory results, are often used to assess prevalence, treatment practices, and costs related to cardiovascular risk factors such as hyperlipidemia, hypertension, and diabetes. However, current data sources often lack key measures of cardiovascular risk, including smoking status, blood pressure, body mass index, and family history of heart disease. These measures and others are available through HRAs. In this workshop, we will outline the role of HRAs in health promotion and their usefulness as a tool in outcomes research. We will describe the process by which HRAs are linked to administrative claims data, and carefully appraise the advantages and limitations of the combined data source. Published examples and case studies will illustrate the use of linked HRA-claims data to examine cardiovascular treatment practices and associated costs of care.

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W27: THE ROLE OF OUTCOMES RESEARCHERS IN DESIGNING PATIENT REGISTRIES: CREATING PROGRAMS TO MEET BROAD ORGANZIATIONAL OBJECTIVES
Larson LR1, Malamis P2, Gordon MJ1, Trotter JP1
1Ovation Research Group, Highland Park, IL, USA; 2Galt Associates, Sterling, VA, USA

Purpose: Participants will gain an understanding of: 1) registry strategy and design principles; 2) trends in regulatory requirements for post-marketing safety-surveillance registries; and 3) opportunities for integrating outcomes measures within these programs.
Who Would Benefit: Outcomes researchers interested in exploring the design and application of patient registries to meet outcomes, commercial, and safety-surveillance objectives. Those with responsibility for implementing peri-approval programs to meet the growing demand for integrated commercial/risk management/outcomes data would also benefit.
Workshop Description: Growing demands from regulators, including the FDA and the EMEA, as well as from global commercial markets, to better document the real-world effects of new and marketed products are contributing to the necessity for – and proliferation of – patient registries. When designed appropriately, these registries represent a valuable new tool allowing outcomes researchers to collaboratively and efficiently meet a broad range of organizational objectives. While the responsibility for initiating a patient registry has traditionally fallen to marketing, clinical, or medical affairs, outcomes research groups are increasingly involved in these programs. Patient registries offer outcomes researchers an opportunity to create and access unique datasets. On a broader scale, patient registries can address a wide range of objectives - from developing a competitive differentiation strategy based on safety and effectiveness, to meeting post-launch regulatory commitments (including risk-management demands), to supporting a new product’s market entry and acceptance. Outcomes researchers can play a prominent role in directing the design and direction of these important programs. This interactive workshop will review registry strategy and design, including a discussion of approaches to incorporating both safety and outcomes measures within an integrated program. Global regulatory and risk-management trends will also be discussed, highlighting the potential value of these programs throughout the sponsor organization.


Compliance issues

W2: PATIENT COMPLIANCE AND PERSISTENCE WITH PRESCRIBED MEDICATION: MEASUREMENT AND HEALTH ECONOMIC IMPACT
Koncz TA1, Hughes D2, Cleemput I3, Annemans L4 for the Med Comp SIG
1Semmelweis University, Budapest, Hungary; AARDEX, Zug, Switzerland; 2University of Liverpool, Liverpool, United Kingdom; 3HEDM, Meise, Belgium; 4Ghent University, HEDM, Meise, Belgium

Purpose: Participants will learn about: 1) the advantages and limitations of various methodologies to measure compliance in clinical trials and practice and the analysis of these data for maximum explanatory power, relative to outcomes; 2) case studies on the health economic impact of variable compliance with prescribed drug regimens; 3) appropriate modelling strategies to represent various patterns of non-compliance in pharmaco-economic evaluations.
Who Would Benefit: Health care services researchers, policy-makers, and payers concerned with medication compliance issues as well as outcomes researchers interested in improving methods to incorporate non-compliance into economic evaluations.
Workshop Description: There is agreement among stakeholders in health care that a large minority of patients are partially or poorly compliant with prescribed drug dosing regimens. However, varying definitions and methods of measurement limit our capability to quantify the effect of variable compliance on outcomes, and to evaluate its health economic impact. Achieving the workshop objectives necessitates that current methods used to measure compliance be reviewed, highlighting their strengths and weaknesses, along with the kind of analytic procedures they support to interpret compliance data relative to outcomes. Second, we will consider the economic impact of medication non-compliance by applying the conventional “cost of non-compliance” approach and another approach potentially more meaningful to decision makers, i.e. that of examining the impact of non-compliance on the cost-effectiveness of pharmaceuticals. Careful attention will be given to assessing the role non-compliance plays in the distinction between efficacy and effectiveness. Examples will be drawn from our own research and published literature. Lastly, we will consider how data on medication non-compliance can be integrated into cost-effectiveness modelling. Participants will be encouraged to comment on presented methods, data, and interpretation. We will conclude with a discussion on how guidelines for economic evaluations could be extended to allow for non-compliance.

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W10: METHODS IN COMPLIANCE EVALUATION: DETERMINING AND ANALYZING APPROPRIATE MEASURES
Nichol MB1, Gwadry-Sridhar F2, Nau DP3, Cramer JA4 for the Med Comp SIG 1University of Southern California, Los Angeles, CA, USA; 2McMaster University, Hamilton, ON, Canada; 3University of Michigan, Ann Arbor, MI, USA; 4Yale University, West Haven, CT, USA

Purpose: This workshop is designed to elicit participation by the audience in the derivation of appropriate measures of medication compliance and evaluation of those measures in a variety of settings and diseases.
Who Would Benefit: Researchers, practitioners, and policy makers interested in the field of compliance would benefit from the discussion at this workshop
Workshop Description: Compliance with medications has become an increasingly important area of research as policy makers have perceived the extent and effect of the problem. Unfortunately, there are no standards in the field for such concepts as the definition of key compliance measures (such as categorical or continuous rates), duration of observation, outcome measures of interest, and the measurement of multiple medications within an observational profile. There are also issues with the analytic methods used in determining important statistical and clinical differences between medications and other interventions (such as violation of observational independence, historical bias, and measurement bias). Specific problems associated with retrospective database analyses will also be considered, including methods for controlling selection, assumptions regarding distributional normality, and approached for defining continuous use. Three members of the ISPOR SIG Analysis Standards Working Group will initiate the discussion of these issues by providing a brief description of alternative solutions to the analytic problems. Where possible, the discussion leaders will provide examples of methods that could alleviate analytic obstacles. Workshop participants will be encouraged to explore the strengths and weaknesses of the alternative approaches.

W18: MEDICATION COMPLIANCE (ADHERENCE): ISSUES AND DEFINITIONS CONSENSUS WORKSHOP
Compliance SIG I&MDWG1, Wong P2, Fairchild C3, Clause S4, Burrell A5, Cramer J6 for the Med Comp SIG
1ISPOR, ISPOR, NJ, USA; 2Good Samaritan Hospital, Dayton, OH, USA; 3Bristol-Myers Squibb, Plainsboro, NJ, USA; 4Albany College of Pharmacy, Troy, NY, USA; 5Aventis, Bridgewater, NJ, USA; 6Yale University School of Medicine, West Haven, CT, USA

Purpose: The objectives of the workshop are 1) to evaluate the applicability of the definitions in various practice settings; 2) to identify issues related to these proposed definitions in conducting and evaluating research; 3) to gather opinions for the further refinement of these definitions; and 4) to build consensus among ISPOR members for the early adoption of these definitions.
Who Would Benefit: Who Would Benefit: Researchers, clinical practitioners, academicians and research sponsors that are interested in medication compliance research and clinical practice.
Workshop Description: In 2003, the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) chartered a Medication Compliance Special Interest Group (SIG) to provide ISPOR members with a forum for discussing issues related to medication compliance (adherence) and treatment persistence. In addition, ISPOR anticipates that this SIG will stimulate research and evaluation on issues related to medication compliance, treatment persistence, and implications for health outcomes. Five working groups were created to reach the SIG’s objectives. These working groups are: 1) Issues & Methods Definition, 2) Analyses Standards, 3) Bibliography, 4) Education, and 5) Economics of Medication Compliance. This workshop is organized by the Issues and Methods Definition Working group. At this workshop, the working group members will provide background information regarding the derivation of the definitions and comparisons with similar definitions in the global medical literature. Participants will engage in small group discussions which will focus on issues with the application of proposed definitions. Examples include use in acute and chronic diseases, in clinical practice, in clinical research, in health policy, as well as various measurement tools (e.g., electronic medical records, electronic monitors, pill counts, serum levels, patient adherence questionnaires, etc). Finally, the small groups will present their recommendations to all the participants for discussion. Key issues raised in 2003 will be addressed and recommendations generated from this workshop will be published at the ISPOR SIG website.

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W26: EDUCATION ISSUES IN MEDICATION ADHERENCE
Adamus AT1, Tran H2 for the Med Comp SIG
1University of Texas M.D. Anderson Cancer Center, Houston, TX, USA; 2Lake Erie College of Osteopathic Medicine, Erie, PA, USA

Purpose: Formulate a plan of action to increase the rate of medication adherence by engaging participants in a discussion of the salient factors associated with medication adherence education and research.
Who Would Benefit: Anyone interested in 1) promoting patient medication adherence; 2) educating the public about medication adherence; 3 developing or integrating a curriculum centered around patient medication adherence; and 4) those responsible for teaching health professionals.
Workshop Description: Purpose: 1) Identify factors thought to lead to medication non-adherence; 2) Appreciate the underlying reasons associated with these factors; 3) Know why these factors and underlying reasons are important to adherence education. Who would benefit: Anyone interested in 1) promoting patient medication adherence; 2) educating the public about medication adherence; 3 developing or integrating a curriculum centered around patient medication adherence; and 4) those responsible for teaching health professionals. Workshop description: There is wide variation in the research on medication adherence making study comparisons and a single educational focus difficult. The development of educational content requires a different level of knowledge be delivered to different groups depending on their level of need. For clinicians, practical strategies to increase medication adherence should be expounded upon while researchers and educators may need more exposure to the measurement and analysis of medication adherence. The goal of the workshop will be to formulate a plan of action to increase the rate of patient medication adherence by discussing educational content and target groups. The workshop will begin with an overview of the most researched and prevailing factors thought to lead to medication non-adherence (e.g. poor communication), underlying causes (e.g. literacy and perception) identified in medication adherence research, and the impact of non-adherence. Workshop facilitators will then present selected target groups who would benefit from education on adherence issues. Participants will be given these target groups as topics to discuss in small groups. The goal of small groups will be to 1) brainstorm a plan of action to educate their assigned target group, 2) identify the most salient educational content for their selected target group. Participants will then present the consensus for their topics to the entire group.


Cost study methodology issues

W3: USE AND INTEGRATION OF FREELY AVAILABLE U.S. PUBLIC USE FILES TO ANSWER PHARMACOECONOMIC QUESTIONS: DECIPHERING THE ALPHABET SOUP
Cisternas M, Noe L
Ovation Research Group, Highland Park, IL, USA

Purpose: Participants will 1) learn about the various public use file (PUF) data sources available in the United States for pharmacoeconomic research, 2) evaluate PUFs for creating prevalence, resource utilization, and cost estimates, 3) identify which PUF sources are available and appropriate to answer disease-specific questions, and 4) learn how to integrate these sources into pharmacoeconomic studies.
Who Would Benefit: Individuals in health economics, outcomes research, marketing, and academic departments interested in utilizing publicly available data as primary or secondary sources for pharmacoeconomic research.
Workshop Description: Public use files can be valuable data sources for conducting pharmacoeconomic research. Databases include information based on patient surveys, medical records, outpatient visits, and inpatient stays, and can represent various patient groups and settings such as Medicare, Medicaid, VA, long-term care, elderly patients, pediatric patients, and more. Information can be used to develop research hypotheses, as well as to help establish the incidence and prevalence of a disease, treatment patterns, resource utilization, productivity and work loss, costs, and variables for sensitivity analysis. While many pharmacoeconomic studies can be strengthened through the analysis of PUF data, others, such as cost of illness studies, can be based solely on such data. This workshop will begin with a guided discussion concerning the questions that can be answered through PUF data. We will describe the various PUF databases available to answer these questions, along with brief examples of their use from the authors’ own work and other published articles. The pitfalls of using PUF data and how to mitigate them will also be discussed, and finally, guidelines for combining estimates from various sources into a single study will be described. Materials summarizing the various data sources, as well as URLs for data download or purchase from federal agencies will be provided.

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W11: AT THE FRONTIER: MAXIMZING THE VALUE OF CLAIMS DATA
Birnbaum H1, Greenberg P2
1Analysis Group, Boston, MA, USA; 2Analysis Group/Economics, Boston, MA, USA

Purpose: Participants will explore innovative claims data analysis methodologies regarding pharmacoeconomic outcomes analysis. The purpose is for participants to gain useful insights regarding analysis of administrative claims data bases containing a variety of components including medical, prescription drug, disability, and workers compensation claims, as well as sick leave and at-work productivity measures. Disease-specific examples will be discussed concerning each of the following categories: •Assessment of the relationship between direct and indirect costs •Examination of the clinical content of claims data for use in economic analysis including attention to proxies that can inform severity of illness adjustments •Adjustments for comorbidities •Analysis of the economic impact on caregivers of dependents’ illness experience •Estimates of the lifetime costs of illness •Identification of patterns of patient outcomes over time. The diseases that will be discussed would include: •Attention deficit hyperactivity disorder •Bipolar depressive disorder •Cancer •Major depression •Diabetes •Osteoporosis •Respiratory infections •Rheumatoid arthritis •Treatment resistant depression The participants will be invited to translate findings into inferences and interpretations. The workshop will conclude by discussing how these approaches can be adapted to the actual needs of audience participants.
Who Would Benefit: Outcomes researchers and research sponsors who are interested in designing and analyzing claims data studies regarding cost of illness and impact of treatment issues.
Workshop Description: At the Frontier: Maximizing the Value of Claims Data Assessing at-work productivity loss associated with the use of pharmaceuticals Learning

W19: QUALITY AND VALIDATION STANDARDS IN HEALTHCARE MODELLING
Beard S1, Davies L2, Payne K3, Earnshaw SR4
1RTI Health Solutions, Manchester, United Kingdom; 2University of Manchester, Manchester, United Kingdom; 3Manchester University, Manchester, Gtr. Manchester, United Kingdom; 4RTI International, RTP, NC, USA

Purpose: Participants will learn methods and practical steps to improve the validity and quality of health care decision models and improve adherence to published guidance on healthcare modelling standards.
Who Would Benefit: Healthcare decision makers and analysts involved in commissioning, developing and analysing or applying the results of computer based decision models to evaluate new and existing healthcare technologies.
Workshop Description: Decision modelling is an increasingly important and frequent approach in establishing the relative economic value and likely budgetary impact of new and existing healthcare technologies. The rationale for a model approach requires that clinical and resource cost data from more than 1 source are synthesised. The process of building the model to represent the clinical decision process also utilises data and opinion from a number of sources and perspectives. These factors lead to variation in the assumptions and biases that underpin the model structure and data selection, quality of data and overall quality of the model that is difficult to assess and interpret. A number of guidance statements have been published in recent years, aimed at providing a common reference point to assess the quality of such models. This workshop will summarise the key common elements of available guidance, then split into 4 facilitator led work groups to highlight practical steps to identify and minimise the impact of implicit biases, maximise the accuracy of the model structure and data, identify and quantify structural and data driven uncertainty and thereby attempt to optimize overall model quality. A number of model scenarios will be used to stimulate these discussions. The groups will reconvene to discuss and summarise the key messages to improve model quality and obstacles to the implementation of these guidelines. This will include presentation of practical examples illustrating different approaches.

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Formulary development research issues

W4: MANAGED CARE DATA: TOOLS AND METRICS FOR FORMULARY DEVELOPMENT
Shaya FT1, Mullins CD1, Wong W2
1University of Maryland, Baltimore, MD, USA; 2CareFirst BlueCross BlueShield, Baltimore, MD, USA

Purpose: This workshop will demonstrate the strengths of managed care claims databases used for formulary development. The aim of the workshop is to provide practical tools to manage and analyze claims data and interpret results used for the cost-effectiveness, value and clinical claims considered by formulary committees.
Who Would Benefit: Stakeholders, decision-makers, researchers and data analysts who have an interest in formulary selection and decision-making. Health care/regulatory professionals who practice in settings of managed care, hospitals, the pharmaceutical industry, consulting, data management, research or academia.
Workshop Description: There has been a growing interest in looking at managed care administrative claims databases - which are designed to process billing claims - to support clinical and cost-effectiveness arguments used in developing formularies. This hands-on workshop will examine practical issues and characteristics of managed care databases, and will outline their strengths and mention their limitations in answering research questions and supporting value claims. With active participation from the audience, we will propose novel tools to develop medical and prescription data queries, addressing data mining, cleaning and operationalization challenges. Participants will be guided in methods to merge and match medical and prescription claims, and will identify validity issues related to properly specifying the data fields (e.g. primary, secondary and tertiary diagnoses, or billed charges versus paid charges etc ..). We will outline bias and confounding concerns, and demonstrate tools to handle them in retrospective claims data, as compared to prospective data. We will propose measures to handle missing data, duplicate data, and data integrity, in the context of patient confidentiality, HIPAA and IRB regulations. Participants will use a case study to interpret the answers to questions of value statement, cost-effectiveness, risk projection, compliance modeling and budget impact. We will assess bias, comparing and contrasting with the clinical trial design. By the end of the workshop, the participants will be able to apply the tools for research geared at formulary development. Purpose: 1)Learn the tools to place a request for managed care administrative claims data to answer a research question, 2)Understand the bias and strengths of arguments supported by claims data analysis, 3)Interpret results from a claims data analysis, in comparison to results from prospective studies, for formulary considerations.

W12: THE FORMULARY DECISION PROCESS: WHAT KIND OF INFORMATION IS TRULY HELPFUL?
Van Den Bos J1, Mullins CD2
1Milliman USA, Denver, CO, USA; 2University of Maryland School of Pharmacy, Baltimore, MD, USA

Purpose: Purpose: Participants will learn: a) about the formulary decision process, based on a review of the literature and on observational study; b) where decision-makers are not being served; and c) changes that can improve the transfer of information to decision-makers from both an academic and an actuarial perspective.
Who Would Benefit: Researchers and their sponsors whose intended audience is MCO formulary decision-makers, and formulary decision-makers.
Workshop Description: A recent report of the ISPOR Task Force on the Use of Pharmacoeconomic/Health Economic Information in Health-Care Decision Making found, unfortunately, that pharmacoeconomic research is not widely used by MCOs. Reasons for this include: the absence of total budget impact analysis, lack of transparency in methods, and models that are not specific to an MCO’s covered population. This workshop is designed to: a) examine the workings of formulary decision-making groups; b) pinpoint where the needs of these groups are not being met; and c) suggest changes that would make pharmacoeconomic information more useful to MCOs. We will begin with a review of the literature on P&T Committees, with special focus on their use of pharmacoeconomic information. We will supplement this with information gleaned from direct observation of P&T Committee meetings and decision-makers. Then we will examine features of economic research that cause P&T Committees to place low value on pharmacoeconomic findings. We will conclude by discussing changes that would make economic research more useful to P&T Committees, such as methodological and reporting improvements. Suggestions will also include an actuarial perspective on the elements of good budget impact modeling, such as consideration of the payer (e.g. commercial, Medicare), benefit design, demographic characteristics, treatment patterns, condition incidence and prevalence, the rate at which the drug will enter the system, medical cost offsets, and degree of medical management.

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W20: THE ART AND SCIENCE OF BUDGETARY IMPACT MODELING FOR FORMULARY SUBMISSIONS
Taylor DC, Thompson D
Innovus Research, Inc, Medford, MA, USA

Purpose: To share tips and techniques for building scientifically rigorous budgetary impact models while maintaining transparency and a user-friendly interface.
Who Would Benefit: Applied researchers or research sponsors interested in including budgetary impact models in dossiers for formulary submissions, as well as potential end users of such models.
Workshop Description: Budgetary impact models (BIM) are increasingly used in formulary submissions to help decision-makers assess the potential financial implications of adding a new therapy to their formulary. These models must incorporate complex marketplace dynamics such as product substitution effects and the potential for market expansion, but need to be flexible enough for users to modify the model parameters to fit their own institution’s population, practice patterns, and costs. The art of creating a successful BIM lies in making it transparent enough so that the user can understand all of the underlying assumptions and calculations, simple enough to be used with little or no training, yet complex enough to capture the cost and science of the disease and its treatment options in a world with and without the treatment being evaluated. This workshop will draw on the presenters’ experience in balancing these sometimes conflicting objectives in a BIM. In true workshop fashion, participants will be asked to share their experiences on a variety of relevant issues, including population selection, event rates, treatment regimens, and cost determination. Discussions will also focus on how methodologic and interface design decisions may affect the accuracy, usability, and perception of the model. Particular attention will be paid to the implications of incorporating the element of time into a BIM, including the need to estimate screening costs, discount rates, discontinuation rates, and plan turnover rates.

W28: ASSESSMENT OF DATASOURCE OPTIONS FOR OUTCOMES RESEARCH AND FORMULATY DECISIONS
Brixner DI1, Tabb K2, Stockdale B1, Holubkov R1
1University of Utah, Salt Lake City, UT, USA; 2GE Medical Systems Information Technologies, Berkeley, CA, USA

Purpose: Participants will learn i) to describe the types of data available from various sources ii) compare and contrast the value of these data sources in conducting outcomes research iii) determine which datasets best answer what type of research question and iv) describe how data from each source can define new product value for formulary decisions
Who Would Benefit: Individuals involved in the conduct or evaluation of outcomes research studies deciding which type of database can best answer specific research questions and decision makers deciding where to go for information in defining value for new products coming to market.
Workshop Description: Various types of datasets will be described including patient registries, public sector databases, clinical trail and observational study datasets, retrospective claims and electronic medical record databases. Each of these sources provides varying degrees of information that is useful in the conduct of outcomes research and in the context of formulary decision making. Patient registries and public sector databases such as the National Health and Examination Survey (NHANES) provide information on the current statistics for a particular disease state. Datasets from clinical trails and observational studies can provide information to project the potential impact of a new product once available in the marketplace. Claims databases are derived from various sources where there are paid reimbursements for medical services, including managed care datasets. These sources may provide accurate information on drug utilization, however few of these databases provide information on outcome measures such as laboratory values, blood pressures or body weight. An electronic medical record can provide extensive data in these areas. The advantages and limitations of each type of dataset will be discussed both for outcomes research and formulary decision making. Assessment of which type of datasource to use for different types of research or value questions will be explored through the presentation of two outcomes research scenarios where the audience will assist in deciding which type of datasource would best answer the questions.

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Healthcare policy development issues using outcomes research

W5: SYSTEMATIC REVIEWS OF COST-EFFECTIVENESS ANALYSES USING ELECTRONIC AND WEB-BASED REGISTRIES
Greenberg D, Neumann PJ
Harvard School of Public Health, Boston, MA, USA

Purpose: The objectives of this workshop are to familiarize participants with various comprehensive electronic sources of economic evaluations. Participants will learn the potential applications and limitations of these sources; discuss the current practice and the main methods used in cost-effectiveness studies, and changes in CEA practice over time. We will also identify the “under-studied” areas of cost-utility and CEA in clinical medicine that will need future attention. Participants will then individually complete a series of web searches of studies of their interest, and explore cost-effectiveness ratios and utility weights for different health states. The workshop will conclude by discussing the documented lack of standard in this field and the potential barriers for using cost-effectiveness data for coverage and reimbursement decision-making.
Who Would Benefit: Outcome researchers (both in academia and pharmaceutical and medical device industry) involved in economic evaluation of health interventions, those interested in conducting reviews of CEAs, public health department officials, and medical directors at different levels of healthcare systems making, reimbursement and resource allocation decisions.
Workshop Description: Cost-effectiveness analysis (CEA) has become a popular technique to support resource allocation decisions, and the number of CEAs published has been increasing steadily in recent years. Due to the growing importance of these studies, several efforts to create comprehensive electronic or web-based sources (e.g., National Health Service Economic Evaluation Database-NHSEED, the Health Economic Evaluation Database-HEED, and the Harvard School of Public Health Cost-Effectiveness Analysis Registry), summarizing data from economic evaluations, have been made. Although many similarities exist, the focus of these databases and the type of information presented vary. The objectives of this workshop are to familiarize participants with various comprehensive electronic sources of economic evaluations. Participants will learn the potential applications and limitations of these sources; discuss the current practice and the main methods used in cost-effectiveness studies, and changes in CEA practice over time. We will also identify the “under-studied” areas of cost-utility and CEA in clinical medicine that will need future attention. Participants will then individually complete a series of web searches of studies of their interest, and explore cost-effectiveness ratios and utility weights for different health states. The workshop will conclude by discussing the documented lack of standard in this field and the potential barriers for using cost-effectiveness data for coverage and reimbursement decision-making.

W13: THE USE OF DISCRETE EVENT SIMULATION TO ASSESS THE VALUE OF HEALTH CARE INTERVENTIONS
Caro JJ1, Huybrechts KF1, Möller J2
1Caro Research Institute, Concord, MA, USA; 2Caro Research Institute, Eslov, Sweden

Purpose: To demonstrate that discrete event simulation is a versatile, worthwhile technology for current outcomes research problems.
Who Would Benefit: Health care decision makers, policy makers and researchers who are responsible for conducting, evaluating and utilizing health economic analyses.
Workshop Description: It is by now well accepted that a model of the disease and its management is required to estimate the value of most health care interventions. Economic models have typically been structured and analyzed using decision trees or Markov models, despite recognition of the major limitations of these techniques. In this workshop, a much more natural, less limiting technique is presented. Although the approach — discrete event simulation (DES) — has a long trajectory in operations research, it has to date been rarely employed in medicine. Through interactive group exercises using an example of a typical problem in valuing a health care intervention, we will illustrate how the needs of decision makers translate to technical requirements for the models and how these can be met with the new technique vis a vis traditional modeling approaches. The key elements — entities and their attributes, events, the simulation clock, resources and queues, and computational algorithms — will be presented, and the process for implementing the models will be illustrated using available software. A DES represents the course of disease very naturally with few restrictions. Neither mutually exclusive branches nor states are required, nor is the cycle fixed. All relevant aspects are incorporated explicitly and efficiently. Flexibility in handling perspectives and carrying out sensitivity analyses, including structural variations, is incorporated and the entire model can be presented very transparently. We propose that DES should be the method of choice in carrying out health care evaluations, particularly those aimed at informing policy makers and at estimating the budget impact of an intervention.

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W21: IMPLICATIONS OF CANADA/US PARALLEL TRADE IN PRESCRIPTION DRUGS: A CANADIAN PERSPECTIVE
Palmer WN
Palmer D'Angelo Consulting Inc, Ottawa, ON, Canada

Purpose: To provide an understanding of Canada/US cross border trade, the factors contributing to lower Canadian prices, implications for Canadian pricing and reimbursement and the outlook for parallel trade in North America.
Who Would Benefit: Health Policy Analysts, Pricing Managers, Reimbursement Managers
Workshop Description: The growth of Canadian internet pharmacies and the debate over the importation into the United States of lower-priced Canadian prescription drugs raises questions of why Canadian drug prices are lower than those in the United States and if these lower prices can be sustained into the future. In Canada both the public and private sectors play an important role in pharmaceutical reimbursement although it is government policies that have limited drug prices since 1987. This workshop is designed to assist decision makers understand Canada’s pricing and reimbursement environment and the impact the cross border trade issues are having on Canadian drug prices. We will discuss the role of the Patented Medicines Prices Review Board (PMPRB), which regulates prices of patented medicines, illustrate pharmaceutical price trends and international price comparisons, and describe the impact of government policies on prices. We will explain provincial reimbursement programs and outline the role and growing importance of pharmaco-economics and outcomes research in light of increasing drug prices. Parallel trade (internet pharmacies, re-importation legislation) with the United States has resulted in upward pressure on Canadian drug prices as well as new distribution arrangements targeted at limiting supplies to Canadian pharmacies. Recently, Canadian pharmaceutical manufacturers have challenged decade old price regulations and Canadian prescription drug prices have begun moving closer to US prices. In light of these and related developments, the workshop will consider the outlook for parallel trade in North America and the implications on pricing and reimbursement in Canada. The workshop will encourage discussion and debate on the desirability of parallel trade and what measure if any policy makers in Canada and the United States should implement to encourage or discourage cross border trade in prescription drugs.
 

W29: DEVELOPING A STRATEGY FOR PROMOTING THE USE OF ECONOMIC EVALUATION IN HEALTH CARE DECISION MAKING IN LATIN AMERICA
Rovira J1, Bertozzi SM2
1The World Bank, Washington, DC, USA; 2Instituto Nacional de Salud Publica, Cuernavaca, Morelos, Mexico

Purpose: To review preliminary evidence and discuss the best strategies for increasing the application of economic evaluation to actual decision making in the health sector
Who Would Benefit: Health care decision makers as well as researchers interested in ensuring the practical application of economic analyses
Workshop Description: In many countries there is a clear disconnect between the analytical work of economic evaluation researchers and the decision-making processes. Although the former are able of producing work of acceptable and high quality, decision makers seem to ignore this work in everyday decisions. The workshop will present the experience of an initiative aimed at bridging that gap in nine LA countries: Argentina, Brazil, Colombia, Cuba, Mexico, Nicaragua, Peru, Uruguay and Venezuela. The key aspects of the strategy were that will be presented are: An assessment of the situation of economic evaluation in each country based on a review of the studies and literature on the topic and an identification of the human and institutional resources available. An analysis of the decision making process and the identification of the potential users of economic evaluations, as well as some analyses carried out in order to eliciting the knowledge and attitudes of these decision makers in relation to economic evaluation tools. The strategy includes the development and dissemination of standard user friendly tools – a software program for economic evaluation and cost databases – that might reduce the cost of carrying out the studies and to increase their transparency and credibility to non expert users. The previous analyses allowed to identify the main bottlenecks that limit a more widespread use of economic evaluations at country level and to define strategies at national level aimed at overcoming the existing obstacles. Workshop participants are expected to provide a critical feedback to the proposed strategy and to share similar experiences aimed at improving the use of tools by decision makers. The work has been the result of a collaborative activity of two projects: NEVALAT “Thematic Network on the Economic Evaluation of Health Programmes and its Application to Decision Making in Latin American Countries” and the RED BOBADILLA.

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Preference-based studies methodology issues including utility studies

W6: ADVANCES IN MAPPING HEALTH-RELATED QUALITY OF LIFE: AN EQ-5D INDEX BASED ON SF-36 DATA
Kind P1, McEwan P2, Currie CJ3, Dixon S4
1Outcomes Research Group, York, United Kingdom; 2Cardiff University, Cardiff, Wales, United Kingdom; 3University of Wales College of Medicine, Cardiff, Wales, United Kingdom; 4Sheffield University, Sheffield, South Yorkshire, United Kingdom

Purpose: To present current methods of mapping alternative measures of health-related quality of life. To discuss the potential benefits of new and more advanced modelling techniqes. To provide examples of the application of these techniques in mapping SF-36 onto EQ-5D.
Who Would Benefit: Those concerned with the analysis of health-related quality of life data for use in cost effectiveness analysis and specifically those with access to SF-36 who require a single-index preference-weighted summary score.
Workshop Description: Single index values are required for use in cost-utility analysis. Where profile measures such as SF-36 have been used in clinical trials it can be extremely difficult to recover useful data for applications that require a preference-weighted summary measure. One option, where utility estimates are not directly available, is to predict such values using mapping methods. This workshop will present examples based on both hospital and non-hospital data to highlight and contrast issues surrounding the mapping of SF-36 data to EQ-5D. The first data set, the Health Outcomes Data Repository (HODaR), contains SF-36 and EQ-5D data on more than 25,000 hospital in-patients alongside targeted disease-specific measures. The second data set, Health Survey for England, contains SF-36 and EQ-5D data on some 16,000 adults drawn from a representative sample of the general population. The workshop will demonstrate the extent to which the EQ-5D domains and utility scores can be predicted from SF-36 data. Results based on mapping using standard regression techniques will be demonstrated and compared with results obtained using more sophisticated methods based on flexible, non-linear models such as neural networks and classification and regression trees (CART). The benefits of indirect mapping to sub-domain scores rather than single utility score will be demonstrated. Improvement in these methods has many applications particularly where utility estimates are absent from costly, large-scale clinical trials

W14: INCORPORATING PREFERENCES INTO OUTCOME MEASURES FOR THE VALUATION OF GENETIC BASED SERVICES
Payne K1, Jarrett J2, Davies LM1
1Manchester University, Manchester, Gtr. Manchester, United Kingdom; 2University of East Anglia, Norwich, United Kingdom

Purpose: Participants will learn about (1) outcome measures currently used to evaluate genetic services (2) the need for and qualitative and quantitative methods to develop tools to measure and value, genetic services.
Who Would Benefit: Researchers and decision-makers responsible for the development, application and interpretation of outcome measures in the evaluation of genetic based services.
Workshop Description: Unlike conventional medicine, genetic care typically does not have a direct effect on the prevention, amelioration or cure of disease symptoms, but affect other aspects of people’s life-choices and well being (e.g. decision about whether to marry or have children). Standard measures of health status and health related quality of life are unlikely to be adequate for the evaluation of genetic services. The nature of the key attributes to include in an array of tools to measure and value genetic services is poorly understood. This workshop will introduce participants to (1) the limitations of current outcome measures and (2) an overview of qualitative and quantitative methods to develop instruments to measure and value the (dis)benefits of genetic services. Using a case study of a genetic counselling service, participants will be asked to form a focus group, and under guidance from the presenters, critique an existing outcome measure. Participants will then identify the factors they feel are important in the delivery and outcomes of this service. The presenters will then demonstrate how these factors can be used to generate attributes and levels to use in the design of a stated preference tool (discrete choice experiment). The workshop will conclude by discussing how the development of stated preference instruments could be taken forward to provide robust measures to inform decision-making.

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W22: QUALITY ADJUSTMENT USING AVERAGE UTILITIES: VIRTUE OR VICE?
Caro JJ, Huybrechts KF
Caro Research Institute, Concord, MA, USA

Purpose: To examine the basis for using average utilities in cost-effectiveness analysis, its appropriateness and its potential impact on decision-making.
Who Would Benefit: Health care decision makers, policy makers and researchers who are responsible for conducting, evaluating and utilizing cost-utility analyses.
Workshop Description: A major challenge in economic analyses has been to find a way to aggregate the disparate health effects into a single measure that can be used comparatively and can be combined with the costs into a single criterion of economic efficiency. The most prominent contender for a solution to this problem has been the construct of quality adjusted life years (QALY). Survival is quality adjusted using weights attached to each of the possible health states. These weights have been estimated as the average obtained from patients (or their surrogates), health professionals or the general public (or by expert opinion!). There are major problems with this use of average weights. A fundamental issue is that it demands an absolute scale across all possible outcomes — an impossible task. More importantly, even if this flaw is ignored, average values mask potentially large individual variations, both cardinal (different people value health states differently) and ordinal (disagreement in terms of ranking). Ignoring the heterogeneity may distort the health effects to such an extent that they misrepresent the value of some outcomes and thus impair optimal decision-making, as will be shown using an example. Throughout the workshop, the theoretical concepts, beginning with the sound original approach to individual decision-making, will be illustrated by means of interactive group exercises and views of the participants will be vigorously elicited. Recommendations for improved application of these concepts will be made.

Quality of life study methodology issues including patient reported outcomes

W7: APPLYING ITEM RESPONSE THEORY MODELING FOR IMPROVING HEALTH OUTCOMES MEASUREMENT
Reeve BB
National Cancer Institute, Bethesda, MD, USA

Purpose: Participants will learn i) the basic concepts and methods of item response theory, ii) what differentiates traditional and modern measurement theory, and iii) potential applications of item response theory modeling for health outcomes measurement.
Who Would Benefit: Health outcomes researchers with interest in scale development or evaluation.
Workshop Description: There is a great need in health outcomes research to develop instruments that accurately measure a person’s health status with minimal response burden. This need for psychometrically sound and clinically meaningful measures calls for better analytical tools beyond the methods available from traditional measurement theory. Applications of item response theory (IRT) modeling have increased considerably because of its utility for instrument development and evaluation, assessment of measurement equivalence, instrument linking, and computerized adaptive testing. IRT models the relationship, in probabilistic terms, between a person’s response to a survey question and their standing on a health construct such as fatigue or depression. This one hour workshop will discuss the basics of IRT models and applications of these models to improve health outcomes measurement. Illustrations will be used throughout the presentation that focuses on measuring key health-related quality of life domains in breast cancer survivors.

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W15: SELECTING OUTCOME MEASURES FOR INTERVENTION STUDIES: MAXIMIZING USE OF WEB-BASED RESOURCES
Balkrishnan R1, Kelly PA2
1University of Texas School of Public Health, Houston, TX, USA; 2Baylor College of Medicine and Houston Center for Quality of Care and Utilization Studies, Houston, TX, USA

Learning Objectives: Participants will learn: 1) utilization of web-based resources to identify appropriate outcome measures for intervention studies 2) develop an understanding of the outcomes framework and aspects of psychometrics that will assist in selection of appropriate measures, and 3) develop a complete understanding of freely available and newly introduced web resources such as the METRIC that have been developed to assist researchers with outcome measure selection.
Who Would Benefit: Applied health services researchers with little background or training in health psychometrics and evaluation of patient centered outcomes, researchers involved in intervention studies, and graduate students interested in outcomes evaluation.
Workshop Description:  Many applied health services researchers with limited background and training in health psychometrics or outcomes evaluation are faced with the dilemma of choosing appropriate patient-centered outcome measures for intervention studies. This workshop provides such researchers the basic tools needed to appropriately and efficiently select such measures making maximum use of available web-based resources available for this purpose. The workshop will begin with a detailed description of the outcomes evaluation framework in intervention research delineating the role of premeditating factors, treatment, and short and long-term outcomes. This will be followed by delineation of key types of generic and condition-specific outcome measures, as well as discussion of issues in development and application of these measures, using examples from the presenters own recent research. This will be followed by a brief discussion of pertinent health psychometric issues such as reliability and validity measurement, as well as practical administration issues. A detailed hands-on demonstration of web-based databases such as the METRIC will follow, where the participants will be able to understand how knowledge of this basic outcome measure selection-related information can be used to identify the appropriate outcome measure for their intervention study. Finally a brief self-evaluative exercise will be provided to participants.

W23: CHALLENGES IN DEVELOPING INSTRUMENTS TO SHOW THE VALUE OF NEW THERAPIES FOR SEVERE MENTAL ILLNESS
Rothman M1, Awad GA2, Revicki D3, Speight J4
1Johnson & Johnson, Raritan, NJ, USA; 2River Regional Hospital, Toronto, Canada; 3MEDTAP International, Inc., Bethesda, MD, USA; 4Oxford Outcomes, Ltd., Cassington, Oxford, UK


Purpose: Participants will gain a greater understanding of the complexities of measuring outcomes in clinical trials of new therapies for severe mental illness. Participants will have the opportunity to participate in discussion of a research agenda to address these issues.

Who Would Benefit: Health outcomes researchers in academia, industry and government interested in assessing outcomes in severe mental illness.

Workshop Description: This workshop is presented by health outcomes researchers with extensive experience in the development of outcome measures used to assess new therapies. There is increasing recognition of the importance of understanding the patient perception of new therapies for many conditions and findings based on this experience are accepted for approval and promotion of products. However, there remain challenges to obtaining credible evidence from persons with severe mental illness that is acceptable to regulatory authorities and clinicians. Examples of these challenges include determining when a respond or a proxy may provide reliable data, identification of an appropriate baseline, outcome specificity and evaluating outcomes in multiple

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W30: COLLECTING PATIENT-REPORTED OUTCOMES DATA OUTSIDE THE CLINICAL TRIAL PARADIGM
Mathias SD, Colwell HH, Matosian ML
Ovation Research Group, Highland Park, IL, USA

Purpose: There are distinct benefits of collecting PROs within non-clinical trial ("real world") study designs. Without disrupting patient care, real world studies can be designed to provide data on the cost and outcomes under usual care conditions. Participants will gain an understanding of recent advances which have resulted in the use of innovative approaches in the collection of PRO data.
Who Would Benefit: The workshop is intended for individuals from pharmaceutical, biotech, and device companies as well as academic researchers responsible for the design and conduct of late phase, real world outcomes studies, including disease and product registries.
Workshop Description: Real world, late phase studies offer many advantages over clinical trials for the collection of outcomes data. These include their increased external validity, reduced burden on both investigators and subjects, the use of more relevant comparators, and the lack of protocol-mandated visits and procedures. Thus without disrupting patient care, real world studies can be designed to provide data on the cost and outcomes under usual care conditions. Recently, technological advances have resulted in the more frequent use of electronic data collection (EDC) methods for the collection of PRO data. This workshop will highlight many of the available options for the collection of PRO data and will address relative strengths and weaknesses of these innovative approaches. Several case studies will be presented which incorporate these new advances in late phase studies. Audience participation will be encouraged through an activity that uses a case study to review and recommend an approach for the collection of PRO data.


W31: CANCER-SPECIFIC PATIENT REPORTED OUTCOMES: DO THEY YIELD USEFUL INFORMATION?
Gondek K1, Kamath CC2, Copley-Merriman K3
1Bayer Pharmaceuticals, West Haven, Connecticut, USA; 2Mayo Clinic, Rochester, Minnesota USA; 3Pfizer, Inc, Ann Arbor, Michigan

Purpose: The participants of this workshop will gain an understanding of the challenges faced in collecting and analyzing patient reported outcomes in cancer. Insight regarding changes necessary to demonstrate the value of patient reported outcomes in cancer.
Who Would Benefit: Health outcomes researchers in academia, industry, government or regulatory bodies. Decision-makers at all levels of clinical practice.
Workshop Description: The potential utility of patient-reported outcomes in cancer has been called into question (e.g., the Goodwin & Gantz paper). The many factors responsible for this include unreasonable expectations, old conceptual foundations as well as poor methodological and statistical rigor. The concept of a clinically significant difference in outcomes is emerging as an aid to clinical decision making and in design of clinical trials. This could potentially contribute to greater utility of these outcomes. But is this another attention-grabber or a concept that holds genuine promise? What will it take to ensure better use of patient-reported outcomes in the clinical care of patients? What will it take to ensure better assessment of patient-reported outcomes in clinical trials?

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Risk assessment/risk management issues

W8: THERAPEUTIC EXPANSION OF THE CLASSICAL Q-TWIST APPROACH
Sherrill B1, Irish W2
1RTI Health Solutions, Research Triangle Park, NC, USA; 2RTI Health Solutions, Durham, NC, USA

Purpose: To provide enough understanding of the Q-TWiST methodology to evaluate treatment differences on successive health states such that researchers can consider it for applications in therapeutic areas not traditionally served with this approach.
Who Would Benefit: Healthcare decisionmakers, outcomes researchers and pharmacoeconomists who are responsible for assessing the relative benefit of time-related treatment effects and balancing treatment advantages against the risks of adverse events.
Workshop Description: Increasingly, researchers are called upon to weigh the clinical and/or quality-of-life benefit of treatments for chronic conditions against the risk of adverse events. Typically, treatment differences in efficacy and safety from clinical trials are evaluated separately. The Q-TWiST methodology represents a unique analytical opportunity to simultaneously evaluate both the quantity and quality of successive health states. Q-TWiST stands for Quality-adjusted Time Without Symptoms or Toxicity and was originally developed for oncology studies. Kaplan-Meier analyses are used to estimate time spent in clinically different health states, usually toxicity, symptom-free period and disease relapse. These states are weighted with utility scores to represent relative value to the patient then summed to provide a Q-TWiST score. Treatments may then be compared for their overall effect on the patient experience with this single measure that encompasses adverse events, time to disease progression or clinical improvement, and relapse or sustained response. We will provide an overview of the analytical approach and demonstrate how the Q-TWiST methodology can be extended to more than three health states. The guided interactive part of the workshop will use a case study in depression, a non-traditional area for Q-TWiST analyses. Participants will discuss interpretation of results and determine when Q-TWiST is applicable. They will outline the pros and cons of Q-TWiST compared to other measures. They will then be invited to brainstorm applications of the Q-TWiST approach in additional therapeutic areas, such as Alzheimer’s Disease.

W16: CAPTURING THE VALUE OF PHARMACEUTICALS: THE ROLE OF OUTCOMES RESEARCH IN PRICING DECISIONS
Tierce JC1, LaPensee KT2, Wierz DJ3, Sugano DS4
1ValueMedics Research, LLC, Arlington, VA, USA; 2Skylands Healthcare Consulting, LLC., formerly of Cambridge Pharma Consultancy, Hampton, NJ, USA; 3Independent Consultant, formerly of Wyeth, Inc, Exton, PA, USA; 4Schering-Plough Corporation, Kenilworth, NJ, USA

Purpose: This workshop helps outcomes researchers assume an active role in pharmaceutical pricing. At the conclusion, attendees will understand the principals for integrating pricing and reimbursement with outcomes research and health economics to determine, demonstrate and capture product value.
Who Would Benefit: This session is for those engaged in pricing, reimbursement, outcomes research and health economics. Anyone with an interest in integrating these disciplines will benefit.
Workshop Description: As medical technologies cost more to develop and launch, health budgets are receiving greater scrutiny for the value for money of each component. While outcomes researchers traditionally assess value of pharmaceuticals, they rarely apply their skills to product pricing. However, outcomes researchers involved in drug development know the most about product value. This sets up a disconnect—or worse, a “train wreck”—between business planning, expectations, and the value that can be credibly demonstrated. Capturing product value within today’s global market is most effectively accomplished by applying the skills of outcomes research and health economics to pricing and reimbursement throughout the product development and commercialization processes. In this workshop, attendees will learn pricing strategies from the perspective of (a) value determination, including market assessment, reimbursement and product/portfolio revenue and profit forecasts, (b) value demonstration, including studies, information considerations, models and pricing research, and (c) value capture, including outcomes research in value-based pricing research and the development of a product pricing strategy, qualitative and quantitative techniques in value-based pricing research, market segment differences and dynamics in pricing and reimbursement and applying the knowledge gained through participating in that process to reimbursement applications and contracting negotiations with payers. The workshop format will include case studies where participants will discuss the appropriate application of outcomes research to pricing decisions. Attendees will also have the opportunity to ask questions and share experiences with integrating these disciplines.

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W24: RISK MANAGEMENT STRATEGIES USING PATIENT REGISTRIES
Tennis P1, Andrews E1, Johnson FR2
1RTI Health Solutions, RTP, NC, USA; 2Research Triangle Institute, Research Triangle Park, NC, USA

Purpose: The objective of this workshop is to demonstrate the utility of patient registries as an integral part of a risk management strategy throughout drug development. We will discuss methodologic and regulatory issues which arise in performing such registries and utilize an audience participation exercise to illustrate these issues.
Who Would Benefit: This workshop is useful for people who wish to learn 1) how information from registries contribute to an effective product strategy, 2) how to evaluate elements of risk management programs (RMPs), and 3) how patient-reported outcomes contribute.
Workshop Description: The objective of this workshop is to demonstrate the utility of patient registries as an integral part of a risk management strategy throughout drug development. We will discuss methodologic and regulatory issues which arise in performing such registries and utilize an exercise to illustrate these issues. Focus on risk management throughout drug development has resulted in closer monitoring of the risk/benefit profiles during the pre-approval phase; and at times, therapies may only be approved with Risk Management Programs (RMPs) requiring education, focused prescribing, or other mandatory elements. This focus on risk management has generated interest in registries of patients with specific diseases and post-approval registries of patients using a therapy. Disease registries can identify the context for safety signals and facilitate rapid response to a shifting product profile during the pre- and post-approval phases. Post-approval therapy registries can generate data to assess whether elements of a RMP are achieving their goals, including whether patients receive and perceive information as intended, whether they utilize medications as prescribed, and whether intended types of patients receive the therapy. Therapy registries can also evaluate the characteristics of patients who most benefit from therapy. Included stated preference surveys of patients quantify their value trade-offs between clinical benefits and risks. Follow-up assessments can be used to assess patients’ perceptions of the impact of therapy (in therapy registries) or disease (in disease registries). Responses from clinicians and patients permit the integration of clinical data and patient-reported outcomes. This workshop is useful for people who wish to learn 1) how information from registries contribute to an effective product strategy, 2) how to evaluate elements of risk management programs (RMPs), and 3) how patient-reported outcomes contribute.

W32: POTENTIAL ROLE OF PRO ASSESSMENTS IN RISK MAMANGEMENT PROGRAMS
Marquis P, Piault E
Mapi Values, Boston, MA, USA

Purpose: To understand the potential role of patient reported outcomes (PRO) in risk management and more specifically to determine 1) when PRO should be evaluated in a clinical development to support the meaningfulness of the drug efficacy, and 2) how the use of PROs will contribute to better assess the benefit in real life for risk management programs.
Who Would Benefit: Outcomes researchers involved in the clinical development and marketing activities, regulatory affair managers.
Workshop Description: The creation of the Study Endpoint and Label Development group (FDA/CDER/OND) and its interaction with the review divisions demonstrates the acceptance of PRO as important endpoints, and also emphasizes the need for quality. Recently, FDA has put a lot of focus on risk management (concept paper, rule for safety reporting requirements). Risk management concentrates mainly on safety issues and little room is given to the evaluation of the treatment benefit. Therefore, regulators are in need for more meaningful endpoints demonstrating the drug benefit particularly from a patient perspective that will complete the data from traditional “objective” endpoints. Indeed the role of PRO in the new risk management paradigm has to be better defined. During the workshop, the emphasis will be put on practical considerations when implementing risk management programs (role of PRO in light of the type of pivotal endpoints, characterization of severity levels, identification of responders…) and strategies will be proposed. Success stories (i.e., Lotronex, Accutane) will be discussed. The workshop will help PRO researchers and regulatory affair managers to better determine the relevant PRO endpoints needed to support drug approval and design efficient risk management program. This would finally contribute to support the availability of those products where the benefit as perceived by the patients is worth the risk of the treatment.

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