Quality of Life/Patient-Reported Outcomes/Valuation Issues
 

WW1	CAN THE SF-36 BE USED IN CALCULATING QALYS?

Lee TA1, Hollingworth W2, Kind P3, 1Hines VA Hospital, Hines, IL, USA; 2University of Washington, Seattle, WA, USA; 3University of York, York, United Kingdom
Learning Objectives: Several algorithms have been developed that use the SF-36 to provide a summary score on a scale from 0 to 1. The intent behind the algorithms was to provide a means by which QALYs can be calculated from SF-36 data in studies not including a preference-based measure of outcome. This workshop aims to: 1) Review published SF-36-to-preference derivation algorithms emphasizing strengths and weaknesses of each approach,
2) Compare SF-36 derived preferences with directly elicited preferences, and 3) Discuss implications of using derivation algorithms for QALY estimation. Following the workshop attendees will be familiar with published algorithms available for estimating preferences from the SF-36, understand differences between the algorithms and appreciate how derived preferences compare to directly measured preferences. They will also be aware of safe limits to use of all these different options.
Who Would Benefit: The target audience is individuals interested in or considering the use of SF-36 derived preferences in pharmacoeconomic studies or those interpreting results from studies using the algorithms. However, anyone involved in cost-utility analysis and preference elicitation would also find the workshop beneficial.
Workshop Description: Not all studies include preference-based outcome measures. However there may still be a desire to conduct a cost-utility analysis in such studies, which has led to the development of several algorithms that seek to estimate health state preferences from SF-36 data. Were this process to become generally accepted, researchers would be able to conduct cost-utility analyses in studies not including a preference-based measure but including the SF-36. However, there is limited information on the performance of these algorithms in comparison to directly elicited preferences. This workshop will review published derivation algorithms and compare methods used in the algorithms. We will compare estimates from derivation algorithms with directly measured preferences and discuss implications of using derivation algorithms to calculate QALYs.  

Fehnel SE, McLeod LD, Williams VSL, RTI Health Solutions, Research Triangle Park, NC, USA.

Learning Objectives: To describe a development process that
combines both qualitative and quantitative techniques to maximize the utility of PRO questionnaires. The value of these methods will be demonstrated through the provision of real-life examples.
Who Would Benefit: Anyone who would like to better understand the techniques used in the development and evaluation of PRO questionnaires could benefit from this workshop.
Workshop Description: This workshop is intended to be an
introduction to the development of questionnaires used to assess patient-reported outcomes. In particular, we will describe in detail a process that combines both qualitative and quantitative techniques to optimize the utility and measurement properties of these instruments, as well as their acceptance to regulatory authorities. The use of qualitative techniques to identify constructs of interest and demonstrate content validity will first be described. Next, methods used to evaluate draft items in an effort to maximize patients' understanding of questions and appropriate use of response scales will be discussed. Relevant principles from the survey methods literature will be provided, along with example protocols and results. Finally, the purpose and general principles behind the psychometric techniques most commonly used to evaluate questionnaires (e.g., factor analysis and item response theory) will be presented. In addition, for each psychometric property such as reliability, validity, and responsiveness, definitions, estimation methods, and criteria appropriate for evaluating the utility of outcomes instruments will be provided. Again, real-life examples will be offered. During the remaining minutes of the workshop, the presenters will entertain any questions the audience may have about any of the qualitative or psychometric techniques discussed.

Healthcare Policy Issues
 

WW3	MEDICARE REIMBURSEMENT TRENDS AND THEIR IMPACT ON PRODUCT PLANNING

Simison D, Robinson S, PharmAnalysis Group, Inc, Alexandria, VA, USA

Learning Objectives: The purpose of this workshop is to review current and future Medicare policies and their implication for product planning and launch. Several Medicare policies will be
discussed, including: the importance of form and site of care, the concept of self-administration and its importance, "least costly alternative", national coverage decisions, hospital outpatient prospective payment, and expected changes in the basis of payment for drugs and biologicals.
Who Would Benefit: This session will be appropriate for those responsible for and involved in bringing new drugs and biologicals to market and who wish to identify long-term strategic reimbursement objectives and tactical programs necessary to make their product readily accessible in the market place; academics and consultants who evaluate the cost-effectiveness of pharmaceuticals and devices; and researchers interested in the United States health system and the elderly population. Examples will be provided as case studies, and attendees will leave with a framework for analysis of the potential for Medicare reimbursement of a new product.
Workshop Description: Reimbursement by third-party payers is essential for a pharmaceutical to be successful in the marketplace. Medicare, a key payer for most pharmaceuticals, is legislation-based and therefore a product must fall within the coverage rules and requirements to be reimbursed by this payer. In response to cost pressures, the Medicare system is changing, resulting in increasingly restrictive coverage and reimbursement policies. This workshop will help the attendee understand some of the new and expected Medicare rules, how they might affect access for drugs in development, what the policy and research implications of these rules might be, and what the attendee can do to work within Medicare requirements for reimbursement.
 

Outcomes Research - Practical Application Issues 

WW4	RISK MANAGEMENT PRIMER AND INTRODUCTION TO NEW INFORMATION SOURCES AND TOOLS

Morris LS, IMS Health, Plymouth Meeting, PA, USA

Learning Objectives: The objectives of this workshop are to provide information about the recent history of risk management and to examine new information sources and methodologies to assess the potential problem and impact physician or patient behavior.
Who Would Benefit: Anyone interested in the area of Risk Management
Workshop Description: In this workshop the participants will learn about how the pharmaceutical industry and the Food and Drug Administration (FDA) are responding to mounting pressure to manage and monitor safe use of pharmaceutical products. This portion will provide a brief history of recent activities and concerns that resulted in the increased emphasis in the area of drug safety. There will be discussion on what drug safety, risk management, and marketing research colleagues are doing to address the growing emphasis on Drug Safety and Risk Management. The relationships between evidence-based risk management and marketing principles of segmentation and targeting to impact behavior will be explored. Finally, newly developed information sources, such as longitudinal prescription information, and tools that aid the assessment and management of drug safety risk will be described. A question and answer session will be included. Depending on the size of the group, the workshop leader will facilitate a roundtable discussion of the information sources and new methodologies.

Brixner DI1, Malone DC2, Avey SG3, 1University of Utah, Salt Lake City, UT, USA; 2University of Arizona, Tucson, AZ, USA; 3Foundation of Managed Care Pharmacy, Alexandria, VA, USA

Learning Objectives: To provide a practical versus theoretical approach to the process of decision analysis through hands on application of the techniques to a clinical decision during the workshop.
Who Would Benefit: Individuals involved in making decisions on the allocation of health care resources. This program will be of particular benefit to those utilizing the Academy of Managed Care Pharmacy "Economic Dossier" in evaluating new drug therapy.
Workshop Description: The session will begin with a review of decision analysis techniques by Dr. Dan Malone. He will also review a robust and objective decisional analysis model from the literature. The model presented will be a part of a cost-effectiveness analysis of selective NSAIDs done by the Canadian Office of Health Technology Assessment. This assessment evaluated both rofecoxib and celecoxib against commonly used non-selective NSAIDs. Dr. Brixner will then present the economic dossiers, completed by the pharmaceutical manufacturer, for rofecoxib and celecoxib. In particular, the section on economic modeling will be reviewed in context of the entire report. Audience members will be asked to compare and contrast the techniques and methodology utilized between the Canadian model and those from the manufacturer. A discussion will occur around how the information would be presented to a formulary committee to make a decision on the use of these products in clinical practice. Dr. Avey, Executive Director of the Foundation of Managed Care Pharmacy and participant in the development of the AMCP economic dossier process; will be available as a faculty member to answer questions in a panel discussion that will conclude the workshop.

Brod M1, Swindle R2, 1The Brod Group, Mill Valley, CA, USA; 2Eli Lilly and Company, Indianapolis, IN, USA

Learning Objectives: To understanding the full range of options for identifying and incorporating health outcomes into all stages of the drug development process. Attendees will be provided with a paradigm to learn to identify the most appropriate health outcome for a given drug development goal, how best to incorporate health outcomes at each phase of the drug development process and how to optimize health outcomes to bridge clinical and product positioning objectives.
Who Would Benefit: This workshop is intended for pharmaceutical, biotech and academic researchers as well as marketing professionals who are interested in optimizing the use of health outcomes for scientific, clinical and/or marketing objectives.
Workshop Description: A well thought out health outcomes strategy can generate powerful data that can bridge clinical and commercialization objectives and optimally position a product for multiple purposes from regulatory review to publication in peer reviewed journals. Successfully incorporating health outcomes at each phase of the drug development process requires an understanding of not only the wide range of options for health outcomes but also issues of study design, objectives, drug and disease characteristics and the intended audiences. This workshop will
provide a paradigm for addressing all of the issues required for a successful health outcomes strategy. The wide range of health outcomes, including quality of life, treatment satisfaction, productivity, functioning and economics will be discussed and each of the factors in the paradigm will be examined in detail. The industry perspective on how best to use health outcomes and case studies that will illustrate the advantages of a well thought out health outcomes strategy will be presented.

Ganguly R1, Martin BC1, Dorfman JH1, Rizzo JA2, 1University of Georgia, Athens, GA, USA; 2Cornell University, Ithaca, NY, USA

Learning Objectives: To gain an understanding of confounding and selection bias and be able to compare and utilize two common strategies to address this bias, propensity scoring and Heckman two stage sample selection models.
Who Would Benefit: Health services researchers, Pharmacoeconomists
Workshop Description: Many studies using observational databases attempt to estimate treatment effects by comparing a treated and an unexposed group. In these studies, the treatment is assigned without the benefit of randomization, which frequently results in differences between the two groups on several observed and unobserved confounding covariates. The propensity scoring technique is an approach to reduce selection bias by matching individuals based on their likelihood (propensity) to enter the treatment or comparison group. However, bias due to unobservable confounders may still exist, especially in the context of administrative databases that have limited direct disease measures. The Heckman two-stage estimation technique may potentially address this concern as it controls for selection bias due to observable confounders in the first stage and may also account for potential bias due to unobservable confounders that may continue to exist in the second stage. These two approaches will be illustrated through a case study of our research where we estimate the effect of using multiple vs. single antipsychotic therapy on health care cost and community tenure in schizophrenia patients utilizing propensity scoring and Heckman two-stage sample selection models. The workshop will be structured into four modules: 1) Introduction: Confounding inherent in observational studies and basic approaches 2) Propensity scoring 3) Heckman two-stage sample selection models 4) Comparison of Propensity scoring and Heckman two-stage models including empirical comparisons. Each module will be followed by a 3-5 minute audience question answer session. The presentation will include a theoretical background to each approach, uses in health services research, a reference bank of related articles, a step by step approach to applying the approaches illustrated through data from our research including sample SAS programs.

Bala MV1, Zarkin GA2, Mauskopf JA2, 1Centocor, Inc, Malvern, PA, USA; 2RTI Health Solutions, Research Triangle Park, NC, USA

Learning Objectives: The objective of this workshop will be to discuss how potential developments in the area of pharmacogenomics could affect economic analysis of new therapies
Who Would Benefit: Analysts or decision-makers involved in the conduct or evaluation of pharmacoeconomic studies
Workshop Description: Pharmacogenomics has the potential to identify optimal drug therapy in terms of efficacy and safety for each patient, based on that patient's individual genetic information. However, advances in pharmacogenomics will also pose new modeling challenges for health economists. We present a decision modeling framework for analyzing cost-effectiveness of genetic testing and pharmacogenomics based therapies. In addition to the heterogeneity in the response-toxicity profile that is addressed by pharmacogenomics, we will examine why incorporating heterogeneity in preferences into the decision modeling framework can lead to greater societal welfare. Thus, in addition to a given individual's genetically based likelihood of experiencing beneficial or adverse outcomes from treatment, the model will also incorporate that individual's preferences for these outcomes to identify optimal therapeutic options. We discuss the theoretical and practical issues related to conducting such individualized economic analysis. We conclude with examples of how cost-effectiveness analysis could be conducted in the era of pharmacogenomics.

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