Quality of Life/Patient-Reported Outcomes/Valuation Issues
 

WW9	VALUING EQ-5D HEALTH STATES: METHODS AND RESULTS

de Charro F1, Kind P2, 1Erasmus University, Rotterdam, Netherlands; 2University of York, York, United Kingdom

Learning Objectives: The measurement of health outcomes is a pre-requisite in any evaluative studies.
Who Would Benefit: Workshop attendees are likely to be researchers and analysts concerned with clinical and economic evaluation, particularly where international collaboration is involved.
Workshop Description: For economic analysis, benefits should be expressed in terms of a single, preference-weighted index. Measures of health-related quality of life can play a central role in such analysis. EQ-5D is one such generic measure that yields a single index value for health status based on self-reported problems on each of 5 dimensions - mobility, self care, usual activity, pain/discomfort, anxiety/depression. Although the descriptive system of the EQ-5D is fixed, the issue of the valuation of EQ-5D health states remains more fluid. This workshop summarises the methods and results obtained in a range of valuation studies designed to elicit values for EQ-5D health states. These studies used a variety of methods including ranking, rating, paired comparisons, Time Trade-Off and Standard Gamble. Valuations from surveys of the general population in several countries will be discussed together with those based on the meta-analysis of a large multi-national dataset. Current advice on the selection of valuation sets for use in different application settings will be presented for discussion by workshop participants. At the conclusion of the workshop participants will be familiar with the valuation options available for EQ-5D.
The issues raised in the Workshop are easily capable of being generalised to other health outcome measurement systems, so that participants will benefit from both an examination of the general methodological issues and the specifics as these relate to the valuation of EQ-5D health states.

Brod M1, Skovlund SE2, 1The Brod Group, Mill Valley, CA, USA; 2Novo Nordisk, Bagsvaerd, Copenhagen, Denmark

Learning Objectives: Treatment satisfaction is increasingly used in clinical trials to evaluate patient reported benefits and disadvantages of new pharmaceutical products. This workshop will review the issues surrounding the use of TS as a legitimate PRO, present a conceptual model that should be used to develop TS measures and provide a framework for psychometric validation of TS measures. Participants will gain an understanding of what is TS, whom should be using TS , why TS data are important, when it's appropriate to use and how to appropriately develop TS measures.
Who Would Benefit: This workshop is directed at individuals from clinical, marketing, health outcomes, health care management and academic departments who are interested in incorporating Treatment Satisfaction into the treatment evaluation process and for people who are considering developing new Treatment Satisfaction measures.
Workshop Description: The adequate evaluation of new pharmaceutical products requires inclusion of Patient Reported Outcomes (PRO's). There has been an increasing interest in Treatment Satisfaction (TS) as a legitimate PRO, distinguishable from HRQoL and TS is increasingly being incorporated into clinical trials to evaluate patient reported benefits. However, the assessment of TS is still in its infancy and a conceptual framework is often missing and/or a scientifically sound instrument development process is not undertaken. As a result, TS as a valid PRO, meeting the standards for publication or regulatory consideration, has been limited. This workshop will review conceptual and methodological issues for appropriately assessing TS from the perspective of the PRO researcher and the pharmaceutical industry. Attendees will learn about the conceptual basis of TS, the key steps for developing a TS measure and evaluating its conceptual and psychometric validity, and the issues related to integrating TS in a product development strategy to support product positioning and optimal clinical usage. Case studies will be used, and participants will have the opportunity to identify treatment satisfaction issues for drugs of interest to them.
 

McKenna SP, Doward LC, Galen Research, Manchester, United Kingdom

Learning Objectives: To provide participants with an introduction to a specific approach to QoL assessment; become better able to distinguish between different types of outcome measure.
Who Would Benefit: Health economic practitioners; professionale involved in patient outcome assessment; instrument developers; QoL specialists
Workshop Description: The purpose of the workshop is to provide participants with an introduction to an approach to quality of life (QoL) assessment that has proven highly effective in the development of disease-specific instruments. While the work has been widely published in academic journals, it has not previously been presented to health economic practitioners as a unified body of work. Traditional QoL assessment focuses on impairments (symptoms) and disability (functioning). Several philosophers and outcome researchers have argued that such an approach fails to appreciate those issues that are of greatest concern to patients, as impairments and disability represent influences on QoL rather than the construct itself. The most active area of QoL instrument development adopts a needs-based model that argues that individuals' QoL equates to the extent to which their needs are met. The model allows the development of instruments that provide an unidimensional index of QoL that offers more accurate and sensitive measurement of outcomes in clinical trials. The workshop will cover the theoretical basis of the needs-based model, how instruments are developed and results from studies in which they have been used. Recent developments related to item response theory, item banking, co-calibration of disease-specific measures, comparability of language versions and the generation of disease-specific utilities from QoL responses will also be reported. Participants will be required to analyse questionnaire items in terms of the outcome construct they represent and to consider data related to alternative methods (statistical and preference-based) by which needs-based items are valued.

Tan-Torres Edejer T, Baltussen R, Lauer J, Hutubessy R, World Health Organization, Geneva, Switzerland

Learning Objectives: 1. Know the framework, methodology, tools and major assumptions used in WHO-CHOICE 2. Understand the issues encountered in applying such a methodology as illustrated by an analysis of a set of interventions versus risk factors for cardiovascular disease 3. Appreciate the importance of comparability of cost-effectiveness estimates across a range of interventions and the generalizability of region-specific estimates.
Who Would Benefit: doers (researchers) and users (policymakers) of cost-effectiveness analysis who are interested in producing or using comparable estimates of cost effectiveness ratios
Workshop Description: WHO has developed a standardized set of methods and tools that can be used to analyze the costs and population health impact of current and possible new interventions at the same time. The WHO-CHOICE (CHOosing Interventions that are Cost-Effective) project will provide regularly updated region-specific databases on the costs and effects of a full range of preventive, curative and rehabilitative health interventions. It is expected that policymakers will contextualize this information to their own setting when deciding on the provision and/or financing of single interventions or packages of services. This session will consist of 3 10-minute presentations on 1. Introduction to the framework, methodology and tools of WHO-CHOICE; (including a description of the "null" counterfactual and its use as the base comparator; 2. Presentation of a cost-effectiveness analysis on interventions
versus cardiovascular risk factors (high blood pressure, high clolesterol, physical inactivity, high body mass index and smoking)
3. Summarized graphical presentation of results for over 170 interventions (aside from the CVD risk factors, malnutrition, zinc and vitamin a deficiency, poor water and sanitation, indoor air pollution, unsafe sex) in different regions, highlighting differences and policy implications. The remaining 30 minutes will be spent on an informal debate on the pros and cons of standardizing methodology for cost-effectiveness analysis of different interventions and the feasibility of modelling techniques to produce region-specific results to complement averages from study-specific CEA derived from trials (multi-center or otherwise).

Shaya FT1, Mullins CD1, Wong W2, 1University of Maryland School of Pharmacy, Baltimore, MD, USA; 2CareFirst BlueCross BlueShield, Baltimore, MD, USA

Learning Objectives: This workshop will demonstrate the impact of using risk management methods on projecting utilization and costs in managed care pharmacy. The aim of the workshop is to outline and apply risk-benefit and cost-effectiveness concepts, and assess their implications for modeling and formulary decision-making.
Who Would Benefit: Stakeholders and decision-makers 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, research or academia.
Workshop Description: In addition to providing evidence of a drug's safety and efficacy, pharmaceutical companies increasingly need to demonstrate cost-effectiveness for getting their drug on the formulary. There has been a paradigm shift from efficacy to effectiveness, underscored by a need for collaboration between
the industry, managed care and health care professionals on risk management strategies and tools. Translating clinical trials to real world settings, as exemplified in Phase IV trials, presents a challenge to patients, practitioners, pharmaceutical companies and managed care decision-makers alike. Concurrently, decisions made by Pharmacy and Therapeutics (P&T) committees guide the treatment options available to a managed care population, and affect both the short run and long run costs of managing diseases. There is an increasing interest in modeling risk from efficacy, as well as monitoring disease events, compliance and deriving the economic impact of therapies. With active participation from the audience, a model will be developed for the formulary selection of a therapy from a managed care perspective, with an examination of the budget impact, considering different types of risk management techniques, e.g. the rule of three, restricted marketing, closed formularies, and using risk measures, e.g. ARR, RRR, NNT for case studies in formulary decision-making.

Schmier JK, Halpern MT, Fernandez JE, Exponent, Alexandria,
VA, USA
Learning Objectives: Providing a brief overview of time and motion methodology, identifying situations in which this type of information can inform decision-making, and enabling participants to develop an activity-based costing study.
Who Would Benefit: Researchers and decision-makers who are familiar with the principles of pharmacoeconomics and are interested in using a micro costing approach.
Workshop Description: Pharmacoeconomic studies routinely consider many characteristics in medical technology assessments, including clinical efficacy, acquisition cost, compliance rates, medical resource use, and lost productivity or workdays. The true cost of an intervention should also consider the time required to administer, monitoring, and documentation. With origins in industrial engineering, the principles of time and motion studies can be applied to a health care setting to evaluate the staff and supplies involved in performing an intervention. Activity-based costing, an application of this methodology focused on comprehensively identifying costs, can be used to compare interventions in which economic differences may be otherwise difficult to detect. The workshop will introduce fundamentals of time and motion studies and then concentrate on their application to evaluation in health care. Key areas to be explored include protocol development, including developing a flow chart of the process, designing a sampling schedule that reflects timing and frequency of the activities, developing a case report form in which to abstract time and resource information, and obtaining appropriate institutional review board approval; observation techniques, including videotaping, manual timing, or computer-assisted methods; and analysis, including ensuring that the analysis plan meets the information needs of the target audience. Workshop attendees will develop a study protocol for an existing activity-based costing study and compare their plans with those implemented in the study.

Menzin J1, Harnett J2, 1Boston Health Economics, Inc, Waltham, MA, USA; 2Pfizer, Inc, New York, NY, USA

Learning Objectives: The objectives of this workshop are to describe data requirements, data sources, and steps for developing budgetary impact models for state Medicaid programs, as well as to describe challenges in model development.
Who Would Benefit: Persons involved in the development of budgetary impact models for use in formulary submissions, as well as those responsible for creating economic dossiers for government agencies, would benefit from this workshop.
Workshop Description: Medicaid is a large U.S. government program that provides medical insurance to over 40 million impoverished adults and children across all 50 states. Medicaid receives both state and federal funding, but each program is administered at the state level. In the past several years, prescription drug expenditures have risen rapidly in many states. To address this problem, state programs are increasingly turning to preferred drug lists. As part of this process, agencies are requesting dossiers from pharmaceutical manufacturers describing the clinical and economic benefits of specific therapies using the AMCP format, which includes development of a budget impact model. There are numerous challenges associated with the creation of models to estimate financial impact. Ideally, a separate model would be developed for each state program requesting a budgetary impact assessment. Using administrative claims, each state's model would specify disease prevalence, current treatment patterns, outcomes and costs. However, this may not always be feasible as there are there practical issues related to data access and conducting analyses of massive claims files within a short period of time. In this workshop, we will discuss key issues for the development of budgetary impact models for state Medicaid programs. Special attention will be paid to sources of data and whether data can be generalized across state Medicaid programs. Examples of the problems encountered in model development will be provided in the form of a case study. The ways in which the main challenges were addressed will also be highlighted.
 

Huse D, McGarry LJ, Innovus Research, Inc, Medford, MA, USA

Learning Objectives: To explore the application of health insurance claims data to the development of clinical pathways models that describe the course of treatment and associated costs for particular diseases.
Who Would Benefit: Persons interested in developing models of the clinical pathways followed by patients with particular diseases, especially as relates to the evaluation of new medical technologies.
Workshop Description: The use of health insurance claims databases in support of developmental technologies has generally been limited to cost-of-illness estimation. Information about the overall average costs of illness, however, has limited usefulness for estimating the economic impact of a novel technology. This requires projecting how the technology will alter the clinical course of illness and its treatment, and estimating the costs associated with each possible course, or "clinical pathway," that a patient might follow. While claims data cannot shed much light on the clinical effects of a technology that is not yet marketed, they are well-suited for identifying and costing the relevant clinical pathways. To accomplish this, a clinical pathways model is developed in the form of a decision tree, with separate branches for patients who do or do not experience particular clinical events and/or undergo specific therapeutic or diagnostic interventions. Using health insurance claims data, patients are grouped retrospectively according to the sequence of clinical events reflected in their claims history (i.e., their clinical pathways) and the costs associated with each observed sequence are estimated. The result is a "map" of the most important disease-specific cost drivers that can support simulation of the economic benefits of a novel medical technology. An approach to clinical pathways modeling will be described, using examples from the presenters' experience. Some of the analytic challenges that may be encountered will be discussed. Workshop participants will be encouraged to contribute examples from their own experience for discussion.
 

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