WORKSHOPS
 
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WORKSHOPS

WORKSHOPS – SESSION IMonday, May 18, 2009
10:30 AM - 11:30 AM

CLINICAL OUTCOMES RESEARCH

W1:

INNOVATIONS IN PHYSIOLOGIC AND PATIENT-REPORTED DATA CAPTURE: IMPLICATIONS FOR STREAMLINING DATA COLLECTION AND LEVERAGING ACCESS TO REAL-TIME DATA
Discussion Leaders: Sonya Eremenco MA, ePRO Manager, United BioSource Corporation, Health Care Analytics, Bethesda, MD, USA; Wilhelm Muehlhausen DVM, Senior Product Manager ePRO, Cardinal Health Germany 234 GmbH, Research Services, Hoechberg, Germany; Lionel Tarassenko FREng, Professor of Electrical Engineering, University of Oxford, Institute of Biomedical Engineering, Oxford, UK; Jill V. Platko PhD, Scientific Advisor, PHT Corporation, Charlestown, MA, USA
PURPOSE: In many therapeutic areas, clinical trial endpoints involve patient collection of physiologic and patient-reported outcome (PRO) data. Technological advances in biometric devices and wireless data transmission have resulted in the ability to collect and transmit these data remotely, from wherever the patient may be. In this workshop, we address the opportunities and challenges of parallel biometric-PRO data collection for gathering empirical evidence of product value through clinical studies conducted pre and post-launch and ways to leverage real-time data access.
DESCRIPTION: Patients with chronic conditions such as asthma and diabetes monitor their conditions regularly using peak flow meters and glucometers. Until recently, these data were collected from patients separately from PRO data during clinical trials, increasing respondent burden and the risk of error associated with patients or sites transposing and manually entering data. With the use of electronic PRO administration (ePRO), development of biometric devices, evolution of data transmission technology, and greater technologic sophistication of consumers, comes an opportunity for parallel electronic data capture, simultaneously capturing and transmitting physiologic and PRO parameters in clinical studies. In this workshop, we discuss using ePRO and biometric technology for parallel data capture emphasizing advantages, disadvantages, execution, and ways to leverage these data. Asthma and diabetes are used as case examples of conditions for which parallel data collection is particularly relevant. The workshop will begin with an overview of these conditions and current EDC technologies, including a single integrated device and separate biometric devices capable of integrating with PDAs or mobile phones. Important practical considerations for implementation in clinical trials including training and compliance will be addressed. The potential for using real-time parallel data for adverse event safety monitoring will also be discussed. Participants will participate in developing a checklist for identifying situations and populations where parallel data collection has greatest value for safety and efficacy.

 

ECONOMIC OUTCOMES RESEARCH

W2:

TRANSFERABILITY OF ECONOMIC EVALUATIONS FROM SETTING TO SETTING WITHIN THE UNITED STATES
Discussion Leaders: Michael F. Drummond DPhil, Professor of Health Economics, Centre for Health Economics, University of York, York, UK; Amy K O'Sullivan PhD, Associate Director, i3 Innovus, Medford, MA, USA; David W. Lee PhD, Senior Director, GE Healthcare, Health Economics and Outcomes Research, Waukesha, WI, USA
PURPOSE: To explore issues of transferability when applied within countries, rather than between countries. Specifically, the workshop will examine the extent to which transferability issues are relevant for economic evaluations conducted in the US for use by different payers across the country.
DESCRIPTION: An ISPOR Good Research Practices Task Force has recently issued guidance on how to address issues of transferability of economic evaluations from one jurisdiction to another. However, the literature on transferability—including the ISPOR Task Force Report—tends to focus on transferability across countries, rather than within countries. In a large country like the US—with its fragmented health care system and multiple payers—transferability issues could be quite important, in that an economic evaluation performed in one region or from one payer's perspective may not be relevant to others'. This has become a key issue in the current comparative effectiveness debate in the US, in which it has been argued that issues of cost and cost-effectiveness should not be included on the comparative effectiveness agenda, as they are better addressed at the local level. This workshop will explore transferability issues that arise when conducting economic evaluations in the US, and whether they can be addressed by the methods proposed by the ISPOR Task Force. An interactive cost-effectiveness case study will be presented and examined from the perspective of at least two US payers. The flow chart published in the ISPOR Task Force Report will be applied and workshop participants will be encouraged to assess whether different US payers could use the cost-effectiveness analysis without major adaptation, or whether additional modeling is required. If the latter is the case, the major threats to transferability will be identified. Finally, the implications of these issues for the current US policy debate on comparative effectiveness will be explored.

PATIENT-REPORTED OUTCOMES/PREFERENCE-BASED RESEARCH

W3: REVISITING THE ESTIMATION OF QALYS
Discussion Leaders: Benjamin M. Craig PhD, Assistant Faculty Member, Health Outcomes and Behavior, Moffitt Cancer Center and Courtesy Associate Professor of Economics, Univ. of South Florida, Moffitt Cancer Center, Health Outcomes and Behavior, Tampa, FL, USA; Jan J. Busschbach PhD, Senior Research Associate, Erasmus Medical Center, Department of Medical Psychology and Psychotherapy, Rotterdam, The Netherlands; Mark Oppe MSc, Researcher, Institute for Medical Technology Assessment, Erasmus MC, Rotterdam, The Netherlands
PURPOSE: To present recent developments in health valuation and discuss the release of adjusted country-specific value sets for EQ-5D states. The workshop will be centered on the implications for cost-utility analyses and related health policy.
DESCRIPTION: Recent evidence suggests the QALY values assigned to EQ-5D health states have been severely underestimated (e.g., by 0.21 QALYs in the UK, and by 0.105 QALYs in the United States). Proposed adjustments are nonlinear, substantial, and meant to correct the arbitrary transformations of worse than death responses. This workshop will present the events leading to these developments, and describe why all currently available QALY estimates may need to be revisited. This development has broad implications, affecting QALY estimates based on time trade-off, standard gamble and person trade-off techniques as well as the valuation of other descriptive systems (e.g., SF-6D). After an introductory review, participants will be invited to comment and discuss the valuation methods, proposed adjustments, and their implications. This workshop will be of interest to ISPOR meeting attendees interested in QALYs, cost-utility analyses, or their use in policy. The workshop will focus on QALY estimation and its implications for setting health policy, not on the construct of a QALY or the position of the EuroQol Group.

USE OF REAL WORLD DATA

W4:

ASSESSING HETEROGENEITY OF TREATMENT EFFECTS
Discussion Leaders: Bijan J. Borah PhD, Senior Researcher, i3 Innovus, Eden Prairie, MN, USA; Nilay D. Shah PhD, Assistant Professor, Mayo Clinic, Division of Health Care Policy and Research, Rochester, MN, USA; Henry Joe Henk PhD, Director, i3 Innovus, Health Economics & Outcomes, Eden Prairie, MN, USA
PURPOSE: The purpose of the workshop is to demonstrate how quantile treatment effects (QTE) can be computed using real world data.
DESCRIPTION: In understanding the impact of a treatment (regimen) on an outcome of interest, researchers often measure average treatment effect (ATE) or average treatment effect on the treated (ATET). However, it might often be of significant import to understand the distributional impacts of the treatment over the entire outcome distribution. For example, the policy maker might be interested in understanding how a specific treatment impacts patients in the lower and upper tail of the outcome distribution. The average treatment effect measures (ATE/ATET) cannot provide such information as they often obscure the heterogeneity of treatment effects along the outcome distribution. QTEs measure the heterogeneous impacts of a treatment on outcome distribution. As an example, in assessing the effect of a treatment (e.g., drug A vs. drug B) on health care costs, ATE measures the mean difference in costs of treatment A vs. treatment B. However, ATE does not reveal the distributional impacts of the treatment on health care cost. QTE, which can be measured for any desired quantile of the outcome distribution, may shed light on whether treatment effect is heterogeneous across the cost distribution. From a managed cared perspective, the understanding of such distributional impacts of a treatment on health care costs might provide valuable information on cost reduction strategies for specific segments of the patient population. We will discuss the pros and cons modeling mean effect vs. the distributional effect. Furthermore, we will also discuss alternative ways of looking at the distributional effects (beyond QTE). The format of the workshop presentation will be interactive so as to encourage optimal audience participation. Both canned and other available software will be discussed that may be used to compute QTE.

 
W5:

KEEPING VULNERABLE POPULATIONS SAFE FROM PERTUSSIS: USING MODELING TOOLS TO IDENTIFY COST-EFFECTIVE INTERVENTIONS FOR WHOOPING COUGH
Discussion Leaders: Amy L. Greer MSc, PhD, Research Fellow, Research Institute of the Hospital for Sick Children, Child Health Evaluative Sciences, Toronto, ON, Canada; David F. Fisman MD, FRCPC, MPH, Medical Epidemiologist and Scientist, Ontario Agency for Health Protection and Promotion and The Hospital for Sick Children, Child Health Evaluative Sciences, Toronto, ON, Canada; Marija Zivkovic Gojovic MSc, PhD Candidate, York University, Centre for Disease Modeling, Toronto, ON, Canada; Laurent Coudeville PhD, Health Economics Deputy Director, Sanofi Pasteur, Lyon, France
PURPOSE: This workshop will introduce modeling techniques that can be used to examine the effectiveness and cost-effectiveness of vaccination strategies for the control of pertussis in North America and discuss how these results can be used to inform healthcare policy. DESCRIPTION: In the decades after pertussis vaccine introduction, morbidity and mortality from pertussis fell sharply. More recently, pertussis incidence has been increasing throughout much of North America. This resurgence is likely multifactorial but may result principally from changed transmission cycles. The resurgence of pertussis is an important public health challenge and requires novel and innovative approaches for developing vaccine policy. Mathematical models of disease transmission can provide insights into the biology of pertussis transmission and a framework for projecting the effectiveness of disease control interventions when real-world experiments are not possible. This is now more crucial because we have a better understanding of the duration of immunity and availability of acellular pertussis vaccines. The first half of this workshop will focus on introducing participants to different disease transmission models (e.g. age-structured compartment models and individual based models) that can be used to identify intervention strategies of public health interest. Discussion leaders will highlight the importance of appropriate model choice and public health context. Next, we will demonstrate how model outputs can be used to develop simple decision analysis trees to conduct health economic analyses for different scenarios. This portion of the workshop will highlight our ability to compare the cost-effectiveness of vaccination scenarios with other health interventions. In the final section, participants will be asked to reflect on the utility of combining transmission models with health economic analyses to address complex infectious disease problems using pertussis as a case-study as well as identifying other key research areas where a similar approach may be useful for moving health outcomes research forward.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W6:

FEASIBILITY OF INDIRECT COMPARISON ANALYSIS TO SUPPORT REIMBURSEMENT DECISIONS
Discussion Leaders: Cornelis Boersma MSc, PhD Student, and Director, University of Groningen, Department of Pharmacy, Unit of PharmaEpidemiology & PharmacoEconomics (PE2)/HECTA B.V, Groningen, The Netherlands; Lieven Annemans PhD, MSc, Professor of Health Economics, University of Ghent, Brussels University, Ghent, Belgium; Maarten J. Postma PhD, Professor Pharmacoeconomics, University of Groningen, Department of Pharmacy, Unit of PharmacoEpidemiology & PharmacoEconomics (PE2), Groningen, The Netherlands
PURPOSE: To evaluate the strengths and weaknesses of applying indirect comparison methodology to support reimbursement decisions.
DESCRIPTION: Decision making procedures for new drugs rely on guidelines, preferences and require evidence-based clinical and health-economic outcomes. As is often the case with new drugs, and especially innovative drugs for very specific (small) patient populations, information on comparative effectiveness, safety and cost-effectiveness is scarce. Lack of ‘head-to-head' trials or difficulties with assigning an appropriate comparator treatment is often the case. Indirect (fixed or mixed treatment) comparison analysis gives the opportunity to synthesize all available relevant information for decision-analytical economic models based on data from a formal meta-analysis. Here, the objective is to evaluate the strengths and weaknesses of applying indirect comparison analysis to support decisions on reimbursements. Antifungal agents in the treatment of severe invasive fungal infections serve as a useful example for evaluating the role of indirect comparison analyses in the decision making procedure as ‘head-to-head' trials are often limited for these drugs. Existing literature was extensively reviewed to include all relevant randomized clinical trials in which antifungal agents are compared for treatment of severe invasive fungal infections. In general, clinical trials are often designed for registration purposes and do therefore not include all relevant comparators or the registered first choice treatment. For those situations, the potentials and limitations of conducting economic evaluations based on results from indirect comparison analysis will be discussed thoroughly based on the existing comparisons, and based on the strengths and weaknesses of different approaches, both frequentist and Bayesian methods. Recommendations will be given on the interpretation of economic outcomes results based on using effectiveness data derived with indirect comparison analysis, in the light of a potential supporting role in health-care decision making procedures concerning drug reimbursement decisions.

W7:

ADHERING TO MULTIPLE GUIDELINES FOR PREPARATION OF FORMULARY SUBMISSION DOSSIERS: ALIGNING WITH PAYER EXPECTATIONS
Discussion Leaders: Carol Gaich PharmD, RPh, HTA-Outcomes Liaison, Eli Lilly and Company, Indianapolis, IN, USA; Lynn Nishida RPh, Director, Clinical Pharmacy Services, The Regence Group/RegenceRx, Portland, OR, USA; Brian Sweet BSPharm, MBA, Chief Clinical Pharmacy Officer, WellPoint, Inc, Grand Island, NY, USA; Melissa Juniper MS, Director, Regulatory and Health Outcomes Strategy, RTI Health Solutions, Research Triangle Park, NC, USA
PURPOSE: The aims of this workshop are (1) to provide an overview of the Academy of Managed Care Pharmacy (AMCP) Format for Formulary Submissions (version 2.1, April 2005), the WellPoint Health Technology Assessment (HTA) Guidelines (revised October 2008), and the RegenceRx Medication Value Appraisal Principles (revised September 2007); (2) to discuss the goals met by these guidelines from payers' perspectives; and (3) to discuss lessons learned from the industry and consultant agency perspectives in coordinating the preparation of dossiers to meet the needs of diverse health plans and payers.
DESCRIPTION: The AMCP Format is the primary guideline used by manufacturers to prepare submissions for US payers. Both WellPoint and RegenceRx require more comprehensive information for formulary submissions. To achieve broad formulary placement, manufacturers planning to market a product in the US may want to understand how to meet AMCP dossier standards while addressing the needs of large health plan payers. Manufacturers also need to manage the logistical challenges of dossier preparation, as it is resource-intensive and requires integration of disease burden and product labeling information, as well as comprehensive clinical and economic evidence—all ideally by product launch. This workshop will begin by providing a broad overview of the AMCP Format, the WellPoint HTA Guidelines, and the RegenceRx Medication Value Appraisal Principles. Representatives from WellPoint and RegenceRx will then discuss the rationale for requesting the information from manufacturers noted in the guidelines, as well as how this information is ultimately used. Representatives from industry and from a consultant agency also will discuss lessons learned from coordinating the preparation of dossiers to meet the standards of multiple formulary submission guidelines. Other topics, such as the appropriate organization, depth, and breadth of information provided in particular sections from the perspectives of the discussion leaders also will be discussed.

 
WORKSHOPS – SESSION IITuesday, May 19, 2009
4:00 PM - 5:00 PM

CLINICAL OUTCOMES RESEARCH

W8: COMPARATIVE EFFECTIVENESS WITHOUT HEAD-TO-HEAD TRIALS: INDIRECT COMPARISONS USING PRIMARY AND PUBLISHED CLINICAL TRIAL DATA
Discussion Leaders: James Signorovitch PhD, Associate, Analysis Group, Inc, Boston, MA, USA; Andrew P. Yu PhD, Manager, Analysis Group, Inc, Boston, MA, USA; Eric Wu PhD, Vice President, Analysis Group, Inc, Boston, MA, USA
PURPOSE: Comparative effectiveness research often requires the comparison of alternative interventions without the benefit of a head-to-head randomized trial. For example, we may wish to compare drug A vs. drug B when randomized trials have only compared each drug to placebo. The Purpose: of this workshop is to review existing statistical methods for indirect comparisons and discuss limitations and gaps in the existing methodology. A new method that fills a key gap will be described and illustrated through an example application.
DESCRIPTION:
Existing statistical methods for indirect comparison include adjusted indirect comparison, mixed treatment comparison (MTC) meta-analyses and meta-regression. Published applications of these methods will be presented and the audience will be invited to discuss the assumptions and limitations underlying each method. The goal will be to identify evidence needs of health care decision makers that cannot be met with existing methods. For example, existing methods generally require data from large numbers of clinical trials. In many important settings however, such as for rare diseases or when new therapies enter the market, there may be too few trials to apply existing methods. The need for assessing comparative effectiveness in these settings can be met by leveraging patient-level clinical trial data. In particular, we describe a new method for matching patients across trials to compare drug A vs. drug B when patient-level data are available from one or more randomized trial of drug A vs. placebo but only published summary results are available from trials of drug B vs. placebo. The new method is illustrated through an indirect comparison of therapies for psoriasis. Workshop participants will be encouraged to evaluate the new methodology and the example application in relation to existing methods.
W9:

ANALYSIS OF EFFECTIVENESS AND COST-EFFECTIVENESS IN PATIENT REGISTRIES
Discussion Leaders*: Maria Malmenäs MSc, Manager Biostatistics, Outcome Surveys, Shire Human Genetic Therapies, Global Medical Affairs, Danderyd, Sweden; Margaret Hux MSc, Lead Analyst, i3 Innovus, Burlington, ON, Canada; Mike Novotny MBA, MA, CEO, Medrio, San Francisco, CA, USA; Lusine Abrahamyan MD, MPH, PhD(c), Graduate Student, University of Toronto, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada
PURPOSE: Observational registries of specific patient groups offer an attractive opportunity to evaluate real-world effectiveness and cost-effectiveness of treatments. Good research practices for data analysis and interpretation will increase our ability to conduct comparative research within patient registries, and this workshop will provide practical guidance on suitable statistical approaches. Researchers currently involved with or interested in the use of registry data for comparison of treatments will want to attend.
DESCRIPTION: Using registry data for comparative analyses of effectiveness or cost-effectiveness is methodologically challenging since these data are lacking the safeguards for comparison included in clinical trials. A key element in comparing treatment effectiveness is assessment for selection bias that is possible in the absence of randomization and for ascertainment and measurement biases that are possible in the absence of standardized assessment procedures. Methods to address these biases include covariate analysis, matching, propensity scoring, and use of instrumental variables. Cost-effectiveness analysis also involves comparing total disease-related cost between treatments. It is important to assess and adjust for potential selection and other biases on both effectiveness and cost. All relevant costs should be included, and methods to address missing data or assumptions to evaluate cost over an equal time in the presence of variable follow-up should be clinically sound and transparent. Selection of a fixed time frame for evaluation must balance the need to impute missing data with having sufficient time for consequences of treatment effectiveness and safety to occur and be measured. Choice of imputation methods will be addressed. Lastly, since many registries are conducted multi-nationally, the cultural and country differences in real-world treatment practice, funding and availability must be considered. Clinical examples from published and unpublished projects will be used for illustration. Audience participation will be ensured through a group exercise and opportunity for question and answer.
* Discussion Leaders represent the ISPOR Patient Registry Design & Operations Working Group’s Data Management & Analysis Project Team. A list of members is available on the ISPOR website.

ECONOMIC OUTCOMES RESEARCH

W10:

UNCERTAINTY AROUND AN EFFICIENCY FRONTIER
Discussion Leaders: J. Jaime Caro MDCM, FRCPC, FAC, Senior Vice President Health Economics, United BioSource Corporation, Health Care Analytics, Lexington, MA, USA; Isao Kamae MD, DrPH, Professor, Keio University Graduate School of Health Management, Graduate School of Health Management, Fujisawa, Kanagawa, Japan; Charalabos-Markos Dintsios MA, MPH, Research Fellow, Institute for Quality and Efficiency in Health Care (IQWiG), Pharmaceutical Products Evaluation, Cologne, Germany
PURPOSE: The new German methods for economic evaluation use the efficiency frontier to inform decision makers about the relative efficiency of interventions in a particular therapeutic area. Projection of the frontier provides guidance for decisions about newer interventions lying above and to the right of the frontier. It is important for decision makers to also understand the uncertainty surrounding the frontier and its projection. In this workshop, we will address various approaches to quantifying this uncertainty.
DESCRIPTION: There are many sources of uncertainty in the efficiency frontier, both for the interventions' benefits and their cost. Moreover, these uncertainties correlate in various ways for each intervention and across interventions. Proper depiction of the efficiency frontier requires incorporating this uncertainty. One approach to this involves parametric estimates of the uncertainty. Another uses a form of probabilistic sensitivity analysis to draw contour lines around the frontier. Special techniques are then required both for depicting the frontier contours and for projection in a consistent way. In the workshop, we will describe and demonstrate these techniques using various case studies. Given the topic's developmental nature, strong discussion will be fostered and the presenters look forward to a vigorous and illuminating debate on what should be done. The decision by IQWiG to implement the efficiency frontier in Germany as the method of choice for economic evaluations means these methodological discussions will have a strong impact on pharmacoeconomics in general.

PATIENT-REPORTED OUTCOMES/PREFERENCE-BASED RESEARCH

W11:

STILL WRESTLING WITH SPECIFYING THE CONCEPTUAL FRAMEWORK OF A PRO INSTRUMENT? TWO TOOLS TO HELP YOU PIN IT DOWN: THE PRO CONCEPT TAXONOMY AND THE PRO INSTRUMENT HIERARCHY
Discussion Leaders: Pennifer Erickson PhD, Co-founder, OLGA, State College, PA, USA; Richard J. Willke PhD, Senior Director/Group Leader, Pfizer, Outcomes Research, Peapack, NJ, USA
PURPOSE: The 2006 FDA draft guidance on patient reported outcomes (PRO) measures states that one of the first steps in the drug approval process is to specify how scores representing concepts measured by an instrument are derived from its items and domains, i.e., to specify the instrument's conceptual framework. Two tools, the PRO Concept Taxonomy and PRO Instrument Hierarchy, have been developed to assist in depicting the conceptual framework and to link this to the intended labeling. This workshop: describes each tool; explains their relationships to existing classification systems and measurement methods; and, illustrates their use based on recently approved FDA labeling.
DESCRIPTION: The PRO Concept Taxonomy depicts an instrument's concepts from lower to higher levels within one or more families. Adding the measurement structure to this diagram indicates how scores represent concepts to be included in the labeling statement. The WHO International Classification of Functioning, Disability and Health (ICF) will be used to motivate the discussion of the taxonomy. The PRO Instrument Hierarchy takes information supplied by the taxonomy and links it to a statement of treatment benefit. In this way, the hierarchy provides an objective tool for determining the fit between an instrument and the proposed labeling. Following this discussion, participants will be presented with case studies based on existing labels and asked to use the taxonomy and hierarchy to develop conceptual frameworks for selected PROs and to evaluate each instrument's potential for supporting the intended claim.

USE OF REAL WORLD DATA

W12:

A TAXONOMY FOR THE DESIGN, DEVELOPMENT AND IMPLEMENTATION OF PATIENT REGISTRIES
Discussion Leaders: Eric K. Gemmen MA, Senior Director, Medical Affairs, Epidemiology & Outcomes Research, Quintiles, Inc, Late Phase & Safety Services, Falls Church, VA, USA; Claudio Faria PharmD, MPH, Associate Director of Clincal Research, University of Massachusetts Medical School, Boston, MA, USA
PURPOSE: The objective of this workshop is to share the culmination of more than one year's work by the ISPOR Patient Registry Classification, Strategy & Design Working Group, Team 2: Design, Development & Implementation, in the characterization, classification, strategy, design, and application of patient registries and the data they generate in health care decisions.
DESCRIPTION:
Patient registries are an important source of data that can aid in the development and evaluation of access and reimbursement strategies. However, registries are not widely understood, and there is not a consensus on their definition, particularly with respect to observational studies, in general. While many health outcomes researchers may become involved in the analysis and interpretation of registry data, fewer are active in the design of registries and in the ongoing management and capture of their data. We will discuss how approaches to the design and conduct can be applied to generate critical information for regulatory and payor requirements, and we will highlight varying perspectives on registries from regulatory authorities in different parts of the world. Key elements of our taxonomy and classification include registry Purpose:, funding and oversight, stakeholders, scope, privacy, regulatory considerations, exposure, feasibility, informed consent, IRB/ethics approval, target population, design characteristics, data elements, data collection materials & methods, registry size and duration, active/passive surveillance, site identification and initiation, regulatory documents, site support and monitoring, database build, data capture & management, mid-study changes and protocol amendments, query resolution, loss to follow-up, source document verification (SDV), data lock, and site close-out. This workshop will interest health outcomes researchers who develop protocols and project plans for registries and those who are responsible for the management of these registries. Participants are encouraged to bring forward their registry design and conduct challenges in the interactive discussion section of the workshop.
* Discussion Leaders represent the ISPOR Patient Registry Special Interest Group, Classification, Strategy & Design Working Group’s Design, Development & Implementation Project Team. A list of members is available on the ISPOR website.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W13: FROM EVIDENCE TO ACCESS: A MORE TRANSPARENT FORMULARY DECISION-MAKING PROCESS
Discussion Leaders: Robert Dubois MD, PhD, Chief Medical Officer, Cerner LifeSciences, Beverly Hills, CA, USA; Sean D. Sullivan PhD, RPh, Professor of Pharmacy and Public Health and Director, University of Washington, Pharmaceutical Outcomes Research and Policy Program, Seattle, WA, USA; Mark Helfand MD, MPH, Director, Oregon Evidence-based Practice Center, Professor of Medicine and Medical Informatics & Clinical Epidemiology, Oregon Health & Science University, Portland, OR, USA; Barbara McNeil MD, PhD, Ridley Watts Professor and Head of Department, Harvard Medical School, Department of Health Care Policy, Boston, MA, USA
PURPOSE: To present a new more transparent approach to formulary decision-making.
DESCRIPTION:
The pharmaceutical industry is improving its ability to provide relevant evidence to health plans in a more transparent manner. Unfortunately, little transparency exists, concordance is low (Anis, Medical Care 2001), and important factors are not explicit regarding the Plans' linkage between such evidence and formulary access decisions. We present a new approach that identifies key decision elements and their importance. Hypothetical scenarios were created based upon permutations of: drug impact (magnitude of survival or QOL benefit vs. current therapy), presence/absence of safety concerns among prior drugs, availability of alternatives, and relative cost. A panel of payers and evidence based medicine experts rated each scenario's effectiveness endpoint (very low to very high importance) and the level of drug “access” (very limited to very open) on 9-point scales under specified availability of safety (low-, medium-, or high evidence certainty) and effectiveness data (low-, medium-, or high evidence certainty). Variability of ratings was observed and was related, in part, to the scenario (median scores ranged from 3-8), certainty of the effectiveness and safety evidence (“low certainty”-median score = 1, “high certainty”-median score = 7), endpoint importance (medians ranged from 1-8), and panelist (medians ranged from 2-5.) This workshop will (1) present the approach, (2) review the influential factors, and (3) enable attendees to rate the ease of drug access for various scenarios initially without this approach (current practice) and later with it. If validated and adopted, this more granular approach could increase formulary policy transparency and make clear to consumers, employers, manufacturers, and the P&T Committee members themselves how decisions are made as well as the relevant factors underlying them.
 
W14:

VALUE BASE PRICING, WHAT IS IT AND IS IT RELEVANT FOR THE US?
Discussion Leaders: Alistair McGuire PhD, Chair in Health Economics, LSE Health and Social Care, London, UK; Monique Martin MSc, MBA, Vice-President UK Operations, i3 Innovus, Uxbridge, Middlesex, UK; Stephen Clark BA, Vice President, Market Access and Reimbursement, i3 Innovus, Eden Prairie, MN, USA
PURPOSE: To present value-based pricing and discuss the relevance to the US.
DESCRIPTION:
Drug reimbursement is a topical subject both in the USA and Europe. The workshop would begin with an overview of the approaches to drug regulation in countries in the developed world. This would be followed by a discussion of the concept of value-based pricing (VBP) which is held by some as a means of delivering both static and dynamic efficiency. As well as discussing the various definitions and criticisms of VBP, including differentiating between ex-ante and ex-post VBP, this approach will be contrasted with other suggested pricing approaches. In particular it is suggested by advocates that VBP can draw upon HTA agencies through the use of economic evaluation; the relationship of VBP to drug evaluation and regulation policies in Europe, specifically NICE (UK) and IQWiG (Germany), will be outlined. Examples will be used to illustrate how VBP, if explicitly introduced as a means of reimbursing pharmaceuticals, might operate in practice. Supporters of VBP also emphasize the ability of this instrument to introduce price differentiation into markets; the efficiency and equity implications of this argument will be addressed. While VBP is most commonly mooted in Europe, and used in Sweden as a basis for reimbursement, the workshop will also discuss the possible implications for the US market if VBP were to be used instead of the currently used approach of differential pricing for different purchasers. The workshop will encourage audience participation through exploring the audience’s views on, definitions of and reactions to VBP. The audience will be asked at the start of the workshop to outline their own definition of an optimal pricing scheme for pharmaceutical reimbursement.

   
WORKSHOPS SESSION IIIWednesday, May 20, 2009
8:30 AM - 9:30 AM

Clinical Outcomes Research

W15:

UPDATING SYSTEMATIC REVIEWS - THEIR IMPORTANCE AND TECHNIQUES FOR UPDATING
Discussion Leaders: Alexander Tsertsvadze MD, MSc, Clinical Epidemiology Program, Ottawa Health Research Institute, Ottawa, ON, Canada; Samuel Stoddart PhD, Systematic Review Technical Lead, Heron Evidence Development Ltd, Luton, UK; Melanie Plested BSc, Senior Health Outcomes Analyst, Heron Evidence Development Ltd, Luton, UK
PURPOSE: Outline and discuss the importance of updating systematic reviews. Methodological challenges faced in the process and practical solutions will be covered. Outline the techniques available to assess the need for an update.
DESCRIPTION: Governments and other groups invest heavily in commissioning and using the results of systematic reviews since they are a form of convenient synthesis of evidence for a health care professional. Clinical practice guidelines and health care policies may also be based on results from a systematic review. This reinforces the need for a review to remain up-to-date, so that health care decisions and policy making are not based on outdated and invalid review evidence. Methodological problems are often encountered when updating a review. Apart from the practical difficulties of handling newly discovered literature in tandem with literature retrieved in a previous review, several scientific obstacles may also arise: disease definitions change, new outcome tools are developed, new interventions and dosing are utilised. The practical solutions to these issues will be discussed. The Cochrane collaboration recommends updating a review every two years. This conflict with a previously conducted survival analysis which suggests the average review lifetime is 5.5 years. Interestingly, the percentage of reviews which are updated within two years of their publication is quite low, especially considering recent estimates state that approximately 2500 new systematic reviews are published annually. The survival time of a systematic review will be examined more closely, and techniques to determine the need to update a review will be outlined. A range of factors influencing an ‘appropriate' timing for updating will also be considered. We will close with a discussion of these results, the potential reasons behind the low number of review updates, the degree to which this may be a problem, and what steps might be necessary to address this.

ECONOMIC OUTCOMES RESEARCH

W16:

EARLY PHASE MODELING TO SUPPORT GO/NO GO DECISIONS: INCORPORATING TARGET PRODUCT PROFILES, PRICING VARIABILITY AND DIAGNOSTICS TO FACILITATE INTERNAL DECISION MAKING
Discussion Leaders: Louis P. Garrison PhD, Professor, Pharmaceutical Outcomes Research & Policy Program Department of Pharmacy Health Sciences, University of Washington, Seattle, WA, USA; Jonathan D. Campbell PhD, Senior Fellow, University of Washington, Pharmaceutical Outcomes Research and Policy Program, Seattle, WA, USA; Josh J. Carlson PhD, MPH, Senior Fellow, University of Washington, Pharmaceutical Outcomes Research and Policy Program, Seattle, WA, USA; Tripthi Kamath PhD, Senior Health Economist, Genentech, Inc, South San Francisco, CA, USA
PURPOSE: The purpose of this workshop is to discuss the process, application, communication, and challenges of early phase pharmacoeconomic modeling. We will highlight issues related to design and incorporation of the target product profile (TPP) under conditions of uncertainty, implications of diagnostic co-development, and a format for and dissemination of the findings to internal audiences.
DESCRIPTION: In today's competitive economic environment, well informed strategic decisions related to pre-launch product advancement are critical to a company's success. Early phase pharmacoeconomics modeling can facilitate the efficient allocation of development resources, inform future pricing considerations, and identify key barriers and drivers related to reimbursement. Our workshop will explore a process for modeling the potential incremental value of early phase compounds as compared to the next best therapeutic alternatives. We will emphasize the design and implementation of the TPP under uncertainty, the implications of diagnostic co-development, and communicating the findings to internal company stakeholders. Specifically, Professor Garrison will provide an overview of the Purpose: of early phase modeling including the importance of a structured process and dissemination format. Dr. Campbell will detail the development of a fit-to-Purpose: early phase model and the design and incorporation of the TPP and its competitors using a case example of a rheumatoid arthritis biologic. Dr. Carlson will explore the incorporation of diagnostics using the same case example and will highlight data needs, assumptions for diagnostic TPPs, and interpretation of results. Dr. Kamath will provide an overview of how and to what end the findings are communicated to and used by internal industry stakeholders. The discussion to follow will encourage the audience to share their experiences with early phase modeling, the optimal format and dissemination of the findings, and the challenges and methods for exploring uncertainty in this context.

PATIENT-REPORTED OUTCOMES/PREFERENCE-BASED RESEARCH

W17: COPING WITH THE VALIDATION REQUIREMENTS OF THE FDA PRO GUIDANCE: THE INTERSECTION OF SCIENCE AND PRACTICE
Discussion Leaders: William Lenderking PhD, Senior Research Leader, United BioSource Corporation, Bethesda, MD, USA; Elizabeth Merikle PhD, Research Scientist, United BioSource Corporation, Montreal, QC, Canada; Michelle Stewart PhD, Director, Pfizer, Inc, New London, CT, USA
PURPOSE: Educate participants about FDA PRO draft guidance and help them generate practical solutions to problems they may face in implementing them. Participants will leave the workshop with 2-3 ideas they can use in the day-to-day application of the PRO guidance. DESCRIPTION: This workshop will take off from the draft PRO guidance and consider the practical implications and scientific evidence supporting certain aspects of the guidance. In particular, we will focus on validation of patient reported outcome measures in light of the guidance on conceptual frameworks, conceptual models, and endpoint models. We will also consider the experience of sponsors with submissions to the FDA since the guidance was released. Any updates to the FDA PRO guidance will be incorporated into the workshop. Practical, real-world examples/ research dilemmas will be presented for discussion by participants and we will consider alternative solutions, as well as discussing the solution ultimately chosen. Questions to be considered might include the following: What does the literature say about validation requirements for a scale that has been in use for decades but has obvious limitations? What constitutes sufficient validation? If a scale has been validated in a given study population, what are the validation requirements for applying it to a new population, according to both the FDA and the literature? What if your research team wants to administer a questionnaire on a weekly basis, and it has only been previously developed for monthly administration? Are there different aspects of health and functioning which might be optimally measured over different recall periods? Can measures with different recall periods be compared within the same study? Does it make sense to assess functioning on a daily basis, or should daily measurement be more appropriately reserved for symptoms?

USE OF REAL WORLD DATA

W18:

HOW TO IMPROVE THE QUALITY OF MEDICATION ADHERENCE ESTIMATES DERIVED FROM HEALTHCARE CLAIMS OR ELECTRONIC MEDICAL RECORDS DATABASES
Discussion Leaders: Peter Sun MD, PhD, Chief Health Economist, Kailo Research Group, Indianapolis, IN, USA; Yang Zhao PhD, Sr. Health Outcomes Scientist, Eli Lilly and Company, Indianapolis, IN, USA; Saurabh Ray PhD, Associate Director, Abbott Laboratories, Global Health Economics & Outcomes Research, Abbott Park, IL, USA
PURPOSE: 1. Explore common issues that lead to widespread inconsistency of medication adherence estimates derived from healthcare claims or electronic medical records databases. 2. Discuss and demonstrate various analytical tools that can be used to solve the issues therefore, improve the quality of medication adherence estimates derived from healthcare claims or electronic medical records databases. 3. Enable audience to discern, avoid and solve the common issue that lead to the inconsistency of medication adherence estimates derived from electronic medical records database.
DESCRIPTION: Over last 5 years, the number of publications on medication adherence estimates derived from health claims or electronic medical records databases have grown very rapidly. At the same time, the inconsistency of the same medication adherence estimates across different study has become widespread. This workshop will not only explore the common issues that lead to this widespread inconsistency, but also provide audience with tools that can solve or minimize these issues, therefore, improve the quality of medication adherence estimates. Topics covered in this workshop include a conceptual framework of medication adherence, common issues that lead to widespread inconsistency of medication adherence estimates, tools and techniques that can be used to solve or minimize these issues, and 1 or 2 real world case(s) that will walk audience through the major steps in solving the issues. A 10 or 15 minute interactive discussion or exercise will follow the real world case. At the end of workshop, audience should be able to discern, avoid, and solve the most common issues that lead to inconsistency of medication adherence estimates derived from a health claims or electronic medical record database.

W19: THE UTILIZATION OF OUTPATIENT ELECTRONIC HEALTH RECORD SYSTEMS DATA FOR OUTCOMES AND QUALITY RESEARCH: STRENGTHS, WEAKNESSES AND LESSONS LEARNED: A CASE STUDY USING THE GE CENTRICITY EHR
Discussion Leaders: Joseph Couto PharmD, MBA, Outcomes Research Fellow, Jefferson School of Population Health, Thomas Jefferson University, Philadelphia, PA, USA; Albert G. Crawford PhD, MBA, MSIS, Assistant Professor, Thomas Jefferson University, School of Population Health, Philadelphia, PA, USA; Candace Gunnarsson EdD, President CEO, S2 Statistical Solutions, Inc, Cincinnati, OH, USA; Somesh Nigam PhD, Vice President, Healthcare IT, Johnson & Johnson, Office of Evidence Based Medicine, Medical Device and Diagnostics, New Brunswick, NJ, USA
PURPOSE: The purpose of the workshop is to allow researchers to share lessons learned when working with a large scale patient EHR database in studying obesity and the co-morbid conditions associated with obesity and thoughts about using such data for quality, i.e., HEDIS, reporting.
DESCRIPTION:
A growing proportion of U.S. ambulatory care physician practices are adopting Electronic Health Record (EHR) systems, e.g. Cerner, GE Centricity, GPRD and THIN. GE Centricity is used by more than 20,000 clinicians to manage the medical records of about 30 million patients in 49 states. EHRs represent a rich resource for population health research and outcomes research, particularly when studying the clinical care pathway of a chronic disease such as obesity, and also for quality, i.e., HEDIS, reporting. EHR databases incorporate and document a wide range of diagnostic and therapeutic services, specifically laboratory test results (with exact amounts and units of measurement) and medications ordered and data at multiple points in time, which allows the kind of longitudinal analyses needed for tracking the progression of obesity and the various comorbid conditions associated with it, i.e.: diabetes, hypertension, hyperlipidemia and sleep apnea. EHRs with high utilization are especially useful for obesity research as they contain patient records with recorded BMI at multiple time points. Participants currently using EHR databases to study the clinical care pathway of chronic disease should attend this workshop, as should participants performing medical record review for quality, i.e., HEDIS reporting. Topics will be presented within the context of obesity research that we conducted using the GE Centricity EHR database. There will be ample time for participants to discuss options and exchange ideas about current best practices. Key topics include: Cleaning rules; techniques to avoid duplication of patients and overestimates/underestimates; disease confirmation; external Validity and generalizability.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W20:

UTILIZING RECEIVER OPERATOR CHARACTERISTIC CURVES TO ESTABLISH A MODEL FOR CLINICAL INTERVENTION: A CASE STUDY IN THE PREVENTION OF PROSTATE CANCER
Discussion Leaders: Michael Eaddy PharmD, PhD, Senior Director, Xcenda, Palm Harbor, FL, USA; Michael Goodman PhD, Assistant Professor, University of Utah, College of Pharmacy, Salt Lake City, UT, USA
PURPOSE: This workshop will demonstrate how receiver operator characteristic (ROC) curves can be used to establish optimal threshold models for recommending clinical and/or pharmaceutical intervention.
DESCRIPTION: The use of non-invasive, diagnostic, prognostic, and treatment response monitoring tests have provided clinicians with an opportunity to best determine the need of clinical and/or pharmaceutical interventions. Given the increase in frequency and costs of these tests and pharmaceuticals used to prevent / treat various diseases, healthcare economists are now faced with assessing the optimum threshold for intervening, factoring in the cost of false negatives and false positives. ROC curves can be used as a first step in this process while also statistically evaluating the value of new diagnostic tests. This workshop will utilize a prostate cancer case study to demonstrate the usefulness of using ROC curves in establishing a prostate-specific antigen clinical value that can be used as a point of action for prostate cancer prevention. The objectives of this workshop are to: (1) review aspects of sensitivity and specificity in development of prediction models; (2) provide an understanding of ROC curves and how they are used to determine predictive thresholds; (3) review how ROC curves can be used for statistical comparisons of diagnostic tests; and (4) demonstrate how to determine the clinical and economic implications of utilizing the predictive threshold. Examples will be drawn from the presenters' own research as well as from the published literature. Audience participation will be encouraged through the use of an audience response system.

W21: CURE, CARE, PREVENT - HOW TO SUPPORT DECISIONS ON HEALTH-CARE BUDGETS?
Discussion Leaders: Maarten J. Postma PhD, Professor Pharmacoeconomics, University of Groningen, Department of Pharmacy, Unit of PharmacoEpidemiology & PharmacoEconomics (PE2), Groningen, The Netherlands; Lieven Annemans PhD, MSc, Professor of Health Economics, University of Ghent, Brussels University, Ghent, Belgium; Keith H. Tolley MPhil, Director, Tolley Health Economics Limited, Buxton, Derbyshire, UK; Cornelis Boersma MSc, PhD Student, and Director, University of Groningen, Department of Pharmacy, Unit of PharmaEpidemiology & PharmacoEconomics (PE2)/HECTA B.V, Groningen, The Netherlands
PURPOSE: To evaluate the current role of pharmacoeconomics in health-care decision-making with a glance at future opportunities.
DESCRIPTION: The increasing impact of cost-effectiveness in health-care decision-making often results in complex considerations. Regulatory procedures for reimbursement aim at efficient allocation of existing financial resources based on several guidelines, preferences and pharmacoeconomics outcomes. Difficulties exist with availability and interpretation of efficacy data from clinical trials including related uncertainty. More importantly, the availability of valid data determines the success rate for conducting adequate economic evaluations. In particular, health-care decisions are generally not based on real-life observational data, but solely rely on efficacy data from clinical trials. Finally, other criteria such as medical need and budgetary impact may be of more importance in final decision-making. Here, we address how health-economists can support decisions on health-care budgets. Recently, the Dutch reimbursement authority launched a procedure for (new) expensive in-patient drugs with conditional reimbursement (3 years) based on approximation of the cost-effectiveness, including value-of-information and indirect comparison analyses. To provide proof of value for money, additional data gathering and economic assessments have to be performed within 3 years. One could argue to also apply this procedure to out-patient drugs and prevention strategies (eg. screening, vaccination). This is the case in Belgium, where such a measure exists already since 2002 and includes a second assessment within 1.5 to 3 years. Concerns about uncertainty and limited generalisability from clinical trial outcomes could be reduced with post-launch economic evaluations using real-life data. On the other hand, the cost-effectiveness of such an approach could also be questioned. Based on recent examples from Belgium, The Netherlands and the UK, current ‘country-specific' procedures, guidelines, timing of economic analyses, and the role of pharmacoeconomists in health-care decision-making, will be debated interactively with the audience, as well as possible pitfalls with the “2-stage” assessment.
 
WORKSHOPS SESSION IV Wednesday, May 20, 2009
12:45 PM - 1:45 PM

CLINICAL OUTCOMES RESEARCH

W22: VALUE MESSAGES: DEVELOPING, INCORPORATING, AND MAKING USE OF A CORE STRATEGIC TOOL
Discussion Leaders: Kati Copley-Merriman MBA, MS, Global Head, Regulatory and Health Outcomes Strategy, RTI Health Solutions, Ann Arbor, MI, USA; Amy Barrett MSPH, MA, Director, Regulatory and Health Outcomes Strategy, RTI Health Solutions, Research Triangle Park, NC, USA; Stephanie Barrows MA, MPH, Associate Director, Regulatory and Health Outcomes Strategy, RTI Health Solutions, Ann Arbor, MI, USA; Jennifer Whiteley EdD, MSc, MA, Associate Director, Global Health Outcomes and Strategic Pricing, Genzyme, Cambridge, MA, USA
PURPOSE: The Purposes of this workshop are to discuss 1) the process of developing clear value messages for a product and adapting them for various stakeholders (patients, physicians, pricing and reimbursement authorities, regulatory bodies); 2) building plans for outcomes research strategy and product lifecycle management around value messages; and 3) developing an effective global value dossier for use by country affiliates.
DESCRIPTION:
This workshop is designed to familiarize participants with the process of developing value messages for internal and external stakeholders and incorporating value messages into outcomes research strategy plans and global value dossiers, using a hypothetical drug and disease as an example. Defining and communicating the value of a new drug to stakeholders is important in achieving successful market uptake. Clear value messages provide a roadmap for outcomes research throughout a product's lifecycle and facilitate the appropriate choice of clinical trial endpoints. Global value dossiers also are built around value messages and their supporting evidence, with the goal of summarizing a drug's value story for internal stakeholders and country affiliates. Workshop attendees will participate in evaluating and creating value messages, with the goal of understanding the development and use of these messages.

ECONOMIC OUTCOMES RESEARCH

W23: UNCERTAINTY IN COST EFFECTIVENESS STUDIES: WHEN PICTURE IS WORTH A THOUSAND WORDS
Discussion Leaders: Katia Noyes PhD, MPH, Associate Professor, University of Rochester School of Medicine, Community and Preventive Medicine, Rochester, NY, USA; Elisabeth Fenwick PhD, Lecturer, University of Glasgow, Section of Public Health and Health Policy, Glasgow, UK
PURPOSE: Multiple sources of uncertainty make interpretation of cost-effectiveness analyses (CE) seem weak or ambiguous. Graphical ways of presenting uncertainty, such as confidence ellipses and acceptability curves (CEAC), are very efficient when presenting results of CE evaluations for diverse audience. The participants of the workshop will learn how to 1. build confidence ellipses within the CE plane in Excel using patient-level data; 2. understand the relationship between the scatter plot of incremental costs and incremental effects, location of confidence ellipse in respect to the CE threshold and CEAC; 3. build CEAC using Excel; 4. understand the strengths and weaknesses of alternative methods for presenting the results of CE analyses.
DESCRIPTION:
The instructors will describe the sources of uncertainty in CE studies using patient-level vs. aggregated data, outline current recommendations for presenting uncertainty, and discuss several examples of studies that estimated and presented various types of uncertainty. Using Excel spreadsheet and bootstrapped patient-level data, the instructors will demonstrate how to build confidence ellipses around the ICER, step by step, and how to interpret scatterplots on the cost-effectiveness plane. Similarly, the participants will learn how to generate CEACs for 2 (and more) interventions and recognize the common problem with interpretation of CEACs. The instructors will show how confidence ellipses and CEACs complement each other and help articulate results of CEA and consequences of making decisions based on those results to decision makers at various levels. The session will include an instructor led discussion about the strengths, weaknesses and overall appropriateness of alternative methods for presenting cost-effectiveness results to decision makers. Each participant will receive files containing datasets and templates for generating CEAC and confidence ellipses in Excel and will be able to follow instructors during the workshop or generate these graphs on her/his own at their convenience.
   
W24:

MODELING APPROACHES FOR ASSESSING THE VALUE OF VACCINATION AGAINST INFECTIOUS DISEASES: WHAT ARE THE NEEDS AND WHAT IS THE BEST ANSWER?
Discussion Leaders: Laurent Coudeville PhD, Health Economics Deputy Director, Sanofi Pasteur, Lyon, France; Pascal Crépey PhD, Health Economics Manager, Sanofi Pasteur, Lyon, France; Andrew J. Dunning PhD, Biostatistics Manager, Sanofi Pasteur, Swiftwater, PA, USA; Vicky Ng BSc(Hon), PhD Candidate, The Hospital for Sick Children, Toronto, ON, Canada
PURPOSE: The aim of this workshop is to discuss and illustrate the different approaches that may be used to assess the value of vaccination programs.
DESCRIPTION: Decisions on implementation of new vaccination programs require a good understanding of expected impacts. For new vaccines, evidence related to duration of protection, impact on clinical outcomes and indirect protection (herd immunity) are generally incomplete, but these parameters are key drivers of the decision to adopt vaccination. Modeling provides a means of synthesizing the best available data, allowing decision makers to maximize efficiency and cost-effectiveness in the face of uncertainty. Using real case examples on diseases such as meningococcal meningitis, pertussis, pandemic and seasonal influenza, and dengue, we will illustrate different types of models used to inform optimal vaccine policy. First, we will focus on dynamic transmission models aimed at assessing the direct and indirect effects of vaccination in both large populations, and small sub-populations. We will highlight factors that motivate the choice of a specific modeling approach by presenting examples of age-structured compartmental models, cohort models, community-based and agent-based models. The second part will focus on statistical models using immunogenicity data to evaluate vaccine efficacy, and the duration of protection conferred by vaccines. The third component will focus on the estimation of "intangible" costs and benefits associated with vaccination. Conjoint analysis will be introduced as an approach to measure preferences in the face of multiple trade-offs in the decision to accept vaccination. We explore the possibility to explicitly describe factors driving vaccination acceptance and the implicit "willingness-to-pay" to avoid illness and disease transmission to others. During this workshop, participants will be invited to discuss optimal modeling approaches and interactively evaluate different vaccinations strategies. The workshop will end on a general note on the role of modeling for the evaluation of vaccination policies.

PATIENT-REPORTED OUTCOMES/PREFERENCE-BASED RESEARCH

W25:

CONDUCTING QUALITATIVE RESEARCH TO ASSESS CONTENT VALIDITY: SHARING BEST PRACTICES
Discussion Leaders: Meryl Brod PhD, President, The BROD GROUP, Mill Valley, CA, USA; Torsten Christensen MSc, Senior Health Economist, Novo Nordisk A/S, Bagsværd, Denmark
PURPOSE: Establishing content validity for both new and existing PRO measures is central to a scientifically sound instrument development process. The most appropriate way to ensure content validity is by conducting qualitative research of direct communication with patients to adequately capture their perspective on issues of importance relative to the focus of the PRO measure. Unfortunately, “qualitative research” covers a wide variety of conceptual principles and methodologies, and includes wide variation in terms, concepts, assumptions and analytic principles. Given these variations, it is especially important to maintain the scientific integrity of the research process in order to maintain credibility. Further, a variety of logistical considerations are crucial in conducting this research. Together, conceptual and logistical issues present a challenge to the field as information regarding the best practices or the “nuts and bolts” of using qualitative research to establish content validity, in relationship to PRO development, is scarce and often thought of as a “black box”. The purpose: of this session is to provide an overview on the logistical and conceptual “nuts and bolts” of conducting qualitative research (focus groups and individual interviews) to ensure content validity of new and existing PRO measures These best practices are based on the authors sum of experience with more than 40 years conducting qualitative outcomes research in academia and industry.
DESCRIPTION: The workshop will offer best practices and concrete guidelines for a theoretical framework, IRB issues, developing the interview guides, defining the sample, rules for conducting interviews, interview logistics, documenting reaching saturation, data analysis , item generation and cognitive debriefing for establishing and assessing content validity for both new and existing PRO measures. The industry perspective based on discussions with the FDA regarding assessing and documenting content validity will also be presented. Participants will participate in evaluating the content validity for a hypothetical PRO.

 
W26:

HEALTHY YEARS EQUIVALENTS: A PRACTICAL ALTERNATIVE TO QALYS?
Discussion Leaders*: A. Brett Hauber PhD, Senior Economist and Head, RTI Health Solutions, Health Preference Assessment, Research Triangle Park, NC, USA; F. Reed Johnson PhD, Principal Economist, RTI International, Research Triangle Park, NC, USA; John FP Bridges PhD, Assistant Professor, Johns Hopkins University, Bloomberg School of Public Health, Health Policy and Management,
Baltimore, MD, USA
PURPOSE: The objective of the workshop is to help participants evaluate the practical usefulness of healthy-years equivalents as an alternative to QALYs in health technology assessment.
DESCRIPTION: The quality-adjusted life year (QALY) is the dominant measure of health value in health-technology assessment (HTE) despite well-known concerns about the preference-elicitation methods used to construct health-state utilities and the strong assumptions required to calculate QALY measures using these utilities. The healthy-years equivalent (HYE) was proposed over 15 years ago as an alternative measure of health value that appeared to overcome many QALY problems. The primary argument against the HYE has been that it is difficult to estimate and therefore is impractical for HTE use. After some debate, the QALY clearly won the policy battle; however, the HYE is not yet dead. Researchers recently have begun re-assessing the merits of this less restrictive measure of health values. Among the proposed strategies for obtaining empirical estimates is adapting conjoint-analysis methods. This workshop will provide a review of the original controversy on conceptual and empirical advantages and disadvantages of HYEs relative to QALYs and a review of several recent empirical applications. The presenters will collect data from workshop participants on their quality/duration tradeoff preferences and the presenters will demonstrate how to derive HYEs from such data. Participants also will provide examples of their own experiences in obtaining plausible QALY estimates for cost-utility analyses. The presenters will discuss prospects for using HYEs derived from conjoint data to solve such problems. Because HYEs challenge the widely accepted way of thinking about HTA, this workshop is likely to generate a lively discussion among participants. This workshop will be particularly useful to researchers who have encountered difficulty in estimating QALYs for acute health conditions or eliciting health-state utilities when subjects are unwilling to trade life-years for improvements in health.

USE OF REAL WORLD DATA

W27:

CONSIDERATIONS IN THE USE OF SECONDARY DATA SOURCES FOR ONCOLOGY RESEARCH
Discussion Leaders: Kathy L. Schulman MA, Lead Researcher, Thomson Reuters, Healthcare, Cambridge, MA, USA; Kathleen A. Foley PhD, Project Director, Thomas Jefferson University, School of Population Health, Philadelphia, PA, USA; Mark S. Walker PhD, Director of Scientific Management, Accelerated Community Oncology Research Network, Inc, Memphis, TN, USA
PURPOSE: The objective of this workshop will be to educate the audience on methodological considerations in conducting oncology research using healthcare claims or clinical practice databases.
DESCRIPTION: Secondary data sources are increasingly being used to profile oncology treatment patterns; assess outcomes across therapeutic regimens and/or quantify the cost associated with care. However, these data systems often lack the degree of clinical specificity needed either for sample selection or measurement of select clinical or economic outcomes. Development of appropriate algorithms or proxies requires both a clinical understanding of the type of cancer being studied as well as an understanding of the reimbursement or practice management system that has generated the data. This workshop will review methodologies for selecting the appropriate population for study while minimizing the measurement error inherent in secondary data sources. The workshop will review the following: 1) sources of error in electronically retrieved data, 2) algorithms used in the identification of primary tumor type, disease stage, and treatment setting, 3) the construction of chemotherapy treatment episodes, 4) considerations in the identification of drug related adverse events, including use of patient reported outcomes collected as part of routine care, and 5) validation of extracted and proxy data. Examples will be drawn from published and ongoing projects. Proposals on key issues for future work to validate claims based algorithms will be presented. Workshop participants will be encouraged to offer their perspectives and recommendations.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W28: DECISION-MAKING PROCESS BY PAYERS IN EUROPE: A MULTIPLE CRITERIA PROCESS
Discussion Leaders: Eline Kogels MSc, Consultant, Danone Medical Nutrition, Health Economic & Market Access Manager, Amsterdam, The Netherlands; Mark JC Nuijten MD, PhD, MBA, Consultant, Ars Accessus Medica/Erasmus University Rotterdam, Amsterdam, The Netherlands
PURPOSE: The Purpose of this workshop is to explore the critical data, which are relevant for payers (health insurers) in Europe. We will present an overview of current situation in main European countries, including some case studies, where payers played a crucial role in obtaining market access for new pharmaceuticals.
DESCRIPTION:
Decisions on reimbursement of new pharmaceuticals have been central decisions based on traditional registration data (efficacy, safety). Now also other data are required, which relate to the use of the drug in real daily practice: cost-effectiveness and budget impact. A positive reimbursement decision used to be sufficient for market access and the target audience for prescription was the physician. However the introduction of market mechanisms and the decentralisation of the health care decision making process resulted in an additional hurdle for new pharmaceuticals: the requirement of listing on local formularies of payers. The physicians' freedom of prescribing new pharmaceuticals has been rather limited by these local formularies. On the other hand, payers may decide to reimburse a new drug after negative central reimbursement decision and absorb the additional costs. The decision criteria for central reimbursement are explicitly described in official policy documents and research guidelines, and the process has been embedded in a legal framework. Contrary the decision criteria, which are applied by payers, are not explicitly stated and the process lacks transparency. A more profound understanding of this decision making process of payers will allow a more proactive development of a dossier for health insurers during the drug program phase and faster access on formularies. We also compare decision criteria for payers in Europe with payers in US. Key topics will include: decision criteria, payer, and decision making process.
WORKSHOPS SESSION V Wednesday, May 20, 2009
2:00 PM - 3:00 PM

CLINICAL OUTCOMES RESEARCH

W29: A REVIEW OF CURRENT RISK-BENEFIT ASSESSMENTS FOR DRUG SAFETY
Discussion Leaders: Jeff Jianfei Guo PhD, Associate Professor, University of Cincinnati, College of Pharmacy, Cincinnati, OH, USA; Swapnil Pandey MS, Project Lead of Risk Management, Kendle International, Cincinnati, OH, USA; John J. Doyle DrPH, MPH, Managing Director & Practice Leader, Quintiles Global Consulting, Global Market Access, Hawthorne, NY, USA; Dennis W. Raisch PhD, Professor, College of Pharmacy, University of New Mexico and Associate Center Director, Scientific Affairs, VA Cooperative Studies Program Clinical Research Pharmacy, Albuquerque, NM, USA
PURPOSE: Regulatory authorities often must balance the risks and benefits conferred by a new drug during the evaluation process. This risk-benefit assessment is not typically presented in a consistent and integrated framework. This study is to review and compare the available risk-benefit assessment methods for drug evaluations. Scientific risk-benefit assessment methods would enhance effectiveness of risk-benefit trade-off decision making.
DESCRIPTION:
Using Medline, Cochrane, and other Internet-based search engines, an extensive literature review was performed to identify and evaluate methodologies of risk-benefit assessment. This workshop will discuss both qualitative and quantitative risk-benefit assessment methods that are available in the current literature, focusing on six major risk-benefit assessment methods: 1) quantitative framework for risk and benefit assessment, 2) benefit-less risk analysis (BLRA), 3) number needed to treat (NNT) and number needed to harm (NNH), 4) minimum clinical efficacy (MCE), 5) incremental net health benefit (INHB) and quality adjusted life years (QALY), 6) benefit-risk plane (BRP) and risk-benefit acceptability threshold (RBAT), including incremental risk-benefit ratio (IRBR), and probabilistic simulation method (PSM) –Monte Carlo Simulation. While some methods rely on subjective weighting schemes or non-statistical assessments (e.g., NNT and NNH), other methods are used for obtaining joint distributions of benefit and risk (e.g. BRP and RBAT) with sound mathematical basis for risk-benefit assessments. The implications of these methods will be further discussed for the pharmaceutical industry and regulatory agencies. In conclusion, several scientific methods for quantifying the benefits and risks are available and can be used to reduce the current subjective onus on the regulatory agencies and help guide them towards making a more objectively oriented and transparent decision making in terms of drug risks and benefits. Multiple approaches maybe required to estimate the risk-benefit profile across different therapeutic indications and treatment populations.

ECONOMIC OUTCOMES RESEARCH

W30: THE WILLINGNESS TO PAY FOR THE WELLBEING OF PETS OR HOW TO APPLY COST-EFFECTIVENESS CRITERIA FOR PHARMACEUTICALS FOR COMPANION ANIMALS
Discussion Leaders: Mark JC Nuijten MD, PhD, MBA, Consultant, Ars Accessus Medica/Erasmus University Rotterdam, Amsterdam, The Netherlands; Ilse Van Vlaenderen DVM, MSc, Consultant, Deloitte, Diegem, Belgium; Barbara Poulsen Nautrup DVM, PhD, Consultant, EAH Consulting, Juelich, Northrhine Westf, Germany
PURPOSE: Animal health economics is no longer restricted to food producing animals, but is increasingly gaining importance for treatment decisions in companion animals. First cost-effectiveness analyses have been conducted, supporting the decision making at the level of the veterinarian and pet owner. The aim of this workshop is to explore to what extend cost-effectiveness criteria for human pharmaceuticals are relevant and applicable in veterinary medicine, especially in companion animals. The pros and cons of applying these decision criteria for veterinary pharmaceuticals will be discussed using case studies from Germany and Switzerland.
DESCRIPTION:
Cost-benefit analyses have been conducted for decades to support medical decision making in food producing animals. Recently, also classical cost-effectiveness studies are emerging in companion animals. Although the aim and type of health economic studies in pets are similar to those in human medicine, there are also differences: reimbursement of human pharmaceuticals is mainly based on cost-effectiveness analyses from a third party payer perspective, whereas third party payers do not play a substantial role in animal health, as health insurances for pets are rare in most countries. Accordingly, the actual use of veterinary pharmaceuticals depends on the willingness and ability to pay by the animal owner. The veterinary doctor is also a key contributor in the decision making process because in many countries the veterinarian's income depends not only on the service provided but also on the pharmaceuticals stocked and sold. Case studies will be used to show that standard cost-effectiveness analyses can be performed for veterinary drugs by applying international guidelines for cost-effectiveness analyses. The choice and appropriateness of decision criteria for the use of veterinary pharmaceuticals will be addressed, and finally approaches will be discussed with the audience how to interpret results from cost-effectiveness analyses, and how to approximate the willingness to pay of pet owners.
W31:

ESTABLISHING THE CONTENT VALIDITY OF PRO INSTRUMENTS
Discussion Leaders: Chad Gwaltney PhD, Scientific Consultant, PRO Consulting, Westerly, RI, USA; Mona Martin MPA, Director, Health Research Associates, Seattle, WA, USA
PURPOSE: To increase understanding of how content validity can be examined and documented when developing new patient-reported outcome (PRO) instruments.
DESCRIPTION:
According to the FDA draft guidance on using PROs in clinical development programs, it is essential to establish the content validity of any PRO instrument that will be used to support label claims. The goal of this session is to define content validity and present industry and regulatory perspectives on demonstrating and documenting content validity. In the drug development context, content validity refers to the match between the items in an instrument and the concepts that are targeted for label claims. Establishing content validity involves ensuring that instrument items assess concepts that are relevant for the patient population, cover all important aspects of the targeted concepts, and are understood by patients. Methods for addressing these criteria will be reviewed in the session. In particular, the session will focus on procedures associated with conducting and analyzing qualitative interviews with patients. These interviews are a critical step in developing new PRO instruments, but they can be challenging to complete and the resulting data can be difficult to interpret. Case examples from industry of how patient interviews have been used in developing and validating new PRO instruments will be presented. An interactive exercise will provide audience members the opportunity to review and interpret patient interview data in the context of developing a new instrument.

PATIENT-REPORTED OUTCOMES/PREFERENCE-BASED RESEARCH

W32:

IMPLEMENTING E-PRO MEASURES IN CLINICAL TRIALS: WHAT SPONSORS NEED TO KNOW
Discussion Leaders: Sonya Eremenco MA, ePRO Manager, United BioSource Corporation, Health Care Analytics, Bethesda, MD, USA; Stephen Joel Coons PhD, Professor, University of Arizona, Pharmaceutical Sciences, Tucson, AZ, USA; Ari Gnanasakthy MSc, Director, Novartis Pharmaceuticals, Health Economics and Outcomes Research, East Hanover, NJ, USA
PURPOSE: Using electronic methods (ePRO) to capture patient-reported outcomes data is becoming the norm rather than the exception in multinational clinical trials. The many advantages of ePRO have been demonstrated, but the shift from paper to electronic platforms can present challenges that result in negative, yet largely avoidable, consequences for the trial. The purpose of this workshop is to enable sponsors to become better informed consumers of ePRO technology by identifying key areas for evaluation and key questions to consider during the decision-making and implementation process.
DESCRIPTION: The decision to implement ePRO measures in a clinical trial is a critical and costly one that has major implications for the study's success. The implementation of ePRO measures has also grown potentially more challenging due to the FDA draft PRO guidance and the ISPOR ePRO Task Force report, which have highlighted the importance of scientific principles in the study design and implementation process. Furthermore, PRO instrument developers may have even more stringent requirements for migrating their instruments to ePRO devices and demonstrating equivalence. This workshop will identify and discuss factors that sponsors need to consider during the decision-making process that can facilitate successful implementation of ePRO technologies in clinical trials. Topics include selecting the appropriate ePRO platform, challenges in designing an ePRO interface, the numerous front-loaded activities required before beginning the study, impact of protocol amendments, validating ePRO versions of an instrument, and working with instrument developers to operationalize their measures on electronic platforms. The use of ePRO measures in clinical trials is growing steadily; hence, it is critical for all stakeholders to work together to find solutions that improve the use of the technology and help realize its full potential in terms of efficiency and data quality. Audience participation will be facilitated through ePRO-related case studies and discussion of audience-generated issues and solutions.

USE OF REAL WORLD DATA

W33:

REAL WORLD DATA BEFORE MARKET ACCESS
Discussion Leaders: Martin Nottmeier MSc, Pharm, International Pricing & Market Access Manager, Nycomed, International Pricing & Market Access, Taastrup, Denmark; David Becedas MSc, Pharm, Team Leader Observational Research, Scandinavian Outcomes, Observational Research, Solna, Sweden
PURPOSE: To describe and discuss whether it is feasible to generate real world data before market access when collecting data from a compassionate use programme.
DESCRIPTION: A sound health technology assessment should be based on real world data and not on data from an artificial setting like clinical trials. Payers and policy makers are interested in the consequences that a new health technology will have on budgets – not how a health technology behaves in a controlled artificial setting. The challenge is thus to generate real world data before market access. In some disease areas physicians can treat patients with new promising medicines even before marketing approval in a compassionate use programme. Disease registries are valuable peri- and post-approval tools, providing evidence of the real-world results of medical products, therapies, and services. A separate disease registry running in parallel with a compassionate use programme could thus be used to capture real world treatment data. This workshop will describe and discuss the possibilities of combining a compassionate use programme with a separate, parallel disease registry to generate and collect treatment data prior to marketing approval of a drug. Compassionate use programmes are in place to allow treatment for incurable patients in a therapeutic impasse or patients with life-threatening diseases and are not to replace clinical trials. However, it is not clear whether it is ethical to collect and use data from such a programme in a health technology assessment. Is there any ethical way of generating real world data for payers before market access?

 
W34:

EVALUATION OF CONTINUITY OF CARE BETWEEN INPATIENT AND OUTPATIENT SETTINGS VIA ADMINISTRATIVE CLAIMS DATABASES USING THE EXAMPLE OF VTE PROPHYLAXIS IN TOTAL HIP AND TOTAL KNEE REPLACEMENT
Discussion Leaders: Henry Joe Henk PhD, Director, i3 Innovus, Health Economics & Outcomes, Eden Prairie, MN, USA; Heather P. McDonald MSc, Senior Manager, Health Economics and Outcomes Research, Bayer Inc, Toronto, ON, Canada; Bijan Borah PhD, Senior Researcher, i3 Innovus, Eden Prairie, MN, USA; Michael Nelson, Vice President, Health Economics and Outcomes, i3 Innovus, Eden Prairie, MN, USA  
PURPOSE: The Purpose: of this workshop is to demonstrate the importance of visibility into inpatient and outpatient medication use in assessing outcomes in specific situations, such as post-surgical VTE outcomes.
DESCRIPTION: Administrative claims databases are often used for health outcomes research. The longitudinal nature of such databases provides the ability to track patients' health care utilization patterns and costs over a relatively long period of time. However, in research studies that require tracking of a specific treatment regimen over a continuous period of time, a single data source (e.g. claims databases, electronic medical records) may not contain all necessary information. For example, to analyze the effectiveness of VTE prophylaxis following joint replacement surgery, it is important that patients' health care utilization be followed both during the inpatient stay when the THR/TKR surgery took place and following hospital discharge. Administrative health claims databases typically provide limited information about medications administered during the hospital stay. In addition, mortality is often under reported in administrative claims data. Thus for research studies requiring the pattern of inpatient medication use and mortality, it is important to link claims databases to external sources of information. This improves our ability to assess patients' medication utilization and survival for a continuous period of time, allowing us to better evaluate the effect of medication use on outcomes. This workshop will demonstrate how three databases, one administrative claims database associated with a large US health plan, an inpatient database that provides detailed inpatient records, and the Social Security Death Master File may be linked to construct a sample of patients for whom there are utilization and mortality records during inpatient stay and following discharge from hospital. Methodological and other issues associated with linking of such databases will be discussed. Audience participation will be encouraged through the use of a real-world example.

HEALTH CARE POLICY DEVELOPMENT USING OUTCOMES RESEARCH

W35: A REVISED FRAMEWORK FOR APPRAISING NOVEL MOLECULAR CLASSIFIERS
Discussion Leaders: John Hornberger MD, MS, Principal, CEDAR Associates LLC and Adjunct Clinical Professor of Medicine, Stanford University School of Medicine, Menlo Park, CA, USA; Gary Lyman MD, MPH, Professor of Medicine, Duke University and the Duke Comprehensive Cancer Center, Oncology, Durham, NC, USA
PURPOSE: To illustrate a comprehensive and valid framework for appraising novel molecular classifiers.
DESCRIPTION:
There has been an unprecedented level of investment in developing genomic classifiers intended to improve diagnostic accuracy (e.g., cystic fibrosis, hemochromatosis) and to predict risk of recurrent disease (e.g, thromboembolism, breast cancer), assess pharmacokinetics (e.g., warfarin), and predict response to therapy (e.g., cancer chemotherapy). Experts at the US CDC estimate that more than 1,000 assays may be in development. With the promise of each new assay entering clinical practice comes the relevant question of what components should be included when appraising a classifier's effectiveness, safety, utility, and affordability. The audience for a standardized appraisal processes is large and has diverse backgrounds and interests; it includes regulatory officials, health technology appraisal groups, investors, developers of genomic assays, clinical investigators, payers, physicians, and the general public. In 2006, Ramsey et al. established one of the first frameworks to guide such appraisals. In addition, several health technology assessment groups adapted their existing appraisals processes, many of which were developed initially for evaluating pharmaceuticals, to the appraisal of genomic assays. Despite these important initial steps, no single set of processes has been widely endorsed by stakeholders nor uniformly applied. This leaves considerable uncertainty on the efforts that are necessary and sufficient to properly develop and validate a novel classifier, which also has led to confusion among physicians, patients, and regulators about why some classifiers are viewed as being clinically acceptable while others are not. This workshop will summarize the many issues that have concerned technology assessment groups when appraising selected genomic classifiers; it will also present a comprehensive, revised framework that addresses the myriad questions that have arisen in the appraisal process. Time will be allocated for audience discussion to address the completeness and validity of the proposed framework.

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