WORKSHOPS - SESSION I
Sunday, 25 October 2009: 15:15-16:15
Economic Outcomes Research
W1: USING A CATALOGUE OF EQ-5D SCORES TO MODEL QALYS FOR COST-EFFECTIVENESS ANALYSES
Discussion Leaders: Patrick W. Sullivan PhD, Associate Professor, Regis University, Rueckert-Hartman College for Health Professions, Denver, CO, USA; Mark J Sculpher PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK; Vahram H Ghushchyan PhD, Professional Research Assistant, Pharmaceutical Outcomes Research Program, University of Colorado Denver, Aurora, CO, USA; Julia F. Slejko BA, PhD Student, Pharmaceutical Outcomes Research Program, University of Colorado Denver, Aurora, CO, USA
PURPOSE: To demonstrate how EQ-5D scores from a publicly-available catalogue of chronic condition scores can be used to calculate QALYs for cost-effectiveness analyses.
DESCRIPTION: Guidelines for many countries recommend the use of QALYs as the gold standard for quantifying health gain in cost-effectiveness analyses. The National Institute for Health and Clinical Excellence (NICE) in England and Wales has expressed a preference for the EQ-5D in developing QALYs for cost-effectiveness analyses. However, directly eliciting EQ-5D scores for specific disease states can be cumbersome and expensive. Often, researchers attempt to find utility scores from the published literature to populate their models and calculate QALYs, but in many cases these estimates are not available or are not appropriate. This workshop will begin with an overview of the recent development of a catalogue of several hundred EQ-5D scores for a wide variety of chronic ICD-9 and CCC codes that can be used to estimate QALYs in cost-effectiveness analyses. The catalogue currently provides preferences for the U.K. and U.S. national populations. We will also discuss how to correctly interpret the EQ-5D marginal disutilities from the catalogue and how to adjust for multiple comorbidities. Next, we will demonstrate how to use the existing publicly-available EQ-5D scores from this catalogue to calculate QALYs for cost-effectiveness analyses from the U.K. and the U.S. perspectives. Specific examples of how to utilize the chronic condition EQ-5D scores to calculate QALYs for cost-effectiveness models will be provided. Finally, workshop participants will be encouraged to discuss the general use of the catalogue scores as well as develop questions related to their own specific applications.
W2: QUANTIFYING THE RISK IN RISK SHARING: COLLECTING AND MODELING OUTCOMES OF THE NEW ACCESS STRATEGIES
Discussion Leaders: J. Jaime Caro MDCM, FRCPC, FAC, Senior Vice President, Health Economics, United BioSource Corporation, Health Care Analytics, Lexington, MA, USA; Zeba M. Khan RPh, PhD, Vice President Pricing and Market Access, Celgene Corporation, Summit, NJ, USA; Ian Joseph BA, Research Associate, United BioSource Corporation, Health Care Analytics, Lexington, MA, USA
PURPOSE: To explore the best practices for and implications of applying patient access strategies to budget impact and cost effectiveness models.
DESCRIPTION: As health technology assessment agencies including NICE, PBAC, and IQWiG increasingly require drug companies to demonstrate proof of a minimum return on investment from payers’ pharmaceutical dollars, manufacturers are forced to seek solutions that preserve patient access to therapy. Some have instituted risk sharing pricing agreements in which real world outcomes inform acquisition cost. While these innovative approaches have proven effective in assuring access, knowledge gaps exist around the eventual clinical and economic implications. Unfortunately, pressures to bring the therapeutic to market rarely afford stakeholders sufficient time to understand the implications of a risk sharing scenario prior to initiation. Manufacturers and HTAs must determine if the risk sharing agreement will: preserve patient access; prevent disputes over patient outcomes; require new infrastructure to manage distribution and revenue collection; incentivize and foster R&D in new disease areas. As a surrogate for a stronger knowledge base, manufacturers are beginning to synergize economic models with parameters from their risk sharing agreements through the use of Patient Access Modules. It is paramount that health outcomes researchers develop strong understandings of these techniques. This workshop will cover best practices for developing and assessing Patient Access Modules to inform the initial risk sharing decision and to monitor the clinical and economic outcomes from post-marketing surveillance. Participants will be introduced to commonly used risk sharing agreements and the associated modeling techniques. Techniques for monitoring and evaluating these strategies will also be examined. The viewpoints of patients, payers and manufacturers will be considered. Finally, participants will be presented with an interactive case study in which they must adopt the viewpoint of HTAs or manufactures and agree on a risk sharing agreement for a hypothetical therapeutic.
Health Care Policy Development Using Outcomes Research
W3: ORPHAN DRUG FUNDING: A MODEL FOR PERSONALIZED MEDICINE?
Discussion Leaders: Deirdre Mladsi BA, Global Head, RTI Health Solutions, Research Triangle Park, NC, USA; Salomé de Cambra MD, MBA, Senior Consultant, RTI Health Solutions, Barcelona, Spain; Antoni Gilabert Mr, Head of Pharmaceutical Care Management Office, Catalan Health Service, Barcelona, Spain; Eric C Faulkner MPH, Senior Director, Pricing & Reimbursement, RTI Health Solutions, Research Triangle Park, NC, USA
PURPOSE: The aim of this workshop is to examine the rationale for funding decisions for orphan drugs and to explore the extent to which similar approaches may or may not apply to funding decisions in an era of personalized medicine.
DESCRIPTION: Criteria for orphan-drug designations vary across markets, but typically orphan drugs are targeted for use in small numbers of patients and are associated with high annual treatment costs. As the era of personalized medicine approaches, drugs for more common diseases may take on these characteristics of orphan drugs—greater scientific understanding of disease processes and the increasing availability of sophisticated biomarkers will permit researchers to define more precisely a new drug’s target patient population. To achieve an adequate return on investment spread over a smaller patient base, drug manufacturers will seek relatively higher prices. The extent to which orphan drug reimbursement may serve as a model for reimbursement of drugs in an era of personalized medicine is unclear. Despite similarities in target patient population sizes and drug prices, considerations such as the severity of illness and the availability of alternative treatments may be key drivers in reimbursement decision making. Payers and economists alike have contributed to funding debates for orphan drugs. Considerations include issues of efficiency, equitable access to health care (a justification for both positive and negative decisions), affordability (of increasing concern as the number of orphan drugs increases), cost-effectiveness (for which traditional threshold levels have been alternately challenged and reinforced), and the extent to which current methods for benefit valuation accurately reflect societal preferences. During the workshop, definitions and reimbursement considerations for orphan drugs in various markets will be reviewed. Examples of orphan and non-orphan drugs will be compared and contrasted in terms of reimbursement decisions.
Patient-Reported Outcomes/Preference-based Research
W4: COMPARING APPLES AND ORANGES: USING CONJOINT-ANALYSIS DATA TO OBTAIN SEVEN QUANTITATIVE BENEFIT-RISK MEASURES
Discussion Leaders: A. Brett Hauber PhD, Senior Economist and Global Head, Health Preference Assessment, RTI Health Solutions, Research Triangle Park, NC, USA; F Reed Johnson PhD, Senior Fellow and Principal Economist, Health Preference Assessment, RTI Health Solutions, Research Triangle Park, NC, USA; John FP Bridges PhD, Assistant Professor, Bloomberg School of Public Health, Health Policy and Management, Johns Hopkins University, Baltimore, MD, USA
PURPOSE: This interactive workshop will introduce participants to the conceptual and empirical basis for quantifying benefit-risk tradeoff preferences using conjoint-analysis. The workshop will give participants new tools for assessing stakeholders’ risk tolerance for clinical benefits of new pharmaceuticals.
DESCRIPTION: While much effort has been devoted to developing more accurate measures of efficacy and safety, decision makers are left with the problem of comparing outcome measures expressed in different metrics. Regulatory agencies such as EMEA and the FDA are actively exploring quantitative methods to inform benefit-risk decisions. Efficacy-safety tradeoffs also are critical in drug-development decisions. Choice-format conjoint-analysis surveys (also known as discrete-choice experiments) offer an empirical solution to estimating stakeholders’ willingness to accept such tradeoffs. Data on benefit-risk tradeoff preferences can provide metrics not previously available to decision makers to facilitate direct comparisons of efficacy and safety.
Conjoint analysis is based on eliciting trade-off preferences in response to a set of experimental stimuli. When a conjoint-analysis study obtains preference data on efficacy, treatment-related risks, and time durations, it is possible to derive seven benefit-risk metrics from the same dataset. These metrics include: 1) Minimum acceptable benefit for a given level of risk; 2). Maximum acceptable risk for a given level of benefit; 3) Net effectiveness margin; 4) Net safety margin; 5) Maximum acceptable number needed to treat; 6) Minimum acceptable number needed to harm; 7) Healthy-years equivalents. The workshop will present the conceptual rationale these metrics and illustrate their estimation using data from a general-population study of Alzheimer’s-disease (AD) treatment preferences. An exercise will enable workshop participants to assess their own benefit-risk tradeoff preferences for AD outcomes and compare their preferences with the general-population sample. This workshop is designed for policy makers, clinicians, researchers, patient advocates, students, and industry experts interested in acquiring new tools to inform benefit-risk decisions.
W5: SYMPTOM ASSESSMENT IN CLINICAL TRIALS – BRIDGING THE GAP BETWEEN EMEA AND FDA
Discussion Leaders: Ingela Wiklund PhD, Senior Research Leader, Center for Health Outcomes Research, United BioSource Corporation, London, UK; Olivier Chassany MD, PhD, Medical Leader, Département de la Recherche Clinique et du Développement, Assistance Publique - Hopitaux de Paris, Paris, France; Alastair Glendenning PhD, Health Economics and Outcomes Research, Novartis Pharma, Horsham, West Sussex, UK
PURPOSE: The objective of this workshop is to understand methods of demonstrating the value of symptom improvement using PROs, and the requirements of sponsors and regulators for the evaluation of symptoms. We will also share methods of implementing the collection of valid and reliable data to support treatment benefit claims relating to improvement in symptoms as discussed by the ISPOR SIG.
DESCRIPTION: Symptom assessments are the most commonly used Patient Reported Outcomes (PROs) and are used as a primary efficacy end-points for evaluating treatment benefit in symptomatic conditions such as pain, irritable bowel syndrome, and migraine. In other cases symptoms are key differentiating outcomes supporting treatment benefits. Improvement of symptoms is often proxy for improvement in functioning, feelings and quality of life in general. It is important to capture symptom from the patients’ perspective, using valid, reliable and culturally adapted tools. These tools are used in clinical trials conducted globally with the intent to submit new drug applications in both Europe and in the US. Not only do EMEA and FDA differ in the way they evaluate claims relating to treatment benefits based on symptom end-points, but the agencies also differ in their terminology and requirements relating to PROs. Presentations: 1) Symptom assessment: Value of evaluating symptom endpoints: labels and beyond - Alastair Glendenning, Novartis; 2) Symptom assessment: The regulatory perspective EU and US - Olivier Chassany; 3) Symptom assessment: Meeting regulatory requirements in international trials (including content validation-PRO issues) - Ingela Wiklund; Discussion – All
Use of Real World Data
W6: THE ROLE OF PATIENT REGISTRIES IN EVIDENCE DEVELOPMENT: SIMILARITIES AND DIFFERENCES BETWEEN EUROPE AND NORTH AMERICA
Discussion Leaders: Eric K Gemmen MA, Senior Director, Medical Affairs, Epidemiology & Outcomes Research, Late Phase & Safety Services, Quintiles, Inc, Falls Church, VA, USA; Yvonne Lis PhD, Director, Carter Lis Associates Ltd, Surrey, UK; Dimitris Polygenis PharmD, Vice President, McKesson Specialty, McKesson Canada, Toronto, ON, CA; Christopher M. Blanchette PhD, MS, MA, Associate Scientist and Director, Center for Pharmacoeconomic and Outcomes Research, Lovelace Respiratory Research Institute, Albuquerque, NM, USA; Chris L Pashos PhD, Vice President, Health Economic Research & Quality of Life Evaluation Services (HERQuLES), Abt Bio-Pharma Solutions, Inc., Lexington, MA, USA
Contributors: Shital Kamble MS, PhD, DCRI Research Fellow, Center for Clinical and Genetic Economics, Duke Clinical Research Institute, Durham, NC, USA; Gabriel Sandblom PhD, MD, Ward Physician, Dept of Surgery, University Hospital, Lund, Sweden; Sally Thompson, PhD MSc, Health Economics and Outcomes Research Director, Pfizer, Surrey, UK; Diana Frame MS, Consultant, Frame Research, Brooklyn, NY, USA; Carl J Gibbons BsC, (Psych), Research Analyst, Health Technology Assessment Group, Schering-Plough Ltd, Welwyn, Garden City, UK; Alex Exuzides PhD, Director, ICON Clinical Research, San Francisco, CA, USA
PURPOSE: This workshop will compare and contrast the role of patient registries in evidence development in Europe with that emerging in North America.
DESCRIPTION: It is widely accepted that the strongest form of evidence for the assessment of efficacy comes from randomized controlled trials. Observational studies have been relegated to the lower tiers in commonly used hierarchies of evidence, largely due to the heterogeneity of the real world they are assessing – physician/provider practice, patient characteristics and behaviour, various outcomes – and the resulting challenges in data interpretation. Nevertheless, observational studies, including patient registries, are an important part of evidence development regarding effectiveness, safety, and value in real-world settings. Patient registries can enhance knowledge of clinically heterogeneous populations, clinical practice and longer-term outcomes. Although emphasis on the comparative effectiveness of medical interventions is leading to a growing recognition of the value of registries by regulatory and reimbursement bodies, controversies remain in how best to design them to demonstrate effectiveness and how best to use the evidence they generate. Guidelines and use of patient registries by European agencies in influencing--or not--registration, coverage and reimbursement decisions will be explored and compared with similar actions by decision makers in North America. Key controversies will be discussed, such as prospective registration of observational studies, linking public and private health care databases, and the possible impact of personalized medicine. This workshop will reference the GRACE initiative, which is developing a set of good practice principles for the design, conduct, analysis, and reporting of observational comparative effectiveness studies. Audience input based on experience will be encouraged throughout the session, which will include an interactive discussion at the workshop’s conclusion.
*Discussion leaders and contributors represent the ISPOR Patient Registry Classification, Strategy & Design Working Group. A list of members is available on the ISPOR website.
WORKSHOPS - SESSION II
Sunday, 25 October 2009: 16:30-17:30
Clinical Outcomes Research
W7: HETEROGENEITY IN TREATMENT RESPONSE: WHAT IT IS, WHY IT'S IMPORTANT AND HOW TO FIND IT
Discussion Leaders: Alastair Glendenning PhD, Health Economics and Outcomes Research, Novartis Pharma, Horsham, West Sussex, UK; Kathleen W. Wyrwich PhD, Senior Research Scientist, Center for Health Outcomes Research (CHOR), United BioSource Corporation, Bethesda, MD, USA; Donald Stull PhD, Senior Research Scientist, United BioSource Corporation - Europe, London, UK
PURPOSE: This workshop is designed for researchers who are familiar with statistical analysis of clinical trial and observational data, but who are not necessarily experts in classification methodology. The purpose will be to introduce the concept of heterogeneity in treatment response, describe why it is important from the perspective of patients, providers, payors and manufacturers and outline two innovative methods to identify heterogeneous groups of treatment responders.
DESCRIPTION: Heterogeneity in treatment response is often encountered in clinical research outside the context of pre-defined subgroups. Analytic methods are now available to identify individuals who are more or less responsive to treatment in clinical trials and observational studies. These individuals, once identified, can then be assessed to see if they have common characteristics that may shed light on their differential response to treatment. Identifying heterogeneity in treatment response may play a key role in the development of truly “personalized medicine.” Responder thresholds can be determined through a selection of anchor-and distribution-based methods; once established, patient change over time can be classified as: improved, about the same, and declined. Subsequently, logistic and polytomous regression techniques can identify demographic and clinical characteristics associated with the responder classification membership. A second approach uses factor mixture model (FMM) methodology, which is designed for data that appears to involve heterogeneous populations but may include several latent classes that are homogeneous. FMM combines latent class analysis and common factor analysis. Mixture models are a good choice if it is reasonable to assume that observed variables within each class can be modelled using a common factor model. Following the presentation, the panel will engage the audience in a discussion on the importance of these methods, their strengths and their limitations.
Economic Outcomes Research
W8: ETHICAL IMPLICATIONS OF RESOURCE ALLOCATION IN SITUATIONS OF EXTREME SCARCITY, AS IN PANDEMIC FLU – IS THERE A ROLE FOR THE QALY?
Discussion Leaders: Peter Kolominsky-Rabas MD, PhD, Managing Director, Interdisciplinary Center for Public Health Studies, University of Erlangen-Nuremberg, Erlangen, Germany; Georgia Mitsi PhD, MBA, MSc, Manager, United BioSource Corporation, Lexington, MA, USA; J. Jaime Caro MDCM, FRCPC, FAC, Senior Vice President, Health Economics, Health Care Analytics, United BioSource Corporation, Lexington, MA, USA
PURPOSE: Ideally, every individual in need of medical care would receive the best available. When demand massively exceeds supply – during pandemic flu or massive terrorist attack, for example – difficult decisions must be made regarding allocation of available resources. Despite considerable stress and pressure, this rationing should be ethical. Recently, it has been proposed that the QALY be used as the arbiter. This workshop will address two issues: What are the ethical approaches to resource allocation in this setting? What are the implications of this for prioritization in less extreme situations, such as routine reimbursement decisions?
DESCRIPTION: To better understand the issues involved in resource allocation in situations of extreme scarcity, two scenarios will be described: pandemic flu and a nuclear terrorist attack. Several approaches will be presented, including the recent proposal to use QALY gains, and their implications will be discussed, in relation to the basic first-come, first-served, taking efficiency into account (i.e., avoiding the waste of resources). The ethics of denying treatment to individuals based on specific considerations (e.g., disease severity, individual characteristics, the person’s role in society, life expectancy, and even the expected QALY gain) will be discussed. The idea of “ethical permissibility” will be introduced: As scarcity deepens, efficiency considerations become more important and egalitarianism less so; uncertainty drives this in the opposite direction. The workshops will discuss these issues in light of the two scenarios, while recognizing the gap in the perspectives of the individual practitioners and the system-wide decision makers. The learnings from the extreme context will be juxtaposed with the current use of the QALY to prioritize across the health care system in more routine situations.
Health Care Policy Development Using Outcomes Research
W9: FROM THE BEDSIDE TO THE CABINET: THE VALUE OF STANDARDISED HRQOL MEASUREMENT IN QUANTIFYING POPULATION HEALTH
Discussion Leaders: Paul Kind, Professor, Outcomes Research Group, University of York, York, UK; Xavier Badia MD, PhD, Principal, Health Economics and Outcomes, IMS Health, Barcelona, Spain; Federico A Augustovski MD, MSc, Director, Health Economic Evaluation and Technology Assessment, Instituto de Efectividad Clínica y Sanitaria (IECS), Buenos Aires, Argentina; Stefan Björk PhD, Adjunct Professor, University of Lund, R.R. Institute of Applied Economics, Malmö, Sweden
PURPOSE: The objective of this workshop is to explore the additional benefits that are to be derived from using a standardised measure of health-related quality of life (HrQoL) in applications relevant to a wide range of decision-making settings, including the development of health policy, monitoring performance at the macro and micro level within healthcare systems, gauging productivity and other indices of change, measuring the extent to which the expected benefits resulting from programmes of healthcare are in fact delivered, in post-marketing surveillance of new therapies. In short, a range of applications that is limited only be our imagination and creativity. Guidance for HTA practitioners has been published in many countries, stipulating the requirements for HrQoL measures used in economic evaluation. This guidance can sometimes be seen as unnecessarily restrictive for many non-economic applications. There are a limited number of instruments which could be useful in this pivotal role but material presented in this workshop will focus predominantly, although not exclusively, on EQ-5D data generated in several national surveys.
DESCRIPTION: Workshop leaders will present population-based survey data from several different countries (Argentina, Spain, Sweden and the UK).exemplifying key areas of potential information gain, including the measurement of disease burden, construction of normative reference tables for significant population subgroups, benchmarking provider unit performance using real world data and establishing counterfactual baseline data for economic evaluation. Participants in the workshop will be actively encouraged to contribute to these presentations by offering their own assessment/interpretation of data exhibits. It is anticipated that this workshop will provide a focal point for ISPOR delegates who have access to patient information systems or to population-based survey tools with the expectation that they can collectively provide guidance for other interested parties. A written record of the workshop proceeding will be circulated to attendees after the event.
Patient-Reported Outcomes/Preference-based Research
W10: STATISTICAL METHODS TO HANDLE INCOMPLETE LONGITUDINAL PATIENT-REPORTED OUTCOMES DATA IN ADVANCED DISEASE STAGE TRIALS
Discussion Leaders: Dawn Odom MS, Director, Biostatistics, RTI Health Solutions, Research Triangle Park, NC, USA; Lee Bennett MS, Research Statistician, RTI Health Solutions, Research Triangle Park, NC, USA; William D. Irish PhD, VP Outcomes Research and Biostatistics, CTI Clinical Trial and Consulting Services, Raleigh, NC, USA; Deirdre Mladsi BA, Global Head, Pricing and Reimbursement, RTI Health Solutions, Research Triangle Park, NC, USA
PURPOSE: In clinical trials of advanced-stage disease, missing patient-reported outcome (PRO) assessments due to patient withdrawal is often a concern because the reason for withdrawal may be associated with the outcome of interest. The workshop will review the conventional statistical techniques used to deal with missing observations and explore newer approaches that are designed to reduce potential bias from nonignorable missing data.
DESCRIPTION: Conventional approaches to deal with missing data, such as last-observation-carried-forward, may introduce bias since these approaches assume that observations are missing completely at random (MCAR). When patients drop out of a trial due to toxicity, disease progression, or death, the subsequent missing data cannot be viewed as such. Newer techniques such as mixed-effects models do not require the MCAR assumption and may be more appropriate than the traditional methods. The pattern-mixture model is a type of mixed-effects model that is particularly powerful for nonignorable missing data because observations are grouped according to patterns of missing values, and information about these patterns is incorporated into the statistical model. Other techniques such as multiple imputation and structural equation models will also be discussed. The workshop is targeted toward health outcomes researchers and others who are responsible for analyzing or interpreting patient-reported outcome data. We will use examples to illustrate how analyzing nonignorable missing data using conventional methods can lead to bias and misinterpretation and demonstrate how modeling approaches, specifically pattern-mixture models, provides more reliable results. The audience will be invited to discuss their experiences with informative missing PRO data and the statistical techniques used with particular attention to the advantages and disadvantages of each method.
W11: ASSESSING TREATMENT SATISFACTION DURING A PRODUCT'S LIFECYCLE TO FACILITATE MARKET ACCESS: DEFINITIONS, FRAMEWORKS, AND MEASUREMENT
Discussion Leaders: Diana Rofail MSc, Senior Project Manager, Mapi Values Ltd, Bollington, Cheshire, UK; Antoine Regnault PhD, Research Consultant, Statistics & Psychometrics, Mapi Values France, Lyon, France; Jean-Francois Baladi MBA, Executive Director, Health Economics & Pricing, Global Marketing Oncology, Novartis Pharmaceuticals Corp, Florham Park, NJ, USA; Gilles Berdeaux MD, Associate Director, Conservatoire National des Arts et Métiers, Paris, Hauts de Seine, France
PURPOSE: To show how the assessment of treatment satisfaction can demonstrate the 'added value' of a product from patients' perspectives by capitalising on product differentiation, providing a competitive edge, and ultimately facilitating market access and the product uptake of a pharmaceutical therapy.
DESCRIPTION: This workshop will consider reasons for conducting satisfaction studies and address conceptual issues related to treatment satisfaction including definition of the concept, and how satisfaction relates to expectations, preferences, and adherence to treatment regimens. The patients’ experience will be emphasised and associated factors such as medication characteristics, convenience, and presence and impact of side effects. The theoretical models framing satisfaction will be presented with empirical evidence supporting them. Implications on scientific research and clinical practice such as the choice of the assessment type (generic versus disease-specific or treatment-specific assessments), identification of appropriate time points to assess treatment satisfaction during a product's lifecycle, and designing studies (either as part of clinical trials or as independent cross-sectional or longitudinal studies), will be a focus of the discussion. Case-studies from various application fields (e.g. iron chelation therapy, anticoagulant treatment, and glaucoma treatment) will illustrate the definitions, conceptual frameworks, and measurement issues related to treatment satisfaction. This will include aspects related to research aims, study designs, development and validation of treatment satisfaction instruments, implications for protocols and statistical analyses, as well as interpretation of results. This will be an interactional workshop and participants’ contributions will be encouraged throughout. This session is directed at individuals who design and conduct outcomes research, and those in charge of interpreting study results, but will provide a useful overview for those who analyse outcomes data to support communicating key messages to local and national key stakeholders.
Use of Real World Data
W12: QUALITY ASSURANCE OF EVIDENCE FROM REAL WORLD DATA (RWD) FOR REIMBURSEMENT DECISIONS
Discussion Leaders: Diana Brixner RPh, PhD, Associate Professor, Department of Pharmacotherapy, The University of Utah College of Pharmacy, Salt Lake City, UT, USA; Daniel Malone PhD, RPh, Professor, Pharmacy Practice and Science, University of Arizona, Tucson, AZ, USA; Anke-Peggy Holtorf PhD, MBA, Business Director, Biobridge Strategies, LLC, Basel, UT, Switzerland
PURPOSE: Using results from RWD analysis for decision making requires the ability to assess the quality of the underlying research. Despite of increasing availability of various checklists and guidelines to evaluate these data only a minority of decision makers use any of the currently available quality assessment resources. In parallel, the skepticism of decision makers towards RWD persists due to a perception of inferior quality. The purpose of this workshop is to summarize the current status of RWD quality assessment globally, briefly review available resources, and to present possible tools to assist decision makers in quality assessment of RWD. The workshop will allow for reaction to a proposed consolidated ‘next generation’ tool, suggestions of alternatives and a weighting of options using an audience response tool.
DESCRIPTION: The current status of quality assurance in RWD will be evaluated through the presentation of two publications of a roundtable in the U.S. between researchers and decision makers on this topic. Evaluation in other global regions will be presented based on examples from European countries. This will be followed by a comparison of the currently available checklists and ISPOR good practice guidelines for the assessment of RWD by research and publication. This information will provide the foundation for the presentation of a potential consolidated tool to stratify checklists by category and purpose. The workshop component will allow input and discussion on these options and a weighting of the preferences for these approaches via an audience response tool. The objective of the workshop is to broaden the geographic perspective towards the issue and the proposed solution and to weight and to validate the relevance of the proposed action.
WORKSHOPS - SESSION III
Tuesday, 27 October 2009: 08:30-09:30
Economic Outcomes Research
W13: OPENING THE BLACK BOX: INFORMING MODEL CHOICE FOR HEALTH TECHNOLOGY ASSESSMENT IN CANCER TREATMENT
Discussion Leaders: Andrew Briggs DPhil, Professor, Public Health & Health Policy, University of Glasgow, Glasgow, UK; Mark Sculpher PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK; Neil S Hawkins PhD, Director, Oxford Outcomes (UK), Oxford, UK; Alison Griffiths Mph, Health Economist, Oxford Outcomes, Oxford, UK
PURPOSE: The goal of cost-effectiveness analysis is to develop a simplified version of reality, a model capable of capturing important cost and health differences between a new technology of interest and existing comparators. There are a number of modelling techniques available to analysts including decision trees, Markov models, semi Markov models, area under the curve methods and microsimulation. However, the rationale for choosing a model approach is not always clear. In this workshop we will discuss potential analytical frameworks with a view to selecting suitable approaches in cancer disease modelling. The purpose of this workshop is to explore the link between the clinical context and the relevant economic model structure to enable more informed choice in cancer disease modelling.
DESCRIPTION: The workshop is intended for those engaged in cancer HTA including decision makers, clinicians and modellers and will taken an interactive format. Discussion leaders will present an overview of model approaches. The audience will then be asked to choose a model structure appropriate for a cancer intervention example. This will be followed by a discussion and other cancer model examples. Potential issues associated with structure types and model calibration will be explored before a final summary and take home messages. Suggested workshop format: Introduction; Overview of model frameworks; Cancer intervention example; Which model structure for example – audience perspective; Which model structure for example – panel perspective; Discussion and other cancer model examples; Summary and take home messages.
W14: TRANSMISSION MODELING FOR ECONOMIC ASSESSMENTS OF VACCINES
Discussion Leaders: Julie Roïz MSc, Senior Analyst, i3 Innovus, Uxbridge, UK; Sibilia Quilici MSc, Health Economic Manager, Sanofi Pasteur MSD, Lyon, France; Samuel Aballéa MSc, Senior Lead Analyst, i3 Innovus, Nanterre, France
PURPOSE: Several new vaccines for infectious diseases have recently been developed; with this trend has come an increasing need for modelling the cost-effectiveness and budgetary impact of vaccination programs. Ignoring indirect protection effects of such programs (“herd immunity”) is sometimes a conservative, acceptable approach, but this is not always appropriate. This workshop will provide insight into the dynamics of infectious diseases and implications for economic analyses of prevention programs.
DESCRIPTION: We will present the challenges raised when modelling the impact of vaccination policies, based on a review of published economic evaluations. Basic concepts (e.g., force of infection, basic reproduction number, and herd immunity threshold) and methods (e.g. compartmental models, differential equations) for infectious disease modelling will first be introduced. Specific effects of vaccination programs will then be presented through interactive examples to justify why such programs should be modelled in a dynamic fashion. Herd immunity, the importance of mixing patterns in the transmission of the disease, the potential negative effect of under-vaccinating a population and the possible advantages of targeting small subgroups will be illustrated. As practical examples, we will present simple transmission models, and explore the impact of incorporating these specificities on incremental cost-effectiveness ratios (ICER) and budget impact. We will examine in particular the relationship between vaccination coverage and ICER. Participants will be asked to share their experiences in developing models for evaluating vaccines. It is expected that at the end of the workshop participants will be aware of the positive and negative externalities of vaccination and understand in what situations transmission dynamic models are required in health economics.
Health Care Policy Development Using Outcomes Research
W15: DESIGNING RISK-SHARING SCHEMES: OPPORTUNITIES, PITFALLS AND PRELIMINARY MODELLING
Discussion Leaders: Olivier Ethgen MSc, PhD, Ass. Prof., Department of Public Health Sciences, University of Liège, Liège, Belgium; Ulf Staginnus MA (Economics), Head, Pricing & Health Economics, Novartis Oncology, Region Europe, Barcelona, Spain
PURPOSE: Under continuing economic pressure, cost-effectiveness appraisals are broadly applied to vouch for good value-for-money of new pharmaceuticals. Though, risk-sharing schemes (RSS) are increasingly being considered as a proactive means to ensure market access. This emergence is mainly motivated by the uncertainties in ex-post cost and effectiveness and/or by international price referencing issues. RSS are not widely described in the published literature and evidence on their effects is even sparser. However, RSS are expected to multiply rapidly as a strategy to access more and more stringent markets. The aim of this workshop is to provide the audience with a review of opportunities, pitfalls and modeling approaches in designing RSS.
DESCRIPTION: After a brief overview of the different schemes settled hitherto and publicly disclosed, the workshop will suggest some definitions and descriptions of different types of schemes. The promises of RSS as an innovative market access tool will be discussed. Next, the pitfalls in setting up and running a RSS will be debated. A review of the few modelling approaches considered so far will be provided. In the final section, participants will be invited to react interactively on the workshop’s content and to share their potential lessons learned in designing and running RSS. During the discussion, emphasis will be put on modelling methods in order to aid in the designing of efficient schemes that would benefit all parties at stake i.e., patients, clinicians, payers and manufacturers.
Patient-Reported Outcomes/Preference-based Research
W16: ASSESSING PATIENT ADHERENCE FOR A POSITIVE CHANGE IN HEALTH BEHAVIOUR
Discussion Leaders: Carla Dias Barbosa MSc, Research Manager, Mapi Values France, Lyon, France; Linda Abetz MA, Director - PRO, Mapi Values, Bollington, UK; Francois-Emery Cotte PharmD, MPH, Health Outcomes Researcher, Health Outcomes Studies, GlaxoSmithKline France, Marly le Roi, France; Benoit Arnould PhD, Director, France, Mapi Values, Lyon, France
PURPOSE: Discuss the added value of evaluating adherence to optimise patient management.
DESCRIPTION: Adherence is recognised as a primary determinant of the effectiveness of treatment; poor adherence has direct consequences such as disease complications, impact on patients’ quality of life and increased use of health care resources. Social and economic impact of poor adherence can be great in chronic diseases. With increasing “risk-sharing” requirements by local authorities and payers, adherence becomes an ever-more important issue for pharmaceutical industry. However, adherence is a complex and multifaceted concept that is challenging to understand, measure and improve. Accurate measurement of adherence is nevertheless necessary for efficient assessment of health care interventions and positive change in patient behaviour is critical for optimal management. This workshop is composed of four parts. The first part will define adherence, as opposed to other similar concepts, and will describe the behavioural mechanisms explaining adherence. The second part will demonstrate the social and economic impact of poor adherence and the benefits for pharmaceutical industry of improving adherence to treatment. Third, different methods for measuring adherence will be described and the consistency of these methods discussed. Finally, methods for improving patient adherence will be presented, including development of interactive tools to identify reasons for lack of adherence and physician-patient educational material for use in clinical practice. Both aim to change patients’ illness perception and behaviour. This workshop will address these different points using examples from the presenters’ experiences and from the literature. In particular, the development of specific PRO tools for assessing or improving adherence will be presented and discussed. Participants (academics, policy makers, researchers from the pharmaceutical industry and health-care programmes) will be invited to debate how to improve patient behaviour in order to optimise disease management.
W17: ELECTRONIC CAPTURE OF PROS - EPROS – STATUS TODAY AND TOMORROW?
Discussion Leaders: Tara Symonds PhD, Senior Director, Outcomes Research, Global Market Access, Pfizer Ltd, Sandwich, Kent, UK; Ingela Wiklund PhD, Senior Research Leader, Center for Health Outcomes Research, United BioSource Corporation, London, UK; Alastair Glendenning PhD, Health Economics and Outcomes Research, Novartis Pharma, Horsham, West Sussex, UK; Lucy Abraham MSc, CPsychol, Associate Director, Outcomes Research, Pfizer Ltd, Sandwich, Kent, UK
PURPOSE: The objective of this workshop is to understand benefits and drawbacks involved when using ePROs, how to select the most appropriate EDC platform on relation to study design and type of PRO end-point, methods to demonstrate the value of ePROs, the requirements for further documentation when moving from paper to ePROs as discussed by the ISPOR ePRO Task Force. We will also share the feedback from regulatory agencies regarding their view of ePROs in clinical trials.
DESCRIPTION: The use of ePROs when assessing Patient Reported Outcomes (PROs) in clinical trials is increasing rapidly. Additionally, mixed modes of administration are being employed for reasons driven by practical and logistical needs when trials are moving into non-traditional countries. Whilst daily prospective symptom assessments are commonly captured on PDAs or using IVRS, clinic visit completion of PROs may require another technology. Despite the many benefits of using ePROs comparatively few studies actually use this mode of administration, and this seems to be more commonly the case outside North America. Presentations: 1) Why ePROs? Benefits, drawbacks, whys and when - Alastair Glendenning, Novartis; 2) Moving from paper to ePRO – What type of documentation is required? - Ingela Wiklund, UBC; 3) Industry perspective - Experience from trials and from agency interactions - Lucy Abraham, Pfizer; Discussion – All.
Use of Real World Data
W18: USING SIMULATION TO BOOST EPIDEMIOLOGICAL STUDIES: IT SAVES THE DAY
Discussion Leaders: Georgia Mitsi PhD, MBA, MSc, Manager, United BioSource Corporation, Lexington, MA, USA; Bernd Bruggenjurgen MD, MPH, Head, Alpha Care, Celle, Germany; J. Jaime Caro MDCM, FRCPC, FAC, Senior Vice President Health Economics, Heatlh Care Analytics, United BioSource Corporation, Lexington, MA, USA
PURPOSE: Many countries in Europe now demand, as a condition of coverage, that the manufacturer supply additional data to demonstrate that the product is correctly used in appropriate populations consistent with the indication and cost-effective subgroups. Collecting data on tens of thousands of patients to meet this condition is not very practical, especially if it must be done in multiple countries or regions. SAVES (Simulation Added-Value Epidemiological Study) is a cost-effective methodology which can supply the required information at a fraction of the effort. In this workshop, we will present this novel approach and discuss its advantages and limitations.
DESCRIPTION: To boost the power of an epidemiological study, a simulation is designed initially to reflect all the information available at the time of the health technology assessment and coverage decision, including the particularities of the country or region at issue, for as close as possible approximation to real world scenarios. This initial simulation is extensively analyzed to identify the degree of variation that is likely and the areas where additional information would contribute the most to reducing uncertainty. Targeted epidemiologic studies are then designed to obtain the informative data. As these seek very specific element, they can be much smaller and focused. Nevertheless, as the data will take some time to accrue, several updates of the simulation can be built into the process to revise the projections. This approach can be taken further into a full adaptive Bayesian design.
The SAVES methodology can provide accurate estimates of the public health and economic impact of using a particular intervention in a given country, thus promoting well-informed decisions.
WORKSHOPS - SESSION IV
Tuesday, 27 October 2009: 14:45-15:45
Economic Outcomes Research
W19: MODELING SLOWLY PROGRESSING CHRONIC DISEASES WITH EXACERBATIONS AND THEIR TREATMENTS: CHALLENGES IN MODELING CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD)
Discussion Leaders: Maureen Rutten-van Molken PhD, Associate Professor, Health Economics, Institute for Medical Technology Assessment, Erasmus University and Erasmus MC, Rotterdam, The Netherlands; Talitha Feenstra PhD, Senior Health Economist RIVM and Assistant Professor HTA, Department of Epidemiology, RIVM and Department of Prevention and Health Services Research, University Medical Center Groningen, Bilthoven, The Netherlands; Sixten Borg MSc, Senior Project Manager, The Swedish Institute for Health Economics (IHE), Lund, Sweden
PURPOSE: Understanding the natural progression of a chronic lung disease, both in men and in women, smokers and non/ex-smokers, is essential to interpreting the cost-effectiveness of any therapeutic intervention. Decision-analytic mathematical models are useful tools to simulate disease progression and synthesize evidence on health consequences and costs of different treatments in order to estimate cost-effectiveness. At least five COPD disease-state models have been published, which differ with respect to both structural assumptions and parameter estimates. Some are population-based models that include the incidence of COPD, others are disease progression models starting after COPD has been diagnosed. Some model exacerbations as events, others as disease-states. Some allow back-ward transitions, others do not. Furthermore, considerable differences exist between the models with respect to estimates of disease progression, exacerbation probabilities, utility values, and costs. Part of these differences may reflect differences between patient populations, while others are due to differences in the empirical data used, or the way these data were synthesized. Moreover, despite the increasing evidence that COPD is a systemic disease affecting many organs besides the lungs all models still use lung function decline to model disease severity and disease progression. The purpose of this workshop is to systematically discuss these issues.
DESCRIPTION: More specifically, the aim of this workshop is threefold. First, to compare and discuss the structural assumptions of the existing COPD models. Secondly, to discuss different data sources available to estimate disease progression (e.g. Lung Health Study, Framingham Heart Study), baseline exacerbation rates (e.g. meta-analysis of placebo arms of trials, OLIN data, BOLD data), case-fatality rates, utilities and costs by disease severity. Thirdly, to discuss different methods of fitting models on cross sectional studies that report data by disease severity class, which is important given the limited availability of longitudinal data.
W20: DEVICES & STRATAGEMS: HOW CAN HEALTH ECONOMIC EVIDENCE BE LEVERAGED IN ORDER TO DEMONSTRATE THE VALUE OF MEDICAL DEVICES?
Discussion Leaders: Daniel Jackson MSc, Head of Health Economics - EMEA, GE Healthcare, Chalfont St. Giles, Buckinghamshire, UK; Alison Begg PhD, Senior Consultant, Medaxial Group, London, UK; Adam Plich MSc, Consultant, Medaxial Group, London, UK
PURPOSE: To discuss the particular challenges of demonstrating product value in the medical devices industry.
DESCRIPTION: What constitutes economic evidence in the context of medical devices? What difficulties arise when evidence from clinical trials cannot practically be obtained? What specific challenges are raised when estimating the value of items with a high capital cost? How is the picture complicated by the rapid speed of technological change? Are there common challenges and solutions that apply across the industry, encompassing everything from disposable drapes to million-dollar scanners? This workshop will explore the issues involved in generating and leveraging economic evidence in the medical devices industry. A real-life case example will be presented to show how some of the challenges can be addressed. Questions, comments and discussion of alternative solutions will be encouraged.
Health Care Policy Development Using Outcomes Research
W21: NEW HEALTH CARE SERVICES – THE PATHWAY INTO THE GERMAN SICK FUND MARKET
Discussion Leaders: Olaf Pirk PhD, MD, Principal, Health Economics & Outcomes Research, IMS Health, Nuremberg, Germany; Axel C. Mühlbacher PhD, MSc, Professor, IGM Institut of Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany; Frank-Ulrich Fricke PhD, Principal, IMS Health, Nuremberg, Germany
PURPOSE: The objective of this workshop is to demonstrate how new health care services (e.g. devices, drugs etc.) might enter the German health care market and get reimbursement by the statutory health insurances.
DESCRIPTION: About 90% of the German population is members of the statutory health insurance (SHI). The rest of them are privately health insured. This makes the population of SHI-insurants the larger and therefore the more attractive market than the privately insured persons. In addition, this market is regulated more clearly, as there are mostly collective contracts which apply in the same way for all SHIs. The specification of a new health care service must be listed within the so called “service catalogue” is crucial to gain reimbursement. If the specifications are not included, the ‚Federal Joint Committee’ (Gemeinsamer Bundesausschuss, G-BA) will have to assess and confirm benefit, need and economic efficiency of this innovation in a consultation process. Only then the service can be included into the outpatient benefits catalogue and can be reimbursed on a regular basis (permission reservation, § 135 Abs. 1 SGB V). If the innovation is a therapeutic appliance, the funding by the SHI will only be possible when the product is listed in the ‚German therapeutic appliance register’ led by the “Head association of the statutory health insurance scheme” (“GKV-Spitzenverband”). Overall, the attendees will develop a notion on how to cope with the market access process at an early stage within product development and to continuously consider the requirements of the G-BA and other institutions of the so called self administration in the German health care system on data during the development process of their respective product. Furthermore it will be discussed with the attendees which method for preference setting within the market access process should be preferred.
Patient-Reported Outcomes/Preference-based Research
W22: METHODS OF POOLING DATA FROM PATIENT-REPORTED OUTCOME (PRO) MEASURES FROM GLOBAL TRIALS: IMPLEMENTING RECOMMENDATIONS OF THE ISPOR TASK FORCE
Discussion Leaders: Asha Hareendran PhD, Senior Research Scientist, Health Care Analytics, United BioSource Corporation, London, UK; Angela Williams BSc, RGN, Pricing and Reimbursement Manager, ViroPharma Europe, Maidenhead, Berkshire, UK; Tara Symonds PhD, Senior Director, Global Market Access, Outcomes Research, Pfizer Ltd, Sandwich, Kent, UK
PURPOSE: The purpose is to discuss practical methods of implementing recommendations of the ISPOR PRO Translations Task Force for pooling data from patient-reported outcome (PRO) measures from global trials.
DESCRIPTION: Clinical trials to evaluate treatment benefit of new health technologies are now being conducted across the globe to facilitate faster recruitment. Regulatory authorities such as the EMEA and FDA have queried whether the linguistic translation process for patient-reported outcome (PRO) measures guarantees that they will perform with an acceptable level of similarity across populations to allow pooling of data. The ISPOR Patient-Reported Outcomes (PRO) Translation and Linguistic Validation Task Force recently published its recommendations and encourages scientists to apply a wide variety of quantitative and qualitative approaches to assess measurement equivalence to support pooling data. In reality, the sample sizes for PRO data from clinical trials for each language may be insufficient to conduct some of the methods suggested in the ISPOR Task Force report. The Moderator will introduce and briefly discuss the scientific recommendations of the Task Force, and the first panelist will present through examples the methods used to assess equivalence of a PRO instrument using data from a large multi-country trial. The second panelist will discuss practical considerations from the industry perspective (e.g., sample sizes, complexity of PRO endpoint symptoms vs. HRQL, using country as co-variate). The session will conclude with an interactive session to further discuss the implications when sponsors move more and more trials into non-traditional countries and practical solutions to address requirements of EMEA and FDA for pooling data from PRO tools and for requesting further psychometric documentation.
Use of Real World Data
W23: THE EMERGING ROLE OF OBSERVATIONAL DATA IN EVALUATING PRODUCT VALUE AND PRODUCT SAFETY
Discussion Leaders: Jerome Wilson PhD, Senior Scientific Affairs Director, PRA, Rockville, MD, USA; Thomas F Goss PharmD, Vice President, Boston Healthcare Associates, Inc., Washington, DC, USA; Mark R Vanelli MD, MHS, MBA, Chief Medical Officer, Adheris Inc, Burlington, MA, USA
PURPOSE: The purpose of this workshop is to improve understanding of the strengths, value and limitations of observational data in assessing effectiveness and safety of treatments in real-world use.
DESCRIPTION: Numerous stakeholders (payers, providers, policymakers and manufacturers) are increasingly reliant on the use of evidence-based data to evaluate the safety and effectiveness of therapies that reflect conditions of real-world use. Manufacturers frequently include outcomes-based endpoints in investigational trials, but such endpoints have limited generalizability to routine practice because of differences in the patient populations, illness severity, duration and intensity of follow-up and spending levels that separate clinical trial data from clinical practice experience. Opportunities to create risk evaluation and mitigation strategies (REMS) that integrate data on patient adherence, health economics and outcomes research are enabled in this new environment as patients, providers, payers, policymakers and manufacturers attempt to better assess the quality and cost of care that is actually delivered. While observational designs have limitations, they can be used to generate hypotheses and to assess a number of potential endpoints and to simply describe patterns of care that are delivered; the best such programs usually have a defined (a priori) goal and purpose. In this session, 3-4 speakers will address: 1) The opportunities associated with implementing a REMS program to assess endpoints beyond safety 2) Decision-maker needs for data that can benchmark emerging therapies relative to existing standards of care 3) The patient groups, medication classes, and points in time when medication non-persistence is most likely 4) Strengths/limitations of observational data and the operational considerations that need to be considered when implementing an observational study. Specific case studies will address how data can be used to address key questions from the provider and payer perspectives.
W24: POST-REIMBURSEMENT STUDIES ASSESSING GOOD MEDICATION USE IN REAL PRACTICE: FRENCH SITUATION
Discussion Leaders: Emmanuelle Préaud PharmD, MSc, Research Manager, Health Outcomes and Market Access, Mapi Values France, Lyon, France; Juliette Longin PhD, Msc, Associate Director, Scientic Expertise and Proposal Development, Registrat-Mapi, Lyon, France; Stéphanie Tcherny MD, MSc, Responsable Recherche en pharmacoéconomie et pharmacoépidémiologie, Unité Pharmacoépidémiologie - Département médical, Lilly France, Suresnes, France; Marie-Christine Woronoff-Lemsi PharmD, PhD, Professor, Hospital Pharmacist Doctor, CHU de Besançon, Jean Minjoz Hospital, Besançon, France
PURPOSE: The purpose of this workshop is to present and discuss studies assessing compliance and real-life practice, focusing mainly on the way such studies should be conducted. Examples are based on studies requested by the French Transparency Committee following reimbursement decisions.
DESCRIPTION: French Haute Autorité de Santé (HAS) reimbursement decisions are increasingly completed by real-life data requests. Hundreds of requests have been addressed, focusing on outcomes like impact on the healthcare system, real-life tolerability, and especially real-life conditions for drug prescription and use in addition to real benefits to patients. These data on prescription and especially patients’ experiences and compliance are essential to assess the true benefit provided by new pharmaceutical products. They are generally requested for reimbursement status re-evaluation, to complete information from clinical development programs with evidence from real practice. One of the main challenges in addressing these requests is how to collect valid and reliable information to reflect real-life practice and impact of a new drug. Key data sources are patients and physicians, but many discussions are currently underway regarding the best way to assess such outcomes. Despite the significant number of requests, HAS has issued no clear recommendations on how these studies should be conducted. Some studies use either existing studies or bibliographic references, while some pharmaceutical companies try to develop and conduct ad-hoc studies. The aim of this workshop is to propose a framework to provide relevant answers to HAS’s real-life data requests. The starting point will be the analysis of existing studies provided to the HAS, thoroughly appraising the way they have been performed using leaders’ expertise in patient-reported outcomes. Leaders will also discuss their experiences with two study protocols recently accepted by the HAS, in addition to the audience’s own experiences.
WORKSHOPS - SESSION V
Tuesday, 27 October 2009: 16:00-17:00
Economic Outcomes Research
W25: USING EARLY ECONOMIC MODELLING TO INFORM STRATEGIC PRICING AND CLINICAL STUDY DESIGN
Discussion Leaders: Adam C Lloyd MPhil, Senior Principal, Health Economics and Outcomes Research, IMS Health, London, UK; Michael Chambers MSc, Director of Health Economics, Global Health Outcomes, GlaxoSmithKline, Uxbridge, UK; Joe Caputo BSc, Engagement Manager, Health Economics and Outcomes Research, IMS Health, London, UK
PURPOSE: The audience will be introduced to the application of economic modelling during early stage clinical development, and will review opportunities and challenges applying the findings of such models to strategic planning and study design.
DESCRIPTION: Value for money arguments are frequently required by agencies regulating market access. Products that can demonstrate attractive cost-effectiveness will therefore experience fewer challenges with market access than products that cannot. Despite data scarcity, economic modelling can be conducted at an early stage of drug development, based on a target product profile or a series of scenarios. Cost-effectiveness information generated prior to starting phase III studies is potentially useful to inform commercialisation decisions including “go/no-go” decisions, product positioning, indication sequencing, strategic pricing and clinical study design. Workshop participants will be introduced to analyses based on the economically justifiable price (EJP), defined as the highest price at which a product meets criteria for acceptable value for money. Using worked examples, we will explore how EJP is estimated, and how sensitivity analyses considering target patient group definition, dose, comparators and study endpoints can inform design of studies that optimise EJP. In addition we will show how sensitivity analyses considering disease processes, variation in cost-effectiveness thresholds, management costs and costs of events may contribute to prioritisation of research activities outside the clinical trial programme. We will review the opportunities and challenges of using EJP to engage internal stakeholders and influence decision making within the pharmaceutical industry with the ultimate aim of achieving successful launch and market access for products in development. Participants will work through an anonymised example, based on a real product, of using EJP to refine product strategy and influence study design, and will be invited to contribute to discussion of the challenges and opportunities of applying EJPs in practice.
W26: PRAGMATIC SELECTION OF APPROPRIATE MODELLING APPROACH IN PHARMACOECONOMIC EVALUATION
Discussion Leaders: Sandrine Cure MSc, Project Leader, i3 Innovus, Uxbridge, Middlesex, UK; Lee Moore MSc, Health Economist, Roche Products Limited, Welwyn Garden City, Hertfordshire, UK; David Thompson PhD, Vice President, Global Health Economics, i3 Innovus, Medford, MA, USA
PURPOSE: The purpose of the workshop is to explore the impact of choice of modelling approach on cost-effectiveness results and provide pragmatic advice on selecting the most appropriate method.
DESCRIPTION: In recent years, the diversification and increased complexity of the modelling techniques in pharmacoeconomics have raised the question of how to select the most appropriate modelling approach when conducting a cost-effectiveness analysis. Though there are many workshops and short courses on how to use the traditional modelling techniques (decision tree and Markov model) and advanced techniques (patient-level simulation), advice on the pragmatic selection of appropriate models is lacking. In this workshop, we will provide practical guidance on selecting the right model using the most widely employed methodologies in cost-effectiveness analysis such as 1) decision-analytic models; 2) state-transition models; and 3) patient-level simulation models, including discrete-event simulation. Using real-world modelling examples, we will illustrate the respective strengths and limitations of each model type by contrasting their results when applied to the same disease and interventions. The influence of the disease (acute, chronic or infectious) on the modelling approach will be illustrated through examples. Furthermore we will indicate the effect of model choice on criteria related to disease type; required transparency; data requirements for parameter estimation; adaptability to multiple settings and/or users; need for specialised software; and others. Other aspects such as development time, budget and data availability will also be discussed using real life examples. Participants will be encouraged to share their experiences.
Health Care Policy Development Using Outcomes Research
W27: VALUE-BASED PRICING OF NEW DRUGS IN JAPAN USING THE PRINCIPLE OF INCREMENTAL COST-EFFECTIVENESS RATIO
Discussion Leaders: Isao Kamae MD, DrPH, Professor, Graduate School of Health Management, Keio University, Fujisawa, Kanagawa, Japan; Makoto Kobayashi MEng, Manager, Health Economics Research Group, Crecon Research and Consulting Inc, Shibuya-ku, Tokyo, Japan
PURPOSE: The new pricing and reimbursement methods for drug approval are under discussions in the Japanese government. Despite no official requirements for pharmacoeconomic evidence in Japan, the proposed draft aiming at a reform of the current pricing system mentions narrative phrases suggesting potential applications of the cost-effectiveness methodology. In this workshop, we will identify various potentials for pharmacoeconomic applications in the proposal, and then present suggestive formula for pricing which is specific to Japan. Our objective is the first to demonstrate a local case-study of value-based pricing and further to discuss external applicability of our method to the other countries seeking methods for capturing the value for money.
DESCRIPTION: First of all, we introduce a reform of Japanese pricing system for new drugs. Then the reform draft proposed in the government is carefully reviewed in terms of pharmacoeconomics. Accordingly it clarifies the points to where pharmacoeconomic approaches should be applied. In particular, two policies are identified as the keys associating an incremental cost-effectiveness ratio (ICER) with the new rules, which include being allowed 1) to compare the new drug with an appropriate comparator and 2) to add a premium of price according to the degree of innovation proven by the new drug. The key relation is quantified, beginning with the principle of ICER and resulting in the formula for pricing. The details will be described using examples. Also, given the ICER’s nature of relativity to the comparator, a question on how we could choose a right comparator will be raised and discussed. It especially focuses on the method of efficiency frontier implemented by IQWiG in Germany. The presenters look forward to a vigorous debate on what impact our methods will have upon the other countries beyond the local issues in Japan.
Patient-Reported Outcomes/Preference-based Research
W28: RECENT PROGRESS ON BEST-WORST SCALING: HEALTH-RELATED APPLICATIONS
Discussion Leaders: Terry N Flynn PhD, Senior Research Fellow, Centre for the Study of Choice (CenSoC), University of Technology Sydney, Sydney, NSW, Australia; Jordan J Louviere PhD, Professor, Centre for the Study of Choice (CenSoC), University of Technology Sydney, Sydney, NSW, Australia; Emma McIntosh PhD, Senior Research Officer, Health Economics Research Centre, University of Oxford, Oxford, Oxfordshire, UK
PURPOSE: Discrete choice experiments (DCEs) / conjoint analyses usually lack detailed insights into decision rules used by individual patients, but a relatively new approach, Best worst scaling (BWS), can do this. BWS is consistent with random utility theory; hence can produce valuation measures consistent with economic theory. This workshop has two aims: (1) review recent developments in three types of BWS, (2) discuss applications of BWS to estimate individual respondent preferences and patient reported outcomes.
DESCRIPTION: Case 1 is the classical “maximum-difference” scaling case introduced by Finn & Louviere (1992, Jl of Public Policy & Mark). We discuss several key issues arising in applications, and illustrate it with two empirical applications: a) measuring attitudes of Australian farmers and b) local retailer choice. Case 2 involves choices of attribute levels within DCE/conjoint profiles. Each profile is a choice set, and the choice options are the profile attribute levels. We discuss several issues arising in Case 2 applications, and illustrate it with two empirical applications: a) choice of dental appointments, and b) measuring preferences for quality of life. Case 3 is closest to a traditional DCE: participants not only choose the most preferred (best) profile in each choice set; they also choose the least preferred profile in each of a number of sets of three or more. Compared with “first choice” elicitation, one obtains much more information about preferences from each set. We show how to estimate choice models for single persons applied to mobile phone choices and dermatology appointments. Finally, we will discuss BWS’s ability to (1) estimate individual patient utility functions for patient reported outcomes (PROs) and (2) identify patient subgroups whose preferences for health-related quality of life make them most likely to benefit from new treatments.
Use of Real World Data
W29: PRACTICAL APPLICATIONS OF OBSERVATIONAL STUDIES IN TODAY'S MULTI-FACETED, PERI-APPROVAL ENVIRONMENT
Discussion Leaders: Matthew Gordon BA, Director, Lifecycle Sciences Group, ICON Clinical Research, Chicago, IL, USA; Ehab Hasan MPH, Project Manager, Lifecycle Sciences Group, ICON Clinical Research, Chicago, IL, USA; Zaher El-Assi BA, Global Senior Director, Kika Medical, Boston, MA, USA
PURPOSE: Through careful operational and technological planning, observational studies have the capacity to significantly impact a product’s development and approval. We discuss here how some of these facets have been successfully implemented through the conduct of a global, non-interventional research study.
DESCRIPTION: The increased acceptance of prospective observational studies as scientifically sound vehicles for epidemiologic research and their ability to collect, in an unobtrusive manner, varying patient-reported and clinical data makes these studies ideal for supporting products in the peri-approval process. Leveraging sound project operations with technical support serves to strengthen this ability. Designing an observational study around a validated EDC system and augmenting data collection with for cause on-site monitoring can provide a cost-effective alternative to a Phase IIIb or IV study. Such data can simultaneously be leveraged to support regulatory marketing applications, reimbursement decisions, marketing claims as well as provide strategic market and safety data to sponsors and regulatory authorities. It is critical, during the design phase, to ensure that the differing goals of an observational study do not conflict and, instead, are set up to compliment each other. This workshop will provide attendees with examples of observational studies which have been developed to satisfy multiple peri-approval requirements and research goals. In one such example, an EU registry was able to provide safety data for a U.S. NDA. Participants will also learn how various tools, including active safety data reporting, on-site monitoring and validated EDC systems, while not commonly performed in these types of studies, can add value to the overall program.
W30: TECHNIQUES FOR LINKING ADMINISTRATIVE CLAIMS AND EMR-BASED DATA SOURCES
Discussion Leaders: Erica Danielson PhD, Healthcare Research Manager, GE Healthcare Clinical Data Systems, Hillsboro, OR, USA; Stacey R Long MS, Vice President, Thomson Reuters, Hampden, ME, USA
PURPOSE: This workshop will provide an overview of approaches for linking administrative claims and electronic medical record (EMR) based data sources, and how combined they can be used to support comparative effectiveness research. Emphasis will be placed on linkage techniques when patient identifiable information is not available for matching purposes.
DESCRIPTION: Administrative claims have been a longstanding data source for health outcomes studies, but sometimes they lack the clinical depth needed to support comprehensive research on treatment effectiveness. Historically, studies of real-world clinical effectiveness have involved costly, time-consuming chart abstraction processes. The increased adoption of EMRs has provided a more affordable and timely data source for obtaining clinical detail. EMRs can have their own limitations, however, including the inability to track services outside the EMR network, e.g., hospitalizations. There is growing interest in linking claims and EMR data sources for the purpose of developing a more robust source of information to support comparative effectiveness research. The source of the data, its available variables, and the intended uses are all important factors in determining which techniques are appropriate when combining data assets. Workshop leaders will recap past US government initiatives to link data disparate sources, review the pros and cons of claims and EMR databases and their ability to support comparative effectiveness research, and discuss options for linking claims and EMR data. Lastly, findings from a hypertension case study utilizing a variety of record linking techniques will be reviewed. Throughout this session, attendees are encouraged to ask questions, describe their information needs, and discuss their experience with data linkages.
12th Annual European Congress Main Page
