Workshops
Monday, 31 October 2016
11:15 - 12:15
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

11:15 - 12:15
Room: Hall E1 (L0)

W1: THE NEW NICE CANCER DRUGS FUND TECHNOLOGY APPRAISAL PROCESS: YOUR QUESTIONS ANSWERED

Discussion Leaders:

Meindert Boysen, PharmD, MSc, Professor, Health Services Research & Policy, London School of Hygiene & Tropical Medicine, London, UK

David Tyas, PhD, Associate Director, Health Economics and Outcomes Research, Bristol-Myers Squibb, Uxbridge, UK

Caroline Ling, PhD, Senior Director, Market Access and Outcomes Strategy, RTI Health Solutions, Manchester, UK

Sorrel Wolowacz, PhD, Head, European Health Economics, RTI Health Solutions, Manchester, UK

PURPOSE:

From April 2016, cancer drugs that appear promising in terms of cost-effectiveness, but for which the clinical evidence is not strong enough for routine commissioning, may be recommended by NICE. Such drugs will be made available through the Cancer Drugs Fund (CDF) for a limited time while more evidence is gathered. NICE has implemented a new Technology Appraisal process and methods for the new CDF. This workshop will provide an overview of the new CDF operating model, guidance on how manufacturers can best prepare for NICE’s appraisal for a cancer drug when a conditional recommendation for use within the CDF is anticipated, and an extended Q&A session to answer audience questions about the new process.

DESCRIPTION:

This session will start with an introduction to changes to the Technology Appraisal process to take account of the CDF. The circumstances under which NICE may issue a recommendation for use within the CDF will be described. We then will discuss how manufacturers can best prepare for appraisal if a recommendation for use within the CDF seems likely. Topics covered will address any differences in the content of the manufacturer’s evidence submission; approach to evaluating whether (given uncertainty in the clinical data) the ICER has the “plausible potential” to lie within the range normally considered a cost-effective use of NHS resources; and the assessment and implications of end-of-life criteria. Consideration also will be given to the kind of study or data collection that could be feasible within the limited time period, the review of the guidance after data collection, and the commercial access arrangements under the new CDF. The workshop will include an extended interactive Q&A session, during which the audience will have the opportunity to discuss aspects of the CDF with the panel.

Clinical Outcomes Research

11:15 - 12:15
Room: Hall E2 (L0)

W2: MOVING THE SCIENCE FORWARD: PSYCHOMETRIC CONSIDERATIONS AND STUDY DESIGNS FOR UNDERSTANDING MEANINGFUL CHANGE AND CONDUCTING MIXED METHODS RESEARCH

Discussion Leaders:

Stacie Hudgens, MA, Managing Partner, Strategic Lead, Clinical Outcomes Solutions, Tucson, AZ, USA

Tara Symonds, PhD, COA Strategy Lead & Partner, Clinical Outcomes Solutions Ltd., Folkestone, Kent, UK

Lori McLeod, PhD, Director of Psychometrics, RTI Health Solutions, Research Triangle Park, NC, USA

Cheryl Coon, PhD, Principal, Outcometrix, Tucson, AZ, USA

PURPOSE:

The purpose of this workshop is to address two methodological issues facing the regulatory and scientific community in the field of clinical outcome assessment (COA) – derivation of meaningful change thresholds and mixed methods research – and to examine recent deliberations and present recommendations aimed at building scientific consensus on these issues.

DESCRIPTION:

A panel of measurement specialists was convened to consider these two issues, which were identified by academics, industry, and regulators as scientifically challenging in the context of developing, evaluating, and interpreting data from COA tools. The issues (and specific questions) considered by this panel included: 1. Proposed methods for determining clinically meaningful change and evaluation of potential anchors. What defines a good anchor? How should potential anchors be evaluated? What parameters should be included in the anchor evaluation? How can distribution-based methods be used to support anchor-based analyses to determine meaningful change at the within patient level? Are qualitative studies a more direct way of deriving meaningful change based on patients’ perspectives? What is the appropriate role for cumulative distribution functions? 2. Definition of mixed methods and mixed methods study designs for COA tool development. What is the definition for mixed methods in this field of research? What is the value of mixed methods research in COA tool development? Which research designs should be considered to provide an efficient way of developing content for new measures? When is a qualitative sample sufficient for exploratory quantitative analysis? The discussion leaders will outline the issues and describe the panel discussions, noting areas where agreement was achieved, where agreement is close but not yet achieved, and where additional research is needed. Discussion leaders will engage attendees to share their perspectives, as well as to provide insight gained from their own research, to move toward scientific consensus.

Economic Outcomes Research

11:15 - 12:15
Room: Hall F1 (L0)

W3: UTILITIES IN ECONOMIC EVALUATION: USING BEST PRACTICES WHERE INTERNATIONAL GUIDELINES PROVIDE INSUFFICIENT DETAIL

Discussion Leaders:

John Brazier, PhD, MSc, Professor of Health Economics, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

Roberta Ara, MSc, Senior Research Fellow, School of Health and Related Research, The University of Sheffield, Sheffield, UK

Helene Chevrou-Severac, PhD, Global Head of Health Economics and HTA, Takeda Pharmaceuticals International AG, Glattpark-Opfikon, Switzerland

PURPOSE:

 The workshop will identify gaps in current international guidelines for health economic evaluation in the identification of utility values and the incorporation of these values into economic models. Workshop participants will become familiar with proposed solutions for meeting these gaps based on current best practice and the literature, and critically evaluate these based on their own views and experiences.

DESCRIPTION:

 The workshop will outline the use of best practice to inform the use of utility values in economic models including estimating utility values for comorbidities and adverse events, and encourage audience participation to inform the debate. Professor Brazier will summarize gaps in international guidelines in economic evaluation for identifying the source of utility values and how these utility values should be incorporated into the economic model. Ms. Ara will use current literature, available recommendations and previous submissions to NICE to propose solutions to filling these gaps, focusing upon 1) obtaining source data using clinical trials and/or systematic reviews, 2) incorporating the identified utility values into the economic model including adjustments for comorbidities, adverse events and baseline evidence. Dr. Chevrou-Severac will discuss the solutions from the perspective of an international pharmaceutical company, and outline the importance of providing transparent and consistent international guidelines for economic models. Fifteen minutes at the end of the session will be devoted to audience participation including critical evaluation of the proposals from participants own experiences to achieve an inclusive and international perspective. Workshop participants will gain further understanding of where gaps exist in international guidelines in the identification and usage of utility values, and contribute to the debate on how to best fill these gaps.

14:15 - 15:15
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

14:15 - 15:15
Room: Hall E1 (L0)

W4: UNDERSTANDING THE CHALLENGES FACING THE VALUE ASSESSMENT OF MEDICAL DEVICES

Discussion Leaders:

Richard Charter, MSc, Head of Market Access & Pricing Diabetes Care Europe, Becton Dickinson AG, Allschwil, Switzerland

Carla Zema, PhD, Director, Health Policy and Economics, ZOLL, Pittsburgh, PA, USA

Chantale Lessard, RPh, MSc, PhD, DcomplD, Adjunct Professor, Department of Health Management, Evaluation and Policy, School of Public Health, University of Montreal, Montreal, QC, Canada

PURPOSE:

This workshop will highlight issues in the value assessment of medical devices including differing regulatory and HTA/reimbursement requirements, challenges with assuming that randomized controlled trials (RCTs) are the gold standard for clinical evidence, why pharmaceutical standards are not always appropriate for devices, and the challenges of prescribing similar requirements to the wide variety of medical devices, including implantable devices, capital equipment, diagnostics, medical equipment and supplies, and consumables. Participants will begin to discuss potential solutions to these challenges. This workshop would benefit all stakeholders interested in the value assessment of medical devices including regulatory agencies, HTA organizations, payers and reimbursement organizations, and health care decision-makers.

DESCRIPTION:

While there is no disagreement regarding the importance of value assessment of medical devices, lack of alignment of stakeholder needs and requirements creates many issues and challenges in market access of medical devices. Regulatory agencies focusing on safety and effectiveness have different standards than health technology assessment (HTA) and reimbursement organizations. Furthermore, applying pharmaceutical standards and requirements, such as RCTs that are not always feasible for medical devices, may be a significant barrier. Moreover, one set of standards will likely not be applicable to the range of different types of medical devices. In discussing those challenges, the workshop will not only inform participants but will also provide input to the ISPOR Value Assessment of Medical Device working group's manuscript in addressing these challenges. Presented by the ISPOR Medical Devices and Diagnostics special interest group.


14:15 - 15:15
Room: Hall A (L2)

W5: NEW APPROACHES TO SURVIVAL MODELING IN ONCOLOGY

Discussion Leaders:

Andrew Briggs, DPhil, William R. Lindsay Chair of Health Economics, Health Economics & Health Technology Assessment, University of Glasgow, Glasgow, UK

Jack Ishak, PhD, Senior Research Leader, Modeling & Simulation, Evidera, Montreal, QC, Canada

Federico Felizzi, PhD, HTA Statistician, GPMA, F. Hoffmann La Roche, Basel, Switzerland

Aline Gauthier, MSc, Managing Director, Health Economics and Market Access, Amaris, London, UK

Victoria Federico Paly, MHS, Senior Health Economist, ICON Health Economics & Epidemiology, New York, NY, USA

PURPOSE:

Given the nature of oncology trial design and conduct, overall survival effects of treatments are often not directly measured. However, estimates of these survival effects are important for economic evaluation. This workshop was constructed to provide those interested in this area a selection of recent research efforts that address this issue.

DESCRIPTION:

The four papers in this session reflect separate studies on various aspects of estimating or modeling overall survival effects in oncology treatment. Topics include parametric and simulation modeling tailored to mechanism of action, use of registry data for background survival estimation, adjusting for varying follow-up times and sample sizes, integrated survival modeling across treatments, and applications to cost-effectiveness and budget impact modeling. PROJECTING SURVIVAL WITH IMMUNO-ONCOLOGY THERAPIES Jack Ishak, PhD, Senior Research Leader, Modeling & Simulation, Evidera, Montreal, QC, Canada; Noemi Muszbek, MSc, Senior Research Scientist, Evidera, London, United Kingdom; Irina Proskorovsky, MSc, Director, Principal Statistician, Modeling & Simulation, Evidera, Montreal, QC, Canada; Yiduo Zhang, PhD, Director, Health Economics and Payer Analytics, Astrazeneca, Gaithersburg, MD, USA MODELING LONG-TERM SURVIVAL AND ECONOMIC IMPLICATIONS IN TARGETED CANCER THERAPIES Federico Felizzi, PhD, HTA Statistician, GPMA, F. Hoffmann La Roche, Basel, Switzerland; Anirban Basu, PhD, Professor & Director, Pharmaceutical Outcomes Research and Policy Program, University of Washington, Seattle, WA, USA; Aasthaa Bansal, PhD, Research Assistant Professor, Pharmaceutical Outcomes Research & Policy Program, University of Washington, Seattle, WA, USA; Noman Paracha, MSc, Health Economist, GPMA, F. Hoffman-La Roche, Basel, Switzerland MODELING SURVIVAL EXPERIENCE USING REAL WORLD EVIDENCE: INSIGHTS FOR CLINICAL TRIAL-BASED ECONOMIC EVALUATIONS Andy Briggs, PhD, William R. Lindsey Chair, Health Economics & Health Technology Assessment, University of Glasgow, Glasgow, United Kingdom; Dhvani Shah, MS, Senior Health Economist, Health Economics, ICON Plc, New York, NY, USA; Victoria Federico Paly, MHS, Senior Health Economist, ICON Health Economics & Epidemiology, New York City, NY, USA; Vijayveer Bonthapally, PhD, Senior Director, Formerly Millennium Pharmaceuticals Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, Cambridge, MA, USA USING SURVIVAL ANALYSES TO DEVELOP FLEXIBLE BUDGET IMPACT MODELS IN ONCOLOGY Keith R Abrams, PhD, Professor of Medical Statistics, Department of Health Sciences, University of Leicester, Leicester, United Kingdom; Aline Gauthier, MSC, Managing Director, Health Economics and Market Access, Amaris, London, United Kingdom; Emanuela Castelnuovo, MSc, MA, Director, Health Economics and Market Access, Amaris, London, United Kingdom

15:45 - 16:45
BREAKOUT SESSION

15:45 - 16:45
Room: Hall D (L -2)

W6: BRIDGING THE GAP BETWEEN EFFICACY AND EFFECTIVENESS: METHODS TO IDENTIFY DRIVERS OF EFFECTIVENESS BEFORE LAUNCH

Discussion Leaders:

Chris Chinn, MSc, ACA, Head of Real World Investigations, Sanofi, Guildford, UK

Clementine Nordon, MD, PhD, Senior Epidemiologist, LASER Analytica, Paris, France

Lifang Liu, MD, PhD, Epidemiologist, EORTC, Brussels, Belgium

Mikkel Z. Ankarfeldt, PhD, Post Doc, Novo Nordisk A/S, Soeborg, Denmark

PURPOSE:

Awareness on potential discrepancies between efficacy and effectiveness (“efficacy-effectiveness gap”) of new drugs being launched has grown among Health Technology Assessment and Regulatory bodies. The European Innovative Medicines Initiative launched the GetReal project, in order to provide scientific and operational solutions to inform on the effectiveness of drugs at an earlier stage of drug development. This project involves more than 30 public and private stakeholders (the EMA, the HAS, ZIN, NICE, academic researchers and Pharma Industry) and aims at providing methods to assess the effectiveness of drugs, before and just after launch.

DESCRIPTION:

In the first session (20 minutes), the speakers will introduce the concept of “drivers of effectiveness” which are defined as patient-related, drug use-related or health care system-related factors (e.g. follow up strategies) that interact with the drug’s effect and may account for an efficacy-effectiveness gap, if inadequately assessed or measured before the launch the drug. In the second session (30 minutes), the speakers will present the methods that can be used to identify these “drivers of effectiveness” at an early stage of drug development. These methods include: (1) literature reviews and experts interviews and (2) patient-level or aggregate data analyses. The strengths and limitations of these methods will be discussed in the light of 3 case studies that were conducted for the GetReal project (drivers of effectiveness in glucose-lowering drugs in diabetes, antipsychotic drugs in schizophrenia, and anti-cancer therapeutic strategies in Hodgkin’s Lymphoma over 40 years). The workshop will conclude with a discussion in which workshop participants will be encouraged to share their understanding and perception of the methods presented, as well as the anticipated acceptability by various stakeholders.

Economic Outcomes Research

15:45 - 16:45
Room: Hall E2 (L0)

W7: APPROPRIATE MODEL TIME HORIZONS: THEORY, EMPIRICAL RESULTS, AND THE PERSPECTIVE OF DECISION MAKERS

Discussion Leaders:

Anthony J Hatswell, MSc, Principal Consulting Economist, Department of Statistical Science, University College London, London, UK

Uwe Siebert, MD, MPH, MSc, ScD, Professor & Chair, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Austria, Adjunct Professor, Health Policy and Management, Harvard School of Public Health, Boston, MA, USA, and Director, Division for HTA, ONCOTYROL – Center for Personalized Cancer Medicine, Hall i.T., Austria

Ash Bullement, BSc, Health Economist, BresMed Health Solutions, Sheffield, UK

Matthew Taylor, MSc, PhD, Director, York Health Economics Consortium, Heslington, York, UK

PURPOSE:

 This workshop brings together the theoretical arguments (and guidelines) related to the choice of the time horizon for an economic model, empirical research on the effect of different time horizons on model results, the perspective of different decision makers on the appropriateness of modelled results and the characterization of uncertainty associated with long-term extrapolation.

DESCRIPTION:

 Economic modelling frequently requires extrapolation beyond the time horizon for which evidence is available modelling guidelines suggesting that the appropriate timeline be ‘enough to capture relevant benefits and costs’. Professor Siebert will begin the workshop with an overview of available guidance from modelling guidelines, how these were created, and theoretical arguments that the guidance was based on. Ash Bullement will then present work on the effect of varying time horizons on models and subsequent results in different disease areas (ranging from cancer to vaccinations), which includes alternative implementations of a ‘lifetime’ horizon. Dr. Taylor will then discuss the challenges of the appropriate time horizon from his perspective as a member of the NICE Public Health Advisory Committee. Anthony Hatswell will then lead a roundtable discussion (with audience participation) on what time horizons are appropriate for different models, how these should be practically implemented by modelers, and the challenges faced in the justification of extrapolations to non-economist audiences.

17:00 - 18:00
BREAKOUT SESSION

17:00 - 18:00
Room: Hall A (L2)

W8: THE IMPORTANCE OF RELEVANCE IN HEALTH ECONOMIC EVALUATIONS: CHALLENGES AND WAYS FORWARD

Discussion Leaders:

Herbert J.A. Rolden, PhD, Postdoctoral Researcher, Radboud Institute for Health Sciences, Department for Health Evidence, Radboudumc, Nijmegen, The Netherlands

J. Jaime Caro, MDCM, FACP, FRCPC, Adjunct Professor, Medicine, Epidemiology, and Biostatistics, McGill University, Montreal and Chief Scientist, Evidera, Inc., Lexington, MA, USA

Manuela A. Joore, PhD, Professor of Health Technology Assessment & Decision Making, Department of Clinical Epidemiology and Medical Technology Assessment, School for Public Health and Primary Care (CAPHRI), Faculty of Health, Medicine and Life Sciences, Maastricht University Medical Center, Maastricht University, Maastricht, The Netherlands

Janneke P.C. Grutters, PhD, Assistant Professor, Radboud Institute for Health Sciences, Department for Health Evidence, Radboudumc, Nijmegen, The Netherlands

PURPOSE:

The aim of this workshop is to familiarize participants with the concept of relevance in model-based health economic evaluations. Participants will also gain insight into the main problems we encountered when we reviewed model-based economic evaluations for relevance; as well as ways to avoid these problems in the future.

DESCRIPTION:

As model-based health economic evaluations are used to make decisions that greatly affect citizens, it is not only imperative that the underlying economic models are sound, but also that model inputs are representative of actual clinical practice, or in other words, relevant for the decision context. In the first half of the workshop, Jaime Caro will provide background information on the concept of relevance, and what factors should be taken into account for a health economic evaluation to be relevant. Thereafter, Herbert Rolden will present the results of a systematic review addressing the relevance of model-based economic evaluations of dabigatran for atrial fibrillation. Manuela Joore will then discuss the concept of relevance in relation to framing a health economic evaluation, and will provide insight into the main relevance issues in model-based economic evaluations of trastuzumab for early breast cancer. Afterwards, Jaime Caro will reflect on the previous presentations and together we will discuss ways to increase relevance when performing a health economic evaluation. In the second half of the workshop, participants will be asked to reflect on the problems with relevance, whether they have encountered such problems, and what they propose as ways to avoid these problems. This interactive workshop will prove to be useful for those performing model-based health economic evaluations, both from industry and academia, as well as those reviewing and utilizing such economic evaluations, such as members of reimbursement agencies, peer reviewers, and journal editors.

Health Policy Development Using Outcomes Research

17:00 - 18:00

W9: WITHDRAWN

Discussion Leaders:
PURPOSE:

 This session has been withdrawn from the program.

DESCRIPTION:

 This session has been withdrawn from the program.

Tuesday, 1 November 2016
8:45 - 9:45
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

8:45 - 9:45
Room: Hall A (L2)

W10: THE POTENTIAL AND PITFALLS OF USING MULTI-CRITERIA DECISION ANALYSIS TO SUPPORT HEALTH TECHNOLOGY ASSESSMENT

Discussion Leaders:

Janine A. van Til, PhD, Assistant Professor Preference Research, Health Technology and Services Research, University of Twente, Enschede, The Netherlands

Rob Baltussen, PhD, Associate Professor Health Economics, Department for Health Evidence, Radboud University Medical Center, Nijmegen, The Netherlands

Michele Tringali, MD, Director, HTA Program, Lombardy Region Health Directorate, Milan, Italy

Susanne Schmitz, PhD, Postdoctoral Fellow, Department of Population Health / Health Economics and Evidence Synthesis Research Unit, Luxembourg Institute of Health, Strassen, Luxembourg

PURPOSE:

This workshop will focus on practical examples of supporting health technology assessment and reimbursement decisions with multi-criteria decision analysis (MCDA). In the workshop, both the value of MCDA to support health technology assessment (HTA) decisions, as well as the pitfalls encountered in applying MCDA to real-life decision contexts will be discussed.

DESCRIPTION:

Workshop participants will be familiarized with how and why applications of different methods of MCDA are explored in countries divergent in current HTA structuring, experience, legislation and culture. The workshop will address 1) the fit between MCDA principles and the principles of HTA; 2) the current HTA process in these countries; 3) share experiences of introducing MCDA to the actual HTA decision process and will address 4) whether MCDA keeps its promise to increase structure, transparency and comprehensiveness to the HTA decision process.

Dr. van Til will reflect on the apparent fit between MCDA and HTA decision making, and on actual testing of a mathematical MCDA framework in the Dutch context. Dr. Baltussen will talk about the use of a deliberative approach to MCDA, involving stakeholder consultation, and presents a number of case studies from the Netherlands, Thailand and Indonesia. Dr. Tringaly will explain the information framework aimed at structuring assessment reports for appraisal activities adopted by The Lombardy Healthcare Directorate, and share experiences with implementation. Dr Schmitz will present the results of a retrospective analysis to identify potential important drivers of reimbursement decisions in Ireland; she will illustrate the challenges of using such information to inform an actual MCDA framework.

Participants will be encouraged to share their experience and perspectives during the workshop. This interactive and informative workshop will be valuable to researchers in the field of MCDA and regulators in the HTA context, who are interested in understanding recent developments in the application of MCDA in the HTA context.

Use of Real World Data

8:45 - 9:45
Room: Hall D (L -2)

W11: LET'S GO FLY A KITE - MANAGING THE WINDS OF CHANGE IN REAL WORLD EVIDENCE STUDIES

Discussion Leaders:

Alison Bourke, BSc, MSc, Scientific Director Real-World Evidence Solutions, IMS Health, London, UK

Andrew Bate, PhD, Senior Director, Epidemiology Group Lead, Analytics & Worldwide Safety, Pfizer Ltd, Surrey, UK

Gillian C Hall, PhD, Consultant in Pharmacoepidemiology, Gillian Hall Epidemiology Ltd, London, UK

PURPOSE:

Sustainability issues, such as data continuity and access, and associated methodology can impact on many health economic/outcomes studies. This workshop will highlight these issues and discuss how internal and external factors, such as clinical practice and legal frameworks, should be considered during study design. The workshop will provide guidance on how to minimize the impact of change.

DESCRIPTION:

In our continually evolving research environment, change is inevitable; such changes commonly involve the addition of more recent data, but also may relate to alterations of the data model, tools, data elements or even healthcare environment. These changes can impact our research, but are we good at anticipating and managing such change? The importance of change management is recognized in a recent publication providing guidance and a checklist to help plan for change in observational healthcare database studies (Bourke et al, Pharmacoepi and Drug Safety. In press). A panel of researchers and data providers will first explain change issues and illustrate why sustainable design is a key consideration in real world data (RWD) research. The importance of change management to the validity, transparency and reproducibility of database studies will be outlined, then the audience will be split into Working Groups to use the published checklist as a tool to identify and consider change across simulated HEOR studies. The Discussion Leaders will facilitate an interactive conversation with workshop participants incorporating feedback from the Working Groups on how changes can affect studies and whether such changes can be anticipated. This interactive workshop will help to plan strategies for sustainability for both researchers and database providers. The workshop will provide participants with change management competencies by creating an interactive environment for attendees to learn about the issues and to practice effectively planning RWD studies

Patient-Reported Outcomes & Patient Preference Research

8:45 - 9:45
Room: Hall E2 (L0)

W12: RETHINKING WHAT MISSING PATIENT-REPORTED OUTCOME (PRO) DATA CAN TEACH US ABOUT OUTCOMES AND SURVIVAL IN ONCOLOGY CLINICAL TRIALS

Discussion Leaders:

Mark E. Boye, PhD, Principal Scientist, Eli Lilly and Company, Indianapolis, IN, USA

Katherine Houghton, MSc, Associate Director, Health Economics, RTI Health Solutions, Durham, NC, USA

Donald E Stull, PhD, Head, Data Analytics and Design Strategy, Health Economics, RTI Health Solutions, Durham, NC, USA

PURPOSE:

This workshop will discuss the importance of missing patient-reported outcome (PRO) data in oncology clinical trials; describe why implementation of methods that make correct assumptions is important; present novel methods to identify heterogeneous groups of treatment responders informed by missing PRO data patterns and their link with survival outcomes; and present examples using these methods. The anticipated audience includes those familiar with PRO analyses who want to learn and incorporate methods that show more accurate and compelling results.

DESCRIPTION:

Although much attention has been paid to the development and administration of PRO questionnaires, largely unrecognized is the explicit analysis of missing data and their impact on clinical trials. Pattern mixture models (PMMs) are recommended as a sensitivity analysis when one suspects that data are not missing at random. These models build in covariates to account for differential missing data patterns and allow the derivation of an adjusted mean effect. This workshop presents an extension to PMMs (ePMM) within a latent variable framework: heterogeneity is explicitly examined at the patient level in terms of intercepts and changes over time, while simultaneously building in the effect of missing data patterns. Data-driven subgroups can be identified that allow analysts to determine whether missing data leads to different PRO results. Once identified, survival can be compared between the subgroups. Identifying subgroups who are more, or less, likely to complete oncology clinical trials and assessing the relationship with survival can play a key role in study design and the development of personalized medicine. We will present models, show the effects on survival outcomes, and review randomized clinical trial examples. Following the presentation, the audience will be encouraged to discuss the pros and cons of these methods compared to currently used approaches in the design and analysis of oncology clinical trials.

13:45 - 14:45
BREAKOUT SESSION

13:45 - 14:45
Room: Hall E1 (L0)

W13: CHANGING OUR PERSPECTIVE ON PATIENT-REPORTED OUTCOMES IN ONCOLOGY: IT’S MORE COMPLEX THAN TRADITIONALLY CONCEPTUALIZED OR ANALYZED

Discussion Leaders:

Mark E. Boye, PhD, Principal Scientist, Eli Lilly and Company, Indianapolis, IN, USA

Donald E Stull, PhD, Head, Data Analytics and Design Strategy, Health Economics, RTI Health Solutions, Durham, NC, USA

Claire Ainsworth, BSc, Research Analyst, Health Economics, RTI Health Solutions, MANCHESTER, UK

PURPOSE:

 This workshop will introduce participants to path modeling and its ability to examine direct and indirect effects of symptoms of cancer and its treatment on health-related quality of life (HRQL) outcomes. Workshop participants will become familiar with the concepts of direct, indirect, and total effects and the importance of conceptualizing and analyzing multidimensional phenomena appropriately.

DESCRIPTION:

 It’s not uncommon to find little or no relationship between symptoms or their change and selected HRQL outcomes when analyzing oncology trial data. One reason, we argue, is that the relationships among these variables are more complex than the analyses would imply or can evaluate. That is, the hypothesis that symptoms affect overall quality of life (QoL) may not be supported (or only marginally so) because the analyses assume a direct effect of symptoms on QoL. However, thinking through the logic among these variables suggests a “causal cascade,” whereby intervening variables mediate the causal pathway between symptoms and QoL or other dimensions of HRQL. Thus, as will be demonstrated, symptoms, such as pain or appetite loss, affect fatigue, which affects QoL. Path analyses are needed to evaluate these more complex causal relationships and obtain total effects of symptoms on the various domains of HRQL. The result is a more accurate understanding of treatment effects and how that affects HRQL. Dr. Boye will discuss the implications this conceptual and analytic approach has for refining our understanding of efficacy of oncology treatments. Dr. Stull will provide background on path analysis, and Ms. Ainsworth will give an empirical example from two trials of treatment for lung cancer. The audience will be encouraged to discuss and explore their own examples as they relate to endpoint models and conceptual frameworks and how these methods may help shed light on null or marginal results they have obtained in trials.


13:45 - 14:45
Room: Hall E2 (L0)

W14: CHOICE DEFINES VALUE: HOW TO ANALYSE AND DISSEMINATE EVIDENCE ON THE IMPORTANCE OF MULTIPLE CRITERIA FROM MULTIPLE PERSPECTIVES TO INFORM AN APPRAISAL PROCESS IN HEALTH TECHNOLOGY ASSESSMENT

Discussion Leaders:

Benjamin M. Craig, PhD, Associate Professor, Department of Economics, University of South Florida and Assistant Member, Health Outcomes and Behavior, Moffitt Cancer Center, Tampa, FL, USA

Axel Mühlbacher, PhD, Professor, Health Economics and Health Care Management, IGM, Hochschule Neubrandenburg, Neubrandenburg, Germany

Juan Marcos Gonzalez, PhD, Senior Research Economist, RTI Health Solutions, Research Triangle Park, NC, USA

Juan Manuel Ramos-Goñi, MSc, Senior Researcher, EuroQol Research Foundation, Rotterdam, The Netherlands

PURPOSE:

The paradigm shift in health technology assessment (HTA) towards including multiple perspectives can be fulfilled only when their views are systematically taken into account. To safeguard the public health, HTAs originally assessed health outcomes and relied on the judgment of professionals and policy makers concerning the trade-offs between benefits and risks. More recently, the focus has shifted toward the patient voice and addressing the problems of weighing multiple decision criteria, such as clinical efficacy, severe side effects and varying modes of administration. A key challenge in HTA is weighting multiple criteria from multiple perspectives to inform their decisions.

DESCRIPTION:

At the 21st Annual International Meeting, the presenters gave a workshop on multi criteria decision analysis (MCDA). In complement to their didactic introduction, they conducted a live MCDA using hand-held response system and interpret its results in real time. The proposed workshop has 4 components. (1) Using the previous responses (paired comparison and best-worst scaling [BWS]), they will expand upon the first by demonstrating the analysis, interpretation and dissemination of the results Although conducting MCDA using audience responses has limitations and challenges, this analytical demonstration is clearly relevant and will serve to introduce a deliberative approach adaptable for multiple settings (e.g., expert panel, one-on-one interviews, online). (2) The audience will be given a hands-on exercise regarding the interpretation of MCDA results and the merits between methods (paired comparisons vs. BWS). (3) With this background, the presenters will introduce examples and open discussion to the audience to ask questions and express concerns regarding MCDA. (4) To aid the greater community, the responses and discussion will recorded, transcribed, de-identified, summarized and disseminated to ISPOR members (conditional on acceptance by ISPOR Spotlight) by the presenters. Through audience engagement and demonstration, the proposed workshop will give attendees a hands-on understanding of the underlying issues surrounding MCDA.

15:15 - 16:15
BREAKOUT SESSION
Economic Outcomes Research

15:15 - 16:15
Room: Hall E1 (L0)

W15: BUILDING AND APPLYING CONSTRAINED OPTIMIZATION MODELS IN HEALTH CARE: A CASE STUDY OF OPTIMAL KIDNEY MATCHING

Discussion Leaders:

William H. Crown, PhD, Chief Scientific Officer, Optum Labs, Cambridge, MA, USA

Alec Morton, PhD, Professor, Management Science, University of Strathclyde, Glasgow, UK

William V. Padula, PhD, Assistant Professor, Health Policy & Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA

Praveen Thokala, MASc, PhD, Research Fellow, Health Economics and Decision Science (HEDS), School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

PURPOSE:

To demonstrate the steps and relevance of building constrained optimization models in the context of addressing important health care decisions, such as kidney matching. Session content draws from the second paper of the ISPOR Constrained Optimization Methods in Health Services Research Task Force, which broadly illustrates the development and evaluation of constrained optimization models in health care. The session is intended both for modelers and for consumers of modeling.

DESCRIPTION:

There is growing interest in using optimization modeling to improve clinical and economic outcomes in healthcare, but there are not yet standard guidelines for modeling and reporting. Moreover, most health care applications of optimization methods have focused on traditional operations research problems such as facilities management, minimizing supply costs, etc. that commonly arise in health care operations. The workshop will focus on the role that optimization models can have in improving clinical outcomes. We will illustrate the steps in building and evaluating model performance using problems arriving in organ donation. This case study is drawn from a published influential kidney matching model that has been widely adopted and estimated to have saved hundreds of lives (Anderson et al., 2015). Topics covered will include background on the complexity of kidney matching, structuring the model, estimating key parameters, model validation and sensitivity analysis. We will discuss the importance of communicating results and translating them into action. In the optimal kidney matching case, for example, overcoming skepticism and resistance within the medical community required strong trans-collaboration between teams of physicians and operations researchers. The workshop will include an interactive discussion about the ability of optimization methods to address real-world problems versus oversimplified versions of these instances—in particular, the tension between model simplification and the usefulness of optimization modeling in application. Finally, we will outline open questions and challenges in using optimization methods to model and analyze healthcare decisions. Presented by the ISPOR Optimization Methods in Health Services Research Emerging Good Practices Task Force


15:15 - 16:15
Room: Hall A (L2)

W16: HOW TO TRANSFER ECONOMIC EVIDENCE TO LOW AND MIDDLE-INCOME COUNTRIES? WORKING UNDER CAPACITY RESTRICTIONS

Discussion Leaders:

Zoltan Kalo, MD, PhD, Professor of Health Economics & Head of the Institute of Economics, Eötvös Loránd University, Budapest, Hungary

Johan L. Severens, PhD, Professor of Evaluation in Health Care, Institute of Health Policy & Management, Institute of Medical Technology Assessment (iMTA), Erasmus University Rotterdam, Rotterdam, The Netherlands

Olena Mandrik, PhD, Postdoc Researcher, Institute of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, The Netherlands

Saskia Knies, PhD, Advisor Pharmacoeconomics, National Health Care Institute, Diemen, The Netherlands

PURPOSE:

The workshop is designed to share the theoretical and practical experience of transferring economic data to and between low- and middle- income countries (LMICs). Workshop participants will become familiar on recent developments in transferability of economic evaluations, approaches towards inputs transferability, its facilitators and barriers in LMICs. The participants will be able to critically evaluate the transferability of evidence from the other countries.

DESCRIPTION:

As more decision makers worldwide seek information on economic impact of healthcare interventions, the importance of research transferability grows. LMICs have limited capacity in health technology assessment (HTA), and so have higher needs for research transferability. How to transfer economic evidence under conditions of the limited HTA-resources? How to transfer the published results to the jurisdiction of interest? How to improve transferability of the research conducted in healthcare settings of LMICs? The workshop is devoted to highlight this information. During the workshop participants will receive a contemporary information on: (a) Developments in HTA in LMICs, reasons studies’ transferability, and its basic principles; (b) Availability and quality of economic evidence in LMICs, critical appraisal in healthcare decisions; (c) Transferability of inputs when the relevant guidelines are not conclusive, generalizability of utilities and productivity losses; (d) Approaches in studies’ evaluation and selection the right models for local economic evaluations. Audience participation will include (a) an inquiry of attendees on their experience with data transferability and faced difficulties; (b) a survey on factors important in HTA-based decision making; (c) case-studies in several disease areas. The audience will be proposed to estimate an impact of variability in input parameters on the results of economic studies having variable volume of critical information. The workshop is oriented on any HTA- expert from business, state or academia segment operating in environments with limited HTA-capacity and so requiring good understanding of data transferability.

Health Policy Development Using Outcomes Research

15:15 - 16:15
Room: Hall E2 (L0)

W17: NEW CHALLENGES FOR HIGH-RISK MEDICAL DEVICES IN EUROPE: HOW TO ACHIEVE THE BALANCE BETWEEN THE DEMAND FOR EARLY PRODUCT LAUNCH AND SAFETY?

Discussion Leaders:

Bernard Avouac, MD, Former president of Transparency Commission, Medecin des Hopitaux de Paris, Paris, France

Jenifer Ehreth, PhD, MHA, Executive & Scientific Vice President, LASER Analytica, Paris, France

Rosanna Tarricone, PhD, Professor, Centre for Research on Health and Social Care Management (CERGAS), Bocconi University, Milan, Italy

Katerina Zakrzewska, PhD, Senior Director, Strategic and Medical Affairs, EMEA, Diabetes Care Companies, Johnson & Johnson, Zug, Switzerland

PURPOSE:

This workshop will focus on the challenges that high-risk (class III) medical devices (MD) are confronted with. Workshop participants will be introduced in the certification process of MD, the definition and requirements for different types of new MDs (i.e. me-too, modification, and innovative products) and will learn about the impact of the on-going revision of the Medical Device Directives (MDD) on manufacturers.

DESCRIPTION:

MDs are used to improve the quality of life or to safe lives of patients. To protect patients from threats caused by MDs and to safeguard the intended performance, devices must comply with the EU directives and be CE marked before entering the European market. Bernard Avouac will present the experience of the approval process for the reimbursement of high-risk MDs in France.

During the last years numerous severe issues initiated by implanted medical devices unsettled both physicians and patients, and forced the European Commission to revise the MDD. Based on publicly available examples (e.g. infusible bone grafts) Jenifer Ehreth presents the participants of this workshop examples of class III MDs that had to be withdrawn from the market because of serious adverse events occurring after their launch and the consequences for patients, physicians and manufacturers.

Due to limited or missing clinical data and the short life-cycle of MDs, the level of clinical evidence is frequently not as high as for pharmaceuticals. Rosanna Tarricone will discuss the challenge of clinical evidence for MDs in HTA from a research perspective and Katerina Zakrzewska will discuss the view of a medical device company regarding requirements of the new MDD and illustrates the potential consequences for patients and physicians.

This interactive workshop will be of interest for manufacturers of MDs, clinicians, and HTA analysts, who are interested in understanding recent developments of the evaluation process of MD in the EU.

16:30 - 17:30
BREAKOUT SESSION
Use of Real World Data

16:30 - 17:30
Room: Hall E2 (L0)

W18: METHODOLOGIES FOR EVALUATING GEOGRAPHIC VARIATION IN HEALTH CARE OUTCOMES RESEARCH

Discussion Leaders:

Rajesh Balkrishnan, PhD, Professor, Public Health Sciences, University of Virginia School of Medicine, Charlottesville, VA, USA

Gregoire Mercier, MD, PhD, Head, Economic Evaluation Unit, CHU Montpellier, Montpellier, France

Vera Georgescu, PhD, Head, Economic Evaluation Unit, CHU Montpellier, Montpellier, France

PURPOSE:

In large database studies of health services and outcomes, significant variation in utilization is seen because of differential geographical access. Traditionally used measures of geospatial variation using provider/facility to population ratios or travel times have significant limitations which necessitate the use of newer methodology.

DESCRIPTION:

We will discuss the evaluation of newer measures and techniques like the Two-step Floating Catchment Area (2SFCA), geographic hot spot analyses, and Geographic Weighted Regression (GWR) in evaluating health care outcomes such as medication adherence and preventable hospitalizations. We will present two research examples, one a study examining adjuvant hormone therapy adherence and patient outcomes in the rural Appalachian region of the United States, and the second, an analysis of geographic variation in potentially avoidable hospitalizations in France. The 2SFCA approach first identifies a prescriber catchment area and then computes a prescriber to population ratio for each physician’s catchment. The step one ratios within a population’s catchment are then summed, resulting in an access score for each population location. Geographical hot spot analyses can help identify geographic clustering patterns of medication use. GWR in which weights can vary as a function of geographical location can be used to examine spatial non-stationarity of the relationships between predictors and medication use outcomes. We will present results of comparative analyses and discuss practical approaches to implementing and using these methodologies for healthcare policy analyses and decision making. A methodological shortcoming for most studies on geographic variations using regression analysis is the lack of accounting for spatial autocorrelation across regions. They assume independence between regions, thus ignoring possible interactions. Since the region boundaries are often administrative and not defined according to the studied phenomenon, this assumption of independence is false. Adjusting for spatial autocorrelation often leads to wider confidence intervals, thus studies without such adjustments could be falsely positive.

Economic Outcomes Research

16:30 - 17:30
Room: Hall E1 (L0)

W19: STRENGTHENING THE ROLE OF VALUE OF INFORMATION ANALYSIS IN HEALTH TECHNOLOGY ASSESSMENT: KNOWN CHALLENGES AND PROPOSED SOLUTIONS

Discussion Leaders:

Elisabeth Fenwick, PhD, Principal of Health Economics, ICON Health Economics & Epidemiology, Abingdon, UK

Claire Rothery, PhD, Senior Research Fellow in Health Economics, Centre for Health Economics, University of York, York, UK

Hendrik Koffijberg, PhD, Associate Professor, Department of Health Technology & Services Research, MIRA institute for Biomedical Technology and Technical Medicine, University of Twente, Enschede, The Netherlands

David Glynn, MSc, PhD student, Centre for Health Economics, University of York, York, UK

PURPOSE:

To illustrate the role of value of information analysis for overcoming challenges associated with making research prioritization and coverage with evidence development decisions. The workshop will focus on proposed solutions to more challenging situations: i) the assessment of medical devices, as compared to drugs; ii) making decisions where formal cost-effectiveness analysis is not explicitly used in the decision making process; iii) illustrating the interpretation and comparison of value of information outcomes using a new reporting tool. Participants will be actively engaged in discussion of the challenges and proposed solutions.

DESCRIPTION:

The workshop will set out the challenges for the evaluation of uncertainty in HTA and demonstrate the role of value of information analysis for overcoming these challenges. The workshop will demonstrate that value of information analysis can be used across different types of technologies (e.g. medical devices vs. pharmaceuticals) and health systems (e.g. inclusion of formal cost-effectiveness analysis vs. absence of economic analysis). Dr Fenwick will describe recent developments which have prompted a need to consider the value of further research. Dr Rothery will present a framework for characterising uncertainty and the value of evidence for informing coverage with evidence development decisions for medical devices compared with pharmaceuticals. Consideration will be given to rapid incremental innovation, learning effects, upfront irrecoverable costs, which present a challenge for the evaluation of devices. Mr Glynn will present a framework for characterising uncertainty in the absence of formal cost-effectiveness analysis. Dr Koffijberg will present a novel checklist for reporting value of information outcomes and demonstrate its application to case studies. These new developments will serve as a starting point for engaging interactive discussion on the implications for decision making. The workshop is intended to be valuable to researchers, decision makers, and industry analysts who are involved in supporting and making research prioritization and coverage decisions. Presented by members of the ISPOR Value of Information Analysis for Research Decisions Emerging Good Practices Task Force

Use of Real World Data

16:30 - 17:30
Room: Hall D (L -2)

W20: PREDICTING TREATMENT EFFECTIVENESS OVER TIME IN REAL WORLD FROM TRIAL EFFICACY DATA

Discussion Leaders:

Keith Abrams, PhD, Professor of Medical Statistics, Department of Health Sciences, University of Leicester, Leicester, UK

Mark Belger, BSc, Principal Research Scientist, GSS, Eli Lilly, Ascot, UK

Sarah Garner, PhD, Associate Director for Science Policy and Research, National Institute for Health and Care Excellence, London, UK

Eva-Maria Didden, PhD, Researcher, Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland

PURPOSE:

This workshop will give participants an understanding of important methodological key deliverables achieved by the IMI GetReal team (www.imi-getreal.eu). Part of the project has been devoted to bridging the efficacy-effectiveness gap between treatment outcomes in clinical trial and real-world populations, which includes the development of a predictive modelling framework. With the aid of selected case studies, the workshop leaders will demonstrate how different sources of evidence can be used to predict real-world treatment effects from trial efficacy data.

DESCRIPTION:

Mark Belger will present an approach based on propensity re-weighting of RCT data to more appropriately reflect the baseline characteristics of a real-world population. This approach will be illustrated using a RCT and observational study in Non-Small Cell Lung Cancer (NSCLC). Eva-Maria Didden will present a modelling approach that allows prediction of the effect of a new biologic agent in Rheumatoid Arthritis (RA) patients who will likely receive this agent in the real world of a health care system. The modelling concept relies on the assumption that only trial efficacy data on the new treatment are available, but not yet any observational evidence on its effectiveness. Observational studies on similar biologic RA medications are used to identify treatment decision criteria. Keith Abrams will present an approach to generalizing RCT data to the real world both with respect to population (using propensity re-weighting) and time (using extrapolation of RCT survival data informed by real world data via a Bayesian model averaging approach). This combination of methods will be illustrated with the same NSCLC RCT and observational study. Michael Happich will chair the workshop, and facilitate audience participation with respect to whether various stakeholders could envisage using such approaches in their decision making process, as well as how to communicate the implications of such approaches to especially non-technical decision makers and stakeholders.

Clinical Outcomes Research

16:30 - 17:30
Room: Hall F1 (L0)

W21: DOUBLY ROBUST ESTIMATION FOR COMPARATIVE EFFECTIVENESS RESEARCH: GETTING AN EXTRA SHOT AT CORRECTLY SPECIFYING THE TREATMENT-OUTCOME MODEL

Discussion Leaders:

Patrick Lefebvre, MA, Managing Principal, Groupe d'analyse, Ltée, Montreal, QC, Canada

Maral DerSarkissian, PhD, Associate, Analysis Group, Inc., Los Angeles, CA, USA

Melvin "Skip" Olson, PhD, Head, RWD Strategy and Innovation, Novartis Pharma AG, Basel, Switzerland

Valéry Risson, PhD, MBA, Director, HEOR Excellence, Novartis Pharma AG, Basel, Switzerland

PURPOSE:

 Since randomized controlled trials are not always feasible, observational studies are often relied upon to evaluate the comparative effectiveness of treatments in real-world settings. The ultimate goal of comparative effectiveness research is causal analysis to assess the relationship between a treatment and outcome. Appropriate control of confounding is essential to causal analysis, though correct model specification with respect to covariate selection can be a difficult task. The validity of causal inference based on observational data hinges on this fundamental yet untestable assumption which cannot be verified using statistical tests. Doubly robust (DR) estimation combines a model for the outcome (i.e., outcome regression) and a model for the treatment exposure (i.e., propensity score), offering investigators with two chances to fulfill the correct model specification assumption. Provided one of the models is correctly specified, the DR estimator will be robust to misspecification of the other model. That is, if either the outcome regression model or the propensity score model for the treatment is correctly specified, one can still get unbiased estimates of the association between treatment and outcome.

DESCRIPTION:

 The workshop will consist of four topics. First, confounding bias, model misspecification, general causal assumptions, and different adjustment techniques (including outcome regression and inverse probability of treatment weighting) will be discussed by way of introduction. Second, a conceptual overview of DR estimation will be presented, and estimators with the DR property, including the augmented inverse probability weighted estimator, will be explored. Third, a study using DR estimation to compare the effect of oral versus BRACE therapies on relapse in multiple sclerosis will be presented and discussed. Fourth, audience members will be invited to participate in building DR models (e.g., by suggesting covariates for control) for the case example as workshop moderators run live sensitivity analyses to demonstrate the implementation of DR estimation.

Wednesday, 2 November 2016
8:45 - 9:45
BREAKOUT SESSION
Health Policy Development Using Outcomes Research

8:45 - 9:45
Room: Hall E2 (L0)

W22: VALUE OF INFORMATION ANALYSIS TO SUPPORT COVERAGE WITH EVIDENCE DEVELOPMENT (CED) SCHEMES: INDUSTRY VERSUS PAYER PERSPECTIVE

Discussion Leaders:

Leyla Mohseninejad, MSc, PhD, HEOR Manager, Creativ-Ceutical, Rotterdam, The Netherlands

Saskia Knies, PhD, Policy Advisor Health Economics, National Health Care Institute (ZiN), Diemen, The Netherlands

Mondher Toumi, MD, PhD, MSc, Professor, Public Health Department, Aix-Marseille University, Marseille, France

PURPOSE:

This workshop will discuss the application of value of information (VOI) methods in coverage with evidence development (CED) schemes, where access to the new technology can be only guaranteed in face of new evidence. We will address the policy implications of the VOI methods for both industries and payers in order to support them in setting an optimal CED scheme.

DESCRIPTION:

CED has become increasingly popular to address uncertainties and to secure ongoing coverage for promising technologies at time of launch. VOI provides a robust framework to quantify the uncertainty and to analyze its consequences. However, to date few countries beyond the Netherlands use VOI to inform the nature of evidence to be developed. VOI methods have been recommended in The Netherlands (ZIN) with guidelines developed for implementation. By mimicking a real discussion about a virtual case study between the industry and the payer, we will enlighten participants on challenges and solutions when implementing VOI. Leyla Mohseninejad will represent the industry perspective. She will present a hypothetical case of an economic evaluation followed by VOI results. She will use the VOI to show how evidence is expected to evolve over time in future, making CED valuable for payers. Saskia Knies, the payer representative, will challenge the work presented by industry with regards to barriers in real practice to achieve the expected value. She will point out the remaining gaps in the evidence development scheme suggested by the industry. Mondher Toumi, the academic HTA expert, will conclude the session by balancing the risks and advantages of using VOI methods in CED agreements. He will advise payers and industries on how to optimally use VOI methods in CED schemes. Finally, we will invite the audience to participate in this discussion by providing feedback and sharing their experience on VOI methods and CED schemes.

Economic Outcomes Research

8:45 - 9:45
Room: Hall A (L2)

W23: ALL MODELS ARE WRONG, BUT SOME ARE LESS WRONG: USING A VERIFICATION PROTOCOL TO FIND THESE LESS WRONG ONES ON THE MODEL FARM?

Discussion Leaders:

Maiwenn J. Al, PhD, Assistant Professor, Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, Rotterdam, The Netherlands

Nasuh Buyukkaramikli, PhD, Senior Researcher, Institute for Medical Technology Assessment (iMTA), Erasmus University, Rotterdam, The Netherlands

Rachid Rafia, PhD, Senior Researcher, Sheffield University, Sheffield, UK

Dawn Lee, MMath, MSc, Head of Health Economics Analysis, BresMed Health Solutions, Sheffield, UK

PURPOSE:

The purpose of this workshop is to introduce the audience to model errors/credibility threats, methods for cost effective improvement of model validation/review processes, giving examples from real-life cases. In particular, the workshop will include discussion on how protocols can systemize verification efforts for models and present a new model verification assessment protocol, TECH-VER Protocol 1.0.

DESCRIPTION:

Validation of health-economic models is essential, especially if models are used in decision making. Verification is a fundamental step for model validation, ensuring that the conceptual model is implemented correctly and behaves accordingly to concept without errors. Procedures used for model verification within industry will be presented with focus given to the trade-off between time spent and accuracy. Following this a new operational protocol for model users/reviewers to assess the quality assurance and verification status of the technical implementation for HE decision analytical models will be presented. An initial beta version of the protocol was developed based on the findings from the literature and authors’ previous modelling/appraisal experience. The beta version was iteratively revised based on oral/written feedback from other health economists and after each execution of the protocol on a different model. In this workshop, we will first introduce model error/ credibility threat types through a taxonomy of errors and mitigation strategies to avoid them. Then, we will present the developed protocol, TECH-VER Protocol 1.0. With the help of the examples from implemented cases, we will then demonstrate how the protocol had systemized the verification efforts and helped in the model validation/review process and in identifying model/reporting errors. Finally, the possible added values and cost-effectiveness of such verification protocols will be discussed from different (e.g. academia, consultancy, decision-maker) perspectives with the audience.

Health Policy Development Using Outcomes Research

8:45 - 9:45
Room: Hall F2 (L0)

W24: ADAPTING TO EVOLVING EVIDENCE REQUIREMENTS IN GLOBAL MEDICAL DEVICE DEVELOPMENT PROGRAMS

Discussion Leaders:

Thomas Goss, PharmD, Sr. Vice President, Boston Healthcare Associates, Boston, MA, USA

Mark Campbell, MPhil, Associate Director - Medical Technologies Evaluation Programme, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence, UK, Manchester, UK

Pascale Brasseur, MSc, BSc, Global Health Economics & Reimbursement Director, Spine & Biologics, Medtronic plc, Tolochenaz, Switzerland

Moore Matt, MHA, Director, Global Health Economics and Reimbursement, Edwards Lifesciences, Irvine, CA, USA

PURPOSE:

To discuss changes in clinical and economic evidence requirements for innovative medical technologies and what companies must do to gain market access.

DESCRIPTION:

The global marketplace for medical technology is increasingly focused on value as a prerequisite for market access and reimbursement. The demand for increased evidence to prove clinical or economic value is typically higher in established markets with systematic methods in place to evaluate quality and cost, with a focus on comparative and/or incremental value. Additionally, as risk shifts from payers to providers, more healthcare systems are centralizing purchasing decisions, taking decisions out of the hands of the clinician and seeking to achieve more standardization while balancing cost reduction and quality improvement goals.

Medical device innovators must become more efficient at appraising a product's likely evidence requirements early in development. Considering the likely return on investments in evidence development is becoming a key component in evaluating ongoing development decisions at the portfolio level. Accordingly, innovative medical device companies are enhancing capabilities in evidence-based evaluation to align value determination, demonstration, and capture activities with clinical and economic evidence development. Industry representatives will present case studies using evidence-based value as a key input to portfolio management, product development, and commercial launch processes. Additionally, for innovative devices, Health Technology Assessment bodies increasingly are key stakeholders. An invited speaker from the UK, NICE will discuss how NICE’s approaches to medical technology evaluation can facilitate access through early and sustained engagement with technology developers; a constructive approach to topic selection; flexibility in assessing all relevant information including ‘low-level’ evidence; and investment in addressing evidence gaps which prevent it from making comprehensive recommendations for use. Further, the discussion will examine how medical device innovators are coordinating evidence-based value building activities globally and discuss strategies for integrating evidence development to address the unique features of medical device development.

10:00 - 11:00
BREAKOUT SESSION

10:00 - 11:00
Room: Hall E2 (L0)

W25: SPHERE OF INFLUENCE: RUSSIA'S EMERGING HEALTH TECHNOLOGY ASSESSMENT AND ITS INFLUENCE OVER ACCESS IN CIS COUNTRIES

Discussion Leaders:

Cyrus A. Chowdhury, CEO & Managing Director, CBPartners, New York, NY, USA

Malwina Holownia, MPharm, Senior Health Economist, Moscow Department of Health, Moscow, Russian Federation

Elena Subbotina, MIM, MSc, Consultant, CBPartners, London, UK

PURPOSE:

The purpose of this workshop will be to explore how HTA / pharmacoeconomic policy in Russia influences policy and access decisions on high value / orphan disease therapeutics in CIS countries.

DESCRIPTION:

Although having one of the lowest willingness-to-pay for high-value drugs in the European region, Russia has been characterised as a country with the highest cost-effectiveness threshold within the CIS. Currently, the policy for HTA assessment and methodological developments in Russia are being influenced by several economic and political factors, which include (1) the creation of a common market with harmonised legislation within the Eurasian Economic Commission (ECC), (2) the political shift towards so-called “importozamescheniye” – an increased push from the government to switch to locally produced medicines and support local pharmarmaceutical manufacturers, (3) the ongoing push for more comprehensive HTA methodology for assessing the drugs in the face of a stringent health care budget. During the workshop the participants will be reviewing how such HTA policy developments in Russia may impact policy and decision-making with regard to access for high-value, rare disease therapies in the CIS region. As a market with common rules and a larger population may be of greater interest in the pursuit of access, particular emphasis during the workshop will be paid to explore the current status of HTA policy harmonisation across the countries in the CIS region and the forecasted changes with the establishment of the ECC.


10:00 - 11:00
Room: Hall F1 (L0)

W26: BIOSIMILAR VALUE GENERATION OR VALUE DESTRUCTION? A WORKSHOP DEMONSTRATING UPTAKE TO DATE AND QUANTIFYING SAVINGS MADE

Discussion Leaders:

Michael Drummond, MCom, DPhil, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK bio

Monique Martin, PharmD, MSc, MBA, Managing Director PMA Europe, inVentiv Health, London, UK

PURPOSE:

 Biologics, large molecule drugs derived from living organisms, have made a substantial contribution to health care and have markedly improved the outlook for patients diagnosed with conditions like rheumatoid arthritis, cancer and growth hormone deficiency (GHD). Patents for some of these biologics have now expired or will be expiring soon, allowing the entry of biosimilars. This could potentially allow healthcare systems to offer biologics in a financially more sustainable manner and to use saved funds to provide access to new technologies.

DESCRIPTION:

The workshop will discuss the current paradigm where biosimilars are entering the market in Europe (EU-5), US, Canada and Australia and the issues manufacturers are facing. We will present those biologics where patents have expired, and those nearing patent expiry. A comparison between Market Access and Pricing regulations in Europe and other major HTA markets will be presented and discussed, differences pointed out and implications for manufacturers identified. Then several case studies will be presented in a range of indications based on data from EU-5 countries. Data on general adoption trends, market share development and price changes (originator and biosimilars) will be presented and discussed, demonstrating how varying market conditions can affect market adoption and price levels. The workshop will end by discussing the value of biosimilars to health care in general; We will quantify the savings made from the various examples and discuss with the audience how these funds should be used. A number of options will be provided to facilitate audience participation.

Economic Outcomes Research

10:00 - 11:00
Room: Hall F2 (L0)

W27: GUIDELINES FOR ANALYZING PUBLISHED SUMMARY TIME TO EVENT DATA

Discussion Leaders:

Jack Ishak, PhD, Senior Research Leader, Modeling and Simulation, Evidera, Montreal, QC, Canada

Martin Hoyle, PhD, Associate Professor of Health Economics, Medical School, University of Exeter, Exeter, UK

Arman Altincatal, MS, Senior Statistician, Evidera, Lexington, MA, USA

PURPOSE:

  • To help researchers understand how published Kaplan-Meier curves can be augmented with other statistics (e.g., event counts, numbers at risk, median time-to-event, hazard ratio) to improve estimation of time-to-event data, and
  • To illustrate how various approaches to generating analyzable data from the curves perform in parametric fitting/projection and comparative analyses. 
Examples will be used to demonstrate and compare the various approaches, and to highlight their strengths and limitations. Attendees will be asked to respond to questions based on the presented material via a web-enabled application; a summary of responses will be shown and used for further discussion and clarifications, where necessary.

DESCRIPTION:

 Economic evaluations often rely on published data for model inputs. In particular, time-to-event distributions are often taken from publications and subjected to parametric fitting analyses to project unobserved portions of the curve. A standard, but somewhat crude approach is to perform least-squares regressions on coordinates derived by digitizing the published curves. Two approaches are available, however, to derive, virtual patient-level (VPL) data in the form of interval censored, or specific event/censoring times. VPL data can be used for parametric fitting to project incomplete Kaplan-Meier curves or derive comparative measures in pooled analyses of data from different curves. Analyses can also produce standard errors of parameter estimates, which are used in probabilistic sensitivity analyses. The approach used to generate data from curves can affect the accuracy of derived estimates. For instance, analyses of coordinates using least-squares regression is prone to bias with all types of distributions, while actual event times may be more accurate than interval censored data for log-normal data. Examples will be used to demonstrate the VPL data generation process from curves for which actual patient data are available. Virtual and actual patient data will be analyzed to compare the accuracy of approaches.

13:45 - 14:45
BREAKOUT SESSION

13:45 - 14:45
Room: Hall E2 (L0)

W28: HEALTH-ECONOMIC EVALUATION OF GENOMIC TECHNOLOGIES: WHICH ISSUES ARISE?

Discussion Leaders:

Kjm van Nimwegen, MSc, PhD Student, Department for Health Evidence, Radboud Institute for Health Sciences, Radboud university medical center, Nijmegen, The Netherlands

Rene Sluiter, MSc, PhD Student, Department for Health Evidence, Radboud university medical center, Nijmegen, The Netherlands

Gerardus WJ Frederix, PhD, Post-doc, Julius Centre, Utrecht University, Utrecht, The Netherlands

Sarah Wordsworth, PhD, Associate Professor, Health Economics Research Centre, Nuffield Department of Population Health, University of Oxford, Oxford, UK

PURPOSE:

To present and discuss the issues surrounding the health economic evaluation of genomic technologies. This will be illustrated by evaluations of genomic technologies in both diagnostics and pharmacogenetics.

DESCRIPTION:

Genomic technologies are rapidly advancing and genomic testing is increasingly being discussed for use in clinical practice. However, internationally, adoption rates for innovative technologies vary considerably, which is partly due to a lack of economic evidence. Since technological innovations are known to be a major cost driver in health care, and health care expenditures are rapidly increasing, there is a growing need for health economic evaluation of genomic innovations such as whole-genome sequencing. However, certain specific problems arise in the health economic evaluation of genomic technologies. First, Kirsten van Nimwegen describes the issues in early HTA surrounding genomic diagnostics which might not result in QALY gains. Then, Geert Frederix shows an economic evaluation of whole-exome sequencing in complex paediatric neurology. Subsequently, Rene Sluiter presents a cost-utility analysis of genetic screening as a tool to individualize patient treatment trough medical selection or dose adjustment based on a patient’s genetic profile. Finally, Sarah Wordsworth will reflect on the issues encountered in the presented cases, and elaborate on which issues require special attention if the economic evaluation of genomic technologies is to be successful. Finally, there will be room for discussion in which the audience is encouraged to share their experiences and thoughts on health economic evaluation of genomic technologies.

Use of Real World Data

13:45 - 14:45
Room: Hall F1 (L0)

W29: TRANSLATING VALUE OVER THE LIFECYCLE OF A THERAPY: DEVELOPING EVIDENCE ACROSS MULTIPLE INDICATIONS

Discussion Leaders:

Caroline Solon, MSc, Senior Associate, CBPartners, San Francisco, CA, USA

Meghan Gallagher, Director, Global Health Economics and Outcomes Research, Oncology, Sanofi, Cambridge, MA, USA

Cyrus A. Chowdhury, CEO & Managing Director, CBPartners, New York, NY, USA

PURPOSE:

The purpose of this workshop will be to explore how evidence generation can be optimised across multiple indications pursued over time. The discussion will be conducted based on a series of case studies based on first-hand and secondary experience belonging to the discussion leaders.

DESCRIPTION:

Multi-application healthcare products are increasingly common, and the nature of the different applications of the product is becoming more divergent across disparate indications that deal with varied pathologies and associated healthcare stakeholders. Often, these therapeutics only gain multi-indication labels via post-marketing authorisation expansion - that is, value perception and evidence is developed at different points in time, as is the general availability of evidence for different patient populations. Evidence requirements and decision-making processes for HTA were originally designed for single product, single indication submissions; however, as more products are developed across multiple indications, holistic evidence packages and multi-indication approaches to demonstrating value may offer manufacturers a more efficient evidence generation process and potentially help facilitate access activities over time. Nevertheless, it remains unclear how payers will receive such evidence packages or how far in advance evidence generation strategies must be implemented for holistic packages to be robust and achievable. A variety of topics will be explored in conjunction with perspective shared by the workshop participants, including early consultation on future indication pursuit, phase III clinical study design and implications, and real world evidence generation. Additionally, discussion leaders will explore contradictory value message development and evidence positioning, as well as holistic value story creation and budget impact modelling approaches to mitigate the challenges of multi-indication label evolution. The payer and manufacturer perspective on these issues will be generously discussed by the discussion leaders, and feedback from the audience via written survey and first-hand experience sharing will support a robust workshop.

Patient-Reported Outcomes & Patient Preference Research

13:45 - 14:45
Room: Hall F2 (L0)

W30: MEASURING MULTIPLE MEDICATION ADHERENCE – WHICH MEASURE WHEN?

Discussion Leaders:

Priti Pednekar, MPharm, PhD Student, Health Policy, University of the Sciences, Philadelphia, PA, USA

Tamas Agh, MD, PhD, Principal Researcher, Syreon Research Institute, Budapest, Hungary

Maria Malmenäs, MSc, Director Hemu, HERON Commercialization, PAREXEL International, Stockholm, Sweden

Bryan Bennett, PhD, Director, Patient Centered Outcomes, Adelphi Values Ltd, Bollington, UK

PURPOSE:

 To provide a discussion on the various methods used to estimate adherence to multiple medications (AtMM). Methods currently employed in the AtMM literature will be presented followed by a discussion of pros and cons of the methods and the calculations used to derive the measure of adherence.

DESCRIPTION:

 In a systematic review undertaken by the ISPOR Medication Adherence Special Interest Group identified 151 relevant studies on AtMM. Of these, 81 studies used a form of Morisky adherence scale and 54 used either the proportion of days covered (PDC) or medication possession ratio (MPR), each with varying methods to calculate AtMM. Other measures were also identified and several studies used multiple measures. This workshop will present the statistical methods and identify the different ways of deriving multiple medication adherence measures. . Discussion will be undertaken of whether the measurements affected the results and if there were measurements that provided more accurate results compared to the others. This will be followed by an audience interaction (15 minutes) on the advantages and disadvantages of using different identified methods and calculations. The concluding period will allow for wrap up, opportunities for questions and answers and examine recommendations for the next steps. Presented by the Medication Adherence and Persistence Special Interest Group.

15:00 - 16:00
BREAKOUT SESSION

15:00 - 16:00
Room: Hall E2 (L0)

W31: DETERMINING UTILITY OF MULTI-ATTRIBUTE HEALTH STATES: NEW MEASUREMENT AND ANALYTIC APPROACHES

Discussion Leaders:

Nan Luo, PhD, Associate Professor, Saw Swee Hock School of Public Health, National University of Singapore, Singapore, Singapore

Kim Rand-Hendriksen, PhD, CPsychol, Post-Doctoral Fellow, Department of Health Management and Health Economics, University of Oslo, Oslo, Norway

Mark Oppe, PhD, Senior Researcher, EuroQol Research Foundation, Rotterdam, The Netherlands

Juan Manuel Ramos Goñi, MSc, Senior Researcher, EuroQol Research Foundation, Rotterdam, The Netherlands

PURPOSE:

 This workshop focuses on new measurement and statistical methods for determining the utility of multi-attribute health states such as those defined by preference-based instruments. Workshop attendees will become aware of the challenges in valuation of such health states as well as data analysis and be informed of new approaches to tackle those challenges.

DESCRIPTION:

 Attendees will obtain an overview of recent developments in health-state valuation methods. The workshop will review a) how computer technology can be used to make time trade-off (TTO) procedures easy to administer and understand; b) how to harness computer technology to monitor and improve data quality, and c) new models that may improve the estimation for unmeasured health states. Dr. Nan Luo will demonstrate a new TTO procedure designed for administration with a software program. He will review the development process and discuss with attendees about ways to further improve the procedure. Dr Mark Oppe will introduce a quality control process that has been designed for use in large health-state valuation studies. Dr. Kim Rand-Hendriksen will describe a type of new models for analyzing TTO values of multi-attribute health states defined using similar descriptors. Mr. Juan M. Ramos-Goñi will introduce the concept and rationale of censored models, interval regression models, and ‘hybrid’ models which use both TTO and discrete choice experiment (DCE) data, and demonstrate how to prepare raw valuation data to estimate those models. This workshop will be valuable to researchers who are interested in collecting primary health-state utility data or developing new valuation procedures or preference-based instruments.

Health Policy Development Using Outcomes Research

15:00 - 16:00
Room: Hall F1 (L0)

W32: HOW TO INCORPORATE ECONOMIC EVALUATIONS IN CLINICAL PRACTICE GUIDELINES: A PRACTICAL WORKSHOP ON RESEARCH METHODS

Discussion Leaders:

Jos Kleijnen, MD, PhD, Professor, Kleijnen Systematic Reviews Ltd., York, UK

Ghislaine APG van Mastrigt, PhD, Senior HTA Researcher, Department of Health Services Research, Maastricht University, Maastricht, The Netherlands

Ben Wijnen, MSc, HTA researcher, Department of Health Services Research, Maastricht University, Maastricht, The Netherlands

Frederick Thielen, Msc, HTA Researcher, Institute for Medical Technology Assessment, Erasmus University, Rotterdam, The Netherlands

PURPOSE:

Although it is recommended, economic evaluations (EEs) are not routinely incorporated in clinical practice guidelines. One of the reasons could be lack of knowledge on how to identify EEs and assess their quality by guideline developers. The purpose of this workshop is to provide hands-on training on how to identify relevant EEs and how to evaluate their quality.

DESCRIPTION:

This workshop is based on a series of three papers on “How to Prepare a Systematic Review (SR) of EE for Clinical Practice Guidelines” in which a five-step-approach for conducting a SR of EE is proposed. Participants will first be introduced to the five-step-approach and will be given brief information regarding the several steps. Next, all participants will be asked to develop a search strategy in small groups based on a (pre)defined topic to identify studies regarding clinical effectiveness data combined with a validated search filter for EE identification. There will be guideline experts available to assist them. The most important topic/difficulties encountered in doing these searches will be discussed. Afterwards, an outline will be given on how to evaluate methodological quality of identified EEs and participants will be asked to assess the quality of a predetermined EE. This will be done in small groups supervised by experienced researchers. The last part of the workshop will focus on incorporating EE in clinical guidelines and to on problems/difficulties which one may encounter when performing a SR of EE.

Use of Real World Data

15:00 - 16:00

W33: WITHDRAWN - USE OF ADMINISTRATIVE DATA FROM VARIOUS SOURCES: CASE STUDIES IN DEPRESSION TREATMENT

Discussion Leaders:
PURPOSE:

This session has been withdrawn from the program.

DESCRIPTION:

This session has been withdrawn from the program.

Contact ISPOR @ info@ispor.org | View Legal Disclaimer
© 2018 International Society for Pharmacoeconomics and Outcomes Research.
All rights reserved under International and Pan-American Copyright Conventions.