ISSUES PANELS - SESSION I
Monday, November 7, 15:45-16:45

HEALTH POLICY/HEALTH CARE REIMBURSEMENT/COVERAGE ISSUES

IP1: PAYOR ORIENTED EVIDENCE GUIDELINES
Moderator: Joseph Singer MD, VP, Integrated Research Network, HealthCore, Inc, Wilmington, DE, USA
Panelists: James MacKay MD, Medical Director, Providence Health Plan, Beaverton, OR, USA

ISSUE: To explore and characterize the research requirements of payors in their pursuit of evidence based guidelines and the determination of research needs to support reimbursement criteria for pharmaceuticals, biotechnology products and medical devises on a global basis.

OVERVIEW: This will be the first of three panel group discussions managed by this ISPOR Working Group (Florence, Shanghai and Philadelphia ISPOR meetings) where payors will present their research needs with regards to the development of reimbursement guidelines / criteria. Researchers in the audience will be given an opportunity to interact with panel members after their initial presentations. The recorded discussions will be included in White Papers produced by the Value Based Reimbursement Special Integest Group (Diane Simison, Chair). This panel discussion will run 90 minutes with the initial 60 minutes being presentations from the payors followed by the audience question and answer period.

QOL/PRO METHODOLOGY ISSUES

IP2: MEMORY AND PERCEPTION: THE ISSUE OF RECALL BIAS IN PATIENT-REPORTED OUTCOMES
Moderator: Nancy Kline Leidy PhD, President and CEO, The MEDTAP Institute at UBC, Bethesda, MD, USA
Panelists: Donald E. Stull PhD, Research Scientist, The MEDTAP Institute at UBC, Bethesda, MD, USA; Olivier Chassany, Département de la Recherche Clinique et du Développement Assistance Publique, Hopitaux de Paris Hôpital Saint-Louis, Paris, France

ISSUE: Recall bias in patient-reported outcomes: Fact or fiction?

OVERVIEW: Patient-reported outcomes (PROs) provide essential data for understanding and communicating the value of new pharmaceuticals from the patient's perspective. As their role and importance increase, the validity and reliability of PRO measures and data come under greater scrutiny. Regulatory agencies, such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMEA), rely, to a large extent, on the scientific community for information on the reliability and validity of outcome measures. Concern has been raised about the “best” or “most appropriate” recall period for capturing PROs in pharmaceutical and device trials. This issue has been examined extensively in the survey research field for several decades; only recently has it become a focus in the health outcomes arena. Early findings from this nascent health outcomes research area suggest that there is no single correct answer; magnitude of recall bias varies across PROs, with divergencies particularly apparent in global assessments, health-related quality of life, symptoms (particularly pain), and satisfaction. The complexity of the task, meaning of the event, duration of the recall period, intervening events and patient demographics can each play a role in affecting recall bias. A key question, therefore, is the magnitude of bias present and its impact on trial results and subsequent conclusions. This panel will juxtapose concerns about and implications of recall bias from industry and regulatory perspectives with scientific evidence from the survey and health outcomes literature to inform the burgeoning debate on recall bias in PROs and encourage further empirical work on this important issue.


IP3: SUBMISSION OF PATIENT-REPORTED OUTCOMES TO THE EMEA AND THE FDA: AREAS OF CONVERGENCE OR DIVERGENCE
Moderator: Patrick Marquis MBA, MD, Managing Director, Mapi Values, Boston, MA, USA
Panelists: Laurie Burke RPh, MPH, Director, Study Endpoints and Label Development, Food and Drug Administration, Rockville, MD, US; Giovanni Apolone MD, Head of Translational and Outcomes Research Lab, Mario Negri Institute, Milan, Italy

ISSUE: To discuss FDA and EMEA patient reported outcome (PRO) submissions and reviews. Areas of convergence and divergence will be identified and discussed.

OVERVIEW: The creation of the Study Endpoints and Label Development group (FDA/CDER/OND/SEALD) three years ago and the development of a draft PRO guidance document illustrate the US regulators' commitment towards developing and maintaining consistent criteria review for PRO and other study endpoint evaluations. In the meantime, the EMEA has developed a reflexion paper on health related quality of life. FDA and EMEA have gained experience and examples of industry-reviewer interactions which will be shared in an effort to encourage quality and consistency in the study endpoint review process. During the panel discussion, an emphasis will be put on practical experiences when submitting and assessing PRO data at different stages of the clinical program with a focus on early phases (e.g., development of hypotheses, development and validation of PRO questionnaires, strategy for analysis and interpretation). Convergences and divergences will be underlined. Although most of the recommendations when implementing PROs in global clinical development programs can be agreed upon by different regulators, issues related to definition of concepts, PRO paradigm, PRO as primary end-point, role in evaluation of treatment risk/benefit, cross-cultural issues, and clinical significance, deserve further discussion. A specific focus will be put on oncology.


HEALTH TECHNOLOGY ASSESSMENT ISSUES

IP4: PROGRESS AND UNCERTAINTY IN HEALTH TECHNOLOGY ASSESSMENT
Moderator: Frank Papatheofanis MD, MPH, PhD, Associate Professor, University of California San Diego, San Diego, CA, USA
Panelists: Uwe Siebert MD, MPH, MSc, Director, Cardiovascular Research Program, Harvard Medical School, Boston, MA, USA

ISSUE: To evaluate and compare current definitions of the scope and content of health technology assessments in the US and Europe

OVERVIEW: Health technology assessment (HTA) holds myriad meanings and significance to a wide range of stakeholders. Interest in HTA comes from payers (government and private insurance companies), providers (clinicians and health professionals), policymakers, purchasers, manufacturers, patients and researchers. However, HTA is not universally defined nor consistently applied by these groups. Moreover, inconsistency remains surrounding the methods used to perform a HTA, and the relevance of such methods to those who apply and those who develop and those who apply them. A core agency or organization for monitoring and evaluating HTA content or research does not currently exist in the US or Europe, and assorted parties, with differing incentives, produce such analyses. Consequently, understanding the value of HTA remains obscured because the definition and scope of HTA is unclear to relevant stakeholders and potential exists for conflicting HTA results. The panel will discuss how HTA is currently being performed in the US and Europe, and who the stakeholders are in these settings. The panel will further discuss methodological issues surrounding HTA definition and scope and seek to develop a working definition of HTA that is relevant to stakeholders.


ISSUES ON USE OF HEALTH OUTCOMES RESEARCH INFORMATION BY DECISION-MAKERS

IP5: EVALUATION OF DRUG SAFETY: CAN THE USE OF HEALTH ECONOMICS AND OUTCOMES RESEARCH (HEOR) METHODS PRODUCE BETTER DECISIONS?
Moderator: John Hutton, BSc, BPhil, Senior Research Leader, United BioSource Corporation, London, UK
Panelists: Francis Pang BSc, MSc, PhD, Head of Health Economics, Abbott Laboratories Ltd, Maidenhead, Berkshire, UK; Clive Pritchard PhD, Health Economist, Office of Health Economics, London, UK

ISSUE: Drug safety is evaluated at the time of regulatory approval alongside efficacy and product quality. Regulators make judgments regarding the safety of products without a clear basis and with no formal link to patient or societal preferences. The low probability of a small number of serious adverse events may lead to the rejection of products capable of delivering health benefits to large numbers of patients. HEOR provides a framework within which rational decisions can be made using quantitative data on clinical and social effects and incorporating the preferences of patients. Explicit recognition of the opportunity cost of rejecting “unsafe” products, and a more transparent and integrated debate of efficacy and safety issues, should lead to decisions more in line with social priorities.

OVERVIEW: Drug safety has become a major policy issue with recent high profile cases of drugs being withdrawn from the market when unsuspected longer-term adverse events have occurred. Agencies such as the FDA and its equivalents in Europe are perceived, in some quarters, to have failed to protect the public from dangerous products, through inadequate analysis of data, and the pharmaceutical industry is accused of selectively presenting information. The regulators and the industry argue to the contrary that the fact that the unexpected problems were discovered later means that their systems are working, and that product licenses are modified, or drugs withdrawn, as soon as the safety issues are discovered. Of the many perspectives on this issue the following will be presented by the Panelists: the policy-maker and health system manager's view (Hutton); the view of the industry – minimizing regulatory demands but avoiding costly product withdrawals – (Pang); the patient and societal view (Pritchard). The audience will have the opportunity to challenge these, and introduce other perspectives, in the concluding 30 minute discussion period.


ISSUES PANELS - SESSION II
Tuesday, November 8, 10:45-11:45

HEALTH POLICY/HEALTH CARE REIMBURSEMENT/COVERAGE ISSUES

IP6: DO VACCINES RAISE ADDITIONAL ANALYTIC OR POLICY CHALLENGES?
Moderator: Jeremy Chancellor MSc, Managing Director, Innovus Research (UK) Ltd, High Wycombe, Buckinghamshire, UK
Panelists: Michael Drummond PhD, Vice President, Innovus Research (UK) Ltd, High Wycombe, UK; Joel Calmet MD, MS, Director Public Policy, Aventis Pasteur SA, Lyon, France; Elizabeth Miller OBE, BSc, MB BS, FRCPath, FFPHM, Professor, Immunisation Department, Health Protection Agency, Communicable Disease Surveillance Centre, London UK

ISSUE: To explore whether any additional analytic or policy challenges are raised by vaccines and whether these justify a different policy response.

OVERVIEW: Vaccines are a vital contributor to public health, and their distinct preventive role often complements that of pharmaceuticals. However, in most jurisdictions, decisions about vaccine adoption are made through a process separate from that for pharmaceutical products. There is some evidence that decision-makers apply different criteria when evaluating vaccines and it is possible that vaccines raise different analytic or policy challenges, such as the need to maintain ‘herd immunity' or to prepare for possible pandemics.
Questions to be debated include: How does the cost-effectiveness of vaccines compare with that of medicines more generally? Do standard cost-effectiveness methods capture the full value for money from vaccines? How does the cost-effectiveness of vaccines vary from country to country? What criteria do decision-makers use to evaluate vaccines? Is health economics used in decision making for vaccines (recommendation, reimbursement, pricing)? What use of health economics evidence is made by non-governmental institutions, and do they have specific requirements? Should the economic evidence be different for vaccines than medication? Should variations in cost-effectiveness from country to country affect policy? Should the cost-effectiveness threshold for vaccines be any different from that for medicines in general, and if so, why? What are the main challenges industry faces in generating evidence for different jurisdictions?


QOL/PRO METHODOLOGY ISSUES

IP7: DO WE REALLY KNOW THE DIFFERENCE BETWEEN PROS AND QOL AND WHY IT'S IMPORTANT?
Moderator: Olivier Chassany, Département de la Recherche Clinique et du Développement Assistance Publique Hopitaux de Paris Hôpital Saint-Louis, Paris, France
Panelists: Laurie Burke RPh, MPH, Director, Study Endpoints and Label Development, Food and Drug Administration, Rockville, MD, US; Richard Willke PhD, Senior Director, Global Outcomes Research, Pfizer Inc, Bridgewater, NJ, USA; Pennifer Erickson PhD, Co-founder, O.L.G.A, State College, PA, USA

ISSUE: While the term patient reported outcomes, or PRO, has come into common use, many serious issues remain as to what the term means to the pharmaceutical industry and regulatory agencies and how this differs from the term quality of life. Panelists will discuss alternative types of measures and propose a framework for improving communication between regulators and industry. Examples will drawn from labels of products that have recently been approved by the FDA.

OVERVIEW: Measures that assess the impact of disease and its treatment on patients' lives are increasingly being used to assess pharmaceutical interventions. These measures include many different concepts both generic, multidimensional measures, for example, health status, health-related quality of life, and treatment satisfaction, and specific, single dimension measures, for example, individual symptoms. The FDA has grouped all of these measures of the various concepts, when developed for patient self-report, under the umbrella term "patient reported outcomes" (PRO). Recent experience indicates that this term is misunderstood to represent a general measurement concept and synonymous with the term quality of life, thereby introducing confusion rather than clarity into the discussion about what is being measured and how to interpret the findings. Additionally, a review of product labels indicated that clinician reported outcomes are being used to assess many of the same concepts, frequently without the same level of development and validation work. The panelists will present issues concerning the use of the term PRO and how these issues also pertain to the use of clinician reported outcomes measures. These issues along with the proposed framework aim to generate an interactive discussion about how to improve both clarity of the concepts being measured and quality of how they are being measured.


HEALTH TECHNOLOGY ASSESSMENT ISSUES

IP8: 'FOURTH HURDLE' SYSTEMS: HOW WELL ARE THEY USING HTA IN DECISION-MAKING?
Moderator: Martin Buxton, Professor, Brunel University, Uxbridge, Middx, UK
Panelists: Chris Henshall PhD , Health Technology Assessment International, University of York, York, UK; Clare McGrath BS, Director, Team Leader, Pfizer Ltd, Tadworth, Surrey, UK; John Hutton BSc, BPhil, Senior Research Leader, United BioSource Corporation, London, UK

ISSUE: What are the ways of describing and comparing the operation in theory and in practice of so-called 'fourth hurdle' and related systems concerned with determining the availability and reimbursement of pharmaceuticals and other technologies in different countries?

OVERVIEW: To date no systematic, internationally applicable set of descriptors has been developed to analyse and compare '4th hurdle' systems. The panel will present a project aimed at filling this gap and will discuss the usefulness and potential implications of the tool. The extent to which these systems make appropriate use of scientifically valid approaches to evidence including health technology assessment (HTA)is an important component of 'fourth hurdle' operations. Failure to use research methods such as HTA appropriately may diminish or nullify its impact and put its credibility at risk. Decisions about availability of health technologies need to be aligned with the objectives of the whole health system such as impact on public health or system finances and the expectations of users. The capacity to conduct HTA is limited in governments and industry alike. What reforms would therefore be required to ensure that the use of evidence and decision making is conducted in a way that will meet the expectations of all stakeholders and make proper use of research resources? What should be expected from the industry and governments? Is there a way to reconcile the different perspectives?


ISSUES ON USE OF HEALTH OUTCOMES RESEARCH INFORMATION BY DECISION-MAKERS

IP9: THE QUALITY OF PHARMACO-ECONOMIC REIMBURSEMENT DOSSIERS
Moderator: Annemarie Jansen PhD, Senior Policy Advisor, Health Care Insurance Board (CVZ), Diemen, Netherlands
Panelists: Andreas Engstrom, Health Economist, Pharmaceutical Benefits Board, Solna, Sweden; Karen Lee MA, Health Economist, CCOHTA, Ottawa, Ontario, Canada; Anna Bucsics MD PhD, Main Association of Austrian Security Institutions, Vienna, Austria

ISSUE: To compare the quality of pharmacoeconomic reimbursement dossiers in European countries and Canada, and to investigate how to improve the quality

OVERVIEW: In many European countries and Canada, cost-effectiveness studies are part of the drug reimbursement process. In order to encourage uniformity between pharmacoeconomic (PE) evaluations and the quality of evidence submitted, guidelines were developed and are being used by reimbursement authorities. Although requirements for submitted economic evaluations are described in each countries' guidelines, problems are encountered when putting them into practice. Recently it was concluded in a Swedish publication that the average quality of reimbursement dossiers is not optimal. This is a similar experience for other countries. The conduct of high quality PE submissions appears to be an issue regardless of the size and location of the country – the manpower and budget of companies' affiliates do not appear to be predictors of submission quality. In the panel, we will discuss shortcomings in PE dossiers in European countries and Canada, and we will discuss ways to improve the quality.
 

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