Issue Panels - Tuesday, 23 October



Issue Panel - Session I - 8:30 - 9:30
USE OF HEALTH ECONOMIC / PHARMACOECONOMIC INFORMATION BY DECISION-MAKERS ISSUES
IP1	PHARMACOECONOMICS IN IRELAND, SCOTLAND AND WALES: A CELTIC PERSPECTIVE ON THE APPRAISAL OF PHARMACEUTICALS
	Moderator: Mike F Drummond, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK
	Panelist(s): Dyfrig Hughes, PhD, Senior Research Fellow, University of Wales, Bangor, UK; Máirín Ryan PhD, Director of Health Technology Assessment, Health Information and Quality Authority, Dublin, Ireland; Keith Tolley, MPhil, Research Director, Mapi Values, Bollington, Cheshire, UK
	ISSUE: Pharmaceutical appraisals in Ireland, Scotland and Wales: what do they have in common, and where do they differ?
	OVERVIEW: Pharmacoeconomic activity in Ireland, Scotland and Wales has grown significantly in recent years – primarily as a result of devolution in Scotland and Wales, and an agreement between the Pharmaceutical Healthcare Association and the Department of Health and Children, in Ireland. The Scottish Medicines Consortium (SMC) provides advice on all newly licensed medicines, all new formulations of existing medicines and new indications for established products. This advice is based on an appraisal of evidence on clinical- and cost-effectiveness submitted by the drug manufacturer. Multiple Technology Appraisals conducted by the National Institute for Health and Clinical Excellence (NICE) may supersede SMC advice. The All Wales Medicines Strategy Group (AWMSG) has a remit that is limited to medicines which cost more than £2,000 (€3,000) per patient per year, and medicines for oncology and cardiovascular diseases. AWMSG guidance is superseded by all NICE guidance. The National Centre for Pharmacoeconomics in Ireland reviews the cost-effectiveness and budget impact of individual drugs in the Irish healthcare setting in response to requests from the Department of Health & Children. This issue panel will discuss the commonalities and differences between the Celtic nations in their approaches to the appraisal of pharmaceutical products, including their views on orphan and ultra-orphan drugs, their remits and scope, speed of assessment, and evidence for differences in decisions.

IP2	WHAT IS THE ROLE OF COST-EFFECTIVENESS DATA IN THERAPEUTIC INNOVATION AND INEQUALITIES OF HEALTH CARE?
	Moderator: John Hutton, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK
	Panelist(s): Uwe Siebert, PhD, MPH, MSc, Professor of Public Health, University for Health Sciences, Medical Informatics and Technology, Vienna, Austria; Mark JC Nuijten, MD, PhD, MBA, Consultant, Ars Accessus Medica/Erasmus University Rotterdam, Amsterdam, The Netherlands; Elly A. Stolk PhD, Senior Research Associate, Institute for Medical Technology Assessment, Erasmus Medical Center, Rotterdam, The Netherlands
	ISSUE: As cost-effectiveness data is gaining importance in the overall health care decision making process, it is justified to scrutinize this criterion in more detail, looking at some of the current and future consequences for the society as a whole. A clinically innovative drug may not be reimbursed because of an unfavorable assessment of its cost-effectiveness, a method based on undisclosed CE threshold which also lacks of evidence and it is weakly connected to individual/societal preferences through the indirect representation of their policymakers. Cost-effectiveness outcome is considered as real evidence by decision makers, increasingly becoming the sole decision criterion that affects patients' lives, medical communities' capacity to deliver optimal health care, and equal and fair access to present and future therapeutic innovations. The sole use of CE outcomes with no explicit and evidence-based thresholds is ignoring some of our most appreciated societal values: fair access to care, individual preferences and transparency in policy decision making.
	OVERVIEW: The panel will discuss theoretical and practical elements for and against the use of CE-data as final decision criterion in health care reimbursement decisions, and explore three still conflictive dimensions: the potential methodological strengths and flaws of the CE concept, the individual preferences ‘as a decision-driver' and the equality of access to care. Key questions to be discussed are: what should be the role of CE data in overall decision making process? Should societal values and preferences be included in the process? Is the lack of explicit CE-thresholds the evidence of the inappropriate consideration of individual's preferences? How CE-dominated decision-making is affects access to innovative therapies in Europe? How can we ensure the maximum efficiency of our current health systems, while ensuring access to optimal care and future innovations? How should we deal with the value of information and research by adopting innovative technologies?

CLINICAL OUTCOMES/TREATMENT GUIDELINES ISSUES

IP3	INSTITUTIONAL DEVELOPMENTS IN HEALTH TECHNOLOGY ASSESSMENT AT MICRO AND MACRO LEVELS – IS RAISING STANDARDS THROUGH COMMON METHODOLOGIES AN ISSUE?
	Moderator: Finn Boerlum Kristensen, MD, PhD, Director, National Board of Health, Copenhagen, Denmark
	Panelist(s): Cari Almazan, Catalan Agency for Health Technology Assessment and Research (CAHTA), Barcelona, Spain; Americo Cicchetti, PhD, Professor of Health Care Management, Catholic University of Sacred Heart, Rome, Italy;Harri Sintonen, PhD, Professor, Department of Public Health, University of Helsinki, Helsinki, Finland
	ISSUE: The internationalization of Health Technology Assessment is a response to similarities in health policy and decision making issues across borders. Will overviews of structures and work processes - and common developments of standards – better enable the sharing of methodological frameworks, questions, and, sometimes, results?
	OVERVIEW: The establishment, aims, background, structure, work process and visibility of existing national / regional / local Health Technology Assessment (HTA) institutions has been surveyed by Work Package (WP) 8 in the EUnetHTA Project. An analytic overview of institutions can be established from the point of view of "young agencies/units" (from 2000 to 2007), the "experienced ones" (1990-1999) and the "oldest ones" (1956-1989) in order to describe components related to e.g. scope, structure, and work processes. HTA principles and tools are increasingly used in hospitals and other health care organizations (HCOs) in order to inform both managerial decision-making and clinical practice. The decentralization process occurring in the HTA movement is an emerging phenomenon, and evidence is increasing on the application at HCO level. The phenomenon has been surveyed internationally by an HTAi Special Interest Group on Hospital–Based HTA. In order to contribute to raising standards through common methodology two models (one for medical and surgical interventions and the other for diagnostic technologies) are created in WP 4 of the EUnetHTA project to capture the "core of HTA" that can also be utilized elsewhere than in the setting in which the HTA was originally produced. The models imply that a standard set of issues should be considered in future more transferable HTAs. Does the need for international utilization of HTAs warrant a standardization of HTAs? Is the benefit worth the workload to agencies? Can duplicate work be decreased?

HEALTH CARE REIMBURSEMENT / COVERAGE ISSUES
IP4	REIMBURSEMENT DECISION PROCESSES IN EUROPE – IS THERE A POSSIBILTY OF PARTIALLY HARMONIZED APPROACH?
	Moderator: Fabian Waechter, MD, MSc, Medical Assessor, Project Manager, AGES PharmMed - the Austrian Medicines Agency, Wien, Austria
	Panelist(s): Richard Bergström, Vice Director, LIF Sweden, Stockholm, Sweden
	ISSUE: To evaluate whether there is a chance to harmonize the many different Reimbursement processes so that applications become more practicable for the industry and that assessment work is not conducted multiple times in parallel by different European Authorities.
	OVERVIEW: In recent years Reimbursement decision processes have become more important in many European countries. Due to the heterogeneous nature of these decision processes in different countries a lot of confusion has arisen. In the light of these confusions the European Union has started a couple of initiatives to gain knowledge on this issue as well as to start a process of harmonization. The first panelist will outline the industry perspective on reimbursement decisions. He will describe in depth the problems that arise for the pharmaceutical industry. In particular how the industry, due to the very different approaches, has to duplicate work theoretically up to twenty-seven fold to get reimbursement for a drug in all EU member states. Then the other panel presenter will start with a brief overview of the European legal background regarding Licensing versus Reimbursement decisions. In EU Regulation No 2309/93 most aspect of drug development are outlined. While both processes are subject to certain timelines, the Reimbursement decision process stays a national member state competency. Then he will depict the overall process from market approval (first to third hurdle) to the Reimbursement decision (fourth hurdle). He will then start to explain the general European approach to the fourth hurdle in a generic way. He will then present and discuss what elements should and could be part of these processes. And make proposals how, while keeping the actual decision a national issue, the EU members states could simplify the process to the benefit of all stakeholders by agreeing on certain standards.

HEALTH POLICY ISSUES

IP5	VALUE-BASED PRICING: SOLUTION OR ILLUSION?
	Moderator: Mark Sculpher, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK.
	Panelist(s): Martin Buxton, BSc, Professor of Economics and Director, Health Economics Research Group, Brunel University, London,UK; William Comanor, PhD, Professor, Department of Health Services, UCLA School of Public Health, Los Angeles, CA, USA; John O'Donnell, PhD, Sr. Director Health Policy, External Medical Affairs, AstraZeneca Pharmaceuticals, Wilmington, DE, USA.
	ISSUE: Should jurisdictions in Europe move toward a system whereby medicines are priced according to the value they offer to the healthcare system?
	OVERVIEW: An increasing number of jurisdictions in Europe are requesting assessments of the cost-effectiveness (i.e. value for money) of new products as part of the pricing and reimbursement decisions. However, in most jurisdictions, the decision is made whether or not to reimburse (on the appropriate conditions for reimbursement) given a particular price or prior to a price being set. The price might be the one proposed by the manufacturer, or agreed by another committee applying a different set of criteria. However, a recent report of the Office of Fair Trading (OFT) in the UK argued that the two decisions (reimbursement and pricing) should be linked. Namely, pricing and reimbursement should be value-based. Whether this would, on average, lead to lower or higher prices in Europe, is open to debate. However, it has already been suggested that, given lower prices in Europe (as compared with the US), Europe is already not paying its full part in funding innovation.

Issue Panel - Session II - 10:00 - 11:00
USE OF HEALTH ECONOMIC / PHARMACOECONOMIC INFORMATION BY DECISION-MAKERS ISSUES

IP6	THE HTA “REVOLUTION” IN EUROPE: THE FUTURE IS BRIGHT, THE FUTURE IS COST-EFFECTIVENESS?
	Moderator: Keith Tolley, MPhil, Research Director, Mapi Values, Bollington, Cheshire, UK
	Panelist(s): Carole Longson, PhD, Director, National Institute for Health and Clinical Excellence (NICE), London, UK; Michael Barry, PhD, MD, Clinical Director, National Centre for Pharmacoeconomics, St. James’s Hospital & Senior Lecturer in Clinical Pharmacology, Trinity College Dublin, Dublin, Ireland; Hilda Bastian, Head of Department; Health Information, Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
	ISSUE: To investigate whether in the future formal cost-effectiveness methods will be routinely applied to appraise new pharmaceuticals seeking market access in European countries?
	OVERVIEW: A number of countries in Europe already use formal cost-effectiveness evidence to support reimbursement and pricing decisions. For this issue contrasting perspectives will be put forward from panelists covering countries with institutions that perform appraisals of pharmaceuticals and other technologies on behalf of their respective Ministries of Health, but with differing approaches to the use of formal cost-effectiveness methods. In the UK cost-effectiveness evidence is routinely used by NICE, adopting a cost-utility ‘reference case' approach, and indirect comparisons performed where necessary to compare new interventions with the current standard. In Germany cost-effectiveness evidence has not been routinely used to date in IQWiG appraisals, although new plans to take greater account of such evidence have been put in place. But what form should that data take to inform decisions? The requirements for cost-effectiveness evidence in Ireland and the rapid HTA approach adopted in this country provide a further contrast with the UK and German perspectives. The role of pharmaceutical companies and consultancy agencies in the evidence generation, submission and review process and the future use of economic evaluation methods and HTA in UK, Germany and Ireland and other countries around Europe will be a key topic for audience debate. Also, the issue of timeliness of cost-effectiveness evidence in relation to product launch will be debated. There will be discussion as to whether the “NICE approach” to cost-effectiveness is likely to be increasingly adopted for guideline development or reimbursement purposes in other European countries, and whether applying explicit cost-effectiveness thresholds are useful.

HEALTH POLICY ISSUES
IP7	THE GERMAN WAY IN DEFINING AND APPLYING COST-EFFECTIVENESS-ANALYSES – REFLECTIONS FROM INTERNATIONAL EXPERIENCE
	Moderator: Johann M Von der Schulenburg, PhD, Professor of Health Economics, Leibniz University Hannover, Hannover, Germany
	Panelist(s): Peter T Sawicki, MD, Director, Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany; John Hutton, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK; Ulf Persson, PhD, Program Director, The Swedish Institute for Health Economics, IHE, Lund, Sweden
	ISSUE: Is the German process in developing standards for CEA based on and informed by international standards and experience in the application of health economic methods?
	OVERVIEW: This panel takes place at a time of significant change in the German health care system. The Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) has been around for three years and by law its remit is now being extended to include economic assessments. There has been an extensive discussion on what to acknowledge as current international standards for the economic evaluation of health technologies and assessing their relevance in the German setting. Among the most contentious items are the perspective for the analysis, the measure of health gain (e.g., QALY), and the role of economic modelling. Especially on the latter, there is some hesitation over its use, since additional assumptions would have to be made, in extrapolating beyond the data observed in randomised controlled trials. The introduction of economic assessment should not only be informed by the mere selection of appropriate methods but also by experience from other countries in processing these methods. In considering methodologies, it is apparent that, although methodological guidelines in the various jurisdictions are fairly similar, there are also key differences. Looking at processes, although no national HTA programme is without its problems, the most widely accepted ones appear to be those that encourage broad stakeholder involvement and have a transparent process. This issue panel aims at reflecting on experience, both good and bad, from two countries, Sweden and the UK, that have already implemented a requirement for the economic evaluation of new health technologies. With that in mind German methods will be discussed in the panel.

IP8	DISPARITIES IN ACCESS TO CANCER CARE – THE KAROLINSKA REPORT
	Moderator: Frans Rutten, PhD, Professor, Institute for Medical Technology Assessment (iMTA), Rotterdam, The Netherlands
	Panelist(s): Nils Wilking, MD, PhD, Associate Professor, Karolinska Institute, Stockholm, Sweden; Bengt Jönsson, PhD, Professor, Stockholm School of Economics, Stockholm, Sweden; Mark JC Nuijten, MD, PhD, MBA, Consultant, Ars Accessus Medica/Erasmus University Rotterdam, Amsterdam, The Netherlands
	ISSUE: The Karolinska reports that despite the proven benefits of new innovative treatments options, patients across Europe do not have equality of access to these cancer drugs and the speed at which patients can benefit from them depends to a great extent upon the country in which they live. Patients have to wait too long to obtain the benefits of newer therapies and the biggest hurdle to the uptake of new drugs is the proactive allocation of financial resources and budget in the health care systems by policy and decision makers. Different patterns of uptake were seen in the countries studied, both with respect to speed of uptake and level of use. For some of the most recently approved drugs the variation in uptake is especially marked. The vintage of the cancer drug “arsenal” used also differs significantly between countries. For example, around one sixth of the inter-country differences in 5-year cancer survival rates across 5 major European countries is due to differences in the uptake of newer drugs (post-1985) in each country.
	OVERVIEW: The panel will discuss differences in the health care decision making process between the countries explaining the observed differences in patient's access to innovative cancer drugs. What are the disparities? Why these disparities exist and what are the major drivers? How to close the gap among EU countries? What should be the role of CE data in overall decision making process? Should societal values and preferences be included in the process? Should we take a broader perspective on the benefits and costs of oncology these drugs bring to patients and society, and introduce a more rational system of allocation of resources to the healthcare system?

PHARMACOECONOMIC / HEALTH ECONOMIC STUDY METHODOLOGY ISSUES
IP9	IMPLEMENTING "GOOD REGISTRY PRACTICE" - NEWLY DEFINED BEST PRACTICE RECOMMENDATIONS FOR THE DESIGN, USE, AND EVALUATION OF PATIENT REGISTRIES
	Moderator: Richard Gliklich, MD, President, Outcome, Cambridge, MA, USA
	Panelist(s): Kathryn Starzyk, MS, Associate Director of Scientific Affairs, Outcome, Cambridge, MA, USA; Sarah Garner, PhD, MRPharmS, Technical Adviser, National Institute for Health and Clinical Excellence (NICE), London, UK
	ISSUE: Growing uses of patient registries, the state of “registry science” and evaluating quality in registry operations, data, and interpretations: Are the newly defined best registry practice recommendations relevant in Europe as well as the U.S. and are they achievable?
	OVERVIEW: Physicians, payers and patients are demanding an increasing amount of real-world evidence surrounding the results of medical products, therapies and services in larger and more diverse populations. Whether disease-based or product-focused, registries provide real-world data on the safety and effectiveness of a product and/or insight into the natural history of a disease under standard care practices. Unlike randomized clinical trials, there are no consensus standards for how registries should be developed, managed or evaluated. In October 2005, the U.S. Health and Human Services' Agency for Healthcare Research and Quality launched an 18 month project to collaboratively create a handbook “Registries for Evaluating Patient Outcomes: A User's Guide” (released in May 2007). Speakers will present the background which led to this project, the various stakeholders, and the process undertaken which included external review and several periods for public commentary for outlines as well as draft documents. They will describe the key learnings in creating, operating and evaluating patient registries and share relevant case studies. Speakers will provide information to participants on expectations of registry consumers for different registry purposes and how best to meet those expectations. They will provide an explanation of the framework for evaluating “quality” in registries and the range of recommendations from “sufficient” to best practices and how these recommendations may vary for different purposes. Better planning and design of registries will enable these tools to be used more successfully in support of regulatory goals.

MEDICAL DEVICE, DIAGNOSTICS AND BIOTECHNOLOGY ISSUES
IP10	ARE MEDICAL DEVICES MORE CHALLENGING TO EVALUATE THAN DRUGS?
	Moderator: Mike F Drummond, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK
	Panelist(s): Rosanna Tarricone, DrPH, Economic Affairs Director, EUCOMED, Brussels, Belgium; Adrian Griffin, MSc, BSc, Director of Health Outcomes, Johnson & Johnson, High Wycombe, Buckinghamshire, UK; Rod Taylor, PhD, MSc, Reader, PenTAG, Peninsular Medical School, Exeter, UK
	ISSUE: Are medical devices more challenging to evaluate than drugs?
	OVERVIEW: To inform reimbursement/coverage/guidance decisions, many health care systems are assessing the effectiveness and cost-effectiveness of medical devices in a similar manner to their assessments of pharmaceuticals. However, are there important differences between devices and medicines that make the health technology assessment methods used for medicines maybe inappropriate for devices? A range of potential challenges to the assessment of medical devices will be considered including: sources for outcome and cost data (experimental versus real world data, relevance versus reliability); shorter product life cycle compared to pharmaceuticals; medical devices being only one of the components of interventional procedure; and learning curves and medical devices.