ISPOR 15th Annual European Congress: Issue Panel Presentations
 
ISSUE PANEL PRESENTATIONS
 
ISSUE PANELS - SESSION I
MONDAY, 5 NOVEMBER 2012: 11:00-12:00
IP1: ROADMAP OF HTA IN EUROPE – MOVING HTA FORWARD OR JUST STUCK IN TRAFFIC? (ISPOR Invited Issue Panel)
Moderator:

J.L. (Hans) Severens, PhD, Professor of Evaluation in Health Care, Institute of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, The Netherlands

Panelists:

Finn Borlum-Kristensen, MD, PhD, Professor, Health Services Research & Health Technology Assessment, University of Southern Denmark and Director, EUnetHTA Secretariat, Danish Health and Medicines Authority, Copenhagen, Denmark; Andrzej Rys, MD, Director of Health Systems and Products, European Commission, Brussels, Belgium; Gordana Kalan Živčec, MD, Board Member, Standing Committee of European Doctors (CPME) and President of the Medical Chamber of Slovenia, Ljubljana, Slovenia

ISSUE:

This year the European network for Health Technology Assessment (EUnetHTA) moves into the decisive phase of Joint Action 2 to facilitate the constructive uptake of collaboratively developed HTA tools and information into the practices of the national HTA organisations. In parallel, the European Commission and EU Member States prepare the establishment of a sustainable, voluntary European HTA network in accordance with the Directive on patients’ rights in cross-border healthcare. How will these developments add value to health care decisions in Europe? With emerging health care cost containment policies being put in place during this austere economic period, will this HTA collaborative assure access to quality health care throughout Europe? What are the success opportunities and threats in the current situation and in the near future? 

OVERVIEW: The current health policy challenges across Member States and challenges facing EUnetHTA will be presented. The benefit of this collaboration to health care quality and access will then be debated.
IP2: IS THERE SCOPE FOR MORE HARMONIZATION OF PROCEDURES FOR ASSESSING ORPHAN DRUGS IN EUROPE?
Moderator: Michael Drummond, PhD, Professor of Health Economics, Centre for Health Economics, University of York and Principal Consultant, OptumInsight, Heslington, York, UK
Panelists: Amy K. O'Sullivan, PhD, Director, Health Economics and Outcomes Research, OptumInsight, Medford, MA, USA; Michael Barry, MD, PhD, Clinical Director, National Centre for Pharmacoeconomics, St. James's Hospital, Dublin, Ireland; Francis Pang, MSc, MPhil, Senior Director of Market Access and Public Affairs, Shire Human Genetic Therapies, Basingstoke, UK
ISSUE:

The level of access to orphan drugs differs across European Union (EU) member states. Although differences in access reflect, in part, different member states’ ability to pay for health care, there are also important differences in how orphan drugs are assessed. In some countries they are assessed in the same manner as conventional treatments; in others they are given special consideration. How are orphans treated by HTA agencies across the EU?  What factors contribute to reimbursement decisions? Would there be benefits from greater collaboration within the EU in orphan drug policies?  The moderator will present an overview of the issue, and set the stage for debate; Amy O’Sullivan will present, from a research perspective, a review of different HTA approaches for orphans within the EU; Michael Barry will represent the decision maker’s perspective in Ireland, where orphan drugs are considered similarly to other drugs; and Francis Pang will provide an industry perspective, drawing on his experience of how orphan drugs are assessed in different European countries.

OVERVIEW:

Orphan drugs differ from conventional medicines in that they are used to treat extremely rare conditions for which few treatments are available. Pharmaceutical companies have less financial incentive to develop orphan drugs, as target populations are too small to recoup R&D costs. Therefore, when orphan drugs do enter the market, they are priced relatively high in efforts to recover at least some of the economic investment.  High acquisition costs often yield unattractive cost-effectiveness ratios for orphan drugs, irrespective of efficacy. While HTA requirements for conventional treatments are clear in many countries, requirements for orphan drugs are less so.  The panel will review HTA processes for orphans across the EU, consider the alternative approaches for assessing orphans, and debate the possibilities for more harmonization and collaboration in orphan drug policies in Europe.

IP3: HOW TRANSFERABLE IS CLINICAL EVIDENCE: DOES ONE-SIZE FIT ALL WHEN ASSESSING THE RELATIVE EFFECTIVENESS OF PHARMACEUTICALS IN EUROPE?
Moderator:

Keith Abrams, MSc, PhD, Professor of Medical Statistics, Department of Health Sciences, University of Leicester, Leicester, United Kingdom

Panelists:

Panelists: Antje Behring, PhD, Desk Officer, Pharmaceuticals Department, Federal Joint Committee (G-BA), Berlin, Germany; Elisabeth George, PhD, Associate Director, Technology Appraisals, National Institute for Health and Clinical Excellence, London, UK; Clare McGrath, PhD, Global Head, HTA Policy, AstraZeneca, Greater Manchester, UK

ISSUE: The three panellists will discuss the incentives and challenges of harmonising the relative effectiveness assessment of new medicines in the context of HTA from the perspectives of a national reimbursement decision-maker, HTA agency and EUnetHTA partner, and the pharmaceutical industry.
OVERVIEW:

Health technology assessment (HTA) harmonisation efforts throughout Europe, such as the European network for Health Technology Assessment (EUnetHTA) Joint Action, have focused on developing ‘core’ sets of methodological standards to improve common review processes and alleviate the local evidential and financial burden of HTA. Often assimilated to comparative effectiveness research in the US, the relative effectiveness assessment (REA) of pharmaceuticals is an integral part of the HTA process and directly informs reimbursement decisions, particularly in countries such as Germany where a cost-effectiveness analysis is not required for submission. The purpose of this session is to discuss the importance, feasibility, and limitations of a standardised REA to support national and regional HTA efforts within the European Union. Findings from the EUnetHTA REA public consultation and pilot assessment, as well as recent work from the University of Leicester in REA methodologies, will be used to initiate discussion. The three panellists will speak on issues including: (i) the incentives for harmonisation from a reimbursement decision-maker,  HTA agency, and pharmaceutical industry perspective, (ii) the transferability of clinical evidence across countries, (iii) the limitations of a ‘one-size’ fits-all model, and (iv) the evidential and methodological challenges a standardised process may entail for stakeholders.

IP4: HOW IS IQWIG MEETING THE CHALLENGES OF THE NEW GERMAN HEALTH CARE REFORM?
Moderator: Alric Ruether, MD, PhD, Head, Department of Health Care Quality, Institute of Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
Panelists:

Andreas Gerber, PhD, MD, Head, Department of Health Economics, Institute of Quality and Efficiency in Health Care (IQWiG), Cologne, Germany, Stefan Sauerland, MD, MPH, Head, Department of Non-Drug Interventions, Institute of Quality and Efficiency in Health Care (IQWiG), Cologne, Germany; TBD, German Ministry of Health, Berlin, Germany; Jos Kleijnen, MD, PhD, Owner, Kleijnen Systematic Reviews, Professor, Systematic Reviews in Health Care, Maastricht University and Clinical Professor, Joanna Briggs Institute, Faculty of Health Sciences, University of Adelaide, York, UK

ISSUE: In 2004, the Institute for Quality and Efficiency in Health Care was created (IQWiG) as a consequence of a German Health reform. The Institute is responsible for assessing the benefits and harms or the costs of medical interventions. In Germany, the Institute is well established as an independent provider of high-quality information necessary for German decision makers in health care. However, is the information provided adequate and timely for the German health care decisions?
OVERVIEW: During this Issue Panel, representatives from IQWiG will focus on the latest challenges conducting health technology assessment (HTA) within the new German health care reform, AMNOG (such as coverage of medical devices with evidence development). A representative from the Ministry of Health (the final German decision-maker concerning reference pricing groups and reimbursement) will then provide their perspective on the health care information available to make care decisions. In addition, an independent expert in health care systems and international developments from outside Germany will provide his perspective on HTA and health care decisions. The future role of HTA agencies in the context of health care decision making will then be discussed, especially in the context of international developments towards cross-border HTA networks.
IP5: POLICY STRATEGIES UNDER AUSTERE CONDITIONS IN EUROPEAN COUNTRIES - DOING LESS WITH LESS AT WHAT COST? (ISPOR Invited Issue Panel)
Moderator:

Carolin Miltenburger, PhD, Senior Director, Global Health Economics & Reimbursement, Medtronic, Tolochenaz, Switzerland

Panelists:

László Gulácsi, MD, PhD, Professor, Head, Health Economics and Health Technology Assessment Research Centre, HunHTA, Corvinus University of Budapest, Budapest, Hungary; Mary Geitona, PhD, Professor, School of Social Policy, University of Peloponnese, Korinth, Greece; Pedro Pita Barros, PhD, Professor of Economics, Nova School of Business and Economics, Lisbon, Portugal; Niek S. Klazinga, MD, PhD, Professor & Chair, Department of Social Medicine, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands and Senior Policy Advisor & Head, Health Care Quality Indicator Programme, Organization for Economic Co-Operation and Development (OECD), Paris, France

ISSUE: Health economics and HTA have developed to assist decision making under sustainable growth. However, for many European countries, health care budgets have been drastically cut, due to economic crisis, resulting in a focus primarily on costs. In these countries, it is not clear what opportunity exists for stakeholder engagement, prioritization and the use of technology value information in health care decisions. What implications will this economic crisis have on the use of HTA, including technology value information, on health care decisions in Europe?  What are the implications on the quality and safety of patient care? Finally, what implications will this development have for harmonization and convergence of HTA and health economics in Europe?
OVERVIEW:

This panel will present the challenges faced by several EU country-specific health care systems (as examples) to operate under austerity conditions, while aiming to maintain access and quality of health care services. The different cost containment approaches applied to pharmaceuticals, other technologies, and health care services will be presented. The cost containment approaches and their implications on public health, quality and safety of patient care will then be debated.

ISSUE PANELS - SESSION II
TUESDAY, 6 NOVEMBER 2012: 13:45-14:45
IP6: REWARDING THE VALUE ADDED BY NEW DRUGS: WHICH OF TWO EUROPEAN APPROACHES IS SUPERIOR?
Moderator: Matthias Graf von der Schulenburg, PhD, Director, Institute for Risk and Insurance, Leibniz University, Hannover, Germany
Panelists: Elizabeth C Jones, MSc, Senior Research Analyst, HEOR, OptumInsight, Uxbridge, UK; Gerard de Pouvourville, PhD, Professor, Department of Health Care Management and Chair, Health Economics Institute, ESSEC Business School, Cergy Pontoise, France; Michael Drummond, PhD, Professor of Health Economics, Centre for Health Economics, University of York and Principal Consultant, OptumInsight, Heslington, York, UK
ISSUE: Within Europe, two contrasting approaches have emerged for rewarding the value added by new drugs. In the Netherlands, Sweden and the UK, the price of, and access to, a new drug has to be justified by the health gain it delivers compared with current therapy, typically expressed as the incremental cost per quality-adjusted life-year (QALY) gained. By contrast, in France and Germany, the assessment of added benefit is expressed on an ordinal scale ( eg 1-5), based on an assessment of the added clinical benefit as compared with existing care. This assessment of added benefit then influences the subsequent price negotiations. The existence of these contrasting approaches raise questions about the pros and cons of each, in terms of the costs, timeliness, practical feasibility and transparency of the processes involved and the resulting outcomes in terms of patient access to, and price of, the drugs concerned.
OVERVIEW:

Two contrasting approaches have emerged for rewarding the value added by new drugs in Europe. In some countries, the price of, and access to, a new drug has to be justified by the health gain it delivers compared with current therapy, typically expressed as the incremental cost per quality-adjusted life-year (QALY) gained. By contrast, in other countries, the assessment of added benefit is expressed on an ordinal scale ( eg 1-5), based on an assessment of the added clinical benefit as compared with existing care. Elizabeth Jones will adopt a research perspective and report on the relationship between the incremental cost per QALY of 38 anti-cancer drugs in the UK and the corresponding  ASMRs awarded in France. Gerard de Pouvourville and Michael Drummond will then adopt contrasting perspectives on the pros and cons of the French and UK approaches to assessing the value added by new drugs.

IP7: SHOULD MULTIPLE CRITERIA DECISION ANALYSIS (MCDA) REPLACE COST-EFFECTIVE ANALYSIS (CEA)?
Moderator:

J. Jaime Caro, MDCM, FRCPC, FACP, Senior Vice President, Research, United BioSource Corporation, Lexington, MA, USA

Panelists: Karl Claxton, PhD, Professor of Economics, Centre for Health Economics, University of York, Heslington, York, UK; Andreas Gerber, PhD, MD, Head, Department of Health Economics, Institute of Quality and Efficiency in Health Care (IQWiG), Cologne, Germany; Warren Cowell, MSc, HTA Policy, Pfizer, Tadworth, Surrey, UK
ISSUE:

What should be the role of Multi-criteria Decision Analysis (MCDA) in the HTA process? The panel will address the issues raised by this question from the perspective of industry (Warren Cowell), HTA bodies (Andreas Gerber), and academia (Karl Claxton).

OVERVIEW: HTAs often focus today on efficiency, quantified as incremental cost-effectiveness. Efficiency does not consider, however, many criteria that citizens consider important, such as severity of illness, fairness, incentives for innovation and so on. This debate has now entered formal policy discussions, such as those about the introduction of Value Based Pricing in the UK, and the Swedish Government’s National Pharmaceutical Strategy. MCDA offers an approach to formally weighing multiple factors in the HTA. Incorporating MCDA into HTA, however, raises a number of process and methodological questions. Panellists will address the following topics: What factors should decision makers consider? Is MCDA necessary to address these? How should MCDA be incorporated into HTA? Who should carry out these MCDA? What type of MCDA approach is most appropriate to HTA What criteria should be included and/or how should relevant criteria be identified? Will decision makers use the results of MCDA? What are the implications of such approaches for manufacturers, researchers and policy makers? The panel will debate the issues before the audience is invited to participate in the discussion.
IP8: HOW DO YOU ASSESS THE VALUE OF CO-DEPENDENT TECHNOLOGIES?
Moderator: Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK
Panelists: Martina Garau, BA, (HONS), MSc, Senior Economist, Office of Health Economics, London, UK; Diego F. Ossa, MD, MSc, Global Head, HEOR MDx, Novartis Pharma AG, Basel, Switzerland; Mira Pavlovic-Ganascia, MD, Deputy Director for Health Technology Assessment at the Haute Autorité de Santé (HAS), La Plaine Saint Denis, France
ISSUE: Co-dependent technologies include biomarkers, diagnostics and drug treatments used in combination to maximise clinical benefits. The value created is a "joint product" and there are no rules for the attribution of some portion of the value to one or the other. This presents a challenge to the concept and operationalization of pricing and reimbursement (P&R) decisions for both diagnostics and treatments. The first panelist (Martina Garau) will discuss the issue from an economic perspective. Although in principal there is no "correct" way of dividing the joint value of co-dependent technologies, in practice that is done depending on the institutional context. Who "captures" this value influences how much R&D is undertaken and therefore whether the value is likely to be created in the first place. In this context, P&R systems should pay close attention to cost effective use of technologies as well as encouraging innovation. She will set out the alternative ways in which co-dependent technologies are currently assessed and the issues this gives rise to. The second panelist (Diego Ossa) will provide the manufacturer perspective highlighting the need to reward the added value brought in by individual technologies part of the ‘package’, including the diagnostic. The third panelist (François Meyer) will provide the payer perspective, focusing on evidence requirements for the evaluation of co-dependent technologies and issues of process coordination.
OVERVIEW:

P&R process should reward innovation in diagnostics and treatments. However, because those technologies are used in combination, there is a need to coordinate their assessment both in term of methods and processes. The adoption of a consistent approach to assessing the value of those technologies is required. The common methodology should be supported by synergies in decision making processes. For example, diagnostic/treatment combinations launched simultaneously arguably requires one committee to review both technologies in one package.

IP9: EVIDENCE GENERATION VIA CONDITIONAL COVERAGE FOR MEDICAL TECHNOLOGIES - WHAT KIND OF EVIDENCE SHOULD BE GENERATED TO SUPPORT ACCESS TO RELEVANT INNOVATIONS?
Moderator: Bernd H. Brüggenjürgen, MD, MPH, Head, Institute for Health Economics, Steinbeis University Berlin and Vice Chairman, German Association for Health Sciences and Public Health, Berlin, Germany
Panelists: Pippa Anderson, BSc, MSc, Managing Director, Swansea Centre for Health Economics, Swansea University, Swansea, UK; Joseph V. Ferrara, MA, President, Boston Healthcare Associates, Inc., Boston, MA, USA; Annie Chicoye, PhD, Executive Director for Development, Health Management Institute, ESSEC Business School Paris-Singapore, Cergy-Pontoise, France
ISSUE: Evidence on benefits and costs provided to inform decision-makers differ widely between pharmaceuticals, diagnostic or genetic tests and medical devices. Fulfilling all potential evidence requirements prior to approval and coverage decisions will not always be feasible and will impact the access to potential beneficial medical technologies. Several legislations try to overcome the gap with evidence generation programs such as the US “Coverage with evidence development”, the National Institute for Health and Clinical Effectiveness (NICE) in the UK “only with research” or the German recent trial regulation for medical devices and the potential real life assessment according to AMNOG (Arzneimittelmarktneuordnungsgesetz). Some criticism relates to the fact that many technologies would take years after they are cleared to be available to the patient. On the contrary ‘budget holders’ will only be willing to allow for provisional coverage when there is a clear potential of the new technology to prove it will be beneficial. Some critics argue that a clear framework is missing, where the evidence is going to come from and how it should be generated. Was evidence generation feasible, which evidence could be generated and was it perceived as a fair response to conflicting interests from industry and budget holders?  And how can conditional approval be meaningfully used to address adequate access to technologies in health care?
OVERVIEW: Panelist 1 will review the status of conditional coverage and evidence generation.  Panelist 2, the deputy Health Economist for the All Wales Medicines Strategy, will discuss the methods and aims currently utilised in the UK.  Panelists 3 and 4, from the US and France, respectively, will discuss the experience in their countries and lessons learned and strategies planned.   All presenters will engage in a dialogue facilitated by the moderator, and the audience will be able to direct questions to the panelists.
IP10: WHICH TO USE? POPULATION BASED OR INDIVIDUAL PATIENT ASSESSMENTS OF VALUE
Moderator: Stephen James Furniss, BA, Senior Vice President, Market Access, GfK Bridgehead, Melton Mowbray, Leicestershire, UK
Panelists: Ron Akehurst, BSc, Professor of Health Economics, Dean of School of Health & Related Research (ScHARR), University of Sheffield, Sheffield, UK; Sue Kilby, MSc, MBA, BPharm, Network Pharmacist South West London Cancer Network, London, UK
ISSUE: HTA bodies such as NICE take a population-based view of the value of a medicine, but decisions made on that basis may deny access to some patients who could derive much more than the average benefit. Many new oncology therapies have not been recommended by NICE for use in the NHS. In response to pressure from patients and clinicians the Government established the Cancer Drugs Fund (CDF), which makes available £200 million per year to improve patient access to cancer drugs. Most of this funding has been for drugs not recommended by NICE, or for use outside the NICE recommendations. Is there a policy justification for providing such a “safety valve”? How is it working in practice?  Is there a consistent approach across regions? Does this have implications for the planned introduction of Value Based Pricing (VBP) in 2014?  The moderator will present data on the use of the Cancer Drugs Fund. Sue Kilby will describe the processes and criteria adopted by the CDF in two regions. Professor Akehurst will represent a health economist’s perspective and discuss the strengths and weaknesses of the population based approach used by NICE.
OVERVIEW:

The CDF provides a mechanism and funding to enable individual patients to access medicines where the expectation of benefit is highest, even when Health technology Assessment suggests they are not good value for money.  The panel will review data on the use of the CDF, assess the extent to which it has met objectives, and debate with the audience whether such an individualised approach will still be necessary under VBP and could be extended beyond cancer treatments.

ISSUE PANELS - SESSION III
WEDNESDAY, 7 NOVEMBER 2012: 10:00-11:00
IP11: CAN VALUE OF INFORMATION ANALYSIS BE USED ROUTINELY TO INFORM RESEARCH PRIORITIZATION DECISIONS?
Moderator: Mark J. Sculpher, MSc, PhD, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK
Panelists:

Karl Claxton, PhD, Professor of Economics, Centre for Health Economics, University of York, Heslington, York, UK; Lotte MG Steuten, PhD, Associate Professor, Department of Health Technology and Services Research, University of Twente and Director, Health Economics and Reimbursement, PANAXEA b.v., Twente, The Netherlands; Rachael Fleurence, PhD, Director, Patient-Centered Outcomes Research Insitute (PCORI), Washington, DC, USA

ISSUE:

Value of information analysis is a powerful set of tools to quantify the potential value of generating additional evidence through research.  However, the implementation and communication of these technical methods can be challenging.  Can they be used successfully with non-specialists to inform routine research prioritization decisions?

OVERVIEW: Dealing with evidential uncertainty in decision making is a major challenge to all health care systems.  Decision uncertainty can be reduced by investing in additional research, but there are limits on the resources available for research.  Value of information (VOI) analysis is a set of tools to establish the maximum potential value of research relating to specific decisions (expected value of perfect information) and the value of different research designs (expected value of sample information).  There are now numerous published VOI analyses relating to specific clinical areas; but far fewer examples exist of their use to inform routine decisions faced by health systems and research funders.  What explains this limited uptake of VOI methods?  Do different types of decision makers have different issues with VOI, or is there a general challenge to all? Are the methods useful to support decisions in systems that do not formally consider cost effectiveness in making decisions? Are there examples of the successful use of VOI and, if so, can lessons  be drawn for the wider application of the methods?  The Panel will take alternative perspectives on these questions, share experience of using VOI with decision makers and identify a research agenda relating to research prioritization.
IP12: REFLECTING METHODOLOGICAL AND ENVIRONMENTAL CHANGE IN HTA METHODS: IS THE 2012 NICE GUIDE TO THE METHODS FOR TECHNOLOGY APPRAISAL THE ANSWER?
Moderator: Paul Tappenden, MSc, Senior Research Fellow, HEDS, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK
Panelists:

Andreas Gerber, PhD, MD, Head, Health Economics, Institute of Quality and Efficiency in Health Care (IQWiG), Cologne, Germany; Elisabeth George, PhD, Associate Director, Centre for Health Technology Evaluation, National Institute for Health and Clinical Excellence, Manchester, UK; Stephen Palmer, MSc, Professor, Centre for Health Economics, University of York, York, North Yorkshire, UK

ISSUE: The NICE Guide to the Methods for Technology Appraisal sets out the process and methods that NICE uses to undertake technology appraisals and provides guidance for the organisations invited to contribute to these appraisals. The guide enjoys widespread recognition and influence far beyond the Institute in England and Wales that publishes it. The guide is updated every four years and has recently been subjected to an update.
OVERVIEW: This update process has involved a number of stakeholder workshops, reviews of current and emerging methods and discussions amongst an expert working party. A large and diverse range of methodological topics have been addressed within the update, ranging from technical aspects of technology appraisals to more fundamental concerns regarding how NICE’s Appraisal Committees should make decisions about whether to recommend health technologies for routine use in the NHS. The revised draft of the methods guide is currently undergoing a three-month public consultation which is due to close in August 2012. This session intends to reflect and critique some of the key changes in the guide and to consider the alternatives views that have been represented throughout the update process. The speakers have all been involved to some extent in the update process though in very different roles. Particular focus will be reserved for two broad issues considered during stakeholder workshops held in 2011: 1) Multi criteria decision analysis or formal weighting of QALYs versus a deliberative process for incorporating “special factors”. 2) Whether and how to expand the current health service perspective to a broader societal one.
IP13: PERSONALIZED MEDICINE FROM A HEALTH SYSTEMS PERSPECTIVE: HOW CAN WE BETTER LEVERAGE EVIDENCE TO ADDRESS MULTIPLE STAKEHOLDER NEEDS?
Moderator: Eric Faulkner, MPH, Director, Global Market Access, Consulting, Quintiles Global Consulting, Durham, NC, USA
Panelists: Diego F. Ossa, MD, MSc, Global Head, HEOR MDx, Novartis Pharma AG, Basel, Switzerland; Ansgar Hebborn, PhD, Head, Global Payer & HTA Program Policy, F. Hoffmann-La Roche AG, Basel, Switzerland; Uwe Siebert, MD, MPH, MSc, ScD, Professor, Department of Public Health and Health Technology Assessment, UMIT/Oncotyrol/Harvard University, Hall i.T., Austria
ISSUE: Acceptance and use of companion diagnostics and personalized medicines involves engagement of a broader spectrum of health stakeholders versus conventional pharmaceuticals. HTA agencies, payers, physicians, hospital administrators, laboratory directors and patients all have different information needs to inform decision making. To what extent are changes in evidence development and value communication warranted for personalized medicine? How do we address existing gaps? How do we leverage evidence HEOR approaches to address the needs of each stakeholder type and address health system needs? Eric Faulkner will open the debate by providing an overview of evidentiary requirements for key stakeholder types and highlight the common challenges of addressing health system requirements in the Australia, the US and the EU. Diego Ossa will represent a diagnostic manufacturer perspective and discuss the extent to which diagnostic evidence development approaches are evolving to encompass multiple stakeholder needs. Ansgar Hebborn will represent a leading biopharmaceutical manufacturer perspective and discuss issues around alignment of developing plans to balance central and local market health system requirements in this rapidly evolving field. Uwe Siebert will provide a health economics perspective on evolution of HEOR methods and approaches for tackling multi-stakeholder information needs, including examples from ongoing pan-European and global efforts to bring clarity and consistency to personalized medicine evidence requirements.
OVERVIEW:

Emerging diagnostic and personalized medicine approaches promise to improve health care efficiency and outcomes at a time when health systems struggle to balance quality and costs. To ensure adoption of these technologies, a broad variety of health stakeholder evidence information requirements must be addressed, increasing the complexity of value demonstration in the field. The session will debate the extent to which existing evidence development paradigms address health system needs and discuss potential policy and HEOR solutions to support appropriate decision making and uptake.

IP14: WILL THE EUNETHTA MODEL FOR RAPID RELATIVE EFFECTIVENESS ASSESSMENT (REA) OF PHARMACEUTICALS WORK?
Moderator: Wim Goettsch, PhD, Project Leader of EUnetHTA WP5 on Relative Effectiveness of Pharmaceuticals and Health Care Insurance Board (CVZ), Diemen, The Netherlands
Panelists: Mel Walker, MRPharmS, PhD, FRS, Senior Director, GlaxoSmithKline, Brentford, Middlesex, UK; Marianne Klemp, PhD, Director, The Norwegian Knowledge Centre for the Health Services (NOKC), Oslo, Norway; Mirjana Huic, MD, MsC, Assistant Director, Agency for Quality and Accreditation in Health Care and Social Welfare, Zagreb, Croatia
ISSUE: Discussing experiences with the first joint European pilot with the EUnetHTA Model for Rapid Relative Effectiveness Assessment (REA) of pharmaceuticals.
OVERVIEW: The objective of this panel session is to facilitate discussion on the use of the EUnetHTA JA1 model for rapid REA of pharmaceuticals in daily practice. Wim Goettsch as Lead Partner of the WP5 of EUnetHTA will introduce the EUnetHTA model for rapid REA of Pharmaceuticals. Subsequenty, three panelists will discuss the results of the first pilot with pazopanib for treatment of metastatic renal cell carcinoma. Mel Walker from GSK will discuss the experiences with the involvement of GSK in this first pilot assessment. Subsequently, Marianne Klemp from NOKC, as representative of one of the WP5 organisations that was intensively involved in the pilot will reflect on the experiences of a pilot-doer with this first assessment using the model for rapid REA. Finally, Mirjana Huic, from the Croatian HTA department that is responsible for national assessments will discuss the possible value of the results of such pilot for their national assessment and a possible role in national decision making. All panelists will also discuss possible improvements to the structure of the model for rapid REA and in the process using this model for joint assessments.
IP15: CAN THERE BE A COMMON METHODOLOGY FOR COMPARATIVE EFFECTIVENESS IN THE ABSENCE OF RANDOMISED CONTROLLED TRIALS (RCTS)?
Moderator: Pascale Brasseur, MSc, Reimbursement Director, Cardiovascular Reimbursement and Health Economics, Medtronic International Trading Sàrl, Tolochenaz, Switzerland
Panelists: Isabelle Durand-Zaleski, PhD, Professor of Medicine, Head of Public Health, Director of the CRU ECO Ile-De-France, Créteil, France; Rod Taylor, MSc, PhD, Professor in Health Services Research, Peninsula Medical School, Exeter, UK; David A Scott, MSc, MA, Senior Director, Health Economics, Oxford Outcomes, Oxford, UK
ISSUE: RCTs are widely considered to be the gold standard for comparative effectiveness whether or not they are used to assess cost effectiveness. Increasingly products, both devices and pharmaceuticals, enter the market without direct evidence against the comparator of interest, in the patient population of interest.  Reimbursement agencies, however, must make decisions in such situations but few provide methods guidance regarding the preferred approach. Delaying or deferring such decisions because of this lack of evidence may deprive patients of effective treatments. The panelists will represent different viewpoints regarding the value of non-RCT evidence of effectiveness and the value of analytical methods to enhance the usefulness of such evidence. David Scott will explain the analytical methods, Rod Taylor will represent the payer perspective for a system using cost effectivness analysis, Isabelle Durand Zaleski will represent the payer perspective for a system focusing on clinical efficacy, and the moderator, Pascale Brasseur will represent the manufacturers perspective.
OVERVIEW: Network meta-analysis is increasingly used to address a lack of direct RCT evidence. However there are situations in which network meta-analysis does not provide evidence regarding the comparators of interest in the patient population of interest. These include: when no connected network of RCT evidence exists; when data available for a new technology is non-randomised or uncontrolled; or when the technology targets a subpopulation of the patients in the network. The methods and options available for the collection and generation of evidence of comparative efficacy in these situations will be presented. The panel will then consider the advantages and disadvantages of each method and their acceptability to decision makers and manufacturers across jurisdictions.