ISSUE PANELS - SESSION I
Monday, 26 October 2009: 11:30-12:30

IP1: THE UK NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE (NICE): NEW DEVELOPMENTS
Moderator: Stephen Beard MSc, Head of Health Economics, RTI Health Solutions, Manchester, UK
Panelists: Carole Longson PhD, Director, Centre for Health Technology Evaluation, NICE, Manchester, UK; Matt D Stevenson PhD, Technical Director, School of Health and Related Research Technology Assessment Group (ScHARR-TAG), University of Sheffield, Sheffield, UK; Sorrel E Wolowacz PhD, Senior Health Economist, RTI Health Solutions, Manchester, UK
ISSUE: Several new initiatives have been launched by NICE recently.  A revised Methods Guide was issued in June 2008 on the basis of a formal review undertaken in 2007, which included a series of expert workshops exploring specific technical areas (briefing papers now published in Pharmacoeconomics).  Proposed changes to the STA submission template were issued for consultation in early 2009.  A new flexible pricing scheme was agreed under the Pharmaceutical Price Regulation Scheme (PPRS) in November 2008.  The scheme aims to ensure that medicine prices fairly reflect their value to patients, and to provide faster access to new medicines that are clinically and cost effective.  Branded drugs will see their price cut, and industry innovations will be encouraged and rewarded.
OVERVIEW: Recent developments from NICE will be discussed, including changes to Methods Guide specifying the requirements for HTA submissions and changes to the Single Technology Appraisal submission template.  A new flexible pricing scheme agreed between the UK government and the pharmaceutical industry will also be discussed.  The rationale for the implementation of these changes will be described by Dr. Carole Longson (NICE).  Key aspects of the new evidence requirements will be discussed by Dr. Matt Stevenson (ScHARR).  Recommendations for research planning and implementation to fulfill the new requirements will be presented by Dr. Sorrel Wolowacz (RTI-Health Solutions).  The issue panel will conclude with an open session for audience questions, discussion and debate.

IP2: PHARMACEUTICAL POLICIES IN EUROPEAN COUNTRIES: AIMING AT COST CONTAINMENT, COST-EFFECTIVENESS, OR EQUITY?
Moderator: Ivar Sønbø Kristiansen MD, PhD, MPH, Professor, Institute of Health Management and Health Economics, University of Oslo, Oslo, Norway
Panelists: John Hutton PhD, Professor of Health Economics, Department of Health Sciences - Health Economics, University of York, York, UK; Marc Koopmanschap PhD, Researcher, Institute for Medical Technology Assessment, Erasmus MC, Rotterdam, The Netherlands; Ewa Orlewska MD, PhD, Lecturer, Centrum Farmakoekonomiki, Warsaw, Poland
ISSUE: The objective of healthcare systems is typically maximum health within a given budget, subject to equity considerations. European countries have instituted policies to control rising pharmaceutical expenditures, but such policies may themselves incur administrative costs or transfer costs to other parts of healthcare or to patients. Also, policies may result in inequity in health because wealthier patients can pay out-of-pocket while the poor cannot. The issue is first whether pharmaceutical policies are evaluated at all, second whether such evaluation is restricted to elucidate consequences for government expenditure only or costs in a broader perspective, and third whether equity issues are considered.
OVERVIEW: The aim of this issue panel is to present and discuss results of a study among European countries in which the focus was the evaluation of pharmaceutical policies and their consequences.  A survey was developed to obtain information about pharmaceutical policies and their evaluation in 27 European countries.  The survey first captured the main policy options that countries have adopted. It subsequently explored how these policies have been evaluated in terms of health consequences, costs to patients and non-pharmaceutical sectors, and equity consequences. The survey was completed by experts within the fields of pharmaceuticals, economics, and medicine.  Responses were received from Belgium, Denmark, Finland, France, Germany, Hungary, Ireland, The Netherlands, Norway, Poland, Portugal, Sweden, and the UK. Overall, economic evaluation of pharmaceuticals themselves is common in most European countries, but explicit evaluation of the policy consequences in regard to health, societal costs, and equity is largely lacking.  Discussion: The study results indicate that stakeholders need to take the objectives of modern healthcare systems seriously. The question is when and how governments can adopt wider perspectives in the evaluation of their healthcare policies. The issue raised here will require broader research frameworks than those currently in place.

IP3: THE DEVIL IS IN THE DETAILS: INTERNATIONAL COMPARISON OF TECHNOLOGY ASSESSMENT PROCESSES AND PAYER UPTAKE OF PERSONALIZED MEDICINE AND IMPLICATIONS FOR THE FIELD
Moderator: Eric C Faulkner MPH, Senior Director, Pricing & Reimbursement, RTI Health Solutions, Research Triangle Park, NC, USA
Panelists: Lieven Annemans PhD, Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium; Uwe Siebert MD, MPH, MSc, Professor, UMIT – University for Health Sciences, Medical Informatics and Technology, Hall i.T, Austria; Katherine Payne PhD, Senior Research Fellow, University of Manchester, Manchester, UK
ISSUE: As personalized medicine begins to enter global markets, uptake and successful application will depend upon how the diagnostic and treatment components fare in health technology assessment (HTA) and payer decision processes. How consistently have early personalized medicine products been handled in key global markets, assuming similar information on product value is available? How can health outcomes and economics approaches address payer decision challenges and inform appropriate integration of personalized medicine? Eric Faulkner will represent a payer perspective and open the panel with a brief overview of example personalized medicine technologies, HTA recommendations and payer decisions regarding these technologies in the France, Germany, the UK and the United States. Katherine Payne will discuss technology evaluation issues regarding the example technologies from the perspective of a health technology assessor and NICE Appraisal Committee member. Uwe Siebert will represent a decision sciences perspective and explore how decision analysis can be used to accomodate the unique challenges of personalized medicine, using examples from the area of cancer diseases. Lieven Annemans will provide a methodologist perspective on how economics and outcomes approaches may help address practical challenges in assessing personalized medicine scenarios, enable appropriate patient access and support harmonization efforts among global payer decision makers.
OVERVIEW: Emerging personalized medicine approaches promise to improve health care efficiency and outcomes at a time when health systems struggle to balance quality and costs.  Despite this promise, payers face technology assessment and decision challenges because conventional evidence development and evaluation approaches are insufficiently developed for personalized medicine, variably applied or have not kept pace with rapid technology evolution in this field. Alternatively, manufacturers face reimbursement uncertainty and practical challenges of meeting ‘ideal’ payer evidence requirements and diminishing incentives for innovation. Because of these obstacles, health outcomes and economics approaches must adapt to support appropriate payer decisions and product uptake.

IP4: WHAT SHOULD BE THE ROLE OF PATIENT PREFERENCES IN MAKING HEALTHCARE RESOURCE ALLOCATION DECISIONS?
Moderator: Michael F Drummond DPhil, Professor of Health Economics, Centre for Health Economics, University of York, York, UK
Panelists: John FP Bridges PhD, Assistant Professor, Bloomberg School of Public Health, Health Policy and Management, Johns Hopkins University, Baltimore, MD, USA; Deborah A Marshall PhD, Associate Professor, Department of Community Health Sciences, University of Calgary, Calgary, AB, Canada; Mark J Sculpher PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK
ISSUE: It is often argued that patients’ preferences are overlooked in healthcare resource allocation decisions, particularly in managed care settings in the US, or within the socialised healthcare systems existing in Europe and Canada. Therefore the objective of this panel is to discuss the possible role of patients’ preferences in making healthcare resource allocation decisions.
OVERVIEW: In pharmacoeconomic studies, the normal approach for valuing health states is to use the values of members of the community.  In contrast, much of outcomes research focuses on eliciting patient reported outcomes for alternative therapies. Also, in the field of health utility assessment, some argue that health state valuations should be those of individuals  that  have experienced particular health states (eg patients), rather than members of the community, who are typically asked to imagine what given states might be like. There is also evidence that patients’ and the general public’s preferences may differ. Therefore, this panel will discuss whether the role of patient preferences differs in socialised healthcare systems as opposed to those with extensive private market involvement; whether patient preferences should be used to determine levels of co-payment; whether patients’ and the public’s preferences differ substantially and: whether there is a time and place for both patients’ preferences and the community’s values in healthcare decision-making in all settings?

IP5: PATIENT-REPORTED OUTCOMES AS A STANDARD APPROACH FOR HEALTH CARE POLICY MAKERS
Moderator: Andrew Bottomley PhD, Assistant Director, Head Quality of Life Department, EORTC, Brussels, Belgium
Panelists: Chantal Quinten MSc, Researcher, Quality of Life Department, EORTC, Brussels, Belgium; Carolyn Gotay PhD, Professor, University of British Columbia, Vancouver, BC, Canada
ISSUE: The panelists will use their extensive knowledge in quality of life to make researcher aware why (regulatory point of view) and how (different implementation strategies) to incorporate Patient-Reported Outcomes (PRO) from international cancer clinical trials in their health economic evaluations. They will use novel results from the EORTC PROBE project to demonstrate the added value of PRO in clinical decision making.
OVERVIEW: Cancer clinical trials have traditionally assessed clinical end points such as overall survival or progression-free survival. Incorporating PRO has received increasing attention over recent years. Our session will address the views of the FDA, EMEA and EORTC of the rising interest of PRO in oncology and the series of measures and policies these institutions have established.  Additionally, we will compare implementation strategies from different groups to enhance PRO and which lessons can be learnt from them to improve PRO assessment. Finally, the session will use new and novel results gained from the EORTC Patient Reported Outcomes and Behavioural Evidence (PROBE) research program to demonstrate the value of PRO alongside clinical information for health care professionals. It will provide the audience with some tools in how to collect, analyse and interpret PRO data gathered from randomized controlled trials. Adequate collection and interpretation become imperative as it becomes more and more expensive to conduct clinical trials. We will encourage the audience to share their experience, debate with the presenters and help set future priorities for PRO assessment in the oncology outcome research field.

ISSUE PANELS - SESSION II
Tuesday, 27 October 2009: 10:00-11:00

IP6: SOCIETAL VERSUS PAYER PERSPECTIVES FOR COST-EFFECTIVENESS ANALYSIS: CAN WE JUSTIFY VARIATION BETWEEN DECISION MAKERS?
Moderator: Mark J Sculpher PhD, Professor, Centre for Health Economics, University of York, York, UK
Panelists: Karl Claxton PhD, Professor, Centre for Health Economics, University of York, York, UK; Bengt Jonsson PhD, Professor, Department of Economics, Stockholm School of Economics, Stockholm, Sweden
ISSUE: Many jurisdictions now use cost-effectiveness to support decision making regarding the reimbursement of new medical technologies.  However, there is variation in the cost perspective decision making organisations recommend for such analyses.  Some advocate the use of a societal perspective, where all costs are considered regardless of budget constraints; others focus only on costs falling on the health system or payer.  Can this variation be justified?  Should decision makers be seeking harmonisation in cost perspective?
OVERVIEW: Most organisations making decisions about the reimbursement of new technologies state their preferred cost perspective.  However, very few seek to justify their recommendation in terms of economic theory or to relate this to the aims and objectives of the decision making organisation.  The aim of this workshop is to explore whether there is scope for greater harmonisation between jurisdictions in required cost perspective.  The panelists have worked closely in developing and implementing the methods guidelines of decision making organisations in the UK (NICE, Claxton) and Sweden (TLV, Jonsson).  These two organisations take quite different views on the cost perspective for economic evaluation.  In most circumstances, NICE requires all submitted economic analyses to adopt the perspective of the UK NHS. TLV, on the other hand, requires analyses to take a societal perspective and include all costs no matter where they fall.  The session will begin with Sculpher describing the variation between jurisdictions in preferred cost perspective and outlining issues that need to be considered in identifying an appropriate perspective.  Claxton and Jonsson will then describe the position of NICE and TLV, respectively, explore the reasons for their different positions and assess the scope for harmonisation in cost perspective. 

IP7: OUR MONEY OR YOUR RISK? RISK SHARING REIMBURSEMENT AGREEMENTS: IS THIS THE FUTURE?
Moderator: Monique Martin MSc, MBA, Vice-President UK Operations, i3 Innovus, Uxbridge, Middlesex, UK
Panelists: Michael F Drummond DPhil, Professor of Health Economics, University of York, Centre for Health Economics, York, UK; Alistair McGuire PhD, Chair in Health Economics, LSE Health and Social Care, London, UK
ISSUE: Are risk sharing reimbursement agreements the way of the future?
OVERVIEW: There is an increasing movement towards using cost-effectiveness and value for money claims as part of reimbursement packages. While the present trend is to use ex-ante cost-effectiveness, a number of risk-sharing agreements based on post-launch information are being used across different countries. The rationale for these ex-post evaluations is partly that ex-ante cost-effectiveness relies upon clinical trial evidence for effectiveness and may not be representative of the intended standard treatment population. A further reason couples this clinical trial argument with a high price of therapy. Given these issues, reimbursement authorities may therefore consider a two-tier pricing mechanism where the initial reimbursement is set using ex-ante cost-effectiveness, but with the agreement that, once on market, the therapy continues to be evaluated under post-launch conditions. The evidence collected is assessed at some future date to re-evaluate the reimbursement price with the possibility of claw-back from product revenue. Such schemes have been implemented in various ways, for various product types in various European countries including Sweden, France and the UK. These so-called risk sharing agreements raise a number of issues concerning R&D incentives, the design of clinical trials and subsequent follow-up, launch price decisions and the targeted population. The aim of this issues panel is to discuss these topics by providing a speaker who defends the use of risk-sharing agreements and an individual who critiques such agreements. After an initial introduction, which considers the current position in Europe with regards to risk-sharing agreements and discusses potential future developments, Professor Alistair McGuire (LSE Health) will present a defence of a movement towards such risk-sharing agreements while Professor Mike Drummond (University of York) will present a critique. The panel will then open the debate to the floor for further questions and discussion.

IP8: IQWIG AND THE EFFICIENCY FRONTIER – HOW CAN WE MEASURE PATIENT RELEVANT OUTCOMES?
Moderator: F. Reed Johnson PhD, Principal Economist, RTI International, Research Triangle Park, NC, USA
Panelists: Axel C. Mühlbacher PhD, MSc, Professor, Hochschule Neubrandenburg, IGM Institut of Health Economics and Health Care Management, Neubrandenburg, Germany; Olaf Pirk PhD, MD, Principal, Health Economics & Outcomes Research, IMS Health, Nuremberg, Germany; John FP Bridges PhD, Assistant Professor, Bloomberg School of Public Health, Health Policy and Management, Johns Hopkins University, Baltimore, MD, USA
ISSUE: This panel session will discuss approaches for including patient relevant outcomes in the estimation of efficiency frontiers as outlined in IQWiGs cost-benefits analysis. Olaf Pirk will present a brief introduction to the efficiency frontier method and discuss how the notion of patient relevant outcomes differs from traditional measures of outcomes. John FP Bridges will present empirical research demonstrating how various stated preference methods can be used to identify patient relevant outcomes. Axel C. Mühlbacher will discuss practical issues associated with the applying the notion of patient relevant outcomes within the efficiency frontier framework.
OVERVIEW: IQWiG, the Germany health technology assessment agency founded in 2004, has proposed a new method for cost-benefit assessment based on what it calls efficiency frontier. Such efficiency frontiers will look at the relative tradeoffs between cost and so call “patient relevant outcomes” in making recommendations on the economic value of medical interventions. To achieve such cost-benefit assessment a greater understanding of patient relevant outcomes is needed. What is a patient relevant outcome? How is it measured? Can one compare or index different patient relevant outcomes?

IP9: HOW SHOULD HTA METHODS BE ADAPTED TO MEET THE RISING EXPECTATIONS OF DECISION MAKERS FOR MEDICAL DEVICES AND DIAGNOSTICS REIMBURSEMENT?
Moderator: Annie M J Chicoye PhD, Expert, Pricing & Market Access Consulting, IMS Health France, Puteaux, France.
Panelists: Olivier Goeau-Brissoniere MD, Professor, Chairman, Federation des Specialités Medicales, Paris, France; Geoffroy Wilson MD, Reimbursement Leader EMEA, Emea, GE Healthcare, Chalfont St Giles , Buckinghamshire, UK; Adrian Griffin MSc, VP Strategic Affairs, LifeScan EMEA, Johnson & Johnson, High Wycombe, Buckinghamshire, UK
ISSUE: Medical technologies others than pharmaceuticals, ranging from external to implantable medical devices, medical imaging  to molecular diagnostics are increasingly challenged to demonstrate their  clinical and economic value by a variety of decisions makers, depending upon funding and reimbursement processes,  medical practices and affordability. In the frequent absence of regulatory requirement  for comparative clinical effectiveness, and considering how those technologies are positioned in the medical pathways, there is a need for clarifying  whether other outcomes than purely clinical or PROs could be appropriate for assessing their medical  value, whether traditional cost/effectiveness analysis is relevant for admission to reimbursement.
OVERVIEW: Panelists will provide examples of different technologies and discuss the related HTA issues and on which grounds in practice decisions makers at national, regional or local level decide for their reimbursement or dissemination. As a result, disparities for access to those technologies across European countries will be presented.  They will suggest how to move forward for improving dialogue between stakeholders and develop a better understanding of requirements. Discussion will focus on possible innovative approaches adapted to the short life-cycles and continuous incremental improvement characterizing this area. It will also address the relevance and feasibility of developing international guidelines for assessing medical devices and diagnostics and in which setting this development should take place.  ISPOR’s initiatives in this field will be presented as a background for the discussion.

IP10: ESTIMATING THE COST-EFFECTIVENESS OF VACCINES – IS THE HEALTH ECONOMIST'S STANDARD TOOLBOX SUFFICIENTLY EQUIPPED?
Moderator: Stuart Michael Carroll BA, (Hons), MSc, Research Associate and Policy Analyst, Centre for Health Economics and Policy, United BioSource Corporation, London, UK
Panelists: Philippe Beutels BSc, MSc, PhD, Professor, Centre for Health Economics Research & Modeling Infectious Diseases (CHERMID), Centre for the Evaluation of Vaccination (CEV), University of Antwerp, Antwerp, Belgium; John Hutton PhD, Professor of Health Economics, Department of Health Sciences - Health Economics, University of York, York , UK; Carole Omnès PharmD, MSc, Health Economics Service Manager, Senior Market Access and Health Economics, Europe, Sanofi Pasteur MSD, Lyon, France
ISSUE: Economic decisions to fund vaccination programmes in industrial countries are commonly based on the same metric as used for curative/reactive pharmaceuticals.  However, as a group of pharmaceuticals, vaccines are different from most other pharmaceuticals, and are an integral part of preventative public health.  Some have argued that this warrants a different approach for judging the economic value of vaccines.  Should standard cost-effectiveness (CE) analysis be adjusted when specific features exemplified by some vaccines are present?  Should alternative discounting techniques and/or higher CE thresholds be adopted?  What are the implications for optimising resource allocation and equity considerations when different criteria are applied? 
OVERVIEW: Vaccines are highly expensive to develop and test, which results in relatively high prices for the preventative capability conferred to target populations.  Vaccines are often evaluated using standard CE methods.  Depending on disease prevalence and the immediacy of outcomes, some vaccines have been deemed CE and adopted, whilst others have either been considered not CE but adopted due to “public need” or judged not CE and therefore rejected. Some economists have stated that the assessment of CE for vaccination programmes generally underestimates economic value. It has been contended that the use of quality-adjusted life years in short-term illness is methodologically inappropriate; that discounting of distant future events should be varied or lowered; and society’s willingness-to-pay for protection against disease may differ from that for curative/reactive treatment. It has been further suggested that outcomes measures may need to go beyond those immunised to properly account for herd immunity effects (indirect protection at zero additional cost), potential cost offsets from reduced antibiotic resistance, and the potential importance of serogroup displacement. The above panel will debate the problems confronted by health authorities, presenting the pros and cons of different positions to stimulate audience discussion of the merits of alternative approaches.