Issue Panels
Monday, 31 October 2016
11:15 - 12:15
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research Issues

11:15 - 12:15
Room: Hall A (L2)

IP1: PATIENT-CENTERED DECISION-MAKING WITH MULTI-CRITERIA DECISION ANALYSIS: SHOULD WE BE TRYING TO QUANTIFY THE PATIENT VOICE FOR USE IN HEALTH TECHNOLOGY ASSESSMENT?

Moderator:

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

Panelists:

Lars Sandman, PhD, Professor, National Centre for Priority Setting in Health-Care, Linköping University, Linköping, Sweden

Peter L. Kolominsky, MD, PhD, MBA, Professor & Director, Interdisciplinary Centre for Health Technology Assessment (HTA) and Public Health, University of Erlangen - Nürnberg, Germany, Erlangen, Germany

Alaa Hamed, MD, MPH, MBA, Head, Rare Disease, Patient Outcomes and Medical Economics, Genzyme, a Sanofi Company, Cambridge, MA, USA

ISSUE:

Decision makers have been making strides to meaningfully incorporate the patient voice into health technology assessment (HTA). The panel will debate the role of the patient voice in HTA, and if multi-criteria decision analysis (MCDA) can facilitate this use of the patient voice. The panel reflects the diversity of perspectives on the topic. Peter Kolominsky-Rabas presents the case for a greater role for the patient voice as part of HTA, and outline ways achieve this. Lars Sandman provides a critical perspective, problematizing the role of patients in HTA and their ability to address the trade-offs required of HTA. Alaa Hamed provides an industry perspective, illustrating ongoing pilots on systematically capturing the patient voice in the rare disease space. Each panelist will speak for 10 minutes on the opportunities and challenges associated with incorporating the patient voice into HTA. This will be followed by a 15-minute panel discussion, and 15 minutes of Q&A from the audience.

OVERVIEW:

Consideration of patients in healthcare decisions generally sounds like a positive aspiration, supported by reference to central ethical values like autonomy and patients’ best interest (from their own point of view). However, how to best (and the extent to which we should) consider patient perspectives in HTA is still up for debate. MCDA has been proposed as a means to involve patients in determining what factors should be considered in HTA. However, this also raises a number of challenges, including: what legitimacy and role do patient preferences have in HTA in relation to other values; how to deal with patient heterogeneity and the fact that the outcome of the HTA is associated with an alternative cost for other patient groups, which are difficult to identify; which MCDA methods are best for eliciting patients’ preferences; and how to combine patients preferences with those of other stakeholders.

Health Policy Development Using Outcomes Research Issues

11:15 - 12:15
Room: Hall D (L -2)

IP2: NATIONAL HTA PROCESS AND EUROPEAN COOPERATION ON HTA--FIT FOR PURPOSE? (Invited Issue Panel)

Moderator:

Finn Børlum Kristensen, MD, PhD, Professor, University of Southern Denmark, Hilleroed, Denmark bio

Panelists:

Dominik Schnichels, Head of Unit, DG SANTE B4, Medical Products: Quality, Safety, Innovation, European Commission, Brussels, Belgium bio

Zoe Garrett, MRes, MPhil, Senior Technical Adviser, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), Manchester, UK bio

Rui Santos Ivo, Vice-President, Executive Board, National Authority of Medicines and Health Products (INFARMED), Lisbon, Portugal bio

Tomas Tesar, PharmD, PhD, MBA, MSc, Member, Reimbursement Committee, Slovak Ministry of Health, Union Health Insurance Fund, Bratislava, Slovak Republic bio

ISSUE:

 The European cooperation on HTA through the work of EUnetHTA has delivered methodological and process support tools which facilitate joint scientific work and production of the HTA information that can be shared across borders. The HTA Network Strategy and recent Council Conclusions have also provided the political support to strengthen the EU cooperation. A few important questions still need to be answered in order to deliver a permanent solution for a sustainable mechanism of the HTA cooperation in Europe. How can the joint work and output produced at the European level fit into the national HTA – and decision-making processes? How to ensure that the European cooperation process contributes effectively and efficiently to the HTA processes in the participating countries? What are the obstacles, opportunities and concrete plans to address the issues of synergy between the European cooperation on HTA and national HTA and decision-making processes? Regional cooperation efforts between Member States are emerging, how can they be supported by EU cooperation?

OVERVIEW:

The methodological aspects of joint HTA work and scientific process support tools have been addressed and developed in the previous joint actions on HTA, the third Joint Action focuses on strengthening joint production and will also address organizational issues to make proposals for a permanent solution for EU cooperation. Decision-making on patient access to health technologies is and will remain a sole national competence of the EU Member States. The national HTA process informs and adheres to the organisation of the national decision-making processes. These can be similar but also differ substantially between the EU Member States, thus influencing how to cooperate on HTA across borders effectively and realise the full European added value of such cooperation. Perspectives, positions and experiences or plans for specific activities addressing this issue will be presented and discussed by a EUnetHTA Partner organisation, the European Commission and EU Member State healthcare decision-maker.

14:15 - 15:15
BREAKOUT SESSION
Clinical Outcomes Research Issues

14:15 - 15:15
Room: Hall E2 (L0)

IP3: DOES CONSERVATISM BY REGISTRATION AUTHORITIES IN THEIR REQUIREMENTS FOR OUTCOME MEASURES IN PAIN TRIALS HAMPER DRUG DEVELOPMENT IN THAT AREA?

Moderator:

R.L Akehurst, PhD, Strategic Director, Sheffield, BresMed Health Solutions, Sheffield, UK

Panelists:

Will Dunlop, MEc, Head of Market Access, Mundipharma International Ltd, Cambridge, UK

C. Daniel Mullins, PhD, Professor & Chair, Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, MD, USA

Ben van Hout, Professor of Health Economics, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

ISSUE:

Does conservatism by registration authorities in their requirements for outcome measures in pain trials hamper drug development in that area?

OVERVIEW:

In the therapeutic area of pain relief, a recent survey commissioned by Mundipharma International Limited has shown that superiority trials are in a minority and the outcome measures available are often not helpful in making economic cases for new drugs (Rycroft et al. 2015, Value in Health). The study was based on a literature review that identified 356 suitable pain studies. Only 46% of the ongoing/recently completed trials that did have an active comparator were designed as superiority studies. Payer-relevant end points were not reported in the majority of published and ongoing/recently completed studies: preference-weighted quality-of-life (36% and 42%), resource use (2% and 8%), physical function (28% and 39%), and psychological function (25% and 24%). The lack of payer relevant outcomes seen in pain studies could be seen as a state of affairs caused by registration authorities being conservative about both trial designs and choice of outcome measures. But it could also reflect unique challenges with pain therapy; including the possibility to titrate comparative therapies and the lack of appropriate outcome measures. Declaration of financial interests: Profs Mullins and Akehurst have received honoraria for themselves or their company for participation in seminars and research in this area conducted by Mundipharma International Limited. Will Dunlop is an employee of Mundipharma International Limited. Prof van Hout has no interests to declare.

Use of Real World Data Issues

14:15 - 15:15
Room: Hall D (L -2)

IP4: DOES EUROPEAN REAL WORLD DATA REQUIRE A ‘DIGITAL SCHENGEN’ TO SUPPORT FEDERATED ACCESS, ASSESSMENT, AND (RE)USE, OPENING BORDERS AT THE INSTITUTE TO CROSS-COUNTRY?

Moderator:

Ömer Saka, MD, MSc, Partner, Market Access Strategy and HEOR, Deloitte, Diegem, Belgium

Panelists:

Sarah Garner, PhD, BPharm, Associate Director of Science Policy and Research, National Institute for Health and Care Excellence (NICE), London, UK

Hans-Georg Eichler, MD, MSc, Senior Medical Officer, European Medicines Agency, London, UK

Johan van der Lei, MD, PhD, Chair of the Department of Medical Informatics, Medical Informatics, Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands

ISSUE:

Europe needs to see breakthroughs in data access and sharing within and across its borders to enable opportunities of real world data, as well as changes to address territorialism, lack of data interoperability, need for common data models, privacy concerns and building of sustainable platforms. Without such initiatives Europe will see both a holding back of basic research and insights into health outcomes, as well as its competitiveness on the global arena of real world data utilisation.

OVERVIEW:

Europe/The EU is in a relatively unique position of increasing harmonisation of political, economic and social aspects of society, inclusive of healthcare, but fraught with challenges in all domains, in comparison to the United States, Asia Pacific and other regions worldwide. Despite moves to address fragmentation of both governance and application, such as the recent General Data Protection Regulation (GDPR), moves to a common digital market and greater collaboration on research and allied programmes, such as within the Innovative Medicines Initiative (IMI), we remain far from in an ideal position to see greater access, aggregation and utilisation of health data for research across the pharmaceutical industry and life sciences, and academia. Though two key drivers for use of real world data, pharmacovigilance and safety surveillance, and outcomes research for reimbursement, have been significant in their impact and focus for some key stakeholders, access to data and biological material to support Discovery and Development, as well as intra-disease area and inter-therapeutic area research remain very challenging. With a data tsunami from disparate sources and technologies, to a methods and outputs blizzard we also face an expertise drought that is not helping solve the issue of widespread data sharing in Europe, and to improving the efficiency of regulatory and HTA processes.

15:45 - 16:45
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

15:45 - 16:45
Room: Hall A (L2)

IP5: A "FEDERAL" STANDARD FOR VALUING HEALTH IN A POST-REFERENDUM EUROPE?

Moderator:

Meindert Boysen, PharmD, MSc, Professor, Health Services Research & Policy, London School of Hygiene & Tropical Medicine, London, and Programme Director, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), London, UK

Panelists:

Paul Kind, Professor, Centre for Health Economics, Management and Policy, HSE University, St. Petersburg, Russia

Ben van Hout, PhD, Professor of Health Economics, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

ISSUE:

Should there be a standard European-wide method of determining the value of health benefits?

OVERVIEW:

By now the result of the UK referendum is a matter of fact. A far-reaching debate about EU membership failed to grapple with one central issue that ought to concern citizens across Europe, namely the methods used to determine the value of health benefits. National decisions made by regulatory agencies that include the evaluation of cost-effectiveness depend upon two key factors – the availability of appropriate evidence and the capacity to analyse it. Given the constraints on both these factors it would be reasonable to consider ways of improving the efficiency of economic evaluation by moving towards a standard “reference case” approach that could be required in all jurisdictions. As a first step in achieving this it would be useful to consider adopting a standard European approach towards the valuation of health benefits. Three key questions will be considered during this Issues Panel. Which method should be adopted as the standard mechanism for determining the “value” of health? How should values from participating countries be aggregated? Should the issue be left to the general public, or to the informed elite? As with the UK referendum, there are (at least) two points of view for all three questions. Underlying this debate is a European challenge - do we keep acting as we do as part of some type of laissez-faire arrangement or do we adopt a more positive system of cross-national working? The session will conclude with a referendum in which participants will be able to choose between two alternatives: the right one and the wrong one.

Patient-Reported Outcomes & Patient Preference Research Issues

15:45 - 16:45
Room: Hall E1 (L0)

IP6: FROM TESTIMONIALS TO QUALITATIVE RESEARCH EMBEDDED IN CLINICAL TRIALS: HOW DO HEALTH TECHNOLOGY ASSESSMENT BODIES CONSIDER THE VOICE OF RARE DISEASE PATIENTS WHEN GRANTING ACCESS TO ORPHAN DRUGS?

Moderator:

Benoit Arnould, PhD, Senior Director, Patient-Centered Outcomes, Mapi Group, Lyon, France

Panelists:

Sheela Upadhyaya, MSc, Associate Director, Centre for Health Technology Evaluation, Highly Specialised Technologies, National Institute for Health and Care Excellence, London, UK

Christine Lavery, MBE, Group Chief Executive, The Society for Mucopolysaccharide Diseases, London, UK

Samantha Parker, MBA, Senior Vice President, Patient Access Officer, Lysogene, Neuilly sur Seine, France

ISSUE:

The panelists, from an HTA body, a Patient Advocacy group, and a Pharmaceutical company, will be invited to debate the following questions: To what extent do patient testimonials impact health technology assessment (HTA) decisions? Is this sufficient to ensure reliable decision making? Would qualitative research fill a gap?

OVERVIEW:

The process resulting in market authorization for orphan drugs is driven by specific considerations, including: 1) critical unmet needs, 2) importance of giving patients a chance to slow or stop the progression of their disease, and 3) limited number of patients. As a result HTA bodies are presented dossiers that frequently lack essential information concerning clinical outcomes. When clinical outcomes are documented, they may lack comprehensiveness, specificity, and documented clinical meaningfulness. Patients and their representatives play an essential role in sharing their experience in terms of priorities, risk/benefit assessment, and meaningfulness of outcomes. However, there is a gap in the way this information is provided to the authorities: in the absence of evidence, based on appropriate standardized Patient-Centered Outcome measures, patients (or their representatives) frequently present their perspective in the form of testimonials. In the near future, this gap might trigger an increased level of rejection by HTA bodies of potentially important innovative therapeutics. There is a growing acknowledgement in the value of including disease specific qualitative research in trial design. This approach – which pertains to the rapidly progressing field of Mixed-Methods Research (MMR) - might provide a critical contribution of scientific based evidence.

17:00 - 18:00
BREAKOUT SESSION
Economic Outcomes Research Issues

17:00 - 18:00
Room: Hall D (L -2)

IP7: HOW SHOULD BIOSIMILARS BE VALUED AND SHOULD THEY UNDERGO HEALTH TECHNOLOGY ASSESSMENT?

Moderator:

Jeanette Kusel, MSci, MSc, Head of HTA and Health Economics, Costello Medical Consulting Ltd, Cambridge, UK

Panelists:

Frank McKenna, MD, FRCP, Consultant Rheumatologist, Trafford General Hospital, Central Manchester University Hospitals, Manchester, UK

Andrew Walker, PhD, Health Economist, Robertson Centre for Biostatistics, University of Glasgow, Glasgow, UK

Emi Psachoulia, MSc, PhD, Senior Manager Market Access, Biogen International GmbH, Zug, Switzerland

ISSUE:

The issue under debate will be whether biosimilars should undergo health technology assessment (HTA) and if they should, then what form this should take. The perspectives of a biosimilar manufacturer, prescriber and HTA body will be covered.

OVERVIEW:

Due to the reduced requirement for clinical evidence for regulatory approval, biosimilars can be priced lower than the originator brand and therefore have the potential to deliver significant cost savings. How payers value biosimilars is likely to differ across countries. Being cost-saving does not necessarily mean they will automatically be cost-effective, as biosimilars still have high price tags compared to many other medicines. For example in the UK, the infliximab biosimilars have been recently rejected by NICE for the treatment of moderate rheumatoid arthritis. HTA agencies already have different approaches to handling biosimilars; for example, within the UK, NICE only assesses biosimilars through multiple technology appraisals, whereas in Scotland biosimilars whose reference product is not recommended are assessed individually. Some countries, such as Poland, do not have a published process. The panelists will give their views on how biosimilars should be valued, whether they should undergo HTA and how this might differ across markets. If HTA is deemed necessary, then the panelists will expand on whether this is only in certain cases and when this should occur with respect to marketing authorisation. The experts will be challenged on whether appraising biosimilars is an effective use of public money and what data should be required by payers. Additionally the panelists will be asked how they think the patient and clinician perspective should be incorporated into decision making. The audience will get a chance to share their own views and pose their questions to the panel. There will be an audience vote on whether biosimilars should undergo HTA.

Use of Real World Data Issues

17:00 - 18:00
Room: Hall E1 (L0)

IP8: IS THE GOAL OF UNIVERSAL COMPARATIVE EFFECTIVENESS EVIDENCE ACROSS JURISDICTIONS ACHIEVABLE?

Moderator:

Pall Jonsson, PhD, Senior Scientific Adviser, Science Policy and Research Programme, National Institute for Health and Care Excellence (NICE), Manchester, UK

Panelists:

Rob Thwaites, MA, Senior Director, Takeda, London, UK

Hans-Georg Eichler, MD, MSc, Senior Medical Officer EMA, European Medicines Agency, London, UK

Sarah Garner, PhD, BPharm, Associate Director of Science Policy and Research, National Institute for Health and Care Excellence (NICE), London, UK

ISSUE:

A key aspiration that is shared between regulators, health technology assessment (HTA) agencies and the pharmaceutical industry is to have evidence that will allow patients earlier access to new effective medicines across all jurisdictions. This requires a global development strategy that produces evidence of effectiveness to meet the differing needs of regulatory and HTA agencies. The panel will debate the minimum requirements for this to be achievable, making reference to current and recent collaborative initiatives. Pall Jonsson will moderate and introduce key themes and issues. Rob Thwaites will represent the industry perspective and describe how the Innovative Medicine Initiative's GetReal project is exploring the acceptability of real-world evidence (RWE) in effectiveness research. Hans-Georg Eichler will discuss the regulatory perspective and whether RWE has a role in regulation. Sarah Garner will provide a HTA perspective and discuss whether collaborative solutions can be developed.

OVERVIEW:

Evidence on comparative effectiveness is a necessary condition for effective and cost-effective health care. In order for the development of such evidence to be viable in different jurisdictions, a number of minimum requirements have to be met, including: agreement on outcomes; infrastructure to capture patient-level outcomes; methodologies to analyse the data; and agreement that the data are acceptable and what role they will play in decision-making. Views on relevance and acceptability of different types of real-world data, evidence and methods vary across stakeholders and jurisdictions. The panel will reflect on their different perspectives and debate with the audience whether a convergence of real-world data methods and polices can be achieved across jurisdictions.

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

8:45 - 9:45
Room: Hall E1 (L0)

IP9: INCENTIVISING RESEARCH INTO THE EFFECTIVENESS OF MEDICAL DEVICES

Moderator:

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

Panelists:

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

Mirella Marlow, MBA, MA, Programme Director, Devices and Diagnostics Systems, NICE (National Institute for Health and Clinical Excellence), London, UK

Adrian Griffin, MSc, Vice President HTA & Reimbursement Policy, Johnson & Johnson, High Wycombe, UK

ISSUE:

Medical devices (MDs) often obtain market authorization with less evidence on effectiveness than other health technologies. This is mainly because of the lack of a legal requirement to conduct adequately controlled clinical studies, as is the case for pharmaceuticals. The lack of effectiveness evidence, especially comparative effectiveness evidence, limits the possibilities for health technology assessment (HTA). Therefore, it is important to discuss what incentives can be given to improve the quality and quantity of the effectiveness evidence for MDs.

OVERVIEW:

Medical devices (MDs) often obtain market authorization with much less evidence on clinical effectiveness than other health technologies. This is mainly because of the lack of a legal requirement to conduct adequately controlled clinical studies. Also, HTA agencies often accept claims of equivalence, which further reduces the incentives to manufacturers to conduct adequately controlled clinical studies, since they could spend time and effort collecting clinical evidence that a ‘fast-followers’ can claim also applies equally to their own product. Therefore, although device manufacturers have patent protection, they are not granted data exclusivity in the same way as pharmaceuticals. The lack of effectiveness evidence, especially comparative effectiveness evidence, limits the possibilities for health technology assessment, although effectiveness evidence can be gathered pre-market (i.e. through conducting controlled clinical trials in an experimental setting), or post-market, through clinical studies undertaken in regular clinical practice. Post-market effectiveness research may be more important for MDs than pharmaceuticals, as the performance of the device often depends on the interaction with the user (the so-called learning curve). This suggests that the solution to the problem of inadequate effectiveness evidence should address the issue of conducting clinical research in both the pre- and post-market phase. This panel will discuss the incentives that could be given to increase the range and quality of effectiveness evidence both pre- and post-marketing of MDs.

13:45 - 14:45
BREAKOUT SESSION

13:45 - 14:45
Room: Hall A (L2)

IP10: ADAPTIVE PATHWAYS AND PATIENT ACCESS – PUSHING PAYER BOUNDARIES OR FACILITATING NEW PAYMENT MODELS?

Moderator:

Jacoline Bouvy, PhD, Scientific Adviser, National Institute for Health and Care Excellence (NICE), London, UK

Panelists:

Hans-Georg Eichler, MD, MSc, Senior Medical Officer, European Medicine Agency, London, UK

Ad Schuurman, MSc, MBA, President of MEDEV, Zorginstituut Nederland, Diemen, The Netherlands

Claudine Sapede, PharmD, MSc, Global HTA & Payment Policy Lead, F. Hoffmann-La Roche Ltd, Basel, Switzerland

ISSUE:

 Adaptive pathways to medicines development could enable patients with earlier access to promising treatments in areas of high unmet medical needs. However, the implications of such pathways for access decisions at the national or regional levels remain to be explored. How health technology assessment (HTA) bodies, payers and manufacturers will deal with product value uncertainty and how this may affect solutions in the form of managed-entry agreements (MEAs)? In this panel session, learnings from payers, HTA agencies, and industry’s experience with MEAs will be shared along with their perspectives on the challenges and enablers on applicability of MEAs to possible adaptive scenarios. Jacoline Bouvy will talk about IMI ADAPT-SMART, where such questions are currently being explored. Hans-Georg Eichler will share the regulatory perspective on adaptive pathways. Ad Schuurman will provide the payer perspective and Claudine Sapede will represent the industry perspective.

OVERVIEW:

 Medicines Adaptive Pathways to Patients (MAPPS) seeks to foster access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion. From an access perspective, the iterative process of MAPPS implies that stakeholders (regulators, HTA agencies and payers, manufacturers, patient groups, clinicians, etc.) find implementable solutions for decision on pricing and coverage while dealing with remaining uncertainties regarding the product therapeutic and economic value, warranting additional evidence generation in real life. Solutions may include more flexible approaches to pricing and reimbursement than those currently used. Will there be more use of outcome-based agreements that tie the price of the medicine to its results? These are questions that the panel will discuss.


13:45 - 14:45
Room: Hall D (L -2)

IP11: EMERGING US VALUE FRAMEWORKS: ARE THERE LESSONS FROM - OR FOR - EUROPE? (Invited Issue Panel)

Moderator:

Richard J. Willke, PhD, Chief Science Officer, International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Lawrenceville, NJ, USA bio

Panelists:

Peter J. Neumann, ScD, Professor & Director, Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts University, Boston, MA, USA bio

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

Finn Børlum Kristensen, MD, PhD, Professor, University of Southern Denmark, Hilleroed, Denmark bio

ISSUE:

 An ISPOR Special Task Force is currently working on an initiative to identify and discuss key methodological issues in defining and applying value frameworks to health care resource allocation issues, and has convened a Special Task Force (STF) that will collaborate to produce a white paper that advises on the appropriate definition and use of high-quality value frameworks. A number of methodological issues have been identified that have created concerns about the validity of current US value frameworks for decision-making from various perspectives. However, a number of European countries have essentially been using types of value assessments for decision-making, and many of these same concerns have already been considered in those countries. How should the ISPOR STF consider European approaches and pick the most applicable from among them, and are there areas where the US should adapt and potentially improve on, European approaches, in order to address the concerns that have stimulated the creation of these frameworks?

OVERVIEW:

 In recent years a number of US-based organizations have put forth Value Assessment Frameworks, employing a variety of methodologies and perspectives. Most, if not all, of the issues they have been addressing have already been considered in existing European HTA guidelines, such as the Eunetha Core Model, although some US-based frameworks have adopted considerably different approaches, more oriented towards shared clinician-patient decision-making. Based on these approaches, it appears that conventional cost-utility analyses will not completely account for all the factors felt to be important in US value frameworks.

15:15 - 16:15
BREAKOUT SESSION

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

IP12: VALUING TRANSFORMATIVE MEDICINES IN RARE DISEASES: METHODS AND MADNESS

Moderator:

Paul Hodgkins, PhD, Vice President, Vertex Pharmaceuticals Incorporated, Boston, MA, USA

Panelists:

Ron Akehurst, DSc, Strategic Director, BresMed Health Solutions, Sheffield, UK

Alastair Kent, OBE, Director, Genetic Alliance UK, London, UK

Maarten Postma, PhD, Professor Pharmacoeconomics, Pharmacy Dept, University of Groningen, Groningen, The Netherlands

ISSUE:

Innovative medicines for rare diseases are often transformative for patients, especially when they are the first medicines designed to treat the underlying cause of disease. They may offer significant value to patients, caregivers and society. However, the ability to demonstrate clinical benefit and cost effectiveness at the time of regulatory approval using existing methodologies can be challenging. Paul Hodgkins (moderator) will introduce this issue and the panel. Ron Akehurst will present the case for largely adhering to current methods and evidence standards. Maarten Postma will consider alternative methodological approaches and data sources that have been and can be used. Alastair Kent will focus on patient engagement and the justification for adaptation of existing HTA processes to better evaluate innovative medicines.

OVERVIEW:

Evolution of the regulatory pathways for transformative therapies is enabling access to medicines at an earlier stage than ever before. To benefit from advances in clinical research in rare diseases and to take advantage of this evolving regulatory framework, an appropriately timed and conducted value assessment of these therapies is required. A balance is needed between early access, evidence of clinical benefit, acceptable cost effectiveness and the application of integrative and iterative methodologies for assessment. The issues faced in rare diseases are exemplified when a therapy may primarily offer survival benefits that are many years in the future and potentially subject to relevant uncertainty but costs accrue immediately. Can faster patient access to medicines be achieved whilst satisfying the need for demonstration of clinical and economic value of transformative medicines at time of approval? What can be learned from recent HTAs of rare diseases? Does this require decision makers to accept higher degrees of uncertainty within a broader context of benefits? This panel will review the opportunities and challenges for assessment of innovative medicines for rare diseases.

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

16:30 - 17:30
Room: Hall A (L2)

IP13: REAL WORLD EVIDENCE TO SUPPORT VALUE PROPOSITION AND REIMBURSEMENT AT LAUNCH: THE ASPIRATIONAL MEETS THE IMPRACTICAL?

Moderator:

Kari Edwards, PhD, Managing Partner, Harbinger Health, LLC, Los Angeles, CA, USA

Panelists:

Adrian R. Levy, PhD, Professor & Head, Community Health and Epidemiology, Dalhousie University, Nova Scotia, AL, Canada

Arie Barlev, PharmD, MS, Senior Director, Health Economics and Outcomes Research, Medivation, Inc., San Francisco, CA, USA

Andrew Briggs, DPhil, William R Lindsay Chair of Health Economics and Professor of Health Economics, University of Glasgow, Glasgow, UK

ISSUE:

Real World Evidence (RWE) is the latest in a series of buzzwords among teams responsible for demonstrating the value of new products at launch. Driven by requirements of payers for RWE that demonstrates effectiveness and safety among populations in which products will be used, such requirements are highly aspirational. We know that quality evidence on effectiveness and safety of an intervention, compared to the relevant comparator, in the population of interest, outside the randomized controlled trial (RCT) setting, is ideally what reimbursement authorities seek. But just how practical is this as a requirement in the face of the methodological challenges and competing demands faced by companies bringing new products to market?

OVERVIEW:

In this panel, we explore the tension between the aspirational and practical elements of generating RWE in a timely fashion to support reimbursement at product launch. Kari Edwards will moderate and provide context for the debate through several illustrative case studies. Adrian Levy will provide the payer perspective, arguing in favor of the provision of RWE to allow timely assessment of likely effectiveness to support reimbursement submissions upon product launch. Arie Barlev will provide an industry perspective on practical challenges associated with collecting evidence within a short post-approval timeframe and will highlight the problem of selection bias when measuring relative effect in populations exposed to newly approved agents compared with standards of care. Andrew Briggs will represent the health economics perspective, demonstrating the use of modelling techniques to exploit the rigorous nature of clinical trial data while making the reimbursement story fit for purpose - including the incorporation of RWE into modelling approaches to further demonstrate product value.

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

8:45 - 9:45
Room: Hall F1 (L0)

IP14: EXTENDING THE USE OF BIOSIMILAR DRUGS: ARE WE WILLING TO ACCEPT THE UNCERTAINTY RELATED TO SWITCHING IN ORDER TO IMPROVE PATIENT ACCESS TO MODERN MEDICINES?

Moderator:

Rok Hren, PhD, MSc IHP(HE), Assistant Professor, University of Ljubljana, Ljubljana, Slovenia

Panelists:

Tomas Tesar, PharmD, PhD, MBA, Member of the Reimbursement Committee of the Slovak Ministry of Health, Union Health Insurance Fund, Bratislava, Slovakia

Andras Inotai, PharmD, PhD, Senior Pharmacoeconomist, Syreon Research Institute, Budapest, Hungary

Duša Hlade Zore, MD, Representative of Patients’ Rights, Ministry of Health, Government of the Republic of Slovenia, Ljubljana, Slovenia

ISSUE:

As prices of modern biological treatments are established in high income countries, they are often not justifiable in Central Eastern Europe (CEE). Therefore many of these drugs are only available with various access limits in the region. Increased use of biosimilar drugs, especially in countries with limited healthcare resources may contribute to improved patient access. Biosimilars offer therapeutic equivalence with original biologics at reduced drug price for treatment naive patients. However, as opposed to small-molecule generic drugs, biosimilars cannot be considered identical to their originators. Therefore, switching patients receiving maintenance drug therapy with original biological drugs to biosimilars may not be an obvious option for clinicians, regulators and payers due to fear of adverse immunological reactions or reduced therapeutic effect.

OVERVIEW:

Mandatory phase III trials test equivalent efficacy and safety of biosimilar drugs compared to original biological drugs in treatment naive patients in one indication. Even if biosimilars are tested among maintenance patients, these trials may not be adequately powered to detect adverse immunologic reactions due to limited number of patients and follow-up period. Extensive use of biosimilars may pose a trade-off for decision makers: either taking the risk of uncertain effects related to switching biopharmaceuticals or accepting the opportunity cost from unrealised savings and improvement in patient access. Panelists will discuss: 1. how to improve the evidence-based reimbursement decision-making on biosimilars, 2. how to balance handling risks of switching and benefits from potential savings, and 3. how to implement this decision-making process in practice. The first panelist is a payer and member of reimbursement committee of a CEE country. The second panelist will represent the perspective of a researcher. The third panelist is a patients’ rights expert appointed by government from one of the most developed jurisdictions in CEE and who will present the perspective of patients.


8:45 - 9:45
Room: Hall E1 (L0)

IP15: IS BALANCING VALUE DEMONSTRATION FOR PAYER AND PATIENT INTERESTS A FEASIBLE NOTION?

Moderator:

Kathleen E. Hughes, MBA, Vice President, Health Economics and Outcomes Research, Avalere Health LLC, Washington, DC, USA

Panelists:

Sachin Kamal-Bahl, PhD, Vice President & Head, Global Health & Value Innovation Center, Pfizer, Collegeville, PA, USA

Adrian Towse, MA, MPhil, Director, Office of Health Economics, London, UK

Alastair Kent, OBE, Director, Genetic Alliance UK, London, UK

ISSUE:

Maintaining patient focus in healthcare decision-making is a universally-accepted approach to ensuring positive outcomes. “Value frameworks” share building blocks: health outcomes of interest; evidence to measure effect of treatment on outcomes; combination of outcomes into single health benefit measures; and comparison of care costs with health benefits. ESMO framework is exceptional in excluding costs. Framework developers and health technology assessment (HTA) organizations claim differences in “valuation” tool orientation to patient interests. Even frameworks claiming patient-centricity are criticized for over-emphasizing methods to incorporate payer versus patient perspectives. While tension between payer and patient perspectives is inevitable, many agree frameworks can be improved and diverse interests reconciled therein. Others claim two viewpoints cannot be reconciled in a single framework and (a) separate patient-centric framework(s) developed.

OVERVIEW:

Moderator will provide a brief overview of existing value frameworks and HTA approaches, characterizing methodology and payer/patient perspective balance. Panelists will critique frameworks/methods on modifications inclusive of patient-focused elements and/or patient-centric use and possibility of reconciling patient and payer interests within a framework. Finally, panelists will debate how addressing payer and patient interests simultaneously could impact research functions: broader evidence generation, patient-reported outcomes development, and establishment of quality measures encompassing payer/patient perspectives.

10:00 - 11:00
BREAKOUT SESSION
Economic Outcomes Research Issues

10:00 - 11:00
Room: Hall E1 (L0)

IP16: SHOULD HEALTH TECHNOLOGY ASSESSMENT GUIDELINES RECOMMEND INCLUSION OF FUTURE MEDICAL COSTS?

Moderator:

Andrew Briggs, DPhil, MSc, William R Lindsay Chair of Health Economics and Professor of Health Economics, University of Glasgow, Glasgow, UK

Panelists:

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

Pieter van Baal, PhD, Associate Professor, Institute for Health Policy and Management (iBMG), Erasmus University Rotterdam, Rotterdam, The Netherlands

Ad Antonisse, MSc, Director Economic Affairs (Market Access & Public Affairs), AstraZeneca, Zoetermeer, The Netherlands

ISSUE:

New medical technologies that prolong life result in additional health care use in life years gained. Currently, many health technology assessment (HTA) guidelines recommend that “unrelated future costs” of a medical technology are excluded from an appraisal (so the costs of caring for a patient’s dementia after preventing their death from stroke would not be included in a cost effectiveness analysis of thrombolysis treatment). Recently, Dutch guidelines for HTA have changed and recommend inclusion of future unrelated medical costs. This begs the question whether other HTA guidelines should also recommend inclusion of future unrelated medical costs?

OVERVIEW:

The panelists will present and discuss the pros and cons of including future unrelated costs in HTA. Alec Morton will summarize theoretical arguments for and against inclusion of future unrelated medical costs. Pieter van Baal will discuss how these costs can be estimated in practice. Finally, Ad Antonisse will present the industry point of view. The audience will be invited to participate in the discussion whether HTA guidelines should recommend inclusion of future unrelated medical costs.

Patient-Reported Outcomes & Patient Preference Research Issues

10:00 - 11:00
Room: Hall A (L2)

IP17: CAN WE REALLY COMPARE AND AGGREGATE PATIENT-REPORTED OUTCOME DATA BETWEEN PEOPLE AND SETTINGS? IMPLICATIONS FOR CLINICAL TRIALS AND HEALTH TECHNOLOGY ASSESSMENT

Moderator:

Nancy Devlin, PhD, Director of Research, Office of Health Economics, London, UK

Panelists:

Michael Herdman, MSc, Director, Insight Consulting and Research, Mataró, Spain

Paula Lorgelly, PhD, Deputy Director, Office of Health Economics, London, UK

Andrea Manca, MSc, PhD, Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK

ISSUE:

Patient-reported outcomes (PROs) are routinely collected in clinical trials, cohort studies and population health surveys. They are used to compare health across heterogeneous populations, e.g. across countries, different cultural settings and socio-demographic groups. This panel looks at whether PRO data are truly comparable across different groups of individuals and the implications e.g. for aggregating PRO data from multi-country randomized controlled trials (RCTs) to provide an overall assessment of treatment efficacy. We examine how comparability can be improved and discuss the implications for using PRO evidence to inform health policy.

OVERVIEW:

 This panel will discuss the comparability of PROs from their conceptualization through statistical methods. Using examples from the literature and his own experience in adapting PRO instruments, particularly the EQ-5D, for use in other countries, Mike Herdman will discuss the importance of taking into account conceptual and linguistic issues when using instruments cross-culturally. He will also present methods for assessing and improving comparability, particularly in relation to preference-based measures, and will discuss possible reasons for a lack of comparability and implications for cross-cultural comparisons and data transferability within the context of HTA. Further, PRO data may not be comparable if groups of individuals systematically differ in their use of response categories, due to e.g. response-scale heterogeneity or differential item functioning (DIF). Paula Lorgelly will present her work using anchoring vignettes to identify response bias and DIF in self-assessed health and the EQ-5D, in order to improve comparability. Andrea Manca will further explore the issue of heterogeneity, what specifically drives heterogeneity in PROs, and what data we need to be able to disentangle that heterogeneity. The panel will conclude with a discussion on the importance of evaluating and enhancing the comparability of PRO instruments and results across different cultural and socio-demographic groups and how the issue should be handled, given an increasingly global context for RCTs and HTA.

13:45 - 14:45
BREAKOUT SESSION

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

IP18: PATIENT PREFERENCES IN DRUG EVALUATION: WHICH METHOD SHOULD WE USE?

Moderator:

Heather L. Gelhorn, PhD, Senior Research Scientist, Outcomes Research, Evidera, Bethesda, MD, USA

Panelists:

Tommi Tervonen, PhD, Senior Research Associate, Evidera Ltd, London, UK

Axel C. Mühlbacher, PhD, MBA, Professor, Health Economics and Health Care Management, Institute Health Economics and Health Care Management, IGM, Hochschule Neubrandenburg, Neubrandenburg, Germany

Douwe Postmus, PhD, Researcher, Department of Epidemiology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

ISSUE:

 How should patient preferences be elicited for use in comparative treatment evaluation?

OVERVIEW:

 Evaluating treatments based on clinical profiles and convenience characteristics brings treatment choice closer to the patient than generic evaluation frameworks such as quality-adjusted life year. When no dominant treatment option exists, such valuation exercises must establish trade-offs between profiles of competing treatments. Various health authorities such as the United States Food and Drug Administration and the Institute for Quality and Efficiency in Healthcare have highlighted the importance of eliciting the trade-offs directly from patients. Multiple techniques such as discrete choice experiments and swing weighting are applicable for eliciting patient trade-off preferences. Currently, there is no guidance on which elicitation method is most appropriate in different circumstances. This panel asks which elicit method is most appropriate, and when, taking into account of both the theoretical and practical differences between the methods. Panelists will draw on their experience from ISPOR conjoint analysis experimental design task force (Mühlbacher), European Medicines Agency preference-based benefit-risk pilot (Postmus) and methodological development (Tervonen) to defend their points of view.

Use of Real World Data Issues

13:45 - 14:45
Room: Hall A (L2)

IP19: USING OBSERVATIONAL (‘REAL WORLD’) DATA IN HEALTH TECHNOLOGY ASSESSMENT: ROUTE TO CONFUSION OR BETTER DECISIONS?

Moderator:

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

Panelists:

Mark Sculpher, PhD, Professor of Health Economics, Centre for Health Economics & Director, Programme on Economic Evaluation and Health Technology Assessment, University of York, Heslington, York, UK

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

Michael Seewald, PhD, Global Head Real World Evidence, Novartis, Basel, Germany

ISSUE:

Regulators are increasingly planning to rely on observational (‘real world’) evidence to inform licensing, which also becomes central to funding decisions. This promises a low cost means of shortening the period before a new medicine is available. However, the lack of randomization in these datasets risks biased estimates of treatment (cost)-effectiveness. The session will debate the strengths and weaknesses of increasing use of real world evidence. Garner will moderate and set out the evolving policy landscape and opportunities offered by such data. Sculpher will argue that there are risks associated with non-randomized evidence and methods are needed to incentivize appropriate studies. Abrams will outline novel methods to facilitate reliable use of real world evidence, particularly when randomized controlled trials (RCTs) are challenging to conduct. Seewald will provide a manufacturer’s perspective and highlight that appropriate use of real world evidence can reduce the costs of drug development.

OVERVIEW:

To make access to some medicines more timely, regulators are licensing some products (e.g. those with high unmet need) without RCT evidence. They may make such decisions conditional on further data collection that is generally observational in nature. Health systems are obliged to rely on effectiveness evidence that is generated for regulatory purposes, whether RCTs or observational in nature. Furthermore, many systems are hoping that uncertainty associated with the value of products at launch can be addressed by using routine observational (‘real world`) data (e.g. the reformed Cancer Drugs Fund in England). Observational data offer potential strengths in terms of low costs and representativeness. However, they risk providing biased estimates of treatment effectiveness. The impact of real world data on the quality of funding decisions is a matter of debate. Risks could be reduced by better methods to analyze such data and/or by seeking novel ways to incorporate routine data in RCTs.

15:00 - 16:00
BREAKOUT SESSION

15:00 - 16:00
Room: Hall F2 (L0)

IP20: IS REAL-WORLD DATA AS AN INDISPENSABLE SOURCE FOR THE ASSESSMENT OF NEW ONCOLOGY TREATMENTS POSSIBLE?

Moderator:

Stefano Capri, PhD, Adjunct Professor, School of Economics and Management, LIUC University, Castellanza VA, Italy

Panelists:

David Chao, BMBCh, FRCP, DPhil, Consultant in Medical Oncology, Royal Free Hospital, London, UK

Ken Redekop, PhD, Associate Professor, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands

ISSUE:

 Randomized controlled trials (RCTs) are the gold standard for assessing efficacy and safety of new treatments for health technology assessment (HTA) bodies and payers. However, RCTs may not always provide sufficient evidence to validate a treatment’s benefit, particularly in an era of numerous therapeutic advances, where approvals are granted more rapidly and with less mature data. Real-world data (RWD) are seen as a means of reducing perceived uncertainty in RCT data, achieving long-term outcomes or providing additional information on the value of treatments; nevertheless, they also raise new challenges, including the potential for multiple decision points, and deciding which sorts of RWD are most appropriate for HTA. Should RWD be required in all circumstances, or only where further evidence is required?

OVERVIEW:

The rapidly evolving landscape of oncology therapies raises challenges for HTA bodies and payers. Increased demand from patients to gain earlier access to innovative treatments has triggered faster regulatory approvals, meaning new therapies are becoming available with less mature RCT data or non-comparative phase 2 trials. Constrained healthcare resources and an ageing population place ever-greater importance on pricing and reimbursement decisions. Harnessing RWD provides an opportunity to gain powerful insight into how a therapy can perform in real-life clinical settings, and where to accelerate access. However, use of RWD has its challenges, with both trial design and decision-making. In many cases, RWD studies aim to provide better understanding of the distribution of costs during the different phases of care, according to the main variables from epidemiological and resource utilization perspectives. Stefano Capri will moderate and provide comments based on Italy’s extensive experience with RWD in HTA; David Chao will present what is currently being implemented in England with the Cancer Drugs Fund and the Systemic Anti-Cancer Therapy dataset; and Ken Redekop will describe the Netherlands’ approach.

Health Policy Development Using Outcomes Research Issues

15:00 - 16:00
Room: Hall E1 (L0)

IP21: IS OFF-LABEL DRUG USE ENHANCING OR LIMITING ACCESS FOR PATIENTS WITH RARE DISEASES?

Moderator:

Annabel Griffiths, PhD, Consultant, Costello Medical Consulting Ltd, Cambridge, UK

Panelists:

Jack Scannell, DPhil, Co-head European Pharmaceuticals, UBS Investment Bank, London, UK

Oliver Timmis, MA, CEO, AKU Society, Cambridge, UK

Pilar Pinilla-Dominguez, MSc, Technical Adviser, NICE Scientific Advice, National Institute for Health and Care Excellence, London, UK

ISSUE:

Of the 7,000 recognized rare diseases, only ~400 have currently licensed treatments, meaning clinicians often have no choice but to prescribe drugs off-label. Although providing a short-term solution for some individuals, off-label use can theoretically have a negative impact on long-term patient access, by potentially reducing the incentive to conduct time-consuming and costly evidence generation processes in order to inform licensing or reimbursement applications. The potential failings in the current system in terms of off-label drug use in rare diseases, and their impact on patient access will be discussed, and strategies suggested to improve the process at the stages of drug development, clinical practice and reimbursement. Annabel Griffiths will moderate and provide an overview of the current system. Jack Scannell will present the financial perspective and the role of off-label treatments in drug development for rare diseases. Oliver Timmis will voice the patient perspective, highlighting challenges faced by the rare diseases patient community due to the use of off-label treatments. Pilar Pinilla-Dominguez will represent the HTA perspective, and discuss the impact of off-label drugs on the methodologies NICE uses for evaluations.

OVERVIEW:

Off-label prescriptions are particularly common in rare diseases, with estimates suggesting that 90% of all prescribed treatments for rare diseases lack specific FDA approval. The use of off-label treatments can, however, be detrimental to long-term patient access, limiting the patient population eligible for inclusion in clinical trials and possibly reducing the incentive for formal licensing and reimbursement. This issue panel will explore the impact of off-label prescriptions on patient access in rare diseases, highlighting issues faced by industry, clinicians and payers, and asking what changes could improve the system for all stakeholders. The audience will vote on their opinion on the title question at the beginning and end of the issue panel; discussion time will be included, and questions from the audience encouraged throughout.

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