Issue Panels
Monday, 6 November 2017
11:15 - 12:15
BREAKOUT SESSION
Use of Real World Data Issues

11:15 - 12:15
Room: Hall 3

IP1: PRAGMATIC CLINICAL TRIALS TO ESTIMATE TREATMENT EFFECTS: ARE THEY WORTH THE EFFORT?

Moderator:

Rita M. Kristy, MS, Director - Statistics, Clinical and Outcomes Sciences, Astellas Pharma Global Development, Northbrook, IL, USA

Panelists:

Keith R. Abrams, PhD, CStat, Professor of Medical Statistics, NIHR Senior Investigator Emeritus & Head, Biostatistics Research Group, Department of Health Sciences, University of Leicester, Leicester, UK

Christoph Gerlinger, PD, Senior Director, Development, Pharmaceuticals, Bayer AG, Berlin, Germany

Helene Karcher, PhD, Managing Vice-President, Analytica Laser, Loerrach, Germany

ISSUE:

Pragmatic trials are designed to reflect real-world product use more accurately than highly controlled explanatory trials. Unfortunately, real world applicability also brings methodological challenges resulting in threats to valid inference. Consequently, pragmatic trials are often not appropriate for regulatory submission. However, information regarding the real-world product can provide useful information for payors, patients and providers. This issue panel will outline several pros and cons of pragmatic trials and discuss innovative (and likely controversial) methodological approaches to address their shortcomings. Advocates for the use of pragmatic clinical trials argue that they provide more generalizable and decision-relevant results than explanatory trials. There has been an increase in publications of studies that are termed ‘pragmatic’ in recent years. This issue panel will discuss the appropriate uses of different study designs, depending upon the endpoint/hypothesis tested.

OVERVIEW:

Advocates for the use of pragmatic clinical trials argue that they provide more generalizable and decision-relevant results than explanatory trials. There has been an increase in publications of studies that are termed ‘pragmatic’ in recent years. This issue panel will discuss the appropriate uses of different study designs, depending upon the endpoint/hypothesis tested.

Presented by the ISPOR Statistical Methods in HEOR Special Interest Group

Health Policy Development Using Outcomes Research Issues

11:15 - 12:15
Room: Clyde Auditorium (Armadillo)

IP2: HOW TO IMPROVE CONSISTENCY OF ORPHAN DRUG PRICING AND REIMBURSEMENT IN EUROPE? APPLICATION OF THE 'ORPH-VAL' PRINCIPLES IN GERMANY, FRANCE, AND THE UNITED KINGDOM

Moderator:

Lieven Annemans, PhD, MSc, Senior Full Professor of Health Economics, ICHER (Interuniversity Center for Health Economics Research), Ghent University - Brussels University, Ghent, Belgium

Panelists:

Karina Ehrig, PhD, Scientific advisor, Pharmaceuticals Department, Federal Joint Committee (G-BA), Berlin, Germany

Francois Meyer, PhD, Advisor to the President, International Affairs, Haute Autorité de Santé (HAS), Saint-Denis, France

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

ISSUE:

Rare diseases represent a major public health issue in Europe. Orphan medicinal product (OMP) legislation introduced in 2000 has led to an increase in new OMPs being developed, but patient access to these medicines is imperfect and variable between countries. This variation partly reflects challenges in pricing and reimbursement for OMPs and different approaches across Europe. In March 2017, the ORPH-VAL Working Group, a European multi-stakeholder group of rare disease experts, published a set of principles for value assessment and funding processes in rare diseases. These principles aim to help improve the quality and consistency of OMP P&R processes in Europe and ensure that value assessment and P&R processes reflect the specificities of rare diseases. This session will consider how the P&R systems for OMPs in Germany, France and UK align with the principles and explore opportunities for improvement and consistency.

OVERVIEW:

Professor Lieven Annemans, the chair of the ORPH-VAL working group will provide an overview of the 9 principles and the results from an assessment of the alignment of the principles with the P&R systems currently used in Germany, France and UK to assess OMPs. He will be accompanied by three payers from the countries of interest. The panelists will be invited to respond to the analysis and discuss the areas of alignment or misalignment in their countries and the potential need for change. They will subsequently be asked to think about how the OPRH-VAL principles could be implemented in practice in their respective country by 1) identifying opportunities and pathways they would recommend in order to align with the principles, 2) highlighting challenges or hurdles that may exist, and recommend pathways for improvements and potential reforms. Professor Lieven Annemans will summarise the arguments and invite the audience to provide their perspective.

14:15 - 15:15
BREAKOUT SESSION

14:15 - 15:15
Room: Hall 2

IP3: UNMET (MEDICAL) NEED: SHOULD STAKEHOLDERS ALIGN ON A DEFINITION?

Moderator:

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

Panelists:

Nicola Bedlington, Secretary General, European Patients’ Forum and Co-Founder, The Patient Access Partnership PACT, Brussels, Belgium

Neil McAuslane, PhD, Director, Center for Innovation in Regulatory Science, London, UK

Jo De Cock, MS, Administrateur General, INAMI - Institut National de l'Assurance Maladie-Invalidité, Brussels, Belgium

ISSUE:

Addressing unmet medical need is a key criterion for medicine prioritization and one of the requirements for a molecule candidate to qualify for an adaptive pathways approach and a number of accelerated regulatory review processes such as PRIME. Yet, there is not a common definition of the term and different qualifiers may be used to describe its application. The interpretations may also vary between stakeholder groups (regulators, payers, patients, medicine developers). In this context achieving stakeholder alignment on a medicine’s eligibility for accelerated development and patient access pathways remain challenging. To what extend is unmet medical need considered in “value frameworks” and reimbursement decision-making? Would a commonly-agreed framework (content and process) be a potential approach? Would this be a valuable and desirable outcome? In this panel session, representatives from different stakeholders groups will share their perspectives.

OVERVIEW:

The term “unmet medical need” is used in different contexts in the medicines lifecycle, including for making decisions by industry, regulatory, HTA bodies, payers, patients, policy makers. The definitions and interpretations of the term may also vary between countries and constituents. The lack of common understanding leads to potentially inconsistent signals between incentives for research and development models and the actual patient’s access to new treatment options. These are agenda items also identified by the WHO and the European Parliament which calls on the EU Commission to define clearly the concept of unmet medical need and call on stakeholders to explore new ways of delivering affordable medicines.


14:15 - 15:15
Room: Clyde Auditorium (Armadillo)

IP4: VALUE ADDED MEDICINES: TIME TO ADJUST THE HEALTH TECHNOLOGY ASSESSMENT DECISION FRAMEWORKS?

Moderator:

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

Panelists:

Ulf Persson, PhD, Professor, The Swedish Institute for Health Economics (IHE), Lund, Sweden

Luigi Burgio, MSc, Head of Market Access & Government Affairs, Market Access, Teva Italia S.r.l., Assago (MI), Italy

Mondher Toumi, MD, PhD, MSc, Professor, Public Health, Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille University, Marseille, France

ISSUE:

Value added medicines (VAMs) are medicines based on known molecules that follow 3 drug repurposing models, i.e., reformulation, repositioning and combination. While VAMs deliver substantial value to patients and society, they receive little acknowledgement from European health technology assessment (HTA) decision frameworks. The panel will debate the current challenges in capturing the full value of VAMs through HTA decision frameworks, and on key recommendations for HTA policy changes. Mr L. Burgio will provide the pharmaceutical industry’s perspective, starting with a presentation of key examples of VAMs and the potential value delivered to patients and society, and describing the current challenges in capturing this value in HTA decision frameworks. Pr. M. Toumi, an expert in the HTA field, will propose key recommendations for improved recognition of the value provided by VAMs. Pr. U.Persson, a HTA representative, will discuss how these changes could be integrated in various HTA models and the challenges involved. Each panelist will speak for 10 minutes, followed by a 15-minute panel discussion, and a 15 minutes Q&A from the audience.

OVERVIEW:

VAMs may match patients’ preferences and bring substantial value to patients, contributing to enhanced adherence, health outcomes or quality of life. VAMs may also bring substantial value to society by addressing a number of medicine-related healthcare inefficiencies, improving healthcare provision and organisation, and contributing to sustainability of healthcare systems. However, current HTA decision frameworks struggle to fully acknowledge VAMs’ value, calling for policy changes. For example, VAMs may not have the opportunity to demonstrate their added value, due to ineligibility for HTA in some countries. Some of VAMs’ benefits may be difficult to demonstrate, such as improvement in patient adherence, and are not currently well-acknowledged in the final HTA deliberative process. VAMs may also highly benefit from early HTA advice, but are not frequently eligible.

15:45 - 16:45
BREAKOUT SESSION

15:45 - 16:45
Room: Hall 2

IP5: EUROPEAN HTA 2020: WHAT IS THE ROAD MAP TO EFFECTIVE PERMANENT COOPERATION? (INVITED ISSUE PANEL)

Moderator:

Tim Wilsdon, Msc, Vice President London Office, Charles Rivers Associates, London, UK

Panelists:

Wim Goettsch, PhD, Director, EUnetHTA JA3 Directorate, The National Healthcare Institute (ZIN), Diemen, The Netherlands

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

Nicola Bedlington, BA Hons, Secretary General, European Patients Forum (EPF), Brussels, Belgium

Flora Giorgio, Head of Sector, Health Technology Assessment, Directorate General for Health and Food Safety (DG SANTE), European Commission, Brussels, Belgium

ISSUE:

After 10 years of EUnetHTA collaborative project work and the launch of impact assessment process in 2016 by the European Commission, it is a perfect time to identify pragmatic solutions not only for making the European cooperation on HTA permanent after 2020 but also to support implementation of joint work at national level. The results of the impact assessment process and formal decision are expected by the end of 2017, thus making ISPOR’s Glasgow conference an ideal opportunity to review progress and understand stakeholder groups’ positions, challenges and opportunities for successfully working together towards a common goal in 2020 and beyond. What still needs to be done by 2020 to support a sustainable EU cooperation on HTA and ensure adequate uptake of joint HTA work at national level? What needs to be changed in how stakeholders work within and across each other? And what are the most important lessons from EUnetHTA for the next stage of the EU cooperation on HTA?

OVERVIEW:

Any way forward should be informed both by the needs of the parties participating in the cooperation and by previous experiences in the national and EUnetHTA HTA processes, taking into account the policy processes within and across European countries. Concrete experiences and suggestions on how to improve the processes of joint work on HTA and how to implement the results of this work in national contexts would be the focus of the panel discussion. The panel will also address the opportunities and challenges of implementation for preferred policy option(s) initially outlined by the European Commission – each panelist will present the view of the constituency they represent: EUnetHTA/national HTA agencies, patients and industry. Issue Panel is organized in cooperation with EUnetHTA (European network for HTA).


15:45 - 16:45
Room: Hall 3

IP6: ARE EXISTING HEALTH TECHNOLOGY ASSESSMENT REQUIREMENTS INADEQUATE FOR ESTABLISHING VALUE FOR POTENTIALLY TRANSFORMATIVE GENE THERAPIES?

Moderator:

Kellie Meyer, PharmD, MPH, Senior Director, Global Health Economics, Xcenda, Palm Harbor, FL, USA

Panelists:

Ron Akehurst, PhD, Chairman, BresMed Health Solutions, Sheffield, UK

Clark Paramore, MSPH, Head of Value Demonstration, bluebird bio, Cambridge, MA, USA

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

ISSUE:

Viewpoints from different stakeholders will be addressed during this issue panel session related to approaches for assessing the value of gene therapies. Akehurst (National Institute for Health and Care Excellence [NICE] Highly Specialised Technologies Evaluation Committee [HSTEC] member) will identify issues particular to gene therapy and present the case that HTA approaches currently adopted are adequate for assessment. Paramore (pharmaceutical industry) will discuss challenges involved in bringing gene therapies to market, considering possible role of adaptive pathways and obstacles presented by current HTA systems. Towse (health economist) will share his perspective on approaches to evaluating cost-effectiveness of gene therapies, considering the high up-front costs and potential long-term and/or transformative benefits, as discussed at the Institute for Clinical and Economic Review (ICER) Gene Therapy Summit.

OVERVIEW:

Gene therapies offer promise of one-time treatment leading to transformative and lifelong benefits for patients with rare/orphan diseases. Evaluating effectiveness of gene therapies at launch is daunting using existing approaches to assess benefit. A solution may be use of adaptive pathways, which involve approval based on early data/surrogate endpoints, supplemental collection of real-world data, and discussions with patients and HTA agencies throughout the development process. High cost of gene therapies may make it difficult to evaluate value, due to concerns related to affordability by payers/society. Questions have been raised regarding need for a new model of pricing/reimbursement for gene therapies, and need for new/modified HTA methodologies. Two gene therapies, Glybera (alipogene tiparvovec) and Strimvelis (GSK2696273), have been approved by European Medicines Agency (EMA) and no gene therapy has been approved by Food and Drug Administration (FDA). Glybera has not been reviewed by NICE and has now been withdrawn from the market. Strimvelis is currently undergoing evaluation by NICE through HSTEC. ICER announced it will evaluate the first gene therapy submitted to the FDA, voretigene neparvovec.

17:00 - 18:00
BREAKOUT SESSION

17:00 - 18:00
Room: Clyde Auditorium (Armadillo)

IP7: 'MIND THE GAP!': HOW SHOULD WE MANAGE THE DIFFERENCE BETWEEN REGULATORY AND REIMBURSEMENT EVIDENCE REQUIREMENTS FOR MEDICAL DEVICES?

Moderator:

Bjoern Schwander, RN, BSc, MA, General Manager, Agency for Health Economic Assessment and Dissemination, AHEAD GmbH, Loerrach, Germany

Panelists:

Richard Charter, MSc, Head of Market Access & Pricing, EMEA, Becton Dickinson, Basel, Switzerland

Karen Facey, PhD, Honorary Research Fellow and Evidence Based Healthcare Policy Consultant, University of Edinburgh, Edinburgh, UK

Nneka C. Onwudiwe, PhD, PharmD, MBA, PRO/PE Regulatory Review Officer, U.S. Government, Silver Spring, MD, USA

ISSUE:

For most medical devices, evidence requirements for regulatory approval are significantly less than evidence required for reimbursement approval. Regulatory agencies are re-examining the evidence required as demonstrated in US and in Europe with the new Medical Device Regulation. In contrast, medical devices are more likely to be developed by small/medium companies, who can struggle to finance large-scale clinical trials; moreover, short product life cycles make significant investments in lengthy evidence generation impractical. How can we balance the desire to increase the evidence required for regulatory approval without having a negative impact on device development, innovation and patient access? How can patient involvement in HTA/evidence facilitate the trade-off between ensuring quality, safety and having access to innovations? The core question is how should we 'mind the gap' in evidence requirements between medical device regulation and reimbursement requirements?

OVERVIEW:

In April 2017, the new Medical Device Directive was approved in Europe: REGULATION (EU) 2017/745 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL. This will impact patient access for medical devices. The regulatory/reimbursement perspective including evidence requirements and timelines to fulfil them will be debated. How payers plan to implement decisions based on changing requirements will also be discussed. The perspective of medical device manufacturers will highlight what this means to R&D and innovation of medical technologies, and how they will overcome the challenges in producing higher evidence, including the role of real world evidence (RWE) and risk sharing agreements. Last, the panel will include an often overlooked perspective on evidence and reimbursement: patient inolvement in HTA. We will discuss what increased evidence means to patient involvement in HTA, as patients are the ones impacted by the changing access to medical devices and the goal of increased quality and safety of medical devices.

Presented by the ISPOR Medical Device and Diagnostics Special Interest Group


17:00 - 18:00
Room: Lomond Auditorium (Loch Suite)

IP8: FORECASTING PHARMACEUTICAL EXPENDITURE IN EUROPE: IS IT SUSTAINABLE?

Moderator:

Jorge Mestre-Ferrandiz, PhD, Economist, Independent Economics Consultant, Madrid, Spain

Panelists:

Tim Davis, MSc, Senior Principle, Consulting, QuintilesIMS, Basel, Switzerland

Claudio Jommi, PhD, Associate Professor, Department of Pharmaceutical Sciences, Università del Piemonte Orientale, Cergas (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management, Milano, Italy

Panos Kanavos, PhD, Associate Professor in International Health Policy, LSE Health, Medical Technology Research Group, London School of Economics and Political Science, London, UK

ISSUE:

Healthcare systems are under pressure to contain expenditure in the face of demographic and technological change. Policy-makers are increasingly focused on pharmaceutical prices as a source of the pressure on healthcare budgets. Data on historic and forecast growth of total pharmaceutical expenditure is therefore an important factor in this debate.Tim Davis will discuss historical and forecast pharma expenditure data covering a period from 2011 to 2021. This data is based on QuintilesIMS (QI) methodology using list prices and audited pharmaceutical volumes. Claudio Jommi will discuss the recent trends for managed entry agreements to set an increasing divergence between list and net prices in Europe. The impact on the expenditure data forecasts will be presented and set in the context of the broader macroeconomic situation. Panos Kanavos will discuss the broader implication of the analyses and address the question of what level of pharmaceutical expenditure in Europe is sustainable, and the implications for healthcare and pharmaceutical policy in Europe

OVERVIEW:

Forecasts for pharmaceutical expenditure are notably absent from the policy debate on drug prices in Europe. There are several sources of expenditure (e.g. OECD) but few forecasts which policy-makers and budget holders can use to, as accurately as possible, predict future needs. QuintilesIMS provide forecast data for the big 5 European pharmaceutical markets based on publicly available list prices of pharmaceuticals applied to audited volume data. This data shows a compound annual growth rate (CAGR) of 3% over the next 5 years. An initial assessment based on OECD and QI data suggests that the net figure could be 1 to 2% lower. The implications for pharmaceutical policy will be explored by the panelists and the question of what constitutes a sustainable growth rate in expenditure on pharmaceuticals will be set against the broader question of how much society should spend on healthcare.

Tuesday, 7 November 2017
8:45 - 9:45
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

8:45 - 9:45
Room: Lomond Auditorium (Loch Suite)

IP9: SHOULD RARE ONCOLOGY TREATMENTS BE CONSIDERED TRUE ORPHANS?

Moderator:

Annabel Griffiths, PhD, Consultant - Rare Diseases Lead, Costello Medical Consulting Ltd, Cambridge, UK

Panelists:

Elangovan Gajraj, MSc, Senior Technical Adviser, Scientific Advice, National Institute for Health and Care Excellence, London, UK

Pan Pantziarka, PhD, Project Co-ordinator, Anti-Cancer Fund, Strombeek-Bever, Belgium

Ivana Cattaneo, MBA, Public Affairs Director, Novartis Oncology Europe, Novartis Farma S.p.A, Origgio (VA), Italy

ISSUE:

Controversially, several high-profile, mass-market oncology drugs have been approved for the treatment of much smaller sub-indications (a practice known as 'salami-slicing'). In doing so, these drugs achieve orphan status based on the rare indication and benefit from incentives provided by the EU Regulation for Orphan Medicinal Products (ROMP). Such use of this legislation has been accused of wrongly allocating resources that could be used more effectively in non-oncological rare diseases. However, many regard incentives to be crucial for orphan oncology drug development. This panel will discuss whether the application of orphan drug regulation in this way is an unfair monopoly or a lifeline to patients.

Annabel Griffiths will moderate and provide an overview of the current situation. Elangovan Gajraj will represent the HTA perspective, discussing the rare cancer reimbursement process. Pan Pantziarka will provide a patient and not-for-profit perspective, highlighting the need for patient access to new oncology drugs. Ivana Cattaneo will present a pharmaceutical industry perspective and the importance of orphan drug incentives in rare cancer drug development. Discussion time will be included and audience participation encouraged throughout.

OVERVIEW:

Advances in precision medicine have enabled increasing use of biomarkers to define cancer subpopulations. These can be small (22% of EU cancer cases are classified as ‘rare’, i.e. have an incidence of <6/100,000 persons per year) and therefore may qualify for orphan designation from the EMA. As the industry has been historically reluctant to invest time and money into rare disease treatments due to lack of economic profitability, the EU ROMP was instated, allowing orphan drugs to receive incentives (e.g. ten years of market exclusivity and regulatory fee waivers). However, there is ongoing debate as to the definition of 'true orphan' therapies and the most appropriate approach to take as the oncology field moves towards a collection of related rare diseases.

Economic Outcomes Research Issues

8:45 - 9:45
Room: Hall 2

IP10: DOES VALUATION OF INNOVATION IN APPRAISAL OF NEW TECHNOLOGIES PROVIDE APPROPRIATE INCENTIVES FOR MANUFACTURERS AND ACCESS FOR PATIENTS?

Moderator:

Mark Sculpher, PhD, Professor of Health Economics, Centre for Health Economics, University of York, York, UK

Panelists:

Karl Claxton, PhD, Professor of Economics, Centre for Health Economics and Department of Economics, University of York, York, UK

Ross Maclean, MD, SVP, Head of Medical, Precision Health Economics, Los Angeles, CA, USA

Jens Grueger, PhD, Head Global Pricing & Market Access, F. Hoffmann-LaRoche, Division Pharma, Basel, Switzerland

ISSUE:

How can health systems with different levels of funding determine a new technology’s value whilst taking into consideration its affordability and the needs of all patients? How do manufacturers plan their research and development given international variation in available resources, value assessment, systems’ ability to achieve agreed volumes and international reference pricing? Sculpher will moderate and set out the challenges faced by health systems and manufacturers and the range of solutions advocated for valuing innovation. Claxton will propose the use of an evidence-based assessment of the benefits offered by technologies against relevant opportunity costs, and the use of rebate schemes to reflect the differences between international list prices and affordable value-based prices. Goldman will provide a critique of conventional methods of HTA and their failure to reflect the full value of innovations. Grueger will set out an industry perspective and the need for predictable signals of value.

OVERVIEW:

Health care systems internationally face the common problem of determining which new medical technologies should be funded from available resources. All decision-making bodies seek to balance the needs of potential recipients of new innovations with those of others who could benefit from different interventions delivered from the same funding. Many attempt to use HTA as a means of securing appropriate evidence to inform decisions and to reflect these considerations. What types of evidence are needed to support these decisions, and what is missing from standard HTA? To inform development decisions, technology manufacturers need clear signals regarding how health systems value new innovations and the volumes they will purchase over time. How can those signals be revealed given international variation in health system funding, different approaches to value assessment and the challenges faced in setting different prices for each jurisdiction?

14:00 - 15:00
BREAKOUT SESSION

14:00 - 15:00
Room: Hall 2

IP11: DETERMINING VALUE IN HEALTH TECHNOLOGY ASSESSMENT CONSISTENT WITH SOCIETAL AIMS: PURSUE NEW OPTIONS?

Moderator:

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

Panelists:

Alistair McGuire, BA, MLitt, PhD, Professor, Health Economics at the LSE Health and Social Care, Social Policy, London School of Economics and Political Science, London, UK

John Brazier, PhD, MSc, Professor of Health Economics and Dean, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

Michael Schlander, MD, PhD, MBA, Professor of Health Economics at the University of Heidelberg, Head of the Division of Health Economics at the German Cancer Research Center, and Foundation Chairman & Scientific Director, Institute for Innovation & Valuation in Health Care (InnoVal-HC), Wiesbaden, Germany

ISSUE:

To debate alternatives to the common cost per QALY threshold approach to determining whether a new intervention should be funded.

OVERVIEW:

In most countries, the majority of physicians and patients do not have unfettered access to all the interventions that can help diagnose, treat or prevent illness. Even if these have been shown to be effective and reasonably safe, their costs may be judged too high to allow unrestricted use. When appraising the balance between benefits and costs, a common, though not universal, tactic is to compute the ratio of net costs to net health effects and then weigh this ratio against a “threshold”. In the face of growing concerns that this method does not sufficiently capture what society wants, new approaches are urgently needed. This panel will address this challenging topic from widely divergent perspectives with a view to fostering discussion of the challenges and potential alternatives.

Health Policy Development Using Outcomes Research Issues

14:00 - 15:00
Room: Hall 3

IP12: WHAT SHOULD PATIENT-CENTERED CARE MEAN IN THE WELFARE STATES OF EUROPE? THE UNITED KINGDOM AND GERMANY CANNOT BOTH BE RIGHT

Moderator:

F. Reed Johnson, PhD, Professor, Duke Clinical Research Institute, Durham, NC, USA

Panelists:

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

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

ISSUE:

European reimbursement authorities are faced with evaluating difficult tradeoffs between efficiency and equity in provision of health care, given realistic constraints on physical, institutional, and financial resources. Such tradeoffs involve basic value judgments that could vary across national health systems. Inevitably, benefits must be weighed against costs to determine access to new health technologies. UK’s National Institute for Health and Care Excellence (NICE) traditionally has used quality-adjusted life years (QALYs) as the standard metric for quantifying the value to society of health outcomes. In contrast, Germany’s Institute for Efficiency and Quality in Health Care (IQWiG) has rejected QALYs in favor of alternative evidence-based efficiency measures, including direct assessments of patient-relevant endpoints. Thus the NICE approach has focused primarily on societal values, while IQWiG has explored using disease-specific patient preferences to inform decision making. Increased interest in patient-centric decision making by the European Medicines Agency raises questions about the role and importance of patient perspectives in regulatory evaluations. This issues panel evaluates how patient-centric concerns should relate to patient access to new treatments.

OVERVIEW:

Dr. Johnson will serve as moderator. He will summarize recent developments in patient-centered valuations of health-technology benefits and harms and proposals for how such values could be used to inform health-care decisions. The panelists will debate the relevance of patient-centered care from the different perspectives of decision-making standards and priorities in the UK and Germany. Dr. Devlin will discuss the limited relevance of disease-specific patient population preferences for the efficient and equitable allocation of overall societal resources, which ultimately is in the interest of all present and future patients. Dr. Mühlbacher will discuss the limitations of societal preferences and how quantitative measures of patient preferences more accurately indicate the effect of health-care expenditures on both societal welfare and equitable treatment of patients across therapeutic areas.

15:30 - 16:30
BREAKOUT SESSION

15:30 - 16:30
Room: Clyde Auditorium (Armadillo)

IP13: MCDA VALUATION AND ICERS – CAN THEY ALIGN FOR DECISION-MAKING PURPOSES? (INVITED ISSUE PANEL)

Moderator:

Louis P. Garrison, PhD, Professor, Pharmaceutical Outcomes Research & Policy Program, School of Pharmacy University of Washington, Seattle, WA, USA

Panelists:

Charles E. Phelps, PhD, MBA, University Professor & Provost Emeritus, Office of the Provost, University of Rochester, Gualala, CA, USA

Maarten Ijzerman, PhD, MSc, Professor & Vice-Dean, Department of Health Technology & Services Research, University of Twente, Enschede, The Netherlands

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

ISSUE:

Which of several proposed approaches to comparing costs and benefits in the context of multi-criteria decision analysis (MCDA) models have the most promise? One uses direct inclusion of cost as a “negative” attribute with its own weight. A second involves finding other healthcare interventions (with known costs) that could be eliminated and assessing the multi-criteria value of those as a benchmark. The third approach divides the multi-criteria scores of each option by its cost (akin to using the ratio of cost per health benefit). A fourth available alternative assumes an exogenous investment budget, without suggesting how to set its level. A very recently proposed approach involves using the QALY as a component in MCDA and using it as a numeraire to align MCDA-based decision thresholds with ICER thresholds. The panelists will discuss and debate the merits of these various approaches.

OVERVIEW:

MCDA models have received increasing attention of late as an alternative or complement to cost-effectiveness analysis for health technology assessment and decision-making, and have been the subject of two recent ISPOR Task Force reports. However, a key limitation of multi-criteria models is the lack of consensus on the best method to balance costs and benefits because benefit measures in these models purposefully include a wider array of attributes than do standard narrow measures such as quality-adjusted life-years (QALYs). Several approaches to these issues have been discussed, some in the Task Force reports and some elsewhere (we could elaborate further). Each has its strengths and limitations and none of them have been agreed upon as the best way to manage resource allocation using MCDA.

Patient-Reported Outcomes & Patient Preference Research Issues

15:30 - 16:30
Room: Hall 3

IP14: PATIENT-CENTERED DECISION MAKING: HOW DO YOU GENERATE RELEVANT PATIENT-REPORTED OUTCOMES EVIDENCE FOR CHRONIC DISEASE MANAGEMENT AND MARKET-ACCESS DECISION MAKING?

Moderator:

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

Panelists:

Katharine Barnard, PhD CPsychol AFBPsS, Visiting Professor, Bournemouth University, Poole, UK

Simon O'Neill, REG.N., Director of Health Intelligence and Professional Liaison, Diabetes UK, London, UK

Francois Meyer, PhD, Advisor to the President, International Affairs, Haute Autorité de Santé (HAS), Saint-Denis, France

ISSUE:

Clinical relevance, budget impact and cost-effectiveness are important criteria in market access decisions. To what extent do these criteria incorporate and reflect the perspective of the patient? Do Patient Reported Outcomes (PRO) contribute to decision making? Currently, PRO are often poorly reported secondary outcomes in clinical trials and a wide range of different data exist of varying levels of quality. This makes synthesizing them difficult. Furthermore, is the often-used EQ-5D specific enough to capture crucial patient aspects as a basis for cost-utility analyses? So, the key issue is how to generate PRO data that can be appropriately incorporated when informing decision-making and contribute to improved outcomes for patients.

OVERVIEW:

Using PROs in healthcare decisions is a topic of considerable controversy. However, the basis for decisions can be improved by increased clarity on what kind of PRO data may impact market access decisions. The panel focuses on practical aspects of generating and synthesizing PROs, using diabetes care as an example, in the context of market access decision-making.

16:45 - 17:45
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

16:45 - 17:45
Room: Hall 1

IP15: A MODEST PROPOSAL: CAN WE EASE THE BURDEN OF MYRIAD MEASURES WITH A MINIMUM DATA SET THAT ACTUALLY MATTERS TO PATIENTS?

Moderator:

Roman Casciano, MS, Executive Vice President & General Manager, Consulting & Analytics & Global Head, Value & Access, ANALYTICA LASER, New York, NY, USA

Panelists:

Sheela Upadhyaya, MSc, Associate Director - Highly Specialised Technologies, NICE - National Institute for Health and Care Excellence, London, UK

Charles D. Petrie, PhD, Vice President, Outcomes and Evidence, Global Health & Value, Pfizer, Inc., Groton, CT, USA

Bettina Ryll, MD, PhD, Founder, and Researcher, Melanoma Patient Network Europe, and Uppsala University (Department EBC), Uppsala, Sweden

ISSUE:

In the recent years, there has been a common effort to identify and measure outcomes that are be meaningful to patient, regulators, payers and industry. With stakeholders placing different value on the same outcome, it may be challenging to reach consensus and standardization. This multi-stakeholder panel will explore if and how can patient, regulators, payers and industry agree on and implement a universal standard set of outcomes by medical condition?

OVERVIEW:

In a recent NEJM paper, Porter et al highlight that despite the world shifting towards valuing outcomes and performance, measurement of outcomes that matter to patients remains limited. Acknowledging the "patchwork of inconsistent outcomes measures and definitions," authors call upon stakeholders to agree on a minimum set of outcomes for each important medical condition. This multi-stakeholder panel will discuss if and how patient-advocacy groups, industry and payers and regulators can work towards achieving standardization and the opportunities and challenges it raises. Charles Petrie will discuss, from the industry perspective, the feasibility and circumstances that may motivate consistency within and across stakeholders on standard sets of core outcomes by medical condition and how these along with clinician-reported outcomes and administrative data can better support value demonstration. Sheela Upadhyaya will discuss the value of standardization for value appraisal at the HTA level, and how HTA and payers can contribute to these discussion. Bettina Ryll will describe patients’ contributions to the identification, validation and measurement of PRO and what it would take to ensure that outcomes that are meaningful to patients are integrated in decision making processes. After 15 min presentation contrasting the perspectives, panellists will discuss what it would take to accelerate the development and testing of such PROs. Discussions will be followed by 15 minutes of Q&A from the audience.

Reference: Standardizing Patient Outcomes Measurement. Porter M et al. NEJM 2016;374:504-506.


16:45 - 17:45
Room: Hall 3

IP16: MANAGED ENTRY SCHEMES FOR MEDICAL DEVICES: GREAT OPPORTUNITY OR MAJOR CHALLENGE?

Moderator:

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

Panelists:

Aleksandra Torbica, PhD, Professor, Bocconi University, Milano, Italy

Richard Charter, MSc, Head of Market Access & Pricing, Becton Dickinson, Allschwil, Switzerland

Denis Gizzi, BSc, Managing Director, NHS Oldham Clinical Commissioning Group, Sheffield Hallam University, Oldham, UK

Brian Mangan, FCIPS MSc, Deputy Director, NHS NW Procurement Development, Wigan, UK

ISSUE:

Funding decisions are often difficult because of numerous uncertainties around the efficacy, effectiveness, safety or cost-effectiveness of new technologies. In this context, several countries have implemented novel market access schemes, with the aim of allowing market access, closely managed through agreements set up with health technology companies. These policies are labelled as “managed entry agreements” (MEA) and they include all types of “arrangements between a manufacturer and payer/provider that enable coverage or reimbursement of a health technology subject to specific conditions’’. These schemes have been developed mainly for pharmaceuticals to date, but there is a growing interest in expanding the scope of MEAs to medical devices, given the uncertainties concerning their effectiveness and use at the time of market approval. However, do the challenges of agreeing and operating these schemes outweigh the opportunities they provide?

OVERVIEW:

MEAs are generally regarded as an attractive policy option since they facilitate market access for new technologies, to the benefit of patients and technology manufacturers, while limiting the financial risk to the payer. These schemes also enable us to gather a stronger evidence base on the costs and outcomes of new health technologies than would otherwise be possible. Although MEA schemes are widely regarded as a promising policy, they require critical decisions to be made concerning the technologies to which they should be applied, the nature of the study to be undertaken, the funding of the research and the ways in which research findings should influence the final reimbursement and coverage determination. This suggests that manufacturers and other stakeholders need more advice on the design and conduct of these schemes before widely applying them on medical devices. This issue panel explores the perspectives of different stakeholders (manufacturers, policy makers and researchers) on the pros and cons of these schemes.

Wednesday, 8 November 2017
8:45 - 9:45
BREAKOUT SESSION
Patient-Reported Outcomes & Patient Preference Research Issues

8:45 - 9:45
Room: Hall 3

IP17: DERIVING UTILITY MEASURES FROM DISEASE-SPECIFIC QUALITY OF LIFE INSTRUMENTS: DOES MAPPING TO GENERIC UTILITY INSTRUMENTS ADEQUATELY CAPTURE THE PATIENT PERSPECTIVE IN ECONOMIC EVALUATION?

Moderator:

Samuel Aballéa, PhD, Vice President, HEOR, Creativ-Ceutical, Paris, France

Panelists:

Linda Abetz-Webb, MA, Senior Research Director, Patient-Centred Outcomes Assessments, Bollington, UK

Keith Tolley, Director, Tolley Health Economics Ltd., Buxton, UK

Clément François, PhD, Vice President, Health Economics and Outcomes Research, Lundbeck, Deerfield, IL, USA

ISSUE:

There is much discussion about including the patient's perspective in HTA submissions, but do patients' voices get incorporated into cost-effectiveness analysis (CEA) when HTAs increasingly rely on utility measure obtained through mapping from disease specific instruments (DSI) to generic instruments? The panel will debate on the methods used to derive utility measures from DSI, to ensure the patient perspective is included in CEA. Linda will discuss how current generic utility instruments are not capturing the concepts that are relevant to patients and will argue for the development of new disease-specific utility instruments. Keith, an SMC reviewer, will discuss the importance of having a common conceptual framework for utility assessments across diseases in order to make fair comparisons and aid decision making. Clément will address the industry perspective, taking into consideration the costs of developing new utility instruments and making decisions based on an incomplete representation of patient preferences. The panel will include short talks followed by lively debate with the audience.

OVERVIEW:

Generic measures, such as EQ-5D, are known to have limitations (e.g. lack of sensitivity), but are still the preferred method of HTA bodies, such as NICE, to assess utility for use in economic evaluations. The use of mapping functions to obtain utility values adds another layer of uncertainty: decisions are being made using a proxy of a proxy utility measure. In addition, the methods to elicit utilities have evolved since commonly used value sets for generic instruments were developed (e.g. Discrete Choice Experiment - Time Tradeoff). Thus, can we better account for patient preferences in HTA submissions through increased investment in developing utility indices for DSI instead of mapping DSI to the EQ-5D? The influence of investigators and resulting lack of comparability between utility measures for different diseases may be mitigated with the use of rigorous methodological frameworks.

Health Policy Development Using Outcomes Research Issues

8:45 - 9:45
Room: Hall 2

IP18: HOW WILL HEALTH CARE SYSTEMS HANDLE FUTURE ONCOLOGY COMBINATION PRODUCT LAUNCHES?

Moderator:

Bettina Ryll, MD, PhD, Founder, and Researcher, Melanoma Patient Network Europe, and Uppsala University (Department EBC), Uppsala, Sweden

Panelists:

Martina Weiss-Radtke, MBA, Head Negotiation/Reimbursement Pharmaceuticals, Helsana Versicherungen AG, Zurich, Switzerland

Jens Grueger, PhD, Vice President & Head of Global Pricing & Market Access, F. Hoffmann-LaRoche, Division Pharma, Basel, Switzerland

Meindert Boysen, PharmD, MSc, Programme Director Technology Appraisals, National Institute for Health and Care Excellence, Manchester, UK

ISSUE:

Over the last decade, the understanding of disease biology and care, in particular in the oncology field, has greatly increased. It has enabled the development of targeted therapies as well as cancer immunotherapies that stimulate the human body´s immune system to fight cancer. With this increased scientific understanding, researchers discover over time that the same molecular aberrations may not only occur in one, but in several cancer indications, thus suggesting clinical benefits of therapies in multiple indications with the same aberration. This suggests that combining multiple targeted therapies often also in combination with cancer immunotherapies, either simultaneously or sequentially, would provide clinical benefits to patients in fighting cancer.

In the current environment, increased use of medicines in multiple indications and combinations poses an access challenge. Current pricing and reimbursement systems in Europe usually allow for only one price for one molecule across all its indications. In case of combinations in most healthcare systems the price reflects the sum of the monotherapy price regardless of the clinical value delivered. Moreover when products from multiple manufacturers are combined, current pricing and reimbursement systems hamper the ability to negotiate on the basis of the clinical profile of the combination. As a consequence, patient access to innovative combination therapies may be delayed or not granted at all.

OVERVIEW:

Introduction by moderator: Patient view and needs with regards to the scientific and clinical evolution and the resulting evolving treatment paradigm.

Presentation 1: Swiss experiences of methods that allow combination pricing Switzerland

Presentation 2 (Jens Grüger): Industry proposals for sustainable access for cross-company combinations

Presentation 3 (tbc): Building on previous presentations, reflections from a country currently reflecting on solutions to implement


8:45 - 9:45
Room: Lomond Auditorium (Loch Suite)

IP19: CAN THE PATIENT VOICE BE BETTER INCORPORATED INTO THE NICE PROCESS?

Moderator:

Eric Low, NA, Former CEO of Myeloma UK, N/A, East Lothian, UK

Panelists:

Jennifer Lee, MBA, HEMAR & Advocacy Director, Health Economics, Market Access, Reimbursement, & Advocacy, Janssen UK, High Wycombe, UK

Heidi Livingstone, BA, Project Manager, Patient and Public Involvement Manager, NICE, London, UK

Richard Jackson, PGCE, NPQH, Patient Expert (Currently a Primary School Headteacher), NA (Hillview Primary School), NA, UK

ISSUE:

Where is patient input into the NICE Health Technology Assessment (HTA) process most valuable? Patients can provide a perspective on their condition and their experience of treatments, but should they have a role in the decision making process? Can more be done to engage them in the process more effectively?

NICE, Janssen and a patient expert will share their views as to where the patient voice can play a role in the HTA process.

OVERVIEW:

Filling In The Gaps?

Where are patient insights in the HTA process most valuable? NICE has an increasing number of medicines to assess, often with less data following accelerated regulatory approval. Does this offer an opportunity for patient advocacy groups (PAGs) to fill in some important blanks? Where does patient preference for different attributes of new treatments fit in?

Capturing Value?

Can the benefits and risks associated with every condition and new treatment be captured and compared by the QALY framework and the EQ-5D measurement? NICE must have some standardisation to be able to do the job, but standardised measures have limitations. Is this where patient input could make a difference?

Part Of The Decision?

Ultimately, patients want to feel their voice has been heard and this can be no more apparent than when they have a say in the final decision. Most HTA bodies around the globe have yet to formalise how patients have a real say in decisions. Does the recent NICE consultation on public and patient input offer an opportunity to increase patient engagement in the NICE process?

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

10:00 - 11:00
Room: Hall 3

IP20: SHOULD ICER BE NICE (OR NOT)? HOW ICER’S NEW COST-EFFECTIVENESS FRAMEWORK COMPARES WITH NICE’S GUIDELINES

Moderator:

Matthew Sussman, MA, Associate Managing Director/Director, Modeling & Evidence, Boston Health Economics, Waltham, MA, USA

Panelists:

Dan Ollendorf, PhD, Chief Scientific Officer, Institute for Clinical and Economic Review, Boston, MA, USA

Pall Jonsson, PhD, Associate Director, Research and Development, National Institute for Health and Care Excellence (NICE), Manchester, UK

ISSUE:

As health economic practitioners around the globe seek assistance in conducting cost-effectiveness analyses (CEAs), they may turn to the guiding principles made publicly available by the Institute for Clinical and Economic Review (ICER) in the United States and by the National Institute for Health and Care Excellence (NICE) in the United Kingdom. Should global health economists be armed with a unified direction for designing, implementing, and reporting CEAs that has no borders? How do the two sets of principles compare? Among the differences, is there a rationale for regional variations (e.g., due to distinctions in health systems, culture, sociodemographics of the population) and should the differences be reconciled? Panelists will consider the similarities and differences between the two frameworks (Sussman), the rationale for differences from the US perspective (Ollendorf), and the rationale for differences from the UK perspective (Jonsson).

OVERVIEW:

CEAs are a universal technique designed to assess health outcomes and costs of interventions, and ultimately aid in resource allocation decisions. In the United States, CEAs are beginning to emerge from closed health economic circles to the national landscape, due in part to ICER’s attempt to advance the role of CEAs in health policy and insurer coverage decisions. In contrast, in the United Kingdom, CEAs continue to play a vital role in informing treatment adoption, with NICE setting the standard for how national health systems should employ CEAs. In both regional settings, the respective organizations have disseminated their conceptual approach for conducting CEAs for public consumption. The panel will highlight key health economic modeling practices behind ICER’s framework and NICE’s guidelines as well as the similarities and differences between them. Panelists will present their perspectives and debate the reasons for regional differences between the two sets of principles, followed by dialogue with the audience.

Use of Real World Data Issues

10:00 - 11:00
Room: Hall 2

IP21: ADAPTIVE PATHWAYS FOR TRANSFORMATIVE MEDICINAL PRODUCTS: A NEW PARADIGM WITH THE ENHANCED APPLICATION OF REAL-WORLD EVIDENCE?

Moderator:

David Schwicker, MA, Principal, ORPHA Strategy Consulting, Basle, Switzerland

Panelists:

Nicola Bedlington, Secretary General, European Patients’ Forum and Co-Founder, The Patient Access Partnership PACT, Brussels, Belgium

Ad Schuurman, MA, Head of the International Department, National Health Care Institute (ZIN), AH, The Netherlands

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

ISSUE:

The panel will debate, with diverse perspectives, (1) how the spectrum of stakeholders can jointly improve the process of demonstrating benefit/risk and value in the challenging environment of adaptive pathways with greater uncertainty, (2) the role of RWE to support conditional authorisation and market access, and which areas still require further development, (3) the current limitations to implementing value-based MEAs in Europe and potential ways forward, (4) the role of the patient community to enhance early access initiatives as mediators and decision-makers on national and European levels. To allow for a maximum of debate, the panelists will each give a 5-minute introduction, followed by a 30-minute panel discussion and 15 minutes of Q&A.

OVERVIEW:

Patients are often willing to accept greater risks from treatment of life-threatening diseases in return for earlier access. Their rising influence puts focus on rapid development and faster access to potentially transformative medicines. Better defined, smaller patient populations and the need for speed challenge established development pathways and regulatory, HTA and pricing systems. In response, paradigms of biomedical innovation are being transformed with “Adaptive pathways”. In order to achieve equitable and timely access for patients with unmet needs, market access and reimbursement in step with earlier conditional marketing authorisation is essential. However, uncertainty about whether the anticipated clinical benefits will be verified and the possibility of undiscovered risks pose challenges to HTA bodies. With greater uncertainty, a novel medicine’s price should no longer be determined at first market entry, but be governed by more flexible approaches that reflect the emerging evidence generated. Patient-centered RWE presents a significant opportunity to enhance early access regulatory decision-making and adaptive value-based contracting centred on mutually agreed outcomes and set milestones. Nonetheless, opinions on RWE remain diverse. Questions raised by stakeholders concern the methodology, reliability and the usefulness of RWE in decision-making.

Health Policy Development Using Outcomes Research Issues

10:00 - 11:00
Room: Lomond Auditorium (Loch Suite)

IP22: HOW CAN WE ASSESS THE VALUE OF NEW ANTIBIOTICS?

Moderator:

Chris Henshall, PhD, Independent Consultant and Visiting Fellow, Office of Health Economics, London, UK, UK

Panelists:

Alec Morton, PhD, Professor of Management Science, Department of Management Science, Strathclyde Business School, University of Strathclyde, Glasgow, Scotland, UK

Douglas Lundin, PhD, Chief Economist, TLV, Stockholm, Sweden

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

ISSUE:

The world faces a growing threat from antibiotic resistance. There is an urgent need for new antibiotics and for improved stewardship. Following international initiatives to boost R&D efforts, new antibiotics are beginning to come to market. HTA and payer bodies need to be able to assess the value that these new antibiotics offer so that prices and payment mechanisms can be agreed that provide appropriate access for patients and appropriate returns on investment for developers. Assessing the value of new antibiotics presents a number of challenges. The design of clinical trials and the emergence of accelerated regulatory pathways mean that new antibiotics are likely to come to market with evidence of effectiveness that does not fit the pattern that HTA and payer bodies typically expect. Furthermore, conventional HTA and payer approaches are not well suited to assessing the wider value of new antibiotics to the population and society in general (for example by helping to contain the further spread of resistant pathogens, and by enabling other medical procedures), and conventional approaches to paying for drugs are not well suited to rewarding compounds whose value may lie partly in their use being restricted to improve stewardship.

OVERVIEW:

This session will explore the challenges that new antibiotics present, and set out recent thinking on possible ways forward. Adrian Towse will present work from the Office of Health Economics on developing a value framework for antibiotics, building on a recent Discussion Forum in London with key stakeholders. Alec Morton will provide an update on the findings of the DRIVE-AB project funded by the European Commission and EFPIA, which aims to provide an approach for the assessment of the value of an antibiotic. Douglas Lundin will reflect on the challenges for payers, health systems and governments and the ways that these can be addressed.

14:15 - 15:15
BREAKOUT SESSION

14:15 - 15:15
Room: Hall 3

IP23: HOW DO YOU INCORPORATE THE PATIENT PERSPECTIVE INTO HEALTH TECHNOLOGY ASSESSMENTS? DEFINING BEST PRACTICES FOR RIGOROUS SCIENTIFIC EVIDENCE AND IMPACTFUL SUBMISSIONS AND REVIEWS

Moderator:

Judith Leah Rubinstein, MIA, Director of Regulatory Data, Context Matters Inc., New York, NY, USA

Panelists:

Paola Kruger, MA, Patient Expert-EUPATI Fellow, Accademia dei Pazienti/EUPATI Italia, Rome, Italy

Ken Bond, MA, Director, Patient Engagement, Ethics, and International Affairs, CADTH, Ottawa, ON, Canada

Laure Delbecque, PhD, Associate Director, Pharmerit International, Rotterdam, The Netherlands

ISSUE:

HTA bodies have increasingly focused on patients as key stakeholders and partakers in the HTA review process. However, there are no agreed-upon or standard methods to obtain patients’ perspectives. As a result, the provided evidence can remain anecdotal, not meaningful or difficult to interpret. The panel will debate the quality and value of patient testimonials as evidence and input compared to other sources of information included in HTA submissions and during the review process. The panel reflects the diversity of perspectives on the topic. Ken Bond, adopting an HTA’s perspective, will describe the work being done by CDR and pCODR in Canada, stakeholder perceptions, and the impact of current methods on HTA decisions. Paola Kruger, adopting a patient/ advocacy group perspective, will contend that a structured approach to patient engagement can reinforce binding commitments from all stakeholders. Laure Delbecque, adopting a researcher’s perspective, will describe scientific methods to reinforce the patient’s voice and create more parity with other evidence submitted to, and/or reviewed by, HTAs. This will be followed by a panel discussion and Q&A from the audience.

OVERVIEW:

There is growing consensus that patient engagement is an essential part of the drug discovery process to ensure that patient views and needs are taken into account from initial drug development to HTA submission and review. However, the patient engagement in HTAs, particularly during the submission and review process, varies between countries and is minimally regulated. Moreover, few methods have been established to obtain/provide patient evidence in HTA submissions and/or review. As a result, the format and content of patient-centric evidence during the HTA review process is ill-defined and has yet to be considered equivalent with other evidence that is traditionally presented to HTA decision-makers. Rigorous methods are proposed to address these concerns.

Economic Outcomes Research Issues

14:15 - 15:15
Room: Hall 2

IP24: TRUSTING THE RESULTS OF MODEL-BASED ECONOMIC ANALYSES: IS THERE A PRAGMATIC VALIDATION SOLUTION?

Moderator:

J. Jaime Caro, MDCM, FRCPC, FACP, Adjunct Professor of Medicine, Epidemiology and Biostatistics, McGill University, Montreal, QC, Canada

Panelists:

Matt Stevenson, PhD, Professor of Health Technology Assessment, ScHARR, University of Sheffield, Sheffield, UK

Jorgen Moller, MSc, Mech Eng, Vice President of Modeling Technologies, Modelling Technologies and Simulation, Evidera, Hammersmith, UK

Salah Ghabri, PhD, Senior Health Economist, Haute Autorité de Santé, Saint-Denis La Plaine, France

ISSUE:

This panel addresses the crucial topic of trusting the results of economic analyses based on decision analytical models and the resulting requirements for validation and transparency, areas where our field is currently very deficient. Based on their differing perspectives and experiences, the panelists will characterize the situation, pose the challenges and outline requirements for improvement and pragmatic solutions to the problem of inadequate validation.

OVERVIEW:

Health economic models have been used to extend the efficacy and safety information obtained from clinical trials to the broader populations, horizons and outcomes of interest to health technology assessors. Despite the extensive use of these decision-analytic models and repeated calls for their transparency and validation, our field continues to depend on poorly validated models, often constructed under time and other constraints duress. These highly artificial models are often complexly formulated and, thus, difficult to validate; but even if they can be assessed, their ability to predict the outcomes of interest in a reasonably accurate and unbiased way is usually unknown because it has not been tested. In part this situation is a result of the lack of sufficient data to both populate the model and validate it, but with the growing availability of automated datasets this reason is diminishing. Nevertheless, the short timelines and pressures to keep models simple and transparent impair accuracy and its usefulness.

Clinical Outcomes Research Issues

14:15 - 15:15
Room: Forth (Armadillo)

IP25: INNOVATIVE CLINICAL TRIAL DESIGNS: WELCOMED BY REGULATORS BUT WHAT ABOUT THE PAYERS?

Moderator:

Richard Macaulay, PhD, Principal Consultant, PAREXEL Access Consulting, PAREXEL International, London, UK

Panelists:

Leanne Larson, MHA, Vice President & Global Head, Observational Research, PAREXEL International, Waltham, MA, USA

Andrew Walker, PhD, Director, Salus Alba Consulting, Glasgow, UK

Detlev Parow, MD, MBA, Head of Health Care Management Development, D A K - Gesundheit, Hamburg, Germany

ISSUE:

There is an increasing trend to utilize innovative new designs for clinical trials. This panel will explore payer perspectives on the benefits and challenges posed by conducting reimbursement assessments on data coming from these trial designs. Leanne Larson will present an overview of different innovative approaches (focusing on adaptive and umbrella trials) and the opportunities that these offer for the development of new medicines. Detlev Parow and Andrew Walker will present an overview of conducting pricing and reimbursement assessments on drugs utilizing these new clinical trial designs from a German and United Kingdom reimbursement perspective, respectively. Each panelist will speak for 10 minutes and this will be followed by a 15-minute panel discussion, and 15 minutes of Q&A from the audience.

OVERVIEW:

There is a well-recognized long-standing decline in R&D productivity in the pharmaceutical industry which has driven exploration of new approaches to the clinical development of pharmaceuticals. These include innovative randomized controlled trial designs, including adaptive, umbrella, basket, hybrid and pragmatic studies. Adaptive trials, perhaps the best established of these, have already been the subject of guidance documents by both the EMA and FDA. However, the reimbursement hurdle is becoming increasingly difficult for companies to clear and thus the perspective of payers is key. Payers may welcome such innovative approaches if they enable the development of new treatments for diseases of high unmet needs, alongside the prospect of better defined biomarkers and more relevant comparators. However, there may be payer concerns in terms of their acceptability due to the potential for perceived bias alongside their complex Bayesian statistics that may be difficult to independently verify.

15:30 - 16:30
BREAKOUT SESSION
Health Policy Development Using Outcomes Research Issues

15:30 - 16:30
Room: Forth (Armadillo)

IP26: MHEALTH AMONG CLINICIANS AND PATIENTS IN CANCER CARE: HOW TO ADDRESS THE DIGITAL DIVIDE?

Moderator:

Rosanna Tarricone, PhD, Associate Dean, Government & Health Division, SDA Bocconi, Director, Centre for Research on Health and Social Care Management, Associate Professor, Department of Policy Analysis and Public Management, Bocconi University, Milan, Italy

Panelists:

Maria Cucciniello, PhD, Assistant Professor, Centre for Research on Health and Social Care Management (CERGAS) and Department of Policy Analysis and Public Management, Bocconi University, Milan, Italy

Roma Maguire, PhD, MSc, Professor, School of Health Sciences Faculty of Health & Medical Sciences, University of Surrey, Glasgow, UK

Nicola Scalzo, MSc, Policy and Legal Officer, DG for Internal Market, Industry, Entrepreneurship and SMEs Health Technology and Cosmetics Unit GROW D.4, European Commission, Brussels, Belgium

ISSUE:

mHealth is booming in cancer care, but its potential is yet to be fully exploited. The panel will debate the current mHealth utilization in cancer care, its potential and the barriers that currently prevent a more thorough diffusion. Maria Cucciniello, moving from the results of an international e-survey on the use of mhealth, examines the determinants that help explain different mHealth uptake rates among patients and clinicians, and outlines which types of levers can be activated to facilitate its diffusion. Roma Maguire provides an overview of the mHealth potential in empowering patients and facilitating symptom management, providing a critical perspective on the challenges in the patient-clinician relationship. Salvatore Scalzo provides a regulatory perspective, illustrating the current state-of-the-art of the EU regulation applicable to mhealth APPs and how the future of these technologies might look like as a result of the newly adopted EU regulation on medical devices

OVERVIEW:

mHealth, the use of mobile technologies for the delivery of healthcare, has been addressed to as the biggest technology breakthrough of our time. Although its features can be suitably applied to several conditions, mHealth contribution to cancer care is still up for debate. mHealth has been proposed as a means to enhance patient-empowerment and improve the organizational performance, ultimately leading to better clinical outcomes. However, current mHealth diffusion in cancer care is unknown and several challenges still need to be taken on, including: how to facilitate patient-clinician relationship rather than making it more burdensome, how to identify and promote a regulatory model which is fit for such new technologies, how to provide clinicians with the right financial and organizational incentives; and how to generate evidence promptly to keep up with rapid changes in technology.


15:30 - 16:30
Room: Hall 2

IP27: VALUING CHILDREN'S HRQoL FOR HEALTH TECHNOLOGY ASSESSMENT: WHAT ARE THE ISSUES AND CHALLENGES?

Moderator:

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

Panelists:

Oliver Rivero-Arias, PhD, MSc, Senior Health Economist, National Perinatal Epidemiology Unit, University of Oxford, Oxford, UK

Donna Rowen, PhD, Senior Research Fellow, School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UK

Deborah Morrison, MSc, BSc, Senior Scientific Adviser, NICE Scientific Advice, Centre for Health Technology Evaluation, NICE, Manchester, UK

ISSUE:

How should we value HRQoL of children?

OVERVIEW:

The estimation of QALYs for use in HTA relies on utilities for health related quality of life (HRQoL). The methods for eliciting utilities for health states experienced by adults are well established. However, the valuation of children’s health states poses additional challenges, and raises a number of complex methodological and normative issues. For example, while by convention stated preferences are usually elicited from the (adult) general public, the valuation of children’s health states raises the question of what perspective adults should adopt in imagining and valuing children’s HRQoL – and what difference this might make to the values. Might children’s preferences differ from those of adults – if so, should that be considered in HTA? We observe that adults are less willing to trade off life years for children – leading to higher TTO values for children'shealth states. What are the implications of that for HTA? What are the implications for the choice of methods to use in eliciting preferences for children’s HRQoL? If we use different methods to value children and adult HRQoL, does that cause a consistency issue for cost effectiveness models that include both childhood and adulthood states? Addressing these issues is critical to ensuring a robust and defensible basis for HTA of technologies addressing childhood illness.

In this issues panel, we begin by outlining the empirical and normative issues associated with valuing children’s HRQoL. We summarise the evidence available on each issue, with particular reference (but not limited to) stated preference studies for the EQ-5D-Y and CHU-9D. Gaps in methods and evidence are highlighted. We consider current guidance and practice regarding utilities for children’s HRQoL in HTA, and consider what early advice should be given to companies asking about the measurement and valuation of children's HRQoL.

Patient-Reported Outcomes & Patient Preference Research Issues

15:30 - 16:30
Room: Hall 1

IP28: PATIENT PREFERENCES IN EUROPEAN DRUG REGULATION – ARE WE READY?

Moderator:

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

Panelists:

Francesco Pignatti, MD, Head of Oncology, Haematology, Diagnostics, European Medicines Agency, London, UK

Pierre Demolis, MD, PhD, Chairman Oncology Working Party of the CHMP, Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM), Saint-Denis, France

Tommi Tervonen, PhD, Research Scientist, Evidera, London, UK

ISSUE:

There is little experience with patient preference in regulatory decisions. With the increased involvement of patients in different phases of the drug development process, how should the regulators take patient preferences into account?

OVERVIEW:

Patient opinions are now more systematically being considered in regulatory decisions about benefit-risk of drugs in the EU. The panelists will present their personal, sometimes conflicting views on whether patient preferences should be taken into account in informing market authorization decisions, and discuss what the state of the play is organizationally, scientifically, and methodologically. The moderator (Postmus) will briefly introduce the work conducted on patient preference pilots within the European Medicines Agency. Pignatti will argue that although methods for systematic elicitations of preferences are being explored, many questions remain open such as the validity of different methods, generalizability of results, and usefulness in the regulatory context. Demolis will discuss how the views of patients available in a survey may reflect feelings at a given stage of disease, influenced by hopes and fears, experience of failure/success and of adverse effects, which may change over time, and therefore regulators and physicians can better take patients' perspectives into account in a global value judgement. Tervonen will argue that authorization decisions of drugs with marginally positive/negative benefit-risk profiles should be justified with quantitative analyses that incorporate patient preferences, and while collecting preference data is not a trivial task, good research practices using theoretically sound methods allow to collect such data with high precision.

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