Educational Symposia
Saturday, 4 November 2017

17:30 - 18:30
Room: Lomond Auditorium (Loch Suite)
THE EVOLVING APPROACH TO DRUG VALUE ASSESSMENT IN GLOBAL MARKETS
David Rees, Principal of Health Strategies Group, will lead a panel of industry market access marketers in a discussion of the evolving approach to drug assessment in global markets. This interactive session will address how the global access stakeholder’s approach to assessment of drug value is changing, the disparity in value assessments, and how the emerging model is different from the current mode.
Moderator: David Rees, BA, MBA
Principal, Health Strategies Group, Yardley, PA, USA
Speaker: Sanjay Shah, BS, MBA
Principal, Visionary Ventures, LLC, Morganville, NJ, USA
Speaker: Michael Schlander, MD, PhD, MBA
Professor of Economics, University of Heidelberg, Chairman and Scientific Director InnoVal-HC, Institute for Innovation & Valuation in Health Care, Wiesbaden, Germany
Speaker: Andreas Wildi, MD, MLaw
Partner, Walderwyss Attorneys at Law, Zurich, Switzerland
Speaker: Jennifer Cain Birkmose, MBA, MPH, BS
Head of Market Access, Pricing and Policy, Region Europe, Roche, Basel, Switzerland
Speaker: Aris Angelis, PhD
Research Fellow, Medical Technology Research Group, London School of Economics, London, UK
Health Strategies Group
(Sponsored by Health Strategies Group)
Sunday, 5 November 2017

17:30 - 18:30
Room: Lomond Auditorium (Loch Suite)
EVALUATING SURVIVAL BENEFITS IN TECHNOLOGY APPRAISALS OF INNOVATIVE ONCOLOGY DRUGS: CHALLENGES AND PRACTICAL SOLUTIONS
Rapid innovation in hematology/oncology treatments offers new hope to patients with advanced cancers. However, long-term survival data is often not available at the time of regulatory submission. Therefore, the initial regulatory approval is often based on safety and surrogate efficacy endpoints such as response rate or relapse free/progression free survival; Health Technology Assessment (HTA) bodies usually rely on survival prediction to assess the long-term outcomes. The mechanism of innovative treatments may not fit in the traditional model of survival prediction. In addition, issues related to trial design (e.g., single arm trials, treatment crossover) impose additional challenges for payers to evaluate the long-term comparative benefits of new treatments vs. standard of care. In this symposium, attendees will be introduced to the common methodological challenges in modelling survival benefits for innovative oncology drugs. The discussions will cover the following topics: 1) different models to extrapolate survival data, including potentially modeling the effect for drugs that can "cure" the disease, 2) adjusting survival estimates in the presence of treatment switching, and 3) indirect comparison using single-arm trials or under non-proportional hazards assumptions. The presenters will discuss the novel methods to address each challenge, using recent case examples and HTA submissions.
Speaker: Keith Betts, PhD
Vice President, Health Economics and Outcomes Research, Analysis Group, Inc., Los Angeles, CA, USA
Moderator: Jipan Xie, MD, PhD
Vice President, Analysis Group, Inc., Los Angeles, CA, USA
Speaker: Nicholas Latimer, PhD
NIHR Post-Doctoral Research Fellow, Health Economics and Decision Science, ScHARR - University of Sheffield, Sheffield, UK
Speaker: Zheng-Yi Zhou, PhD
Manager, Analysis Group, Inc., London, UK
Analysis Group
(Sponsored by Analysis Group)

18:45 - 19:45
Room: Lomond Auditorium (Loch Suite)
HOW TO USE THE BEACON FRAMEWORK TO HELP GUIDE THE DRUG DEVELOPMENT PROCESS AND ACHIEVE PATIENT ACCESS
Decisions about the kind of study data to be collected are made throughout the clinical development process. Historically, these decisions have not always been made with Health Technology Assessment (HTA) requirements in mind, with often costly results. The BEACON framework was the culmination of a research collaboration between Mundipharma International and a group of payer experts. The objective of the project was to provide a framework for being aware of and therefore addressing payer’s criteria during the drug development process. As part of this, 15 sets of HTA guidelines were reviewed in depth. The manuscript was published in the peer-reviewed journal PharmacoEconomics (Dunlop et al. PharmacoEconomics (2016) 34:1051) and is freely available to access. This educational symposium will explore through presentations and an interactive session how the BEACON research project identified the key HTA categories, and distilled them into six criteria. These six criteria apply throughout the drug development process to account for data requirements, the reimbursement environment and affordability. Importantly, the BEACON framework is designed to be simple and clear, so all stakeholders can understand the payer’s needs. It will open with a presentation from Dr. Andrew Walker outlining some of the misalignments between the requirements of HTA bodies and the data the pharmaceutical industry provides. This will be followed by a presentation from Will Dunlop providing an industry perspective on data collection for HTA, and finally a presentation by Professor Ron Goeree who will explain how the BEACON framework was developed, and how it can be applied. Finally, attendees will get the chance to use the BEACON framework on some hypothetical drug profiles to learn how it can help with clinical development decisions and optimise patient access.
Moderator: Meindert Boysen, PharmD, MSc
Programme Director Technology Appraisals, National Institute for Health and Care Excellence, Manchester, UK
Speaker: William Dunlop, BSc, MSc
Head of Market Access, Mundipharma International Ltd, Cambridge, UK
Speaker: Andrew Walker, PhD
Director, Salus Alba Consulting, Glasgow, UK
Speaker: Ron Goeree, BA, MA
Professor Emeritus, Professor Emeritus, McMaster University and Goeree Consulting Ltd, Hamilton, ON, Canada
(Sponsored by Mundipharma International Ltd)
Monday, 6 November 2017

7:30 - 8:30
Room: Lomond Auditorium (Loch Suite)
REAL-WORLD EVIDENCE TO SUPPORT REGULATORY APPROVALS BASED ON TUMOR BIOMARKERS IN ONCOLOGY RESEARCH
In a historic first, the U.S. Food and Drug Administration (FDA) recently granted accelerated approval for a cancer treatment based on a common biomarker rather than histologic tumor origin. The FDA granted its first tissue/site–agnostic approval to pembrolizumab for adult and pediatric patients with unresectable or metastatic, microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed after prior treatment. The approval was based on data from 149 patients with MSI-H or dMMR solid tumors enrolled across 5 uncontrolled, multi-cohort, multi-center, single-arm clinical trials (90 patients had colorectal cancer, and 59 patients were diagnosed with 1 of 14 other types of cancer). This paradigm-changing approval is at the intersection of precision medicine and immuno-oncology and creates unique challenges for all stakeholders: What demands will be placed on health economists to analyze smaller and smaller patient populations? How will these analyses be conducted in patients with highly variable clinical scenarios whose only commonality is a shared actionable mutation? What information will patients, providers, and payers need to become comfortable in this new reality? Through this symposium, we will ask key stakeholders to weigh in on a robust discussion that will include real-world evidence (RWE) beyond claims and medical records, prospective outcomes research beyond registries, linking patient-reported outcomes to clinical outcomes, and how RWE research studies can be designed to effectively capture the required clinical biomarker data to support such approvals in the future.
Moderator: Bruce Feinberg, DO
Vice President, Chief Medical Officer, Cardinal Health Specialty Solutions, Dublin, OH, USA
Speaker: Hui Huang, PhD, MBA
Executive Director, Head of Global Outcomes Research Oncology, Takeda, Cambridge, MA, USA
Speaker: Samuel Wagner, PhD, RPh
Executive Director, Worldwide HEOR, Global Health Economics and Outcomes Research, Bristol-Myers Squibb, Washington Crossing, PA, USA
Speaker: Joshua Ray, MSc
Group Head Health Economics, F. Hoffmann-La Roche, Basel, Switzerland
Speaker: Jonathan Kish, PhD
Director, HEOR, Cardinal Health Specialty Solutions, Dublin, OH, USA
Cardinal Health
(Sponsored by Cardinal Health)

12:45 - 13:45
Room: Clyde Auditorium (Armadillo)
HTA V2.0: THE RELEVANCE OF REAL-WORLD EVIDENCE IN VALUE FRAMEWORKS AND ITS RELATION WITH REGULATORY AND HEALTH TECHNOLOGY ASSESSMENT CONVERGENCE
Innovation is enabling earlier HTA insights. Classical HTA approaches rely on translating clinical efficacy outcomes into cost-effectiveness assessments. Innovative study designs are evolving to achieve relevant insights earlier and at lower cost, facilitating measures such as conditional reimbursements, adaptive pathways and rapid access schemes, and driving stakeholder alignment. New HTA requirements demand new evidence approaches. Healthcare technologies and stakeholder needs are also evolving, with challenges for classical value assessment frameworks. Newer evidence-driven value frameworks, including Multi Criteria Decision Analysis (MCDA), require data typically not collected during clinical development. Innovative and integrated real-world evidence (RWE) approaches are crucial to address this demand in a cost-effective and timely way. An integrated RWE strategy is imperative. Join us to explore with our panel of senior level stakeholders from academia, the industry and QuintilesIMS, the evolving HTA environment, newer technological and methodological approaches for innovative evidence generation, and how to drive a more integrated RWE strategy.
Moderator: Jacco Keja, PhD
Vice President, Global Head Outcomes Research, QuintilesIMS, London, UK
Speaker: Maureen Rutten-van Mölken, PhD, MSc
Professor, Economic Evaluations of Innovative Health Care for Chronic Diseases, Erasmus School of Health Policy & Management (ESHPM), Rotterdam, The Netherlands
Speaker: Dana Vigier, MD
Vice President, Market Access France, BMS, Rueil-Malmaison, France
Speaker: Elizabeth Powers, MBA
Senior Principal, QuintilesIMS, New York, NY, USA
QuintilesIMS
(Sponsored by QuintilesIMS)
Tuesday, 7 November 2017

7:30 - 8:30
Room: Lomond Auditorium (Loch Suite)
CHANGING THE HEALTH TECHNOLOGY ASSESSMENT PARADIGM FOR COMBINATION THERAPIES
Recent advances in genomics are enabling the development of innovative and targeted therapies in a range of disease areas, particularly in oncology. Targeting antitumor immune response at multiple levels may prove more effective than monotherapy and pharmaceutical companies are increasingly seeking Health Technology Assessment (HTA) approval for innovative drugs used in combination. Due to the complexity and nature of innovative drugs (e.g. extended treatment duration due to delayed disease progression), companies face multiple challenges, including high-costs and establishing an appropriate pricing structure/strategy. The growing potential for innovative combination therapies is likely to generate further challenges within reimbursement systems. In Europe, payers are faced with increasing budgetary pressure to improve their allocation of scarce funding. Similarly in the U.S., there is a growing willingness to take action against prices deemed excessively high. This Takeda-sponsored symposium‘s key objective is to capture expert insights on how to overcome HTA challenges specific to brand-on-brand combination therapies in various disease areas, including oncology. The panel will acknowledge the variability of current HTA practices (e.g. single cost-effectiveness threshold and/or the use of multiple criteria) in assessing high-cost combination therapies (oncology and combination oncology drugs), the factors driving these differences (e.g. national reimbursement systems, decision rules), the discrepancies in HTA outcomes (e.g. approved, rejected) across countries and the implications these may have on patient access to medicines. European representatives will debate on how combination therapies are evaluated and identify the challenges experienced during the evaluation process, if any, in these respective markets. For example, in the UK, how does the use of cost-effectiveness thresholds impact on brand-on-brand combination therapies and what are the issues with this approach? The panel will discuss how best to price combination treatments, i.e. consideration of the combination cost or only the addition cost, based on each market's set negotiation methods/discounts? They will also consider whether implementing therapeutic-area specific threshold/multi-indication pricing are feasible and appropriate. The panel will also explore other potential approaches to harmonise the HTA process and discuss ways to make new therapies affordable to independent health care systems to ensure patient access to innovative drugs now and in the future. • Are current HTA frameworks used to assess brand-on-brand combination therapies suitable and do these lead to unequal patient access between jurisdictions? • What are the pricing and reimbursement challenges for high-cost brand-on-brand combination therapies? • What are the implications of current HTA decision rules for pricing? • What makes an effective pricing strategy in order to secure reimbursement of combination therapies in multiple markets? • Are current frameworks sustainable in the future? How are these expected to evolve? • Do HTA agencies need to use other approaches (e.g. therapeutic-area specific thresholds/multiple criteria) to assess combination therapies in oncology?
Moderator: Mark Ratcliffe, PhD
Chief Executive Officer, PHMR Ltd, London, UK
Speaker: Andrew Walker, PhD
Director, Salus Alba Consulting, Glasgow, UK
Speaker: Claudio Jommi, MSc
Professor, CERGAS, CERGAS Bocconi University, Milan, Italy
Speaker: Hanim Edoo, MSc
Early Engagement & Patient Access Consultant, Medi-Qualite Omega, Paris, France
Speaker: J. Jaime Caro, MDCM, FRCPC, FACP
Chief Scientist, Evidera, Bethesda, MD, USA
Takeda Pharmaceuticals International AG
(Sponsored by Takeda Pharmaceuticals International AG)

12:30 - 13:30
Room: Clyde Auditorium (Armadillo)
PRACTICAL IMPLICATIONS OF VALUE-BASED PRICING AND EMERGING VALUE FRAMEWORKS IN HEALTH TECHNOLOGY ASSESSMENT
In light of growing competition for innovative therapies in addition to rising cost of treatments, manufacturers are pressured to demonstrate product value both clinically and economically. One leading payment model that has led such efforts to align with this objective is value-based pricing (VBP), in which financial risk is shared between manufacturers and payers. VBP has emerged as a front-runner in addressing product value by associating product prices with actual patient health outcomes. However, efforts to implement the model have been limited and proven challenging. Organizations have also taken various approaches to defining drug value, including the American Society of Clinical Oncology (ASCO) Value Framework, the European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, the Institute for Clinical and Economic Review (ICER) Evidence Reports, and the Memorial Sloan Kettering Cancer Center (MSKCC) Drug Abacus. ASCO, ESMO, and MSKCC tools have focuses on the rising cost of oncology therapies, whereas the ICER reports span across multiple therapeutic areas. Due to varying methodologies in these approaches and the limited, proprietary nature of current VBP agreements, the durability of VBP remains to be determined. We will discuss the practical implications of VBP, expand on the highlights and shortcomings of these value frameworks, and explore the implications for stakeholders.
Moderator: Jay Jackson, PharmD, MPH
Senior Vice President, Consulting Services, Xcenda, Palm Harbor, FL, USA
Speaker: Andrew Briggs, DPhil
Visiting Investigator, Center for Health Policy and Outcomes, Department of Epidemiology & Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, NY, USA
Speaker: Ansgar Hebborn, PhD
Head Global Market Access Policy, Global Pricing & Market Access (GPMA), F.Hoffmann-La-Roche AG, Basel, Switzerland
Speaker: Ken O'Day, PhD, MPA
Director, Global Health Economics, Xcenda, Palm Harbor, FL, USA
Xcenda
(Sponsored by Xcenda)
Wednesday, 8 November 2017

7:30 - 8:30
Room: Lomond Auditorium (Loch Suite)
UNIVERSAL VALUE: THE WAY FORWARD?
In this interactive symposium, we will explore and discuss the evolution of VALUE from the perspective of patients, clinicians and payers. How do we reconcile universal value when it can mean different things to different stakeholders?
Speaker: Michael Blackney, PhD
Principal Consultant, Covance Market Access, London, UK
Speaker: Ruth Zeidman, PhD
Director, Covance Market Access, London, UK
Speaker: Emanuela Castelnuovo, MSc
Director, Covance Market Access, London, UK
Speaker: Michael F. Drummond, MCom, DPhil
Professor of Health Economics, Centre for Health Economics, University of York, Heslington, York, UK
Speaker: Don Redding, BA
Director of Policy, National Voices, London, UK
Covance
(Sponsored by Covance)

13:00 - 14:00
Room: Lomond Auditorium (Loch Suite)
DOES ACCELERATED APPROVAL DELIVER ACCELERATED ACCESS? EXPERIENCES, CHALLENGES AND PRIORITIES
Accelerated approval mechanisms exist to speed particular new treatments to patients who need them, but rapid regulatory approval does not guarantee rapid patient access. This symposium will explore ongoing London School of Economics (LSE) research into different early access to medicines pathways and the approaches and timelines for reviewing for reimbursement the cancer drugs that go through these pathways. Differences in approach and evidence requirements will be discussed and a specific industry example will be shared of approaches taken to try to meet HTA agency requirements where clinical trial data is incomplete. Perspectives of payers, healthcare professionals, patients and pharma will be considered and attendees will be invited to contribute to determination of challenges and priorities for ensuring sustainable solutions that provide appropriate patient access to innovative treatments.
Moderator: Victoria Tzouma, MSc
Associate Director in Health Economics and Policy, LSE Health, London School of Economics and Political Science, London, UK
Speaker: Panos Kanavos, PhD
Associate Professor in International Health Policy, LSE Health, Medical Technology Research Group, London School of Economics and Political Science, London, UK
Speaker: Chris Hoyle, PhD
Director Health Economics & Payer Analytics (Oncology), AstraZeneca, Cambridge, UK
(Sponsored by AstraZeneca)
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