Educational Symposia & HEOR Theater

* Program subject to change

Mon 7 Nov11:30 - 12:30Educational SymposiaFrom Cradle to Grave: Can We Measure and Value HRQoL Consistently Over the Lifespan?In-person & Virtual

The EuroQol Group has developed a portfolio of instruments, enabling health to be measured and valued across the entire lifespan. EQ-5D-3L and EQ-5D-5L are instruments aimed at measuring health in adults, while EQ-5D-Y-3L and EQ-5D-Y-5L are aimed at measuring health in children and adolescents. An instrument for even younger populations (0–3-year-olds) is currently in development. All these instruments are short, simple to use and preference-weighted, which means they can be used to both assess health and to calculate QALYs.

However, hurdles remain if the instruments are to be used in a coordinated fashion. Obtaining and using preference weights in pediatric populations is a relatively new area of research with its own challenges including the need to transition between value sets for different age groups. Furthermore, while using age-appropriate instruments may improve measurement within an age group it can also produce discontinuities in measurements across age groups.

This symposium aims to summarize the EuroQol instruments developed to measure HRQoL across different ages and describes the conceptual relationship between those instruments. We will discuss the empirical evidence on how the descriptions of HRQoL generated from EuroQol instruments compare and the resulting comparability of results before and after transitioning to another measure of health based on age. Furthermore, an increasing number of value sets are becoming available for the EQ-5D-Y-3L and appear to have distinctive characteristics compared to adult EQ-5D values. We discuss the implications for use of these values in QALY estimation and other applications, focusing on understanding the transition between age-specific measures.

We conclude by describing the potential advantages for users and decision-makers of a system which allows for the reliable measurement and valuation of health from infancy, childhood, adolescence and into adulthood, identifying the remaining challenges and evidence needed to achieve that goal.

SponsorEuroQol Research FoundationModerators

Elly Stolk, PhD

EuroQol Research Foundation, Rotterdam, Netherlands

Elly Stolk is Scientific Director at the EuroQol research foundation. Elly also is founding co-director of the Erasmus Choice Modelling Centre (ECMC). Her main areas of expertise are measurement and valuation of health and assessment of quality of life using patient reported outcome measures (PROMs). Her core responsibilities are managing the EuroQol research funding program, high quality project delivery, and to provide support and guidance to Office team members and other researchers. She has over 100 publications in this area.

Speakers

Nancy Devlin, PhD

University of Melbourne, Melbourne, VIC, Australia

Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. She is Chair of the Board of the EuroQol Research Foundation and served as President of ISPOR (2019-2020). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money in health care; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published >150 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.

Michael Herdman, MSc

Insight Consulting & Research, Mataró, Spain

Mike has worked in the field of health technology assessment and outcomes research for over 25 years. He currently runs his own consulting and research company in Spain and is Honorary Visiting Associate Professor at the Saw Swee Hock School of Public Health at the National University of Singapore. His main areas of interest are the development, validation, application, and interpretation of patient reported outcome (PRO) measures. He has led or collaborated on numerous national and international projects involving the use and development of PRO instruments and in recent years has focused on the measurement and valuation of health and quality of life in younger populations. He was part of the development team that produced the most widely used generic preference-weighted measure of health status, the EQ-5D-5L, and its equivalent for use in younger populations, the EQ-5D-Y. He has been a member of the EuroQol Group for 25 years, is Chair of that Group’s Younger Populations Working Group, and currently serves on its Executive Committee. He has published over 100 original peer-reviewed journal articles on a range of topics, from the use of utilities in health economic assessments in areas such as vaccines and cancer to widely-cited papers on the cross-cultural measurement of health-related quality of life.

Janine Verstraete, PhD

Department of Paediatrics and Child Health, University of Cape Town, Cape Town, South Africa

Janine Verstraete is a paediatric clinician researcher based at the University of Cape Town, South Africa. Janine is a member of the EuroQol Younger Populations Working Group. Her research interests include clinical research in paediatric pulmonology, physiotherapy and Paediatric Outcome Measurement. She has led or collaborated on numerous projects involving the EQ-5D-Y and developed the EuroQol Toddler and Infant Populations (EQ-TIPS) Health Related Quality of Life measure for children aged 0-3 years.

Quantifying Bias in Real-World Studies: A New Hope for RWD Acceptance or Are HTAers Gonna Hate?In-person & Virtual

Medicinal development for rare-disease has become increasingly reliant on single-arm trials, which poses challenges for health technology assessment (HTA). While external or synthetic control arms (ECAs or SCAs) using real-world data (RWD) are potential tools to aid decision-making for drug reimbursement by providing comparative data, there are understandable concerns with non-randomised comparisons. Statistical techniques such as quantitative bias analysis (QBA) can give decision-makers a measure of uncertainty around the effect estimates from a non-randomised comparison. However the practical utility of QBA in a HTA setting is currently unclear.

The QBASEL study aimed to investigate the utility of QBA methods for assessing uncertainty in RWD SCA- trial comparisons. This demonstration project investigated 14 trials for advanced non-small cell lung cancer with SCAs being generated from RWD from oncology electronic health records from the United States. The trials which differed by sample size, geographical coverage, biomarker usage and line of therapy provide allowing an understanding of when and how QBA methods may be most useful.

This session will have a varied discussion on the merits of QBA, led by a range of professionals across academic, HTA, and industry settings. First, there will be an overview of the general context and implementation of QBA methodology in an SCA setting. Second, panelists will detail the results from the QBASEL study. The session will conclude with a discussion on the applied and pragmatic implications of the QBASEL results on HTA decision-making.

SponsorCytelModerators

Sreeram Ramagopalan, PhD

F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland

Sreeram (Ram) Ramagopalan is the Global Head for Real World Evidence for Market Access at Roche. Dr Ramagopalan’s team strategically plans and executes real world research studies to obtain and maintain access for Roche medicines. Dr Ramagopalan holds a PhD in Epidemiology from the University of Oxford, as well as an MSc in Health Economics from the London School of Economics. Prior to Roche, Dr Ramagopalan led European Real World Evidence teams at Bristol Myers Squibb and the life-sciences consultancy Evidera. He is an international expert in real world evidence with over 295 peer reviewed publications.

Speakers

Miguel Hernán, PhD, MD, MPH, ScM

Harvard University, Boston, MA, USA

Miguel Hernán uses health data and causal inference methods to learn what works. As Director of CAUSALab at Harvard, he and his collaborators repurpose real world data into scientific evidence for the prevention and treatment of infectious diseases, cancer, cardiovascular disease, and mental illness. His work shapes health policy and research methodology worldwide. As the Kolokotrones Professor of Biostatistics and Epidemiology, he teaches at the Harvard T.H. Chan School of Public Health and at the Harvard-MIT Division of Health Sciences and Technology, where he has mentored dozens of trainees and students. His free online course “Causal Diagrams” and book “Causal Inference: What If”, co-authored with James Robins, are widely used for the training of researchers. Miguel has received many awards for his work, including the Rousseeuw Prize for Statistics, the Rothman Epidemiology Prize, and a MERIT award from the National Institutes of Health. He is Fellow of the American Association for the Advancement of Science and the American Statistical Association, Associate Editor of Annals of Internal Medicine, Editor Emeritus of Epidemiology, and past Associate Editor of Biometrics, American Journal of Epidemiology, and Journal of the American Statistical Association.

Seamus Kent, PhD

National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom

Seamus is a Senior Adviser in Data & Analytics at NICE. He is leading the development of a framework for the use of data and analytics in the development of NICE guidance and has participated in several conferences discussing the role of RWE tools in delivering value to patients and health systems.

Pilar Pinilla, MSc

National Institute for Health and Care Excellence, London, United Kingdom

Pilar is the Associate Director of the NICE International team. She is responsible for the strategic objectives and direction of the NICE International services, oversees all the international services provided by the team and contributes to the delivery of different international engagements through workshops, educational seminars, and international consultancy projects. She has extensive experience in delivering these services at a national and international scale and represents NICE in a series of NHS International groups. Pilar also works in scientific advice projects in the NICE Scientific Advice team, where she contributes to the standard service providing advice on behalf of NICE and also collaborates in parallel scientific advice with other Health Technology Assessment (HTA) and regulatory agencies. She joined NICE in 2012 and worked in the Technology Appraisals and the Highly Specialised Technologies programme until 2016, when she became part of the Scientific Advice team. Pilar holds a University degree in Business Administration and Management from University of La Laguna (Spain), and an MSc in Health Economics, Policy and Law with specialisation in Health Economics from the Erasmus University of Rotterdam (The Netherlands). She is currently conducting a PhD at University of Las Palmas de Gran Canaria (Spain) focusing on HTA. Before joining NICE, Pilar worked as a Researcher in one of the Spanish regional HTA units (SESCS, Canary Islands) conducting systematic reviews and economic evaluations for the Spanish Healthcare System. She is currently on the Board of Directors for the Spanish Health Economics Association (AES).

Kristian Thorlund, PhD

Cytel, Inc., Waltham, MA, USA

Kristian Thorlund is a trained biostatistician with expertise in Bayesian statistics, meta-analysis methods, clinical trial monitoring, and complex models for evidence synthesis, and health care decision-making. He currently serves as a senior principal scientist at Cytel. Between 2016 to 2020 Kristian ranked among the top 1% cited researchers globally in the past decade. He has published well over 150 scientific articles in referred journals and written several book chapters and led the development of multiple open-source statistical software. He has also led over 50 seminars and workshops at prestigious international conferences and has been invited as speaker or chair at more than 100 panel sessions.

12:45 - 13:15HEOR TheaterSequential Models – When Are They the Right Choice?In-person

Sequential modelling has been used in health technology appraisals, clinical pathway and strategic models to support payer, clinical and manufacturer decision-making across several disease areas, including autoimmune diseases and oncology. Building sequential models can shed light on questions that simple treatment line specific models cannot, including the clinically and economically optimal positioning of a therapy in a treatment sequence especially in disease areas with therapies indicated across several lines. These models, however, can prove technically challenging to implement, require comprehensive clinical data sources, and interpretation of results of sequencing models are often more demanding than for line specific models.

During this presentation we will provide an overview of the reasons to consider and develop sequential models. We will outline the decision problems, key challenges and circumstances under which sequential modelling is likely a good investment, and also discuss when this type of modelling may not be the right approach. Across the presentation, multiple recent applications will be leveraged.

Learning Objectives:

Consider what decision problems would require true sequential modelling and cannot be answered by a simpler approach – from a clinical, manufacturer and payer point of view
Understand the sweet spot for sequential models from the perspective of the treatment landscape for a given therapy and therapy portfolio
Suggest methods for executing sequential models – leveraging other pieces of standardly generated evidence

SponsorEvideraSpeaker

Ágnes Benedict, MSc, MA

Evidera, London, LON, United Kingdom

Simone Rivolo, PhD

Evidera, London, LON, United Kingdom

Simone Rivolo, PhD, is a Research Scientist with Evidera in the Modeling and Simulation group in London. Dr. Rivolo has significant experience in developing pharmacoeconomic models and value demonstration tools across multiple disease areas, including cardiovascular disease (atrial fibrillation), oncology (RCC, ovarian/endometrial cancer, breast cancer, NSCLC, gastric cancer, bladder cancer, DLBCL), neurology (Lewy body dementia), infectious diseases (pneumonia, cSSTI, acne), inflammatory bowel disease (ulcerative colitis) and rare diseases (growth hormone deficiency). Dr. Rivolo experience spans a wide range of mathematical modeling, simulation and statistical analysis approaches, including machine learning. Specifically, during his academic career, he focused on patient-specific modeling and cardiovascular hemodynamics, along with large-scale statistical analysis of high-dimensional data for clinical outcome prediction. Prior to joining Evidera, Dr. Rivolo worked as a researcher in the H&VM group at King’s College London. His work focused on the development of innovative image and computational derived biomarkers to enhance patient stratification and optimize treatment for patients affected by heart failure. Dr. Rivolo obtained his PhD from King’s College London where his research addressed cathlab (cardiac catheterization) diagnostics, from developing novel methodologies for signal processing to personalized blood flow modeling for rapid clinical decision. Dr. Rivolo holds a master’s degree in mathematical modeling in engineering and a bachelor’s degree in mathematics for engineering, both obtained from Politecnico di Torino. Dr. Rivolo has authored papers in a number of respected journals, including Journal of medical economics, IEEE Transactions on Biomedical Engineering, AJP: Heart and Circulatory Physiology, Circulation: Cardiovascular Interventions, Journal of Biomechanics, Artery Research and BJOG: An International Journal of Obstetrics & Gynaecology. Dr. Rivolo has also authored multiple conference disseminations, with conference proceedings published in Value in Health, Annals of Oncology, Heart and EHA.

14:45 - 15:15EU Joint Clinical Assessments: What Happens When Gold-Standard Evidence Is Out of Reach?In-person

Regulation (EU) 2021/2282, published in December 2021, aims to harmonize health technology assessment (HTA) across the European Union by launching the EU-wide Joint Clinical Assessment (JCA). As more information emerges on JCA methods, the direction of travel appears to be towards accepting only gold-standard evidence such as randomized clinical trials (RCTs), even when these may not be feasible for ethical or practical reasons. This could create challenges for some health technologies, such as highly innovative treatments, to meet JCA evidence requirements.

In this presentation, Suzette will provide an overview of the new EU regulation and the potential challenges we envisage based on the draft guideline documents published to date. Miranda will then explore the issues relating to indirect treatment comparisons, particularly in the context of a high number of comparators, before James highlights a potential role for structured expert elicitation in filling evidence gaps where the collection of empirical evidence is unfeasible. We will conclude by highlighting the risks of setting the bar too high for evidence generation, particularly for innovative technologies and rare diseases.

Learning objectives:

Understand the current direction of travel for EU JCA and its possible implications
Dissect the challenges and risks posed by the current evidence requirements for JCA
Highlight the value of alternative methodologies where gold-standard evidence is unattainable

SponsorLumanitySpeaker

Miranda Cooper, MSc

Lumanity, Sheffield, DBY, United Kingdom

Miranda is the Head of Statistics at Lumanity, HEOR and has over 8 years experience in HEOR consultancy. Her focus is on developing the strategy, design and implementation of statistical analyses to support global HEOR activities, health technology assessment and standalone statistical research. Her experience covers methods for indirect comparisons such as NMA, population adjustment and time varying treatment effect comparisons, as well as the statistical analyses of clinical trial data across a range of oncology and non-oncology indications.

James Horscroft, PhD

Lumanity, Sheffield, United Kingdom

James has 6 years of experience in providing health economics and outcomes research support for health technologies. Most of his work focuses on health technology assessment strategic planning and gathering evidence from experts, for example, via structured expert elicitation. He has experience in designing and conducting qualitative and quantitative expert elicitation/opinion studies, including the Sheffield Elicitation Framework, Delphi panels, and burden of illness studies. James graduated from a collaborative PhD in Physiology between the University of Cambridge and Zealand Pharma in 2016.

Suzette Matthijsse, PhD

Lumanity, Sheffield, DBY, United Kingdom

Suzette is Senior Director and EU Head of Health Economics Analysis at Lumanity. She has over 10 years’ experience in disease modelling, starting off with a PhD and postdoctoral research, before joining Lumanity over 4 years ago. Since then, she has been involved in a large range of indications, developing health economic models to be used for submissions globally, primarily in various European markets. She has also provided several trainings and workshops to different audiences on the new EU regulation on health technology assessment, and took part in the public consultations of EUnetHTA 21.

15:00 - 16:00Educational SymposiaLeveraging Real-World Evidence for Health Technology Assessment – Using Big Data to Enable Patient AccessIn-person & Virtual

The evolution of the healthcare ecosystem over the past decade has resulted in the increasing availability of big data – i.e., real-world data (RWD) for patients. As a corollary, real-world evidence (RWE) derived from the analysis of RWD is becoming a critical component throughout the product development lifecycle, especially in support of health technology assessment (HTA) and reimbursement discussions. The potential to evaluate the relative effectiveness of therapies in actual use as well as augment clinical trial data where there are few patients or short time horizons makes RWE a compelling solution for evidence generation. However, there is still some uncertainty around the HTA agency requirements, types of RWD, and the analytic approaches to synthesizing such data. The symposium will explore the use of RWE to enable patient access, drawing contrasts across borders to highlight potential strategies for respective HTA agencies.

Specifically,

Matthew Gordon will provide an overview of RWE and growth of application across the product development lifecycle in his opening comments
Jackie Vanderpuye-Orgle will present considerations for analyzing RWD, current evidence gaps in HTA, and some analytic approaches for addressing these gaps.
Emtiyaz Chowdhury will present NICE’s recent real-world evidence framework and provide a case study of how RWE has been used in a recent NICE HTA submission for an anti-CMV drug in post-transplant patients.
Gianluca Biao will discuss using RWE to augment clinical trial data and potential use cases.
Nick Latimer will present a case study on estimating cost-effectiveness using cancer registry data.

SponsorParexel Moderators

Matthew Gordon, BA

Parexel International, Los Angeles, CA, USA

Matthew has over 20 years’ experience developing global, real-world evidence solutions to support the development and commercialization of pharmaceutical / biotechnology products and devices. His experience covers a wide-range of therapeutic areas, as well as strategic objectives from natural history programs, external control arm studies for regulatory approval, and global safety-surveillance mandates. His technical expertise includes program strategy and operational structure in disease and product registries, prospective pharmacoeconomic studies, and systematic literature reviews. Prior to joining Parexel, Matthew served as Vice President, Real-World Evidence at Worldwide Clinical Trials, where he led a global team responsible for all observational research programs. Matthew has also held senior management roles at inVentiv Health Clincal, Outcome Sciences, ICON Clinical Research (formerly Ovation Research Group), and MetaWorks, Inc. Matthew holds a Bachelor of Arts in Sociology from Boston University.

Speakers

Gianluca Baio, PhD

University College London, London, United Kingdom

Gianluca graduated in Statistics and Economics from the University of Florence (Italy). He then completed a PhD programme in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA); he then worked as a Research Fellow and then Temporary Lecturer in the Department of Statistical Sciences at University College London (UK). Gianluca's main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach. Gianluca leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science at University College London.

Emtiyaz Chowdhury, BSc, MA

Parexel, HEOR Modeling, London, United Kingdom

Emtiyaz is a manager in health economics at Parexel. He directs teams of economists and statisticians to prepare HTA submissions and develop global health economic models. Prior to his work in market access consulting, Emtiyaz worked as a health economist at the Royal College of Physicians (National Guidelines Centre) who were commissioned by NICE to develop clinical guidelines. Emtiyaz is the life science industry member on the Highly Specialised Technology Evaluation committee which is NICE’s dedicated HTA programme for rare diseases. Emtiyaz holds an MA from University College London in Philosophy, Politics and Economics of Health and a BSc in Biological Sciences (Physiology and Pharmacology) from the University of Leicester.

Nicholas Latimer, MSc, PhD

The University of Sheffield, Sheffield, DBY, Great Britain

Nick is a Professor of Health Economics at the University of Sheffield, and a Yorkshire Cancer Research Senior Fellow. He is a member of NICE Technology Appraisal Committee B and of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.

Jacqueline Vanderpuye-Orgle, MSc, PhD

Parexel International LLC, Massachusetts, MA, USA

Jackie is a Vice President and the Global Head of the Advanced Analytics Unit with experience in conducting empirical research to underscore the differentiated value of assets across a range of therapeutic areas. In her role at Parexel, Jackie is responsible for providing technical expertise and oversight on network meta-analysis, AI, and general statistical analysis workstreams. Jackie is a health economist with over 20 years of progressive experience in the application of econometric principles and statistical analysis to both clinical and real-world data. Prior to joining Parexel, she worked as a Senior Manager in Global Health Economics at Amgen. Jackie also worked as a Director of Research Operations and Senior Economist at Precision Health Economics where she was responsible for developing proposals and executing projects on several oncology and immune-oncology assets to support regulatory approval, value messaging, and market access in various regions. In addition, Jackie has held research and consulting roles at Endurance Reinsurance, Analysis Group, and the Public Economics and Macroeconomics Divisions of the World Bank in Washington DC. Jackie completed her PhD at Cornell University following a pre-doctoral in economics at Yale University. She is currently Chair of the ISPOR Biosimilars Special Interest Group.

Inclusion of the Caregiver Perspective in Health Technology Assessment: The Case of Alzheimer’s DiseaseIn-person & Virtual

Alzheimer’s disease (AD) has a large humanistic and economic burden, and the historical absence of new AD treatments has resulted in considerable unmet need. New disease-modifying drugs have emerged and have stimulated discussions on how to best to measure the value of these novel therapies.

Broad debates on whether and how to include caregiver perspectives in health technology assessment (HTA) continue. However, AD is atypical in the large potential effects treatments may have on caregivers.

From academic, patient organisation, payer, and industry perspectives, this session will explore the importance of including caregiver impacts when evaluating AD interventions and discuss the key challenges in including them in health technology assessments.

The session will be introduced with a summary of definitions of caregiver outcomes and current recommendations from HTA agencies surrounding their inclusion. Speakers will then discuss: (i) how the high prevalence of AD, the large unmet need, the substantial equity impacts of AD, and its large effects on caregivers’ health and work-related outcomes supports the importance of routine inclusion of caregiver outcomes in assessments of AD therapies; (ii) the key methodological challenges for including caregiver outcomes in HTA for AD, including data availability, risk of double-counting impacts on caregiver and patient health, the presence of multiple caregivers, and whether to differentially weight the QoL outcomes of caregivers and patients; and (iii) what changes in HTA frameworks are required to fully capture the impacts of AD treatments on caregivers, such as taking a wider perspective where important costs and benefits are not currently recommended for inclusion, improving methodological guidance where inclusion is already recommended, and the inclusion of impacts on caregivers qualitatively or separately, rather than pushing to integrate them quantitatively within traditional cost-effectiveness approaches.

SponsorF. Hoffmann-La Roche LtdModerators

Peter Neumann, ScD

Tufts Medical Center, Boston, MA, USA

Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine. He is the founder and director of the Cost-Effectiveness Registry, a comprehensive database of cost-effectiveness analyses in health care. Dr. Neumann has written widely on the role of clinical and economic evidence in pharmaceutical decision making and on regulatory and reimbursement issues in health care. He served as co-chair of the 2nd Panel on Cost-Effectiveness in Health and Medicine. He is the author or co-author of over 300 papers in the medical literature, and the author or co-author of 3 books: Using Cost-Effectiveness Analysis to Improve Health Care (Oxford University Press, 2005) ; Cost-Effectiveness in Health and Medicine, 2nd Edition (Oxford University Press, 2017); The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press, 2021). Dr. Neumann has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). He is a member of the editorial advisory board of Health Affairs and the health policy advisory board for the Congressional Budget Office. He has also held several policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration. He received his doctorate in health policy and management from Harvard University.

Speakers

Hareth Al-Janabi, PhD

University of Birmingham, Birmingham, United Kingdom

Hareth is a Professor of health economics and Wellcome Trust Investigator at the University of Birmingham. His research interests include: decision-making, the role of family (informal) care in the economic evaluation, and research methods (especially qualitative methods) in health economics. He has developed and applied a range of techniques to include informal care in economic evaluation and has worked across a number of clinical areas (including dementia).

Samantha Benham-Hermetz, .

Alzheimer's Research UK, Cambridge, CAM, United Kingdom

Samantha Benham-Hermetz is Director of Policy and Public Affairs at Alzheimer’s Research UK. Sam sits on the UK Government’s Ministerial Programme Board for Dementia, and has worked to influence the forthcoming UK 10 Year Plan and Dementia Mission. She is Chair of the Charity Medicines Access Partnership and attended the Patient Access to Medicines Partnership. In 2020, she represented patients on the NICE Methods Working Group seeking to ensure that patient perspectives were incorporated in value and assessment of new medicines.

Jacoline Bouvy, PhD

National Institute for Health and Care Excellence, London, LON, United Kingdom

Jacoline is the Technical Director for NICE Scientific Advice. She signs off and quality assures work across all services offered by the team and chairs national and parallel scientific advice meetings with medical device and pharmaceutical companies. She also works on the joint NICE, NHS England, and Department of Health and Social Care (DHSC) project that is developing new payment models for antimicrobials and is the chief analyst for NICE on the DHSC’s Health and Care Appraisal Working Group. Previously, she worked in NICE’s Science Policy and Research team where she has led NICE’s work on the European Health Data and Evidence Network, HTA of histology independent cancer drugs, the use of patient preference studies in HTA, and several other pan-European research projects. Before joining NICE in 2015, Jacoline worked at the European Medicines Agency where she was involved in the EMA registries initiative. Before that, she held postdoctoral positions at Erasmus University Rotterdam and Utrecht University in the Netherlands. She has a PhD from Utrecht University, the Netherlands and has a MSc in health economics from Erasmus University Rotterdam, the Netherlands.

Sreeram Ramagopalan, PhD

F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland

15:15 - 15:35HEOR TheaterAvalere's Perspective: Value and Evidence Determination Post-IRAIn-person & Virtual

The recently passed Inflation Reduction Act (IRA) allows the Medicare program to negotiate drug prices for the first time in program history. Importantly, the process of setting prices will include a review of comparative effectiveness evidence, which is part of a broader US trend to rely increasingly on this type of analysis. Understanding the implications of these changes for the evolving value ecosystem is critical for evidence strategy and R&D investment decision-making.

In this presentation, Avalere Health experts will discuss how value and evidence strategies and investment decision-making need to evolve in an post-IRA world.

Learning objectives:

How IRA drug pricing negotiation provisions will change the value landscape
The impacts of IRA on R&D investment decision-making
How data can be leveraged to improve value positioning in a post-IRA world

SponsorPRMA ConsultingModerators

David Sykes, MSc

PRMA Consulting, a member of Fishawack Health, London, DC, United Kingdom

Speakers

Sarah Alwardt, PhD

Avalere Health, a member of Fishawack Health, Washington, DC, USA

Michael Ciarametaro, MBA

Avalere Health, a member of Fishawack Health, Washington, DC, USA

15:45 - 16:15Generating High Quality Real-World Evidence for Regulatory Decision Making in EuropeIn-person

The role of real-world data in clinical research is growing, and researchers need regulatory-grade real-world evidence. Understand the challenges that RWE researchers face while navigating the regulatory landscape in Europe, and how we can meet those challenges.

SponsorTriNetXSpeaker

Lenka Kellermann, MScEcon

TriNetX Oncology, Freiburg, Germany

Tue 8 Nov11:30 - 12:30Educational SymposiaUnlock Real-World Data with Machine LearningIn-person & Virtual

An increasing demand for real-world evidence (RWE) to support regulatory submissions and payer negotiation has accelerated the big-data revolution in health care. Major strides have been made in improving the availability and usability of real-world data (RWD), and the application of machine learning (ML) in RWD is an essential step forward to augment meaningful insights into the care pathway. This symposium will introduce several creative ML approaches to establish consistency across data sources, harness the research value of RWD, and discuss the application of transparent and interpretable ML methods.

First, a case study will illustrate how ML methods are used to improve the consistency, transparency, and traceability of RWD across hospitals in China, where RWE research has traditionally faced many challenges. In a large-scale multi-center hematology study, researchers established a disease model to incorporate both consensus-based decision logic and ML-based data-driven optimization to unify data standards and definitions. This tool was applied to mechanize information integration across top hematology centers in China to enable result synchronization, which is essential in the generation of high-quality evidence to support China’s National Reimbursement Drug List’s negotiation and post-launch activities.

The second presentation will demonstrate how ML tools help to empower data transformation while ensuring relevance and validity. The raw RWD captured by hospital information systems (HIS) are fragmentary and lack an integrated picture of the care pathway. To close this gap, researchers developed and validated progression algorithms, drawn from China’s National Longitudinal Cohort of Hematological Diseases (NICHE), to reconstruct the complex patient journey through a hematologic condition. The progression algorithm was then incorporated into an automated data capture system to obtain, filter, and process traceable data, allowing researchers to supply important information that is not readily available from the HIS, including patients' treatment response by line of therapy.

Lastly, the presenters will demonstrate new transparent and interpretable ML approaches using case studies. Generalized linear models (GLM) are often preferred over ML models because of concerns regarding the lack of interpretability of complex ML algorithms. State-of-the-art interpretable ML methodologies will be discussed, along with their applications to a wide-ranging set of ML models. The objective of these approaches is to describe both general model behavior and the logic behind individual data unit predictions. These methods provide an improved level of interpretability and transparency that is both informative and, to some extent, more granular than GLM.

We hope this symposium can introduce these recent creative examples in the intersection of ML and RWD to the audience and stimulate discussions to further advance RWE research and methodology.

SponsorAnalysis Group, Inc.Moderators

Eric Q. Wu, PhD

Analysis Group, Inc., Boston, MA, USA

Dr. Wu is a health economist with expertise in health economics and outcomes research (HEOR), market access, and scientific evidence strategy. He has conducted research in more than 30 countries on behalf of pharmaceutical and medical device companies, payers, providers, and government agencies. Dr. Wu has contributed to over 200 publications across dozens of therapeutic areas, including regenerative therapies (gene and stem cell), rare and ultra-rare diseases, biologics, and immuno-oncology. He spends a significant portion of his practice developing new scientific methods to address challenges in health care research. Dr. Wu has developed client-focused solutions based on the use of artificial intelligence (AI), medical big data, real-world evidence, and innovative comparative-effectiveness research methodologies.

Speakers

Max Leroux, MSc

Analysis Group, Inc., Quebec, QC, Canada

Mr. Leroux specializes in the application of machine learning (ML) and big data technologies to complex problems related to health care, finance, marketing analytics, fraud detection, client engagement, process automation, and credit and market risk. His experience includes developing data-focused strategies and campaign targeting models; developing predictive response event and time-to-event models, analyzing investment and finance portfolios; building predictive, next-best-offer, and behavioral analytics models; leading information technology projects from proof-of-concept stage to enterprise level; and designing, developing, and implementing automated credit scoring and fraud detection systems. Mr. Leroux has also overseen the implementation of ML solutions and big data technologies in numerous domains for both on-premises infrastructure and major cloud platforms.

Jimmy Royer, PhD

Analysis Group, Inc., Montreal, QC, Canada

Dr. Royer, a principal at Analysis Group, has extensive experience applying his skills to health care and pharmacoeconomics. His work includes predicting the potential future onset of rare or undiagnosed conditions with machine learning models applied to large datasets, assessing the socioeconomic impact of a drug, and conducting HEOR data analysis to evaluate the cost effectiveness of disease treatments. Dr. Royer has worked with pharmaceutical companies to assess the competitive impact of generic pharmaceuticals and has assisted with statistical issues associated with clinical drug trials. A previous presenter on artificial intelligence and machine learning topics at ISPOR, he has also published in journals such as Neurology, Current Medical Research and Opinion, Dermatology and Therapy, and eBioMedicine. Dr. Royer serves on the scientific committee for Le Québec Économique, a consortium that strives to broaden the understanding of economic issues in Canada.

Xiaochen Zhang, MS

Beijing Huashu Yihui Technology Co., Ltd., Bejing, China

Ms. Zhang is a biostatistician who applies statistics and big data analysis to research real-world medical health issues. Her medical and statistical background give her rich, interdisciplinary experience in topics such as registered queue research, big data analysis, and data visualization, with a focus on hematology, epidemiology, health economics, and other fields. Ms. Zhang is also proficient in a variety of statistical models and research methods, as well as using statistical modeling and big data analysis to solve practical problems in drug efficacy and safety evaluation and cohort study design. Earlier in her career, she worked as a bioinformatics analyst at a large medical company, where she gained rich project experience in data analysis, algorithm design, big data visualization, consulting reports, medical health knowledge support, and biological information analysis.

Jia Zhong, ScD

Analysis Group, Inc., Boston, MA, USA

Dr. Jia Zhong, a Manager at Analysis Group, is an epidemiologist who specializes in clinical health economics and outcomes research (HEOR), clinical trials, and large-scale epidemiological studies. She has more than 10 years’ research experience in disease areas such as pulmonary diseases, diabetes, cardiovascular diseases, oncology, rare diseases, and immunology. Dr. Zhong’s expertise includes comparative efficacy and safety, individualized medicine, and longitudinal analysis. Her recent work includes leading large-scale prospective cohort studies, evaluating the comparative efficacy of immuno-oncology treatments for regulatory submissions, quantifying direct and indirect resource utilization and costs, and evaluating real-world effectiveness using patient reported outcomes (PROs). Her research has been published in Nature Immunology, Immunity, Proceedings of the National Academy of Sciences, Circulation, Circulation Research, Hypertension, and the International Journal of Epidemiology, among other publications. Prior to joining Analysis Group, Dr. Zhong was a research scientist at the Columbia University Mailman School of Public Health, where she developed data-based therapeutic target identification frameworks.

Gene Therapies: Where High Promise Meets High Uncertainty, How Should HTA Methodologies Appropriately Value and Enable Access?In-person & Virtual

Gene therapies represent a new era of medicine, offering the potential for truly transformational health gains and further benefits for society and health systems. Health Technology Assessment (HTA) of gene therapies can be challenging for a variety of reasons, including uncertainty regarding long-term outcomes, initial assessment of clinical value, assessment of costs and narrow perspectives. These challenges can result in the potential benefits of these therapies being overlooked and undervalued. This symposium will provide an overview of the challenges faced in the HTA of gene therapies and discuss potential solutions to overcome them. Panelists will provide diverse perspectives and practical examples of solution implementation. The recommendations provided by the panel will demonstrate the practical tools available to address the HTA challenges and work towards unlocking the potentially transformational promise of gene therapy for patients and society.

SponsorPfizerModerators

Adrian Towse, MA, MPhil

Office of Health Economics, London, United Kingdom

Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare. A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term. Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.

Speakers

Amanda Cole, PhD

The Office of Health Economics, London, LON, United Kingdom

Amanda is a Senior Principal Economist at the Office of Health Economics. Amanda’s research interests include the economics of innovation, use of real-world evidence to support HTA and product development; novel pricing and reimbursement mechanisms for pharmaceuticals, and the interaction between HTA policy and optimal R&D decisions by industry. Before joining OHE in 2014, Amanda was a research fellow at the University of Birmingham where she focused on the HTA of medical devices and embedding health economic evaluation early in product development. Amanda holds a PhD in health economics from the University of Birmingham.

Josie Godfrey, MA

JG Zebra Consulting, Hammersmith, United Kingdom

Josie has worked in rare diseases and innovative therapies for over 12 years. She currently runs a consultancy business specialising in strategic market access, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning global collaboration developing evidence and tools to support HTAs for new treatments for Duchenne Muscular Dystrophy. Josie is also co-founder and joint CEO of Realise Advocacy, which supports patient involvement in drug development and access processes. She previously led work at NICE to establish the Highly Specialised Technologies programme.

Ruth Kim, PharmD, MPH

Pfizer Inc, New York, NY, USA

Ruth Kim is an intrapreneur in developing unique launch plans for pharmaceutical companies. During her tenure in industry, she successfully led a team launching multiple immuno-oncology assets, with extensive publications in oncology. Ruth established companywide real-world evidence and epidemiology functions at Medivation and Bristol Myers Squibb. Now at Pfizer, she is the Health Economics and Outcomes Research (HEOR) Team Leader for Rare Disease and Internal Medicine. Ruth is a pharmacist by training with a master’s in public health. She completed her post-doctoral fellowship in HEOR and Epidemiology focusing on Haemophilia and Hepatocellular Carcinoma. Prior to joining industry, she was an adjunct professor in physical chemistry, pharmacoeconomics and pharmacy technics.

Oriol Sola Morales, MD, MSc, PhD

The HiTT Foundation, Barcelona, Spain

I have built a career in the planning, policy and decision-making environment (Regional HTA Agency director (2007-11); Innovation and Strategy Director in several hospitals, member of the Regional Research Advisory Council and policy advisor to 2 Health Ministers). In 2011 I founded HITT, transformed in 2019 into the HiTT Foundation to strive for a sustainable healthcare. I am an assistant professor in Health Economics at Universitat Ramon Llull -IQS, and teach in several courses nationally and internationally and a Research Fellow at the OHE (office for Health Economics). I am an MD specialised in Internal Medicine. I earned an MSc from the London School of Economics and Political Science (LSE) and the London School of Hygiene and Tropical Medicine (LSHTM) and a PhD from Universitat Rovira i Virgili (URV).

11:40 - 12:00HEOR TheaterHow AI Unlocks RWE in Belgium, France, Germany – Use Case in Rare Diseases (hATTR and ATTR-CM)In-person

Multi-center RWE Hubs are enabling large-scale evidence generation across various therapeutic areas (i.e. cardio, onco, neuro) in Belgium, France, and Germany. Georges De Feu (LynxCare) and Jean-Baptiste Angeloglou (PSIH) will share how these federated clinical data networks create value for patients, clinicians, and researchers. Clara Oeste (LynxCare) will showcase how these insights are being used to identify patients at-risk of rare diseases through A.I. and NLP. LynxCare works with leading hospitals in Europe and the United States, and facilitates scientific research for Life Sciences companies such as AstraZeneca, Pfizer and Novartis within a strict ethical and legal framework.

SponsorLynxCareSpeaker

Jean-Baptiste Angeloglou, MSc

PSIH Group, Lyon, France

Jean-Baptiste Angeloglou is the Managing Director of PSIH Group, the leader in Business Intelligence in hospitals in France. After obtaining his MSc in Law and Taxation from the Panthéon-Assas University in Paris, France, he held positions as Deputy Managing Director at CEGEDIM, Head of Sales at CMPMedica, and Commercial Director and member of the ComEx of IQVIA France. PSIH has been supporting healthcare organisations for over 20 years in analyzing their structured data and is active in more than 1,000 hospitals. PSIH also manages databases such as the French hospital discharge database (PMSI) and French nation-wide healthcare data system (SNDS), which is the largest RWD source in Europe.

Georges De Feu, PharmD

LynxCare, Leuven, Belgium

Georges De Feu is a 3x successful start/scale-up founder & exec in HealthTech. Georges is the CEO and founder of LynxCare, the Big Data market leader in healthcare. With an AI-driven data platform, they help hospitals unlock previously unavailable clinical data for better patient care and scientific research. They work with leading hospitals in Europe and the United States, and facilitate scientific research for Life Sciences companies such as AstraZeneca, Pfizer and Novartis within a strict ethical and legal framework.

Clara Oeste, PhD

LynxCare, Lyon, France

Clara L. Oeste obtained her BSc and MSc in Biochemistry and Molecular Biology at the Complutense University in Madrid, Spain. Her PhD at the Spanish National Research Council focused on the dynamics of RAS proteins, and during her 5+ years as a postdoc, she focused on their role in leukaemia and breast cancer. She has co-authored numerous scientific papers, editorials, and book chapters. Recently, as a medical writer and scientific advisor, she has focused on the value of real-world evidence (RWE) in diverse therapeutic areas to foster AI-driven clinical insights and patient outcomes research.

12:45 - 13:15Using RWD to Deliver Better Therapies and Outcomes – A Focus on Rare DiseaseIn-person

Pharma has much to gain if it can work out how to use anonymized RWD to help tackle rare disease. Using RWD can be used to help scope reliable evidence (Real-World Evidence, RWE) to support better decision-making and develop new therapies for rare diseases. RWE can expand on disease epidemiology, progression, and patient pathways, and help gain clarity on the safety and effectiveness of novel therapies both in development and following marketing authorisation.

But while the opportunities are clear, there are many challenges to realising these. This is new territory. Pharma needs to improve skills and capabilities internally in order to realise these opportunities. It also needs robust, well-structured and high-quality data, as well as academic and data partners with whom it can work to derive the insights required from data. Then there is the question of how able HTAs are to accept RWD.

SponsorCegedim Health DataSpeaker

Gilles Paubert, MA, MBA

Cegedim Health Data, Boulogne-Billancourt, France

Gilles Paubert is the Global Head of Cegedim Health Data, which provides Real World Data and Evidence (RWD-E) to drive cutting-edge improvements in patient outcomes, and boasts a data history of over 25 years and millions of anonymized patient records in Europe that are immediately accessible via the THIN®️ (The Health Improvement Network) database. Gilles has a scientific and business school background and nearly two decades experience in life science data analysis and consulting. He provides innovative solutions and advice to help the life science industry manage big data, statistics and analytics.

14:45 - 15:15Generating Payer Insights and Evidence: Meeting the Challenges of the New Evidence ParadigmIn-person

Overview
A new evidence paradigm is emerging with the availability of additional real-world data sources facilitated by technological innovation and the evolving adoption of real-world evidence (RWE) in regulatory, reimbursement and other health care decisions. The intent of this presentation is to describe the new evidence paradigm and why generating payer insights and relevant, impactful evidence is now a ‘must have’. The presentation will describe opportunities, including tech-enabled solutions, which can add speed, agility, and scientific rigor to the generation of payer insights and evidence generation.

Description
In an evolving healthcare environment with continuous economic pressures, clinical innovation alone is not enough to achieve optimal reimbursement and patient access. Payers, regulator, and health technology assessment (HTA) agencies prioritize products that demonstrate the most compelling clinical and economic value, including improvements in patient outcomes.

Timely payer insights can inform a relevant evidence package and development strategy by defining the data required to support an optimal place in therapy/treatment pathways and by describing the value of the product in populations of unmet need. However, heterogeneous evidence requirements from different HTA agencies make it challenging to use the same evidence package across markets. HTA policies and stringent data requirements mean payer insights are critical throughout the product life cycle to understand what is most meaningful to them. When products come to market with a limited evidence package, HTA agencies and payers are likely to turn to credible and robust RWE to assess the value of a product post-launch. This adds complexity because companies have a need to gather payer insights and customize each evidence package to support the value assessments at launch and then update evidence for post-launch reviews.

In demonstrating the full value of a product and customizing evidence packages, consideration should be given to emerging RWE data sources to capture the data elements required to differentiate products in competitive disease areas. Many if these emerging data sources include supplementary elements such as patient outcomes, genomics, biomarkers, etc., which may allow companies to address research questions with greater precision. Opportunities also exist to integrate data across sources; however, this requires complex analytical RWE expertise. Clearly defining the analytical approaches using reliable scientific methods is critical for gaining acceptance among regulatory and HTA agencies, especially when RWE is being considered as confirmatory evidence.

In summary, the dynamic global healthcare environment elevates the need for scientifically rigorous and fit-for-purpose evidence generation, informed by payer insights, throughout the product development life cycle and continually updated following product launch. This iterative process of insight collection and evidence generation is critical to build an evidence package that is relevant and informative for decision-makers.

SponsorGenesis ResearchSpeaker

Tijana Ignjatovic, PhD

Market Access Transformation, Short Hills, NJ, USA

Tijana Ignjatovic has over 15-years of consultancy experience within market access, having conducted over 100 pieces of research across a range of therapy areas during her time at MAT. This experience has given Tijana in-depth knowledge on how to optimize research methodologies to meet the strategic intent of payer research and provide actionable recommendations.

Priti Jhingran, PhD

Genesis Research, Hoboken, NJ, USA

With over two decades in the pharma industry, Priti Jhingran has focused on understanding evidence needs and delivering tools/solutions for access decision makers/HTAs. She has led multiple enterprise level initiatives; launched 15+ products; and developed diverse teams of scientists dedicated to the generation, dissemination, and communication of evidence.

15:00 - 16:00Educational SymposiaNew Pan-EU HTA: How Can Biopharma Navigate the Joint Clinical Assessment?In-person & Virtual

The EU Joint HTA regulation was enacted in January 2022 with the primary objective of streamlining the clinical assessment process for EU member states’ HTA bodies. Given the newness of the assessment process and lack of precedent on which to draw conclusions, health technology developers such as biopharma are seeking guidance on clinical development and market access processes in this uncharted environment.

In this educational symposium, we will discuss the components of the EU joint HTA regulation, share anticipated impacts at the level of the joint and individual EU member HTA body, and provide key considerations for health technology developers as they prepare for market authorisation and HTA submission in this new and evolving process.

SponsorAmerisourceBergenModerators

Tommy Bramley, PhD

AmerisourceBergen, London, United Kingdom

Thomas J. Bramley, PhD, is Senior Vice President of Global Consulting at AmerisourceBergen where he is responsible for delivery and expansion of global market access, real-world evidence (RWE), and health economics and outcomes (HEOR) research activities across international markets. Prior to his current role, Tommy was President of Lash Group and served as President of Xcenda, both business units at AB, where he has dedicated his 20+ year career to enhancing the delivery and efficiency of healthcare to improve patient outcomes. Tommy has been an invited speaker at many international conferences and has authored numerous peer-reviewed articles. He serves on the Board of Directors for valued charities, including The AmerisourceBergen Foundation and Claire’s Army.

Speakers

Michael Drummond, PhD

University of York, York, YOR, United Kingdom

Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.

Mihai Rotaru, MSc

European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium

Mihai Rotaru works as a Senior Manager Market Access at EFPIA (European Federation of Pharmaceutical Industries and Associations) where he is currently responsible for overall industry engagement in the implementation of the HTA Regulation at EU level. In addition, Mihai is also responsible for oncology policy working with the EFPIA Oncology Platform to drive industry engagement at EU and national levels, biotherapeutics as well as various supply chain issues (Falsified Medicines Directive, shortages and anticounterfeiting efforts).

Alberto Rubio, MBA

PharmaLex, Madrid, Spain

Alberto Rubio is a Senior Director at PharmaLex and Head of International Market Access. He has over 15 years of experience in the life sciences industry where he has held increasingly senior roles in market access and government affairs. Alberto’s expertise is in healthcare innovation projects, with focuses on eHealth, value-based healthcare, innovative contracting schemes and public-private collaborations. Previously, Alberto held managerial roles in other industries, including legal and financial services. He holds a BS in Economics from Texas Christian University and an MBA from the University of Phoenix.

Are We Turning a Blind Eye? Challenges in Value Assessment of Gene Therapies for Inherited Retinal DiseaseIn-person & Virtual

Gene therapies have the potential to deliver transformative improvement in patient outcomes but challenge the status quo of value assessment by HTA bodies and affordability for health care systems.

In the value assessment of gene therapies, which typically target severe diseases, various challenges are uniquely combined. For example, for diseases with early-onset and a slow progression such as inherited retinal disease (IRD) X-linked retinitis pigmentosa, multiple issues exist in measuring and quantifying the quality of life over a long time horizon. Furthermore, while some gene therapies may have relatively large upfront costs, their potentially large long-term benefits for patients, informal carers, and families are uncertain. Their expected value is also affected significantly by the various discounting approaches used internationally. Understanding and, where possible, overcoming these and other value assessment challenges for gene therapies like IRD X-linked retinitis pigmentosa is critical for all concerned with value-driven innovation, payment, and access models.

This educational session will be focused primarily on those within the HEOR community that aim to expand their knowledge base regarding the challenges faced by gene therapies. It will provide an introductory overview of how traditional value assessment is challenged when evaluating gene therapies, using IRD X-linked retinitis pigmentosa as one example, and what solutions may exist based on learnings from various countries and/or other technologies. These challenges and solutions will be discussed by representatives from a patient organisation, an HTA-organisation, and industry. A moderated debate between the speakers and the audience will conclude the session.

SponsorOffice of Health EconomicsModerators

Lotte Steuten, PhD, MSc

Office of Health Economics, London, LON, United Kingdom

Lotte Steuten, PhD, is Deputy Chief Executive at the Office of Health Economics and Honorary Visiting Professor at City, University of London, UK. After graduating cum laude with her PhD from Maastricht University, the Netherlands, Lotte has been active in the HEOR field for over 15 years in various academic roles and executive functions (non-profit and for-profit). To inform better decisions based on independent research and analysis, Lotte worked effectively with pharmaceutical industry, technology assessors, payers and policy makers, (academic) researchers, clinical and patient representatives as well as capital investors. Notably, her international career-path, including the UK, the US and the Netherlands, has provided her with deep insights in the fundamental differences as well as commonalities between the role of HEOR in different healthcare systems, cultural and societal values. Over the past decade, Lotte has been leading diverse teams; developing strategies and delivering on program or company missions, values and objectives; acquiring funding and managing budgets; and been responsible for executive decision-making as well as legal and fiduciary matters. In her current role at the Office of Health Economics she is responsible for the research-led consulting program, making sure to maintaining OHE's stellar reputation for objective, innovative and high-quality research and analysis globally, as well as meeting its charitable objectives. In addition, she is a professor at City, University of London, contributing to their Master and PhD programs. Before joining OHE, Lotte worked as an Associate Member at the Fred Hutch Cancer Research Institute and Associate Professor at the University of Washington, Seattle (US), where she currently holds affiliate appointments.

Speakers

Simon Brassel, MSc, Dipl.Ing

Office of Health Economics, London, LON, United Kingdom

Simon is a Health Economist and Electrical Engineer and works as a Principal Economist at the Office of Health Economics in London. He has over eight years of professional experience in international healthcare markets covering the whole spectrum of health technologies, from pharmaceuticals to digital health technologies. Before joining the OHE, Simon worked at the EMEA headquarters of Fresenius Medical Care, the world’s largest provider of products and services for patients suffering from chronic kidney disease. There, he held various roles within the field of therapy marketing and product management and in the field of Health Economics, Market Access and Policy Affairs. Before Fresenius, Simon worked as a technology consultant at the VDI Technologiezentrum in Berlin. As part of a small team, he worked on the National Strategy Process on Innovations in Medical Technology, which three German Federal Ministries initiated. He also served as a grants officer and helped fund a range of collaborative research projects in the health-tech field with support from the German Federal Ministry of Research and Education. Simon also holds an MSc in Health Policy (Health Economics) from the London School of Economics and Political Sciences (LSE), a Dipl.-Ing. in Electrical Engineering from the Technische Universität München (TUM), and a joint Diploma in General Management from the University of Rochester and the University of Bern.

Caroline Bregman, PharmD, MSc

NICE, Manchester, LAN, United Kingdom

Caroline is a Senior Scientific Adviser in the NICE Scientific Advice team. Caroline is involved in a range of scientific advice projects and parallel scientific advice procedures with other health technology assessment and regulatory agencies, as well as delivery of educational seminars. Previously, Caroline worked in the Technology Appraisals Programme at NICE. Before joining NICE, Caroline worked in a consultancy within the life science sector where she led on the development of health technology assessment dossiers for submissions in the UK and in France. Caroline holds a Doctorate in Pharmacy from Paris Descartes University and an MSc in Health Economics from Paris Dauphine University.

Avril Daly, BSc

Retina International, Zürich, Switzerland

Avril Daly is CEO of the patient-led global umbrella organisation, Retina International, which represents the voice of charities and foundations concerned with the promotion of retina research and related health policy in 43 countries. Avril has worked in health policy for rare and common eye disease since 2000 and was CEO of the Irish NGO, Fighting Blindness for eight years. She was Chairperson of Rare Disease Ireland from 2006-2019. In 2009 she was elected to the Board of Directors of Rare Disease Europe – EURORDIS, and has been the organisations Vice-President since 2012. In 2011 Avril was appointed by the Irish Minister for Health to a steering committee working towards the development of the Irish National Plan for Rare Diseases. The plan was published in 2014. She was appointed to the Irish Clinical Programme for Rare Diseases at the Health Service Executive (HSE) in Ireland in 2014 and was recently appointed to the Rare Disease Technology Review Committee and the National Centre of Pharmacoeconomics (NCPE) to review orphan therapies that fail initial Health Technologies Assessment (HTA) for statutory reimbursement. Avril has held board positions with the European Patient Forum, The European Platform for Patient Organisation Science and Industry (EPPOSI), the HRCI (formerly Medical Research Charities Group) in Ireland and IPPOSI, the Irish Platform for Patients Organisation Science and industry. She believes that patients have a critical role to play in aiding the development of appropriate interventions from concept to delivery. Avril holds a Bachelors Degree in Business and International relations, she is affected by Retinitis Pigmentosa.

Jennifer M Lee, MBA, BSc

Janssen-Cilag Denmark, Birkerod, 84, Denmark

Jennifer is a trained health economist and biochemist. She completed her BSc in Biology/Biochemistry at the University of British Columbia, her MBA at the University of Calgary in Canada and her Postgraduate in Pharmacoeconomics in Melbourne, Australia. She began her career as a health economist in consulting in 2004 in Sydney, Australia, before moving onto a senior health economist role at Sanofi in Australia. She then moved to a regional market access role based in Singapore with Sanofi, prior to moving to the UK and joining Janssen in 2012. She led the Janssen UK Operating Company team responsible for market access, government affairs, policy and patient engagement as the Director of HEMAR and Advocacy until December 2019. She is currently the EMEA Therapy Area Market Access Leader for Immunology, Retina, CVM and VOT based in Copenhagen, Denmark.

15:45 - 16:15HEOR TheaterLiveHTA; Concept and Implementation Tools for the Future of Health Technology AssessmentsIn-person

In response to the recent changes (COVID-19 pandemic, precision medicine, increased healthcare expenditure, expanded use of real-world data), healthcare decision-making bodies searched for new ways to implement more responsive, innovative processes for the technologies’ assessment. These changes along the increased recognition of current HTA challenges (evidence availability and quickly out of date evidence reviews, health care system sustainability, budgetary constraints) have opened a window of opportunity for decision makers to consider moving towards a fully, “living” approach to health technology assessment (HTA). Furthermore, these challenges are even magnified in the context of a general drive for faster patient access to effective health technologies.

This theatre will discuss how the traditional methods for synthesizing comparative clinical and economic evidence for the assessment of new health technologies may be problematic and how the exploration of more reactive and flexible “liveHTA” approaches can mitigate these challenges. The speakers will also discuss implementation tools to make liveHTA a reality for health care decision-makers.

SponsorCytelModerators

Bart Heeg, MSc, PhD

Cytel, Rotterdam, ZH, Netherlands

Bart has more than 20 years of experience in leading and conducting health economic and outcomes research consulting projects for pharmaceutical industry, such as trial analyses, economic modelling, and indirect treatment comparisons for HTA submissions. Prior to merging with Cytel Bart co-founded Ingress-health in 2015. Bart is passionate about developing or extending methodologies such as Bayesian survival network meta-analyses, developing tumor agnostic models and models for early/adjuvant oncology therapies. Bart has a PhD in health economics from the University of Groningen.

Speakers

Anna Forsythe, PharmD, MSc, MBA

Cytel, Waltham, MA, USA

Prior to joining Cytel, Anna Forsythe cofounded PSE with the vision of providing curated evidence and economic modeling to support global HTA. In addition to her PharmD and MBA, Anna earned a MS in Health Economics and Public Policy from the University of Birmingham.

Grammati Sarri, PhD, MSc, DiDS

Cytel, London, United Kingdom

Grammati worked in private consultancies leading HEOR projects and providing strategic guidance on evidence synthesis and clinical effectiveness analyses for early product value demonstration and HTA submissions. Grammati has also a strong experience in understanding the payer perspective and requirements through her senior leadership role in the development of NICE Guidelines and closely working with clinicians, patient's and NICE executives.

Andre Verhoek, MSc

Cytel, Rotterdam, ZH, Netherlands

Using his background in econometrics, Andre focuses on advanced analytics in HEOR. He has a wealth of experience in developing global cost-effectiveness models, Bayesian network meta-analyses and working with innovative HEOR technology products within Cytel’s LiveHTA suite.

ISPOR Europe 2022

6 - 9 November

Educational Symposia & HEOR Theater

The Professional Society for
Health Economics and Outcomes Research