* Program subject to change
In-person & Virtual
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.
EuroQol Research Foundation
Elly Stolk, PhD
EuroQol Research Foundation, Rotterdam, Netherlands
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Michael Herdman, MSc
Insight Consulting & Research, Mataró, Spain
Janine Verstraete, PhD
Department of Paediatrics and Child Health, University of Cape Town, Cape Town, South Africa
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.
Sreeram Ramagopalan, PhD
F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland
Miguel Hernán, PhD, MD, MPH, ScM
Harvard University, Boston, MA, USA
Seamus Kent, PhD
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Pilar Pinilla, MSc
Universidad de Las Palmas de Gran Canaria, London, United Kingdom
Kristian Thorlund, PhD
Cytel, Inc., Waltham, MA, USA
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.
Ágnes Benedict, MSc, MA
Evidera, London, LON, United Kingdom
Simone Rivolo, PhD
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.
Miranda Cooper, MSc
Lumanity, Sheffield, DBY, United Kingdom
James Horscroft, PhD
Lumanity, Sheffield, United Kingdom
Suzette Matthijsse, PhD
Matthew Gordon, BA
Parexel International, Los Angeles, CA, USA
Gianluca Baio, PhD
University College London, London, United Kingdom
Emtiyaz Chowdhury, BSc, MA
Parexel, HEOR Modeling, London, United Kingdom
Nicholas Latimer, MSc, PhD
The University of Sheffield, Sheffield, DBY, Great Britain
Jacqueline Vanderpuye-Orgle, MSc, PhD
Parexel International LLC, Massachusetts, MA, USA
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.
F. Hoffmann-La Roche Ltd
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Hareth Al-Janabi, PhD
University of Birmingham, Birmingham, United Kingdom
Samantha Benham-Hermetz, .
Alzheimer's Research UK, Cambridge, CAM, United Kingdom
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
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.
David Sykes, MSc
PRMA Consulting, a member of Fishawack Health, London, DC, United Kingdom
Sarah Alwardt, PhD
Avalere Health, a member of Fishawack Health, Washington, DC, USA
Michael Ciarametaro, MBA
Lenka Kellermann, MScEcon
TriNetX Oncology, Freiburg, Germany
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.
Analysis Group, Inc.
Eric Q. Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Max Leroux, MSc
Analysis Group, Inc., Quebec, QC, Canada
Jimmy Royer, PhD
Analysis Group, Inc., Montreal, QC, Canada
Xiaochen Zhang, MS
Beijing Huashu Yihui Technology Co., Ltd., Bejing, China
Jia Zhong, ScD
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Amanda Cole, PhD
The Office of Health Economics, London, LON, United Kingdom
Josie Godfrey, MA
JG Zebra Consulting, Hammersmith, United Kingdom
Ruth Kim, PharmD, MPH
Pfizer Inc, New York, NY, USA
Oriol Sola Morales, MD, MSc, PhD
The HiTT Foundation, Barcelona, Spain
Jean-Baptiste Angeloglou, MSc
PSIH Group, Lyon, France
Georges De Feu, PharmD
LynxCare, Leuven, Belgium
Clara Oeste, PhD
LynxCare, Lyon, France
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.
Cegedim Health Data
Gilles Paubert, MA, MBA
Cegedim Health Data, Boulogne-Billancourt, France
DescriptionIn 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.
Tijana Ignjatovic, PhD
Market Access Transformation, Short Hills, NJ, USA
Priti Jhingran, PhD
Genesis Research, Hoboken, NJ, USA
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.
Tommy Bramley, PhD
AmerisourceBergen, London, United Kingdom
Michael Drummond, PhD
University of York, York, YOR, United Kingdom
Mihai Rotaru, MSc
European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium
Alberto Rubio, MBA
PharmaLex, Madrid, Spain
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.
Office of Health Economics
Lotte Steuten, PhD, MSc
Office of Health Economics, London, LON, United Kingdom
Simon Brassel, MSc, Dipl.Ing
Caroline Bregman, PharmD, MSc
NICE, Manchester, LAN, United Kingdom
Avril Daly, BSc
Retina International, Zürich, Switzerland
Jennifer M Lee, MBA, BSc
Janssen-Cilag Denmark, Birkerod, 84, Denmark
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.
Bart Heeg, MSc, PhD
Cytel, Rotterdam, ZH, Netherlands
Anna Forsythe, PharmD, MSc, MBA
Cytel, Waltham, MA, USA
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, United Kingdom
Andre Verhoek, MSc