* Program subject to change
Level: Intermediate
Track: Health Policy & Regulatory
Afschin Gandjour, MD, PhD, MA, MBA
Frankfurt School of Finance & Management, Frankfurt, Germany
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Marlene Gyldmark, MPhil
Idorsia Pharmaceuticals, Allschwill, Switzerland
Mark Nuijten, MBA, PhD, MD
Ben Gurion University, Be'er Sheva, Israel
Fred W. Sorenson, MSc
Cencora, Basel, Switzerland
Level: Introductory
Track: Methodological & Statistical Research
Sven Klijn, MSc
Bristol Myers Squibb, Utrecht, ZH, Netherlands
William Rawlinson, MPhysPhil
Estima Scientific Ltd, London, United Kingdom
Tim Reason, MSc
Estima Scientific Ltd, South Ruislip, LON, United Kingdom
Track: Health Technology Assessment
William Canestaro, PhD, MSc
Washington Research Foundation, Seattle, WA, USA
Erik Landaas, PhD, MPH
W. L. Gore & Associates, Inc., Flagstaff, AZ, USA
Lotte Steuten, MSc, PhD
Office of Health Economics, London, LON, United Kingdom
Level: Experienced
Track: Real World Data & Information Systems
Felicitas Kuehne, MSc
UMIT - University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Innsbruck, Austria
Nicholas R Latimer, PhD, MSc
University of Sheffield & Delta Hat Limited, Sheffield, United Kingdom
Uwe Siebert, MD, MPH, MSc, ScD
UMIT TIROL - University for Health Sciences and Technology Hall in Tirol, Austria and Harvard Chan School of Public Health Harvard University, Boston, MA, USA
Ulka Campbell, PhD, MPH
Aetion, Inc, New York, NY, USA
Doug Foster, MBA
Advanced Data Sciences, San Francisco, CA, USA
Aaron Kamauu, MPH, MS, MD
Navidence LLC, Bountiful, UT, USA
Leanne Larson, MHA
ZS Associates, Wilmette, IL, USA
Track: Economic Evaluation
Gianluca Baio, PhD
University College London, London, United Kingdom
Rose Hart, PhD
Lumanity Inc., Sheffield, United Kingdom
Felicity Lamrock, PhD
Queens University Belfast, Belfast, ANT, United Kingdom
Howard Thom, MSc, PhD
University of Bristol, Bristol, United Kingdom
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Sebastian Schneeweiss, MD, ScD
Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA
Shirley Wang, PhD
Brigham & Women's Hospital, Harvard Medical School, Boston, MA, USA
Lorne Basskin, PharmD
Strategic Economics Ltd., Cary, NC, USA
Karen L Rascati, PhD, RPh
The University of Texas at Austin, Austin, TX, USA
Mondher Toumi, MD, PhD, MSc
Laboratoire de Santé Publique Aix-Marseille University, Marseille, France
Devin Incerti, PhD
EntityRisk, Inc., San Francisco, CA, USA
Jeroen Jansen, PhD
Department of Clinical Pharmacy, School of Pharmacy, University of California – San Francisco, USA; PrecisionHEOR, Oakland, CA, USA
Track: Study Approaches
Turgay Ayer, PhD
Georgia Institute of Technology, Atlanta, GA, USA
Jagpreet Chhatwal, PhD
Harvard Medical School, Boston, MA, USA
Xiaoyan Wang, PhD
Tulane Univeristy, New Orleans, LA, USA
Hua Xu, PhD
Yale University School of Medicine, New Haven, CT, USA
Josh Byrnes, PhD, MHEcon, BEcon
Griffith University, Brisbane, QLD, Australia
Hwee-Lin Wee, PhD
Saw Swee Hock School of Public Health, National University of Singapore, Singapore, Singapore
Track: Patient-Centered Research
Rebecca Crawford, MA
RTI Health Solutions, Manchester, United Kingdom
Lynda Doward, MRes
Arinesalingam Gnanasakthy, MSc, MBA
RTI Health Solutions, Succasunna, NJ, USA
Shanshan Qin, PhD
RTI Health Solutions, Durham, NC, USA
Nicholas J. Rockwood, PhD
RTI Health Solutions, Bend, OR, USA
Wei-Hsuan Jenny Lo-Ciganic, PhD, MSPharm, MS
University of Pittsburgh, Pittsburgh, PA, USA
William Padula, PhD, MSc, MS
University of Southern California, Los Angeles, CA, USA
Elisabeth Fenwick, PhD
OPEN Health Evidence & Access, Oxford, United Kingdom
Bristol-Myers Squibb, Utrecht, ZH, Netherlands
Claire Simons, PhD, MSc, MMATH
OPEN Health - HEOR and Market Access, York, United Kingdom
Digital Conference Pass
Yannis Natsis, MA, BA
European Social Insurance Platform (ESIP), Brussels, Belgium
Richard Bergstrom, .
IQVIA, Zug, Switzerland
Pedro Facon, .
National Institute for Health and Disability Insurance, Brussels, Belgium
Birgitte Klindt Poulsen, .
Danish Medicines Council, Copenhagen, Denmark
Valérie Paris, MSc
OECD, Paris, Ile-de-France, France
ISSUE: Global drug pricing stands as a critical and hotly debated issue in the healthcare industry; its complexity underscores the need for in-depth discussion and debate amongst key stakeholders, especially considering the evolving EU Joint Clinical Assessment (JCA) process and presence of Institute of Clinical and Economic Review (ICER). This issue panel aims to ignite a debate on how manufacturers can build informed pricing strategies, grounded in economically justified prices supported by economic models, robust willingness-to-pay research, and extensive analysis of the analogue landscape.
OVERVIEW:
Priti Jhingran, PhD
Genesis Research Group, Hoboken, NJ, USA
Christopher Blanchette, PhD, MBA
Novo Nordisk, Plainsboro, NJ, USA
Frank-Ulrich Fricke, PhD
Technische Hochschule Nürnberg, Nürnberg, Germany
Adam Weston, MSc
Genesis Research Group, London, England, United Kingdom
ISSUE:
Chris Skedgel, PhD
Office of Health Economics, London, United Kingdom
Saskia Knies, PhD
Zorginstituut Nederland, Diemen, Netherlands
Tara Lavelle, PhD
Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA
Becky Pennington, MSc
University of Sheffield, USA
ISSUE: Individual preferences with respect to the characteristics and possible outcomes of a healthcare intervention may vary. Some patients may have a preference for maximising the probability of response to treatment, whereas others may have a preference for avoiding certain adverse events. Some patients may have a preference to avoid injections, whereas others may wish to avoid frequent oral medication.
However, the standard decision rules for cost-effectiveness analysis identify a single technology as maximising expected health benefit. Decisions made on this basis may fail to recognise variation in preferences and circumstance and be economically inefficient failing to maximise patient utilities, compromise treatment adherence, and worsen health inequalities.
In this issue panel we will explore how heterogeneity in individual preferences might be investigated using qualitative and quantitative methods, including elicitation of personal utility functions, and accounted for in decision-making in terms of deliberation and ‘decision-rules’.
OVERVIEW: Ben Van Hout will discuss evolving methods to solicit more accurate estimates of utility weights using personal utility functions and their potential use in decision-making.
Neil Hawkins will moderate and will provide context by exploring how the ‘rules’ of cost-effectiveness analysis might be adapted to account for preference heterogeneity including the role of cost-minimisation, net-benefit, and ‘individualised cost-effectiveness” approaches.
Shelagh Szabo will describe methodologies that have been used to explore heterogeneity in patient preferences. These include qualitative methods using grounded theory and quantitative methods such as discrete choice experiments with latent class analysis.
Amanda Adler, as a clinician and an ex-chair of a NICE technology appraisal committee, will moderate and share her experiences in considering heterogeneity in preferences in real-world decision making. We will use real-time polling to allow the audience to express their own heterogeneity in preferences with respect to this topic and to allow the audience to act as a reimbursement ‘decision-maker’.
Amanda Adler, MD, PhD, FRCP
Churchill Hospital, Oxford, Oxford, United Kingdom
Neil Hawkins, MSc, PhD, MBA, CStat
University of Glasgow, Glasgow, United Kingdom
Shelagh Szabo, MSc
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Ben A Van Hout, PhD
University of Sheffield, Sheffield, United Kingdom
Sean Sullivan, BScPharm, MSc, PhD
CHOICE Institute, University of Washington, Seattle, WA, USA
Jens Grueger, PhD
Boston Consulting Group, Zürich, Zürich, Switzerland
Ansgar Hebborn, PhD
F. Hoffmann-La-Roche AG, Basel, Switzerland
Inmaculada Hernandez, PharmD, PhD
University of California San Diego, La Jolla, CA, USA
ISSUE: Creating educational content on the HTAR for patients while the implementation is still ongoing.
Maria Dutarte, MS
EUPATI, Utrecht, Netherlands
Lotte Klim, EUPATI Fellow
EUPATI, Copenhagen, 84, Denmark
Finn McCartney, BA, MA
EUPATI, Amsterdam , NH, Netherlands
Mitchell Silva, PhD
EUPATI Belgium vzw, Brussels, Belgium
PURPOSE:
DESCRIPTION:
Joe Hollinghurst, PhD, MSc, BSc
HEOR ltd, Cardiff, CRF, United Kingdom
Nia C Jenkins, MSc
Health Economics and Outcomes Research Ltd., Cardiff, United Kingdom
Alik Vodyanov, MSc
HEOR ltd, Cardiff, Cardiff, United Kingdom
Gemma Taylor, PhD
University of Bath, Bath, Avon, United Kingdom
Mitchell Higashi, PhD
ISPOR, Lawrenceville, NJ, USA
Rachael Fleurence, PhD
NIH, Washington, DC, USA
Ganhui Lan, PhD
Pfizer, New York, NY, USA
IMO Health, Rosemont, IL, USA
Sylwia Bujkiewicz, PhD, MSc
University of Leicester, Leicester, United Kingdom
Georgios Nikolaidis, PhD
IQVIA, Sheffield, United Kingdom
Anastasios Papanikos, PhD
GlaxoSmithKline, Stevenage, UK, United Kingdom
Janharpreet Singh, PhD
University of Leicester, Leicester, LCE, United Kingdom
Megan John, MBBS, BSc, MRCGP
National Health Service and Chair of NICE Appraisal Committee D, Frimley, Surrey, United Kingdom
Federico Felizzi, PhD
Menarini GmbH, Basel, BS, Switzerland
Nicholas Latimer, PhD, MSc
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Grace Hampson, MSc
Tom Denee, PharmD, MBA
Johnson & Johnson’ Innovative Medicine Europe, Zeist, Netherlands
Scott Ramsey, MD, PhD
Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Center, Seattle, WA, USA
David Sánchez González, BA
Retinosis FARPE, Madrid, Madrid, Spain
Hugo Pedder, PhD, MSc, BSc
University of Bristol, Bristol, BST, United Kingdom
Mark Harries, BPharm (Hons), MSc, MRPharmS
Ipsen Ltd, London, United Kingdom
University of California – San Francisco, San Francisco, CA, USA
Dawn Lee, MMath, MSc
University of Exeter, Exeter, United Kingdom
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Jag Chhatwal, PhD
Harvard Medical School and Value Analytics Labs, Wilmington, MA, USA
Diana Delnoij, PhD
Dutch National Health Care Institute, Diemen, Netherlands
Ipek Ozer Stillman, MS, MBA
Takeda Pharmaceuticals, Cambridge, MA, USA
Eleanor Perfetto, PhD, RPh, MS
University of Maryland School of Pharmacy, Baltimore, MD, USA
Michelle Campbell, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Gunnar Esiason, MBA, MPH
Boomer Esiason Foundation, New Canaan, CT, USA
Angela Rylands, PhD, CPsychol
Kyowa Kirin International plc, Marlow, BKM, United Kingdom
PURPOSE: To present the benefits and applications of integrating synthetic preference data into digital twins for enhancing personalized healthcare decision-making. Synthetic data may solve data scarcity, privacy concerns, and evidence transportability. Synthetic data generation methods promise to replicate real-world scenario complexity and representativeness. Researchers can simulate alternative treatment plans and explore resulting outcomes to optimize decision-making by enabling the creation of digital twins. We will explore the innovative use of synthetic data for real-time elicitation of patient preferences in a wide range of decision-making settings. The application of digital twins in the studies of patient behavior and statistical methods available to develop synthetic preference data will be presented.
DESCRIPTION: Workshop attendees will obtain a working knowledge of the fundamentals of digital twins and the application of synthetic preference data. The workshop will review a). the basics of synthetic data, b). methodological advancement needed to develop synthetic data for preference studies c). practical applications of synthetic preference data in the further development of personalized healthcare. Dr Mühlbacher will chair the session, introducing key definitions and contextualizing the applicability of synthetic data in the field of preference studies (10 min). Dr Manca will illustrate the causal inference underpinning the validity of the models that produce predictions to generate synthetic data and make generalizable statements (15 min). Dr Kolasa and Dr Zah will exemplify the benefits of incorporating synthetic patient preferences in personalized healthcare, enhancing medication adherence, and managing chronic diseases through personalized interventions (15 min). The highlight of the workshop is the audience participation, involving the identification of problems and solutions for a hypothetical case study in surgical planning and risk management (20 min). This interactive workshop will be valuable for researchers, clinicians, and industry analysts interested in integrating synthetic data with real-world data for a broader range of decision-making applications.
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, Germany
Katarzyna Kolasa, PhD
Óbuda University, Budapest, PE, Hungary
Andrea Manca, MSc, PhD
University of York, Heslington, York, North Yorkshire, United Kingdom
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Mario Ouwens, PhD
AstraZeneca, Mölndal, Västra Götaland, Sweden
Rob Abbott
Eberechukwu Onukwugha, BA, MSc, PhD
Brian O'Rourke, PhD
RCMS, Ottawa, ON, Canada
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
ISSUE: Global oncology spending is continuously rising. Simultaneously, drugs are increasingly approved based on less comprehensive evidence to ensure timely access, hampering assessments of their added benefit and often resulting in negative ratings. Concerns are growing regarding the financial strain on health systems and misalignment between incentives in the pharmaceutical market and patient interests, i.e. access to beneficial drugs for critical needs.
OVERVIEW: First, Francine Brinkhuis will present findings (~10 minutes) from a BMJ-published study*, co-authored by herself and Wim Goettsch, revealing that many EMA-approved oncology drugs (1995-2020) lack demonstrated added benefit, especially those approved via expedited pathways. These pathways aim to accelerate access to drugs for unmet medical needs but often lead to expensive drugs without substantial clinical benefits. Further, while revenues seemingly align with added benefit, most oncology drugs recover R&D costs within a few years. All this underscores the policy gap between drug approval/reimbursement and patient/societal benefit.
https://www.bmj.com/content/384/bmj.q511
Wim Goettsch, PhD
Utrecht University, Utrecht, Utrecht, Netherlands
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Uppsala, Sweden
Tina Taube, Master
EFPIA, Brussels, Belgium
Steffen Thirstrup, MD, PhD, CMO
European Medicines Agency, Amsterdam, Netherlands
ISSUE: As demands upon healthcare resources increase, informal caregiving has gained greater consideration in determining the impact of health conditions. However, not all informal caregiving is the same. Variation exists within and across health conditions. Three key areas of debate will be discussed. 1) conceptualizing informal caregiver HRQoL for use within economic models. 2) measurement of informal caregiver HRQoL. 3) valuation and incorporation of informal caregiver HRQoL in economic evaluations. This session will reflect upon lived experiences of being an informal caregiver of a person with a rare, progressive life-limiting condition, explore the differences of informal caregiving across the different groups, and consider how to account for these differences when selecting measures and creating economic models to ensure the correct values are captured, and reflect upon whether values attributed to informal caregiving should be the same across health conditions.
OVERVIEW: Fleur Chandler will first offer an overview of the lived experience and variability of being a caregiver (15 minutes), reflecting upon key domains of HRQoL affected. Jill Carlton will discuss the suitability of existing instruments to measure informal caregiver HRQoL, focusing upon content validity and acceptability. She will reflect upon whether societal views of informal caregiver HRQoL impact should differ according to issues such as rare conditions, and/or life-limiting conditions. Alice Biggane will discuss the need for wider conversation and collaboration between sponsors and payers to ensure alignment on how to capture caregiver HRQoL and the impact of potential treatments in health technology assessments and cost-effectiveness analyses, without leaving value behind (10 minutes). Alice Biggane, Jill Carlton and Fleur Chandler will be giving their thoughts on these areas from the different perspectives; industry, academia and from lived experience during a panel discussion lead by Sally Vincent. Sally will also facilitate questions from the audience (20 minutes).
Sally-Anne Vincent, BSc, MSc
Vitaccess, Reading, RDG, United Kingdom
Alice Biggane, MPH, PhD
Pfizer, Bath, SOM, United Kingdom
Jill Carlton, BMedSci, MMedSci, PhD
University of Sheffield, Sheffield, Yorkshire, United Kingdom
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain
ISSUE: Stakeholders from across the healthcare and life science spectrum are watching as the European Parliament and the Member State representatives in the Council debate the legislative proposal of the Commission to substantially review the EU’s legislative framework for pharmaceuticals. The common goal is to ensure better and faster access, availability and affordability of new medicines in all EU Member States. But how well are the needs of patients and the member states’ healthcare systems addressed? Will the proposals and counter-proposals really meet their needs?
Petra Wilson, PhD
Health Connect Partners, FTI Consulting, Brussels, Belgium
Virginie Bros-Facer, MSC PHD neuroscience
EURORDIS-Rare Diseases Europe, Paris, France
Johan Pontén, MA
Medicine Evaluation Committee (MEDEV), Stockholm, Sweden
Valentina Strammiello, MA, BA
European Patients’ Forum, Brussels, Brussels-Capital Region, Belgium
ISSUE: After decades of investment in the HEOR function, the biopharma industry appears to have entered a stage in which it is actively questioning what it has gotten in return. To date, little has been done to demonstrate the return on investment (ROI) of spending on HEOR personnel and research activities. This contrasts sharply with other components of biopharma spending, in which measurement of financial impact and calculation of ROI are routine. Do we need to demonstrate the ROI of HEOR? Can we?
OVERVIEW: Since individual health economists first started getting hired by industry in the 1980s, the HEOR function has grown to become a sizable component of the typical biopharma company org structure and annual budget. Core elements of HEOR—economic modeling, patient-reported outcomes, real-world evidence, and the like—have become fundamental for demonstrating product value to health system stakeholders. The beneficial impact of HEOR has largely been taken as a given rather than open to question, amid the common notion that HEOR activities and data are table stakes to successful launch and market uptake of biopharmaceutical products. This issue panel will consider the importance of demonstrating the ROI of HEOR and the difficulties in framing the calculation. Laura Pizzi, the panel moderator will outline key questions for the discussion, including: What is currently known about the ROI of HEOR? How can we formalize ROI calculations for HEOR work? Sissi Pham will provide an overview of current thinking within the biopharma industry on the role and value of the HEOR function. David Thompson will outline alternative approaches to ROI calculation and corresponding estimation challenges. Jens Grueger will provide a management consulting perspective on how HEOR and related functions can be championed within biopharma organizations. The moderator will solicit audience interaction and feedback on the alternative viewpoints.
Sissi V Pham, Pharm.D.
AESARA, Chapel Hill, NC, USA
David Thompson, PhD
Rubidoux Research LLC, Manchester, MA, USA
ISSUE: EU JCA aims at establishing a framework that promotes research, innovation, and more equitable access to products with large unmet medical needs. Process and timelines for submission and review have been recently finalized. While the 27 member states are still finalizing national processes and regulations, the fundamental question remains: is the designed process going to expedite patients’ access in EU?
OVERVIEW: provided by Cristina Masseria. Processes and timelines were designed to guarantee high quality and a voice for all member states, providing an opportunity for raising the EU perspective, maximizing synergies, and eliminating inequity across the EU. However, the maximum number of Population, Intervention, Comparison and Outcomes (PICOs) has not been set and will vary by therapeutic areas. The potential requirements of Indirect Treatment Comparison to meet all the PICO requirements may be substantial in terms of additional RCT statistical analyses and data gathering. The opportunity for the pharmaceutical company to engage with the EU is limited, with a very tight submission timeline. Companies are incentivized to initiate local reimbursement at the same time in all member states following EU JCA complete assessment, removing the possibility for accelerated submission. Moreover, there is risk of losing innovation funding for certain therapeutic areas with implications on pipeline selection.
Cristina Masseria, MSc, PhD
ISPOR, NEW YORK, NY, USA
Fabrizio Gianfrate, PhD, MSc
University of Ferrara, Ferrara, Italy
Francis Pang, MBA
Orchard Therapeutics Ltd, London, LON, United Kingdom
Gergana Zlateva, BA, MPA, PhD
Pfizer Inc., New York, NY, USA
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, LAN, United Kingdom
Matthew Dixon, PharmD, PhD
Bristol Myers Squibb, Lawrenceville, NJ, USA
Miguel Hernán, PhD, MD, MPH, ScM
Harvard University, Boston, MA, USA
Grace Hsu, MSc
Cytel Inc., Waltham, MA, USA
ISSUE: Wide variations in clinical practice and the availability of treatments across the EU will likely result in many PICOs being identified when scoping Joint Clinical Assessments (JCAs). This has been confirmed in multiple PICO simulation exercises including the ones undertaken in the EFPIA and Evidera report. Whilst good-quality direct comparative evidence is optimal, the likely number of PICOs mean this will not fully address the scope of many JCAs underscoring the need to understand the experience and current use of state-of-the-art evidence synthesis methods, and their strengths and limitations to facilitate decision-making in the context of JCAs.
OVERVIEW: EFPIA and Evidera conducted a simulation to explore the impact of the proposed EUnetHTA21 methodologies on several widely reimbursed oncology treatments. The simulation of the scoping process resulted in many potential PICOs for each product, ranging from 16 up to 57 depending on the indication and line of treatment.
Context is key to the use of different evidence synthesis methodologies, the simulation demonstrated the central role indirect treatment comparisons, real-world evidence, single-arm studies, and oncology-relevant endpoints play in country appraisals to help address uncertainty. The simulation found that a wide range of evidence synthesis approaches will be essential to conduct comprehensive JCAs and underlined the importance of considering the totality of evidence or endpoints available at the time of submission. With the publication of methodological guidelines over the next few months, it will be important for assessors and technology developers to understand how best to use state-of-the-art methodologies in JCAs and apply learnings from these previous experiences.
The issue panel will start with a short presentation highlighting the issues identified followed by a structured panel discussion with recommendations and audience questions. This panel will be beneficial to stakeholders involved in evidence generation, HTA, and JCA processes.
Martin Parkinson, MRPharmS
Evidera, part of PPD clinical research business, Thermo Fisher Scientific, London, London, United Kingdom
Anna Chaimani, PhD
Inserm, Université Paris Cité, Paris, France
Niklas Hedberg, MSc
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, AB, Sweden
James Ryan, MSc
AstraZeneca, Cambridge, CAM, United Kingdom
PURPOSE: This workshop aims to discuss best practices and challenges in expert elicitation for long-term survival estimates. We will present a comprehensive, step-by-step elicitation framework, providing attendees with essential practical knowledge of this method, its benefits, and implementation considerations. Perspectives from industry and health technology assessment (HTA) agencies will also be discussed.
Jessica Forsyth, PhD
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Min-Hua Jen, PhD
Eli Lilly and Company, Uxbridge, England, United Kingdom
Kate Ren, PhD
University of Sheffield|ConnectHEOR, Sheffield|London, England, United Kingdom
Richard Willke, PhD
Scintegral Health Economics, Chattangooga, TN, USA
Jason Shafrin, PhD
Lotte Steuten, PhD
Anne Willemsen, MSc
Dutch National Healthcare Institute, Diemen, Netherlands
Roisin Adams, MPharm, MSc., PhD
National Centre for Pharmacoeconomics, Dublin, Ireland & Discipline of Pharmacology and Therapeutics, Trinity College Dublin, Dublin, Ireland
Vanessa Schaub, PhD
F. Hoffmann-La Roche Ltd, Basel, Switzerland
Michal Stanak, Dr.phil, AKC
National Institute for Value and Technologies in Healthcare (NIH), Bratislava, Slovakia
Solange Corriol-Rohou, MD, PhD
Pharmaceutical Company: AstraZeneca, GMD, Paris, 75, France
Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency (EMA), Amsterdam, Netherlands
ISSUE: The European Union (EU) Health Technology Assessment Regulation (HTAR) will be applied from January 2025. From that date, all advanced therapy medicinal products and medical devices will have their relative clinical efficacy evaluated through a new EU Joint Clinical Assessment (JCA) pathway. Indirect treatment comparisons (ITCs) are expected to be frequently required to address the numerous PICOs (Population, Intervention, Comparator, Outcomes) requested in the JCA scope.
Sophia van Beekhuizen, MSc, MPhil
Cytel, Rotterdam, Netherlands
Mahmoud Hashim, PhD, MD
Johnson & Johnson, Rotterdam, Rotterdam, Netherlands
Iñaki Gutiérrez Ibarluzea, MSc, PhD
Fundación Vasca de Innovación e Investigación Sanitarias (BIOEF), Bilbao, Bizkaia, Spain
Amanda Cole, PhD
Claudio Jommi, M.Sc.
Università del Piemonte Orientale, Novara, Italy
Julien Patris, MA
argenx, Brussels, Belgium
Center for Health Decision Science and Dept. of Epidemiology and Health Policy & Management, Harvard T.H. Chan School of Public Health, Institute for Technology Assessment and Department of Radiology; Massachusetts General Hospital; Harvard Medical School, Boston, USA
Beate Jahn, Assoc.Prof. Dipl.Math. oec. Dr.rer.soc.oec
Institute of Public Health, Medical Decision Making and Health Technology Assessment; Department of Public Health, Health Services Research and Health Technology Assessment, UMIT TIROL – University for Health Sciences and Technology, Hall in Tyrol, Tyrol, Austria
Patrícia C. Soarez, MPH, PhD
University of Sao Paulo, Sao Paulo, Sao Paulo, Brazil
Michael Urban, Dr. rer. nat. MBA, MSc
MED-EL Hearing Technology GmbH, Innsbruck, Tyrol, Austria
Danny Yeh, PhD
AESARA, Burlingame, CA, USA
Fang-Ju Irene Lin, PhD
National Taiwan University, Taipei, Taiwan
David Shum, PharmD, MBA, MSc
Hoffmann-La Roche Canada, Mississauga, ON, Canada
Anouchka Cecilia Vidal, PharmD, MBA
F. Hoffmann-La Roche LTD, Basel, Switzerland
Meindert Boysen, PharmD, MSc
Health Technology Assessment International, Manchester, Manchester, United Kingdom
Elizabeth Kopits, PhD
US Environmental Protection Agency, Washington, DC, USA
PURPOSE: To educate health economics and outcomes research (HEOR) professionals on the Patient Experience Mapping Toolbox (PEMT), a set of publicly available resources to help researchers engage and document patients’ experiences, its real-world applications, and how it complements other patient initiatives.
DESCRIPTION: Patient experience data (PED) and patient engagement are critical to patient-centered outcomes in healthcare delivery research, drug development, and regulation. Meaningful patient engagement is bi-directional, and patients must be included as equal partners from the inception. Developed by patients, the PEMT can be used to collect disease-specific PED to inform a range of uses, including drug development and joint clinical assessments. To date, the PEMT has been applied to studies that ascertain patients’ treatment tradeoffs, as well as the experiences of diverse patients with food allergies and chronic kidney disease in the U.S. The use of this tool could be beneficial to broader global audiences. Through this workshop, HEOR-EU professionals will hear from patient engagement experts demonstrating applications of the PEMT and identify opportunities to enhance their patient-centered research methods.
Silke Schoch, MA
National Health Council, Washington, DC, USA
Derick Mitchell, PhD
IPPOSI, Dublin, Ireland
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Delta Hat Ltd, Nottingham, UK; University of Sheffield, Sheffield, Great Britain
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Adam Brooke, MSc
The National Institute for Health and Care Excellence (NICE), London, United Kingdom
Jen-Yu Chang, MSc
University of Sheffield, Sheffield, YOR, Great Britain
Shahid Hanif, PhD, MSc
GetReal Institute, Utrecht, Netherlands
Kapil M Khambholja, PhD
Catalyst Clinical Research, Thiruvananthapuram, Kerala, India
Jessica Santos, PhD, Ma, Bsc, CIPP
Oracle Life Sciences, Cambridge, Cambridgeshire, United Kingdom
Sebastian Heidenreich, PhD
Evidera, London, LON, United Kingdom
Divya Mohan, PhD
OPEN Health, London, London City, United Kingdom
Paul Schneider, MD, PhD, Msc
Valorem Health, Bochum, NRW, Germany
PURPOSE: Health Technology Assessment (HTA) bodies increasingly consider supplementary evidence from real-world data (RWD) alongside traditional data sources like RCTs. However there remain concerns about bias and the applicability of RWD from other countries to the local settings. Further complicated by the uneven availability of high-quality RWD across different geographical regions. This workshop will discuss how transportability analysis, like methods used to adjust for confounding factors, can help bridge this gap. Recently NICE updated their real-world evidence framework to include transportability methods as a potential approach to adjust for external validity concerns.
DESCRIPTION: Workshop attendees will hear from different stakeholders across the industry including decision makers on how transportability methods can address the lack of quality local data within an HTA submission. The workshop will a) present a transportability framework and methods, and current stakeholder guidance and b) address how transportability methods can be applied in practice. Philani Mpofu will open the workshop with a brief introduction on the concepts (5 min). Alexandra Starry will draw learnings from previous NICE and G-BA appraisals and introduce a transportability analysis framework (15 min). Marian Eberl will discuss challenges from the industry perspective to address requirements for local data and present a pilot transportability analysis in lung cancer using US and German data as an example explaining how the study was adapted to the previously outlined framework (15 min). Stephen J. Duffield will discuss key considerations for the acceptability of international data and transportability analysis approaches from a decision maker perspective (10 min). Then Philani Mpofu will lead the audience participation, by walking them through a hypothetical case study, asking them to identify differences that should be considered across a multi-country transportability project (15 min). This interactive and informative workshop will inform researchers and industry analysts on how to design their own transportability study.
Philani Mpofu, PhD
Marian Eberl, MSc
Daiichi Sankyo Europe, Munich, Bavaria, Germany
Alexandra Starry, MPP
Cytel, Berlin, Germany
Dalia Dawoud, PhD
National Institute of Health and Care Excellence, London, United Kingdom
National Institutes of Health, Bethesda, MD, USA
IMO health, Rosemont, IL, USA
Chris Cameron, MSc, PhD
EVERSANA, Sydney, NS, Canada
Shannon Cope, MSc
Precision AQ, Vancouver, BC, Canada
Justin Doan, MSc, MPH
Pfizer, Inc., New York, NY, USA
ISSUE: The role of HEOR and Market access is growing in importance. We are witnessing many reorganizations across pharma companies with the aim of better integrating access into the overall product strategy. Tatia Woodward and Indranil Bagchi, representing respectively global HEOR and Market Access, will discuss how these functions need to evolve and collaborate to create a holistic strategy that demonstrate the value of innovative products from pipeline until patent expiration. They will share their direct experience in terms of the impact and evolution of their roles, as well as perspectives on what more is needed to elevate the access function within the company. Brian O’Rourke will provide the HTA perspective in terms of evidence required for value demonstration and highlight how institutions like ISPOR can support this evolution.
OVERVIEW: We are witnessing a fundamental transformation of the global HTA landscape. Center for Medicare and Medicaid Services (CMS) is empowered to negotiate drug prices, the EU is creating a singular, comprehensive HTA process, Canada and the UK are updating their frameworks focusing on sustainability and innovation, and collaborations such as the Access Consortium are emerging in various markets. The need to demonstrate the value of new medicines, vaccines, and devices across the entire product lifecycle is becoming more urgent than ever. Real World Evidence (RWE) has emerged as a pillar to demonstrate effectiveness, long term safety, durability, and risk/benefit profile in populations not included in clinical trials, though acceptance of RWE across stakeholder groups remain variable. Value-based pricing is becoming a standard access strategy at launch, for expensive medications that obtain accelerated regulatory approval. The need to reconsider clinical trials program design, often designed just for fast regulatory approval, is forefront to allow collection of data also necessary to inform optimized access and pricing.
AESARA Inc., Madrid, M, Spain
Indranil Bagchi, PhD
GSK, Philadelphia, PA, USA
ISPOR, Lawrenceville, GA, USA
Tatia Chay Woodward, MPH, MSc
ISSUE: More clinical trials and real-world evidence studies are using digital endpoints, which are outcomes collected using digital tools such as wearables. These endpoints can provide useful information on how patients are functioning between clinic visits and do not rely on patient recall or opinion. There is little experience with them, however, so lack of validation and uncertainty over patient compliance mean they are not commonly considered by HTA bodies. This panel will discuss whether there are any scenarios where digital endpoints are ready to be considered in HTA, what the pros and cons are of using digital endpoints, and what researchers and HTA bodies need to do to allow digital endpoints to be used for decision making in the future.
OVERVIEW: Each panellist will have 10 minutes to outline their case. This will be followed by a debate amongst panellists, moderated by Jeanette Kusel. The audience will have the opportunity to pose questions to the panellists and polls will be used to elicit audience opinion. Dr Jacoline Bouvy will explain why HTA bodies are excited about the prospect of digital endpoint, but currently cautious about their use to inform decisions. She will discuss what is needed in terms of validation and links to health-related quality of life for HTA bodies to be more reassured of their use. Prof Lynn Rochester will highlight recent efforts that support cases where digital endpoints have been developed, validated and can be used in decision-making, highlighting the case study of the Mobilise-D initiative. Dr Kai Langel will highlight the work that is ongoing through the DEEP initiative to make digital endpoints fit for purpose for decision making. This panel will be of interest to all those involved in technology development and evaluation.
Kai Langel, BSc
Deep Measures, Helsinki, Helsinki, Finland
Lynn Rochester, PhD
Newcastle University, Newcastle, United Kingdom
Kate Hanman, BSc, MSc
Costello Medical, Boston, MA, USA
Annabel Griffiths, PhD
Formerly at Chronic Granulomatous Disease (CGD) Society, Dartford, Kent, United Kingdom
Mohit Jain, PhD, MBA
BioMarin Pharmaceutical, London, United Kingdom
Arjan Lankester, PhD, MD
Leiden University Medical Center, Leiden, South Holland, Netherlands
Min Huang, PhD
Merck & Co., Inc., Rahway, NJ, USA, Rahway, NJ, USA
Michael Drummond, PhD
University of York, Lichfield, United Kingdom
Guido España, PhD
Centers for Disease Control and Prevention, Atlanta, GA, USA
Rachel Oidtman, PhD
Malwina Holownia-Voloskova, MSc, PhD
Certara, Cracow, n/a, Poland
Gergo Meresz, Ph.D.
MediConcept Ltd., Budapest, PE, Hungary
Oresta Piniazhko, PhD
State Expert Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine, Kyiv, Ukraine
Eva Turk, PhD, MBA
Ministry of Health, Ljubljana, Slovenia
Jennifer Bright, MPA
International Consortium for Health Outcomes Measurement (ICHOM), Alexandria, VA, USA
Ian Patton, PhD
Obesity Action Canada, Quebec, QC, Canada
ISSUE: The pharmacoeconomic evidence provided by the BIA and CEA plays an important role in decision-making within healthcare systems. Despite these two instruments, systems do not always use both analyses or have different interpretations and criteria. To what extent this impacts the access to innovative medicines and how it can support their clinical assessment remains challenging. Efforts have been made to synergize the use of both BIA and CEA, for example, in a “three-dimensional pharmacoeconomic model” or “lock,” while some question the need for CEA. In the Netherlands, drugs or treatments with a high price or financial risk are placed in the lock, meaning the national authorities require both analyses for a decision. Are then BIA and CEA sufficient to address innovative medicines?
OVERVIEW: This panel will discuss the importance and the need to standardize the rules for a logical interplay between BIA and CEA. The moderator, Cornelis Boersma, will reflect on the situation (5min) and provoke debate based on several questions (15min): Is CEA always the appropriate instrument, and how far that depends on the BIA?; Could the development of a tool linking BIA and CEA support the decision-making of innovative drugs in the phase of their clinical assessment? How can industry, academia, and health authority contribute and benefit from such a tool? The panelists Josephine Mauskopf, Jaime Caro, Thea van Asselt, will address the perspectives mentioned in 10 min presentation per panelist. The audience will actively engage in the debate via online polls and open questions (10min).
Cornelis Boersma, PhD
University of Groningen, Department of Health Sciences, UMCG; Open University, Heerlen, Department of Management Sciences and Health-Ecore Ltd, Zeist, The Netherlands, Zeist, UT, Netherlands
J. Jaime Caro, MDCM, FACP, FRCPC
McGill University, London School of Economics, Evidera, Lincoln, MA, USA
Josephine Mauskopf, PhD, MHA, MA
RTI International, Research Triangle Park, NC, USA
Thea van Asselt, PhD
University Medical Center Groningen/ University of Groningen/ National Healthcare Institut in Netherlands/ The Netherlands Organisation for Health Research and Development, Groningen, GR, Netherlands
Thilo Schaufler, Ph.D.
CSL Vifor, Glattbrugg, ZH, Switzerland
Sue Harnan, MSc
The University of Sheffield, SHEFFIELD, United Kingdom
Helene Vioix, PharmD, MSc
Evidence and Value Development, Merck Healthcare KGaA, Darmstadt, Germany
Vicki Young, PhD
Cytel Statistical Services Ltd, London, LON, United Kingdom
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Rohini Sen, PhD
AbbVie, Arlington, MA, USA
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, LON, United Kingdom
Luis G. Hernandez, PhD, MPH, MSc
Takeda Pharmaceuticals America, Inc., Westford, MA, USA
Seamus Kent, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Varun Ektare, MPh
Indence Health, Mumbai, MH, India
Aikaterini Fameli, PhD, MBA
European Federation of Pharmaceutical Industries and Associations, LONDON, GLS, United Kingdom
Salah Ghabri, PhD, HDR
French National Authority for Health (HAS), Paris, 75, France
Florent Guelfucci, MSc, PhD
Syneos Health, RENNES, 35, France
Noemi Kreif, MA, MS, PhD
Imke Mayer Mayer, PhD
Owkin, PARIS, France
Juan García Burgos, MD
TLV, Tandvårds- och läkemedelsförmånsverket / Dental and Pharmaceutical Benefits Agency, Stockholm, Sweden
Francois Houyez, Patient Advocate
EURORDIS - Rare Disease Europe, Paris, France
OPEN Health Communications, London, LON, United Kingdom
Renske Ten Ham, PhD, PharmD, MSc
University Medical Center Utrecht, Utrecht, Utrecht, Netherlands
David Thomson, BSc(Hons), MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
Julie Roiz, MSc
Lumanity, London, DBY, United Kingdom
Maiwenn Al, PhD
Erasmus School of Health Policy & Management, Rotterdam, DBY, Netherlands
Miranda Cooper, MSc
AstraZeneca, Sheffield, DBY, United Kingdom
Tracy Westley, MScPH
Lumanity, Bethesda, MD, USA
Nan Qiao, PhD, MPH, MSc, MBBS
Merck, North Wales, PA, USA
Andrew Briggs, DPhil, MSc, MSc, BA
London School of Hygiene and Tropical Medicine, London, London, United Kingdom
Anke-Peggy Holtorf, PhD, MBA
PCIG @ HTAi.org, Basel, Basel-Stadt, Switzerland
OVERVIEW: Timeframe 45 min: moderator and panelists introducing their perspective in a motivational speech (5 minutes each), 15 minutes discussion on selected topics and 10 minutes on questions from auditorium
Stephanie Rosenfeld, Dr. med.
Bayer AG, Berlin, Germany
Michael Berntgen, MSc
Inka Heikkinen, MSc, MBA
Lundbeck Pharma, Copenhagen, Copenhagen, Denmark
Zorginstituut Nederland (ZIN), Diemen, Netherlands
ISSUE: JCA will roll out in 2025, starting with all new oncology products. This will expand to include products with orphan designation in 2028. Assessment of rare diseases will face additional challenges compared with non-orphan products in terms of clinical trial design, low patient numbers, heterogenous populations, and differing standards of care across the 27 European Union member states. It is important that companies start preparing now to ensure studies are designed appropriately and to understand the PICOs (population, intervention, comparator, and outcomes) that will apply. It may be appropriate to use pragmatic approaches to reduce the research questions to be appraised and innovative methodology to consider the available evidence, both from clinical trials and real-world data. Understanding the potential options to efficiently consider all relevant populations and to ensure that a voice is given to patients across these populations within rare diseases will be vital. All stakeholders will need to collaborate to balance challenges due to the constraints of the time and data available with the needs of patients.
OVERVIEW: This panel discussion will convene experts in rare diseases, patient advocacy, and statistics to share their insights and experiences regarding JCA requirements. Caroline Ling will moderate the session, introducing the topic and the panellists. Abigail Stevenson will explore the considerations and potential challenges associated with evidence generation across all 27 European Union member states for rare diseases. Emma Hawe will consider efficient approaches to evidence generation from the perspective of literature reviews and indirect comparisons where multiple PICOs exist. The patient advocate will articulate the significance of giving a voice to specific populations in rare diseases, emphasizing the urgency of access to new treatments and importance of patient-relevant outcomes. The panel presentations will span 45 minutes, followed by a 15-minute question-and-answer session, allowing the audience to engage with the panellists.
Caroline Ling, PhD
Emma Hawe, M.Sc.
Abigail Stevenson, DPhil
Chiesi, Manchester, LAN, United Kingdom
Tbc Tbc, TBC
TBC, TBC, LON, United Kingdom
PURPOSE: Equity continues to be a significant area of HEOR research. This workshop will describe what methods are available to address equity in health technology assessment (HTA) and discuss how and to what extent HTA agencies should consider equity. It will also explore the opportunities and challenges of incorporating health equity in HTA.
DESCRIPTION: Although there is an ever-growing body of research on equity, including methods such as distributional cost-effectiveness analysis (DCEA), the incorporation of equity in HTA is inconsistent and is not well defined. How HTA agencies consider equity is a shifting landscape, and is one that will likely evolve in the coming years.
In this workshop, Ms. Cresswell will introduce the topic of equity in HTA, including how NICE was created to end the ‘post-code lottery’ and its work to date around equity (10 minutes). Dr. Jansen (methodologist perspective) will introduce the concept of DCEA as a quantitative method to quantify the health equity impact of a technology using an illustrative model-based example, and will highlight potential implementation challenges (10 minutes). Dr. Whittington (HTA perspective) will discuss the practical and ethical challenges of conducting a DCEA within a health technology assessment, and will describe other non-DCEA opportunities to incorporate equity within HTA (10 minutes). Dr. Gsteiger (industry perspective) will explore the application of equity considerations and DCEA in clinical development programs, and will discuss current challenges (including collecting the data needed for DCEA), opportunities, and prerequisites for a more widespread use in practice (10 minutes). The workshop will allocate 20 minutes for an interactive audience discussion, including question and answer sessions. Audience polling will be employed throughout to encourage active participation and engagement. The workshop will culminate by asking the audience how they think equity should be considered in HTA.
Katharine Cresswell, BSc (Int), MPH
National Institute for Health and Care Excelllence, Manchester, LAN, United Kingdom
Sandro Gsteiger, PhD
F. Hoffmann-La Roche Ltd, Basel, BS, Switzerland
PURPOSE: ‘Value measurement: Assessing Value and QALY Alternatives’ remains a key trend in health economics and outcomes research. The goal of this workshop is to raise awareness of the EQ-HWB (EQ Health and Wellbeing instrument). The workshop will also explore the opportunities and strengths perceived by the speakers and audience on using the EQ-HWB-S, both from the perspective of health technology assessment (HTA) agencies and industry.
DESCRIPTION: The EQ-HWB has been designed as a standardised measure of broader aspects of quality of life related to health, social care and informal carers enabling assessment of interventions that are delivered in health, including public health, and social care. A short version, the EQ-HWB-S, can be used to generate QALYs for HTA. The instrument has experimental status which has enabled collaborators and researchers to assess its performance in diverse populations. ZIN, the HTA agency for the Netherlands, has named it as a potential measure for use when evaluating interventions in chronic conditions. Understanding the views of those who will use the tool and appraise it are important to consider.
Clara Mukuria, MA, MSc, PhD
University of Sheffield, Sheffield, South Yorkshire, United Kingdom
Jeremy Dietz, MSc
National Institute for Health and Care Excelllence, Bristol, BST, United Kingdom
Reva Efe, MD, MSc
Zorginstituut Nederland, Diemen, NH, Netherlands
Andrew Lloyd, DPhil
Acaster Lloyd, London, London, United Kingdom
Neil Grubert, MA
Independent, Basildon, United Kingdom
National Institute for Health and Care Excellence (NICE), London, United Kingdom
ISSUE: In our increasingly connected world, patients find information and support from their peers online, via social media and forums. These platforms provide a rich tapestry of lived patient experiences, capturing potentially valuable insights on topics such as disease burden, quality of life, and treatment outcomes. Until recently, the information within social media platforms remained under-utilised; however, capturing these data via social media listening (SML) has garnered attention as an assessable, low-burden, real-time source of real-world data (RWD). Historically, barriers to the wider adoption of SML have included lack of acceptance/guidance from key stakeholders, challenges with handling large unstructured data, and issues regarding bias, user privacy and content reliability. However, SML is now regaining attention with the advent of new evidence standard frameworks for digital health technologies and a growing interest in leveraging artificial intelligence to analyse big data, making it timely to re-examine its capacity in enhancing patient-centric real-world evidence. This session examines the promises and limitations of SML as a medium of RWD, sitting at the crossroads of many “hot topics” in the pharmaceutical industry.
OVERVIEW: Siddharth will open with a 5-minute balanced overview of SML as a medium of RWD. Next, Jackie will discuss the mechanics, value, and impact of SML and the use of AI to bring efficiencies to SML analysis. Following this, Mary Jo will examine challenges encountered in the use of AI from a clinical development/operations perspective and illustrate use cases of AI/SML informing adverse event reporting. Finally, Jeanette will provide a decision-maker’s perspective on the evidence standards that SML would need to meet to be useful for health technology assessment and acceptable use cases. Speakers are each allotted 10-minutes, followed by 20-minutes discussion time with audience participation encouraged throughout. Stakeholders from academia, industry and patient advisory groups would benefit from attending.
Siddharth Ramanan, PhD
Costello Medical, Cambridge, CAM, United Kingdom
Jackie Cuyvers, MSc
Convosphere, London, London, United Kingdom
Mary Jo Lamberti, PhD
Tufts University, Boston, MA, USA
ISSUE: The EC proposed pharmaceutical directive will require developers to be transparent regarding any direct financial support received from public bodies for R&D of medicines. This issue panel explores the implications and reactions of academia, industry, and payers. How should companies report the public funding received? How can payers use this information in HTA and price negotiations? How does it affect incentives to finance and conduct R&D?
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
David Mark Epstein, Phd
University of Granada, Granada, Spain
Carole Longson, MBE
Carole Longson Consultant, Manchester, United Kingdom
Mark Sculpher, PhD
University of York, York, United Kingdom
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Dan Ollendorf, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Beth Woods, BA, MSc
Nicholas Adlard, MA MSc MBA
Novartis Pharmaceuticals UK Ltd, Busserach, SO, Switzerland
Stefan Lange, MD
Dierks+Company, Berlin, Germany
Olivia Wu, PhD
PURPOSE: To test the feasibility of accounting for carbon emissions in HTA and go beyond theoretical considerations by showing and discussing worked examples for carbon footprint (CF) assessment inspired by methods used in clinical trial conduct.
DESCRIPTION: Previous discussions at ISPOR addressed the normative aspect of incorporating environmental impacts in HTA, but they stopped short of practical consideration of how this could be implemented. HEOR is not the only field grappling with methodological and data availability challenges related to assessing greenhouse gas (GHG) emissions, and we can learn from initiatives in other parts of the industry, such as clinical trial conduct. This session will focus on practical examples of methods to incorporation of carbon footprint assessment into HTA.
Ruth Chapman, PhD
Evidera Ltd, London, LON, United Kingdom
Michael J Cohen, MSc, MBA, SEA
PPD, Miami, FL, USA
Kinga Marczell, PhD
Evidera, London, United Kingdom
Harlan Campbell, PhD
University of British Columbia, Vancouver, BC, Canada
PURPOSE: To debate whether new and emerging policies and guidance on the use of real-world evidence (RWE), should be focusing on harmonisation between regulatory and Health Technology Assessment (HTA). Through interactive discussions and real-time polling, we seek to identify the benefits and opportunities of harmonisation, but also the challenges and hindrances this could pose in regard to future policy development.
DESCRIPTION: In recent years, there has been an increasing number of policies on the use of RWE within regulatory and HTA healthcare decision-making. As more policies emerge with differing methods and recommendations, it raises the question of whether there is a greater need for harmonization between policy for regulators and HTA? Differing organizational remits of regulators and HTA agencies will likely place some limits on harmonization, but to what extent should there be a drive to harmonize other elements including between medicines and MedTech? Does harmonization support future RWE policy development or will it hinder efforts to develop policy by encouraging guidance that is too broad and non-specific?
In this workshop, Rita Peeters will introduce the topic by discussing the evolution of RWE policies across regulatory/HTA organisations (10 minutes). Katharine Cresswell will discuss the context of the Innovative Health Initiative - Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IHI-IDERHA) project and what degree of harmonization was found in their recent global review of these policies (10 minutes). Patrice Verpillat will provide the regulatory perspective discussing perceived gaps and opportunities for future development and harmonization (10 minutes). Niklas Hedberg will discuss the HTA perspective and where harmonization may be challenging (10minutes).
A 20-minute interactive audience discussion, facilitated by real-time polling will allow discussion of critical arguments for and against harmonisation, and priority areas for future policy. Poll results will be displayed, allowing participants to see points of consensus and differentiation.
Rita Peeters, PhD, MBA
J&J MedTech, Antwerp, Flanders, Belgium
We, regulators, HTA agencies, payers and pharmaceutical industry, are all working closely with patients to ensure that innovative drugs can benefit patients as early as possible. This collaboration is guided by the ambition to enable and use the generation of reliable and valid evidence that will address and answer different questions, e.g. for benefit/risk assessment, for relative effectiveness assessment... We are relying on the totality of evidence generated, either from randomized controlled trials, or from other designs such as studies using real-world data. Even if differences in scope may exist, there are overlapping commonalities in what constitutes meaningful evidence generation. We also must deal with (remaining) uncertainties at different stages of decision making. How can we limit these uncertainties, and ensure that at the end of the overall approval process, all stakeholders get the needed evidence to take an informed decision and ensure an innovative drug reach the patients if appropriate?
Nikos Dedes, BA
Greek Patients' Association, Athens, Attika, Greece
Almath Spooner, .
Abbvie, Dublin, Ireland
