Sponsored by EORTC

Description:

HRQL research has been evolving, not only when it comes to the modes of administration or methods for ensuring validity, but also in how we approach the concept itself. In this plenary, we explored how the concept of HRQL has changed over the decades due to the emergence of parallel ideas and altered contextual conditions, from the initial conceptual framework that served for the development of foundational measures, to current programs of research and application, and HRQL’s possible future evolution.

Speakers:

Galina Velikova, MD PhD FRCP, University of Leeds, Leeds, United Kingdom

I am an academic Medical Oncologist at the University of Leeds/Leeds Teaching Hospitals, UK with a track record of patient-centred research using electronic Patient-Reported Outcome Measures in daily practice and clinical trials and lead collaborative research, both nationally and internationally. I led a NIHR Programme Grant for applied research on patient self-reported symptoms and toxicity using an online reporting system (QTool), uniquely integrated in electronic records, along re-designed care pathways for remote monitoring during cancer treatment to improve patient safety. Further research projects evaluate the use of the electronic system for remote monitoring and follow-up of cancer patients after treatment.

Amylou C. Dueck, PhD, Mayo Clinic, Scottsdale, Arizona, United States

Dr. Amylou Dueck is an Associate Professor of Biostatistics at Mayo Clinic in Arizona in the US. She is an expert in the statistical analysis of patient-reported outcomes (PROs) in cancer clinical trials and has contributed to the development of PRO measures, including the PRO-CTCAE and MPN-SAF. Dr. Dueck is the Co-chair of the Health Outcomes Committee of the Alliance for Clinical Trials in Oncology. In this role, Dr. Dueck partners with clinical investigators to integrate, monitor, analyze, and report PROs in multicenter cancer trials.

Kevin Weinfurt, PhD, Duke University School of Medicine, Durham, North Carolina, United States

Kevin Weinfurt, PhD is the James B. Duke Distinguished Professor at Duke University Medical Center. He is also a Professor of Psychology and Neuroscience, Psychiatry and Behavioral Sciences, and Biostatistics and Bioinformatics. Dr. Weinfurt co-directs the Center for Health Measurement in the Duke University School of Medicine. He recently completed 4 years working part-time for the FDA developing guidance for the Patient-Focused Drug Development initiative. He received his PhD in psychology from Georgetown University and did graduate work in the history of science and philosophy of mind at Linacre College, Oxford.

Moderator:

Mogens Groenvold, MD PhD, University of Copenhagen, Copenhagen, Denmark

Sponsored by Eli Lilly

Description:

The Cutting Edge Research plenary session featured some of the highest-ranked, innovative research from ISOQOL abstract submissions. In particular, these abstracts reflected research that truly “pushes the ISOQOL envelope” in providing new and different ways to look at quality of life.

Speakers:

Jinxiang (Fran) Hu, University of Kansas Medical Center, Kansas City, Kansas, United States

Explainable AI predicting Alzheimer’s Disease with Multimodal Deep Neural Networks

Jinxiang Hu is an Associate professor in the Department of Biostatistics & Data Science at the University of Kansas Medical Center. Having completed a Ph.D. degree at University of Florida in Psychometrics and a postdoctoral fellowship at National Institutes of Health, she is experienced in latent variable analysis/measurement models, and Machine Learning/Deep Learning. Jinxiang also collaborates extensively in researching clinical outcomes assessments, quality of life, cancer, Alzheimer’s Disease, pain studies, and cardiology.

Lindsey Murray, MPH PhD, Critical Path Institute, Tucson, Arizona, United States

Launch of the Rare Disease Clinical Outcome Assessment Resource, a Tool to Aid in COA-derived Endpoint Selection

Dr. Murray is the Executive Director of the Rare Disease Clinical Outcome Assessment (COA) Consortium at Critical Path Institute. Dr. Murray has nearly 20 years of experience in health outcomes research. She specializes in quantifying the patient’s perspective of health, illness, and treatment through COA development, psychometric testing, and the design and analysis of clinical trials involving COAs. For the last 5 years, her research has focused on identifying COAs that can be used across multiple rare disease clinical trial programs.

Angela Stover, PhD, University of North Carolina, Chapel Hill, North Carolina, United States

Pharmacist-facilitated PROM monitoring: reach, equity, and adoption of an EHR-integrated, telehealth PROM care model where patients do not need internet access

Dr. Angela Stover is an Associate Professor in the Department of Health Policy and Management at UNC Chapel Hill in the US. She also co-directs the Implementation Science Methods Unit for UNC’s Clinical and Translational Science Award from NIH. Her research program on patient-reported outcome measures (PROMs) and implementation science has spanned 20 years, including developing PROMs and helping clinics implement PROMs. Dr. Stover teaches courses on PROMs and implementation science. Dr. Stover has published more than 60 peer-reviewed articles, and her work has been cited more than 6,000 times.

Jin-ah Sim, PhD, Hallym University, Chuncheon-si, Gangwon-do, South Korea

Application of Generative Pre-trained Transformer (ChatGPT) to Analyze Qualitative Patient-Reported Outcomes Data for Cancer Survivors: A Cross-Validation Study

Dr. Jin-ah Sim is an Assistant Professor at the Department of AI Convergence specialized to Medical AI from Hallym University in Republic of Korea. She holds a Ph.D. in Biomedical Science from Seoul National University and specializes in patient-reported outcomes for cancer patients using advanced technologies such as natural language processing and machine learning. She had finished her post-doctoral fellowship at St. Jude Children’s Research Hospital and has received the New Investigator Outstanding Oral award from ISOQOL in 2020. Her recent work emphasizes developing AI methods to improve healthcare delivery for cancer survivors.

Moderator:

Andrea Pusic, MD MHS FACS FRCSC, Brigham and Women’s Hospital and Harvard Medical School, Boston, Massachusetts, United States

Sponsored by GSK

Description:

The digitization of healthcare, clinical workflows and outpatient monitoring brings with it multiple legal, ethical and regulatory challenges. Moreover, this changing landscape raises questions concerning the appropriate adoption and application of digital health measures, given that regulatory frameworks and acceptance of digital health measures vary widely across countries. There is a need to provide clarity to stakeholders from research and industry trying to navigate this complex regulatory landscape. This plenary brought together different perspectives and discussed some of the most debated issues and challenges in the adoption and use of digital health measures, with a focus on those collected in the context of clinical trials.

Speakers:

Bill Byrom, PhD, Signant Health, Nottingham, United Kingdom

Bill has worked in the Pharmaceutical industry for 30+ years, and is a recognized industry leader in eClinical product strategy and electronic clinical outcome assessments. He is extensively published in the field of COAs, and the author of two industry textbooks on ePRO. His current work with sensor data includes serving on the ISPOR task force on PerfOs, the C-Path working group on Chronic Heart Failure, and the Executive Advisory Board of the EU-funded Learning Network for Advanced Behavioral Data Analysis (LABDA) research program researching novel methods for advanced sensor-derived movement behavior data analysis. Bill is Senior Visiting Professor within Nottingham Trent University’s Medical Engineering Design Research Group.

Jessica Braid, MSc, Roche Products Ltd, Welwyn Garden City, United Kingdom

Jessica Braid has worked in patient-centered outcomes research for 10 years across a variety of therapeutic areas. Most recently, she has specialized in the development and validation of digitally-derived outcome measures and leads the Digital Innovation Chapter within the Patient-Centered Outcomes Research team at Roche.

Stefan Sauerland, MD MPH, Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany

Stefan Sauerland joined IQWiG (Institute for Quality and Efficiency in Healthcare) in 2010. Heading the Non-Drug Interventions Department, he oversees the scientific evaluation of non-drug therapies (including medical devices), diagnostic tests, and screening procedures. Within the German health care system, IQWiG’s health technology assessment (HTA) reports guide reimbursement decisions on a national level. Prof Sauerland has 12 years of experience in clinical research on surgical topics. He holds an M.D. from University of Cologne and an M.P.H. from Düsseldorf University. In 2019, he was granted an adjunct professorship by University of Cologne, where he teaches evidence-based medicine.

Moderator:

Megan Turner, GSK, Collegeville, Pennsylvania, United States

Description:

In recent years, health outcome assessment beyond patient-reported outcomes measures has become increasingly important and diversified. Besides different types of widely established clinical outcome assessments, some new developments emerged: family-reported outcome measures were introduced and digital health outcome measures are on the rise. This session took a closer look at these developments, reflecting on their implications, potentials and limitations for health outcome assessment as well as how these relate to almost existing approaches in terms of complementary insights and added value.

Speakers:

Sam S. Salek, PhD RPh FFPM FRPS, University of Hertfordshire, Gerrards Cross, United Kingdom

Sam Salek is Emeritus Professor of Pharmacoepidemiology in the School of Life and Medical Sciences, University of Hertfordshire, UK where he leads the Public Health & Patient Safety research group and is a visiting Professor at the State of Hessen, Germany. He is the co-founder and the past chair of the Patient Engagement Sig of the ISOQOL, co-chairs the European Hematology Association (EHA) Scientific Working Group for QoL and Symptoms. He has developed12 general and disease-specific patient-reported and family-reported outcome/HRQoL measures. He is a member of five Editorial boards and has published 19 books and over 750+ articles and abstracts.

Piper Fromy, PhD, SeeingTheta, Saumur, France

Piper has worked in the patient reported outcome field for around 8 years, and previously focused on assessing the measurement properties of instruments included in clinical trials. This ranges from patient reported outcomes to digital health technologies. She develops new methods to address the challenges that arise from increasingly complex data and associated questions. Across all her roles she specializes in the development and use of clinical outcomes assessments and digital health technology as applied to clinical trials and clinical practice.

Roya Sherafat, Mapi Research Trust, Boston, Massachusetts, United States

Dr. Roya Sherafat has over a decade experience in clinical research, specifically on long-term cardiometabolic outcomes, and heterogeneity of treatment effects in clinical trials. Subsequently, her research focused on evaluation of patient outcomes in large healthcare systems. Converging on intersection of Patient-Reported Outcome Measures and Value-Based HealthCare. This entailed conceptualization of quality of healthcare, patient-centric performance assessment, and benchmarking methodologies. Currently, Dr. Sherafat is the Scientific Director at Mapi Research Trust which has the mission of disseminating the science of Clinical Outcome Assessment and facilitating access to these instruments and to promote best practices in COA science.

Moderator:

Jan R. Boehnke, PhD, University of Dundee, Dundee, United Kingdom

Contributor:

Caroline Anfray, Lyon, France

Description:

Health literacy and quality of life have been assessed using different measurement models. How are these models useful for making equitable decisions about people’s health? Are the assumptions underpinning these models appropriate in different contexts? What should we be thinking about when we measure health literacy and/or quality of life? This roundtable discussion delved into the ways in which we conceptualise health literacy and quality of life and how this influences the ways in which we measure these concepts.

Host:

Melanie Hawkins, PhD
Swinburne University of Technology,
Hawthorn, Victoria, Australia

Description:

Although the use of patient-reported outcome and experience measures (PROMs/PREMs, respectively) is becoming more prevalent, they are typically absent in health professions education (i.e., degree, certificate, or training necessary to be licensed) or continuing education (i.e., learning to retain license or certification by a professional regulatory body). At this round table, participants were invited to explore opportunities within this area; share experiences, examples, and resources to design curricula; and discuss any questions you have. Note: This includes pre-licensure education for any health professionals such as nurses, doctors, physiotherapists, occupational therapists, social workers, dieticians, pharmacists etc.

Host:

Angela Wolff, PhD RN
Trinity Western University,
Langley, British Columbia, Canada

Description:

The flexibility afforded by PRO item libraries has helped to ensure that PROMs remain relevant for different populations, even as clinical approaches evolve. However, with this added flexibility comes the need to establish validity of customized PROMs, minimize the potential for bias in their design, and support their integration alongside standard PROMs. In this roundtable, participants were invited to share their experiences of using item libraries, including opportunities and challenges, and discuss methods to encourage best practices, highlighting areas for future work.

Host:

Claire Piccinin, MSc
EORTC,
Brussels, Belgium

Description:

In the context of translation and linguistic validation of clinical outcome assessments (COAs), concept definitions provide an important tool which can guide stakeholders in the process of ensuring COA translations are meaningful and as equivalent as possible to the source language. In this roundtable we discussed the Translation and Cultural Adaptation Special Interest Group’s (TCA-SIG) recommendations for the process of developing concept definitions for COAs, including what they consist of, when they should be developed, and who should be involved in the development process. Roundtable participants were invited to share their experience and perspectives on the topic.

Host:

Benjamin Arnold, MA
FACITtrans,
Florida, United States

Description:

Hosted by Sandra Nolte of the University of Melbourne, Australia, who has first-hand experience working for academia, the non-profit sector, PRO consulting and industry, participants of this roundtable discussed strategies of how to get a job in industry if currently working in academia and vice versa. In addition, both the opportunities and the limitations of working for each type of employer were reviewed critically.

Host:

Sandra Nolte, PhD
The University of Melbourne,
Melbourne, Victoria, Australia

​Description:

An opportunity to share experiences and discuss the unique challenges that arise in the conduct of qualitative research in pediatric populations. Who is best to include in such research: children themselves, parents/caregivers, clinicians, all of the above?  What concepts can you discuss with children and how can you best elicit qualitative data that you can interpret with confidence?

Hosts:

Amy M. Jones, MSc
Adelphi Values Ltd. – Patient Centered Outcomes,
Cheshire, United Kingdom

Adam Gater
Adelphi Values Ltd,
Bollington, United Kingdom

Moderator:

Karon Cook, PhD, Retired, Nacogdoches, Texas, United States

Symposium Synopsis:

In this symposium, we explained the use of score “vignettes” to communicate levels of symptoms and outcomes as measured by patient-reported outcome measures (PROMs). The first presentation laid the foundation for the other three by explaining the science and craft of creating score vignettes. Available tools for vignette item selection were demonstrated and important considerations were noted. The second presentation described how score vignettes were used to develop a tool for clinicians that helps them understand patients’ PROMIS fatigue and physical function scores. In the third study, score vignettes were used in four, independent, in-person bookmarking panels to assign interpretative labels to ranges of scores on PROMIS measures of sleep disturbance and pain interference. Variations among the panels were discussed. The fourth study used score vignettes in an Idioscale Judgment study with patients living with multiple sclerosis. This study derived thresholds for meaningful score change on PROMIS measures of fatigue and physical function based on levels of change participants endorsed as meaningful to them. Predictive modeling was used to define estimates.

Together, the presentations had three goals. The first was to educate attendees, didactically, on this novel approach for communicating PROM scores as “stories”. The second was to expand on this education by presenting three demonstrations of how score vignettes were used in three different methods and for three different purposes, i.e., clinical interpretation of scores, labeling ranges of scales with interpretable levels (e.g., none, mild, moderate, severe), and estimating the magnitude of score changes that patients perceive as meaningful to their everyday lives. The final goal was to provide attendees with a base of understanding of this approach so that they are equipped for their own explorations and expansions of methods for interpreting PROM scores.

Individual Presentations:

Writing PROM Score Stories: Concepts, Applications, and future directions.

Karon Cook, PhD, Retired, Nacogdoches, Texas, United States

Development of a novel story-based score interpretation tool for PROMIS Fatigue and Physical Function scores

Dagmar Amtmann, PhD, University of Washington, Seattle, Washington, United States

Bookmarking panels with people living with chronic pain to interpret PROMIS pain interference and sleep disturbance scores

Nan Rothrock, PhD, Northwestern University, Chicago, Illinois, United States

An evaluation of meaningful within-person change for PROMIS fatigue and physical function scores among people with MS: an idioscale judgement study

Paul Kamudoni, MSc PhD GFMD, Merck Healthcare KGaA, Darmstadt, Germany

Moderator:

Lotte Haverman, PhD, Emma Children’s Hospital, Amsterdam UMC, Amsterdam, Netherlands

Symposium Synopsis:

Historically, Patient-Reported Outcome Measures (PROMs) and Shared Decision Making (SDM) were often viewed as separate (research) domains. PROMs are designed to measure outcomes that hold significance for patients, while SDM focusses on engaging patients in decision-making processes concerning their care. Nevertheless, there is a growing acknowledgment that these two approaches can complement and mutually reinforce one another.

Yet, there remains an uncertainty on how to integrate these domains both theoretically and practically. Although PROMs and SDM have the potential to facilitate a more comprehensive and patient-centered approach to healthcare, the specifics of their integration are not clear. This symposium aimed to address this gap by exploring how and why to effectively integrate both worlds.

The following topics were presented during this symposium:

1. The Amsterdam PROM Implementation Strategy: Policy and Pathway: An example of implementing PROMs in daily clinical care, with a focus on barriers in their actual use in communication between healthcare providers and patients.
2. Shared Decision Making: an inclusive model for practice : An introduction to SDM and SDM models, and a description of the development of a new, inclusive SDM model for use in clinical practice.
3. Shared Decision Making and PROMs: Start with WHY: An attempt to conceptualize the similarities and challenges involved in integrating SDM and PROMs.
4. How to Use PROMs for Shared Decision-Making: A Conceptual Framework: Utilizing mixed-methods, we made an effort to develop a conceptual framework illustrating the synergy between PROMs and SDM, showcasing their collaborative utilization.

The conclusion of the symposium featured a panel discussion with all presenters, providing an opportunity for questions from the audience.

Individual Presentations:

The Amsterdam PROM implementation strategy: policy and pathway

Maud van Muilekom, PhD, Emma Children’s Hospital, Amsterdam UMC, Amsterdam, Netherlands

Shared Decision Making: an inclusive model for practice

Olga Damman, Amsterdam UMC, Amsterdam, Netherlands

Shared Decision Making and PROMs: Start with WHY

Anna (Sandra) Beurskens, Maastricht University, Maastricht, Netherlands

How to use PROMs for Shared Decision-Making: a conceptual framework

Anouk Groenewegen, MSc, Department of Child and Adolescent Psychiatry and Psychosocial Care, Amsterdam UMC, University of Amsterdam, Emma Children’s Hospital, Amsterdam, the Netherlands

Sponsored by RWS

Moderator:

Huda Shalhoub, PhD Psychology, Bayer, Berlin, Germany

Symposium Synopsis:

The field of health outcomes is increasingly standardizing ‘de novo’ clinical outcome assessments (COAs) to be created electronically or expecting old COAs to be migrated to electronic formats (e.g., mobile phones, tablets, and so forth.). In this digital age, it has additionally become more important than ever to be globally inclusive in clinical research. Focusing only on English language and English-speaking COAs is no longer adequate. In light of these trends, translating and culturally adapting electronic COAs concurrently can bring forth a multitude of advantages. It can create process efficiencies that can improve timelines, lead to higher data quality and result in higher scientific integrity.

The moderator initiated by introducing this topic to the audience and laying the rationale of the findings and the methodology used. A roundtable discussion led by two of the co-authors further illuminated the audience on practical and scientific approaches on concurrent implementation of eCOAs and translations. The symposium discussions were based on a TCA-SIG collaborative workgroup effort providing best practices on this topic. The symposium was of special interest to eCOA and translation vendors, COA developers and copyright holders, patient-centered scientific experts, regulators, academics and the pharmaceutical industry.

The symposium findings are planned to be submitted and published in the Journal of Patient Reported Outcomes (JPRO) in 2024. The discussions concluded with an opportunity for the audience to ask any questions to the moderator and presenters.

Individual Presentations:

Addressing the concerns of instrument developers and copyright holders regarding electronic implementation of their measures

Sonya Eremenco, MA, Critical Path Institute, Tucson, Arizona, United States

The Two-Step Dance of Concurrent Translation and Implementation of eCOAs

Megan Turner, BA, GSK, Collegeville, Pennsylvania, United States

Moderator and Discussant:

Leah McClimans, PhD, University of South Carolina, Columbia, South Carolina, United States

Symposium Synopsis:

Patient-reported measurement is the idea that patient perspectives should play an evidentiary role in determining how effective a drug is taken to be, the degree to which a hospital provides good quality care or improvements in patient-clinician communication. This idea may sound prosaic, but in fact it’s nothing short of revolutionary. It says, patient views matter-not as an afterthought, and not only at the bedside, but in the nuts and bolts of creating our evidence base, and thus in health-care decision-making. But patient-reported measures present a puzzle: How can measurement, which relies on standardization, represent patient perspectives, which, if not idiosyncratic are at least various and changeable? This tension is explored in Patient-Centered Measurement (McClimans, L 2024, Oxford University Press) a recent book that combines philosophy and conceptual questions from HRQoL research. This symposium brought “all different together” four HRQoL researchers and four philosophers into dialogue with one another as they discuss their different perspectives on four chapters of this book.

The first chapter considers this tension from a HRQoL perspective. Some HRQoL researchers have argued that QoL research needs better theories of what we measure. This chapter argues that instead of better theories, we need better recognition of the values and uncertainty that are inherent to HRQoL research. The third and fourth chapters develop a theory of patient-reported measures that aim to resolve this tension. This theory is not a theory of HRQoL constructs but rather a theory about how patients, people with disabilities and other ill persons’ contributions should shape our understanding of HRQoL constructs. The final chapter considered in this symposium looks at a possible criticism of this theory. Patient-Centered Measurement champions the use of patient perspectives in defining HRQoL constructs to ensure these constructs are sensitive to patient needs. But what about pharmaceutical companies that use patient representatives to further their own economic needs? Do we need to be worried?

This symposium thus created a multi-directional dialogue: between HRQoL researchers and this text, between HRQoL researchers and other philosophers, and between the HRQoL community and philosophical concepts.

Individual Presentations:

The philosophy and practice of patient-centered measurement: An interdisciplinary discussion

Melanie Hawkins, PhD, Swinburne University of Technology, Melbourne, Australia
Jan R. Boehnke, PhD, School of Health Sciences, University of Dundee, Dundee, United Kingdom
Richard Sawatzky, PhD RN, Trinity Western University, Langley, British Columbia, Canada
Kevin Weinfurt, PhD, Duke University School of Medicine, Durham, North Carolina, United States
Alessandra Basso, PhD, Department of History and Philosophy of Science, University of Cambridge, Cambridge, United Kingdom
Sebastian Rodriguez Duque, PhD Candidate, McGill University, Montreal, Quebec, Canada
Rebecca L. Jackson, PhD, Indiana University—Bloomington, Bloomington, Indiana, United States
Leah McClimans, PhD, University of South Carolina, Columbia, South Carolina, United States

Moderator and Discussant:

Calvin Ho, PhD, AstraZeneca, Gaithersburg, Maryland, United States

Symposium Synopsis:

Patient-reported symptom and quality of life data are increasingly central to the evaluation of drugs in major markets. While PROs and other patient experience data were initially included in regulators’ assessments of drug efficacy and safety, they are also now increasingly central to health technology assessment (HTA). Patient-reported data can also be valuable for individual healthcare providers and patients in making treatment decisions.

In this symposium, jointly organized by the Regulatory and Health Technology Assessment Engagement SIG and the Industry SIG, panelists with varied experiences across academia, HTA bodies, and clinical practice discussed how patient experience data are being used after drugs receive regulatory approval, and opportunities for future applications given the evolving landscape.

The symposium was primarily discussion-based. The four panelists gave 10-minute presentations on their areas of expertise, including global trends in HTA, specific perspectives of reviewing agencies, and use of patient-reported data in clinical practice. The moderator provided 5 minutes of remarks, then led 30 minutes of discussion with the panelists and audience.

This topic is of great interest to ISOQOL’s academic, industry, and patient partner members, and is especially important in this year’s host country of Germany. The Institute for Quality and Efficiency in Health Care (IQWiG) is a leader in the inclusion of patient-reported outcomes in HTA. We expect that the symposium will attract many attendees from Germany and other European countries where HTA bodies have taken varied approaches to including patient experience data in their evaluations.

Individual Presentations:

Value of Patient Experience Data to Health Technology Assessment Agencies and Clinical Practitioners

Lynda Doward, MRes, RTI Health Solutions, Didsbury, Manchester, United Kingdom

Patient-reported Outcomes Data in Health Technology Assessment: Use in Germany and Beyond

Beate Wieseler, Dr. rer. nat., Institute for Quality and Efficiency in Health Care (IQWIG), Cologne, Germany

Viewpoint and expectations of the French HTA Agency regarding Patient Experience Data

Olivier Chassany, MD PhD, Université Paris Cité, Paris, France

Development and feasibility of the self-report quantified tuberous sclerosis complex-associated neuropsychiatric disorders checklist (TAND-SQ) and the TAND Toolkit App

Anna C. Jansen, MD PhD, Antwerp University Hospital, Belgium

Moderator and Discussant:

Andrew Trigg, MSc, Bayer plc, Reading, United Kingdom

Symposium Synopsis:

Patient-reported outcome (PRO) measures commonly inform judgements on the relative impacts of different interventions on health outcomes in randomised clinical trials. Determining if group-level differences in health outcomes, the cornerstone of clinical trials, are meaningful has been neglected in favour of determining if individuals have experienced a meaningful change. However, the interpretation of these group-level differences is critical, as it governs our access to potentially life-changing medicines. In this symposium, we critically evaluate this issue, exploring questions such as: ‘What is a relevant effect?’, ‘Who should judge this?’ and ‘Is there a one-size-fits-all approach?’.

We have convened a panel of measurement experts, with experience in academia, instrument development, and pharmaceutical research, to present their recent research and views.

The first presentation focused on distinctions between interpreting scores for an individual versus a group, challenging the view that such interpretations are interchangeable. The need for interpretative guidelines around group differences, providing an enriched context of understanding beyond statistical significance, is also justified. Consideration of the comparator group, measurement error, and the ‘number needed to treat’ was discussed.

The second presentation presented recent work to conceptualize what properties a threshold for meaningful between-group differences should possess. Detailed simulations explore illustrative contrasting viewpoints, raising issues of whether such thresholds can in fact be estimated purely through empirical methods.

The third presentation concerned eliciting expert opinion to help interpret the magnitude of between-group differences, where the experts could be clinicians or patients. This is mainly discussed in the context of research to establish group-level thresholds for two widely used cancer PRO measures. The research piloted cognitive interviews with patients and compared those results to results from interviews conducted with clinicians familiar with the PRO scores and published literature.

The fourth presentation discussed the use of anchors other than those based on patient or clinician judgement, to contextualise group-level differences. This is drawn from many years of research supporting a well-established PRO measure across a variety of populations.

Time was set aside for a thorough discussion of the issues raised with audience members, aiming to encourage active debate on this important topic.

Individual Presentations:

Differentiating group-level and individual thresholds used for interpreting the meaningfulness of COA scores

Cheryl Coon, PhD, Critical Path Institute, Tucson, Arizona, United States

Conceptualizing what it means for groups to meaningfully differ by exploring quantitative definitions of between-group thresholds

Nicolai D. Ayasse, PhD, Critical Path Institute, Tucson, Arizona, United States

Exploring the size of between-group score differences with patients and clinicians.

Kim Cocks, PhD, Adelphi Values, Cheshire, United Kingdom

Developing benchmarks to help interpretation of group-level differences in patient-reported outcomes

Jakob Bjorner, MD PhD, QualityMetric, Copenhagen, Denmark

Moderator:

Julia Garcia, PhD MS, AbbVie, Irvine, California, United States

Symposium Synopsis:

A sensor-based outcome (SBO) is derived from a digital device or platform that includes a sensor to measure a patient’s lived experience continuously through real-time data collection. With appropriate interpretation, SBOs that measure how patients feel, function, or survive may be considered novel types of clinical outcome assessments (COAs). Key examples are SBOs that measure a patient’s physical activity or sleep (e.g., wearable accelerometers, under-mattress pressure mats monitoring sleep). SBOs offer a means to gather high-quality clinical data efficiently and directly from patients in real-world settings. These devices have the potential to enhance our understanding of patient’s experiences with a disease or condition and increase patient centricity in medical product development to improve health outcomes. However, aligning SBO development with current patient-focused drug development (PFDD) guidance focused on traditional COA development presents unique regulatory challenges. Researchers beginning development of a SBO face challenges of identifying how the device will fit into the traditional COA development process and how and when to engage patients. Helpful frameworks have been developed by several organizations (C-Path, DiMe, Transcelerate) to guide development and adoption of SBOs, and guidance on digital health technologies exists from FDA and EMA, but there is an opportunity to integrate best practices for development of COAs for SBOs. With these principles in mind, a new approach developed by COA and Digital Health experts will be discussed that further refines and expands on existing frameworks to better align SBO development with traditional patient-centered COA development and current PFDD guidance, with an emphasis on opportunities for patient engagement. In Part 1 of this symposium, regulatory challenge areas were explored including identifying concepts that may be beneficial to measure with an SBO, identifying an SBO to measure concepts of interest for context of use, confirming measures capture meaningful aspects of health, and identifying thresholds for meaningful change. In Part 2, the framework was described with real-world examples outlining steps in the framework, best practices, and considerations for how researchers can best navigate the challenge areas. Attendees took part in interactive polling and shared their thoughts on challenges and opportunities.

Individual Presentations:

Developing Sensor-Based Outcome Measures to Enhance Understanding of the Patient Experience in Medical Product Development

Elektra Papadopoulos, AbbVie, Gaithersburg, Maryland, United States

A framework for sensor-based outcome measure development

Bill Byrom, PhD, Signant Health, Nottingham, United Kingdom

Moderator:

Michelle Carty, PhD, QualityMetric an IQVIA business, Johnston, Rhode Island, United States

Discussants:

Dorothee Oberdhan, Otsuka, Germany
Holly Krasa, MSc, Blue Persimmon Group, Washington, DC, United States

Symposium Synopsis:

As patient-reported outcome (PRO) diaries become increasingly popular in clinical trials for medical product development, an examination of the benefits and challenges associated with their use is needed. This symposium used case studies and a review of label claims to illustrate key considerations, decisions, and challenges related to diary development, testing and validation, and implementation when using daily, event-based, episodic, and hybrid PRO diaries in clinical trials. This adds to research presented at a 2022 ISOQOL symposium that examined methodological issues related to the use of daily diary measures. Speaker 1 described the development of a hybrid daily and event-based urination diary, focusing on design decisions that balanced the needs of researchers and the unique experiences and preferences of users. Speaker 2 presented challenges encountered in implementing a binge eating diary in a clinical trial, focusing on electronic PRO operationalization and the importance of site instruction and monitoring. Speaker 3 used an example from a sickle cell pain crisis diary to present an approach to overcoming challenges associated with complex episodic diaries, focusing on the use of multi-level factor analysis to test the diary’s hypothesized measurement model. Speaker 4 reviewed examples of patient-reported diaries that were used to support endpoints (and thus label claims) in clinical trials, identifying key trends, approaches, and decisions to help inform future use of diary-based trial endpoints. The discussants summarized and expanded upon the presentations, suggesting areas for future research. Time was allowed for questions and discussion.

Individual Presentations:

Designing a hybrid daily and event-based urination diary: Lessons learned from a content validity and feasibility/usability study

Meaghan O’Connor, MTS MPH, QualityMetric, Inc., Johnston, Rhode Island, United States

Mitigating challenges of daily diary implementation in a clinical trial

Jessica T. Markowitz, PhD, Blue Persimmon Group, Arlington, Massachusetts, United States

Using multi-level modeling to test measurement models for complex episodic diaries with nested data

Avery Rizio, PhD, QualityMetric Incorporated, LLC, Johnston, Rhode Island, United States

FDA labeling based on patient-reported outcome diaries

Michelle Carty, PhD, QualityMetric an IQVIA business, Johnston, Rhode Island, United States

Moderator:

Adam Gater, Adelphi Values Ltd, Bollington, United Kingdom

Symposium Synopsis:

Throughout their lifetime, women may require medical support and intervention simply due to natural life events. From navigating menstrual health, contraception, infertility, pregnancy, childbirth, postpartum health, HPV vaccines, cervical screenings, mammograms and the menopause, women are likely to experience medical challenges without ever being diagnosed with anything other than being a woman. All of these life events are associated with physical symptoms and can have a huge spectrum of short and long-term HRQoL impacts. The symptom experience during these life events varies significantly between women and can also vary for an individual across time, making it difficult to manage both immediate symptoms and long-term wellbeing. As women experience many of these symptoms on a cyclical basis, the symptoms may present less as a medical problem and can be seen as a routine part of life. As a result, women and health care professionals may attribute these symptoms and HRQoL impacts to ‘being a woman’ and they may not be managed in the same way as other ‘patients’. The lack of diagnosis and variation in the way women engage with medical support for these events are important factors to consider when conducting women’s health research. Researchers must adapt their style to engage with women and when talking about these health conditions. A more ‘person-centred’ approach to understanding the experiences, perspectives and needs of women could support research, diagnosis and treatment and research would help to ensure women are heard and managed as people and not patients. Using case studies from a range of conditions, this symposium provided a multi-stakeholder perspective on person-centered approaches to women’s health research. The first presentation outlined methodological considerations for optimising research from a researcher perspective. In the second presentation, person-centered research to inform trial design and selection of clinically meaningful endpoints was presented. The third presentation provided an industry perspective regarding experiences and unique considerations when developing clinical outcome assessment strategies for women’s health conditions. Finally, the fourth presentation discussed person-centered approaches to research from a patient advocacy perspective. Individual presentations were followed by an open panel discussion and audience Q&A.

Individual Presentations:

Methodological considerations for conducting meaningful and impactful research in women’s health

Laura Grant, Adelphi Values Ltd, Bollington, United Kingdom

Advancing the Therapeutic Landscape for Spontaneous Preterm Labor through an Understanding of the Maternal and Neonatal Experience

Elizabeth Gargon, PhD, Patient-Centered Outcomes, Adelphi Values Ltd, Bollington, United Kingdom

Patient-focussed drug development in women’s health: industry case examples from endometriosis, uterine fibroids and menopause

Claudia Haberland, PhD MSc, Bayer AG, Berlin, Germany

Person-centered approach to navigating the menopause: patient advocate perspective

Malar Subramaniam, BAYER AG, Berlin, Germany

Moderators:

Madeline Pe, PhD, EORTC Quality of Life Department, Brussels, Belgium
Ashley Wilder Smith, PhD MPH, National Cancer Institute, Bethesda, Maryland, United States

Discussant:

Claire Snyder, PhD, John Hopkins University, Baltimore, Maryland, United States

Symposium Synopsis:

While there has been long-standing recognition of the need to capture patient-reported outcomes (PROs) from cancer patients in clinical trials, there has also been an aspirational goal to include PROs to monitor health and inform care in clinical practice. More recently, there has been a greater emphasis on the need to understand the impact of cancer treatments and disease effects in real-world conditions, and to create an evidence base that supports the use of PROs (such as symptoms, functioning and general quality of life) in applied cancer care delivery. There are multiple challenges when including PROs in such settings, such as what, when, and how to capture and integrate PROs in practice to best understand patient health and inform clinical decisions. Addressing methodological challenges is critical to ensure they can provide information on the real-world impact of cancer and related therapies on PROs. Since these questions are currently relevant in the European Union (EU) and United States (US), the proposed symposium aimed to bring together researchers from the EU and the US to talk about their experiences integrating PROs in cancer care delivery. The goal was to understand how hospitals and clinics have integrated PROs in clinical practice, the challenges they have encountered and what their plans are to successfully implement PROs. In this symposium, we provided an overview of the need for PRO implementation to manage cancer and its consequences in Europe and the US as well as methodological considerations unique to deployment of PROs in clinical practice. We then provided two examples from the EU, one focused on PRO collection in a large Austrian hospital and one focused on PRO actionability in cancer care in the Netherlands. Finally, we provided an example of the adaptation needed to deploy PROs in low resourced cancer care settings in Florida. These presentations were followed by a discussion of best practices for PRO integration and how to address challenges raised to improve cancer outcomes. This supported shared knowledge to advance the field and to continue to promote the relevance and utility of collecting PROs in clinical practice.

Individual Presentations:

Implementing PROMs in routine cancer clinical practice: lessons learned from the Medical University of Innsbruck, Austria.

Bernhard Holzner, Prof.Dr., Medical University of Innsbruck, Innsbruck, Austria

PROMS in clinical practice: How to make them actionable for the individual patient?

Lonneke van de Poll-Franse, PhD, Netherlands Cancer Institute, Amsterdam, Netherlands

Implementation of Patient Reported Outcomes in Oncology Care: Adaptation for Low Resource Settings in the United States

Frank Penedo, PhD, University of Miami, Miami, Florida, United States

Moderator:

Kathleen Wyrwich, PhD, United States

Speakers:

Natalie Aldhouse, MSc, Clarivate, United Kingdom
Milena Anatchkova, PhD, Evidera, United States
Ana Maria Rodriguez-Leboeuf, PhD, IQVIA, Canada
Tom Willgoss, PhD, Roche, United Kingdom

The Food and Drug Administration (FDA) Patient-Focused Drug Development Draft Guidance 3 (FDA, 2022) recommends that sponsors provide rationale for the selection of the type of clinical outcome assessments (COAs) and recommends the creation of a conceptual framework that includes the conceptual model and the measurement model. This Guidance provides illustrations of COA conceptual frameworks; however, in-depth discussion for the creation and justification of these frameworks is limited. The ISOQOL Industry SIG has explored best practices and practical recommendations to guide the development of conceptual frameworks for regulatory purposes. This Symposium presented working groups’ recommendations for these best practices with four focused presentations, followed by attendees’ input and discussion.

1. How far do we go on conceptual model detail?
2. How do we learn from past models of health and disease experience?
3. Which type of COA?
4. How to fund and collaborate to create the best conceptual frameworks?

Moderator:

Scottie Kern, BSc. (Hons), Critical Path Institute, United States

Discussants:

Bryan McDowell, MSc MBA, Clario, Switzerland
Florence Mowlem, PhD, uMotif, United Kingdom
Alisa Heinzman, MFA, RWS Regulated Industries, United States
Estelle Haenel, PharmD PhD, Kayentis, France
Jens Lehmann, PhD, Medical University of Innsbruck, Austria

Speakers:

Bryan McDowell, MSc MBA, Clario, Switzerland
Florence Mowlem, PhD, uMotif, United Kingdom
Alisa Heinzman, MFA, RWS Regulated Industries, United States
Estelle Haenel, PharmD PhD, Kayentis, France

As the value of and reliance upon patient-reported outcomes (PRO) in the medical product development process has increased, collaborative science-based efforts aiming to better capture the patient’s voice have all aimed to drive collective progress. The increasingly evident benefits of electronic approaches for PRO data collection, along with deeper engagement and alignment with regulators, has unquestionably reaped dividends. Yet the question we must ask ourselves is this: as an industry, are we at last in a place where legacy thinking, unjustified risk aversion, and preaching without actually practicing true patient centricity are consigned to the past?

A series of informative yet interrogatory presentations alongside an expert panel discussion explored where we really are in terms of optimising PRO data collection, where we should be, and plot the path towards optimality.

Moderator:

Kim Cocks, PhD, Adelphi Values, United Kingdom

Speakers:

Jessica Roydhouse, PhD, Menzies Institute for Medical Research, University of Tasmania, Australia
Devin Peipert, PhD, Northwestern University Feinberg School of Medicine, United States
Antoine Regnault, PhD, Modus Outcomes, France
Jennifer Beaumont, MS, Clinical Outcomes Solutions, United States
Libby Floden, PhD MPH, Evinova, United States

Tolerability is increasingly important in oncology and other therapeutic areas and an overall side effect impact summary item was recommended as a core oncology trial outcome in recent regulatory guidance. A single item for capturing important aspects of tolerability is minimally burdensome and can generate useful data, but several analytic and interpretation issues must be addressed. This symposium provided a series of talks on core statistical issues relating to a single item for tolerability, including operationalization, developing tolerability estimands, novel methods of data collection, addressing statistical challenges and considerations when opting for single- or multi-item measurement of tolerability. The session included speakers from industry and academia from a diverse range of countries and was intended for anyone interested in clinical trials and measurement research.

Moderators:

Monika Sztankay, PhD, Medical University of Innsbruck & University Hospital Innsbruck, Psychiatry II, Austria
Klara Greffin, PhD, Department of Psychology, University of Greifswald, Germany & School of Psychology, University of Queensland, Australia

Speakers:

Alizé Rogge, PhD MSc BA, Charite Universitätsmedizin Berlin, Germany
Lukas Schöner, MSc, Technische Universität Berlin, Germany
Christine Blome, PhD, University Medical Center Hamburg-Eppendorf, Germany
Stefanos Boudouroglou-Walter, MSc, University Medical Center Hamburg-Eppendorf | Hamburg Center for Health Economics, Germany
Mina Luetkins, MSc, Germany

This symposium, organized by the German-speaking Countries Special Interest Group, aimed to provide an insight into the evolving landscape of patient-reported outcomes (PROs) and their impact on healthcare decision-making in German-speaking countries by exemplifying use cases going beyond single provider implementation.

Alizé Rogge (Charité, Berlin) reported on the Health Outcomes Observatory (H2O) launched by the EU-funded Innovative Medicines Initiative and the Patient-Centered Outcomes Research within the Medical Informatics Initiative connecting all 35 German academic medical centers within one national data sharing platform. Viktoria Steinbeck (Berlin University of Technology) showcased examples of PRO use in public reporting, benchmarking and PRO-based payment. Christine Blome and Stefanos Boudouroglou-Walter (University Medical Center Hamburg-Eppendorf and the Hamburg Center for Health Economics) shared a novel theoretical approach to PROMs use in the clinical context, embedding PRO instruments in the microeconomic principal-agent problem to explore ways to reduce information asymmetries within the patient-physician relationship.

Sunday, 13 October | 9:00 am – 4:00 pm

Workshop level: Basic

Workshop Goals:

Personal technologies have given us many new and convenient ways of discovering ourselves and our health. This new availability is not only poised to improve our personal lives but also has huge potential to broaden our research toolkit and modify our clinical interactions and clinical trial design to facilitate access and benefit.

The goals of this workshop are to leverage personal technologies to illustrate 1) how digital health technologies (DHTs) can be used to collect data in human subject studies, 2) how DHTs reflect individual’s feelings and states, and 3) how DHTs data can be analyzed for research practices. This workshop links technologies in a bidirectional way to life quality assessments.

The content of this workshop is informed by several frameworks, including the Wilson-Cleary model of patient outcomes, a newly proposed model of terminology for DHTs, and a novel method for causality estimation from observational data.

Intended Audience

Individuals working in or interested in digital health/medicine technologies, health outcomes researchers working with PROs, and individuals interested in learning about connections between these two environments by leveraging personal technologies.

Presentation Format

Lecture 20%. Hands-on: 60%. Q&A 20%.

Overview/Outline

Initially, participants will learn about digital health technology (DHT), including challenges and a newly proposed terminology model. This includes demonstrating the Heel2Toe sensor, a wearable technology for gait analysis. Group work will involve case studies applying this terminology.

The second part involves experimental hypothesis testing with the Heel2Toe sensor. Participants will simulate various moods, fatigue levels, and pain states to observe their impact on gait markers. Real-time biofeedback will be provided to examine its effect on gait quality under different health states. Data from this exercise will be analyzed later and shared.

The final part includes a hands-on analysis of DHT data. This includes population-level analysis and individual causality estimation. Optionally, participants can collect personal data using a wearable device during the conference. The workshop concludes with discussions on technology experiences, PRO linkages, and their research and clinical practice implications.

Learning Objectives

1. By the end of the conference, participants will be able to identify the theoretical and practical challenges and opportunities in implementing DHT as a method of data collection. Participants will accomplish this goal through information sessions, case studies, and experiential learning in acquisition and analysis. Accomplishing this goal will give participants enhanced competency and confidence in implementing DHTs in their own research.
2. By the end of the session, participants will be able to explain/articulate the relationship between digital biomarker data and health states. Participants will accomplish this through playing an active role in a real-time proof of concept experience. Accomplishing this goal will allow participants the skills needed to justify the use of digital biomarker data to identify health states and changes in them.
3. By the end of the session, participants will gain experience and know-how on how to analyze and interpret DHT data. Participants will accomplish this goal by analyzing real-world, collected sensor-based DHT data at the group and individual levels. This will equip the participants with a toolkit to replicate the analysis with their own data collected throughout the conference, as well as in their own work roles.

Organizers:

Igor Matias, MSc PhD student, University of Geneva, Quality of Life Technologies Lab, Geneva, Switzerland

Nancy Mayo, BSc(PT) MSc PhD, McGill University, Montreal, Quebec, Canada

Piper Fromy, PhD, SeeingTheta, Saumur, France

Katarzyna Wac, PhD in Information Systems, University of Geneva, Quality of Life Technologies Lab, Geneva, Switzerland

Sunday, 13 October | 9:00 am – 12:00 pm

Workshop level: Basic

Workshop Goals:

1. Understand the importance and complexities of patient engagement in quality-of-life research among paediatric populations.
2. Identify appropriate strategies to engage children, youth and parents (for example, digital storytelling, citizen juries, photo elicitation, video reflexive ethnography).
3. Be able to develop an evaluation plan of the impact of these strategies in research.

Intended Audience

Researchers, clinicians and patient partners who have an interest in quality of life research and patient engagement.  No previous knowledge or experience is required.

Presentation Format

33% lecture, 33% hands-on learning, 33% question and answer.

Overview/Outline

There is no evidence of the effectiveness of guidelines regarding young people’s engagement in the context of child health research. To date, the most reported strategies include qualitative methods, such as focus groups, meetings, interviews, scientific cafes, priority settings activities, expert panels/working groups/Young Peoples Advisory Groups (YPAG).

This workshop aims to explore the current state of the art and illustrate with case studies from three continents the strategies and methods that have been used to involve young people in health outcomes research. We will look at how these are used and evaluate their effectiveness. Researchers with current experience drawn from their thesis work will provide case studies, experts involved in the promotion of patient engagement will illustrate strategies being used to optimise and stimulate patient engagement, advice/guidelines from regulators will be presented and patient partners will describe their experience of being a part of the research process. Together we will attempt to identify which tools/resources and strategies are most effective.

Participants will develop an understanding of the strategies and methods for child patient engagement, learn recruitment strategies, knowledge and training requirements, activity plans and methods of integration and feedback. Knowing what strategies are most appropriate and successful for what age group will help inform researchers on what strategies to use in their research process. The workshop will include presentations of case studies, hands-on work using worksheets to develop recruitment, training and activity plans. Participants will also have an opportunity to present their own experiences and the workshop will promote discussion around ways of ensuring meaningful and successful youth patient engagement.

Learning Objectives

1. Understand the importance and complexities of patient engagement in quality-of-life research among paediatric populations
2. Identify appropriate strategies to engage children, youth and parents (for example, digital storytelling, citizen juries, photo elicitation, video reflexive ethnography).
3. Be able to develop an evaluation plan of the impact of these strategies in research.

Organizers:

Sumedh Bele, PhD MBBS MPH, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, Oxford, Alberta, United Kingdom

Harpreet Chhina, PhD, University of British Columbia, Vancouver, British Columbia, Canada

Sadia Ahmed, MSc, University of Calgary, Calgary, Alberta, Canada

Contributor:

• John Chaplin, PhD, University of Gothenburg, Gothenburg, Sweden

Sunday, 13 October | 9:00 am – 12:00 pm

Workshop level: Basic

Workshop Goals:

The goal of this workshop is to educate attendees about LGBTQIA+ (Lesbian, Gay, Bisexual, Transgender, Queer, Intersex, Asexual/Aromantic, + indicating other sexualities and gender identities exist) representation in Patient Reported Outcomes (PRO) research and the role this plays in achieving broader diversity and inclusion in clinical research. The workshop aims to show practical steps to maximise inclusivity and minimise cis-heteronormative biases in PRO measure development and evaluation.

Intended Audience

This workshop is intended for those interested in the ways that clinical research can be diverse and inclusive, particularly for the LGBTQIA+ community. This includes industry and academic researchers, regulatory bodies, and other stakeholders invested in clinical outcomes and PRO research. A basic knowledge of PROs is assumed; no prior knowledge of working with LGBTQIA+ populations is required.

Presentation Format

The workshop combines direct teaching (50%) and interactive tasks (40%) for all attendees. Participants will be encouraged to participate in group discussion, including commenting on the presented topics and asking questions to the facilitators (10%).

Overview/Outline

Led by Clinical Outcome Assessment (COA) consultants, academic and industry representatives, with extensive experience in diversity, equity and inclusion initiatives, including work with gender minorities, this workshop will focus on expanding diversity and inclusion in PRO research to consider LGBTQIA+ communities. This includes demographic categories of sex assigned at birth, gender identity and sexual orientation. Current state of play of inclusion of PRO research and future opportunities for LGBTQIA+ inclusion will be covered, including why LGBTQIA+ inclusion in clinical and PRO research is important for all stakeholders. Terminology used in PRO measures will be discussed with a focus on identifying non-inclusive or cis-heteronormative language and how language can be modified to allow for LGBTQIA+ inclusion. Specific examples will be evaluated and developed, in relation to items capturing sexual and social impacts, allowing for tangible demonstration of the discussed concepts to be explored. Ways of maximising LGBTQIA+ inclusivity in PRO research will be discussed in relation to planning for inclusive, diverse, and representative samples, including appropriate recruitment techniques and terminology used in patient/participant-facing documentation and interview techniques.

Learning Objectives

1. To establish why LGBTQIA+ inclusion in PRO research matters This section will establish the current landscape of LGBTQIA+ (Lesbian, Gay, Bisexual, Transgender, Queer, Intersex, Asexual/Aromantic, + indicating other sexualities and gender identities exist) inclusion in clinical and PRO research. We will cover why treatments should be tested in patient samples reflecting the target population, including LGBTQIA+ communities, to allow generalizability of safety and efficacy results. We will review historical exclusion of such groups, particularly transgender and gender-diverse individuals. We will discuss why diverse LGBTQIA+ representation should matter to various stakeholders, including research sponsors, regulators, health technology assessments, and payers. Workshop participants will learn about the bi-directional relationship between inclusive practices and (un)willingness of marginalized populations to trust in/contribute to research, grounded in theoretical knowledge (e.g., Minority Stress Theory). We will introduce how inclusive PRO measures can enable inclusive clinical research, with the goal of improving LGBTQIA+ representation across research contexts.

2. To evaluate and develop LGBTQIA+ inclusive PRO measure items and instructions Workshop participants will learn how to maximize LGBTQIA+ inclusivity when developing or selecting a PRO measure(s). Using examples from the literature, participants will learn how to evaluate PRO measure(s) for their suitability for use in LGBTQIA+ populations, as well as evaluate the sample used to develop the measure. Good practice for item and instruction generation in the development of new PRO measures that are inclusive to LGBTQIA+ communities will be considered. Participants will learn about identifying and avoiding pitfalls that lead to using cis-heteronormative language and concepts in item generation. An interactive task will involve reviewing and developing new PRO items relating to sexual and social impacts.

3. To illustrate how to plan PRO studies that are inclusive to LGBTQIA+ communities Workshop participants will gain an understanding of considerations and good practices for planning a PRO development/evaluation study that is inclusive to LGBTQIA+ communities. Components of developing an inclusive PRO study will be highlighted, including developing suitable demographic questions and sampling targets, appropriate recruitment techniques, terminology used in participant-facing documentation, qualitative interview techniques, and analysis methods. Specific examples will focus on developing suitable demographic questions and interviewing techniques. Workshop participants will learn from recent research when and how to capture relevant demographic information most appropriately, such as sex assigned at birth, gender identity, and sexual orientation. An interactive exercise will challenge unconscious biases when designing and conducting qualitative interviews to promote inclusivity, quality of data collected, and to improve research participants’ experiences and encourage engagement in future research.

Organizers:

Amy M Jones, MSc, Adelphi Values Ltd. – Patient Centered Outcomes, Cheshire, United Kingdom

Sam Wratten, PhD, Clarivate – Clinical Outcome Assessments, Macclesfield, United Kingdom

Mitchell R Lunn, MD MAS, Stanford University School of Medicine, Stanford, California, United States

Manraj Kaur, PhD, Harvard Medical School, Brigham and Women’s Hospital, Boston, Massachusetts, United States

Jane R Wells, MSc, Sanofi – Patient Informed Development & Health Value Translation, Reading, United Kingdom

Sunday, 13 October | 9:00 am – 12:00 pm

Workshop level: Basic

Workshop Goals:

Present details on how to develop an end-to-end patient-relevant endpoint strategy (ES) to support product development and clinical care that includes:

• Starting with patients to identify aspects of their disease and treatment that are most important
• Engaging diverse stakeholders across multiple development timepoints to align the endpoints to support regulatory, reimbursement, and clinical care needs
• Implementing the ES beginning in early development through phase 3, regulatory submission, and product launch
• Communicating the patient-relevant endpoint evidence to clinicians and caregivers to bridge clinical trial data to clinical care and improve physician-patient discussions

Intended Audience

Early and mid-career outcomes research scientists who are responsible for clinical outcome assessment (COA) measure development and/or selection and strategy.

Presentation Format

½ day workshop with 40% lecture and 60% hands-on work/group discussion.

Overview/Outline

During the development of a new pharmaceutical product, a significant amount of evidence is generated to support product approval and launch. A well-defined patient-relevant ES starts with understanding directly from patients their unmet needs, experience with disease- and treatment-related symptoms, and impact of the disease and treatment on their lives. Understanding what matters most to patients, the requirements of regulators and reimbursement agencies, and the needs of clinicians is critical for building an end-to-end endpoint strategy and work plan. The workshop will be taught by experienced patient-centered outcomes research scientists who will incorporate principles from regulatory guidance (e.g., PFDD), best practices, and the ISOQOL Introduction to Patient-Centered Outcomes Research for the Pharma/Biotech Industry (IPCOR) course. The presenters will discuss the importance of generating and evaluating information early in clinical development (phase 1) which provides the framework for creating a robust patient-relevant ES that can be included in phase 2 and refined for phase 3. The presenters will also highlight collaborations with internal partners, and strategic timepoints to interact with regulatory and reimbursement agencies as well as clinicians and caregivers. Attendees will be given case studies and work in groups to create a work plan focusing on research to understand the patient experience, the development of a patient-relevant ES, and value messages and a communication plan geared towards improving clinician and patient discussions about the approved product.

Learning Objectives

1. Understand the importance of developing patient-relevant endpoint strategies to support product development and regulatory, health technology assessment (HTA), patient, and provider acceptance
2. Learn methods, approaches, and tools to create patient-relevant endpoint strategies and how to communicate trial results to clinicians, caregivers and patients
3. Work collaboratively in groups to apply the learnings to a real-life case study, using qualitative patient data, the target product profile, and clinical trial results.

Organizers:

Allison Nguyen, MS, Merck, Sharp & Dohme LLC, West Point, Pennsylvania, United States

Helen Kitchen, MSc, Clarivate, Manchester, United Kingdom

Tom Willgoss, PhD MSc, Roche Products Ltd, Welwyn Garden City, United Kingdom

Kelly McQuarrie, Merck, Sharp & Dohme LLC, West Point, Pennsylvania, United States

Sunday, 13 October | 1:00 pm – 4:00 pm

Workshop level: Basic

Workshop Goals:

Personalized clinical outcome assessments, particularly goal attainment scaling, have been increasingly used in academic and clinical research settings. In accordance with increased efforts in patient-focused drug development, it has recently been recommended by regulators, especially as a way to assess meaningful change following treatment for conditions with heterogeneous symptoms and impacts. In their draft guidance published in 2023, the FDA has underlined its strengths (i.e. highly responsive, inherently clinically meaningful, non-arbitrary and reveal what is important to patients), but also pointed out the need to standardize the goal-setting process. In this workshop we aim to address this need and introduce different methods to implement GAS.  Our goal is to give researchers an understanding of the value of this personalized endpoint and provide an introduction to the tool, along with methods that standardize its implementation.

Intended Audience

Are you currently planning to incorporate personalized endpoints into your research project? Are you looking for a responsive outcome assessment that will successfully assess treatment response? Are you interested in exploring clinical meaningfulness at the patient level and learning more about your treatment/intervention?  Are you interested in what matters most to patients/caregivers and how they relate to other standardized outcome assessments?

Presentation Format

45% lecture; 40% interactive activities; 15% admin (intro, breaks, conclusion).

Overview/Outline

The workshop will introduce goal attainment scaling within the context of clinical research and will outline why, when and how it can be utilized.  The objectives are:

1. To introduce the rationale and value of personalized endpoints, particularly goal attainment scaling (GAS) and to illustrate the contexts where it is most suitable and powerful (why/when).
2. To help attendees gain the knowledge/skills to use GAS themselves and deploy it in their own research program. This includes a step-by-step hands-on exercise where participants develop multiple goal scales and assess attainment by calculating the total score (how).
3. To introduce different approaches to GAS implementation (e.g. paper forms vs. eCOA, advantages and disadvantages of goal inventories, training of GAS raters) and GAS techniques (e.g. developing a patient orientation handout, quality assurance methods). This includes a step-by-step hands-on exercise where participants assess the quality of the developed scales (how).

Learning Objectives

1. To introduce the rationale and value of personalized endpoints, particularly goal attainment scaling (GAS) and to illustrate the contexts where it is most suitable and powerful (why/when).
2. To help attendees gain the knowledge/skills to use GAS themselves and deploy it in their own research program (how). This includes a step-by-step hands-on exercise where participants develop multiple goal scales and assess attainment by calculating the total score.
3. To introduce different approaches to GAS implementation (paper forms vs. eCOA, advantages and disadvantages of goal inventories, training of GAS raters) and GAS techniques (e.g. developing a patient orientation handout, quality assurance methods). This includes a step-by-step hands-on exercise where participants assess the quality of the developed scales(how).

Organizers:

Gunes Sevinc, PhD, Ardea Outcomes, Halifax, Nova Scotia, Canada

Chere Chapmann, MBA MHSC, Ardea Outcomes, CEO, Halifax, Nova Scotia, Canada

Kellee Howard, MSc, IQVIA, Senior Principal, Halifax, Nova Scotia, Canada

Ana Maria Rodriguez-Leboeuf, PhD, McGill University, Montreal, Quebec, Canada

Sunday, 13 October | 1:00 pm – 4:00 pm

Workshop level: Basic

Workshop Goals:

Qualitative research (QALR) is an essential component to understand the lived experiences of patients with respect to their condition and treatment and ensure accurate measurement of relevant health-related outcomes. Today, QALR is also used to inform decision-making that aims to meet patients where they are and improve patient experiences in clinical research and real-world settings. This workshop will help participants understand application of QALR in their own work. Specifically:

• Learning about varied contexts where qualitative data has been applied to derive patient-centered scientific evidence for decision makers;
• Gaining an understanding of the types of research questions that can be answered through QALR, recommended good QALR practices, and key methodological considerations and limitations.

Intended Audience

Academic researchers, clinicians, patient advocates, and outcomes researchers interested in QALR. No prior knowledge required.

Presentation Format

An interactive workshop including expert presentation, discussion, and small group activities using illustrative examples. Presenters will discuss the topic, reflect on learnings, and encourage group discussion to empower participants to make informed decisions around QALR. Interactive break-out activities will be included to enable consolidation of learnings.

Overview/Outline

• Welcome (~15 min): Introduction, audience poll, agenda, objectives
• Introduction to QALR: (~45 minutes)
  • Importance of QALR
  • Basics of conducting QALR
    • Crafting QALR questions
    • Key considerations and best practices for design and conduct:
      • Objectives, study design, methods, implementation, analysis, reporting
    • Reflections from past experiences about limitations and practical considerations
  • Resources for further learning
• Break (~10 min)
• QALR Application (~45 minutes): Presenters will provide traditional and novel illustrative examples where QALR is leveraged
• Break (~10 min)
• Interactive Activity (~25 minutes): Participants will break into small groups and complete a practical exercise in applying the concepts learned. Case study vignettes that highlight varied scenarios where QALR could be applied will be used. Participants will work together to develop high-level components of a QALR study synopsis (e.g., research question, objectives, methodology, interview questions)
• Guided Group Discussion (~20 minutes): Focused on learnings and outputs from interactive activity
• Wrap-up (~5 minutes): Evaluation, thank you, closing remarks

Learning Objectives

1. Workshop participants will gain an understanding of the various types of research questions that can be answered through qualitative research (QALR) and experience in crafting research questions appropriate for QALR.
2. Workshop participants will critically examine varied example contexts where evidence from QALR has be used to support scientific decision making, such as: a.Patient-centered outcomes research strategies; b.Study design and implementation; c.Decision making about the value of health care interventions.
3. Workshop participants will learn good research practices for QALR, including methodological considerations when designing and implementing qualitative research studies. Participants will gain: a.An ability to critically appraise qualitative research studies; b.A fundamental understanding of how to design and conduct QALR.

Organizers:

Sally Mannix, AstraZeneca, Gaithersburg, Maryland, United States

Asha Hareendran, PhD, UCB Biopharma Srl, Slough, United Kingdom

Haylee Andrews, MPH, Evinova, Gaithersburg, Maryland, United States

Organized by the New Investigator SIG

Speakers
Jae-Yung Kwon, PhD RN, Lotte van der Weijst, PhD, Jan R. Boehnke, PhD, and Rasa Ruseckaite, PhD MCompSc BCompSc

Trends in Citation Patterns for New Investigators and Manuscript Writing for Health-related Quality of Life Research

For early-career researchers, including graduate students, recent doctoral graduates, and faculty members, in Quality of Life research, navigating the publication process can be daunting. This session first delved into the citation trajectories and burgeoning domains of interest from the ISOQOL New Investigator Special Interest Group members. The session then drew on expertise of editors of ISOQOL journals to provide advice for early career researchers to enhance the publication of their patient-reported outcome papers. QLR and JPRO editors shared advice and their view on trends in the field from 2017-2024. This session also included an interactive discussion about skills needed in writing better academic papers.

Chocolate Museum

The 2024 social event took place at the Chocolate Museum, one of the most famous and visited places in Cologne. Attendees joined us in this unique setting to experience the whole world of chocolate and cocoa.

The Experience Cologne Social Event at the 31st Annual Conference was an exciting way to socialize with your colleagues and build new connections.

Come meet with us to discuss the challenges and solutions related to strategic and operational management of PROMs and other COAs, as well as the keys to increase the visibility of your COAs and improve the COA user experience (and satisfaction!) – all to ensure the increased use of COAs worldwide.

*This was an Ancillary Event hosted by Mapi Research Trust and was not an ISOQOL event. Pre-registration was required.