Measuring What Matters 2022

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Symposium Theme

Measuring What Matters in Child Health: Challenges, Developments and Opportunities

There are many challenges that arise in the use of patient-reported outcome measures (PROMs) in pediatric populations. These include age and developmental differences, response shift associated with developmental changes and illness trajectories, identifying the best informant(s) (the child, a parent/caregiver, healthcare professional), and understanding what concepts are important to children and at what age they can reliably self-report. The aim of this symposium is to provide a forum to share and debate current conceptualizations of quality of life in children, methodological and operational challenges commonly encountered in measurement and best practice methods for addressing these.

​Measuring What Matters Symposium Committee Co-Chairs

Nalin Payakachat, BPharm MS PhD

Eli Lilly and Company
Arkansas, United States



Rob Arbuckle, MSc

Adelphi Values
Patient-Centered Outcomes
United Kingdom



Session I: Does one size actually fit all? Conceptual fuzziness of QoL/HRQoL/functioning in pediatrics

Monday, 18 July | 8:10 am – 9:10 am CDT



Many health conditions in childhood affect children from birth through to adulthood, impacting their quality of life (QoL). Any definition of QoL for children must include the family context and developmental, environmental and societal factors associated with growing up with a health condition. Some theoretical models have been developed to understand QoL, but they were not developed specifically for the pediatric population. For QoL to be accurately measured in children, pediatric QoL must first be conceptualized. In this session, we will discuss current theoretical models of QoL and health related quality of life (HRQoL) in children, the theoretical challenges of measuring QoL in children and adolescents and discuss possible solutions to overcome these challenges.


Christopher Forrest, MD PhD, Children’s Hospital of Philadelphia, Pennsylvania, United States

Dr. Forrest is a pediatrician and health services and outcomes researcher. He has developed over 50 pediatric patient-reported outcome measures, many of which are part of PROMIS, and serves as ISOQOL’s Secretary/Treasurer. Chris is a Professor of Pediatrics, the Executive Director and Founder of PEDSnet (, a national pediatric learning health system, and Director of the CHOP Applied Clinical Research Center, which is the institutional home for PEDSnet. He co-edited the new Handbook of Life Course Health Development, which has been downloaded over 650,000 times from the Springer web site.


Bryce Reeve, PhD, Duke University School of Medicine, Durham, North Carolina, United States

Dr. Bryce Reeve is a Professor of Population Health Sciences and Pediatrics at Duke University, where he serves as Director of the Center for Health Measurement. Trained in psychometric methods, Dr. Reeve’s work focuses on assessing the impact of disease and treatments on the lives of patients and their caregivers. This includes the development of clinical outcome assessments using both qualitative and quantitative methods, and the integration of patient-centered data in research and healthcare delivery settings to inform decision-making.

Session II: Measurement framework and approaches for different age ranges and conditions in pediatric populations

Monday, 18 July | 9:20 am – 10:20 am CDT



From childhood to adolescence, every child experiences changes in their roles and responsibilities and this in turn, changes the perceptions of their own functioning. This developmental change can affect the weight of the domains that make up the construct measured, or interpretation of the construct measured. This session aims to discuss measurement frameworks, response shift and factors associated with measuring/collecting QoL/HRQoL in pediatric population.


Christopher Forrest, MD PhD, Children’s Hospital of Philadelphia, Pennsylvania, United States

Dr. Forrest is a pediatrician and health services and outcomes researcher. He has developed over 50 pediatric patient-reported outcome measures, many of which are part of PROMIS, and serves as ISOQOL’s Secretary/Treasurer. Chris is a Professor of Pediatrics, the Executive Director and Founder of PEDSnet (, a national pediatric learning health system, and Director of the CHOP Applied Clinical Research Center, which is the institutional home for PEDSnet. He co-edited the new Handbook of Life Course Health Development, which has been downloaded over 650,000 times from the Springer web site.


Nikki Ow, PhD, The University of British Columbia, Vancouver, British Columbia, Canada

Nikki Ow is currently a Postdoctoral Fellow at the University of British Columbia in Canada. She graduated from McGill University where she was involved in work related to pediatric rare diseases, patient engagement, and response shift. Her current work involves research in youth mental health services, mental health outcomes, and the relationship between social media and youth mental health. Nikki is also a pediatric occupational therapist who has worked with children and adolescents with developmental disabilities.


Naomi Knoble, PhD, U.S. Food and Drug Administration, Washington, District Of Columbia, United States

Naomi Knoble, PhD, is a reviewer in the Division of Clinical Outcome Assessment (DCOA) at FDA. Prior to joining FDA, Dr. Knoble was a research scientist and global healthcare consultant specializing in patient-centered outcomes and measurement. She is also a psychologist specializing in pediatric neuropsychology. Her research focuses on pediatric rare disease clinical trial measurement.

Session III: How to meaningfully discuss meaningful change with children: Qualitative approaches

Monday, 18 July | 10:30 am – 11:30 am CDT



The FDA is increasingly interested in patient perspectives of what level of change is important to support traditional quantitative methods of evaluating meaningful change and deriving thresholds. This has led to the emergent of qualitative methodologies to explore changes experienced by patients and the meaning of changes. In pediatric populations this throws up additional challenges: who is the best reporter? How to discuss in a way that children can understand the topic? This session will provide case-studies of different qualitative methodologies for capturing and contextualizing changes experienced within clinical trials. Learnings will be shared and illustrative examples presented.


Hannah Staunton, MSc, Roche Products Ltd, Welwyn Garden City, United Kingdom

Hannah Staunton is a Senior Principal Outcomes Research Scientist at Roche Products Ltd in the Patient Centered Outcomes Research (PCOR) team in Product Development. Hannah has been working in the field of patient-centered outcomes for the last 8 years, specialising in the development, validation and interpretation of patient, caregiver, clinician and performance outcomes. Hannah has more recently worked on novel wearable devices, an area of research that is evolving and presents new opportunities to understand the patient experience. As part of her role at Roche, Hannah is responsible for overseeing PCOR activities in the Neurodevelopment and Psychiatry therapeutic areas.


Claire Burbridge, MSc, Clinical Outcomes Solutions, Folkestone, United Kingdom

Claire is Executive Director, Clinical Outcomes Assessments (COA) at Clinical Outcomes Solutions (COS) and has been working in COA research for twenty years. Most recently in consultancy, and previously in the pharmaceutical industry, supporting compounds at all stages of the product lifecycle across multiple therapeutic areas; leading projects designed to capture the patient voice and explore the real meaning and value of treatments. Claire is a Chartered Psychologist and an Associate Fellow of the British Psychological Society and the BPS Division of Health Psychology. She has an MSc in Health Psychology from University College London.

Session IV: Per aspera ad astra: Advancement in measuring child health

Tuesday, 19 July | 8:10 am – 9:10 am CDT



Measuring health of children and adolescents has substantially progressed but remains a challenging adventure. In this session, the potentials and limitations of selected, advanced approaches to health status assessment in different pediatric populations will be presented. These novel solutions are related to alternative forms in which data are collected (e.g. using cartoon tools), what kind of data are recorded (e.g. behavioral data such as laughter), or which metrics are applied (e.g. multiple-group item response theory). Implications for interpretation and practice are discussed.


Holger Muehlan, PhD, University of Greifswald, Greifswald, Germany

Holger Muehlan, PhD, is a Senior Research Fellow in the Department of Health & Prevention at the University of Greifswald, Germany. Since his thesis on pediatric quality of life assessment his interest in health outcome assessment has continued to grow, especially in regard to pediatric health indicators and digital health applications. Some of his current research activities are directed toward the development of setting-sensitive outcome assessments in digital health contexts, the use of behavioral data for health assessment in small children, as well as ecological momentary assessment. Currently he serves as Chair of the ISOQOL Child Health SIG.


Aaron Kaat, PhD, Northwestern University, Chicago, Illinois, United States

Dr. Kaat is an Assistant Professor in the Department of Medical Social Sciences at Northwestern University. His expertise is in measure development, adaptation, and validation for chronic and developmental conditions typically diagnosed in childhood—especially intellectual and developmental disabilities. Dr. Kaat is particularly interested in novel solutions to the measurement challenges associated with younger children and individuals with cognitive impairments: for example, multiple-group psychometric models, and methods to account for informant effects. He is particularly interested in how the purpose of assessment should guide measure selection and how modern measurement theory can improve these practices.

Session V: Why is it so challenging to measure preference-based HRQoL in children? Potential measurement problems and challenges

Tuesday, 19 July | 9:20 am – 10:20 am CDT



Preference-based HRQoL instrument that are used to generate quality-adjusted life years (QALYs) are preferred by funding decision-makers because they generate a universal metric that presents an overall summary of health and HRQoL. There is a paucity of reliable and valid preference-based measures for measuring health states of children and adolescents. In recent years, new pediatric instruments have been developed but some still rely on adult value sets to generate preference-based scores (utilities) for children and adolescents. Importantly, there is no clear guidance for valuing and measuring health and preference-based HRQoL in pediatric populations.


J Mick Tilford, PhD, Department of Health Policy and Management, Little Rock, Arkansas, United States

J. Mick Tilford serves as a Professor and Chair of Health Policy and Management, Fay W. Boozman College of Public Health at the University of Arkansas for Medical Sciences. His research focuses on methods for the economic evaluation of child health services. He has studied the cost-effectiveness of improving outcomes in children with traumatic brain injuries and quality-adjusted life years in children with chronic conditions, especially children with autism. Recent areas of interest include the development of methods for incorporating family effects in economic evaluations. He received his Ph.D. in health economics from Wayne State University.


Lisa Prosser, PhD, University of Michigan – Susan B. Meister Child Health Evaluation and Research Center, Ann Arbor, Michigan, United States

Dr. Prosser is the Marilyn Fisher Blanch Research Professor of Pediatrics and Director of the Susan B. Meister Child Health Evaluation and Research Center. Her research focuses on measuring the value of childhood health interventions using methods of decision sciences and economics. Current research topics include newborn screening programs, vaccination programs, and methods for valuing family spillover effects of illness. Her work evaluating the cost-effectiveness of vaccination programs has been used in setting national vaccine policy. She currently serves as the Associate Vice President for Research-Health Sciences at the University of Michigan.


Wendy Ungar, MSc PhD, The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada

Wendy J. Ungar, M.Sc., Ph.D. is a Senior Scientist, Child Health Evaluative Sciences, Hospital for Sick Children Research Institute, Professor, Institute of Health Policy, Management and Evaluation, University of Toronto, and Affiliate Scientist, Institute for Clinical Evaluative Sciences (IC/ES). Dr. Ungar holds the Canada Research Chair in Economic Evaluation and Technology Assessment in Child Health. In 2007 Dr. Ungar founded TASK (Technology Assessment at Sick Kids), where she and her team conduct research applying health economic methods to child health with a focus on genomics and neurodevelopmental disorders. She and her team maintain the PEDE database, a user-friendly online database of pediatric economic evaluations published since 1980 used by HTA agencies around the world. Her book, Economic Evaluation in Child Health was published by Oxford University Press in 2010.

Session VI: Stronger together: Patient engagement and integration of PROs in pediatric clinical practice and research prioritization

Tuesday, 19 July | 10:30 am – 11:30 am CDT



This session will provide case-studies of a) engagement of children and caregivers in the co-design of an ePRO solution in clinical care in a children’s hospital in Calgary, Alberta, Canada; b) implementation of ePROs in clinical care, the KLIK in the Netherlands; c) illustrative example of the assembly of a youth panel for research prioritization and design. It is anticipated that there will be a youth partner in attendance to describe their experiences.


Maria Santana, PhD, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada

Dr. Maria J. Santana is a health services researcher, patient and family-centred care scientist, an Associate Professor in the departments of Pediatrics and Community Health Sciences at the Cumming School of Medicine, University of Calgary. Dr. Santana has received training in clinical pharmacy (BPharm, MPharm, London School of Pharmacy, UK, Universidad La Laguna, Spain), public health and clinical epidemiology (PhD, University of Alberta, Canada). She is the provincial lead, Patient Engagement for the Alberta Strategy for Patient-oriented Research ( and she is the academic leader of the Patient and Community Engagement in Research (PaCER,


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

Lotte Haverman is a child/adolescent psychologists, working in the Emma Children’s Hospital in Amsterdam. She is the director of the KLIK PROM portal. An online PROM portal to measure outcomes in children and parents. This portal is implementated as part of standard care in over 30 hospitals in the Netherlands. Lotte Haverman is member of the Dutch Flemish PROMIS group and translated, validated and collected norm data for the Dutch PROMIS item banks. Her research line mainly focuses on outcomes and intervention for children with chronic health condition and their parents.

The International Society for Quality of Life Research (ISOQOL) is a global community of researchers, clinicians, health care professionals, industry professionals, consultants, and patient research partners advancing health related quality of life research (HRQL).

Together, we are creating a future in which patient perspective is integral to health research, care and policy.