EClinicalMedicine (Oct 2024)
Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials – insights from an expert virtual roundtable
- Christina Yap,
- Olalekan Lee Aiyegbusi,
- Emily Alger,
- Ethan Basch,
- Jill Bell,
- Vishal Bhatnagar,
- David Cella,
- Philip Collis,
- Amylou C. Dueck,
- Alexandra Gilbert,
- Ari Gnanasakthy,
- Alastair Greystoke,
- Aaron R. Hansen,
- Paul Kamudoni,
- Olga Kholmanskikh,
- Bellinda L. King-Kallimanis,
- Harlan Krumholz,
- Anna Minchom,
- Daniel O'Connor,
- Joan Petrie,
- Claire Piccinin,
- Khadija Rerhou Rantell,
- Saaeha Rauz,
- Ameeta Retzer,
- Steven Rizk,
- Lynne Wagner,
- Maxime Sasseville,
- Lesley K. Seymour,
- Harald A. Weber,
- Roger Wilson,
- Melanie Calvert,
- John Devin Peipert
Affiliations
- Christina Yap
- Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK; Corresponding author. Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, SM2 5NG, UK.
- Olalekan Lee Aiyegbusi
- Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration West Midlands, University of Birmingham, Birmingham, UK; NIHR Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK
- Emily Alger
- Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK
- Ethan Basch
- Lineberger Comprehensive Cancer Center, Chapel Hill, NC, 27514, USA; Division of Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC, 27514, USA
- Jill Bell
- AstraZeneca, Oncology Research and Development, Gaithersburg, MD, USA
- Vishal Bhatnagar
- Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, USA
- David Cella
- Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
- Philip Collis
- Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK
- Amylou C. Dueck
- Department of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ, USA
- Alexandra Gilbert
- Leeds Institute for Medical Research, University of Leeds, St James's University Hospital, Leeds, UK
- Ari Gnanasakthy
- RTI Health Solutions, Patient Centered Outcomes Research, Research Triangle Park, NC, USA
- Alastair Greystoke
- NU Cancer, Newcastle University, Newcastle upon Tyne, UK
- Aaron R. Hansen
- Princess Alexandra Hospital, Cancer Services, Brisbane, Queensland, Australia; University of Queensland, Faculty of Medicine, Australia
- Paul Kamudoni
- Merck KGaA, Darmstadt, Germany
- Olga Kholmanskikh
- Federal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium
- Bellinda L. King-Kallimanis
- LUNGevity Foundation, Bethesda, MD, USA
- Harlan Krumholz
- Center for Outcomes Research and Evaluation, Yale New Haven Hospital, New Haven, CT, USA
- Anna Minchom
- Drug Development Unit, Royal Marsden/Institute of Cancer Research, Sutton, UK
- Daniel O'Connor
- The Association of the British Pharmaceutical Industry, UK
- Joan Petrie
- Canadian Cancer Trials Group (CCTG), Kingston, Ontario, Canada
- Claire Piccinin
- Quality of Life Department, European Organisation for Research and Treatment of Cancer, Brussels, Belgium
- Khadija Rerhou Rantell
- Medicines and Healthcare Products Regulatory Agency (MHRA), UK
- Saaeha Rauz
- Academic Unit of Ophthalmology, Institute of Inflammation and Ageing, University of Birmingham, UK; Birmingham and Midland Eye Centre, Sandwell and West Birmingham Hospitals NHS Trust, Birmingham, UK
- Ameeta Retzer
- Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK; National Institute of Health and Care Research (NIHR) Birmingham Biomedical Research Centre, University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK
- Steven Rizk
- Veloxis Pharmaceuticals, Cary, NC, USA
- Lynne Wagner
- University of North Carolina Gillings School of Global Public Health, Chapel Hill, NC, USA
- Maxime Sasseville
- Health Canada, Ottawa, Ontario, Canada
- Lesley K. Seymour
- Canadian Cancer Trials Group (CCTG), Kingston, Ontario, Canada
- Harald A. Weber
- Pfizer Oncology, Global Medical Affairs/Early-Stage Development, Zug, Switzerland
- Roger Wilson
- Cancer Research Advocates Forum UK, Sarcoma Patient Advocacy Global Network, UK
- Melanie Calvert
- Centre for Patient-Reported Outcomes Research (CPROR), Institute of Applied Health Research, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC), University Hospital Birmingham and University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration West Midlands, University of Birmingham, Birmingham, UK; NIHR Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK
- John Devin Peipert
- Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
- Journal volume & issue
-
Vol. 76
p. 102838
Abstract
Summary: Early phase clinical trials provide an initial evaluation of therapies’ risks and benefits to patients, including safety and tolerability, which typically relies on reporting outcomes by investigator and laboratory assessments. Use of patient-reported outcomes (PROs) to inform risks (tolerability) and benefits (improvement in disease symptoms) is more common in later than early phase trials. We convened a two-day expert roundtable covering: (1) the necessity and feasibility of a universal PRO core conceptual model for early phase trials; (2) the practical integration of PROs in early phase trials to inform tolerability assessment, guide dose decisions, or as real-time safety alerts to enhance investigator-reported adverse events. Participants (n = 22) included: patient advocates, regulators, clinicians, statisticians, pharmaceutical representatives, and PRO methodologists working across diverse clinical areas. In this manuscript, we report major recommendations resulting from the roundtable discussions corresponding to each theme. Additionally, we highlight priority areas necessitating further investigation.
