Comparable Real-World Patient-Reported Outcomes Data Across Health Conditions, Settings, and Countries: The PROMIS International Collaboration
Abstract
The patient voice is critical to achieving value-based care, improving health outcomes, and advancing medical research. However, a key challenge is how to translate this “voice” into scientifically valid data that can inform evidence-based clinical decisions. One of the biggest barriers is the sheer variety of available patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs) and the associated challenges of translation, validation, implementation, and interpretation, making it difficult to obtain valid and comparable health outcomes. The authors present a harmonized global approach to international standardization of PROs and PROMs. This approach has the potential to accelerate patient-centered care by facilitating the collection of accurate and comparable real-world evidence on health outcomes that matter most to patients. This proposed approach consists of two elements: a data collection process based on a common set of PROs and a state-of-the-art measurement approach based on item response theory. First, there is growing evidence that outcomes such as pain, fatigue, anxiety, depression, sleep disturbance, physical function, and the ability to participate in social roles and activities are relevant for most people, irrespective of their health condition. Measuring these outcomes routinely in all patients could increase outcome comparability and utility for a range of stakeholders. Second, a measurement strategy based on a state-of-the-art psychometric approach — using item response theory (IRT)-based item banks — offers short, flexible, sustainable, and universally applicable PROMs with robust measurement properties and a common measurement scale. The unique integration of these two elements offers the potential to collect comparable PROM data across patients and providers to support shared decision-making, which may lead to better outcomes. The Patient-Reported Outcomes Measurement Information System (PROMIS) is a globally used example of such an approach. The PROMIS Profile measures serve as a resource for measuring a harmonized core set of PROs across medical conditions, languages, and countries. To meet the United Nations Sustainable Development Goal of ensuring healthy lives and promoting well-being for all, at all ages, a collaborative effort is needed to achieve consensus on international standardization of PROs and PROMs to accelerate patient-centered care across health conditions, settings, and countries. The authors propose to routinely measure a core set of broadly relevant PROs in all patients, regardless of their health condition, with universally applicable IRT-based PROMs.
Notes
We thank Dr. Judy Baumhauer for her leading role in promoting the use of PROMIS in daily clinical practice and sharing her experience and data (Figure 4) for this paper.
Rehab Alhasani, Juhee Cho, Heeseung Choi, Helena Correia, Fabio Efficace, Wojciech Glinkowski, Danbee Kang, Rebecca Mercieca-Bebber, Takeko Oishi, Ay-Woan Pan, Leo D. Roorda, Matthias Rose, Benjamin D. Schalet, Sein Schmidt, Melissa Tinsley, Gemma Vilagut, and Changrong Yuan have nothing to disclose. Caroline B. Terwee, Sara Ahmed, Jordi Alonso, Susan J. Bartlett, David Cella, John E. Chaplin and Felix Fischer are past board members of the PROMIS Health Organization (PHO). Dr. Bartlett is current President of PHO. All authors are members of a PROMIS national center. Dr. Terwee received grants from the Dutch Ministry of Health, Welfare, and Sport, and the Netherlands Organisation for Health Research and Development. Dr. Alonso reports grants from the following public institutions: Instituto de Salud Carlos III (ISCIII/FEDER), Spain; and Agency for Management of University and Research Grants (AGAUR) and Department de Salut, Catalonia, Spain. Dr. Alonso’s institution has received consultation funding from Ionis Pharmaceuticals Inc., USA. Dr. Chaplin is supported by investigator-initiated grants from Gothenburg Centre for Person-Centred Care and from Research and Development in West Sweden and by Horizon Europe funding. Istvan Mucsi is supported by investigator-initiated grants from the Canadian Institutes of Health Research, the Kidney Foundation of Canada, the Canadian Donation and Transplantation Research Program, the Health Canada Health Care Policy Contribution Program, and the Mount Sinai Hospital-University Health Network Academic Medical Organization Innovation Funding. Dr. Mucsi also received an unrestricted education grant from Astellas Canada and from Paladin Labs Inc., Canada. Jose M. Valderas is supported by grants from the National Medical Research Council; the National University Health System; the Yong Loo Lin School of Medicine, National University of Singapore (Singapore); the National Institute for Health Research (United Kingdom); the Health Research Fund (Spain); and the Organisation for Economic Co-operation and Development. Dr. Cella is supported by investigator-initiated grants from the U.S. National Institutes of Health, the U.S. Food and Drug Administration, and the Michael J. Fox Foundation. No funding was obtained for writing this paper.
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NEJM Catalyst Innovations in Care Delivery
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Copyright © 2024 Massachusetts Medical Society.
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Published online: August 21, 2024
Published in issue: August 21, 2024
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