A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH).

The importance of patient centricity and keeping the patient at the heart of research design is now well recognised within the healthcare community. The involvement of patient, caregiver and clinician representatives in the study design process may help researchers to achieve this goal and to ensure robust and meaningful data generation. Real-world data collection allows for a more flexible and patient-centred research approach for gaining important insights into the experience of disease and treatments, which is acutely relevant for rare diseases where knowledge about the disease is more likely to be limited. Here, we describe a practical example of a patient-centric, multi-stakeholder approach that led to the co-design of a prospective observational study investigating the lived experience of adolescents with the rare disease, X-linked hypophosphataemia. Specifically, we describe how the knowledge and expertise of a diverse research team, which included expert physicians, research and technology specialists, patients and caregivers, were applied in order to identify the relevant research questions and to ensure the robustness of the study design and its appropriateness to the population of interest within the context of the current clinical landscape. We also demonstrate how a structured patient engagement exercise was key to informing the selection of appropriate outcome measures, data sources, timing of data collection, and to assessing the feasibility and acceptability of the proposed data collection approach.
Competing Interests: The authors have read the journal’s policy and have the following competing interests: Vrinda Saraff received an honorarium for lectures and advisory board participation from Kyowa Kirin International and served as Lead Study Advisor for this study. Annemieke M. Boot has received research grants from Ultragenyx and Kyowa Kirin, and payment for lectures and consulting fees from Kyowa Kirin. Agnès Linglart’s academic institution has received a research grant, payment for lectures and consulting fees provided by A. Linglart from Kyowa Kirin. Angela Williams is an employee of Kyowa Kirin (study sponsor). Karen M.A. Bailey is an employee of OPEN Health, who has received payment from Kyowa Kirin to support the activities described in this article and to provide medical writing support for this manuscript. Fiona Glen is an employee of OPEN Health, who has received payment from Kyowa Kirin to support the activities described in this article and to provide medical writing support for this manuscript. Elin Haf Davies is an employee of Aparito, who has received payment from Kyowa Kirin to provide technological services to support an ongoing study sponsored by Kyowa Kirin. Sue Wood is a former employee of Kyowa Kirin (study sponsor). Stephen Greentree is a former employee of Kyowa Kirin (study sponsor). Angela J Rylands is an employee of Kyowa Kirin (study sponsor). The study is sponsored by Kyowa Kirin International. Karen M.A. Bailey and Fiona Glen are employed by OPEN Health and funded by Kyowa Kirin International. Medical writing and editorial support for the preparation of the manuscript was also provided by Afsaneh Khetrapal of ApotheCom, London, UK, and was funded by Kyowa Kirin International, in accordance with Good Publication Practice (GPP3) guidance (Ann Intern Med. 2015;163:461-464). This does not alter our adherence to PLOS ONE policies on sharing data and materials. There are no patents, products in development or marketed products associated with this research to declare."
(Copyright: © 2024 Saraff et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.)