Document is current

Article History

Accepted: 10 November 2025

First Online: 20 November 2025

Declarations

:

: Adriana Boateng-Kuffour, Lauren Lilly, Jennifer Drahos, and Nanxin Li are employees of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. Melanie Calvert is Director of the Centre for Patient-Reported Outcomes Research and Deputy Director Birmingham Health Partners Centre for Regulatory Science and Innovation and a National Institute for Health and Care Research (NIHR) senior investigator; has received funding from Anthony Nolan, European Regional Development Fund, Gilead, GSK, LifeArc, Merck, MHRA, NIHR, NIHR ARC WM, NIHR Birmingham BRC, NIHR BTRU Precision and Cellular Therapeutics, UKRI; and has received consultancy fees from Aparito, Beta6, Boehringer Ingelheim, CIS Oncology, Gilead, Halfloop, ICON, Merck, Northwestern University, Pfizer, Shionogi and Vertex Pharmaceuticals Incorporated. Ashley Valentine has received consulting fees for Atlas Clarity Consulting Firm and Vertex Pharmaceuticals Incorporated; acted as Board of Director for IVI; and acted as an advisory board member for PCORI. Anthony Mason has received consulting fees for Vertex Pharmaceuticals Incorporated. Zahra Pakbaz has received research grants from Amgen, Forma Therapeutics, Global Blood Therapeutics, Novartis, Novo Nordisk, and Pfizer; received consulting fees from Agio, Alexion, Amgen, Global Blood Therapeutics, Novartis, Sobi, and Vertex Pharmaceuticals Incorporated; received speaker fees from SOBI, Agio, GPT, Aplastic Anemia MDS foundation, Cayenne Wellness Center and Child Foundation, and acted as a CME course director for the Cayenne Wellness Center and Child Foundation and planning committee member for their annual education symposium. Farrukh Shah has received research grants from IQVIA, Novartis Pharma AG, Pharmacosmos, and Vertex Pharmaceuticals Incorporated; received honoraria from Biologix FZ co, Bristol Myers Squibb, Chiesi Ltd, and Novartis Pharma AG; served as an advisory board or committee member for Agios, bluebird bio, Bristol Myers Squibb, Silence Therapeutics Plc, and Vertex Pharmaceuticals Incorporated; and acted as Chair for the UK Forum on Haemoglobin Disorders. Nick Ainsworth is an employee of QC Medica and Antony P. Martin is a partner of QC Medica which was funded by Vertex Pharmaceuticals Incorporated to perform this research.

: Raw data is not publicly available in order to protect participant privacy. Requests for data can be made to the corresponding author.

: All authors contributed to the study conception and design. Material preparations were performed by all authors. Data collections were performed by Antony P. Martin and Nick Ainsworth. Analyses were performed by Adriana Boateng-Kuffour, Lauren Lilly, Jennifer Drahos, Melanie Calvert, Nanxin Li, Nick Ainsworth, and Antony P. Martin. All authors critically reviewed, contributed to, and approved the final manuscript.

: Ethical approval was not required for this study and exemption for this study was granted by WIRB-Copernicus Group Institutional Review Board.

: The authors affirm that human research participants provided informed consent for publication.

: Not applicable.

: All analyses were performed using R (version 4.3.1) and R Studio (version 2023.06.1+524) software. No custom code was developed for this study.