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Article History

Accepted: 23 March 2025

First Online: 17 April 2025

Declarations

:

: 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.

: Jennifer Drahos, Adriana Boateng-Kuffour, and Nanxin Li are employees of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. Melanie Calvert is Director of Birmingham Health Partners Centre for Regulatory Science and Innovation and Centre for Patient Reported Outcomes Research and a National Institute for Health and Care Research (NIHR) senior investigator; has received funding from Anthony Nolan, European Regional Development Fund-Demand Hub and Health Data Research UK, Gilead, GlaxoSmithKline, Janssen, Macmillan Cancer Support, Merck, NIHR, NIHR ARC WM, NIHR Birmingham BRC, NIHR BTRU Precision and Cellular Therapeutics, UCB Pharma, UKRI, and UK SPINE; and has received consultancy fees from Aparito, Astellas, Boehringer Ingelheim, CIS Oncology, Daiichi Sankyo, Gilead, Glaukos, GlaxoSmithKline, HalfLoop, Merck, Patient-Centered Outcomes Research Institute, Pfizer, Takeda, and Vertex Pharmaceuticals Incorporated. Laurice Levine has received honoraria from Editas and Vertex Pharmaceuticals Incorporated; received consulting fees from Agios and bluebird bio; received support for congress participation from Agios; and has served as an advisory board member for Editas. Neelam Dongha has received consulting fees from 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, Amgen, Dova, Global Blood Therapeutics, Guide point, Novartis, Sanofi, Sobi, and Vertex Pharmaceuticals Incorporated; received honoraria from Cayenne Wellness Center and Child Foundation, Dova, and Global Blood Therapeutics Inc; served as an advisory board or committee member for Alexion, Sanofi, and Sobi; 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 who were funded by Vertex Pharmaceuticals Incorporated to perform this research.