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Article History

Accepted: 3 December 2024

First Online: 3 January 2025

Declarations

:

: The study and publication fees were supported by Vertex Pharmaceuticals Incorporated.

: Chuka Udeze, Nanxin Li, Jessica Baldwin, and Petra Tuzin are employees of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. Colin Kunzweiler is a former employee of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. Sebastian Dietmar Zingel, Céline Vetter, and Silvia Dombrowski are employees of IQVIA Commercial GmbH & Co. and may hold stock or stock options in the company. Céline Vetter received consulting fees for the National Institutes of Mental Health. Elena Georgiadou-Schmidt and Aranzazu Alba are employees of Team Gesundheit GmbH, which received funding from IQVIA for conducting the statistical analysis in this study. Roland Meisel has received reimbursement for clinical trial participation from Vertex Pharmaceuticals Incorporated; received consulting fees for bluebird bio and Vertex Pharmaceuticals Incorporated; served as an advisory board member for bluebird bio and Vertex Pharmaceuticals Incorporated; and acted as Board Member for the Pediatric Diseases Working Party and the Hemoglobinopathy Working Party of the European Society for Blood and Marrow Transplantation (EMBT).

: Data for this study were obtained from the Betriebskrankenkassen (BKKs) German Sickness Fund Database.

: Not applicable.

: Not applicable.

: Not applicable.

: Not applicable.

: All authors contributed to the study conceptualization, design, and interpretation of the results per ICJME guidelines. Initial data analysis was performed by Sebastian Dietmar Zingel, Céline Vetter, Silvia Dombrowski, Elena Georgiadou-Schmidt, and Aranzazu Alba in consultation with Chuka Udeze, Colin Kunzweiler, Nanxin Li, Jessica Baldwin, Petra Tuzin, and Roland Meisel. All authors have reviewed the results and have read and approved the final manuscript.