Document is current

Article History

Received: 18 July 2025

Accepted: 17 September 2025

First Online: 3 October 2025

Declarations

:

: Edward Wells, James Turnbull, and Matthew Reaney are employees of IQVIA, which received funding from Sanofi to conduct this study. Laurence Lucats, Joyce R. Talavera, Sophie R. Van Tomme, Benoit Arnould, Catherine Coulouvrat, and Paul Cordero are employees of Sanofi and may hold stocks or stock options in the company. Giovanni Abbadessa was an employee of Sanofi when this study was conducted and may hold stocks or stock options in the company.

: This study was conducted in accordance with the ethical principles that have their origin in the Declaration of Helsinki 1964 and its later amendments and is consistent with Good Clinical Practice and applicable regulatory requirements. Centralized approval was obtained from the Western IRB-Copernicus Group (WCG) IRB, including for all recruitment materials. IQVIA is responsible for submitting the application and all study documents for study approval to the IRB. The study was initiated only after IRB approval was granted, including the use of all recruitment materials.

: Informed consent was obtained prior to performing any study-related procedures, including screening, and patients provided consent via the online consent form. Patients were informed that, if the results of the study were published for scientific purposes, the patient’s identity would remain confidential. Clinicians who participated in the CD interviews provided verbal consent at the start of the interview after the objectives and logistics of the interview were shared with them. They provided verbal consent to both participate and to also be audio-recorded.A standard ethics approved informed consent process was followed for all patients involved in this study. This required their review and signature of an informed consent form document that clearly outlined the study background, objectives, and procedures (including screening, risks and benefits, compensation, confidentiality, information use and data sharing, and voluntary participation and withdrawal). Completed and signed forms were reviewed by the study team for completeness prior to any interviews being conducted. For participating clinicians, informed consent was obtained verbally at the start of each interview. Prior to providing consent, clinicians were informed of the study background and objectives, interview logistics, and information regarding their confidentiality and how their data will be used.

: The authors would like to thank SparkCures and Rare Patient Voice for their patient advocacy and support during interviews and the patients for their contributions to the study.