Document is current

Article History

Received: 26 February 2026

Accepted: 6 August 2026

First Online: 30 September 2026

Declarations

Conflicts of Interest: Ayo Adeyemi and Michelle Erdmann are employees of, and own stocks in, Alexion, AstraZeneca Rare Disease. At the time of the study, Susanna Burckhardt was an employee of Alexion, AstraZeneca Rare Disease and still owns stocks in AstraZeneca. Abby Crites is an employee of IQVIA and has conducted market research studies with patients, healthcare professionals, and payers on behalf of Alexion, AstraZeneca Rare Disease. Rosalie Ferner is a medical advisor for Alexion. Riccardo Buttarelli, Brendon Pommier, Geting Su, and Hui Zhang declare that they have no competing interests.

Ethical Approval: Review by an Institutional Review Board (IRB) was not required, as this study aligns with established international ethical standards for non-interventional qualitative research and, therefore, meets the criteria for exemption from formal institutional ethics board review. Several globally recognized frameworks, including the European Pharmaceutical Market Research Association [EPHMRA] guidelines and code of conduct [ ], the British Healthcare Business Intelligence Association [BHBIA] guidelines and legislation [ ], COPE guidance and tools [ ], and ESOMAR Global Voice of Research, Data and Insights professional research guidelines [ ], consistently affirm that anonymized, opinion-based, qualitative research with no clinical intervention does not constitute human subjects research requiring formal IRB review. The study is therefore appropriately classified as exempt under widely recognized international ethical standards governing healthcare market and outcomes research. Honoraria were provided at fair market value for participation in 45-min interviews. Informed consent was obtained from every participant prior to participation in the study before the interviews started. Participants were explicitly informed about how their data would be used, including their right to withdraw from the study at any time. While participants were not specifically told that the study results would be published, they were clearly informed that their responses would be reported only in aggregate form and that their identities would remain anonymous throughout all stages of analysis and dissemination.