Ethics approval and consent to participate

The study was conducted in accordance with all ethical principles having their origin in the Declaration of Helsinki. All participants provided written, informed consent. The study did not entail any prospective methodology involving patient treatment. All data were de-identified. The study protocol and documents were approved by a central institutional review board (IRB) (Advarra) and local IRBs, as required.

Consent for publication

Not applicable.

Competing interests

WL reports consultant arrangements with Astria, BioCryst, BioMarin, CSL Behring, Express Scripts/CVS, Fresenius Kabi, Intellia, KalVista, Magellan, Optum, Pharming, Pharvaris, and Shire/Takeda; speakers’ bureau for BioCryst, CSL Behring, Optinose, Pharming, Shire/Takeda, Grifols, Astra Zeneca, Sanofi/Regeneron, and GSK; and grants/research support from Astria, BioMarin, CSL Behring, Grifols, Intellia, Ionis, KalVista, and Shire/Takeda. TC reports research, speaking and consulting relationships with BioMarin, CSL Behring, and Takeda; research activities with Astoria, GSK, Intellia, KalVista, and Pharvaris; service as a speaker for Grifols; and consultant activities for BioCryst. JA has received personal fees for steering committee, advisory board, speaker bureau, and/or clinical research activity from BioCryst, BioMarin, CSL Behring, Cycle Pharmaceuticals, KalVista, Pharming, Pharvaris, and Shire/Takeda. MR reports research support from BioCryst, BioMarin, CSL Behring, Ionis Pharmaceuticals, KalVista, Pharvaris, and Takeda; and consultancy fees from Astria, BioCryst, BioMarin, CSL Behring, Cycle Pharma, Grifols, Intellia, KalVista, Ono Pharma, Pfizer, Pharming, Pharvaris, Sanofi-Regeneron, and Takeda. HL has been a speaker for CSL Behring, BioCryst, Takeda, and Pharming; consultant/advisory board member for BioCryst, BioMarin, CSL Behring, Intellia, KalVista, Pharming, Pharvaris, and Takeda; and has undertaken sponsored research for BioCryst, BioMarin, CSL Behring, Intellia, KalVista, Pharming, Pharvaris, and Takeda. RT reports speaking, advisory, and research fees from BioCryst, CSL Behring, and Pharming; research and advisory honoraria from KalVista; and research fees from Ionis and Pharvaris. MM has been a research/principal investigator for BioCryst, BioMarin, CSL Behring, Ionis, KalVista, Pharming, Pharvaris, and Takeda; a speaker for BioCryst, CSL Behring, Pharming, and Takeda; and a consultant to BioCryst, CSL Behring, KalVista, Pharming, and Takeda. PB is an employee and shareholder of CSL Behring, sponsors of the study. FR’s company, Churchill Outcomes Research, was funded by CSL Behring to serve as the contract research organization for this study and to provide assistance with medical writing and editing of this report. SW is an employee of ICON plc, which conducted the qualitative research component of the study, funded by CSL Behring. TRS has no conflicts of interest to report. JAB has been a primary investigator and consultant for BioCryst, BioMarin, CSL Behring, Ionis, KalVista, Pharming, and Takeda/Shire; and a consultant to Astria, Intellia, and Pharvaris.