Funding for this research was provided by:
Vertex Pharmaceuticals Incorporated (NA)
Received: 18 March 2019
Accepted: 26 June 2019
First Online: 13 August 2019
Ethics approval and consent to participate
: Central ethics approval of the study protocol was obtained in the UK (national approval via the National Research Ethics Service Committee South Central, reference no. 14/SC/0297) and France (ethics exemption provided by the <i>Comité de Protection des Personnes</i>), while institution-specific ethics approval was obtained at each of the participating institutions from Germany (institutional ethics review committee at Universitätsklinikum Jena [reference no. not available]), and approval by the Ethics Commission of LMU Munich, reference no. 390–14), Australia (local institutional review board at Princess Margaret Hospital, Scientific Advisory Sub-committee, reference no. 2014096/EP, and Human Research Ethics Committee of Prince Charles Hospital, Metro North Hospital and Health Services, reference no. HREC/14/QPCH/113), and Ireland (Temple St Children’s University Hospital, Ethics Committee, Department of Research, reference no 14.042).Written informed participant consent was obtained by participants or their guardians. All participants, or their parent or guardian if aged younger than 18 years, provided written informed consent, or assent where appropriate, before enrolment.
: Not applicable.
: SCB is a site principal investigator, advisory board member, and writing group member; he has received travel support from Vertex Pharmaceuticals Incorporated to attend investigator meetings. He also has been a site principal investigator and received travel support from Galapagos and Flatley to attend investigator meetings and attended advisory board meetings for AbbVie/Galapagos. JGM has participated in clinical trials and has received funding for investigator-initiated research and advisory boards for Vertex Pharmaceuticals Incorporated. GM has received funding for investigator-led research, participated in clinical trials, and attended paid advisory board meetings for Vertex Pharmaceuticals Incorporated. SM is a former advisory board member and has received funding and travel support from Vertex Pharmaceuticals Incorporated. JM has participated in clinical trials and received funding for advisory boards from Vertex Pharmaceuticals Incorporated. MF is a consultant for Vertex Pharmaceuticals Incorporated. EDS is a former employee of Vertex Pharmaceuticals Incorporated and may own stock or stock options in that company. SN is an employee of Decision Resources Group, has provided consulting services for Vertex Pharmaceuticals Incorporated, and has received funding to execute the observational study discussed in this manuscript. NK is an employee of Vertex Pharmaceuticals Incorporated and may own stock or stock options in that company.