The authors thank all the people with CF who took part in the study and their families, as well as the principal investigators and research coordinators at each site. The authors thank all of the site investigators and clinical research associates who collected data at study sites.
Collaborators: BRIO Study Group: Sophie Ramel, Philippe Vigneron, Veronique Storni, Natacha Remus, Laurence Bassinet, Isabelle Durieu, Muriel Laurans, Cinthia Rames, Françoise Troussier, Marie-Laure Dalphin, Frédéric Huet, Isabelle Pin, Boubou Camara, Annabelle Payet, Jeanne Languepin, Isabelle Danner-Boucher, Marc Albertini, Julie Mankikian, Nathalie Wizla, Laure Cosson, Eric Deneuville, Marie Mittaine, Philippe Reix, Nadine Dufeu, Valérie David, Thierry Langin, Stéphanie Bui, Raphael Chiron, Dominique Grenet, Frédérique Chedevergne, Chantal Belleguic, and Laurent Mely.
This study was supported by Vertex Pharmaceuticals Incorporated. The sponsor was involved in the study design, analysis, and interpretation of the data, with collaboration from the authors. The sponsor helped develop the report with input, review, and approval from the authors. Vertex Pharmaceuticals Incorporated also funded the journal’s Rapid Service Fee.
Medical Writing and/or Editorial Assistance
Editorial coordination and support were provided by Francesca Francois, PharmD, MPH, of Vertex Pharmaceuticals Incorporated; Francesca Francois may own stock or stock options in that company. Medical writing and editorial support were provided under the direction of the authors by JoAnna Anderson, PhD, CMPP, and Karen Kaluza Smith, PhD, CMPP. JoAnna Anderson and Karen Kaluza Smith are employees of ArticulateScience LLC, which received funding from Vertex Pharmaceuticals Incorporated.
All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.
All authors contributed to data interpretation, conception, drafting, and/or revisions to the manuscript, and all approved the final version that was submitted for publication.
The final analysis of the BRIO study has not been previously presented or published. Interim analyses from this study have been previously presented at: 32nd Annual North American Cystic Fibrosis Conference, October 18–20, 2018, Denver, CO, USA. ISPOR Europe 2018: New Perspectives for Improving 21st Century Health Systems, November 10–14, 2018, Barcelona, Spain. 23e Congrès de Pneumologie de Langue Française 2019 (23rd French Language Pulmonology Congress 2019), January 25–27, 2019, Marseille, France.
All authors received nonfinancial support (assistance with manuscript preparation) from ArticulateScience LLC, which received funding from Vertex Pharmaceuticals Incorporated. Additional disclosures are as follows: Dominique Hubert discloses personal fees from Vertex Pharmaceuticals Incorporated. Christophe Marguet discloses personal fees and nonfinancial support from Vertex Pharmaceuticals Incorporated. Jacques Benichou has no additional disclosures. Cynthia DeSouza is a former employee of Vertex Pharmaceuticals Incorporated and may own stock or stock options in that company; she is currently an independent consultant. Catherine Payen-Champenois, Nils Kinnman, and Keval Chandarana are employees of Vertex Pharmaceuticals Incorporated and may own stock or stock options in that company. Anne Munck discloses personal fees from Vertex Pharmaceuticals Incorporated. Isabelle Fajac discloses grants and personal fees from Vertex Pharmaceuticals Incorporated.
Compliance with Ethics Guidelines
This study was performed in accordance with the Helsinki Declaration of 1964, and its later amendments. Each investigator used International Society for Pharmacoepidemiology guidelines for good pharmacoepidemiology practice (2007) and good pharmacovigilance practices. This was an epidemiological observational study not modifying in any way the medical management of persons entering the study, not harming their physical or mental integrity, and not requiring any special monitoring visits. There were no additional or unusual diagnostic or surveillance procedures and no changes to the medications prescribed for the participants. Under these conditions, this study did not fall within the scope of French Research Planning Law 2006–450 of 18 April 2006 nor French Law 2004–806 of 09 August 2004, Article 88, Chapter II, Article L.1121–1, and the project, therefore, did not require submission to the Agence Nationale de Sécurité du Médicament et des Produits de Santé or the Ethics Committee. Written informed consent was obtained from each pwCF or their parent or legal guardian, as appropriate, and assent was obtained from each child (if applicable).
Vertex Pharmaceuticals Incorporated is committed to advancing medical science and improving the health of people with cystic fibrosis. This includes the responsible sharing of clinical trial data with qualified researchers. Proposals for the use of these data will be reviewed by a scientific board. Approvals are at the discretion of Vertex Pharmaceuticals Incorporated and will be dependent on the nature of the request, the merit of the research proposed, and the intended use of the data. Please contact CTDS@vrtx.com if you would like to submit a proposal or need more information.