Labisa, Pedro
Andreozzi, Valeska http://orcid.org/0000-0001-6478-8456
Mota, Melina
Monteiro, Susana
Alves, Rita
Almeida, João
Vandewalle, Björn
Felix, Jorge
Buesch, Katharina
Canhão, Hugo
Beitia Ortiz de Zarate, Igor
Funding for this research was provided by:
PTC Therapeutics
Article History
Accepted: 5 September 2021
First Online: 3 October 2021
Declarations
:
: PL, VA, MM, SM, RA, JA, BV, and JF are employees of Exigo Consultores. Exigo Consultores provides consulting services to PTC Therapeutics under a fee-for-services contract. KB, HC, and IBOZ are employees of PTC Therapeutics.
: This study was funded by PTC Therapeutics. Authors affiliated with Exigo are employees of an independent consulting firm and maintained independent scientific control over the study, including data analysis and interpretation of final results.
: COIDUCH was developed following a direct request by the National Authority of Medicines and Health Products (INFARMED) after the reimbursement decision for Translarna® in Portugal. Following this request, the study protocol was developed and sent to INFARMED, which validated it. Note that, since 2014, the Ethics Committee for Clinical Investigation (CEIC) has been working with INFARMED to approve nationwide studies whenever a local ethics committee for health (CES) has not been designated for the purpose. This was the case with COIDUCH, as it did not involve any hospital (where CES are often located). Ethical principles and good clinical research practices guided the execution of this study.
: Prior written informed consent was obtained from participants. The anonymity of the participants was guaranteed. All personal data were managed according to the General Data Protection Regulation.
: Not applicable as the manuscript does not contain any personal data related to an identified or identifiable participant.
: An Excel database was developed in which to register the original data from the questionnaire, and the software R was used to analyze the data. All aggregate data generated or analysed are included in this published article. No individual data are available to protect the anonymity of individual patients.
: Not applicable.
: PL and MM designed the study, collected the data, and drafted the manuscript. VA designed the study, analysed the data, and drafted the manuscript. SM and JA collected the data. JF and BV designed the study and contributed to the interpretation of the data. KB, HC, and IBOZ supported the planning phase of the interviews. All authors read and approved the final manuscript.