Qualitative evaluation of the Canadian Fabry Disease Initiative using key informant interviews.

摘要

The objectives of this study were to evaluate the Canadian Fabry Disease Initiative (CFDI) using input from key informants. A qualitative strategy composed of interview transcripts, a holistic-inductive design and content analysis was used. Within group analysis suggested: (1) patients are concerned about the restrictions on access to therapy; (2) CFDI investigators believe the database and monitoring are essential components to treating rare diseases, but the CFDI is not the ideal model; (3) Provincial representatives believe research should not be a foundation for drug access; and (4) pharmaceutical representatives perceived the CFDI as a poorly designed answer to a reimbursement problem. Between group analysis revealed that the CFDI as an important initiative in Canada. However, it is not the solution to many of the issues related to orphan drug reimbursement. Overall, no group was completely satisfied with the CFDI therefore it should be redesigned to better accommodate each group's needs.