Presently, there are no validated regulatory endpoints to advance new therapies for populations with non-cystic fibrosis bronchiectasis (NCFBE) with or without non-tuberculous mycobacterial (NTM) lung infection. Previous drug development in NCFBE populations has not met consistently endpoints related to pulmonary exacerbations or to existing patient reported outcomes (PRO) instruments, e.g. Quality of Life Bronchiectasis, or St George?s Respiratory Questionnaire (SGRQ) tools. This project will work within the framework of the drug development tool qualification process at the FDA to advance a novel PRO to ultimately qualify for drug development and regulatory decision making. A content-valid draft PRO instrument will be developed via preliminary PRO instrument development activities, undertaken through targeted literature reviews, expert clinical input, qualitative concept elicitation, and evaluation and refinement via cognitive interviews (CI), translatability assessment, and ePRO usability testing. Because the exact nature of the relationship between the symptom-related experiences of NCFBE patients with and without NTM infection is currently unknown, evidence arising from the proposed PRO instrument development process will help determine whether a single PRO instrument can be created that is content valid for both patient groups via qualitative examination of unique aspects of NTM infection. Psychometric validation of the new PRO(s) will be achieved via: 1) a non-interventional validation study (NIVS) to collect longitudinal daily diary data to validate the scale including NCFBE patients with and without NTM infection, and 2) planning for further evaluation in clinical trial settings to assess the instrument?s sensitivity to detect change in patient state indicative of clinically-meaningful responses to therapeutic intervention.
The project aims to develop a novel patient reported outcomes (PRO) instrument on symptoms from patients with NCFBE with or without NTM that is advanced to the stage of readiness to allow qualification for drug development and regulatory decision making. This project will conduct qualitative research to develop a content valid draft PRO. This will be followed by a quantitative non-interventional study to validate and to advance the draft PRO instrument to a stage of readiness for future interventional trials and final qualification.
