Table 3. Considerations for combining CF and NCFB disease populations for CF therapeutic development.

Summarized responses from workshop attendees surveyed regarding considerations when combining CF and NCFB populations for clinical trials of symptomatic therapies. Table indicates rank order of preferences in which more than 50% of respondents felt that consideration is important. NCFB=non-CF bronchiectasis; NTM=non-tuberculous mycobacterium; IV= intravenous; FEV₁=forced expiratory volume in one second.

	Hydrators/Muco-active Therapies	Anti-inflammatories	Inhaled antimicrobials	NTM therapies
Target Characteristics within the CF population
	Produces mucopurulent sputum on a regular basis (at least 3X/week) Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Requires IV antibiotics at least yearly	Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Requires IV antibiotics at least yearly Produces mucopurulent sputum on a regular basis (at least 3X/week)	Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Requires IV antibiotics at least yearly Produces mucopurulent sputum on a regular basis (at least 3X/week)	Persistent NTM infection
Target Characteristics in the NCFB population
	Produces mucopurulent sputum on a regular basis (at least 3X/week) Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Multi-lobe involvement by CT scan or x-ray Requires IV antibiotics at least yearly	Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Requires IV antibiotics at least yearly Produces mucopurulent sputum on a regular basis (at least 3X/week)	Produces mucopurulent sputum on a regular basis (at least 3X/week) Requires acute antibiotic interventions (oral, inhaled or IV) at least twice yearly Requires IV antibiotics at least yearly Continues to perform airway clearance and use other inhaled maintenance therapies daily	Persistent NTM infection
Recommended Sub-populations to Exclude in NCFB population
	Traction bronchiectasis in setting of fibrosis Bronchiectasis associated with chronic aspiration Bronchiectasis associated with active mycobacterial disease Bronchiectasis due to COPD Bronchiectasis due to immune deficiency or connective tissue disease	Traction bronchiectasis in setting of fibrosis Bronchiectasis associated with chronic aspiration Bronchiectasis associated with active mycobacterial disease Bronchiectasis due to COPD Bronchiectasis due to immune deficiency or connective tissue disease	Traction bronchiectasis in setting of fibrosis Bronchiectasis associated with chronic aspiration Bronchiectasis associated with active mycobacterial disease Bronchiectasis due to COPD Bronchiectasis due to immune deficiency or connective tissue disease	Traction bronchiectasis in setting of fibrosis
Novel/Unique and Common Endpoints to Consider
	Lung clearance index Mucociliary clearance FEV1 HRCT/imaging Sputum analytes (e.g., rheology, etc)	Mucociliary clearance Lung clearance index Pulmonary exacerbation rate FEV1 Sputum analytes (e.g., rheology, etc)	Mucociliary clearance Sputum analytes (e.g., rheology, etc) Pulmonary exacerbation rate FEV1 Lung clearance index HRCT/imaging	Sputum microbiology HRCT/imaging Research biomarkers (e.g., urine tests)