Table 1.

Executive Summary of the Workshop to Support Global CF Therapeutic Development

Kev Priorities to Support Future Global CF Therapeutic Development Efforts

The future of CF drug development will depend on frequent and transparent communication between stakeholders across industry, academia, patient organizations, regulatory agencies, and community representatives to define pragmatic yet rigorous, clinically informative pathways for future drug approval. CF clinical trial groups must take an increased role in providing industry sponsors data across multiple therapeutic development programs to support early development planning, including: Contemporary assessments of the CF population targeted by a new therapy Descriptions of relevant and, if applicable, competing standard of care (SOC) across geographic regions Estimates of the size and composition of the target population(s) as supported by global registries Input from the broader CF community on the willingness of providers and persons with CF to participate in future clinical trials In particular for studies requiring periods of interrupted SOC Input on the most relevant clinical endpoints to establish efficacy of the candidate therapy and how these endpoints are impacted by changing or differential SOC Input on the clinically meaningful effect sizes both in the presence and absence of relevant SOC Development of pragmatic, ethical and feasible regulatory packages of new candidate therapies should include: Individual studies that will support the requirements of multiple regulatory agencies in order to efficiently utilize a population with an orphan disease Study designs that minimize or avoid SOC interruption, with feasibility and safety of these studies supported by formal input from the CF community and available data on the clinical ramifications of SOC withdrawal Sensitive biomarkers to support mechanism of action and dosing decisions Clinical endpoints that can be measured over durations that align with feasible and ethical study designs Expedient approaches to enable extrapolation of an initial approved indication to populations who can safely benefit from the new therapy, in particular for pediatric populations and those ineligible for current effective therapies Innovative pathways for drug development in small sub-populations for whom traditional trial designs are not feasible