Building global development strategies for cf therapeutics during a transitional cftr modulator era

Area of Science:

• Pulmonology
• Pharmacology
• Clinical Trial Management

Background:

• Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulator therapies are revolutionizing CF care.
• Unequal global access to these therapies and evolving care standards present challenges for developing new CF treatments.
• A robust pipeline of CF therapeutics is essential for maximizing patient benefits.

Purpose of the Study:

• To identify global uncertainties in CFTR modulator drug development.
• To explore paths forward for advancing CF therapeutic development.
• To foster collaboration within the global CF community for optimizing therapy evaluation and approval.

Main Methods:

• Summarized counsel from international core advisors and clinical trial networks.
• Convened a one-day workshop in October 2019.
• Focused on the transitional era of CFTR modulator availability.

Main Results:

• Identified areas lacking global alignment in CF drug development.
• Highlighted the need for harmonization to improve study designs.
• Recognized the importance of collaboration for future therapy advancement.

Conclusions:

• Global collaboration is essential for advancing cystic fibrosis therapeutics.
• Harmonizing drug development processes will enable more efficient and ethical clinical trials.
• Addressing access disparities and evolving care standards is key to ensuring maximal benefits for all people with CF.