Conclusion
The year 2020 has brought many exciting developments in the field of CFTR modulators, including the use of currently available modulators for other mutations, and exploration of the efficacy of modulators on alternative endpoints, such as LCI, nitric oxide, cardiopulmonary exercise testing, and intestinal organoids. Special populations studied included those with FEV1pp<40% and FEV1pp≥90%. Adherence and real-world studies were reviewed. The effects of CFTR modulators on pancreatic function, diabetes, sex differences and fertility were also reported in various studies. Due to the cost analysis and worldwide outcome data suggesting differential efficacy and eligibility for currently available CFTR modulators, there is a continued need for new therapeutic avenues and unique research designs. Real world evidence and patient involvement in research priorities will continue to be important, as ways to increase access to appropriate and effective therapy is the overarching goal of CF care.