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.