Research Milestone Forecasting in Cystic Fibrosis

:Background: Cystic fibrosis (CF) has seen important breakthroughs in the last few years. Physicians, experts, and patients anticipate further treatment advances in the coming years.Methods: In October 2021, CF experts in the US and Canada were asked to provide forecasts about 8 milestones in cystic fibrosis research in an online survey (Canadian experts provided forecasts about two additional milestones about healthcare system outcomes). CF experts were identified using lists of CF clinics available from major CF advocacy groups in the US and Canada. We randomized experts into two different experimental arms, comparing two different forecast elicitation approaches. Results: 81 experts completed the survey. Forecasts were not particularly optimistic about near term breakthroughs, though many events, including approval of anti-inflammatory drugs and approval of an amplifier drug were viewed as having at least a 40% chance of happening in the next 6 years. Experts were still less sanguine about other events, including FDA approval of effective antibiotics for B. Cepacia, FDA approval of a therapy for those with stop mutations on both alleles and a Phase II trial testing a gene therapy (25%, 27%, and 27% probability of not occurring at all within the next 10 years). Overall, both elicitation methods produced very similar forecasts. Conclusions: Experts regard important advances like genetic therapy as unlikely to occur within the decade. However, experts are very optimistic about healthcare system access to new treatments, at least in Canada.