Core Outcome Set Taskforce for Cystic Fibrosis (COST-CF): a patient-initiated core outcome set for CF

People living with cystic fibrosis (CF) want meaningful and consistent outcomes in clinical trials. Clinical trials compare a drug with a dummy treatment, or one treatment with another. To decide which treatment works best, researchers choose outcome measures – just as the fastest time decides who wins the 100-metre sprint. Outcome measures, used in CF trials, include spirometry, weight and quality of life. Many CF trials have few participants and lack statistical power. We can combine small trials to have a large enough sample size to answer the question but only if the same outcome measures were used in each trial. This often does not happen. The student will jointly deliver a project to agree a universal core outcome set for CF: a shortlist of outcome measures to be used in every CF trial. Our universal core outcome set will allow trials to be compared and results combined. This will help decide more easily which treatments work best. This project will be co-produced with the patient community and will also involve stakeholders from academic, industry and healthcare delivery. Once the universal core outcome set is agreed, we will work make our core outcomes the norm in every CF clinical trial

Background. The COST-CF project arose because people living with cystic fibrosis (CF) said they want meaningful and consistent outcomes in clinical trials. Clinical trials compare a drug with a dummy treatment, or one treatment with another. To decide which treatment works best, researchers choose outcome measures – just as the fastest time decides who wins the 100-metre sprint. Examples of outcome measures, used in CF trials, include spirometry, weight and quality of life. Many CF trials have few participants. Larger numbers are needed to find the best treatments. We can combine small trials to answer the question but only if the same outcome measures (measured in the same way) were used in each trial. This often does not happen. Furthermore, now that modulator drugs are widely used, we may need more sensitive outcome measures to compare treatments.  

Aims. We will agree a universal core outcome set for CF: a shortlist of outcome measures to be used in every CF trial. Others may devise core outcome sets for specific types of trials (e.g. for CF-related diabetes) which would contain our universal outcomes plus other specific measures (e.g. blood sugar) – see figure 1. Our universal core outcome set will allow trials to be compared and results combined. This will help decide more easily which treatments work best. 

Design & Methods. This project will be co-produced with the patient community. We will involve a range of people including: patients and families; health professionals; researchers; drug companies; editors of medical journals; healthcare funders; and drug regulators. When we have agreed which outcomes are most important, we will decide how they should be measured. 

Dissemination. Once the universal core outcome set is agreed, we will work with everyone in the CF and scientific communities to make our core outcomes the norm in every CF clinical trial.