Kalydeco, Creon, even physiotherapy - none of these amazing developments in cystic fibrosis (CF) treatments and care could have happened without clinical trials.
A lifetime of change
On day one of CF Week, we spoke about campaigning and how we are working with the community through our Stopping the Clock campaign to support access to vital CF treatments. But Stopping the Clock isn't just about campaigning for access - it means working hard to support the clinical trials that are vital for ensuring new treatments make it to market.
In 1999 we began our London CF Collaborative Study, which helped doctors to predict lung function decline in adolescence and identify when medical intervention was necessary.
Six years later in 2005, the Trust-funded TOPIC study helped cut down treatments for patients after discovering that intravenous antibiotics could be given once a day instead of three times.
Just last year the first UK CF Registry-based clinical trial, CF START, was launched – recruitment has begun and the trial will be assessing whether prescribing preventative antibiotics to infants with CF reduces infection, or risks exposing children to earlier infection from harmful bacteria. And let’s not forget that all of this was made possible by people with CF agreeing to take part in clinical trials.
Find out more about the impact of clinical trials in our video on five treatments that have transformed CF care.
We launched our CF Insight Survey in 2016 to help build a picture of what CF looks like to the people living with it every day. Feedback from almost 900 people with CF and their friends and families told us that 40% of adults had participated in at least one clinical trial. Not only that, but over 50% of those same adults reported that they found participating in a clinical trial to be a generally positive experience.
However, we learnt that poor communication during the trial process was an issue for people taking part, and contributed to 23% of adults reporting that their experiences of clinical trials were not positive.
During last year’s UK Cystic Fibrosis Conference, we launched the Clinical Trials Accelerator Platform, an initiative taking place across the UK that is working with CF centres to increase participation in clinical trials, make them more beneficial to take part in and ensure that they are easily accessible. To increase communication with people taking part in trials, we are recruiting Trial Coordinators at selected centres and are making trial result summaries available through our brand new Trials Tracker database, which will be available as of autumn this year.
The development of precision medicines is one of the biggest advances we’ve seen in CF treatment so far, and drugs like Kalydeco and Orkambi could never have been made if it weren’t for clinical trials. Check out our brand new animation for more about the different mutations of the faulty CF gene, and how precision medicines can work to help treat some of them.
We’re committed to putting people with CF at the heart of our journey towards making clinical trials more accessible. Patient and Public Involvement, or PPI, is one of the major elements driving our Trials Accelerator.
We are still recruiting people from the community for two of our three PPI groups – Peer Advocates and the Focus Group (our Lay Reviewer group is already underway). We spoke to Lorna Allen, PPI Coordinator at the Trust, about how PPI works and how people with CF can get involved and really make a difference to clinical trials.
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Don’t forget to check back tomorrow for day five of CF Week, when we'll be celebrating Wear Yellow Day in style!
Setting up a regular gift today helps to support our campaigns and ensures we can plan ahead and react to CF breakthroughs as they happen.