The Kids researchers have been awarded over $3.4 million for a new trial to pioneer improved ways for managing cystic fibrosis (‘CF’).
The BEAT-CF (‘Bayesian Evidence-Adaptive Trial to optimise management of Cystic Fibrosis’) trial has been awarded a 5-year grant by the Australian Government’s Medical Research Future Fund through The University of Western Australia.
This is the first WA research project to receive a Medical Research Future Fund grant.
In a watershed approach to optimising care of people with rare and complex diseases, the BEAT CF trial will simultaneously evaluate a range of treatment strategies used in CF and will quickly eliminate those found to be inferior to pave way for more effective available alternatives.
CF treatment typically involves management across a range of therapeutic domains – including antibiotics, clearance of airway mucus, immune modulation, and nutritional.
To date, traditional trials have only assessed two treatment strategies at a time, which can needlessly delay improvements in survival and quality of life for participants.
Dr Tom Snelling from the Wesfarmers Centre of Vaccines and Infectious Diseases at The Kids said that this type of clinical trial is the best way to determine which treatments work, and which ones don’t.
“We are pioneering a new approach to clinical trials, called an adaptive platform trial. This adaptive trial is a type of ‘learning health care’ model, in which we aim to learn as quickly as possible which treatments are best, which treatments aren’t, and in whom.
“BEAT CF aims to not only directly improve the care of those who take part in the trial – but hopes to provide lasting benefits to CF patients in the future.”
BEAT CF will be a collaboration involving patients with CF from across Australia. The trial aims to make participation available to as many children and adults with CF – and those who participate will agree to allow their treatment data to be used to work out which CF treatments are best.
Dr Andre Schultz, Director of the Cystic Fibrosis Centre at Princess Margaret Hospital for Children, said BEAT CF will revolutionise the management of cystic fibrosis and the MRFF funding will make it possible.
Despite gradual improvements in outcomes for people with cystic fibrosis (CF), as for many rare and complex diseases, there are still many uncertainties and much variation in management.
The average life expectancy of a child born today with CF is still under 50 years, and some will die in early adulthood. It’s hoped this trial will improve this.
“While the life expectancy for a child born with CF today is much better than 20 years ago, there is still a lot of opportunity for improvement in care if more patients can be encouraged to participate in trials,” Dr Snelling said.
Read Minister Hunt’s media release here.