The COVID-19 pandemic has led to a surge in clinical trials around the world, but also set critical challenges for researchers.
The pandemic represents a moving target, as countries see cases spike and decline in unpredictable ways, making it difficult for trials to enrol patients. At the same time, hospitals are struggling to accommodate research while also managing critical staff and equipment shortages.
It’s in this shifting and challenging context that a state-of-the-art Australian trial, REMAP-CAP, supported with a $2 million grant from Minderoo Foundation alongside the NHMRC and other funders, has managed to demonstrate the power of a new, cutting-edge approach to clinical trials to deliver results.
REMAP, which is an acronym for Randomised, Embedded, Multifactorial, Adaptive Platform trial, was designed with the flexibility and agility in mind to respond to a global pandemic. It utilises a Bayesian statistical model to constantly tweak and improve the therapies it tests on patients, which in turn allows faster results with less participants.
This intensively data-driven approach represents the next generation of clinical trial design, and is a model Minderoo Foundation has supported in other areas of medical research, including Australia’s $100 million Global Brain Cancer Mission.
It delivers faster, more efficient breakthroughs, as well as benefiting patients in the trials.
Using this process, multiple drugs can be tested at once. Patient data is continually assessed to identify treatments that are working, and to drop other treatments that show no benefit. This feedback loop radically speeds up results, and also means patients in adaptive trials are more likely to receive treatments that show some promise of working.
This contrasts with old, linear trial designs where a drug was tested across an entire patient sample even if early results demonstrated little or no benefit.
REMAP-CAP’s research team at Monash University have already enrolled around 900 patients to test potential COVID-19 therapies, at over 250 sites around the world including in Australia, Canada, France, Ireland, the Netherlands, New Zealand, the United Kingdom, and the United States. In a few short months they have been able to set up COVID-19 trials to test antivirals, immune modulators, macrolides, anticoagulation, convalescent plasma, and vitamin C.
Last month, data from around 400 ICU patients in the trial who were given the common steroid hydrocortisone showed strong evidence that these cheap drugs can save the lives of critically ill COVID-19 patients. Using this data, combined with published and pre-publication data from other similar trials, the World Health Organisation has revised its clinical guidelines and recommend corticosteroids as a standard treatment for COVID-19.
REMAP-CAP’s data comes at a time of increasing concern about shortages of the drugs dexamethasone and Remdesevir, adding another weapon in the arsenal of front line healthcare workers, particularly in lower-and-middle-income countries.
The principal investigator for REMAP-CAP, Professor Steve Webb, has also been nominated by Minderoo Foundation to serve on the scientific advisory committee for the International COVID-19 Data Research Alliance, a global effort to improve research collaboration.
Data sharing is a pillar of Minderoo Foundation’s approach to health research. In September last year Minderoo Foundation launched a collaboration with Harvard and MIT based research institutes to collect and aggregate genomic data from brain cancer patients, called Count Me In.
The COVID-19 Data Research Alliance has likewise built a trusted and secure cloud-based platform that allows scientists to share, compare, and analyse research findings, supported by the Gates Foundation, Wellcome Trust, Minderoo Foundation and other partners.
These new models promise to not only deliver breakthroughs for COVID-19, but also generate a positive legacy from this crisis, cementing the next generation of sophisticated, collaborative research efforts to accelerate medical science.