MoreTrials: A public campaign to make it much easier to do randomised trials
My colleague, Professor Richard Haynes has contributed to an excellent article on the ground-breaking RECOVERY trial. In the article he made the following important point to keep in mind when designing randomised trials:
You can read the full article here
The Good Clinical Trials Collaborative have set out the key principles that underpin the effective development of a Covid-19 vaccine:
You can read the full piece here.
“The Safer Medicines Trust believes that the provision and completion of robust, randomized clinical trials (RCTs) for medicines underpins successful healthcare decisions and patient safety. Where trials are not started, fully completed or statistically under-powered so results are at best unreliable, patients may suffer unnecessarily. Safer Medicines Trust supports the MoreTrials campaign for transparency in the guidance, governance, execution and reporting of RCTs on behalf of all patients.”
The Safer Medicines Trust
“MoreTrials isn’t a nice to have; it’s a must have. People are increasingly taking control of their health so that we can be co-pilots in our care – rightly so. But current trial set up is a huge barrier to this. Trials take too long to set up, some never start, trial results are often unreliable, few outcome measures reflect what truly matters to the people living with the disease, and some never get reported at all. we’re delighted to be part of the MoreTrials campaign to change this.”
The Brains Trust
“New drug development is always vastly expensive – often, prohibitively so for rare conditions like GIST, where the number of potential patients may not justify the development expense. However, sometimes sometimes it’s not necessary to start from scratch: quite often, drugs for one cancer can be repurposed for another – that’s how imatinib, originally developed for leukemia, transformed treatment of GIST. So, clinical trials of existing drugs for new purposes can be invaluable in abbreviating the development process – and reducing the associated costs.”
GIST (Gastrointestinal Stromal Tumours) Support UK
“As a rare disease patient support charity, we recognise that clinical trials are a crucial but difficult part of our journey as we look for better management and curative options for our community. We need good data to steer our world renowned guidance and advice but we also need to get there faster . Therefore we support the initiative to modernise and simplify the governance of clinical trials which we believe will have a positive impact on our work.”
Chronic Granulomatous Disorder Society
“As a patient support charity group (The Pituitary Foundation) we support MoreTrials initiative. We realise that many trials that should get done don’t even get started, or that too many trials don’t get completed. There is also the added problem that too many trials produce results that are not reliable. The development within MoreTrials of new trial guidelines with an open and transparent manner, can only be helpful to all patients.”
The Pituitary Foundation
“We signed up to More Trials because there is a chance that something might come from it in terms of increasing patient input in trial design. Patients will only get appropriate treatment if clinical trials are well designed. We don’t need more trials, we need better trials!”
The Lyme Disease Action
“As a rare disease patient organisation, Reverse Rett supports the MoreTrials campaign because too many trials that need to be done to alleviate disease aren’t even being started and other trials which do start are often not completed. There are also trials of potential therapies which could infer benefit but which do not demonstrate reliable results, with the opportunity to ameliorate devastating symptoms, lost to those patients forever. This is unacceptable when there are so many devastating diseases, like Rett Syndrome, for which there are no currently no approved treatments. The ultimate beneficiaries of clinical trials are patients; the ultimate contributors to the development of new treatments are patients. Therefore patient voice should and must be at the heart of this campaign.”
Reverse Rett
“We primarily signed up to MoreTrials because I agree that more randomized trials need to be done and that pragmatic randomized trials should be made easier. More efficient pragmatic trials addressing relevant clinical questions – and I would add, using the therapeutic tools we already have – is the best way to improve the health of the sick in my opinion.”
The Anticancer Fund
“The Stroke Association is committed to raising the profile of, and increasing the amount of investment in high-quality stroke research that can make the greatest difference to people affected by stroke. It’s important that we continue to innovate in research to ensure trials have the most appropriate design to answer the question at hand, and innovate our evaluation of evidence to ensure important findings can translate to clinical practice.”
The Stroke Association
“Meningitis Now supports any initiative that will improve the treatment and outcomes for anyone affected by meningitis or meningococcal septicaemia. The MoreTrials campaign is vital to ensure that clinical trials are efficient, effective and produce results that are reliable and useful.”
Meningitis Now
“We support MoreTrials because we all need excellent data to make excellent decisions. Without trials being started, completed and the results peer-reviewed and published, we simply won’t have the data we need to make reliable evidence-based decisions.”
Group B Strep Support
“PID UK supports the MoreTrials campaign because we believe that changes need to be made to how clinical trials are conducted. Many trials that should get done don’t even start and for some the study design lacks the robustness to ensure that meaningful data is produced. Patients should also be embedded in the design of trials from their inception and there needs to open and transparent reporting of negative results so that duplication of effort and resource spend is minimised. The MoreTrials campaign aims to tackle these issues. “
Primary Immunodeficiency UK
“89% of people with visual impairment or blindness live in low or middle income countries. However, almost all the research that tests and develops new treatments for blinding diseases is carried out in rich countries. We urgently need more clinical trials of treatments that can prevent avoidable blindness in the developing world. To be sure that the results of these studies are relevant to real world conditions, the trials must be undertaken in low and middle income countries. Since its inception, The British Council for Prevention of Blindness has supported clinical trials to improve eye care in the poorest countries, and we are proud to support the MoreTrials campaign.”
British Council for Prevention of Blindness
“As an advocacy organization seeking new and improved treatments for mesothelioma and other rare asbestos-related illnesses, we understand the role randomized clinical trials play in realizing that goal in an unbiased manner. Our hope is that the MoreTrials campaign can raise awareness, research and funding needed to start and run randomized trials to completion, which ultimately bring us closer to a cure.”
The Mesothelioma Center at Asbestos.com
Action Duchenne
CRPS (Complex Regional Pain Syndrome) UK
“Thyroid disorders are common and can be highly debilitating. While most people are easily treated, we know that at least 200,000+ UK thyroid patients – and many more worldwide – remain symptomatic. Many tell us they are unable to function with their condition, with very poor quality of life, loss of income and significant physical and cognitive impairment. Trials to date have tended to be small scale and unreliable. We have joined More Trials, determined to have patients’ voices heard in the development of new, well designed trials, which will include patients concerns from the outset and address the issues that patients care about – delivering results that will have real life benefits.”
Professor Rob Califf, Duke and former commissioner at the FDA recently outlined his thoughts on what the clinical trial space might look like after the Covid-19 pandemic. In the talk, Rob outlines the problems with doing large-scale trials and sets an optimistic tone on what might emerge after the pandemic. You can watch the full talk here.
Below are a few highlights of the talk:
“If we are going to treat people well and optimise their chances of a good health outcome, we need to have evidence that is high-quality that we can depend on. Otherwise, we are suffering from ‘eminence based medicine’ where doctors somehow think they know what the best thing is despite the absence of evidence.”
“In cardiovascular disease only 8.5% of major recommendations in clinical practice guidelines are based on high-quality evidence.”
“There are two options following the Covid crisis. We can either make the changes we need now to deal with the emergency hoping we can revert back to the ‘good old days’, or we can take the path of learning from what is happening now and implementing changes in the system that are permanent and move us to much more quickly to the system that is envisioned but not yet enacted.”
Rob Califf
“Clinicians involved in pragmatic trials don’t all have to complete GCP training”
Anonymous
“The value of randomization is undermined by poorly designed and underpowered trials”
Dr Fauci
The talk featured the RECOVERY trial run across the UK which is evaluating a number of treatments for Covid-19. In a recent talk for CTTI – which you can find here (the talk starts at 1:02), Professor Martin Landray presented some of the key findings that have already emerged from this ground breaking trial:
None of us know yet how things will be at the end of the pandemic. That said, perhaps, trials like RECOVERY – focusing on the fundamental principles to answer a question reliably and keep patients’ (and staff) safe – are giving us a glimpse of how trials might get done in the future.