It may not have escaped your notice as you travel between different areas of the hospitals in which you work that there appear to be some things that have more clinical trial activity going on than others. There have been many things written on why this might be, including a very persuasive paper* that argues for the reduction in health waste by the better integration of clinical care and clinical trials, and a claim that trials are an ethical imperative.
Yet not an awful lot of folk are on-trial. Why?
Obsessive, picky and self-centred as some paediatric oncologists are, they’ve also got a massive history of undertaking stunningly effective studies across the heterogeneity and rarity of malignancies that affect children. They, with such a richness of trial heritage, complain about the inadequacies of trial uptake. And with the sort of terrier-like determination that has improved survival from ~10% to ~80%, they have dug into what might be a problem.
There’s a wider literature from trials in general, and a focus on trials in children and teenagers/young adults that suggest that studies need to be:
In order to enter a study, there needs to be one. If no-one wants to look to see how to improve the management of chronic constipation, there will be no trial, and likely to be little improvement in managing the condition.
Trials need to be accessible – that is, open to enough folk who can reasonably be on them. That may be geographical, or it may be age limited. As recently modified in the Experimental Cancer Medicine Centres in the UK, there is now a strong requirement to biologically justify any exclusion made on the basis of age.
An open study will be empty is no-one knows it’s there. So trials need to be sold, like drugs and guidelines and new books.
And when it comes to trials for children and young people, they need to be manageable, without onerous testing or outcome measurements detracting from the quality of life of the participant.
When it comes to looking at really experimental stuff, the phase 1 and phase 2 trials, then the stakes are even higher when it comes to rarity, “first do no harm” and expectation. There is really very very little examining why young people enter such studies, but if we take what adults say as being an approximation, then the themes seem to be:
- Potential for health benefit by ‘cutting edge’ treatment
- Trust in the clinical team
The elements that work against trial participation include
- Financial or practical barriers (geography, multiple trips for tests, awkwardness of timings)
- Community attitudes (of family members or community leaders)
- Lack of accessibility through inappropriate co-morbidity exclusions, age bars or requirements for literacy
If we are to improve the access to clinical trials we need to take seriously these issues and work to make ourselves aware of the studies on offer, engage in making new studies appropriate and accessible, and use our status as trusted brokers to offer – not force – our patients the opportunities to take part. Doing this will make the evidence we practice on richer, relevant and more plentiful so we don’t so often need to ask what to do when there is no evidence.
* Massive CoI: I’m an author on the paper.