14/52 Patients need the best possible research
A common patient advocacy ask is: more research into our condition. While there is an obvious logic to it- we will not progress in areas where we don't conduct research- there are also obvious limitations. Firstly, has anyone ever had 'enough' or 'more than enough' research? So, this is a bottomless pit. Then, even if societies increased their research budgets, these will always be limited in nature. And then, it invariably pitches conditions against each other- an approach I call the 'no one loves us' approach ( the classic 'x money gets spent on condition y that only concerns z amount of people, while no one spends money on us').
And research prioritisation into areas of high unmet need aside- this goes beyond single conditions and is about fairness, justice and resilience- there is obviously Einstein's famous
'The definition of insanity is doing more of the same and expecting different results'.
As patient advocates, that means we should not only be aiming for research into undeserved areas but above all, for the *best possible* research in our condition.
*Best possible* is obviously hard to define but covers what is researched and how it is researched and then, how those research findings are used.
In our thinking, 'best possible' means research
that addresses a problem addressing important patient need,
that is conducted in a way ensuring maximal clinical relevance, optimises translation and resource utilisation
whose results can be and are used where they are needed
Addressing the relevant problem is not only a matter of research prioritisation 'what to study' but also about how to accurately phrase the research problem. Most research into resistance to treatment- one of the biggest challenges for us in Melanoma- currently addresses the question: 'what is a possible mechanism for Melanoma to become resistant to therapy?' rather than the relevant one which would be 'what is the clinically relevant resistance mechanism in Melanoma that we can target'?
Anticipating translation and introduction into the healthcare system are critical steps to turn research findings into patient benefit and are critically determined by experimental design, including the choice of model systems, sufficient statistical power and steps to ensure clinical validation of findings. My pet peeve in this area would be 'exploratory biomarkers' in a tiny number of samples without any thought or strategy given to validation. This will definitely be another post!!
The utilisation of research results might not seem an obvious topic that a research project should address. This however becomes a dangerous misconception when it comes to e.g. clinical research. If know that cost is a barrier for patients to access new treatments or diagnostics and we want the largest possible impact of our solution, it has to be accessible for the largest possible number of patients and that means our solution cannot be costly.
If we want to develop a new diagnostic tool to inform treatment choice in advanced cancer, we have to keep in mind that advanced cancer can be growing very fast and that any clinician would like to initiate treatment as fast as possible, either directly or the next day. This means that the ideal diagnostic for these patients should give a clinically actionable information within 24h- not in 6 months. There is actually quite some literature out there how clearly defined constraints can help, rather than inhibit innovation- surely something we as patient advocates should care about.
It is important to remember that failure is an important part of research, that research does not always deliver the expected results (but then, quite something else), that one needs both curiosity-driven (solution in want of a problem) AND problem-driven (problem in want of a solution) research and activity across the entire research spectrum, from very basic over applied to implementation research.