This is the second of my two thoughts about pressures being faced by patient advocacy.
There have been a number of recent articles in journals lamenting the failure of patient advocates to respect the principles of evidence-based medicine (EBM). It is a consistent low grumble from the science community, to which a small group are deeply wedded, while a majority probably have some sympathy with the point but also have some sympathy with the stance being taken by patient advocates.
There has been a change in the attitudes of regulators. Not that long ago they too were wedded to the evidence-base and they would maintain that this principle still underpins their decision-making. But in that decision-making, and therefore in the processes and procedures they employ, they are being more pragmatic. That has been partly driven by patient advocacy.
Some voices in the regulatory community are also identifying the issue which I believe needs to be addressed and which EBM protagonists seem to be studiously ignoring. It is quite a big question.
Are we gathering the right evidence in clinical trials and in the way we measure and report real-world clinical care?
No-one in medical research seems to be addressing this effectively. A few are tinkering around the edge of the issue and for some, the idea that we are not gathering the right evidence, is like voicing heresy.
Cancer clinical trials are focussed on some measure of survival or tumour response to treatment. The so-called ‘gold standard’ measure of Overall Survival becomes difficult when a treatment is one of a succession of different therapies. A wide range of surrogate measures are used as a substitute. These are often dependent on some degree of investigator interpretation so are inherently unreliable, a factor which it is convenient to forget. Side effects of treatment are an important issue and these are reported too, usually using an investigator applied judgement of severity against the CTCAE standards.
All this is a focus on objective measurement of the disease and the effect the treatment has on the disease. No-one is denying that this is important but where are the measures focussed on the patients themselves and the effect on them, their subjective reporting.
This is what patient advocates have been pursuing with their regulatory campaigns which the EBM stalwarts claim ignore the evidence-base. The advocates have an evidence-base in patient stories and anecdotes, but it is not one which they are in a position to measure, the professionals are not measuring it and have been ignoring it for more than twenty years. So the EBM complainants, writing stridently about patient advocates not playing their game, would do well to look at themselves and their colleagues.
Are they asking all the right questions: are they gathering all the right evidence?
There is a side issue here about patient involvement. It is an issue which patient involvement cannot easily address and could be used as an excuse for inaction. Untrained involved patients do not necessarily see this ‘right evidence’ issue, they are often overwhelmed with understanding the planned outcomes and endpoints in a study. This assumes, of course, that they are consulted at a timepoint when they can actually input such ideas.
The second side issue is that we have much better ‘big data’ now than we have ever had. The ability to look at clinical practice across years, aggregating the experience of thousands of patients, means that retrospective analysis can provide valuable information and new insights. The question is, are we asking all the right questions in these analyses if the right questions were not asked when the data was being first gathered?
So the challenge for the data analysts is not to turn EBM into a self-justifying paralysis of ideas which ignores the patient?