One of the challenges of patient advocacy is developing a balance between addressing immediate patient needs and creating a future which addresses the issues which patients see as important but which take time to change.
A good current example of the latter is the demand for earlier diagnosis. We have been voicing the need for twenty years to my knowledge and now it is government policy, cancer charities support sit, Public Health England has taken note, and the NHS is starting to consider how it can be done. It will take another five years before technologies, manpower and systems are fully in tune with the concept. One of the bigger changes involved is shifting the behaviour of some GPs.
When we discuss patient involvement we do not draw any distinctions between patient advocacy for the short term and the longer term. The opportunities for involvement in research range from reviewing patient information for a simple research study to providing patient perspectives on multi-million pound strategic projects, with just about anything in between.
Lets be clear, the review of information about a ‘simple’ research study is important and valuable, just as informing those making multi-million pound decisions is valuable. Similarly the support for a patient having problems accessing a new treatment is valuable. These are all roles in the whole context of patient advocacy.
There is no scale of value in this but we do need to understand the differences and the implications that these differences bring with them. The main ones are the ability of the advocate, the training they have had and perhaps most importantly, their life experience prior to being involved in work as a patient. We do not assess these when we embark on involvement, and perhaps we should not do so as many people will grow into roles which are initially unfamiliar and their skills will rapidly develop given the chance to do so.
So I can hear you ask, why are you raising this issue?
There is one area where we are approaching a crunch. The dichotomy between patient advocacy viewpoints of short term issues of access to new treatments and the long term issues of the value of new treatments. We want a patient who believes there is value in a new treatment and whose doctor agrees, to have access to that treatment. At the same time we want to see the evidence of value in that treatment so that it can be appropriately used in the clinic in the future. Regulation is moving fast to accept data from short term Phase 2 trials with ‘surrogate’ endpoints, rather than favouring randomised Phase 3 trials (RCTs) offering statistical significance of effect based on an Overall Survival endpoint (OS). A short versus long term conflicting view.
Seeing new treatments come faster into clinical use meets the demand of most patient advocates. However most of them also fail to consider the cost in terms of long term understanding, the increase in risk which is implied, and the inability to control the cost of treatment through a meaningful understanding of value. Many advocates also welcome the reduction in the numbers of RCTs with placebo comparators; the placebo is generally disliked and there are questions about using data from such trials in the wider treatment context, something which information technology is increasingly enabling.
How do we move forward through this dichotomy? Quick and easy access versus long term validity and value.
The route being proposed by professional bodies, supported it must be said by many patient groups, is to develop means and methods to gather ‘real world evidence’. The idea is that a treatment can be given early access to clinical use. Data gathered from that use is analysed and used to review value, changing authorisations, pricing etc as seems fit. Many patients are stunned when told that does not already happen, so making it happen could be best described as ‘common sense’.
The means and methods do not currently exist. Let us see whether policy makers and regulators can make it happen. Maybe there will be subtle resistance from the pharmaceutical industry which currently gets it own way in such decision-making. I believe it is in patient interests to make it happen.
That is a long-term advocacy project.