Those with an eye on developments in healthcare and pharmaceuticals know that personalised medicine – where treatments are tailored to individual patients, rather than administered in a standard way across a group of patients – is already with us, and acknowledged as a huge challenge. Yesterday, I realised that ‘huge’ doesn’t do go nearly far enough…
As researchers learn more about the biology of what is currently recognised as one disease they necessarily split it up and re-define it as multiple new diseases. Sadly, I am without a PhD, but I gather that the science points to this genetically inspired process creating literally billions of ‘new’ diseases and that we won’t be talking about disease ‘types’ for much longer.
Yesterday I attended the Pancreatic Cancer UK “Summit” in snow-bound central London (http://flres.uk/2oBu8oq) . A leading researcher talked about how they are discovering, as they break down the genetics of pancreatic cancer, that “we are probably talking about 10 or more different diseases, not just one”. Regrettably, there is almost no useful way for clinicians and patients to react to this news: existing treatments are few; the R&D pipeline is more or less empty; outcomes have not improved significantly in 50 years; and it remains amongst the least survivable cancers.
On the way home I read that diabetes might best be thought of as five diseases rather than two (http://flres.uk/2FdDujX), and that according to UCL based oncologist Dr Victoria Salem, “… it may well be that worldwide there are 500 subgroups depending on genetic and local environment effects. Their analysis has five clusters, but that may grow.”
Clinicians, patients, charities, researchers, and carers always welcome such news but what of the lag time between these laboratory based revelations and the delivery of personalised medicine to patients. Whilst an R&D lag has always existed, the landscape is now more confusing than ever, and the implications are potentially profound:
- The astoundingly complex science has to be decoded by the pharmaceutical and medical industries and then jump multiple clinical trial and regulatory hurdles. It takes years to do this for one medicine, with no established method of batch processing! In any case, approving and manufacturing billions of drugs obviously can’t work, so presumably it is the process by which personalised medicine is turned from genetics into a deliverable that would receive formal approval in future, rather than the end product itself?
- In the UK, we have a health system under immense budgetary pressure, set to increase as the population ages, presenting a funding crisis. We can’t afford personalised medicine, but even if we could, saving more lives self evidently increments our collective longevity, which I’m pretty sure would only widen and deepen the money part of the problem?
- There is already a knowledge deficit amongst health professionals in the face of a torrent of new clinical information, which personalised medicine intensifies. In order to cope – doctors habitually mentally pigeon-hole patients into de-facto groups, and treat at that level. This behaviour has both a psychological and cultural dimension, and will be difficult to resolve. In any case, a bigger question emerges as to the role of medical opinion in a world with billions of diseases: the outcome of a patient’s genetics will predetermine precisely his or her treatment, so what role for the physician?
- And the rest..!
The science is light years ahead of our ability, as a society, to keep up. Political leaders of all persuasions will, rightly, choose to focus on diseases which affect the many (like diabetes), rather than the few (like pancreatic cancer). And yet I suspect even with this funneling of resource, we will need something truly revolutionary to happen somewhere along the R&D >> approval >> delivery pathway. In the past this has been the cue for innovative pharmaceutical companies to step in and save the day, and of course they are deeply engaged with personalised medicine both clinically and commercially. Yet the reality for them, I think, is that it is a complex and long term strategic issue competing with several very threatening short to medium term ones (like Biosimilars, Amazon & co, NICE etc.).
For those in-the-know this piece may have been without fresh insight. I felt motivated to express my view because the more I reflect on what personalised medicine might ultimately mean, the more it transitions from ‘huge’ and to ‘awesome’ in my mind. I’d go so far as to label it the biggest strategic problem I’ve seen in 20+ years of trying to understand the healthcare and pharma landscape.
John Aitchison, MD at First Line Research, firstname.lastname@example.org