Why the research community needs to rethink – Medical Innovation Bill

By Prof Andy Hall ­–   Professor of Experimental Haematology, Newcastle University

There is a deep-rooted and complacent culture across substantial sections of the medical research community that all is rosy in the garden, that we scientists know what we’re doing and we don’t need any help from outsiders.

Men and women of science should, of course, welcome challenge. It is the grist to the scientific mill – to propose a thesis, and test it, to see if it stands up or falls down. This is what science is about.

But when it comes to medical research and cancer research in particular, there is a sensitivity to challenge that tends towards destructive defensiveness and at times a pig-headed aggression.

For those who want to find new treatments for cancers, this is a problem. Progress in many areas of cancer research has been painfully slow. This is not for want of trying, or of bad intentions, there really are “no villains in this story”. It is because cancers are incredibly hard to tackle.

Many researchers have focused on developing therapies that target individual gene mutations within a tumour. But tumours are often heterogeneous with multiple mutations that will never respond to just one drug.

This focus on the individual gene mutation is in part a legacy of the phenomenal – but one-off – success scientists and doctors had in finding a cure for Chronic Myeloid Leukaemia a form of cancer which is almost unique in having one driver mutation.

Many of us have become fixated on this  success and we have become accustomed to a method of research designed to repeat it.

But sadly it will probably never happen. Unfortunately this means the science community is stuck in a rut. A culture of group-think has taken over and it is reinforced by an endless round of conferences where we clever laboratory kings and queens speak only to each other swapping high-minded clever research papers – all based on the same research methodology.

We need to break out, we need to be challenged and we need to accept that too little progress has been made in finding new cures for cancers.

But the problem is fundamental. My generation, who are now leaders in our research labs, hold our beliefs so dear that any challenge feels like an assault on us as people. Our work and our beliefs are bound up with our identity.

When pushed, doctors and researchers can be genuinely unpleasant to those who challenge them  – I’ve seen this at first hand. But to repel contrary thinking is a derogation of the scientific principle.

One area that must be challenged is the singular and exclusive reliance on old-school and large randomized clinical trials.

Of course, they have their place. But they are slow, cumbersome, expensive and at times wholly inappropriate to the needs of science and, more importantly, patients.

We also need smaller, faster, lighter trials with fewer participants. Trials that are easier to set up, when – as in the case of rare diseases and diseases affecting children – which can be set up quickly, and, crucially, can be adapted quickly during the trial.

We must no longer only rely on trials designed to detect small changes, which need large numbers of patients to find only marginal.benefits.

We need more, smaller, cheaper trials where we look for big effects. And we must record and share the data of success and, very importantly, of the inevitable failures.

Patients deserve this. Particularly, patients with rare diseases where there are never enough patients to fill a large and traditional randomized controlled trial

We need to challenge ourselves and we need to change.

Fortunately new ideas are emerging which are provoking debate and new ways of thinking.

One is the  Medical Innovation Bill. It will encourage doctors to try novel treatments in a clinical environment.

It is not the only answer, though it will certainly help encourage innovation. It is just one solution to a raft of problems that must be resolved. But my interest and support for the Bill is based on this – it is a start.

It is new thinking. It is challenging, it has  inspired debate – as well as anger among some conservative colleagues – that is driving scientists to question themselves and their ways of working.

This has to be good for patients.

About Prof Andy Hall
→READ:  Read his biog

The views in this blog post are Prof Andy Hall’s personal views and do not represent those of Newcastle University.