World Cancer Leaders’ Summit 2015

Issues surrounding trials for rare cancers

Prof Matt Seymour - National Institute for Health Research, UK

The reason I’m here is that I’m the Clinical Director of the National Cancer Research Institute in the UK and as part of that role I’ve been involved in setting up and running an International Rare Cancers Initiative.

So, Matt, could you tell me about the International Rare Cancers Initiative? First of all why is it needed?

About five years ago we got a lot of people together from around the world because a lot of us simultaneously had realised that rare cancers were falling behind. Over the past twenty years or so there has been an explosion of knowledge and new treatment opportunities for patients with common cancers but over the same time rare cancers have often been left out of the game and they’ve been left out mainly because it’s very difficult in practical terms to organise clinical research projects for cancers which are so rare that in a single country there aren’t enough patients to make a trial feasible.

Could you give me an example of an unmet need where perhaps there is big potential for clinical progress if only it were focussed on?

So, for example, we’ve been concentrating on trials of rarity but where there is potential to improve outcomes. So we’ve looked, for example, at ocular melanoma, so that’s melanoma which affects the back of the eyeball, cancer of the penis, small bowel cancer and cancer of the salivary glands and a number of other cancers around the body where individual countries are, by and large, not able to run the sort of practice-changing randomised trials that are needed to take the field forward.

What typically is done when these cancers are encountered, then, right at the moment?

At the moment those types of cancers are managed partly by anecdote, that is to say, ‘What did we do last time?’, partly by looking to the common cancers which are nearby, so for example in the small bowel people by and large manage those cancers in the same way that they would large bowel cancer even though they don’t have clear evidence that that’s necessarily the right thing to do.

So a lot of them are not getting the right treatment, what are the practical issues needed to implement the sorts of studies that you need to put this on a good footing?

Firstly we need international organisation and that’s why the International Rare Cancers Initiative was set up, to try to bring together researchers from around the world to reach agreement on what the important questions are and to get trials written and set up. But, of course, getting those trials set up is then technically difficult. We need to get collaboration for trials to run between different countries, that means that we have to have agreement of how the trials should be structured and a way of funding the trial and sponsoring the trial which makes it reasonably simple to actually get up and running. Then at the hospital level there are major disincentives to researchers to do research for rare cancers. I’m an oncologist in Leeds, if I set up a trial for large bowel cancer I may put in the hours of work that it takes to get that trial through my regulatory system in the hospital and then I can put in maybe tens or dozens or even hundreds of patients into that trial over a period of a few years. But if I set up a trial for small bowel cancer it will take me just the same amount of work and resources to get it set up and I may find one patient or two patients. So there’s a disincentive for clinicians to get involved.

So what would you say to cancer doctors all over the world about what they can do to help this initiative?

We have to recognise that for every patient, whether they have a rare cancer or a common cancer, they have the same right to evidence based medicine. So it’s vitally important that we do that, that’s the first thing. Secondly we have to recognise that because rare cancers are under-researched then it means that the amount of benefit that comes out of any new trial that we do is that much more; because we’re starting from a situation of very little knowledge, anything that we do will make a big difference. Whereas in a common cancer very often the trials that we do have very small incremental effects.

So potentially there can be a big gain, it’s just that you won’t get too many patients. What can you say to doctors about how to plug into this right now and what they should be doing with each patient?

We have to collaborate nationally and we have to collaborate internationally. Most countries now in the developed world have set up some form of networking for the way that we run clinical research. In the UK we have a National Cancer Research Network which has been up and running, quite well established, for thirteen years now but the same type of model is being developed around the world. So we have to use those networks but we have to link them together so that the work that we do in the UK is linked with the EORTC, is linked with North America and many other countries around the world.

And you’re doing this now?

That’s exactly what the International Rare Cancers Initiative has been set up to try to achieve.