Rare cancers are a good model for personalised medicine because in the end the main issue with precision medicine, personalised medicine, is the numbers. The numbers are low, because when you go to a subset of patients the numbers decrease, and so you face more difficulties doing studies, you face more uncertainty in clinical decision-making and so on. Rare cancers are rare so they pose a big problem of numbers by definition. I think that if we are able to work out innovative solutions for rare cancers we may also provide some help to shaping better personalised medicine and precision medicine. If we are able to provide innovative solutions for rare cancers probably we are also working for personalised medicine, precision medicine, because in the end the solutions could be the same.
Are there some potential solutions?
If you look at precision medicine, personalised medicine, you have at least two big problems. One has to do with your clinical method, and the other has to do with the regulatory environment. The problem is in both cases, which degrees of freedom do you have in order to personalise your clinical decisions? Because, from the clinical method perspective, if you see evidence-based medicine as something which is based on the results of clinical trials and clinical trials say to you yes or no on a new therapy, and that’s it, basically you’re not free to personalise anything. Then, if you achieve a personalised clinical decision on a new drug, for example, and that new drug is not available, you’re not free to give it. So I think that these two areas should be dealt with if we want to pursue actually a personalised approach to our clinical decisions.
This is all the more true with rare cancers, because they are rare as tumours. If you go to subgroups with a rare cancer by definition you will have small, small numbers. I think these are the problems. As Rare Cancers Europe, we tried to put together some recommendations in a consensus paper on innovative solutions for clinical research on rare cancers. We put inside also something of the clinical method because it’s a continuum. You should improve research, you should improve your clinical method, and finally, hopefully, you will be able to personalise your decisions a little more. Of course, that paper also included some solutions which could be useful for the regulatory landscape in which medicine is practised. Of course here I am talking of the European Union, because clearly the regulatory environments, globally speaking, are very discrepant. In the European Union the main problem today is this gap between the centralised approval of new drugs and then the reimbursement for these drugs, which takes place nationally or regionally even.
How to fix this issue? How to bridge this gap?
I think that a harmonised assessment of efficacy should be a solution, even if then you may have a different willingness to pay at the national or regional level, of course. But at least the efficacy assessment should be harmonised, which doesn’t happen today. Of course, again in rare cancers in particular, the assessment of efficacy is problematic because you have more uncertainty. We always say in the rare cancer community that in order not to discriminate against rare cancer patients you need to accept a higher degree of uncertainty. In a sense this is the problem. If you want to do precision medicine and so on, you must accept a higher degree of uncertainty. This is the problem.
