Ranking the clinical benefit of anti-cancer drugs is certainly a very difficult challenge. I have been fascinated by this topic in the last five years and I personally have published three or four papers on this that have considered the basis for some initial work that the Task Force on Clinical Benefit Evaluation of the ESMO has worked on. Essentially, in the last three years with ESMO we have put out one of these scales of magnitude of clinical benefit, “we” being five or six medical oncologists and a statistician from ESMO, have outlined a rather complicated system of ranking the clinical benefit of new drugs in oncology. There are a lot of merits to this publication and this scale. Essentially, it represents the first step ahead in this prohibitive task of categorising and ranking the clinical benefit of the new agents.
At the same time, of course, being the first step, it certainly needs some corrections and some new developments. During my talk I highlighted some of these corrections that could be done to the scale and the first point I made is in line with the title of the meeting that is patient empowerment. One of the problems that, looking back, I recognise in the work we have done is the fact that the committee did not include a patient advocate, a patient representative, and the committee also did not include any expert in public health. In a way that is a problem, because what we have worked out is a system that suits very much our perspectives, the perspective of the clinical researchers, of the doctors.
Stakeholders in this business can be identified at least along three different avenues: one, the patient; two, the doctor; three, the payer; four, the regulatory people; and five, the drug companies. Drug companies set the price, they are out. Regulatory people approve drugs, and in a way they are part of the business, but they say yes or no. So we remain with patients, doctors and payers. Payers have a general public health perspective. Patients and doctors have an individualised perspective. The problem of the ESMO scale is that it has a public health perspective. It declares it wants to rank the drugs in terms of clinical benefit in such a way that all the most powerful, the most valuable drugs, should be available in those countries where they are not now. So this is a clear public health perspective.
At the same time, it was elaborated by people like me that are clinical researchers without expertise in public health. That’s one of the problems.
The patient, once part of the committee, they could certainly have contributed in emphasising or de-emphasising certain aspects of clinical benefit. That’s one. Number two, certainly patients are not so interested in a single trial and that’s a limitation, a mistake, I would say, that we have done in putting out that scale. Essentially, we rate only trials whereas patients are interested in the benefit and value of a drug, not of that drug within one trial. That’s a problem. This lack of overall view of the problem, certainly that is something that patients would have raised.
There are some more technical problems within that scale. There is a paper that, along with my statistician, we have written and is in press on the European Journal of Cancer, will come out in a couple of months from now, is already in press, and it highlights some of the problems of the scale that continues to hammer issues that are very technical and that do not take into consideration the overall picture. That, to me, is a direction that should be corrected, essentially, at this point. So the contribution of empowering patients even within committees with such a challenging task is certainly a valuable step to be taking.
How does this influence your rating of drugs?
It’s the opposite. The rating should homogenise the availability of drugs. It depends on the perspective. If you consider Europe as Western culture, you should expect that all European countries benefit from availability of all the clinically relevant drugs. There are some countries that do not benefit from some of these agents. This ranking system should allow, in the end, to recognise those that you cannot do without, those drugs that you cannot give up, and that the government should pay for them.
