War on Cancer
How should decision makers assess the value of a medicine?
Dr Hans-Georg Eichler - Senior Medical Officer, European Medicines Agency
What was your presentation at the Economist War on Cancer meeting about?
It was about this idea of medicines adaptive pathways to patients. The background to this is that in our modern healthcare environment we are facing a number of competing objectives that are all legitimate but they are very different to reconcile with each other. For example, we know that some patients, today’s patients, have an urgency to get access to promising new treatments; at the same time we want to ensure that the drugs we allow on the market as regulators are well tried and tested and that creates tension. We also know that the future, certainly in oncology, is not a one-size fits all, single drug treatment, but will likely consist of personalised treatments and, even more difficult, personalised treatment combinations or personalised treatment sequences. That is an opportunity but is also a methodological challenge – how will you demonstrate efficacy in this myriad of clinical situations and scenarios? So we will have to broaden our armamentarium of research methodologies.
Then we know that there is, of course, the financial issue. On the one hand we want to support the innovation engine, we want to keep incentives for innovation out there, but we know that our healthcare systems are under financial stress, how do you reconcile that? So that is the foundation why this medicines adaptive pathways to patients idea was born and is now pursued by a number of stakeholders.
How does adaptive licensing work?
We no longer call it adaptive licensing because we recognise that it is more than licensing; licensing does not get you to the patient or, if you are a company, does not get you to market. It is all the decision-makers along the row that need to be aligned if we want to achieve success. Now, how will it work out in practice? The idea is that you define usually a narrow treatment eligible population that you will focus your development on, you can allow the drug on the market, hopefully early, to satisfy the need of those patients who are in most urgent need. But then you continue the learning process, the evidence generation has to continue over time so it’s not over once the drug is out on the market. That means that also payers will have to come to grips with that idea that in the beginning we may know a little less about this product so we have to narrow the treatment eligible population also on the market. But as we know more we could widen the treatment eligible population and that is important, we will widen from a regulator’s point of view the indication and from a payer’s point of view we may set a new price. That is why all of us need flexibility. So it is about repeat cycles of evidence generation and re-assessing and re-evaluating the value or otherwise of a new product.
How does this benefit the patient?
The idea here is to strike the balance between those patients who are in need today, so those who are sick patients today would benefit by having access to something hopefully that will help them that they wouldn’t otherwise under a traditional system where you take years and years and years of learning before you allow it on the market. If, on the other hand, you are a patient who is not yet sick but who will likely be a patient in ten, fifteen, twenty years, which is something that will happen to all of us, then you will want a thoroughly tested and vetted drug. And we are trying to square that circle by having this lifespan approach to learning and evidence generation and modifying the licence and the usage.
How long will it take to implement these things?
This is very difficult. We haven’t, at the EMEA, given ourselves a timeline because we know that this is a learning curve that is a journey that we will all go together. The pressure to keep up the momentum comes from patients because patients keep telling us, ‘You’re not good enough. Why do I have to wait?’ We’re in constant interaction with patients who tell us to keep up the pressure. I could not predict the future; I hope we will get there, I’m confident that this is the right approach to our problems but I would be very hesitant to give you a timeline.
Is there anything else you’d like to mention?
Only to say that this requires the contribution of all the decision makers, and when I say decision makers I mean first the proxy decision makers, they are regulators, health technology assessment bodies and payers, but then also the individual level decision makers and that is first and foremost patients and their clinicians. They all have to be on board and of course the industry who is developing these new products. Second, I think it requires all of us to understand also the concept of uncertainty. Some of the critics of this idea point out that this is a lowering of the evidence standards. I totally disagree with that notion; it is not a lowering of the evidence standards, it is an acceptance of initial higher uncertainty in order to provide benefit to some patients but that higher uncertainty will progressively be reduced over time.
