So we are in times where we cannot rely on the big trial data for evidence generation anymore. Because we have a lot of subgroups, patient subgroups, diagnostic subgroups. So the groups we are treating are getting much smaller and smaller. And that’s why we have to think about novel ways of getting trials up and running. So the regular way to create evidence of course, is doing a randomized phase 3 trials with a few hundred patients, sometimes a thousand patients, as it is in coagulation trials for instance...
So we are in times where we cannot rely on the big trial data for evidence generation anymore. Because we have a lot of subgroups, patient subgroups, diagnostic subgroups. So the groups we are treating are getting much smaller and smaller. And that’s why we have to think about novel ways of getting trials up and running. So the regular way to create evidence of course, is doing a randomized phase 3 trials with a few hundred patients, sometimes a thousand patients, as it is in coagulation trials for instance. And that’s not going to work in the future anymore. At least for our patients who had a relapse, or patients with high risk disease and rare diseases. So what’s been done in recent years is then the basket trials, where, let’s say you have one drug and you test this drug in several indications who all have for instance mutations in a certain pathway where this drug is active.
And now in personalized medicine we are fighting with the opposite problem. We have one patient, but we have diagnostic tools to let’s say open up 50 different treatment possibilities for this patient, personalized treatment. How are you going to put this in a clinical trial? And that’s a challenge for the agencies like EMA. That’s a challenge for the ethics committee. And that’s also a challenge of course for us who are planning clinical trials. And that’s quite an interesting question from regulatory and clinical point of view. And also from the point of view of the patients who are also involved in this type of action, how can we plan these trials.
