Sramana Mitra: What you just said is very interesting from a computer science point of view. So far, it seems like pharma has been developing drugs with clinical trials that have relatively small numbers of people whose data is going into trial input. The big data opportunity is to do this at a much higher scale.
You can collect data from millions of patients in the process of figuring out what works, whys, on what segments, and what kinds of biometric characterization. All of that is very viable. So far, it hasn’t been that viable. So far, you take small samples and you extrapolate. But it’s all becoming very viable and potentially, even cost effective to do this at scale. Could you comment on this trend?
Tarek Sherif: First of all, I do think it makes a lot of sense. You do want to use real-world data and real-world evidence to help in the decision-making process. I don’t think we’re going to lose control groups. I don’t think we’re going to lose clinical trials anytime soon. If you look at the regulatory environment, there’s a lot of thought and a lot of discussion being put to informing clinical trials with the use of real-world evidence.
But you’re not going to supplant them with it because I don’t think anybody is in favor of coming out with a drug, putting it out in the population, trying it out on millions of people, and seeing what happens.
Sramana Mitra: That’s not what I meant. I meant that the clinical trials today are done on much fewer numbers of people. Then you extrapolate from there. That has limitations, right? One of the problems that we have in the drug industry is that the level of side effects is huge. If you can run trials on a much larger dataset, you stand to be able to take all of that in stride and have much more accurate and much more precise understanding of what that drug does and in what circumstances. Is that not correct?
Tarek Sherif: No, I actually disagree in a sense. Historically, the paradigm has been that you run clinical trials in three phases (actually four phases but let’s call it three phases) before you bring them to market or before you bring them to a regulator who opines on whether it’s safe and efficacious. In phase one, you used to give the drug to a very small sub-population of patients who are very sick. You would see if it has any impact and what the toxicity was.
In your phase two, you would start to give it to more people and you would start to figure out what’s the right dose and what’s the impact of this drug on patients. Then in phase three, you start to look a little bit more at broader populations, maybe thousands of patients, depending on what kind of drug you’re developing, maybe hundreds if it’s for a rare disease. In there, you were looking for adverse events or safety issues.
We’re seeing this already happen. Regulators are saying, “Based on a limited number of trials and a limited number of patients that you’ve done the drug development with, we will give you an approval. But the approval of your drug is contingent on you continuing to follow what happens in the broader world when you give it to many more patients.”
I’m not disagreeing necessarily with what you said. What I’m saying is you won’t take a drug from research and then look at what the impact is on a lot of patients on day one. You might bring a drug to market faster and then have to track what the safety profile of that drug looks like.