Steven Coutre, MD: There’s always the dynamic between clinical trials and what actually happens in practice. And, of course, in many cases, drugs may be approved from a certain clinical trial, but it’s not really addressing the issue of how that drug really compares to, say, a different standard therapy. It’s more of a trial that’s designed from a regulatory perspective to get a drug approved rather than from the perspective of a physician who would look at that and say, “I understand those results. That really applies to when I’m making decisions in practice.” And the ideal situation, of course, is to have the two together: a well-designed trial that may allow a novel therapy to be approved, but also is a believable trial to the practicing physician.
And, so, yes, it is important to gather data from “real-world” experience. I think individual physicians do that every day in practice. That’s what they have to do. They’re really dependent on their own anecdotal experience in many cases. But, it’s also very important to continue to collect data in a more controlled setting to really address issues about things like late toxicities or development of resistance, things that may not show up in an individual physician’s practice. When one looks at a much larger group as a whole, then you get that information.
So, we do gather some of that information from the real-world, if you will, in terms of safety. The companies have to keep integrative safety databases. Not only safety reported from the trials, but safety reported from postmarketing trials, from expanded access programs, and even just patients receiving drugs who have adverse events. I think it’s really important to have the combination of the two.
Value-based care is obviously a very topical area right now. It speaks for itself. It’s hard to argue against the concept. The challenges are often demonstrating what value is. How do you measure value? Is it measured in terms of patient satisfaction? Is it measured in terms of cost? Is it measured in terms of outcomes? It’s a complex issue. I think we do have to focus on patient satisfaction, patient quality of life issues. Quality of life issues are very important and are, fortunately, usually components of any trials that we do now. So, just as it’s important to report out efficacy and safety issues, it’s important to report out quality-of-life issues when we’re looking at therapies. I think demonstrating the value of these newer therapies, because they’re inevitably expensive, is very important. We have to show that not only are they better therapies, perhaps than our standard treatment, but that they add value in terms of satisfaction, in terms of quality, and hopefully in the big picture of our healthcare costs in terms of financial benefit, as well. We often overlook some of the consequences of other therapies—hospitalizations, infections, the need to visit physicians’ offices frequently. Those things don’t get captured in the total equation sometimes.