Segment:1 Real-world evidence in regenerative medicine with Shane Shapiro.
Segment:2 Please tell us about yourself.
SHANE SHAPIRO: My name is Shane Shapiro. I'm a physician and scientist at Mayo Clinic in Florida and Mayo Clinic Center for Regenerative Medicine.
Segment:3 What is real-world evidence?.
SHANE SHAPIRO: Well, real world evidence has been around for a while, and that simply means utilizing data that comes, not just designed from a clinical research trial, but also from the patient's health medical record, the insurance claims. In the modern era, we now have all sorts of information on smartphones and tablets that patients can provide us with through web portals on the internet, and we want to combine all of this into data that can be used to help understand whether or not regenerative therapies truly work for a particular disease condition.
Segment:4 What are the challenges of this approach?.
SHANE SHAPIRO: And so, we know that this is going to be a challenge, because it's not as pure as a typical drug approval pathway randomized controlled trial, but randomized controlled trials have their shortcomings also. We know that it is a challenge to prove efficacy in a novel therapy, because of how tightly we have to control our patient population, our experimental conditions, and other factors. So we want to be able to use both of these when it comes to exploring regenerative therapies.
SHANE SHAPIRO: So that means conducting responsible, rigorous scientific clinical trials, but at the same time, collecting data that we can get elsewhere from the patient, about the patient's medical condition.
Segment:5 How does the FDA view real-world data?.
SHANE SHAPIRO: I really think that we're going to have to use both pieces of evidence. And one of the more exciting talks that I got to hear at the World Stem Cell Summit was representation from the FDA talking about how they are ready to accept real world evidence, and this is actually written into the 21st Century Cures Act, where we talk about-- well, the legislation talks about modern trial design and charges the FDA with accepting real world evidence.
Segment:6 How is real-world evidence being used in the field of regenerative medicine?.
SHANE SHAPIRO: And this is where it really gets important for us to synthesize this data, because there's so many treatments that are being performed at this point, and a lot of uncertainty into which type of cells may work for which medical conditions, and for some of the point of care procedures that are being performed out there, particularly in the orthopedic space, and in the pain management space, this gives us the opportunity to see which of these treatments that are being performed work the best for which patients.
SHANE SHAPIRO: And then, the other thing we're able to do with this digital platform, is take our output in the data sets that we are generating and combine those with other investigators around this new specialty, because we know that there are really limited opportunities at this point to perform some regenerative medicine procedures, and that if we take small numbers and try and glean answers from those small numbers, we're not going to have the power to detect outcomes.
SHANE SHAPIRO: But if we're able to combine with other investigators in this space and pool our data, which we will be able to do with this platform, then there'll be strength in numbers. [MUSIC PLAYING]