The 21st Century Cures Act, passed by Congress in late 2016, actually promotes real-world evidence. The practice offers a way for a medtech company to prove safety and efficacy without necessarily having to conduct a clinical trial. But there are also worries that it exposes a device company to accusations that it is promoting off-label use of a device.
Mike Drues, president of Vascular Sciences, is a Boston area regulatory consultant who has worked with both medical device companies and FDA. He recently spoke with MDO about what people in the industry need to consider when it comes to real-word evidence.
MDO: What exactly is real-world evidence?
Drues: It has different meanings to different people. … But here’s what it means to me. Real-world evidence means data collected and associated with the medical device or drug when used in the “real world” that is not necessarily in a clinical trial. This has been a topic of much debate and much controversy for a long time – especially at the FDA because oftentimes real-world evidence or real-world data is synonymous with off-label use. And as you and your audience know, historically FDA has not been keen on considering data from off label use as part of the submission.
That is starting to change a little bit – very, very slowly. As you know, FDA put out a guidance on this last summer, but I can tell you the pill is difficult to swallow for a lot of my FDA reviewer friends. I’ll give you a very quick example. As you know, I do a lot of [presubmissions] at the FDA. We did a presub just a few months ago which was basically the label expansion. That is, this particular medical device was already on the market. … It’s actually got a few 510(k)s on it, and we were doing a label expansion to add a different indication. We went into the FDA with a whole pile of real-world evidence because, after all, this device has been on the market for several years and has been used with several thousand patients already.
FDA was insisting that we do a clinical trial. Historically, the randomized clinical trial or RCT has been considered the gold standard, and we basically said, “With all due respect, we don’t need to do that because we’ve already got a ton of real-world evidence on it.” Which, I would argue is actually much better than the “gold standard” of the randomized clinical trials because it’s data that was collected in real life use as opposed to some very controlled, very artificial clinical trial. Anyway, long story short, FDA was really not swallowing that pill and was really pushing back hard that they wanted clinical trial data.
I anticipated this. Coincidentally, we [were doing the presubmission] shortly after this particular guidance came out. I anticipated this kind of response. I had a hard copy of the guidance with me, and I literally, I was out in front of them, and I said, “Look, here’s a guidance that just came out … basically saying how we should do exactly what we are proposing on doing.” Bottom line, even though the guidance has come out, one of the things that your audience has to understand for a variety of reasons is [that] it’s a difficult pill, to use that metaphor, for a lot of my FDA reviewer friends to swallow.