Spotlight on Research:
Dr. Michael Lauer Co-Publishes Perspective Piece on Randomized Registry Trials


Robin Ferrier, NHLBI Office of Communications: Welcome to the Spotlight on Research from the National Heart, Lung, and Blood Institute’s Office of Communications. I’m Robin Ferrier and my guest today is Dr. Michael Lauer, director of the Division of Cardiovascular Sciences.

Dr. Lauer:  Good morning Robin.

Ferrier:  To start, why don’t you provide us with an overview of the perspective piece you published in the New England Journal of Medicine.

Dr. Lauer:  Yes, Ralph D'Agostino and I published a piece about something called “The Randomized Registry Trial.” Briefly, what this is about is a trial, which was published over the weekend in which a group of investigators used a registry, that is an observational group of patients, to build a clinical trial. 

And let me briefly explain what this means. Normally when people do clinical trials, they find a group of patients with a particular problem, and then they randomize them to one arm or the other, and they see what happens. The problem with this approach, although it’s been used for a long time, is it is very expensive. It takes a lot of time and money to find all these people. 

Now we have another world called registries. In registries, people are routinely collecting information about patients who are coming in with common problems, and what these investigators did was, is they said well let us take advantage of this registry. These patients are already here. We know about them. We don’t have to go looking for them. And furthermore, the registry is collecting a lot of information about them, so we don’t have to collect any information about them. We can just use the data from the registry. So this way they were able to find 7,000 patients and do this in almost no time, do it very quickly, and they were able to answer an important clinical question at a remarkably low cost.

Ferrier:  And this happened – where was this trial again?

Dr. Lauer:  This trial was done in Scandinavia. It was done primarily in Sweden. Sweden, for a long time, has had a registry in which essentially every patient with a heart attack and every patient going to the heart catheterization lab for treatment of a heart attack gets included. It’s a very high quality registry. It’s been around for at least 10 to 15 years. It’sbeen used for very high quality observational research. This is the first time that it’s been used for a large-scale trial. During this trial, they took about 10,000 patients who were having a heart attack, and randomized just over 7,000 of them to a test of something called Thrombus Aspiration. Thrombus is a clot and so what they did here was they randomized them to have their clot sucked out before they put in a stent versus not getting sucked out, which is the standard approach. The trial found that this aspiration of the clot did not work, that it didn’t lead to an improvement of outcome. 

It was a well-done trial. The results are robust. But what’s interesting is how the trial was actually done. This trial cost $300,000, which for people who don’t know, in trial speak, that’s almost next to nothing. That’s a very low cost. Typically, a trial like this could cost in the tens of millions or even hundreds of millions of dollars.

Ferrier:  So there are real advantages both in terms of the lower cost of this new paradigm that we call the Registry Based Randomized Trial, and in terms of clinical trial recruitment, which has been a challenge. So is this a model that would work in the United States?

Dr. Lauer:  Well, I think so. Now, there are challenges to doing it in the United States. Our information, our health information systems in the United States, are not as well integrated and as robust as they are in European countries. People get their health care from multiple different sources so they are more difficult to follow over time. None the less, it is being done. There is a trial that’s being run out of Duke University called the SAFE-PCI Trial. This is a trial in which women who are getting stents are being randomized to have the stent put in by the femoral approach, which is the standard approach, or via the wrist. The thinking is that it might be safer to do it through the wrist, because there’s a lower risk of bleeding. And this trial is being built on a registry, which has been established by the American College of Cardiology. It’s called the National Cardiovascular Data Registry. 

It’s a similar story. They’re able to do this trial at very low cost. I’ll admit it is more difficult to do a trial like this here in the United States than it is in Europe, but I don’t think it’s impossible. We have a number of very high quality registries here in the United States. We do have increasing integration of health care with health care reform. So we can take advantage of these to stimulate this model along. 

Ferrier:  Are there some examples of research that wouldn’t be able to use this model?

Dr. Lauer:  Well where you wouldn’t be able to do it is if you’re looking at patients who are not  going to be included in our registry. So, for example, if you were to do a very complicated trial of metabolic interventions, say in a feeding kitchen. That kind of trial, which is more of a physiology trial, you couldn’t do that kind of a trial based on a registry. In a trial like that you’re going to be collecting lots of data, which your registry would not collect. It may be more difficult to do this with rare diseases. Tt depends upon the disease. Some rare diseases have set up registries so you could do that. But this methodology lends itself particularly well for common diseases.

Ferrier:  So we have the one study that’s happened now in Scandinavia and the one ongoing study here in the U.S. Do you think this new model is going to be embraced by the U.S. Research community?

Dr. Lauer:  Well, I think one of the roles we have in NIH is to help stimulate that. And one way we can help stimulate that is by creating a funding environment in which we reward this kind of behavior. We have already funded research, which has leveraged the registries. And we are also pushing methods for conducting trials at low cost. An example of that is our common fund collaboratory, in which we are funding trials that leverage digital platforms and do these trials at very low cost. 

So we’re engaged in conversations amongst ourselves as well as with thought leaders in the American research community, and think about ways in which we can stimulate this kind of trial to happen.

Ferrier:  Great. Is there anything else you would like to say about this new paradigm of the Registry-Based Randomized Trial?

Dr. Lauer:  I think this is a case where we could potentially look at our budget constraints as an opportunity. There’s an old saying that when people lack resources, they become resourceful. We are seeing ourselves in the research community as increasingly lacking in resources. And particularly lacking in resources for funding expensive studies. This is an opportunity for all of us to become resourceful, and this particular trial is a beautiful example of how people have become resourceful and may be something for us to emulate.

Ferrier:  Wonderful. Thank you so much for your time. 

Dr. Lauer:  Great, thanks Robin.