ISCTR: Translating Cardiovascular Research to All Concerned

Ileana L. Piña, MD, MPH; Anthony N. DeMaria, MD


December 11, 2017

Ileana L. Piña, MD, MPH: Hello. I'm Ileana Piña from the Albert Einstein College of Medicine in New York and the Montefiore Einstein Medical Center, and this is my blog.

I want to welcome everyone to Transcatheter Cardiovascular Therapeutics (TCT) 2017 at the Denver Convention Center, where it has turned very cold today. I am thrilled to have one of my guests today, Anthony DeMaria, who is the past editor of the Journal of the American College of Cardiology (JACC).

Tony is at the University of California in San Diego, where he has been for how many years now?

Anthony N. DeMaria, MD: Twenty-five years.

Dr Piña: Twenty-five years, and a luminary in cardiology and someone that we look up to and respect. Welcome, Tony.

Dr DeMaria: Thank you.

Dr Piña: Tell us about ISCTR yesterday, and what is that organization?

Dr DeMaria: That's the International Society of Cardiovascular Translational Research, with the emphasis on translational.

Dr Piña: What does that mean?

Dr DeMaria: That is a good question. The definition of translational research is very variable. I think in its simplest form, it's taking discoveries and getting them to patients. There are lots of discoveries that occur that languish in laboratories or get caught up in the process of being translated into a product.

The ISCTR is dedicated to facilitating that process. We have engineers, we have basic scientists, we have funders, we have major industry, and of course we have clinical investigators and regulators—of note, the US Food and Drug Administration (FDA). The idea is to bring all of these groups that are so fundamentally important to translating discoveries into products, to bring them together to see how we can make that happen faster.

Dr Piña: I notice the FDA was there, Centers for Medicare & Medicaid Services was there, as were some other payers and people that know about the payment system—which, of course, is getting so complex. If we don't understand it, how can we expect patients to?

We had patients there yesterday, which I found incredibly interesting, and there's a new focus in the clinical trial world on patient-reported outcomes. I know the Patient-Centered Outcomes Research Institute (PCORI) has been very dedicated to patient-reported outcomes. We spent a lot of time listening to the patients. What struck you about those patients?

Dr DeMaria: What was very interesting is that, as trialists and as regulators who approve products and payers who reimburse products and what not, we've had this focus on hard incomes, such as mortality or serious morbidity, infarction, or stroke. The patients said, "The most important thing for me was that I felt better and that I was independent, that I could go home and live on my own. That's what's really important to me, or it's important to me that I don't have to go to the hospital every month and get injections or whatever."

Mortality wasn't on the scoreboard. It put things in a very different perspective, especially in your field, in congestive heart failure, where traditionally we've asked, "Does this drug change mortality?" What is much more important is, "Does this drug or device change the quality of life for the patient?" That's what they care about.

Dr Piña: We are limited in our ability to measure some of these things. We've had a couple of health outcomes instruments; the Minnesota Living With Heart Failure Questionnaire, which was crafted in the late '80s when Jay Cohn did V-HeFT I and II,[1,2] asked questions about work. Most of my patients who are that ill haven't worked in a while. They can't make that assessment. Sometimes when they get a left ventricular assist device (LVAD), or they get transplanted, they come back with an infection, or they have a bleed, and it's very disruptive to their lives.

One of the things that I took away is that we need better instruments to capture and quantify quality of life. It's so hard to quantify because what may be important may be totally different from one person to another. We heard from three different people who had very different syndromes. What do you think about where the science is going with this?

Dr DeMaria:I had two thoughts about that. The first one was about our guidelines because we're progressively guideline-driven. When I make rounds with the house staff, they focus on the class Ia guideline recommendations, which are very few.

Dr Piña: We don't have that many things that are Ia's.

Dr DeMaria: The Class IIa and IIb recommendations are where clinical judgement comes in—things that might be reasonable to do under certain circumstances. That's where you and I as clinicians say that for this patient, the class IIa or even class IIb indication is preferable. I'm going to use that approach, not the other one.

A word of caution is that patients are very susceptible to a placebo effect. One of my favorite stories is about a patient who insisted on going for catheter ablation for atrial fibrillation (AFib), even though he was doing perfectly well. He came back from ablation saying, "I'm glad I had it—I should have had it a long time ago," only he was still in AFib.

Dr Piña: He was still in AFib?

Dr DeMaria: Patient input is subjective. As an endpoint for a clinical trial, it has to be viewed with some caution, but I think it's been given much more importance than in the past.

Dr Piña: I use a couple of these quality-of-life questionnaires in my own clinic, and we make sure that whoever gives it to the patient to fill out isn't part of the clinical team. There's always that bias that patients want to please their providers. They want you to be happy because you're doing a good job with them; and they may feel terrible, but they want you to feel well [so they say they feel fine].

We give it to them to fill out before they have had any contact with the clinical team. At the FDA, we have tried to incorporate some of those things when anyone comes to us with a composite endpoint.

For example, in the area of LVADs, we don't have a great instrument that will capture what goes on with these patients. They are usually so sick by the time they come to us that they haven't been walking and doing things. What you want to capture is: How do they feel about being in the hospital? I can't imagine anybody loves being in the hospital. How do you quantify that? I think we have a big challenge on our hands.

Dr DeMaria:A major challenge. That will be an important direction. Maybe the professional societies can help foster this. The ISCTR symposium shed some important light on this. The FDA, the payers, the trialists, and certainly I came away thinking more about this.

Dr Piña: I did too, and I was struck by the patient who wanted his LVAD and didn't want transplant. Yet, we clinicians think of transplant as better because all of the hardware is out. Yes, you have to take these medicines, but we have a history of a longevity with transplant. He didn't want that. He liked his LVAD.

Dr DeMaria: It is really interesting. You think the last thing in the world you'd want is to be tethered to a device that, if it malfunctioned, would be the end of you, but he has gone 3 years with it.

Dr Piña: Listening to the patients—what an unusual thing to do, right?

I want to thank my audience for joining us today from TCT. This is Ileana Piña, signing off. Have a great day.


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