Real-World Data and Outcomes For Patients with NVAF

OCTOBER 03, 2018


Gary M. Besinque, PharmD, FCSHP; Jaime E. Murillo, MD; Juvairiya Pulicharam, MD; and Peter Salgo, MD, discuss how real-world data surrounding the management of nonvalvular atrial fibrillation with novel oral anticoagulant therapy can be relayed back to various stakeholders, including drug manufacturers, to help to address gaps and improve outcomes for patients.

Peter Salgo, MD: Let’s talk about how you collect data. It’s not just a moment to talk to the patient about it, it’s a moment to sit down and say, “OK, let’s have a look at you. What are your issues? What drugs are you on?” Then we can establish a registry to be able to do this.

Juvairiya Pulicharam, MD: Yes. We are doing that. In heart disease, it’s important to learn what is happening in the real world. With clinical trials, it’s a closed system. It’s not real-world data. We want to know what is really happening. You can really look at gaps. I think it’s very important that this information is shared with specialty physicians, primary care physicians, and maybe even with patients—some of this data and the outcomes—and even manufacturers, because they are a partner to this. 

Peter Salgo, MD: What you’re going to hear, I suspect, when you try to share this data is, “Well, I saw the control study. That wasn’t all-comers. You didn’t control the entry into this study.” You pointed out that studies are not real-world. But, the real world is not a study.

Juvairiya Pulicharam, MD: I just need to correct myself. Clinical trials are not real-world scenarios. There are still patients, but it’s a selection of patients, right?
Peter Salgo, MD:That’s right.

Juvairiya Pulicharam, MD: There’s criteria. You’re really trying to look at the safety of your product, and then efficacy, or noninferiority. Once it’s approved and you are prescribing it without any criteria, you’re not doing a lot of follow up and changing. If they don’t take a dose, maybe they’re out of the study. You want to look at what’s happening in the real world. Your registry studies are still under the umbrella of a study because you want to enroll them into the registry. There’s informed consent. They’re aware of that in the registry. But, it’s more of a real-world case because we want to see what’s happening and we follow these patients for 2 or 3 years. They’re still in the registry. You can always look at retrospective data if a question arises. Or, you can look at prospective data and a lot of questions can be answered in registries. Maybe it’s not a registry of very few patients. You get new patients. It’s de-identified. They’re all have a unique identification.

Gary M. Besinque, PharmD, FCSHP: Well, the good news is that the real-world evidence for the novel oral anticoagulants (NOACs) and the initial pivotal registration trials are coming together and they agree with one another. The very narrowly scoped pivotal trials were actually validated by what’s coming out of the pharmacy claims database information that we have.

Peter Salgo, MD: Is it valid though? It’s nice to see that this synchronizes, if you will. But, again, isn’t there some self-selection here? Those that don’t seem to do very well, or don’t like these drugs, sort of drop off? And, it’s not as well-controlled as a double-blind placebo-controlled study.

Juvairiya Pulicharam, MD: In real-world registries, there are different ways of keeping a registry, right? You can even look at another control arm. They’re not a part of the registry, but you can look at what’s happening. Or, in the registry, you can have an intervention arm. You explain things to them. You educated them. And then, you can have another arm in which you don’t give much education and you see what’s happening. So, there are different ways that you can design these registries. 

Jaime E. Murillo, MD: The good news about the future, without going into too much detail, is that now that we have intelligence, the way we study medications in the real world is going to change. Now we have the ability to obtain data in a way that is faster and accurate, truly reflecting what’s happening on a daily basis. You can put big data together. You can merge systems. Then, you come up with a lot of protocols based on that data.

Juvairiya Pulicharam, MD: Yes, I agree with that. It’s also important because there are so many different NOACs out there, right? They have different, fancy names. For a primary care physician or a patient, it’s important to know what the differences are. “Why am I not on that drug? Why am I on this drug?” Having the real-world evidence, we can follow patients and say, “These patients were on this drug, but it’s not drug-specific. What’s happening with these patients?” And also, maybe some of the manufacturers, I’m hoping, or some health systems can help create some care pathways. Now you can identify these patients and say, “This is what is good for this patient.” The manufacturers, or the health systems, or the payers’ involvement is very key if you really want to….

Peter Salgo, MD: It’s key, but are the drug manufacturers really interested? They’ve got FDA [Food and Drug Administration] approval. They’re out there. There are drugs in the real world. Do they want to hear back from you? 

Juvairiya Pulicharam, MD: That’s a very good question. Maybe in the past I wasn’t paying attention to that so much, but what I’m seeing recently, because of the whole value-based medicine and accountable care organizations, is that manufacturers are now becoming more interested. A few are showing more interest and are saying, “How can we be part of it? How can we support that? How can we be one thing, at the end of the day?”

Peter Salgo, MD: Doesn’t that cost the manufacturer money, especially if you’re giving him stuff that he doesn’t want to hear?

Gary M. Besinque, PharmD, FCSHP: Not too much money. 

Peter Salgo, MD: What are you trying to say?

Juvairiya Pulicharam, MD: Otherwise they won’t make any money, right?

Gary M. Besinque, PharmD, FCSHP: This helps them seek a larger market. The information that we develop together will definitely pay off for them. It will be more than they’re spending to get it.

Peter Salgo, MD: OK. If I understand correctly, they’re not spending much to get it at all. They’re not really funding that, are they?

Juvairiya Pulicharam, MD: At least when we work with organizations, it’s really an internal question. We want to know how we can better educate our physicians so we have the knowledge. How can we really look into our data? So, it’s really for work that we are doing together, and I think the manufacturers are interested.

Peter Salgo, MD: OK.

Juvairiya Pulicharam, MD: Physicians and patients.

Peter Salgo, MD: But you’ve developed this internally, right?

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: And you’re going to follow your patients and keep some sort of database.

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: And you’re going to change and modify your practices based on what you see.

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: Then, one day, somebody says, “Oh, the light bulb went on. Let’s take all of this, fold it into 3 pieces, put it in an envelope, and put a stamp on it and say, “Send it to the manufacturer.” Do they want to get it?

Juvairiya Pulicharam, MD: The way you packaged it, it looks very good. For the manufacturer, what I’m seeing is they’re very interested in knowing the burden of the illness. They are very interested in knowing where the gaps are. 

Peter Salgo, MD: So they are interested. 

Juvairiya Pulicharam, MD: I am seeing that more and more manufacturers, and health systems, and providers are coming together for the first time. Everybody knows that we can’t do it alone.

Peter Salgo, MD: I’ve been in the medical business for a long time. I’ve talked to a lot of manufacturers. I’ve talked to a lot of doctors. This demonization of one group versus another group doesn’t do anybody any good. Aren’t we all on the same page? I don’t want to just join hands and say kumbaya here. The drug manufacturers don’t want to hurt anybody. They would like for their drugs to work, and they’d like to know how best to do that. So, they should be interested. Is that your experience?

Jaime E. Murillo, MD: Yes, the data will be out there. You have to be a part of the system if you want to be moving along with the system. Otherwise, you’re going to be isolated. The fact that you don’t participate in the process doesn’t mean the data won’t be there. It will be there.

Peter Salgo, MD: It’s coming whether you’re ready for it or not.

Jaime E. Murillo, MD: Yes.

Peter Salgo, MD: So, you might as well get on the train.

Jaime E. Murillo, MD: Correct.

 
 


Gary M. Besinque, PharmD, FCSHP; Jaime E. Murillo, MD; Juvairiya Pulicharam, MD; and Peter Salgo, MD, discuss how real-world data surrounding the management of nonvalvular atrial fibrillation with novel oral anticoagulant therapy can be relayed back to various stakeholders, including drug manufacturers, to help to address gaps and improve outcomes for patients.

Peter Salgo, MD: Let’s talk about how you collect data. It’s not just a moment to talk to the patient about it, it’s a moment to sit down and say, “OK, let’s have a look at you. What are your issues? What drugs are you on?” Then we can establish a registry to be able to do this.

Juvairiya Pulicharam, MD: Yes. We are doing that. In heart disease, it’s important to learn what is happening in the real world. With clinical trials, it’s a closed system. It’s not real-world data. We want to know what is really happening. You can really look at gaps. I think it’s very important that this information is shared with specialty physicians, primary care physicians, and maybe even with patients—some of this data and the outcomes—and even manufacturers, because they are a partner to this. 

Peter Salgo, MD: What you’re going to hear, I suspect, when you try to share this data is, “Well, I saw the control study. That wasn’t all-comers. You didn’t control the entry into this study.” You pointed out that studies are not real-world. But, the real world is not a study.

Juvairiya Pulicharam, MD: I just need to correct myself. Clinical trials are not real-world scenarios. There are still patients, but it’s a selection of patients, right?
Peter Salgo, MD:That’s right.

Juvairiya Pulicharam, MD: There’s criteria. You’re really trying to look at the safety of your product, and then efficacy, or noninferiority. Once it’s approved and you are prescribing it without any criteria, you’re not doing a lot of follow up and changing. If they don’t take a dose, maybe they’re out of the study. You want to look at what’s happening in the real world. Your registry studies are still under the umbrella of a study because you want to enroll them into the registry. There’s informed consent. They’re aware of that in the registry. But, it’s more of a real-world case because we want to see what’s happening and we follow these patients for 2 or 3 years. They’re still in the registry. You can always look at retrospective data if a question arises. Or, you can look at prospective data and a lot of questions can be answered in registries. Maybe it’s not a registry of very few patients. You get new patients. It’s de-identified. They’re all have a unique identification.

Gary M. Besinque, PharmD, FCSHP: Well, the good news is that the real-world evidence for the novel oral anticoagulants (NOACs) and the initial pivotal registration trials are coming together and they agree with one another. The very narrowly scoped pivotal trials were actually validated by what’s coming out of the pharmacy claims database information that we have.

Peter Salgo, MD: Is it valid though? It’s nice to see that this synchronizes, if you will. But, again, isn’t there some self-selection here? Those that don’t seem to do very well, or don’t like these drugs, sort of drop off? And, it’s not as well-controlled as a double-blind placebo-controlled study.

Juvairiya Pulicharam, MD: In real-world registries, there are different ways of keeping a registry, right? You can even look at another control arm. They’re not a part of the registry, but you can look at what’s happening. Or, in the registry, you can have an intervention arm. You explain things to them. You educated them. And then, you can have another arm in which you don’t give much education and you see what’s happening. So, there are different ways that you can design these registries. 

Jaime E. Murillo, MD: The good news about the future, without going into too much detail, is that now that we have intelligence, the way we study medications in the real world is going to change. Now we have the ability to obtain data in a way that is faster and accurate, truly reflecting what’s happening on a daily basis. You can put big data together. You can merge systems. Then, you come up with a lot of protocols based on that data.

Juvairiya Pulicharam, MD: Yes, I agree with that. It’s also important because there are so many different NOACs out there, right? They have different, fancy names. For a primary care physician or a patient, it’s important to know what the differences are. “Why am I not on that drug? Why am I on this drug?” Having the real-world evidence, we can follow patients and say, “These patients were on this drug, but it’s not drug-specific. What’s happening with these patients?” And also, maybe some of the manufacturers, I’m hoping, or some health systems can help create some care pathways. Now you can identify these patients and say, “This is what is good for this patient.” The manufacturers, or the health systems, or the payers’ involvement is very key if you really want to….

Peter Salgo, MD: It’s key, but are the drug manufacturers really interested? They’ve got FDA [Food and Drug Administration] approval. They’re out there. There are drugs in the real world. Do they want to hear back from you? 

Juvairiya Pulicharam, MD: That’s a very good question. Maybe in the past I wasn’t paying attention to that so much, but what I’m seeing recently, because of the whole value-based medicine and accountable care organizations, is that manufacturers are now becoming more interested. A few are showing more interest and are saying, “How can we be part of it? How can we support that? How can we be one thing, at the end of the day?”

Peter Salgo, MD: Doesn’t that cost the manufacturer money, especially if you’re giving him stuff that he doesn’t want to hear?

Gary M. Besinque, PharmD, FCSHP: Not too much money. 

Peter Salgo, MD: What are you trying to say?

Juvairiya Pulicharam, MD: Otherwise they won’t make any money, right?

Gary M. Besinque, PharmD, FCSHP: This helps them seek a larger market. The information that we develop together will definitely pay off for them. It will be more than they’re spending to get it.

Peter Salgo, MD: OK. If I understand correctly, they’re not spending much to get it at all. They’re not really funding that, are they?

Juvairiya Pulicharam, MD: At least when we work with organizations, it’s really an internal question. We want to know how we can better educate our physicians so we have the knowledge. How can we really look into our data? So, it’s really for work that we are doing together, and I think the manufacturers are interested.

Peter Salgo, MD: OK.

Juvairiya Pulicharam, MD: Physicians and patients.

Peter Salgo, MD: But you’ve developed this internally, right?

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: And you’re going to follow your patients and keep some sort of database.

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: And you’re going to change and modify your practices based on what you see.

Juvairiya Pulicharam, MD: Yes.

Peter Salgo, MD: Then, one day, somebody says, “Oh, the light bulb went on. Let’s take all of this, fold it into 3 pieces, put it in an envelope, and put a stamp on it and say, “Send it to the manufacturer.” Do they want to get it?

Juvairiya Pulicharam, MD: The way you packaged it, it looks very good. For the manufacturer, what I’m seeing is they’re very interested in knowing the burden of the illness. They are very interested in knowing where the gaps are. 

Peter Salgo, MD: So they are interested. 

Juvairiya Pulicharam, MD: I am seeing that more and more manufacturers, and health systems, and providers are coming together for the first time. Everybody knows that we can’t do it alone.

Peter Salgo, MD: I’ve been in the medical business for a long time. I’ve talked to a lot of manufacturers. I’ve talked to a lot of doctors. This demonization of one group versus another group doesn’t do anybody any good. Aren’t we all on the same page? I don’t want to just join hands and say kumbaya here. The drug manufacturers don’t want to hurt anybody. They would like for their drugs to work, and they’d like to know how best to do that. So, they should be interested. Is that your experience?

Jaime E. Murillo, MD: Yes, the data will be out there. You have to be a part of the system if you want to be moving along with the system. Otherwise, you’re going to be isolated. The fact that you don’t participate in the process doesn’t mean the data won’t be there. It will be there.

Peter Salgo, MD: It’s coming whether you’re ready for it or not.

Jaime E. Murillo, MD: Yes.

Peter Salgo, MD: So, you might as well get on the train.

Jaime E. Murillo, MD: Correct.

 
 
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