The rare, chronic condition requires investigators to turn to unique registries and other patient populations to access therapy benefits.
Patients who present with symptoms of pulmonary arterial hypertension could land on any spot of a spectrum which indicates minimal to significant risk of comorbidities, and even mortality. The uncommon, burdensome condition often requires that patients are receiving optimized therapy early and aggressively following diagnosis.
That puts pressure on investigators to broaden their access to clinical data. In an interview with MD Magazine®, Rich Channick, MD, director of the Acute and Thromboembolic Disease Program at the UCLA Medical Center, and Prof. Sean Gaine, director of the National Pulmonary Hypertension Unit in Ireland, explained the utility of national, international registries of PAH patients, and how it influences their ability to identify low- and high-risk patients.
MD Mag: How critical are broad database in assessing care of patients with a rare, chronic condition such as PAH?
Channick: Yeah, I mean there's a lot of creative ways to get data. A lot of people are using claims data, insurance claims, instead of getting a big data. You can find a lot of patience. And now with coding, diagnostic codes, you look at pulmonary hypertension as a code for an insurance claim.
Obviously, there's a lot of different insurance companies. It's different in other countries, but you can really then look at different testing that has been done, what drugs are getting used, and patterns of use. You often can't get very granular, as we say, a higher view. But you can look at it a lot of patients.
Gaine: Yeah, in fact, we saw it at our session today, that we were presenting the GRIPHON data from the VA (Veterans Affairs). So the VA is an enormous database, and patients who attend VA have pulmonary hypertension—some of them—and you can gather data from there. So there are those are national registries that we pour through, not just registries from drug trials and seeing how patients are using them, but the national registries. And we learn a lot from them.
We’ve learned in the last few years that PAH as a disease seems to be sort of shifting—or at least what we're seeing is older patients that we wouldn't have seen previously. So we have to reset where to look for the disease. We used to say, “Oh, this is a disease of idiopathaty from the arterial hypertension.” Formerly, primary pulmonary hypertension was a disease in young women in their mid-30s. So you knew where to look, in the sense of when a young woman arrived in breathless. You thought, this is unusual, maybe it's primary pulmonary hypertension, maybe it's idiopathic pulmonary hypertension.
Now we've learned from these registries that it’s not necessarily always the case. You may have a 55 year old person who comes in says, “I'm breathless.” You’re thinking, well you're not doing any exercise, that's why you're breathless. But, in fact—normal pulmonary function, normal chest, X-ray, ECG. They need to go for an echo, and the echo is the number 1 screening tool to see if a person has problems with the right ventricle. So again, in primary care, someone comes in and it just doesn't fit, think of an echo as a really valuable screening tool.
Channick: Yeah, the last thing I’d say about registries—especially all the registries in Europe and elsewhere—is they have allowed us to start to look at things like risk stratification. One of the big mantras in pulmonary hypertension is determining what risk category are patients in, based on composite parameters—coming up with scores where we can actually profile a patient.
And when you treat a patient with these therapies, they determine whether they are into what we call the low-risk category, meaning they'll have a good prognosis. We quantify that. So, these registries have really allowed us both to develop some of these scores, and then validate them to show that it actually are predictive.
And that's really how we're treating. We have the GRIPHON subgroup to really look at the ability of the agent to lower risk, to put people into a low-risk category—that's really important in our treatment.