PH and PAH: An Overview of Treatment Options and Coverage

A detailed overview of treatment used in PH and PAH, followed by considerations for the coverage of specific strategies.


Charles D. Burger, MD: Group 1 pulmonary arterial hypertension [PAH] includes pulmonary hypertension [PH] associated with connective tissue disease. All of the medications that have been approved for Group 1 PAH can be used for patients with collagen vascular disease, whether it be scleroderma, rheumatoid arthritis, or systemic lupus. There really aren’t any limitations to use of the FDA-approved Group 1 PAH medications in that subpopulation. In liver disease, it’s a bit more challenging because some of the medications have had a history of potentially affecting the liver in an adverse way. It’s a rarer diagnosis, so not all of the studies of medications for pulmonary arterial hypertension have included portopulmonary hypertension in the efficacy studies to show benefit.

Our information is a bit more limited. We do however know—based on some fairly recent studies—that macitentan, which is an endothelin receptor antagonist, has shown benefit in a study called PORTICO. There are some open-label studies with the other medications that have shown benefit. We have experience in pulmonary hypertension centers with all of these medications, generally, with one or two exceptions for portopulmonary hypertension. But it does require some additional judgment and individualization to the patient.

For chronic thromboembolic disease pulmonary hypertension, Group 4 PH, the only medication that has FDA approval is riociguat. It’s very important to understand that a significant number of those patients can be cured with surgery. The initial part of the evaluation for Group 4 PH is to see whether a patient might be eligible for surgery, which has an 85% or greater chance of curing the pulmonary hypertension. If they’re not eligible for surgery, or if they may have had surgery and they’re one of the 15% who had some persistence of pulmonary hypertension, then they would be eligible for consideration for treatment with riociguat as the only FDA-approved medication. There are some other medications that have shown benefit in trials. Macitentan was shown to have some benefit in the MERIT-1 trial, but it does not have an FDA approval, so that would be off label.

Derek van Amerongen, MD, MS, FACOG: In terms of coverage for the management of various aspects of PH and PAH, including subpopulations, that’s not really the role of the health plan. Our job is to make sure that we’re covering the standard-of-care treatments and that those agents—especially when we’re talking about expensive, complex, and powerful drugs that are used in these conditions—are being used in a targeted way to address the patient groups that the drugs were developed for. We look at the materials and methods sections of all of the pivotal trials to understand who the individuals are that the researchers identified as eligible candidates and who are most likely to respond and do well with these drugs. It is not really the role of the health plan to get into the weeds on how we treat one patient versus another. That’s a clinician’s responsibility.

At the end of the day when we’re talking about subpopulations or different aspects of PAH and PH, as well as all other conditions, those are critical decisions for the clinician. The role of the health plan is to make sure that those requests, those treatments are being used within the context of the guidelines and certainly within the context of the FDA label and the pivotal trial results, which should tell all of us who the ideal candidates are.

In terms of the data we look at to make medical policy and coverage decisions, we’re always looking at the evidence-based literature. What is the information from the pivotal trial results? Who were the patients that the researchers identified as being first, eligible for the treatment; and second, most likely to respond and do well? All of us want to make sure that these treatments are being targeted as accurately as possible, so that we’re going to get the maximum and optimal clinical outcomes.

We are constantly going back to the literature. We’re going back to the FDA as labels may change and expand as new indications or modifications of those indications are added. We’re always looking at guidelines from national societies, although I would say that guidelines tend to lag—both regarding evidence-based literature and the FDA labels—because there is a period of time that professional societies need to incorporate new evidence into their guidelines. But the ultimate goal is to make sure that our policies reflect the current standard of care and the current status of the literature.

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