Early Intervention: Better Clinical and Economic Outcomes

by Phyllis Hanlon, Contributing Writer  

Researchers who study WHO Group 1 PH (PAH, pulmonary arterial hypertension) have shown that drugs may delay progression of the disease, reduce symptoms and improve quality of life. More recently, scientists have found that using more than one drug early when a patient is newly diagnosed can produce an even better result. But what about the financial costs associated with these drugs? Do the financial costs of taking more than one drug outweigh the costs that might have been incurred if the patient was, for example, hospitalized because the disease was not as well treated? In 2017, a research team embarked on a study to find some answers.

Charles D. Burger, M.D., Division of Pulmonary, Allergy and Sleep Medicine at the Mayo Clinic in Jacksonville, Florida, led a team of authors in reviewing studies published from January 2005 to August 2017 and included in reputable medical journal databases to determine how PAH drugs affect health and health care costs. They also examined the impact of early intervention on the disease. The authors hail from the Mayo Clinic and Allegheny Health Network in Pittsburg, which are both Pulmonary Hypertension Association (PHA)-accredited centers.

The EARLY study of bosentan (Tracleer® compared to placebo) was the first clinical trial to suggest that early treatment of the disease, rather than waiting for a patient’s disease to progress before treating, was beneficial for patients. In this study, patients with mild PAH symptoms were randomized to receive either treatment with bosentan or a placebo. It found that bosentan decreased pulmonary vascular resistance (PVR) by 23 percent, and that 18 percent of patients’ functional class was improved on follow-up at 3.6 years. Six-minute walk distance (6MWD), however, was not statistically significantly increased in the treatment group.

The AMBITION trial used an upfront combination of ambrisentan (Letairis®) and tadalafil (Adcirca®) compared to the drugs used individually, and included patients who had not previously received PH-targeted therapy. The results showed that patients receiving the two-drug combination did better and had fewer disease-related events compared to patients taking either drug individually. The benefit was observed regardless of the subtype of PAH such as in those patients with connective tissue disease-associated PAH, like scleroderma. According to the authors, the AMBITION trial “…may change the [pattern] of PAH therapy.”

The SERAPHIN trial tested the effect of macitentan compared to placebo and found that patients in World Health Organization (WHO) functional class (FC) II, III or IV who were treated with macitentan had fewer hospitalizations and delayed worsening of their disease, in both the patients who were on the drug alone or combined with a PDE5-inhibitor or an oral or inhaled prostanoid.

Similarly, selexipag used in the GRIPHON trial demonstrated delayed disease progression and reduced hospital admissions. The PATENT-1 study examined the use of riociguat and found an increase in 6MWD and a 29 percent reduction in PVR. Patients in both of these studies were receiving background therapy.

The authors also examined health care-related costs of PAH treatment. Dr. Burger and his colleagues reported that specialty prescriptions, which include drugs for PAH, account for more than one-quarter of all prescription drug costs in the U.S., despite only a few patients taking them. The spending on these drugs continues to grow, with specialty drug cost growth doubling the growth in traditional drugs 2016, i.e., 8.4 percent versus 3.4 percent. However, they pointed out that some studies they reviewed suggested that fewer hospitalizations might offset the high price of PAH drugs.

While it’s difficult to pin down exact cost of “healthcare utilization” in the United States—due to the variability in health systems and health insurance to name only two—

the authors affirm that drug costs can take a huge financial toll. For instance, Dr. Burger asserted that the annual wholesale cost for the individual PAH-specific medications currently available ranges between $25,000 and $250,000. Combination treatment raises the price tag, e.g., ambrisentan and tadalafil together could cost $125,802 per year; the cost for the patient varies depending on health insurance and availability of charitable assistance.

When the authors examined hospital costs for patients with PAH, they found a significant discrepancy between PAH-associated costs with those for other primary diagnoses. For example, patients admitted to the hospital due to PAH spent $61,922, while patients hospitalized for other diagnoses paid $42,455 in hospital costs. But when the PAH drugs reduced hospitalization and length of hospital stay, those costs were significantly reduced.

Dr. Burger and his fellow researchers suggested that more innovative approaches to treatment would offer patients a clinical benefit and would also be kinder to the wallet. They pointed out that nanotechnology and individualized medicine is helping to create better and more targeted delivery systems. Also, implantable monitors are helping clinicians keep a closer eye on a patient’s condition and enable better disease management in other diseases. These advances may also help reduce the cost of PAH-related drugs, they noted.


Each PH patient is different. It is essential that you talk to your own doctor about what treatment options are best for you. For more information on finding a doctor or an accredited care center, visit https://www.phassociation.org/PHCareCenters/Patients

2018-05-09T18:26:10+00:00 May 9th, 2018|