Fact checked byKristen Dowd

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September 05, 2024
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96% of patients with PAH prefer a single-tablet combination therapy

Fact checked byKristen Dowd
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Key takeaways:

  • Out-of-pocket costs and dosing frequency play a big part in combination therapy adoption among patients.
  • One common perceived benefit of single-tablet combination therapy is the idea of taking fewer pills.

When presented with four unlabeled treatment profiles showing five treatment attributes, 96% of patients with pulmonary arterial hypertension chose a single-tablet vs. a multi-tablet combination therapy, according to a poster.

This poster was presented during the Pulmonary Hypertension Association International PH Conference and Scientific Sessions.

Infographic showing perceived benefits of single-tablet PAH combination therapy
Data were derived from Wilson M, et al. Patient preferences regarding the use of combination ERA + PDE5i for the treatment of pulmonary arterial hypertension: Results from a discrete choice experiment. Presented at: Pulmonary Hypertension Association International Pulmonary Hypertension Conference; Aug. 15-18, 2024; Indianapolis.
Sean Studer

“Patients have indicated their preference for a single-tablet combination therapy (STCT) in this study; these findings may help guide the everyday clinician to offer the STCT to patients,” Sean Studer, MD, MSc, vice president of medical affairs, pulmonary hypertension at Johnson & Johnson, told Healio. “If clinicians become aware that a study showed that the vast majority of patients are inclined to STCT, there may be more proactive discussions regarding this option.”

Through an online survey and supplemental questionnaire, Melisa Wilson, DNP, APRN, BC, PH program coordinator and Advanced Lung Disease Institute clinical operations director at AdventHealth Medical Group, and colleagues analyzed responses from 201 patients (88.6% women; 86.1% white; 70.2% did not indicate employment outside the household; 38.3% with a disability) with self-reported PAH to find out what attributes impact patients’ willingness to adopt endothelin receptor antagonist (ERA) + phosphodiesterase 5 inhibitor (PDE5i) therapy, as well as what patients think of a single-tablet form for ERA + PDE5i adoption.

The seven treatment attributes evaluated in this study included: out-of-pocket costs, dosing frequency, number of prior authorizations, number of pharmacies, discontinuation due to side effects, dose increase (titration) and patient support program availability.

More than one-third of the total cohort (36.8%) was currently taking PDE5i + ERA + prostacyclin. Other frequently reported treatment regimens were PDE5i + ERA (17.9%) and PDE5i by itself (15.4%), according to researchers.

After calculating the relative importance for each of the attributes, researchers found higher scores for out-of-pocket costs (33.7) and dosing frequency (31.5) vs. the remaining five attributes. This suggests that these two attributes play a big part in ERA + PDE5i adoption among patients.

Within the dosing frequency category, patients showed willingness to adopt the combination therapy when it consisted of one pill taken once daily, which was the lowest frequency dosing regimen.

Additionally, the out-of-pocket costs attribute was divided into five levels, and patients demonstrated more willingness to take up ERA + PDE5i at the two lowest levels (less than $20 and $20 to $50).

When presented with four unlabeled treatment profiles (three being loose dose combos and one being a fixed-dose combo) showing dosing frequency, number of dose increases, discontinuation due to side effects, pharmacies required and prior authorizations, 96% of patients chose the fixed-dose STCT of macitentan-tadalafil.

“Some health care professionals did not expect that 96% of respondents would prefer single-tablet therapy,” Studer told Healio. “This emphasizes the key role of including the patient’s voice, as their viewpoint is not always accurately estimated.”

Notably, the FDA approved Opsynvi (Johnson & Johnson), a once-daily tablet that includes both macitentan and tadalafil, to treat adults with PAH in March.

“Now that the first STCT for PAH is available, the opportunity exists to convert current patients taking multi-tablet therapy to STCT, as well as to initiate STCT in newly diagnosed patients,” Studer said. “Patient knowledge of STCT may also help drive these discussions with their prescribers.”

The perceived benefits of STCT most commonly reported by patients included that it would mean taking fewer pills (83.1%) and less time managing prescriptions (68.7%), according to the poster.

Further, 42.3% responded, “I would find it easier to remember when to take my medication and stick to my medication schedule.” A similar proportion of patients believed that STCT would mean the possibility of spending less money on medication (39.8%) and the possibility of fewer daily doses (37.3%).

Lastly, 39.3% said STCT would result in fewer doses missed, and 34.8% said they would have started therapy sooner if given STCT.

“The two big takeaways from this study were that patients prefer single-tablet treatment vs. multi-tablet treatment and that costs are an important factor that determines patient choice when considering a single-tablet therapy,” Studer told Healio.

“Future studies may include utilizing the emerging body of real-world data related to STCT in PAH to assess patients’ ability to better adhere to their prescribed regimen and the effect this may have on outcomes,” Studer added.