OBJECTIVES: PAH is a rare, chronic disease that should be treated with double/triple therapy in most cases, requiring multiple tablets. A single-tablet combination therapy (STCT) of macitentan 10 mg and tadalafil 40 mg compared to equivalent monotherapies is being evaluated in a Phase 3 trial (A DUE, NCT03904693, AC-077A301): patients received 4 tablets during double-blind and 1 tablet (STCT) during open-label treatment. Interviews during the ongoing open-label extension are exploring patient and clinician experience with the STCT.

METHODS: Qualitative, one-on-one, semi-structured web-assisted interviews are being conducted across multiple countries with A DUE trial participants and site investigators. Interim data on their experience of open-label versus double-blind treatment is presented from 4 US patients and 7 clinicians (4 US, 1 Poland, 1 Spain, 1 South Africa).

RESULTS: All participants provided positive feedback on the STCT and preferred the open-label STCT to the four tablets in the double-blind phase. Patients stated the STCT was convenient, aided adherence, and had a positive impact on their day-to-day lives. Patients discussed how taking more tablets made them feel more “sick” compared with STCT. In comparison, the STCT improved their psychological well-being and reduced the stress of managing multiple tablets. Clinicians noted that the typically high pill burden in PAH causes their patients emotional distress, whereas patients had higher treatment satisfaction with the STCT. Clinicians predict the STCT will be well received in clinical practice and endorsed prescribing STCT for treatment-naïve patients.

CONCLUSIONS: Interim analysis suggests that the STCT was well received by patients and clinicians. The reduced pill burden minimized stress, simplified the treatment regimen, and facilitated adherence, which could improve patient outcomes and quality of life. Clinician endorsement of STCT for treatment-naïve patients increases the likelihood that these patients will receive guideline-recommended care.