Preferences for pulmonary arterial hypertension (PAH) medications: plain language summary on insights from interviews with patients and doctors in the A DUE phase 3 clinical study

Plain Language Summary: What is this summary about? This article presents insights from interviews with 26 patients and 18 doctors who took part in a phase 3 clinical study, called the A DUE study. This study investigated a single-tablet (or single-pill) combination therapy in people living with pulmonary arterial hypertension (PAH), a severe and rare progressive lung condition. The single-pill combination therapy contains 2 medications to treat PAH (macitentan and tadalafil) and is now approved in several countries. In the clinical study, patients were split into 3 different groups (macitentan only, tadalafil only, or single-pill combination therapy only) for 16 weeks. Patients in all groups were given 4 pills a day to treat PAH. Some of these were placebo pills. This was to ensure nobody knew which medication they were taking until the end of the study, to avoid biased results. After 16 weeks, all patients received only the single-pill combination therapy (1 pill per day) for up to 2 years. The interviews occurred during or within 2 weeks after this 2-year period finished. What was this interview study about? Patients and doctors from the clinical study were asked about their thoughts on PAH treatment, their experience with the single-pill combination therapy compared with the 4 pills required earlier in the study, and how it might impact patients taking medication as recommended (known as adherence). What were the key takeaways? The interviews with patients and doctors revealed that taking 1 pill a day for PAH was considered easier than taking multiple pills per day (4 in this study) and reduced stress and worries around managing multiple pills. Both patients and doctors felt that 1 pill would reduce the pill burden and help patients take their medication as recommended. The findings are relevant for shared decision-making on treatment options for PAH. Shared decision-making describes the process where patients and doctors work together to make decisions that reflect what matters most to the patient - their values, priorities and preferences. In summary, these results provide support from both patients and doctors for the use of the single-pill combination therapy in the medication of PAH.