It has been shown that initial combination therapies in PAH can improve clinical outcomes in patients [1]. Although treatment guidelines may recommend certain prescribing patterns for patients, data from Ipsos’ Global PAH Therapy Monitor (TM; a retrospective medical chart review with physicians reporting on the last five PAH patients seen within the last 3 months) suggest there has been a gradual shift in recent years. In fact, more newly diagnosed patients (defined as patients who have been diagnosed within the last 12 months) are receiving a combination therapy whilst there is a decline in the prescribing of monotherapies for these patients [2].
The objective of the study by Allie et al. was to explore treatment patterns of newly diagnosed PAH patients in the SUSA vs 3 EU countries: the United Kingdom, Germany, and Italy. To do so, data from the Ipsos’ Global PAH TM study were used, totalling 783 patients from 2016, 523 patients from 2017, and 509 patients from 2018; retrospective patient record forms across the 3 EU countries and the US were collected (online). Also, a specific data set was analysed which focused on newly diagnosed PAH patients in the 3 EU countries (2016 n=214; 2017 n=117; 2018 n=64) and the US (2016 n=142; 2017 n=118; 2018 n=74). Eligibility criteria included physicians having at least 5 PAH patients currently under their direct care who are currently prescribed PAH-specific drug therapy. Also, physicians must also have initiated at least one PAH patient onto their current treatment.
The results showed that treatment practices of newly diagnosed patients have changed since 2016; there has been a decrease in the prescription of monotherapies and a shift towards dual therapy. The use of combination therapy in newly diagnosed patients in the US showed a significant increase from 42% in 2016 to 74% in 2018. The use of dual therapy in newly diagnosed patients in the 3 EU countries has gradually increased. In the US, there has also been a growth in dual therapy usage since 2016, with a significant increase from 2017 to 2018 (48% to 70%). With an increase in dual therapy treatment, the use of monotherapy has decreased in both the 3 EU countries since 2016. The 3 EU countries show a gradual decline and in the US there was a significant decline from 2016 to 2018 (58% to 26%). When comparing the US to the 3 EU countries, it is clear that in 2018 there was a significantly lower proportion of newly diagnosed US patients being prescribed a monotherapy and a significantly higher proportion prescribed a dual therapy when compared to the 3 EU countries. In the same year, of those newly diagnosed patients in the US, 61% were prescribed a dual therapy consisting of an ERA and PDE-5i (see Figure).
Figure: PAH-specific regimens in EU and USA in 2018 [2]

Figure used with permission from Ipsos – data are © Ipsos 2019, all rights reserved.
These findings support that there has been a consistent increase in dual therapy and a decrease in monotherapy in both the US and the 3 EU countries. In the US, the majority of newly diagnosed patients were being treated with a dual therapy in 2018, showing a shift in the treatment of these patients since 2016. Of those newly diagnosed patients receiving a dual therapy in 2018, 61% are receiving a combination consisting of an endothelin receptor antagonist (ERA)+ phosphodiesterase type 5 inhibitor (PDE-5i). It is assumed this increase can be attributed to the AMBITION trial. Data should be monitored in subsequent years to examine whether this uptake is a future continual upward trend and if the same level of uptake will occur in the 3 EU countries. Limitations of the study are that – in order to meet the screening criteria of the TM – participating physicians are medium to high prescribers and that only patients receiving PAH-specific drug therapy were included in the study.
1. VA Office of Research and Development. 2016. Retrieved from https://clinicaltrials.gov/ct2/show/NCT01178073.
2. Allie N, et al. Abstract 1242-492. ACC 2019, 16-18 March, New Orleans, USA.
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