TYVASO External Comparator Study
Research type
Research Study
Full title
Real-World Comparative Effectiveness Study of TYVASO (Inhaled Treprostinil) in the Treatment of PH-ILD
IRAS ID
335576
Contact name
Laura Price
Contact email
Sponsor organisation
Ferrer
Duration of Study in the UK
1 years, 9 months, 21 days
Research summary
Pulmonary hypertension (PH) is a pathophysiological disorder characterised by elevated mean pulmonary arterial pressure that can lead to cardiac dysfunction and failure. WHO Group 3 PH patients – those with PH associated with lung disease and/or hypoxia – experience the most severe outcomes and mortality. Interstitial lung disease (ILD) is one factor that can contribute to the development of PH, with varying prevalence rates depending on the type and severity of ILD.
PH in ILD patients is associated with increased need for supplemental oxygen, reduced mobility, and decreased survival, but given the significant overlap in symptoms in ILD patients with and without PH, diagnosis is difficult. Currently, there are no approved medical treatments for PH-ILD in Europe, however, the recent INCREASE trial demonstrated significant improvement in exercise capacity with inhaled treprostinil, a prostacyclin analogue that reduces pulmonary pressure and improves cardiac function in these patients.
This study will be a multi-country, multi-centre retrospective observational study comparing routinely collected clinical data collected by COMPERA, REHAR and UKRB pulmonary hypertension registries/datasets with existing clinical trial data from INCREASE and INCREASE OLE studies. The aim is to generate evidence on the comparative efficacy and safety of inhaled treprostinil versus standard of care (SOC) in Europe which includes 2 arms: (1) an external comparator group of treatment naïve patients; (2) an external comparator group of patients treated off-label with pulmonary arterial hypertension (PAH) therapy.
By emulating the clinical trials with external comparator group of PH-ILD patients from disease specific registries (COMPERA, REHAR, and UKRB), this study will aim to generate evidence to gauge the applicability of the pivotal INCREASE study to the European setting. Further, this real-world-evidence (RWE) study will provide comparative effectiveness evidence for a substantially longer follow-up window of 28 and 52-weeks, as compared to the placebo-controlled 16-week follow-up of INCREASE.
REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
23/EM/0269
Date of REC Opinion
27 Nov 2023
REC opinion
Favourable Opinion