Thomas R. Fleming

TL;DR: Among the 222 patients completing one year of treatment with sildenafil monotherapy, the improvement from baseline at one year in the distance walked in six minutes was 51 m, and the incidence of clinical worsening did not differ significantly between the patients treated with s Bildenafils and those treated with placebo.

TL;DR: Surrogate end points are rarely, if ever, adequate substitutes for the definitive clinical outcome in phase 3 trials and proper justification for such replacement requires that the effect of the intervention on the surrogate end point predicts the effect on the clinical outcome.

TL;DR: The updated Nice classification for PH has been enhanced to include a greater depth of CHD and emphasizes persistent PH of the newborn and developmental lung diseases, such as bronchopulmonary dysplasia and congenital diaphragmatic hernia.

TL;DR: In insight into why indirect measures such as biomarkers may fail to provide reliable evidence about the benefit-to-risk profile of interventions, the definitions of clinically meaningful endpoints and surrogate endpoints are discussed, and examples from recent clinical trials are provided.

TL;DR: An overview of the opportunities and challenges that await the development of novel treatments for PAH is provided and several potential targets in varied stages of drug development exist.