Why the study?
Phase 2 and 3 trial designs in pulmonary hypertension need to diversify beyond traditional randomized double-blind designs to evaluate novel therapeutic approaches.
Key points are not available for this paper at this time.
Design
Review article and expert debate platform
Trial designs in pulmonary hypertension should diversify beyond traditional RCTs; leaves open optimal endpoints and populations for future studies.
This article on clinical trial design incorporates the broad experience of members of the Pulmonary Vascular Research Institute's (PVRI) Innovative Drug Development Initiative (IDDI) as an open debate platform for academia, the pharmaceutical industry and regulatory experts surrounding the future design of clinical trials in pulmonary hypertension. It is increasingly clear that the design of phase 2 and 3 trials in pulmonary hypertension will have to diversify from the traditional randomised double-blind design, given the anticipated need to trial novel therapeutic approaches in the immediate future. This article reviews a wide range of differing approaches and places these into context within the field of pulmonary hypertension.
No takes yet. Share an insight, caveat, or question.
Nikkho et al. (2020) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: