This article is not freely available but a pdf version can be purchased on the Lancet web page at this link
A summary of the article is provided which explains that pulmonary arterial hypertension trial design has shifted from short-term sub-maximal exercise capacity as a primary endpoint, to larger clinical event-driven trial outcomes. Event-driven pulmonary arterial hypertension trials could face feasibility and efficiency issues in the future, say the authors, because increasing sample sizes and longer follow-up durations are needed, which would be problematic in such a rare disease. Enrichment strategies, innovative and alternative trial designs, and novel trial endpoints are potential solutions that could improve the efficiency of future pulmonary arterial hypertension trials while maintaining robustness and clinically meaningful evidence.
Jiason Weatherald, Athénaïs Boucly, Anthony Peters, David Montani, Krishna Prasad, Mitchell A Psotka, Faiez Zannad, Mardi Gomberg-Maitland, Vallerie McLaughlin, Gérald Simonneau, Marc Humbert, The evolving landscape of pulmonary arterial hypertension clinical trials, The Lancet, Volume 400, Issue 10366, 2022, Pages 1884-1898, ISSN 0140-6736, https://doi.org/10.1016/S0140-6736(22)01601-4.