An article titled “Future treatment paradigms in pulmonary arterial hypertension: a personal view from physicians, health authorities, and patients”, was published in The Lancet on February 20, 2025. It reports on discussions held between a group of international experts at the 20th Global CardioVascular Clinical Trialists Forum, CVCT (Washington DC, USA), patients’ representatives, and members of global regulatory agencies to discuss future treatment objectives and trial designs in pulmonary arterial hypertension. Important questions were raised around the challenges in recognising a treatment as “disease modifying”, as this implies showing a persisting treatment effect after drug withdrawal, which has not yet been studied in pulmonary arterial hypertension, and which poses ethical concerns.
The representatives of the regulatory agencies indicated they are moving away from making the disease modifying designation a priority in pulmonary arterial hypertension and are more interested in trials that advance interventions with significant pathophysiological effect, in particular showing reverse pulmonary vascular remodelling, which is likely to bring about more potent clinical response, partial or complete remission, and perhaps even a cure.
What is more important to patients? The designation of a drug as a disease modifying agent? Or outcomes that are directly relevant to them such as how they feel, function, and survive? The concept of partial and complete remission might better suit the needs of this disease area. This article addresses these issues and discusses possible definitions of what might constitute a partial or full remission in pulmonary arterial hypertension.
Read the full article at this link on The Lancet Respiratory Medicine
Citation
Franck F Rahaghi, Marc Humbert, Marius M Hoeper, R James White, Robert P Frantz, Paul M Hassoun, Anna R Hemnes, Steven M Kawut, Vallerie V McLaughlin, Gergely Meszaros, Peter G M Mol, Steven D Nathan, Mitchel A Psotka, Farbod N Rahaghi, Olivier Sitbon, Norman Stockbridge, Jason Weatherald, Faiez Zannad, Sandeep Sahay,
Future treatment paradigms in pulmonary arterial hypertension: a personal view from physicians, health authorities, and patients, The Lancet Respiratory Medicine, 2025, ISSN 2213-2600, https://doi.org/10.1016/S2213-2600(24)00425-9
(https://www.sciencedirect.com/science/article/pii/S2213260024004259)