Pulmonary arterial hypertension (PAH) is a condition characterized by high blood pressure in the pulmonary artery that carries blood from the heart to the lungs. PAH is a heterogeneous disease, meaning it has several root causes, and how the disease manifests is determined by a number of factors, such as PAH subtype, genetic background, age, sex, and comorbidities. Although PAH is a rare disease, there have been several developments in effective therapies and therapeutic strategies over the past 30 years that have improved the management and outcomes of the disease. Despite these advancements, many patients still face a poor prognosis.
In a recent paper published in The Lancet Respiratory Medicine, co-authored by CVC Associate Faculty member Dr. Jason Weatherald, researchers sought to provide a comprehensive summary of advances in PAH pathophysiology, current therapeutic approaches, and emerging treatment options, along with a forward-looking dialogue on innovative solutions for future PAH trials. They determine that increased awareness of the mechanisms and pathways involved in the origin and development of PAH has led to progress translating clinical discoveries into patient care and an expanding list of treatment options. Although ongoing and future PAH clinical trials face distinct challenges, the researchers suggest these trials will benefit from efforts to improve representation from diverse paediatric and adult patient groups, as well as the implementation of emerging technologies, innovative clinical trial designs, and novel new therapies.
In summary, Dr. Weatherald says “There are exciting new treatment options on the horizon for patients with PAH. Yet, we see several important challenges to future clinical trial design and conduct. To continue to move the field forward, we will need to develop innovative solutions to improve PAH clinical trial efficiency, some of which are discussed in this article. We illustrate the need to improve accessibility for patients to participate in trials. By doing this researchers may also improve the diversity of participants, so that future trials are not only more feasible but have better representation, with results that apply more broadly to patients in the real-world.”