Opinion
Video
Author(s):
Panelists discuss how riociguat targets the nitric oxide pathway and is approved for both Group 1 PAH and Group 4 chronic thromboembolic pulmonary hypertension, with specific patient selection criteria based on risk stratification and surgical candidacy.
Current Therapeutic Landscape and Limitations
The current PAH therapeutic arsenal includes approximately 15 approved treatments in the United States, representing a dramatic evolution from the single-therapy era of two decades ago. These medications target different pathways including endothelin receptor antagonism, nitric oxide-cGMP signaling, prostacyclin pathways, and the newer activin signaling inhibition. The diversity of available treatments enables individualized therapy selection and combination approaches that can provide synergistic benefits.
Despite therapeutic advances, significant limitations persist in current treatment options, particularly regarding delivery methods, side effect profiles, and complexity of administration. Treatment options range from simple once-daily oral medications to complex 24-hour continuous infusions requiring specialized monitoring and administration. Unfortunately, healthcare inequities and access limitations sometimes result in patients receiving simpler but potentially less appropriate therapies rather than optimal treatment regimens.
The field has addressed some therapeutic limitations through aggressive upfront combination therapy, typically initiating at least two medications simultaneously and sometimes three drugs in severely ill patients. This approach maximizes the probability of targeting effective pathways in individual patients while overcoming the limitation of not having precision medicine approaches. Aggressive monitoring and therapy escalation strategies further help ensure that patients receive effective treatment regimens despite inherent therapeutic challenges.
Stay ahead of policy, cost, and value—subscribe to AJMC for expert insights at the intersection of clinical care and health economics.