Key result
Biomarkers and adaptive trial designs address key challenges in developing novel pulmonary arterial hypertension therapies.
This review highlights the challenges in developing novel treatments for pulmonary arterial hypertension, emphasizing the need for better target validation, dose selection, and adaptive trial designs.
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May guide future PAH trial designs with biomarkers and adaptive methods; leaves open optimal validation strategies.
Developing new treatments for pulmonary arterial hypertension (PAH) is a challenge. We have enjoyed success with regulatory approvals for three drug classes-prostanoids, endothelin receptor antagonists and phosphodiesterase type 5 inhibitors. But we have also seen some disappointing results, for example, from studies with vasoactive intestinal polypeptide, statins and tergolide. Animal models are an unreliable predictor of efficacy in humans. The best model for the disease is the patient. This review discusses three major issues facing the evaluation of drugs in PAH patients-target validation, choosing the right dose, and early trial design.
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Martin R. Wilkins (2013) conducted a review in Pulmonary Arterial Hypertension. Developing new treatments for pulmonary arterial hypertension requires overcoming challenges in target validation, dose selection, and trial design through the use of biomarkers and adaptive designs.
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