Clinical Trial Design Principles and Endpoint Definitions for Transcatheter Mitral Valve Repair and Replacement: Part 1: Clinical Trial Design Principles
Why the study?
The introduction of transcatheter mitral valve therapies has highlighted the need for a consensus approach to pragmatic clinical trial design and uniform endpoint definitions to evaluate outcomes in patients with mitral regurgitation.
Structured PICO
P
Population
Patients with primary (organic) or secondary (functional) mitral regurgitation (MR)
I
Intervention
Transcatheter mitral valve repair and replacement therapies
Key expert perspectives
Captured external expert commentary on this paper, strongest first. Original sources are linked where available.
“Appropriate adoption of the common definitional framework and trial design recommendations put forward in these documents will help avoid future problems with trial design that can adversely affect the ability to analyze data. Failure to identify and classify appropriate study populations or failure to define and use suitable individual or composite endpoints that are adequate for providing reasonable assurance of safety or effectiveness can be obstacles to device approval. The MVARC documents aim to address these issues proactively.”
“I think that if the clinician takes the time to read these documents, they'll develop greater understanding of the pathophysiology and approach to mitral regurgitation, current standard of care, therapies, prognosis, as well as the options that are available to their patients both today and tomorrow.”
Implication
Provides standardized consensus recommendations from the Mitral Valve Academic Research Consortium for clinical trial design to evaluate transcatheter and surgical therapies for mitral regurgitation.
Abstract
Mitral regurgitation (MR) is one of the most prevalent valve disorders and has numerous etiologies, including primary (organic) MR, due to underlying degenerative/structural mitral valve (MV) pathology, and secondary (functional) MR, which is principally caused by global or regional left ventricular remodeling and/or severe left atrial dilation. Diagnosis and optimal management of MR requires integration of valve disease and heart failure specialists, MV cardiac surgeons, interventional cardiologists with expertise in structural heart disease, and imaging experts. The introduction of transcatheter MV therapies has highlighted the need for a consensus approach to pragmatic clinical trial design and uniform endpoint definitions to evaluate outcomes in patients with MR. The Mitral Valve Academic Research Consortium is a collaboration between leading academic research organizations and physician-scientists specializing in MV disease from the United States and Europe. Three in-person meetings were held in Virginia and New York during which 44 heart failure, valve, and imaging experts, MV surgeons and interventional cardiologists, clinical trial specialists and statisticians, and representatives from the U.S. Food and Drug Administration considered all aspects of MV pathophysiology, prognosis, and therapies, culminating in a 2-part document describing consensus recommendations for clinical trial design (Part 1) and endpoint definitions (Part 2) to guide evaluation of transcatheter and surgical therapies for MR. The adoption of these recommendations will afford robustness and consistency in the comparative effectiveness evaluation of new devices and approaches to treat MR. These principles may be useful for regulatory assessment of new transcatheter MV devices, as well as for monitoring local and regional outcomes to guide quality improvement initiatives.
Discussion
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Cite This Study
Stone et al. (2015) conducted a review in Mitral regurgitation. Transcatheter mitral valve repair and replacement vs. Surgical therapies for mitral regurgitation was evaluated. The Mitral Valve Academic Research Consortium panel of 44 experts developed a 2-part consensus framework to standardize clinical trial design and endpoint definitions for transcatheter MV therapies.