About The Study

Biomarkers in Heart Failure

Challenge: 

Heart failure (HF) is a leading cause of morbidity and mortality worldwide. On the basis of data from NHANES 2007–2010, an estimated 5.1 million Americans ≥20 years of age are diagnosed with HF, which is the only cardiovascular diagnosis currently on the rise. Projections show that by 2030, the prevalence of HF will increase 25% from 2013 estimates (AHA computation based on methodology described in Heidenreich et al). HF is the underlying cause of >55,000 annual deaths in the US, and the cause of > 1,000,000 annual hospital visits. Despite medical progress, the 5-year mortality toll from newly diagnosed HF remains approximately 50%.[1]

Key factors in the optimal care of HF are the correct diagnosis of the syndrome, accurate assessment of disease severity, as well as fine tuning its treatment.  Under the leadership of James Januzzi, MD and Hanna Gaggin, MD, The Baim Institute has focused its attention on designing and running biomarker studies, including pivotal studies focused on NT-proBNP. 

The chief difficulty that many sponsors of biomarker studies encounter is the lack of consensus around what the FDA requires for the design of such studies, and then identifying the clinical sites that will be the best fit to enroll the study.

Solution:

The Baim Institute, working with faculty leadership continues to interface with sponsors and the FDA to optimally design high quality clinical trials meeting expectation for regulatory approval, using innovative strategies for efficient and successful trial completion.  For example, for one trial of NT-proBNP testing for acute HF diagnosis and prognosis, the Baim Institute introduced an innovative web based patient informed consent process that documented better patient engagement in the recruitment process.[GS1]  The Baim Institute, serving as the data and clinical coordinating center for the study, and with the leadership of expert faculty, identified study sites that were most interested in the scientific value of this study. 

Outcome:

Through our collaborative approach to study design and Dr. Januzzi’s reputation at FDA, we designed a study that satisfied FDA requirements.  Also, the input of Drs. Januzzi and Gaggin for the initial identification of quality sites allowed us to start with a solid list early on, thereby reducing the need of our Site Management team to filter out many sites and expediting a completed site list. 

Drs. Januzzi and Gaggin have also been the key to the success of the study’s enrollment.  Often, sites in studies encounter enrollment fatigue. However, the commitment of our Faculty to frequent site contact helped to reinforce the scientific importance of the study. This provided the necessary encouragement to continue to bring patients into the study and helped to keep sites motivated to meet enrollment milestones.

These examples of the hands-on engagement of our Faculty on studies designed and run by the Baim Institute, point to the benefits of employing a well-integrated academic leadership plan to run clinical studies. 



[1] - Go AS, Mozaffarian D, Roger VL, Benjamin EJ, et al.; American Heart Association Statistics

Committee and Stroke Statistics Subcommittee. Executive summary: heart disease and stroke statistics—

2013 update: a report from the American Heart Association. Circulation. 2013 Jan 1;127(1):143-52.