The Medicare Evidence Development and Coverage Advisory Committee voted it had low-to-intermediate confidence that HF hospitalizations are standalone, meaningful primary outcomes in studies, and it had intermediate confidence in the use of HF hospitalizations and events equivalent to hospitalizations such as outpatient IV diuretic therapy.
The committee was asked to advise CMS on which outcomes should be included in studies of technologies for HF treatment. This information will eventually help CMS decide what evidence it prefers to see before agreeing to cover HF technologies.
Regarding the use of surrogate and intermediate endpoints, the panel voted that it had low-to-intermediate confidence on the following: HF with preserved ejection fraction, HF secondary to mitral regurgitation where the focus is the repair or replacement of the mitral valve and HF with reduced ejection fraction.
The panel voted it had intermediate-to-high confidence that quality-of-life measures are an adequate reflection of patient experience, though they had intermediate confidence regarding use of either as a standalone outcome or a composite outcome.
Functionality assessments such as the 6-minute walk test received a vote of intermediate confidence from the panel. Panelists voted that they had low-to-intermediate confidence for its use as a standalone primary outcome vs. intermediate confidence for composite primary outcome.
Philip B. Adamson, MD, MSc, FAAC, vice president of medical affairs and medical director at Abbott, said identifying the areas of unmet medical needs through these studies will allow physicians to focus on meaningful patient outcomes while improving patient experience.
Philip B. Adamson
Benefits such as quality of life and freedom from hospitalization should also be considered as endpoints, John D. Carroll, MD, professor of medicine at the University of Colorado School of Medicine and director of interventional cardiology at University of Colorado Hospital, said to the panel. He said it’s important to measure patient-reported health status, as it will allow physicians to best decide what will best optimize their patient’s survival and health.
John D. Carroll
“If we want to help patients make informed decisions about new [HF] technologies, then we need their input from the start,” Larry A. Allen, MD, MHS, medical director of advanced heart failure at the University of Colorado, said to the panel. “These treatment decisions not only affect the patient, but also their families and caregivers, and can often be emotional.”
Quality of life
Clinton A. Brawner, PhD, clinical exercise physiologist at the Henry Ford Hospital, who represented the Cardiovascular Research Foundation, told the panel about the benefits of peak VO2 and the 6-Minute Walk Test in clinical studies. “[They’re] closely related to outcomes across the spectrum of [CVD], particularly in HF,” Brawner said.
Adding other measures of quality of life including the Kansas City Cardiomyopathy Questionnaire is clinically significant, according to Nancy Sweitzer, MD, director of the Sarver Heart Center at the University of Arizona. “Failure to include measure of quality of life is failure to comprehensively study an intervention,” Sweitzer said.
“Current and future therapies must focus not only on mortality, but perhaps even more on HF and related outcomes,” Srihari S. Naidu, MD, FACC, FSCAI, director of the hypertrophic cardiomyopathy program and interventional cardiologist at Westchester Medical Center in Valhalla, New York, and associate professor of medicine at New York Medical College, told the panel. He said endpoints such as days alive and out of the hospital or number of hospitalizations should be considered in studies, as they provide value to all involved, including the health care system.
Srihari S. Naidu
Rita Redberg, MD, MSc, chair of the committee, professor of medicine at the University of California, San Francisco, School of Medicine, and a member of the Cardiology Today Editorial Board, said caution must be exercised before encouraging use of implantable technologies.
“I don’t know of any devices that get pulled off the market when we find out that they actually didn’t work or they’re harmful, and I don’t think right now, post-marketing … [evidence is] robust at all,” she said. “So that means essentially, we’re putting untested or minimally tested and possibly unsafe devices in patients that are now permanently implanted.”– by Darlene Dobkowski
Disclosure: MEDCAC panel members reported the following financial disclosures: Michael J. Fisch, MD, MPH, FACP reports employment with AIM Specialty Health; Eileen Hsich, MD, reports receiving institutional research support from Novartis and St. Jude Medical, and unpaid consulting for Medtronic and St. Jude Medical; and Adi Renbaum, MBA, reports being an industry advisor representative working for variety of medical device companies. Adamson is an employee of Abbott. Allen reports being a member of the advisory committee for the National Quality Forum; consulting for Janssen, Novartis, and ZS Pharma; and serving on boards for Boettcher Foundation and PCPI. Brawner reports laboratory contracts with Actelion, Covance and Heart Metabolics. Naidu reports serving on advisory boards for Abbott Vascular and MyoKardia. Sweitzer reports leading clinical trials sponsored by Merck and Novartis, and consulting for Acorda Therapeutics and Medtronic.