The Gastroenterology and Urology Devices Panel (the Panel) met on June 10, 2009 at the Holiday Inn Gaithersburg located at Two Montgomery Village Avenue in Gaithersburg, Maryland. The Panel met to discuss and provide recommendations regarding general issues related to the use of ultrafiltration devices in the treatment of extracellular fluid overload with a focus in patients experiencing heart failure. Specifically, the panel addressed the use of these devices in the following terms: identifying the most appropriate heart failure patients for whom these treatments should be indicated; determining where these treatments fit within the spectrum of treatment options; and defining what level of clinical evidence is necessary to adequately evaluate and provide labeling for these devices.
The meeting began with an FDA update to the Panel, followed by the FDA presentations on the general issues topic, and an open public hearing. The FDA presentations provided the objectives of the meeting, an overview of ultrafiltration devices and systems, the labeling of current legally marketed devices for the treatment of fluid overload, clinical management of fluid overload, the contemporary treatment of acute decompensated heart failure, and an analysis of reported adverse events.
The Panel discussed three FDA questions related to the use of ultrafiltration devices in the treatment of fluid overload in general, and specifically, the use of ultrafiltration in the treatment of heart failure. The Panel believed that the treatment of fluid overload is not the same in all patient populations that could benefit from ultrafiltration, due to the varying underlying etiologies and the different physiological conditions which make it inappropriate to treat all patients with fluid overload the same. The Panel believed that there is a need for more data from prospective randomized controlled studies for indications for use that specify a disease or condition such as, for the treatment of heart failure. Also, the Panel felt that ultrafiltration therapy should be performed as an inpatient therapy due to the lack of data on its use in alternative settings. The Panel believed that there is inadequate data available to support the use of ultrafiltration as an adjunct or alternative therapy to treat heart failure, but the panel felt that the use of ultrafiltration as a tool to treat fluid overload in general was reasonable for the physician to consider when standard medical care options were exhausted. The panel agreed that there was no data from randomized controlled studies demonstrating ultrafiltration was safe and effective for first line therapy in the treatment of heart failure.
The Panel discussed the level of evidence that would be needed to adequately evaluate the use of an ultrafiltration device for the treatment of heart failure, and generally believed that prospective, randomized controlled trials with appropriate comparators are necessary. With respect to study design, the Panel felt that a sponsor should study the specific patient population that would be reflected in the indications for use of the device, and the Panel recommended primary endpoints of safety, quality of life and other measures of patient benefit, such as rehospitalization. The Panel also recommended that mortality be considered as a secondary endpoint. In addition, the Panel generally believed that it is important to measure kidney function throughout the course of the trial, and that patients be followed for one year to assess the safety of the treatment. The Panel examined inclusion criteria from an ongoing study as potential markers for volume overload, and generally believed that the information provided a starting point for defining volume overload. Lastly, the Panel again reiterated the strong need for safety data and appropriate study designs intended to provide adequate supporting data for an indication for use for a specific disease or patient population, or for use in an outpatient setting.
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