Case Study: Using the MDIC Patient Centered Benefit-Risk Framework to Support an Expanded Indication

Feb 1, 2019Patient Centered Benefit-Risk (PCBR), Regulatory Decision

Patient Introduction

Some patients may require dialysis if their own bodies are unable to filter toxins from their blood. Hemodialysis is a type of dialysis in which blood is pumped from the body through a filter and then back into the body. Typically, in-clinic treatment requires patients to travel multiple times per week to a dialysis center to receive dialysis. In the option of home hemodialysis (HHD), the treatment takes place at the patient’s home (or outside of a clinical setting). Home hemodialysis can be a more convenient option for some patients because they do not have to travel to the clinic and can have a flexible schedule to dialyze on their own time. Previously, FDA had only cleared HHD for use outside the clinical setting when a patient was with a care partner. Patients that lived alone or lost access to a care partner were not eligible for HHD and could only receive dialysis in-center. Home hemodialysis cleared for solo use (solo HHD) could increase treatment options for those dialysis patients who do not have care partners present and willing to accept the associated risks of solo treatment. This case study discusses how the patient preference survey data were considered in the FDA CDRH’s decision to clear an expanded indication for NxStage’s home hemodialysis device.

Summary

Home hemodialysis device manufacturer, NxStage, used the MDIC Patient-Centered Benefit-Risk (PCBR) Framework as a guide in developing a patient preference survey to quantify the acceptable risk of the patient performing hemodialysis at home alone versus at a dialysis center. Data from that survey were considered in the FDA CDRH’s decision to clear an expanded indication for NxStage’s home hemodialysis device. This article reviews how NxStage incorporated quantitative patient preference data in its 510(k) submission, which ultimately received clearance from the FDA.

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