Stimulating Patient Engagement in Medical Device Development in Kidney Disease: A Report of a Kidney Health Initiative Workshop.

New technologies challenge current dialysis treatment paradigms as devices become smaller, more portable, and increasingly used outside the dialysis clinic. It is unclear how patients will view this care transition, and it will be important to consider patient and care partner perspectives during all aspects of development for novel dialysis therapies, from design and clinical trials to regulatory approval. To gain insight into this area, the Kidney Health Initiative, a public-private partnership between the American Society of Nephrology, the US Food and Drug Administration, and nearly 80 member organizations and companies dedicated to enhancing patient safety and fostering innovation in kidney disease, convened a workshop of patients, care partners, and other kidney community stakeholders. The workshop included background presentations followed by focused small group discussions in 3 areas (device design, clinical trials, and regulatory approval). Participants explored how to involve patients throughout the life cycle of a medical device, including discussions of how patients can influence device design, assist in the planning and implementation of clinical trials, and provide input to affect regulatory decisions. Patients were engaged in the workshop discussion and interested in sharing their perspectives, but they recommended additional efforts around education, communication, and outreach in these areas.

Overcoming Barriers in Kidney Health-Forging a Platform for Innovation.

Innovation in kidney diseases is not commensurate with the effect of these diseases on human health and mortality or innovation in other key therapeutic areas. A primary cause of the dearth in innovation is that kidney diseases disproportionately affect a demographic that is largely disenfranchised, lacking sufficient advocacy, public attention, and funding. A secondary and likely consequent cause is that the existing infrastructure supporting nephrology research pales in comparison with those for other internal medicine specialties, especially cardiology and oncology. Citing such inequities, however, is not enough. Changing the status quo will require a coordinated effort to identify and redress the existing deficits. Specifically, these deficits relate to the need to further develop and improve the following: understanding of the disease mechanisms and pathophysiology, patient engagement and activism, clinical trial infrastructure, and investigational clinical trial designs as well as coordinated efforts among critical stakeholders. This paper identifies potential solutions to these barriers, some of which are already underway through the Kidney Health Initiative. The Kidney Health Initiative is unique and will serve as a current and future platform from which to overcome these barriers to innovation in nephrology.

Incorporating patient-preference evidence into regulatory decision making.

BACKGROUND: Patients have a unique role in deciding what treatments should be available for them and regulatory agencies should take their preferences into account when making treatment approval decisions. This is the first study designed to obtain quantitative patient-preference evidence to inform regulatory approval decisions by the Food and Drug Administration Center for Devices and Radiological Health. METHODS: Five-hundred and forty United States adults with body mass index (BMI) ≥ 30 kg/m(2) evaluated tradeoffs among effectiveness, safety, and other attributes of weight-loss devices in a scientific survey. Discrete-choice experiments were used to quantify the importance of safety, effectiveness, and other attributes of weight-loss devices to obese respondents. A tool based on these measures is being used to inform benefit-risk assessments for premarket approval of medical devices. RESULTS: Respondent choices yielded preference scores indicating their relative value for attributes of weight-loss devices in this study. We developed a tool to estimate the minimum weight loss acceptable by a patient to receive a device with a given risk profile and the maximum mortality risk tolerable in exchange for a given weight loss. For example, to accept a device with 0.01 % mortality risk, a risk tolerant patient will require about 10 % total body weight loss lasting 5 years. CONCLUSIONS: Patient preference evidence was used make regulatory decision making more patient-centered. In addition, we captured the heterogeneity of patient preferences allowing market approval of effective devices for risk tolerant patients. CDRH is using the study tool to define minimum clinical effectiveness to evaluate new weight-loss devices. The methods presented can be applied to a wide variety of medical products. This study supports the ongoing development of a guidance document on incorporating patient preferences into medical-device premarket approval decisions.

Development of a Patient Preference Survey for Wearable Kidney Replacement Therapy Devices.

Background: Recent innovations have the potential to disrupt the current paradigm for kidney failure treatment. The US Food and Drug Administration is committed to incorporating valid scientific evidence about how patients weigh the benefits and risks of new devices into their decision making, but to date, premarket submission of patient preference information (PPI) has been limited for kidney devices. With input from stakeholders, we developed a survey intended to yield valid PPI, capturing how patients trade off the potential benefits and risks of wearable dialysis devices and in-center hemodialysis. Methods: We conducted concept elicitation interviews with individuals receiving dialysis to inform instrument content. After instrument drafting, we conducted two rounds of pretest interviews to evaluate survey face validity, comprehensibility, and perceived relevance. We pilot tested the survey with in-center hemodialysis patients to assess comprehensibility and usability further. Throughout, we used participant input to guide survey refinements. Results: Thirty-six individuals receiving in-center or home dialysis participated in concept elicitation (N=20) and pretest (N=16) interviews. Participants identified reduced fatigue, lower treatment burden, and enhanced freedom as important benefits of a wearable device, and many expressed concerns about risks related to device disconnection-specifically bleeding and infection. We drafted a survey that included descriptions of the risks of serious bleeding and serious infection and an assessment of respondent willingness to wait for a safer device. Input from pretest interviewees led to various instrument modifications, including treatment descriptions, item wording, and risk-level explanations. Pilot testing of the updated survey among 24 in-center hemodialysis patients demonstrated acceptable survey comprehensibility and usability, although 50% of patients required some assistance. Conclusions: The final survey is a 54-item web-based instrument that will yield estimates of the maximal acceptable risk for the described wearable device and willingness to wait for wearable devices with lower risk.

U.S. Food and Drug Administration and off-label use of expandable metal biliary stents within the peripheral vasculature.

Expandable metal stents are used to maintain the patency of compromised ducts, lumens, and vessels. As medical devices, there products are regulated by the Center for Devices and Radiological Health of the U.S. Food and Drug Administration (FDA). During the past several years, the FDA has become aware of the increasing prevalence of off-label use of expandable metal stents cleared for biliary use within the peripheral vasculature. The authors provide an overview of how the FDA regulates medical devices and expandable stents and summarize safety issues reported to the Agency with regard to the off-label use of these biliary stents.