Special ReportPatient-Reported Outcomes in Clinical Trials of CKD-Related Therapies: Report of a Symposium Sponsored by the National Kidney Foundation and the US Food and Drug Administration
Section snippets
Overview
The constellation of criteria that typically define nephrotic syndrome are proteinuria with protein excretion >3.5 g/d in adults or >40 mg/h/m2 in children, hypoalbuminemia with serum albumin level <3.5 g/dL, hypercholesterolemia, lipiduria, and edema.11 Nephrotic syndrome is a complex condition affecting both children and adults that may result from a variety of primary and secondary glomerular disorders. Patients with nephrotic syndrome also are at higher risk for related conditions, such as
Overview
C-Path is a private not-for-profit organization created in 2005 by the University of Arizona and the FDA with the mission of supporting the FDA's Critical Path Initiative. In late 2008, working with the FDA and the medical products industry, C-Path established the PRO Consortium to develop, test, and qualify PRO instruments as measures of primary or key secondary efficacy end points in clinical trials of new medical products.53 PRO instrument qualification through the FDA's drug development
Conclusion
The participants in the workshop agreed that while there are challenges (Box 1) to developing and using PRO instruments in this population, there would be significant value in using these end points in CKD trials. They supported that there is an opportunity for PRO end points in nephrotic syndrome to improve therapy and care and that the development of PRO instruments is feasible although complex due primarily to the heterogeneity of the condition. Fundamental research examining the experience
Acknowledgements
Support: C-Path's PRO Consortium is supported by grant U01FD003865 from the US FDA and by Science Foundation Arizona under grant SRG 0335-08. The NKF gratefully acknowledges Abbott, Affymax, AMAG Pharmaceuticals, Amgen, Boehringer Ingelheim, Centocor Ortho Biotech, FibroGen, and Takeda, which provided grants to support the conference. Kerry Willis, PhD, and Tom Manley from the NKF assisted in meeting logistics and manuscript preparation.
Financial Disclosure: Dr Perrone has received research
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2022, Kidney MedicineCitation Excerpt :The purpose of this paper is to report qualitative findings on the burden of disease in adolescents, which were obtained during interviews conducted as part of the instrument development process. The methods conformed to recommended guidance concerning the conduct of qualitative research for the development of PRO tools.12-14 The research team included individuals with expertise in qualitative research, PRO development and validation (D.O., J.C.C., A.C.P.), and pediatric nephrology (F.S., L.G.W.); none of the researchers had any prior association with the participants before the study.
Patient-Reported Outcomes in Children With Chronic Kidney Disease
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2019, American Journal of Kidney DiseasesCitation Excerpt :The need to develop PROMs specific to kidney disease subtypes is being prioritized both in the United States and abroad. During a symposium between the National Kidney Foundation and the FDA consisting of nephrologists, patients, and representatives from the pharmaceutical industry and the National Institutes of Health, ADPKD and nephrotic syndrome were determined to be conditions amenable to future measure development.88 The Outcome Measures in Rheumatology Vasculitis Working Group is exploring the utility of PROMIS measures and PROMs specific to antineutrophil cytoplasmic antibody–associated vasculitis.89
Originally published online August 28, 2013.
Because a quorum could not be reached after those editors with potential conflicts recused themselves from consideration of this manuscript, the peer-review and decision-making processes were handled entirely by an Associate Editor (Kevin C. Abbott, MD, MPH) who served as Acting Editor-in-Chief. Details of the journal's procedures for potential editor conflicts are given in the Editorial Policies section of the AJKD website.