TY - JOUR
T1 - Drug Development in Kidney Disease
T2 - Proceedings From a Multistakeholder Conference
AU - Edmonston, Daniel L.
AU - Roe, Matthew T.
AU - Block, Geoffrey
AU - Conway, Paul T.
AU - Dember, Laura M.
AU - DiBattiste, Peter M.
AU - Greene, Tom
AU - Hariri, Ali
AU - Inker, Lesley A.
AU - Isakova, Tamara
AU - Montez-Rath, Maria E.
AU - Nkulikiyinka, Richard
AU - Polidori, David
AU - Roessig, Lothar
AU - Tangri, Navdeep
AU - Wyatt, Christina
AU - Chertow, Glenn M.
AU - Wolf, Myles
N1 - Funding Information:
Dr Edmonston reports consulting for Akebia. Dr Roe reports grant support from the American College of Cardiology, American Heart Association, AstraZeneca, Familial Hypercholesterolemia Association, Ferring Pharmaceuticals, Janssen Pharmaceuticals, Myokardia, Patient Centered Outcomes Research Institute, and Sanofi-Aventis; and consulting fees from Amgen, Ardea Biosciences, AstraZeneca, Eli Lilly & Co, Elsevier Publishers, Flatiron, Janssen Pharmaceuticals, Novo Nordisk, Pfizer, Regeneron, Roche-Genentech, Sanofi-Aventis, and Signal Path. Dr Block is an employee of Reata; has consulted for or received support from Kyowa Hakko Kirin, Amgen, Keryx, Akebia, OPKO, Vifor, CARA, AstraZeneca Inc, Mitsubishi, Kirin, Ardelyx, and Reata; is a director of Ardelyx, Inc; owns stock in Reata and Ardelyx, and his wife is an employee of US Renal Care. Mr Conway reports his relationships include but are not limited to the following: Member, Board of Directors, and Chair of Public Policy, American Association of Kidney Patients (occasional reimbursement for travel expenses); Patient Editor, Clinical Journal of the American Society of Nephrology (stipend); Member, Board of Directors, Kidney Health Initiative; Member, Kidney Committee, American Board of Internal Medicine (stipend); Co-Chair, Global Kidney Summit, a collaborative of AAKP & the George Washington University School of Medicine and Health Sciences; Chair, FDA Patient Engagement Advisory Committee; Member, External Expert Panel, Kidney Precision Medicine Project; Member, External Expert Panel, US Renal Data System, National Institute of Diabetes and Digestive and Kidney Diseases; Liaison, Healthcare Infection Control Practices Advisory Committee for the Centers for Disease Control and Prevention and US Department of Health and Human Services; Member, Peer Reviewed Medical Research Program, Congressionally Directed Medical Research Programs at the US Department of Defense (stipend); Speaker, Bayer (2019, speaker fee and travel). Dr Dember receives compensation from the NKF for serving as a Deputy Editor of the American Journal of Kidney Diseases and reports consulting fees from Merck, Cara Therapeutics, Proteon Therapeutics, and GlaxoSmithKline. Dr DiBattiste was a full-time employee of Janssen and shareholder in Johnson & Johnson at the time of this conference. Dr Greene reports personal consulting fees from Janssen Pharmaceuticals, DURECT Corp, and Pfizer Inc, and grant support from AstraZeneca Inc, CSL, and Boehringer Ingleheim. Dr Inker reports funding from the NIH, NKF, Retrophin, Omeros, Dialysis Clinics, Inc, and Reata Pharmaceuticals for research and contracts to Tufts Medical Center; and consulting agreements with Tricida and Omeros Corp. Dr Nkulikiyinka is a full-time employee and stock holder of Bayer AG. Dr Polidori is a full-time employee of Janssen Research & Development and a shareholder of Johnson & Johnson. Dr Roessig is a full-time employee of Bayer AG. Dr Tangri reports grants and personal fees from AstraZeneca Inc; personal fees from Otsuka Inc, Janssen, and Boehringer Ingelheim/Eli Lilly & Co; and grants, personal fees, and other from Tricida Inc. Dr Wyatt reports Academic Steering Committee membership for Allena Pharmaceuticals. Dr Chertow serves on the Board of Directors of Satellite Healthcare, a not-for-profit dialysis provider; has served or currently serves as an advisor or member of a clinical trial Steering Committee with Akebia, Amgen, Ardelyx, AstraZeneca, Baxter, CloudCath, Cricket, DiaMedica, DURECT Corp, Gilead, Outset, Reata, Sanifit, and Vertex; and has served or currently serves on Data Safety and Monitoring Boards with Angion, Bayer, and ReCor. Dr Wolf reports consulting for Akebia, Amag, Amgen, Ardelyx, Diasorin, Luitpold, and Pharmacosmos. All other authors declare that they have no relevant financial interests.
Publisher Copyright:
© 2020 National Kidney Foundation, Inc.
Copyright:
Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2020/12
Y1 - 2020/12
N2 - Occasional bursts of discovery and innovation have appeared during the otherwise stagnant past several decades of drug development in nephrology. Among other recent drug discoveries, the unexpected kidney benefits observed with sodium/glucose cotransporter 2 inhibitors may herald a renaissance of drug development in kidney disease. This recent progress highlights the need to further promote and stimulate research and development of promising therapies that may ameliorate the morbidity and mortality associated with kidney disease. To help identify and address barriers to drug development in nephrology, the Duke Clinical Research Institute convened a conference in April 2019 that included stakeholders from academia, industry, government agencies, and patient advocacy. From these discussions, several opportunities were identified to improve every stage of drug development for kidney disease from early discovery to implementation into practice. Key topics reviewed in this article are the utility of interconnected data and site research networks, surrogate end points, pragmatic and adaptive trial designs, the promising uses of real-world data, and methods to improve the generalizability of trial results and uptake of approved drugs for kidney-related diseases.
AB - Occasional bursts of discovery and innovation have appeared during the otherwise stagnant past several decades of drug development in nephrology. Among other recent drug discoveries, the unexpected kidney benefits observed with sodium/glucose cotransporter 2 inhibitors may herald a renaissance of drug development in kidney disease. This recent progress highlights the need to further promote and stimulate research and development of promising therapies that may ameliorate the morbidity and mortality associated with kidney disease. To help identify and address barriers to drug development in nephrology, the Duke Clinical Research Institute convened a conference in April 2019 that included stakeholders from academia, industry, government agencies, and patient advocacy. From these discussions, several opportunities were identified to improve every stage of drug development for kidney disease from early discovery to implementation into practice. Key topics reviewed in this article are the utility of interconnected data and site research networks, surrogate end points, pragmatic and adaptive trial designs, the promising uses of real-world data, and methods to improve the generalizability of trial results and uptake of approved drugs for kidney-related diseases.
KW - Drug development
KW - administrative data sets
KW - clinical investigation
KW - clinical trial design
KW - conference report
KW - data integration
KW - disease network
KW - drug discovery
KW - electronic health record (EHR)
KW - genetic determinants of disease
KW - health care innovation
KW - kidney disease end points
KW - nephrology research
KW - patient-centered endpoints (PCEs)
KW - post-approval trials
KW - post-marketing studies
KW - pragmatic trial
KW - real-world evidence
KW - surrogate end points
KW - therapeutic target
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U2 - 10.1053/j.ajkd.2020.05.026
DO - 10.1053/j.ajkd.2020.05.026
M3 - Article
C2 - 32768631
AN - SCOPUS:85092221355
VL - 76
SP - 842
EP - 850
JO - American Journal of Kidney Diseases
JF - American Journal of Kidney Diseases
SN - 0272-6386
IS - 6
ER -