TY - JOUR
T1 - Use of biomarkers to improve immunosuppressive drug development and outcomes in renal organ transplantation
T2 - A meeting report
AU - Mannon, Roslyn B.
AU - Morris, Randall E.
AU - Abecassis, Michael
AU - Axelrod, David
AU - Bala, Shukal
AU - Friedman, Gary S.
AU - Heeger, Peter S.
AU - Lentine, Krista L.
AU - Loupy, Alexandre
AU - Murphy, Barbara
AU - Nickerson, Peter
AU - Sarwal, Minnie
AU - O'Doherty, Inish
AU - Spear, Nicole
AU - Karpen, Stephen R.
N1 - Funding Information:
The authors of this manuscript have conflict of interests to disclose as described by the American Journal of Transplantation. Roslyn B. Mannon: research grants from Transplant Genomics Inc and Care Dx, Inc. Michael Abecassis: co‐founder, shareholder, and consultant—Transplant Genomics Inc. David Axelrod: XynManagement owner/shareholder. Consulting work for CareDx, Inc. Gary Steven Friedman: employee of Pfizer and Pfizer stockholder. Krista L. Lentine: consulting for CareDx, Inc. Alexandre Loupy: holds shares in Cibiltech, a company that develops software. Barbara Murphy: RenalytixAI board member and holds equity in company. Minnie Sarwal: sponsored research grants from Genentech, Bristol Meters Squibb, and Natera. The KIT urine test for early detection of transplant rejection is licensed from the Regents, the University of California by NephroSant, where Dr. Sarwal is a co‐founder and chair of the Scientific Advisory Board. Inish O'Doherty, Nicole Spear, and Stephen R. Karpen: TTC is supported in part by grant number U18FD005320 from the US Food and Drug Administration (FDA) to the Critical Path Institute ( http://c‐path.org ) and through annual dues of member companies. The other authors have no conflicts of interest to disclose.
Publisher Copyright:
© 2020 The American Society of Transplantation and the American Society of Transplant Surgeons
PY - 2020/6/1
Y1 - 2020/6/1
N2 - On September 27-28, 2018 the Food and Drug Administration (FDA) and the Critical Path Institute's Transplant Therapeutics Consortium convened a public workshop titled "Evidence-Based Treatment Decisions in Transplantation: The Right Dose & Regimen for the Right Patient/Individualized Treatment.” The workshop facilitated cooperative engagement of transplant community stakeholders, including pharmaceutical industry, academic researchers, clinicians, patients, and regulators to discuss methods to advance the development of novel immunosuppressive drugs for use in solid organ transplantation. Day 1 focused on the utility of biomarkers in drug development, with considerations for seeking regulatory endorsement for use in clinical trials. Biomarkers add value to drug development by improving patient selection criteria, safety monitoring, endpoint selection, and more. Regulatory endorsement through the FDA Biomarker Qualification Program encourages the use of biomarkers in drug development by instilling confidence and consistency in biomarker interpretation across trials. Public–private partnerships or consortia allow stakeholders to share expertise, resources, and data in pursuit of biomarker qualification. Biomarkers relevant to pretransplant risk assessment, early posttransplant care, and assessment of immune response, immunosuppressive drug efficacy, and graft function as discussed on day 1 of the workshop are described.
AB - On September 27-28, 2018 the Food and Drug Administration (FDA) and the Critical Path Institute's Transplant Therapeutics Consortium convened a public workshop titled "Evidence-Based Treatment Decisions in Transplantation: The Right Dose & Regimen for the Right Patient/Individualized Treatment.” The workshop facilitated cooperative engagement of transplant community stakeholders, including pharmaceutical industry, academic researchers, clinicians, patients, and regulators to discuss methods to advance the development of novel immunosuppressive drugs for use in solid organ transplantation. Day 1 focused on the utility of biomarkers in drug development, with considerations for seeking regulatory endorsement for use in clinical trials. Biomarkers add value to drug development by improving patient selection criteria, safety monitoring, endpoint selection, and more. Regulatory endorsement through the FDA Biomarker Qualification Program encourages the use of biomarkers in drug development by instilling confidence and consistency in biomarker interpretation across trials. Public–private partnerships or consortia allow stakeholders to share expertise, resources, and data in pursuit of biomarker qualification. Biomarkers relevant to pretransplant risk assessment, early posttransplant care, and assessment of immune response, immunosuppressive drug efficacy, and graft function as discussed on day 1 of the workshop are described.
KW - biomarker
KW - clinical trial design
KW - immunosuppressant
KW - kidney transplantation/ nephrology
KW - off-label drug use
KW - translational research/science
UR - http://www.scopus.com/inward/record.url?scp=85081884558&partnerID=8YFLogxK
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U2 - 10.1111/ajt.15833
DO - 10.1111/ajt.15833
M3 - Article
C2 - 32090461
AN - SCOPUS:85081884558
VL - 20
SP - 1495
EP - 1502
JO - American Journal of Transplantation
JF - American Journal of Transplantation
SN - 1600-6135
IS - 6
ER -