The importance of drug safety and tolerability in the development of new immunosuppressive therapy for transplant recipients: The Transplant Therapeutics Consortium's position statement

Am J Transplant. 2019 Mar;19(3):625-632 doi: 10.1111/ajt.15214.
Abstract

The Transplant Therapeutics Consortium (TTC) is a public-private partnership between the US Food and Drug Administration and the transplantation community including the transplantation societies and members of the biopharmaceutical industry. The TTC was formed to accelerate the process of developing new medical products for transplant patients. The initial goals of this collaboration are the following: (a) To define which aspects of the kidney transplant drug-development process have clear needs for improvement from an industry and regulatory perspective; (b) to define which of the unmet needs in the process could be positively impacted through the development of specific drug-development tools based on available data; and (c) to determine the most appropriate pathway to achieve regulatory acceptance of the proposed process-accelerating tools. The TTC has identified 2 major areas of emphasis: new biomarkers or endpoints for determining the efficacy of new therapies and new tools to assess the safety or tolerability of new therapies. This article presents the rationale and planned approach to develop new tools to assess safety and tolerability of therapies for transplant patients. We also discuss how similar efforts might support the continued development of patient-reported outcome measures in the future.

Metadata
Organisation: Stegall, Mark D. Mayo Clinic, Rochester, Minnesota. Troy Somerville, Kenneth. Sanofi Genzyme, Medical Affairs, Cambridge, Massachusetts. Everly, Matthew J. Terasaki Research Institute, Los Angeles, California. Mannon, Roslyn B. University of Alabama at Birmingham, Birmingham, Alabama. Gaber, A Osama. Houston Methodist, Houston, Texas. First, M Roy. Transplant Genomics Inc., Mansfield, Massachusetts. First, M Roy. Comprehensive Transplant Center, Northwestern University, Chicago, Illinois. Agashivala, Neetu. Novartis Pharmaceuticals Corporation, East Hanover, New Jersey. Perez, Vanessa. Astellas Pharma Global Development, Inc., Northbrook, Illinois. Newell, Kenneth A. Emory University School of Medicine, Atlanta, Georgia. Morris, Randall E. Stanford University School of Medicine, Stanford, California. Sudan, Debra. Duke University, Durham, North Carolina. Romero, Klaus. Critical Path Institute, Tucson, Arizona. Eremenco, Sonya. Critical Path Institute, Tucson, Arizona. Mattera, Maria. Critical Path Institute, Tucson, Arizona. Spear, Nicole. Critical Path Institute, Tucson, Arizona. Porter, Amy C. Critical Path Institute, Tucson, Arizona. O'Doherty, Inish. Critical Path Institute, Tucson, Arizona.
Funding: Industry funding
Publication type: Guideline
Organ: Various; Kidney; Liver; Heart; Lung; Pancreas; Intestine; Simultaneous Kidney/Pancreas; Heart/Lung
Language: English
MeSH terms: Consensus; Drug-Related Side Effects and Adverse Reactions; Humans; Immunosuppressive Agents; Maximum Tolerated Dose; Organ Transplantation; Patient Safety; Prognosis; Risk Assessment; Societies, Medical; Transplant Recipients; 0 (Immunosuppressive Agents); Heart Transplantation; Kidney Transplantation; Liver Transplantation; Heart-Lung Transplantation; Lung Transplantation; Pancreas Transplantation