A Review of Pediatric Drug Development Programs and FDA Labeling for Immunosuppressive Drugs in Organ Transplant Patients
1Office of Clinical Pharmacology, Office of Translational Science, CDER, US Food and Drug Administration, Silver Spring, MD
2Division of Transplant and Opthalmology Products, Office of New Drugs, CDER, US Food and Drug Administration, Silver Spring, MD
3Tauro University, New York, NY
4University of North Carolina, Chapel Hill, NC.
Meeting: 2018 American Transplant Congress
Abstract number: B245
Keywords: Dosage, Heart transplant patients, Kidney transplantation, Liver transplantation
Session Information
Session Name: Poster Session B: Kidney: Pediatrics
Session Type: Poster Session
Date: Sunday, June 3, 2018
Session Time: 6:00pm-7:00pm
Presentation Time: 6:00pm-7:00pm
Location: Hall 4EF
The development of drugs for organ transplantation spanned the time before and after pediatric regulatory requirements under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act. Therefore pediatric labeling for these drugs may be inconsistent. The objective of this review was to (a) document present FDA labeling for transplant agents in relation to pediatric indications and the Physician Labeling Rule requirements, and (b) to assess published organ transplant pediatric studies for their ability to contribute to pediatric labeling.
Methods: For objective (a), transplant drugs were identified in current or previous clinical use were identified, and the FDA labels were examined as found on Labels@FDA for pediatric content and Physician Labeling Rule (PLR) format. For objective (b), Medline was searched for all articles containing “pediatric” and “transplant” for each drug. Study data was collected for each of the publications reviewed.
Results: The FDA labels for 12 immunosuppressive drugs were examined. Five out of the 12 labels are in PLR format, and 7 labels (58%) contained at least one pediatric indication for the drug in organ transplantation. For those products not labeled for pediatric use, between 8 and 249 (median of 46) published articles were identified with the “pediatric” and “transplant” search for each drug. Tacrolimus had 731 pediatric publications, but is not directly approved in pediatric renal or cardiac transplantation.
Discussion: The current BPCA initiative for labeling off-patent off-label drugs for pediatric use has not included any drugs for organ transplantation. Given the considerable published information on most of the drugs either without a pediatric indication or limited indications, the BPCA program should consider the inclusion of the transplant medications for FDA labeling and a PLR update.
Disclaimer: The material presented represents the thoughts and opinions of the authors, and should not be interpreted as the position of the US Food and Drug Administration.
CITATION INFORMATION: Burckart G., Cavaillé-Coll M., Duong A., Hothem L., Albrecht R. A Review of Pediatric Drug Development Programs and FDA Labeling for Immunosuppressive Drugs in Organ Transplant Patients Am J Transplant. 2017;17 (suppl 3).
To cite this abstract in AMA style:
Burckart G, Cavaillé-Coll M, Duong A, Hothem L, Albrecht R. A Review of Pediatric Drug Development Programs and FDA Labeling for Immunosuppressive Drugs in Organ Transplant Patients [abstract]. https://atcmeetingabstracts.com/abstract/a-review-of-pediatric-drug-development-programs-and-fda-labeling-for-immunosuppressive-drugs-in-organ-transplant-patients/. Accessed May 8, 2025.« Back to 2018 American Transplant Congress