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Qualification of biomarkers for drug development in organ transplantation
Gilbert J Burckart1, Shashi Amur, Federico M Goodsaid
1School of Pharmacy, University of Southern California, Los Angeles, CA, USA. burckart@usc.edu
Abstract:
The drug development process is dependent upon having established end points for measuring drug efficacy and adverse effects. New drug development in organ transplantation suffers from having end points which are either outdated or which do not serve the purpose of addressing the current critical drug therapy problems. Numerous biomarkers have been examined in organ transplantation, but almost all would be classified as exploratory for drug development purposes. Some of the possible pathways out of this dilemma include investigator- or consortium-initiated research that would qualify the biomarkers as either probable or known valid biomarkers, help in identification of new end points in transplantation and their associated biomarkers, co-development of a new biomarker and drug for transplantation and the use of new clinical trial design methods which facilitate enriched or stratified transplant patient populations. With new biomarkers and new study design methodologies for drug development, improvement in the drug development process for transplantation is a real possibility that the transplant clinical and research community can help to bring about.
Insights
Drug development for organ transplantation needs better endpoints. New biomarkers and clinical trial designs can improve drug efficacy and safety assessments for transplant patients.
Area of Science:
- Pharmacology and Transplant Medicine
- Biomarker Discovery and Validation
- Clinical Trial Design
Background:
- Current drug development for organ transplantation faces challenges due to outdated or inadequate endpoints for measuring drug efficacy and adverse effects.
- Existing biomarkers in transplantation are largely exploratory and not yet validated for drug development purposes.
- Critical drug therapy problems in transplantation remain unaddressed by current assessment methods.
Purpose of the Study:
- To highlight the limitations of current drug development endpoints in organ transplantation.
- To propose strategies for improving the drug development process in transplantation.
- To emphasize the role of novel biomarkers and advanced clinical trial designs.
Main Methods:
- Review of existing challenges in organ transplant drug development endpoints.
- Exploration of potential solutions including investigator-initiated research and consortium efforts.
- Consideration of biomarker qualification, identification of new endpoints, and co-development strategies.
- Evaluation of novel clinical trial designs, such as enriched or stratified patient populations.
Main Results:
- Identified a critical need for validated biomarkers and relevant endpoints in transplant drug development.
- Proposed pathways include biomarker qualification, new endpoint discovery, and integrated drug-biomarker development.
- Highlighted the potential of innovative clinical trial methodologies to enhance patient stratification.
Conclusions:
- Improving drug development for organ transplantation requires the establishment of valid biomarkers and endpoints.
- Collaborative research and novel clinical trial designs are essential for advancing transplant therapies.
- The transplant community can actively contribute to developing better drug assessment strategies.
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