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Updated: Sep 20, 2025
Digital Home-Monitoring of Patients after Kidney Transplantation: The MACCS Platform
Published on: April 12, 2021
Rationale for Surrogate Endpoints and Conditional Marketing Authorization of New Therapies for Kidney Transplantation
Maarten Naesens1, Alexandre Loupy2, Luuk Hilbrands3
1Department of Microbiology, Immunology and Transplantation, KU Leuven, Leuven, Belgium.
Abstract:
Conditional marketing authorization (CMA) facilitates timely access to new drugs for illnesses with unmet clinical needs, such as late graft failure after kidney transplantation. Late graft failure remains a serious, burdensome, and life-threatening condition for recipients. This article has been developed from content prepared by members of a working group within the European Society for Organ Transplantation (ESOT) for a Broad Scientific Advice request, submitted by ESOT to the European Medicines Agency (EMA), and reviewed by the EMA in 2020. The article presents the rationale for using surrogate endpoints in clinical trials aiming at improving late graft failure rates, to enable novel kidney transplantation therapies to be considered for CMA and improve access to medicines. The paper also provides background data to illustrate the relationship between primary and surrogate endpoints. Developing surrogate endpoints and a CMA strategy could be particularly beneficial for studies where the use of primary endpoints would yield insufficient statistical power or insufficient indication of long-term benefit following transplantation.
Insights
Conditional marketing authorization (CMA) can speed up access to new kidney transplant drugs. Using surrogate endpoints in trials helps demonstrate long-term benefits for late graft failure, improving patient access to innovative therapies.
Area of Science:
- Organ transplantation
- Nephrology
- Pharmacovigilance
Background:
- Late graft failure is a critical, life-threatening complication for kidney transplant recipients.
- Existing treatments for late graft failure are limited, highlighting an unmet clinical need.
- Conditional marketing authorization (CMA) offers a pathway for timely drug access.
Purpose of the Study:
- To present the rationale for employing surrogate endpoints in clinical trials for late graft failure.
- To support the use of CMA for novel kidney transplantation therapies.
- To improve patient access to innovative medicines for post-transplant complications.
Main Methods:
- Review of existing data and scientific literature on surrogate endpoints in transplantation.
- Analysis of the relationship between primary and surrogate endpoints in clinical trials.
- Development of a strategy for utilizing CMA in kidney transplantation research.
Main Results:
- Surrogate endpoints can provide sufficient evidence for CMA when primary endpoints lack statistical power.
- A well-defined surrogate endpoint strategy can accelerate the evaluation of new therapies.
- This approach is particularly relevant for conditions like late graft failure where long-term outcomes are challenging to measure.
Conclusions:
- Developing and validating surrogate endpoints is crucial for enabling CMA in kidney transplantation.
- CMA, supported by surrogate endpoints, can significantly improve access to life-saving therapies.
- This strategy addresses the limitations of traditional trial designs for late graft failure.
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