Demonstrating Benefit-Risk Profiles of Novel Therapeutic Strategies in Kidney Transplantation: Opportunities and Challenges of Real-World Evidence

Ilkka Helanterä, Jon Snyder, Anders Åsberg, Josep Maria Cruzado, Samira Bell, Christophe Legendre, Hélio Tedesco-Silva, Giovanna Tedesco Barcelos, Yvonne Geissbühler, Luis Prieto, Jennifer B. Christian, Erik Scalfaro, Nancy A. Dreyer (Lead / Corresponding author)

Research output: Contribution to journalComment/debatepeer-review

2 Citations (Scopus)
37 Downloads (Pure)


While great progress has been made in transplantation medicine, long-term graft failure and serious side effects still pose a challenge in kidney transplantation. Effective and safe long-term treatments are needed. Therefore, evidence of the lasting benefit-risk of novel therapies is required. Demonstrating superiority of novel therapies is unlikely via conventional randomized controlled trials, as long-term follow-up in large sample sizes pose statistical and operational challenges. Furthermore, endpoints generally accepted in short-term clinical trials need to be translated to real-world (RW) care settings, enabling robust assessments of novel treatments. Hence, there is an evidence gap that calls for innovative clinical trial designs, with RW evidence (RWE) providing an opportunity to facilitate longitudinal transplant research with timely translation to clinical practice. Nonetheless, the current RWE landscape shows considerable heterogeneity, with few registries capturing detailed data to support the establishment of new endpoints. The main recommendations by leading scientists in the field are increased collaboration between registries for data harmonization and leveraging the development of technology innovations for data sharing under high privacy standards. This will aid the development of clinically meaningful endpoints and data models, enabling future long-term research and ultimately establish optimal long-term outcomes for transplant patients.

Original languageEnglish
Article number10329
Number of pages8
JournalTransplant International
Publication statusPublished - 3 May 2022


  • Clinical Trials as Topic/standards
  • Graft Survival
  • Humans
  • Kidney Transplantation/adverse effects
  • Pragmatic Clinical Trials as Topic/standards
  • Research Design/standards
  • Risk Assessment
  • real-world evidence
  • data harmonization
  • kidney transplantation
  • extension studies
  • registries

ASJC Scopus subject areas

  • Transplantation


Dive into the research topics of 'Demonstrating Benefit-Risk Profiles of Novel Therapeutic Strategies in Kidney Transplantation: Opportunities and Challenges of Real-World Evidence'. Together they form a unique fingerprint.

Cite this