RWE Use in Prograf’s Successful Indication Expansion: Making the Case for Fit-for-Purpose Data and Data Transparency
Astellas faced a high-stakes question: how do you structure a successful application for indication expansion when patients are already relying on your drug off-label? Their answer—fit-for-purpose real-world paired with data transparency to facilitate FDA replication of analyses.
When Astellas aimed to expand the use of Prograf (tacrolimus) to prevent rejection in lung transplant patients, they faced a significant hurdle: conducting a randomized controlled trial wasn’t feasible or ethical. There was no approved immunosuppressive agent to prevent rejection in lung transplant patients, but Prograf was already approved for liver, kidney, and heart recipients—and was already the off-label standard of care for lung. So, the team turned to real-world evidence, drawing on a rich, fit-for-purpose data source—the Scientific Registry for Transplant Recipients (SRTR)—which comprehensively captured outcomes for lung transplant patients across the country. Recognizing the importance of a complete and reliable dataset, Astellas supplemented gaps in the registry, such as dosing details, with carefully selected historical controls and existing clinical trial data from other organ transplant approvals.
Transparency was at the heart of their approach. By submitting patient-level data in standardized formats, Astellas enabled the FDA to replicate and expand upon the analyses, building trust and enabling independent verification. Although the FDA noted challenges like residual confounding and some potential misclassification in outcomes, the large treatment effect observed and the quality of the data provided strong confidence in tacrolimus’s effectiveness. This thoughtful combination of well-chosen, real-world data and transparent sharing exemplifies how sponsors can overcome the limits of traditional trials, especially in complex settings, and successfully support regulatory decisions through real-world evidence.
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