FDA approves new use of transplant drug based on real-world evidence

Today, the U.S. Food and Drug Administration approved a new use for Prograf (tacrolimus) based on a non-interventional (observational) study providing real-world evidence (RWE) of effectiveness. FDA approved Prograf for use in combination with other immunosuppressant drugs to prevent organ rejection in adult and pediatric patients receiving lung transplantation.  

Prograf, originally approved to prevent organ rejection in patients receiving liver transplants, was later approved to prevent organ rejection for kidney and heart transplants as well. The drug has also been routinely used in clinical practice for patients receiving lung transplants. Today’s action marks the first approval of an immunosuppressant drug to prevent rejection in adults and pediatric patients who receive lung transplants. Prograf is the only approved immunosuppressant drug product for this population. 

This approval reflects how a well-designed, non-interventional study relying on fit-for-purpose real-world data (RWD), when compared with a suitable control, can be considered adequate and well-controlled under FDA regulations. Specifically, the non-interventional study supporting approval for this new indication used RWD from the U.S. Scientific Registry of Transplant Recipients (SRTR), supported by the Department of Health and Human Services. The data were collected on all lung transplants in the U.S. and were supplemented by information from the Social Security Administration’s Death Master File as a trusted repository of mortality data. A dramatic improvement in outcomes was observed among lung transplant patients receiving Prograf as part of their immunosuppression medications compared to the well-documented natural history of a transplanted drug with no or minimal immunosuppressive therapy.  

In addition to the RWE from the non-interventional study, randomized controlled trials of Prograf used in other solid organ transplant settings provided confirmatory evidence of effectiveness. Additional clinical trial evidence from research publications supports the independent contribution of Prograf as part of a multidrug immunosuppressive regimen.  

Prograf should only be prescribed by physicians experienced in immunosuppressive therapy and management of organ transplant and patients receiving the drug should be managed in facilities equipped and staffed with adequate laboratory and supportive medical resources. Prograf is associated with increased risk of developing lymphoma and other malignancies and is associated with increased susceptibility to bacterial, viral, fungal, and protozoal, including opportunistic infections.

FDA granted the approval to Astellas Pharma US, Inc.