On July 16, the Food and Drug Administration (FDA) approved a new indication for Prograf (tacrolimus; Astellas Pharma US Inc.) based on observational study data providing real-world evidence (RWE) of effectiveness. It is the first drug approval based on RWE. The newly approved indication is for use in combination with other immunosuppressant drugs to prevent organ rejection in adult and pediatric patients receiving a lung transplant. Prograf is the first and only approved immunosuppressant drug for this indication.

Prograf is a calcineurin-inhibitor immunosuppressant originally approved to prevent organ rejection in patients receiving liver transplants; expanded approvals were later granted for patients receiving kidney and heart transplants. The approval for use in patients receiving lung transplants was based on RWE data collected from the U.S. Scientific Registry of Transplant Recipients (SRTR) sponsored by the Department of Health and Human Services, which demonstrated a substantial improvement in outcomes among lung transplant patients receiving Prograf as part of their immunosuppression regimen.

In addition to this RWE, the FDA also considered the published randomized controlled trials of Prograf that supported previous approvals and confirmed evidence of effectiveness. The FDA also noted that Prograf was already being used routinely off-label to prevent lung transplant rejection.

Food and Drug Administration (FDA). FDA Approves New Use of Transplant Drug Based on Real-World Evidence [news release] July 16, 2021. Available at: click here. Accessed July 21, 2021.