We have heard a lot about the use of real world evidence (RWE) and real world data (RWD) lately in the context of potential approval of new uses of approved drugs.  Well, on July 16, 2021, FDA “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.”  The data ultimately demonstrated that 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.”

The 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” the FDA says.  The Agency also noted that RWE was gleaned from randomized controlled trials of the use of Prograft in other organ transplants.  The full FDA announcement of this approval for a new use (here) provides additional direction on how data might be used to support RWE and RWD for other approvals and emphasizes the need for substantive data to support a determination of efficacy.  Not only is the road map gaining clarity, the reality of RWE/RWD to support approval is confirmed by the FDA action.