From Our Perspective: FDA Approval Demonstrates the Role of Real-World Evidence in Regulatory Decision-Making on Drug Effectiveness In this CDER From Our Perspective, experts discuss the recent FDA approval of Prograf (tacrolimus) in combination with other immunosuppressant drugs for the new indication of preventing organ rejection in adult and pediatric patients receiving lung transplantation. This action marked the first approval of an immunosuppressant drug to prevent lung transplant rejection. The approval is also significant because it reflects how a well-designed, non-interventional (observational) study relying on fit-for-purpose (i.e., reliable and relevant) real-world data (RWD), when compared to a suitable control, can be considered adequate and well-controlled under FDA regulations. As demonstrated by the Prograf approval for the indication of preventing organ rejection in adult and pediatric patients receiving lung transplants, real-world evidence (RWE) can play a significant role in regulatory decision-making when appropriate. According to FDA's definition, RWE is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from the analysis of RWD. FDA defines RWD as data about a patient's health status and/or the delivery of health care routinely collected from a variety of sources, including health care provider records, medical and pharmacy claims, and disease registries. RWD can also be collected outside the health care setting — for instance, data from mobile technologies that gather biometric information. Fit-for-purpose RWD may be used to generate RWE that FDA can consider when making regulatory decisions about the safety and effectiveness of medical products — such as identifying new safety issues with a drug after it is approved or helping to determine the effectiveness of a drug for a new indication or patient population. | 
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