The Food and Drug Administration (FDA) uses real world data (RWD) and real world evidence (RWE) to monitor postmarket safety and adverse events – and also to make regulatory decisions.
The 21st Century Cures Act, passed in 2016, places additional focus on the use of these types of data to support regulatory decision making, including approval of new indications for approved drugs. Congress defined RWE as data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials. FDA has expanded on this definition as discussed below.
Real-world data are the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. RWD can come from a number of sources, for example:
- Electronic health records (EHRs)
- Claims and billing activities
- Product and disease registries
- Patient-generated data including in home-use settings
- Data gathered from other sources that can inform on health status, such as mobile devices
The use of computers, mobile devices, wearables and other biosensors to gather and store huge amounts of health-related data has been rapidly accelerating. This data holds potential to allow researchers to better design and conduct clinical trials and studies in the health care setting to answer questions previously though infeasible. In addition, with the development of sophisticated, new analytical capabilities, scientists are better able to analyze these data and apply the results of our analyses to medical product development and approval.
Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective).
Example: Ibrance
Pfizer’s Ibrance (palbociclib) was approved in 2015. It is a kinase inhibitor, now approved in combination with an aromatase inhibitor as the first hormonal-based therapy in women who have gone through menopause and in men, or with fulvestrant in patients whose disease progressed following hormonal therapy.
Pfizer provided the results of an analysis of real world data (RWD) from electronic health records (EHRs) as additional supportive data to characterize the use of Ibrance in combination with endocrine therapy (aromatase inhibitor or fulvestrant) in male patients with breast cancer based on observed tumor responses in this rare subset of patients with breast cancer.