Real World Data (RWD) are data that are collected outside of the constraints of a conventional clinical trial. RWD is routinely used for regulatory decisions. One example of RWD routinely used is in pharmacovigilance (PV) and safety signal detection, where RWD consists of tens of thousands of reports that may translate into a few dozen validated signals. It takes more than detective work to sift through the noise of RWD. In contrast to a traditional clinical trial, where there is control over what data is measured, the methodology used and the standards by which it will be reported, RWD relies on the locally established standards of care and the local healthcare system. Missing elements or differing standards require interpretation and more than detective work.
In the clinical development of an investigational drug, one of the most challenging aspects is to define endpoints that are relevant for the disease, investigational drug, and target patient population. This is one of the most important steps in protocol development. After weeks or months of struggle - scouring the literature, reviewing available regulatory guidance, and speaking with medical advisors – teams eventually finalize the endpoints and data elements that will support the scientific questions. As hard as this may be, we often find ourselves with trials where the data doesn’t translate into evidence of efficacy. Peer review journals will often publish well designed clinical trials with differing results for a given therapy. These are some reasons why Regulators question RWD data. RWD offers valuable data used to inform, but does not necessarily translate into Real World Evidence (RWE) for regulatory purposes. However, this is an area of innovation and Regulators are increasingly open to discussing transparent and justifiable real world data.
Early Access Care assists pharmaceutical companies in the optimization of RWD solutions in Expanded Access programs. To speak with one of our team members about RWD, contact us at email@example.com.