ABSTRACT
The 21st-century Cures Act passed by the United States Congress in December 2016 requires the FDA shall establish a program to evaluate the potential use of real-world evidence (RWE), which is derived from real-world data (RWD) to (i) support the approval of new indication for a drug approved under section 505 (c) and (ii) satisfy post-approval study requirements. RWE offers the opportunities to develop robust evidence using high-quality data and sophisticated methods for producing causal-effect estimates regardless randomization is feasible. In this chapter, we have demonstrated that the assessment of treatment effect (RWE) based on RWD could be biased due to potential selection and information biases of RWD. Although fit-for-purpose RWE may meet regulatory standards under certain assumptions, it is not the same as substantial evidence (SE) (current regulatory standard). In practice, it is then suggested that when there are gaps between fit-for-purpose RWE and SE, we should make efforts to fill the gaps for an accurate and reliable assessment of the treatment effect.
