In a recent article published in Clinical Cancer Research, investigators analyzed successful submissions of oncology drugs to the Food and Drug Administration (FDA) that utilized real world evidence (RWE). The researchers identified elements criticized by the FDA, including confounding factors, and distinguished between full and accelerated approval.
“All full (vs. accelerated) approvals shared the following characteristics: high magnitude of efficacy in the pivotal trial, designations of orphan disease, breakthrough therapy, and priority review, and no advisory committee meeting held. This study found that findings from external control real-world studies complemented efficacy data from single-arm trials in successful oncology product approvals.” Read more here.
(Source: Arondekar et al., Clinical Cancer Research, 11/3/21)