RWE Biologics License FDA BLA suplements

Real-World Evidence in Support of Oncology Product Registration: A Systematic Review of New Drug Application and Biologics License Application

by Bhakti Arondekar, Mei Sheng Duh, Rachel H. Bhak, Maral DerSarkissian, Lynn Huynh, Kelsey Wang, John Wojciehowski, Melody Wu, Bryon Wornson, Alexander Niyazov, George D. Demetri

Jan 1^st 2022
12 mins
Clinical Cancer Research (2022) 28 (1): 27–35

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Summary

The FDA emphasizes early engagement and transparent protocols in Real-World Evidence (RWE) studies.
Aligning RWE populations with pivotal trial populations is crucial, often achieved through criteria matching and propensity score methodologies.
Major critiques include differences in tumor assessment criteria, the need for concordance analysis, and addressing confounding factors.
FDA critiques are inversely correlated with pivotal trial efficacy; high Objective Response Rate (ORR) and long Duration of Response (DOR) lead to fewer critiques.
Full approvals are associated with high ORR and longer DOR, while accelerated approvals often have mixed ORR and shorter DOR.
Selection of appropriate Real-World Data (RWD), adequate sample sizes, and sound study designs are stressed by the FDA.
Chart reviews are common but limited by selection bias and confounding, and handling missing data thoroughly is recommended.

Regulatory real-world evidence (RWE) has garnered great interest since the passing of the 21st Century Cures Act in 2016, which aimed to accelerate medical product development by supporting approval of new indications for current therapies or satisfying post-approval requirements (1–3).

RWE is especially important for new therapies that target rare, serious illnesses with high unmet needs, such as orphan drugs where single-arm trials are often conducted due to lack of standard of care. Additionally, RWE can play a role in contextualizing or serving as external controls for comparison with an investigational therapy to support accelerated approval, as confirmatory trials for regular approval, or to support a new indication. Using historical control data to support clinical trials is of interest in oncology research, especially given the possibility of decreasing time to approval and increasing availability of therapies...

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