Good Morning ,
It's been a happy hunting ground - can the FDA expand the label to adults based on current evidence- if pediatric gets full approval?
In June 2017, FDA approved a new indication for a medical device without requiring any new clinical trials. This approval marked the onset of a new era in drug and medical device regulation: the systemic use of “Real World Evidence” (RWE).* FDA based its approval on records of the product’s actual patient use rather than on randomized clinical trials.
Specifically, FDA approved a new procedure for a transcatheter aortic valve replacement (TAVR). The manufacturer had obtained its initial marketing approval from FDA in 2011. Upon obtaining this approval, the device manufacturer had established a product registry which resulted in a database containing records of over 100,000 TAVR records. Among these records were 600 records relating to the then off-label use of the new procedure. Based on the product registry data, FDA approved use of the new procedure without requiring traditional, randomized clinical trials (RCTs), saving both time and money.1,2
FDA’s acceptance and use of RWE dramatically expedited the agency’s ability to review and approve the TAVR new procedure. The United States was the 42ndnation to originally approve the device in 2011. However, by using RWE, the U.S. became the first country to approve the new procedure.3
The CIMBTR data is independent- Prof Kurtzberg highlighted this point saying it was not the sponsors data in a video some time back- link anyone? Silviu is now talking about 5 year survival data - the 4 year data was announced end of 2022.
CIMBTR will have all the EAP adult data.
First link below is a paper on why the FDA should consider RWE but has multiple examples where they have.
@Phaedrus you will love the links.
@Wilba32 take the day off - got your back.
https://www.bio.org/sites/default/files/legacy/bioorg/docs/BIO%20White%20paper%20%20-%20Incorporating%20RWE%20Within%20the%20Label_FINAL%202019.pdfhttps://www.fdli.org/2018/08/update-fdas-historical-use-of-real-world-evidence/
(20min delay)