Replies to post #42627 on Relief Therapeutics Holding AG (RLFTF)

[changes_iv]

I tell you from my side why the acceptance for filing is in my eyes so relevant.

I was doubting after all the stories of Dr. J and Dr. Ram that RELIEF is ready to perform a filing act on their own ! The process of EUA/NDA of Zyesami has been so much driven by JJ - I was somewhat getting this impression. (Sorry RLF, I still love you).

Now today seeing RLF to perform a successful FDA filing is very relevant since you see that this company is able to perform this very demanding procedure.

An accepted file for NDA needs many details, starts with preclinical data, toxicology in mice, GMP production details, goes to package insert writing (very relevant) to human phase III data presentation, labeling, supply chain and a postmarketing plan etc.

Now I see that RLF is very capable, even better FDA proven to be capable, to perform such an act.

This is very reassuring and shows to me that RLF can act on its own as independent pharma company !

They have everything in place to advance to Big Pharma!

~ IG, Y@h00 RLFTF finance conversations

[changes_iv]

To better understand the use of external controls to support product development and approval, we reviewed FDA regulatory approval decisions between 2000 and 2019 for drug and biologic products to identify pivotal studies that leveraged external controls, with a focus on select therapeutic areas. Forty-five approvals were identified where FDA accepted external control data in their benefit/risk assessment; they did so for many reasons including the rare nature of the disease, ethical concerns regarding use of a placebo or no-treatment arm, the seriousness of the condition, and the high unmet medical need. Retrospective natural history data, including retrospective reviews of patient records, was the most common source of external control (44%). Other types of external control were baseline control (33%); published data (11%); and data from a previous clinical study (11%). To gain further insights, a comprehensive evaluation of selected approvals utilizing different types of external control is provided to highlight the variety of approaches used by sponsors and the challenges encountered in supporting product development and FDA decision making; particularly, the value and use of retrospective natural history in the development of products for rare diseases.

https://link.springer.com/article/10.1007/s43441-021-00302-y