Ann: FDAs OTAT Agrees to Primary Endpoint for Back Pain, page-168

The issue with the FDA is not whether the treatments are potential therapeutics, but whether they actually have any efficacy for the claimed indications. And if they do have efficacy, how much of it do they have?

So the FDA would like to see the accepted normal randomised controlled trials to be completed and the primary end point to be achieved in order for it to be approved for a particular condition. To help the process to go smoothly they want to discuss a protocol of the trial before it starts to make sure it meets the appropriate standards for approval. So MSB has talked with them about what they want to use for the trial endpoints and the FDA have agreed to them. That is all.

The FDA also wants MSB to complete a randomised controlled clinical trial to show that Ryonicil has efficacy for aGVHD. The first step is to agree with the FDA on a method of assessing the potency of a dose of the therapy so there can be consistency in the amount of drug given to each trial subject which is particularly important if you are intending to treat children because their size varies a lot. Then they could be dosed according to body weight or body surface area or some other parameter to ensure they are getting some sort of safe and scientifically equivalent dose.

So far the same drug underwent two adult clinical trials by the original developer which failed to show efficacy. SI now says it was a potency issue and he has changed the manufacturing process to improve it so that it works. The FDA wants a new RCT to scientifically show that it now does work when before it didn't.