Was Efficacy Bridged between MedTone C and Dreamboat
Mar 31, 2014 13:43:56 GMT -5
ezrasfund, goyocafe, and 2 more like this
Post by silentbob on Mar 31, 2014 13:43:56 GMT -5
I've seen the statement made that Mannkind only bridged safety and not efficacy, and therefore the efficacy evidence may be too light for approval.
I will quote an example by poster Dereklinders on the yahoo forums, who is a smart and balanced poster:
------------------ Dereklinders' post -------------------------
Well, I'm not going to be so silly as to say "Believe it!", but it is clear to me that efficacy has not been bridged between inhalers. Safety has.
The FDA doc specifically states that arm 3 of 171 is not discussed since it was not meant to bridge efficacy. That orphans all Medtone efficacy data.
That means for efficacy, there are about 800 participants across all 171 and 175 arms, for 6 months in total duration - 90 days if you count the experimental phase, and with an abbreviated titration period in 171 to boot, I was surprised to find out about that 1-month titration instead of the typical 3-month, I think there's risk of a requirement for more data.. How big a risk? No idea. Tuesday will tell.
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The statement contains some truth when reducing the context of the FDA remarks.
For example here is an FDA statement:
"Trial 171 was designed to address the deficiency listed in the Cycle 2 Complete Response letter that one of the phase 3 studies with the Gen2 inhaler should
include a MedTone arm so that pulmonary safety of the two inhalers could be directly compared. The trial was not designed to directly compare the efficacy of Afrezza TI using the two devices. This approach was agreed upon at the Cycle 2 End-of-Review meeting held 4 May 2011. "
If you read such a statement in isolation you might accept the statement.
While I respect Dereklinders and his posts, and you should too, in this case it is quite easy to discredit the theory.
To quote the FDA division director, on page 4:
"Gen2 inhaler: Used in the two new pivotal phase 3 studies. This is the to-be-marketed device. In-vitro performance (particle size) and single dose comparative bioavailability (study: MKC - TI 42) suggest device design change would not affect efficacy. In light of these findings, data derived from Phase 3 trials performed with the MedTone C inhaler device although not pivotal may be thought of as providing supportive evidence to inform efficacy and safety of the product."
So all the older efficacy data can certainly be considered supportive evidence.
I will quote an example by poster Dereklinders on the yahoo forums, who is a smart and balanced poster:
------------------ Dereklinders' post -------------------------
Well, I'm not going to be so silly as to say "Believe it!", but it is clear to me that efficacy has not been bridged between inhalers. Safety has.
The FDA doc specifically states that arm 3 of 171 is not discussed since it was not meant to bridge efficacy. That orphans all Medtone efficacy data.
That means for efficacy, there are about 800 participants across all 171 and 175 arms, for 6 months in total duration - 90 days if you count the experimental phase, and with an abbreviated titration period in 171 to boot, I was surprised to find out about that 1-month titration instead of the typical 3-month, I think there's risk of a requirement for more data.. How big a risk? No idea. Tuesday will tell.
--------------------------------------------------------------
The statement contains some truth when reducing the context of the FDA remarks.
For example here is an FDA statement:
"Trial 171 was designed to address the deficiency listed in the Cycle 2 Complete Response letter that one of the phase 3 studies with the Gen2 inhaler should
include a MedTone arm so that pulmonary safety of the two inhalers could be directly compared. The trial was not designed to directly compare the efficacy of Afrezza TI using the two devices. This approach was agreed upon at the Cycle 2 End-of-Review meeting held 4 May 2011. "
If you read such a statement in isolation you might accept the statement.
While I respect Dereklinders and his posts, and you should too, in this case it is quite easy to discredit the theory.
To quote the FDA division director, on page 4:
"Gen2 inhaler: Used in the two new pivotal phase 3 studies. This is the to-be-marketed device. In-vitro performance (particle size) and single dose comparative bioavailability (study: MKC - TI 42) suggest device design change would not affect efficacy. In light of these findings, data derived from Phase 3 trials performed with the MedTone C inhaler device although not pivotal may be thought of as providing supportive evidence to inform efficacy and safety of the product."
So all the older efficacy data can certainly be considered supportive evidence.
