@Street Novelist has a point; from the FDA website regarding FX-322's current Fast Track designation:
A drug that receives
Fast Track designation is eligible for some or all of the following:
- More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval
- More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
- Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
- Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA
Rolling Review may be part of the incentive for the 90-day data release.
On Compassionate Use:
I know this is going to be an unpopular opinion; but I struggle to see from a business standpoint the value of putting any resources into enabling Compassionate Use in 2021/2022 if the drug may be in its pivotal phase. Why go through the work to start limited production with a drug manufacturer, price an experimental drug, deal with the individual paperwork, distribution, and deal with doctors. The price may be astronomical in low volumes for select use. Not to mention QUICKLY educating a workforce of ENTs on the drug and how it works, how to determine dosage, etc. Seems like a lot of resource and logistical problems.
Sorry I should have been clearer. I meant in regards to evaluating and assessing FX-322 after the completion of trials for market release.
Regarding compassionate use access:
I actually agree with you that from a trial, medicine and also a business standpoint that Frequency Therapeutics might not authorise compassionate use then because it might make little sense commercially to do it. It will therefore make much more sense for Frequency Therapeutics to take things slowly and get a completeness about FX-322.
My alternate position is however that it would make sense for Frequency Therapeutics to look at granting compassionate access for the following reasons.
> Granting compassionate (expanded) access means that Frequency Therapeutics can start making money off of FX-322 now through sales and also inflated price sales. Something says to me that there totally would be people that would pay 5 times the price to get it early.
> Frequency Therapeutics would be interested in training professionals how to use FX-322 now, as this would save further delays to the release of FX-322. This is because Frequency Therapeutics could actually commence training interested practices now on how to administer FX-322.
> Frequency Therapeutics can use the patients being treated through expanded access as actual examples to ENT clinics on the benefits of treating people with FX-322. This means that Frequency Therapeutics can demonstrate to practices the benefit of participation as an FX-322 treatment provider.
> Frequency Therapeutics has already got some providers participating in the trials who are versed in dosing FX-322 who would wholly be able to participate in offering expanded access to people.
Thus patients will not be the only ones who will benefit from the provision of FX-322 early through compassionate use. This is because Frequency Therapeutics can utilise this expanded access time to get doctors interested and also understanding how to dose people now before they have to do it in a much bigger and much more complex manner when FX-322 is hopefully released at full scale.
At this stage I think that it is possible that this could go either way. What we have heard and seen from Frequency Therapeutics is that they tend to be preparing and planning hopefully for FX-322 to get released, with the things they have done like preparing pricing and looking at how the distribution and treatment chain will work.
However consequently they could be getting on top of this stuff early so they can get it right for 2 years time when they theoretically could be finished with the pivotal phase by. I predict that we will know more in March 2021 maybe. My hunch is that depending on how the results are not only will we see Frequency Therapeutics look to submit their rolling review right away but also indicate other steps at that point in time too. Nevertheless I think that we will see Frequency Therapeutics attempt to do everything they can to get FX-322 released.
In all of the tens of thousands of years of human existence, we've never been able to restore our own hearing naturally. Generations of people have come and gone with hearing loss and tinnitus. I'm willing to wait 2-3 more years if that means doing it right.
I do definitely agree. If Frequency Therapeutics turns around and demonstrates that there will be benefit and further gains to patients by waiting till the end of phase 3 then I am in support of this. Better outcomes will probably be obtained by doing it right and I hope that we will widely see massive benefits come March/April when the 90 day release eventuates.