FDA Expanded Access

[markt]

For those excluded from participation in clinical trails due to not meeting one of several diagnostic criteria (a significant portion of the diagnosed Sjogrens populous), this FDA program may be of interest and present an avenue to receiving investigational medications (those of which for Sjogrens are post Phase-I and have an established safety profile).  I am in process of exploring this with my provider at the moment (FDA approves the majority of requests; the worse you can hear is no from the developer/manufacturer). 

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm432757.htm

That said, am I the only one getting the notion that you have to essentially be a "unicorn patient" to participate in a trial; i.e. meet very restrictive criteria thereby alienating a significant portion of those actually diagnosed.  How they get enough people to move forward with a trial is beyond me.  The last time I inquired about a trial, you had to have things as specific as parotid gland involvement, something about impacts to skin/dermis health, be positive for antibodies at screening (okay, I can see that ... but this is an on and off things for a lot of us anyways), etc.  Long story short, there is an entire chapter of Sjogrens patients in my city where the study is being done, unfortunately I can count on one hand the number that actually are participating in it.   Rant off... 

[cccourt1942]

Where do you live?

[Sharon]

Thanks for posting this markt.
Yes, most of us do not meet the criteria required for clinical trial participation.
I just wish there would be at least ONE trial involving seronegative SS patients.

[markt]

Jacksonville, FL.  There is a Phase-II trial for a Novartis biologic being held locally.  Talking with the coordinator, they are having recurring meetings with the principal developer due to the poor enrollment and participation. 

[markt]

The President signed the Right to Try Act (FDA Expanded Access) into Federal Law on Wednesday.

It will be interesting to see how this vehicle matures, as well as how industry, providers, and insurers all respond to the various patient communities submitting claims.

https://www.youtube.com/watch?v=mIaJnnPo-Xs

https://www.youtube.com/watch?v=JJmjjB5Q4wI