self trial of Gileyna

[nebrhahe53]

Sequoya started a thread on here several years ago about self trials of Gileyna. Does anyone know what happened with that? I know it costs .50k/yr off label, so a source at some reasonable cost would have to be found.

 

[lgelb]

Neil/all,
It is true that Gilenya is probably prior authed on most plans because of the first dose monitoring requirement and per-dose cost. However, your doc can accompany the prior auth form with a letter citing FDA approved investigational use, the otherwise dire prognosis, your cardiovascular/ophthalmologic status, etc. It is not unknown for prior auth criteria to be modified with appropriate evidence. I would at least try before giving up, if you really want to do this. In fact, if Max's neuro is willing, the fact that he, i.e. the VA, is rx'ing for this indication can be cited as well.

If the prior auth is denied, you can try Novartis' own patient assistance program. That too requires a neuro to fill out a form (which you can download any time). The worst they can say is no. Gilenya is not currently recruiting for an official ALS trial, ALS doesn't feature a long lifespan, and there is precedent for manufacturers to provide some support at the Phase II milestone.

As Max mentioned, there are clear contraindications for this drug and they are important -- if you value your vision and ability to stay off the floor. It is not like some of the other potential "self-trials." _Don't_ try this at home without clinical support.

 

[Nikki J]

I do not know but suspect cost was a factor. Sequoia ( Sharon ) passed away some time back. So did Ed from ALSTUN. So did Persevering ( Rob) in the ALSTDI thread Sharon linked to. There is an inactive page for als treat us now on face book. If you want to search it you might find more info

 

[nebrhahe53]

Well I guess that says it all-everyone who tried has passed on-they were not cared enough about to be listened to-interesting when you see how the fda fell all over itself making experimental treatments for Ebola available.
I know Gileyna has some nasty side effects, but so does ALS, and if people are willing to try they should have the opportunity.
Either that or how about starting clinical trials with more than 10-20 people in them!

 

[MaxEidswick]

>It is true that Gilenya is probably prior authed on most plans because of the first dose monitoring requirement and per-dose cost. However, your doc can accompany the prior auth form with a letter citing FDA approved investigational use, the otherwise dire prognosis, your cardiovascular/ophthalmologic status, etc. It is not unknown for prior auth criteria to be modified with appropriate evidence. I would at least try before giving up, if you really want to do this. In fact, if Max's neuro is willing, the fact that he, i.e. the VA, is rx'ing for this indication can be cited as well ... It is not like some of the other potential "self-trials." _Don't_ try this at home without clinical support.

Ditto that!

 

[nebrhahe53]

yeah I know you can go blind with this, or get a heart attack. I would vastly prefer the heart attack to slow painful death, and I dont think dead people can see too well either.

 

[lgelb]

Neil,
While I appreciate black humor as much as the next widow, my husband had a very hard time dealing with the inability to move his head coupled with pre-existing blindness in only one eye. Trust me, it's not a combo you want. Either way, Gilenya is not a cure for either MS or ALS, so you won't end up blind and otherwise healthy. Nor are the major cardiovascular risks a nice quick death from MI but rather more arcane and long-term manifestations.

I am not saying not to try -- I outlined how to start earlier -- but please don't bury your head in the sand about the risks of any therapy, rather minimize them as best you can. In the case of Gilenya, that means first dose observation or support and following the contraindications the label provides, both in terms of pre-existing conditions and concomitant drug therapy.

--Laurie

 

[rknt50a]

Pharma company can't subsidize off-label use with its own financial assistance program.

That would have to go through a formal Expanded Access Program.

 

[lgelb]

They can, actually, though they can also opt not to use PAPs for that purpose and go other ways. However, there's no harm in trying door #1 first. It's their product and not illegal to write for ALS. Think of the oncology or AED landscapes, for example, in terms of unrestricted grants and investigator-initiated sponsored trials.

It would be a roll of the dice for them to do an EAP to the extent that could impede further trial recruitment, presuming they intend to pursue the indication. It's possible, with the right incents, but I wouldn't hang around waiting only for that.