thought for ddf508 with upcoming quarterly appt.

[Aboveallislove]

Approval for Orcambi should be by July 5 for those 12 and older. For those who have quarterly appt. before then, had a thought: Wonder if it would make sense to ask the doctor if you can call in for them to start the script/approval process once approved and if there is anything that you need to discuss before then? That way you could get the process rolling right away without waiting until October or September and then a delay with the approval etc. And have in hand, mouth, stomach and blood stream before cold and flu season.
Love to hear what/how your clinics handle if anyone does.

 

[Ratatosk]

Wise advice to get organized and get all your "ducks in a row", so to speak.... Not to be a big downer; however, there may be issues with insurance and it taking months for them to get on board in terms of coverage, adding it to their approved drug lists, availability..... When DS' enzymes were reformulated Pancrease MT to Pancreaze -- the approval happened in the spring and when I went to get a refill before school started in August, I was told by the pharmacy that our insurance wouldn't cover it and I'd have to come up with $2300 out of pocket for a 3 months supply. When I called the insurance company, they referred to their list of drugs and it was non-covered -- only creon was allowable, so I had to switch him. Fortunately, he was able to tolerate creon. To this day our insurance still only covers creon and more recently zenpep, none of the others.

Those who got Kalydeco may be able to offer some insight or suggestions as well. I know a friend of mine goes thru a pre-approval process and has to deal with a mail-order/online pharmacy, which also helps with the copays. Not sure with Orcambi will be able to be sold at local pharmacies or if there will be a certain pharmacy allowed to distribute it.

We have appointments in June, so I fully intend to bring it up. So I'll keep you posted.

 

[Aboveallislove]

Hey,
Totally agree re the insurance delay, which is why I was actually thinking to get script/prior approval started ASAP so that 3 months later it will be resolved. I know with comparable drugs like different enzymes insurance can have a list and deny others, but they can't do that when there isn't a comparison, like with Orcambi. Now, some might try to argue with stable lung function it isn't medically necessary and that might end up delaying, but hopefully not. Vertex has said they have a staff trained to make the process go smoothly and help with all the logistics, so I guess after getting a script that would be my next call too.
I would love to hear from the Kalydeco on label folks about the process they had, but there could be the hang up of Orcambi not have the same extreme benefit. And please do share what you find out from clinic!!

 

[ToriMom]

Hello,
Does anyone know if this new medication is only for double deltas and excludes all heterozygous deltas? My daughter has one DF508 and one other mutation--but in the same class. I will ask at the next clinic.
Thanks

 

[Aboveallislove]

Hey Mom,
I'm swamped at work, so this is quick, but I have lots of thoughts to put together for you later. Could you share the other specific mutation in the meantime, so I can see what info I can find on that and also look to see whether Vertex has in vitro studies of how it responds as well as how it compares to df508. The FDA has not decided on the label yet, so it is unknown whether the label will say "it is not effective for those heterozygous for df508." I think they won't given that the FDA seemed to be backpeddling on that aspect of the label for Kalydeco (Kalydeco said not effective for homozygous df508 and unknown for other mutations and then with the Orcambi results said maybe we just needed a bigger trial and Kalydeco alone works. My "gut" is the FDA will be more cautious and say something like "it is unknown whether Kalydeco is effective for those heterozygous with df508.") Scripting and insurance are different. I'll add more thoughts when I can--there's some info I want to pull/link/check to explain my thoughts.

 

[ToriMom]

Thank you so so much! You have already helped a lot. Her second mutation is R1066C. I looked up if Kalydeco did anything to help this mutation when used alone, and unfortunately it doesn't help. In fact, it looks like it had no effect at all. I thought DF508 and the R1066C were more similar because they both cause misfolding and gating problems. But apparently one is a "substitution" defect and the other is a "deletion" defect. In genetics I guess that's a BIG difference?!
Thanks for anything you can share with me. You can also PM me if you prefer.
Hoping and praying,
Michelle

 

[Aboveallislove]

Still buried so can't dig in like I need to, but if ok I will post here in case others have similar questions. Also, there is a study coming up for those in her situation. I will post link as soon as it is posted o. Clinicaltrials.gov...I'll shoot some prayers up too!

 

[Tom]

My son's prescription for Kalydeco was filled and delivered approximate 5 weeks from the FDA approval date. The case managers at Vertex handle all of the insurance issues (if there are any). We did not have any problems with our insurance and it has changed 4 times since 2012. My son is G551D and DF508. I wonder if Orkambi will have even more benefits that Kalydeco alone. Has anyone come across this type of info?

 

[Aboveallislove]

Tom orcambu should Improve your sons health even more theoretically because vertex has evidence that 661 and kalydeco add about 10% more fev...can't pull study now so that might be off...for those with 551 and df508 and 809 works the same as kalydeco. They are doing a study now for those with your sons mutations for 661 and kalydeco which will replace orca bi in a couple years. He might try getting in that. You might discuss with your doctor having hm do orca bi in the meantime....it might be priced cheaper than kalydeco so I wouldn't think the insurance would bulk and if it did he has kalydeco on label so it doesn't matter...you aren't risking a red flag as those on kalydeco off label. I know 809 has a reaction wi kalydeco so the orcambi dosing is increased over the kalydeco alone so the doctor might not want to do it, but something to discuss.

 

[Aboveallislove]

TorisMom,
Check out slide 79: https://www.nacfconference.org/art/plenaryarchives/2014_Boyle.pdf

That was what I was looking for--to see "how" the combo improved the transport to the cell surface. It says:"Correctors such as Lumacaftor and VX-661 correct folding of CFTR by stabilizing the interaction between NBD1, TMD1, and TMD2."

I'm going to dig in further when I have a few minutes here and there to see if there's anything on your daughter's other mutation that talks about a problem with that interaction. There are several problems with df508 and likely with your daughter's second mutation, but if one of the multiple problems is the stability of that interaction then it would seem to me, Orcambi should have a similar benefit. (I think that stabilizing that interaction has a "capped" benefit, meaning you can only improve CFTR function so much by improving that interaction...so maybe it is the same with the other mutation). Hopefully her doctor can tell you if there is a stability issue for her mutation in that interaction."


That would be part 1 to trying to get her the drug: Getting the doctor to script it. Then the insurance issue.
The trial might be a quicker way to access it (and if she does well that would be added evidence to get it off-label).
More when I can.

 

[Aboveallislove]

Torismom:So I started to research to see how the R1066C mutation works (or rather doesn't). This article does a great job of going into the various issues but is wayyyyyy beyond me: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3434365/
Basically, I think you need a science-type to be able to help figure out if the R1066C mutation has as "an" issue (not necessarily the only issue, since df508 has multiple issues) the thing that Orcambi fixes for df508, which as noted above is to stabilize the interaction between NBD1, TMD1, and TMD2. If so, then there it should work. The article I posted is about 3 years old and there may be better and more research on R1066C and specifically in vitro on Orcambi for it. (If trying to get off label, your doctor can request from Vertex if it has any evidence of its effectiveness on R1066C.) Once it is approved, shoot me a PM and I can help strategerize an approach to trying off label if you can't get your daughter in the trial.
In the meantime, if you have any friends who have a science background and are saying "is thee anything I can do to help," you could try to put them on a literature review to answer the above question.
Good luck mom!
Hugs and Prayers,
Love

 

[ToriMom]

AboveAll,
Thanks so much for taking the time to look up information for me. Thanks for your compassion and your prayers. Her name is Victoria (or Tori) if you want to toss her name into a prayer circle. I am not too knowledgable on the deep science of it all, but I also know that sometimes medicine works with no scientific explanation that makes sense.
Thanks again,
Michelle