Monday, March 19, 2012

Getting a script for Kalydeco off-label

I just called Vertex to speak with them about doctor's prescribing Kalydeco off-label.
Here is what I learned~

Any doctor, any where can write a script RIGHT NOW for Kalydeco for any patient with CF regardless of mutation.  They (vertex) do not require proof of mutation...that is only important if you have G551D and vertex can help get financial assistance for the drug.  The form doc s sign is to help G551d get insurance coverage, that is all!!!!

Vertex is bound by legal obligation to only help (financially) patients who are G551D.  There is actually very little they can say to a CF patient who is not G551D because of FDA REGULATIONS because the approval of the drug only covered G551D. They cannot tell you if it might be beneficial (even if it works great in vitro on your mutation) because it is against the LAW and they will get in trouble.

However, I was informed that if your doc thinks it is an important drug to try and might be helpful, they are absoltuely allowed to prescribe it off-label.  At that point, your pharmacy will do what it can to get the drug covered by insurance.
If your clinic has a problem with that, have them call this number at vertex:

877-752-5933 Option 2


  1. Just so I'm sure...if I get a prescription from my doctor CF Services will try and get the drug approved by insurance? That is where I would get it I assume, because my specialty insurance pharmacy doesn't have it.

    The reason I ask is because CF Services is making my doctor deal with getting TOBI approved by my insurance. I just want my doctor to write the prescription for Kalydeco and nothing else.any other work and he might say no.

  2. They did that for me. I was shocked.

    I have no idea about Tobi because I can't take that drug. All I can tell you is how I got Kalydeco and my doctor did nothing but write the script.

  3. Hello, I'm working on a blog entry about the genetic reason why Kalydeco might work in someone like you, would you mind if I reference your blog in my entry? My goal is to enlighten more people to the possiblity of Kalydeco having benefit for them because of a process called "transcomplementation"--that is, interaction BETWEEN two different CF mutations. It is very exciting research and I predict the pool of eligible Kalydeco candidates to keep growing and growing! Thanks,

  4. Hi- I have your exact mutations! I love the internet. I'll be especially interested to hear about your sinuses and gut, so please keep posting. I had a lung transplant 13 years ago at age 23, so the lung stuff won't really affect me, but dang, this is so awesome to hear. Congratulations and thanks for posting!


  5. Thanks for this information. My doctor refuses to write a prescription for me to try even though my insurance will cover it.

  6. I have the same mutations also, and have been contemplating obtaining Kalydeco for two months and see what happens. I am sure my insurance will not cover it, so I will need to figure out how to pay for it. Can you e-mail me directly?