Thursday, February 9, 2012

Hoop Jumping 101 for Kalydeco Off-Label

They say jump…you say how high?  As far as we are concerned, we received a SECOND miracle yesterday when our insurance company called us with the approval of our off-label claim for Kalydeco for Brady, who is 4 ½.  I know there are parents wondering what we did to get it approved, and so quickly, so I will share every detail! 

First of all, I have been discussing VX-770 with Brady’s CF specialist since he was an infant.  I frequently took newly published info to give to Brady’s Doctor on clinic days to ensure he was in the loop.  I absolutely love Brady’s CF care team, and honestly have to give them the majority of the credit for the approval.  I think the thing that I love most is their responsiveness.  I emailed Brady’s Dr. within a few hours of the FDA approval…and he emailed me right back.  I’ve been on the phone with Brady’s nurse every single day since the approval.  They always call me back right away if they can’t take my call.  The point of this is that you need a good Dr. on your side willing to fight with you.  Brady is lucky enough to have an excellent physician and nurse. 

His prescription was faxed into CF Services Pharmacy on Thursday Feb. 2nd.  We also faxed in our enrollment forms for the VertexGPS program.  We called on Friday to make sure it had been received.  At that point, CF Services said that they had Kalydeco in stock, but were waiting on insurance companies to get the drug in their database before they could start billing.  We called our insurance company (Regence Blue Shield of Idaho) and started bugging them about updating their database.  When we called back Monday morning, Regence had just added Kalydeco.  We called CF Services back and asked them to get moving with our insurance.  Monday afternoon, we heard back from Regence that our claim had been automatically flagged because of the price (any drug over $10K/month gets an automatic flag and a “request for more documentation of medical necessity”), and also because of Brady’s age.  We were expecting this.  We called Brady’s clinic back and asked his Dr. to write a “letter of medical necessity.”  They faxed this letter, along with something called a “pre-authorization form,” and lots of Brady’s medical notes into both the Insurance company and CF Services Pharmacy.  The woman we were working with at CF Services was super helpful to us.  The pharmacy didn’t require all this stuff we were faxing them, but it is helpful for them to have in hand when/if the insurance comes back to them with questions or tries to deny.    

Tips for jumping through flaming hoops:

1)      Call all involved parties every single business day.  The squeaky wheel gets the grease.  When we would call our insurance, our claim would get pulled out of a huge pile of claims and actually get some attention.  It would get marked urgent and sent to a manager. 

2)      Write it all down!  We kept a log of every phone call we made concerning Kalydeco.  Time/Date/Who you were speaking with/their extension/will they be working tomorrow?  Get all the information you can to get back to the same person the next time you call.  Keep fax numbers and phone numbers handy in your log.  That way, if you get your Dr.’s office on the phone, you can easily tell them, “I’ve been working with Roger at Regence.  His phone number is…ext…fax…”  You will care more about this than anyone working with you, so you have to be the one responsible to keep all parties in the loop.

3)      Confirm receipt of everything.  Confirm the pharmacy has received the script.  Confirm the insurance has been billed.  Confirm with insurance that the claim has been received.  Confirm receipt of any faxes, etc…  We had to fax Brady’s “pre-authorization form” in 3 times before it was actually received and put in his file.  Don’t assume that just because it was sent, that they have it.  Always call to make sure! 

4)      Provide as much detail as you can if they request more information.  Brady’s Dr. included a lot of his medical chart notes.  Even with as wonderful as our clinic team is…they get busy.  I would always ask the nurse to please email me and let me know when she sent information into insurance or the pharmacy.  I also requested copies. 

5)      If you are attempting an off-label prescription, don’t worry too much about Vertex.  You will be enrolled in Vertex GPS, but Vertex can’t offer you financial support or much guidance for off-label use.  They don’t really have anything to do with the distribution.  In fact, today our Vertex GPS coordinator called us to tell us we didn’t qualify for financial support because of Brady’s age.  I responded, “Thanks for the call.  We don’t need financial assistance.  Our insurance approved it and we are getting it delivered today.”  The Vertex rep was SHOCKED and happy for us.  Don’t even waste your time worrying about Vertex.   You don’t need any kind of approval to get your Kalydeco.  The main issue is getting insurance coverage.  If you get coverage of your claim…you’re golden.  Also, Kalydeco does NOT have to be obtained only from their "authorized distributors."  If you have insurance coverage through specific pharmacies only...turn it in to them and let them call their wholesalers for details.  They will likely be able to get it. 

We have always assumed that our insurance would deny our off-label claim for Kalydeco, which is why we went about getting the Rx so meticulously.  We figured that we would probably need all these details to give to our attorney for the appeal process.  Even though we didn’t end up needing the lawyer, staying organized about the process was extremely helpful.   By Wednesday morning, the insurance company had all the extra information they had requested.  Later that day, they called us with the approval.  They had already called the pharmacy and our Dr. to inform them of the approval also.  I can’t tell you how shocked I was when Brock came running down the stairs yelling “APPROVED!!”  I mean, totally, utterly shocked.  Everyone, including our attorney, had been telling us that this claim had a snowball’s chance in hell of getting approved.  I can only assume that the letter of medical necessity was a big factor in their decision.  With that being said, I will share what Brady’s Dr. wrote in that letter:
“To Whom It May Concern:

 Brady is an almost 5 year old little boy with Cystic Fibrosis [genotype G551D and deltaF508].  I have recommended and prescribed Kalydeco for him, to be started as soon as available.

 Based on my review of the supportive scientific data, Brady is an ideal candidate for starting the drug.  His genotype predicts that it will be efficacious in preventing or slowing development of lung damage in him.  Kalydeco is the first drug to treat the root cause of CF by correcting underlying CFTR dysfunction.  Every other drug he takes treats only the symptoms. Every day that he spends with dysfunctional CFTR puts him at risk of significant lung deterioration due to CFTR dysfunction, which results in abnormal airway secretions, infection, inflammation, and irreversible damage.  With permanent lung damage being the primary ultimate cause of death in cystic fibrosis, PREVENTION of such damage is our #1 goal.  Brady so far has mild lung involvement, making early institution of the drug all the more important; it is proven that damage can be prevented by this new drug, but unlikely that damage can be reversed by this or any other drug.  Additional benefits of Kalydeco which may accrue include prevention of liver damage and possibly other organ dysfunction, though these benefits are not yet proven.

Brady is slightly younger than study subjects, but close enough in age to allow clinical confidence that a dose of 150mg twice daily will be safe and effective.  Delaying institution of the therapy until he meets current age criteria (about 1.5 years) or until studies with younger subjects are completed and published (unknown timeline) will jeopardize his lung health and ultimate prognosis, as well as increasing ultimate costs of care.

I look forward to a rapid positive determination in this case, and would be glad to provide more information or to discuss Brady’s case by phone if it would be helpful.

Thank you very much.”

Immediately following insurance approval, CF services called us to discuss shipment and co-pay.  Our insurance lists Kalydeco as a brand name non-formulary drug, which carries a 50% co-pay.  However, we have an annual out-of-pocket maximum of $4K for prescriptions.  We always meet this annual max.  This year we will just meet it sooner, rather than later!  Our co-pay this month is about $3K for Kalydeco…then we are maxed out and all his meds (including Kalydeco) will be covered at 100% for the rest of the calendar year.  I wrote this on the drive home and am so exhausted.  I can’t wait to get to the hospital in the morning for Brady’s baseline sweat test and blood work.  He will see his CF specialist after that for his first dose of Kalydeco!!


  1. Wow we all need moms like you to fight for us. Kudos to your persistence. I am curious about the process of getting off label kalydeco for non G551. Your issue was his age precluded him? My question is that kalydeco did show some efficacy with Df508 just not enough to get a drug approved( about a 5-8 % versus 30%+ with G551). So what do you think about that process of getting off lable for just Df508 patients.
    Thanks Rob