Vertex Patient Assistance Program

[Runnergirl227]

I have recently started on Kalydeco. I have two rare genetic mutations and one is a residual function mutation. However, neither are on label because they are very rare. I have Q493* (there are 288 other people in the CFTR2 database with this mutation) and D513G (there are 7 other people in the database with this mutation.) So, the likelihood that they will ever be on the label is slim. My insurance initially denied the claim because it was off label. However, on appeal they decided to cover it for a 6 month trial basis. So, before starting I had a repeat sweat test, a nasal potential test, and a new test called an OCT (basically looking at nasal cilia). I have recently reaquired Pseudomonas and Mycobacterium Avium. So, hopefully after a trial period on Kalydeco, cultures will improve, and we will see improvements in the sweat test, NPD, and OCT.

So, currently I have already met my out of pocket max for this year because I have been on cayston for pseudomonas eradication. For this reason, my copay was $0 for the Kalydeco. However, and with the assumption that Kalydeco is effective for me, in January my co-pay will be 20% of the cost of the drug after meeting my deductible. My cost would be about 5,900. My CF center said that I will not be able to participate in the Vertex Patient Assistance Program because Vertex does not cover anyone using the drug off-label.

Has anyone been able to get copay assistance from Vertex for off label use? I would really only need assistance for the first month because after that I should hit my out of pocket max.

 

[jricci]

Unfortunately, you won't be able to get co-pay assistance for off-label, it's not legally permitted. I know of others in your situation, some who don’t have an out-of-pocket max so their co-pays are exhausting their retirement savings; some have had to stop taking it all together because they can’t afford it. So upsetting that this is the reality for some people and the cost of these drugs are preventing people from receiving a treatment that is literally saving their lives.

Some states have catastrophic illness funds. I don’t know if your situation would qualify. I used to be a nurse in a pediatric hospital and some patients' hospital bills were hundreds of thousands of dollars (after insurance). Those that lived in NJ could apply for a catastrophic illness children’s relief fund. This is only for NJ residents that are 21 years or younger.
There are a few states that have programs that aren’t limited to children, each with specific eligibility requirements. So maybe you could talk to your social worker or CFF’s Compass to see if your state has a program that might cover your deductible.

This would be too late for you, since you already started Kalydeco, but for anyone reading the thread that has a rare mutation that is expected to respond to a modulator:
There's a clinical trial that’s studying theratyping: https://clinicaltrials.gov/ct2/show/NCT03587961?term=off-label&cond=Cystic+Fibrosis&rank=1
It’s not expected to start enrolling until August 2019 and details about study are limited, such as how long will participants have access to drug and who is providing drug. I'm guessing insurance is providing the drug. This study is only offered at one study site, with 20 expected participants.
This is promising and will hopefully one day open the door for label expansion for rare mutations, but why aren’t these studies more extensive and being sponsored by CFF or Vertex and why didn’t they start as soon as FDA made that 2017 landmark approval for select residual function mutations, based in part on laboratory testing, not just clinical evidence? I thought this would have opened the door for N-of-1 trials. But here we are, 2 years later and still no CFF or Vertex sponsored studies for rare missense mutations. Maybe this will change once triple combo is approved and they'll start studies then?

Sorry, I got sidetracked there; I apparently needed to vent. But I hope Kalydeco works for you and I hope you find a solution to affording the co-pay. Keep us updated with how it’s going.

 

[jricci]

I just thought of something else. I'm not sure if you already know about the Healthwell Foundation. I would think they also have the off-label restriction, but it's worth looking into:

https://www.healthwellfoundation.org/fund/cystic-fibrosis-treatments-2/

 

[Runnergirl227]

This would be too late for you, since you already started Kalydeco, but for anyone reading the thread that has a rare mutation that is expected to respond to a modulator:
There's a clinical trial that’s studying theratyping: https://clinicaltrials.gov/ct2/show/NCT03587961?term=off-label&cond=Cystic+Fibrosis&rank=1
It’s not expected to start enrolling until August 2019 and details about study are limited, such as how long will participants have access to drug and who is providing drug. I'm guessing insurance is providing the drug. This study is only offered at one study site, with 20 expected participants.
This is promising and will hopefully one day open the door for label expansion for rare mutations, but why aren’t these studies more extensive and being sponsored by CFF or Vertex and why didn’t they start as soon as FDA made that 2017 landmark approval for select residual function mutations, based in part on laboratory testing, not just clinical evidence? I thought this would have opened the door for N-of-1 trials. But here we are, 2 years later and still no CFF or Vertex sponsored studies for rare missense mutations. Maybe this will change once triple combo is approved and they'll start studies then?
.[/QUTOE]

This is actually my CF center doing this research. I think I am the first person enrolled in this study. I did all of the pre-Kalydeco testing last week and will go back in 6 weeks to repeat it all. They also asked if they could write up my case as a case study to publish now. I said yes, of course. So, maybe this will help in the long run. Drug for this study is coming from my own insurance.

I am 44 and currently live in Louisiana. Healthwell may be an option, but previously I did not qualify. My job situation has changed so maybe I will now but I doubt it.

Thanks for the reply

 

[stephen]

Runnergirl227,

I have some thoughts:

Are you sure Kalydeco will not be covered next year even after you meet the deductible? From what you indicated, they are covering it this year.

I wea on Kalydeko off-label for a number of years. My drug plan also initially approved it for only a limited time. My CF center then submitted data showing Kalydeco improved my condition, and I then had no problems getting approved for it.

Also, if Kalydeco works for you, Symdeko should also. Although it’s not a tremendous difference in cost, Symdeko is less expensive.

Good luck!

 

[jricci]

Here’s a website with different financial assistance programs for CF:

https://www.needymeds.org/copay_diseases.taf?_function=summary&disease_eng=Cystic Fibrosis

No catastrophic fund for Louisiana that I could find. This is a very old document from 2008 that outlines different state funding for catastrophic illness. I couldn’t find anything more recent. Programs have different eligibility requirements depending on the state—some just for children, some for uninsured, some for specific conditions, etc.
https://www.cga.ct.gov/2008/rpt/2008-R-0638.htm

I’d still check with CFF Compass since this is such an outdated document. https://www.cff.org/Assistance-Serv...ce-Plan/Find-Resources-CF-Foundation-Compass/
When asking, I’d be vague and not mention that it’s for off-label Kalydeco-- just ask if there’s any catastrophic illness funding for Louisiana. Because of their financial connections with Vertex (royalty sale), they’re not supposed to give out any information connected to co-pay assistance for off-label use as it’s seen as off-label promotion. When I think about this, this is pretty upsetting that they wouldn’t be able to help you cover costs of a medication that is in a study that will be advancing CF research since this is part of their mission. I’m a strong supporter of the CFF, but there’s a clear conflict of interest going on. A necessary conflict of interest for the greater good of curing CF? That’s what I keep trying to convince myself of.

 

[Runnergirl227]

Thank you jricci for all of that research!

 

[chevbacher]

Hi there. I live in South Africa, my daughter is 11 with CF. We are USA citizens. Does anyone have any idea how I can register to get the 3 in one drug that’s out now? Any info would help! Thank you. Yocheved

 

[kenna2]

The triple combo med isn't out to patients yet. They are still deciding which triple combo med to put out. My doctor told me they should be out for patients towards the end of 2019.

 

[chevbacher]

Thank you Kenna2
i would love to get a head start with enrolling my daughter in a clinic that when it is available she will have access. Any idea how I go about enrolling? Do I just walk in to a local clinic?

 

[kenna2]

Talk to either your doctor or social worker at the clinic and let them know you want to get her on it when it becomes available. Vertex usually sends forms to CF clinic to have us fill out to get the process started with insurance ahead of time. So I'd ask to be contacted when that gets in.