Today show and Kalydeco

JENNYC

New member
I can't believe that no one has posted this yet!!! If you haven't seen this today....you need to take a minute and watch it!! This is a story about how siblings that have the same Dr, same CF genes, and same health insurance got two totally different responses to their Kalydeco request!!

http://www.today.com/news/man-battles-health-insurer-drug-could-save-his-life-6C10636133


This is an amazing story!!! I guess we were on the right end of the russian roulette!! Scary!!!
 
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sdeuber

Guest
I saw it and almost could not believe what I heard and saw. What a major cluster f... with these insurance companies.
We have to be thankful for the patient advocates and the NBC team to bring this to the public.
I would consider it discrimination and that is not allowed in the US as far as I know.
 

JENNYC

New member
I agree!! I hope this opens a lot of eyes and hopefully will make the insurance companies get with the program and stop the russian roulette.
 
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BreathinSteven

Guest
The problem is that it may open a lot of eyes - at insurance companies to tighten their determination as to who will and will not be approved for treatment of this nature. And an issue of this nature will likely cause them to tighten their process - not loosen... As many know, Kalydeco has shown beneficial for two specific mutations - though it appears to have benefits for a few others. It's a relatively small percentage of the CF population who is known to benefit. Neither the sister or brother have the mutations that are known to benefit. They didn't go into detail about how much the sister benefited - and no one knows if the benefits will continue, or if she's just having a lucky-health streak (she hasn't been on the drug very long)... The reviews approving the sister and denying the brother sound incredulous - but they were performed under different protocols. I'm rather shocked that the sister was approved, and not-so-much that the brother was denied. Now - because of the bad press, the insurance company was somewhat cornered into covering the brother. I believe that insurance companies should not be for-profit, and I believe they should not answer to shareholders - but beyond that, insurance companies have an obligation to all those insured to be conservative with the procedures and drugs they allow their policy holders. For the sake of the ability to pay for medical procedures in the future - they need to avoid paying for things that will not work, or will not improve someone's condition. The problem is that we don't know whether something will always work - but the documentation available says it's not likely in this case. For these two - and for the insurance company - it's a $600,000.00 gamble for the first year. If it works - that's wonderful and it was money well spent. If it doesn't - that's $600,000.00 down the drain and not available for other, possibly better treatments. What made the news here is the discrepancy between the sister and brother. If they'd have both been turned down, this never would have made the news. The sad thing is that the insurance companies will likely step more carefully in the future - and that will not be to our benefit. Love, Steve
 

JENNYC

New member
Very true Steve! I wish they would give people an opportunity for a 90 day trial to see if it works. In a perfect world the cost would not be so astronomical and again we wouldn't be having this conversation. But here we are and I am just grateful that my little one had the chance to try and benefit from it. Abby has been on it for 1 year and the difference is amazing. We have not had a hospital stay or surgery since starting. She has become a normal little girl that simply does breathing treatments and takes pills. Thanks for your comment Steve :)
 
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lifeisgood729

Guest
Today Show and Kalydeco

Steve,
My brother and I were the two people in the piece on the Today Show.
Your points are interesting, and I can understand how you might form some of those opinions based on the limited information in the Today Show segment. However, I stand by our decision to ask for this drug off label, and I'd like to clarify.

We did not enter into this decision to ask for a Kalydeco prescription irresponsibly, and I am quite certain our doctor never would have written the prescription for us if we hadn't first gathered plenty of evidence to support our request. We do not have G551D, the FDA "approved" mutation, but we have 3849+10kb C>T, part of a class known as "residual function" mutations. This means that our mutation does produce some functional CFTR. (unlike DF508, which produces none). There are plenty of science journal articles stating that residual function mutations are expected to benefit from a CFTR potentiator like Kalydeco. The science is strong enough that Vertex is currently in Phase 2 trials with Kalydeco and residual function mutations. They would not be investing in these trials if they didn't believe it would work. All of this science was submitted as part of our claims.

We also have DF508, but we have a third mutation, R668C, for which there have been in vitro studies showing it responds to Kalydeco. So, we were able to present scientific papers referencing two of our mutations as likely to benefit.

As for the idea that our appeals were submitted under different protocols, it's much more complicated than the statement MCMC gave to the Today Show would lead you to believe. In reality, my insurance company denied me twice also. Fortunately, there is a system in place so that consumers can go to their state and ask for an independent third party review of the insurance denial. (Insurers fought against that law, by the way, for obvious reasons.) In my third party external appeal, an independent doctor agreed with my analysis of the science and concluded that I was likely to benefit from Kalydeco. Thus, the insurance company was told by the state that they must cover me.

Kevin's story is more complex, and I won't go into all the details. The short version is that they "accidentally" sent his appeal to the division that handles health insurance reviews as opposed to external reviews. i.e., those reviewers are paid by the insurance companies. Guess who they tend to find in favor of most often?

I do agree that our story didn't make any insurance company execs happy, but I doubt that it will cause them to tighten their procedures. They already had all of them in place well before our fight. Within months of Kalydeco being FDA approved, they had policies written stating very clearly who could get the drug. This is why they denied me twice, because I don't have G551D. Their problem is that the state requires them to submit to an impartial external review, and they can't change that system, as much as I'm sure they would like to. In fact, Kevin's contact with a NY State senator has inspired that senator to begin a review of the state appeal agency to make sure that the external review process is working efficiently and fairly. The insurance companies can't change the external review requirement; the best they can do is what they've been doing all along, which is hope that consumers will get tired and give up the fight. (Two good books on the subject: Rainmaker, which is fiction, and Deadly Spin, non-fiction written by an industry insider.)

As for my own improvements, I saw a 6.5% increase in my FEV1 within two weeks of starting the drug, without the use of antibiotics. This came after a year of continually declining numbers. Nothing was bringing them up, in spite of having tried every available treatment. I also saw a drop in my sweat chloride values after starting Kalydeco. I have maintained this increase for ten months now.
We have another brother who won an appeal in his state, and he has been on Kalydeco for a few months. He has also seen an increase in FEV1 and a decrease in sweat chloride values. This is in addition to the subjective improvements we both feel. I have also spoken to people in the Denver residual function trials who are seeing similar results. This is not just a "period of good health." The drug is working with our mutation, and I feel confident that within the next year or two the FDA will add residual function mutations to the labeling.

You seem to imply that asking the insurer to cover this was throwing away money that could have been used for other people and other treatments. Here is my problem with that: If we didn't have Kalydeco to slow our decline, Kevin would be heading for transplant in the not too distant future. That, too, is an extremely expensive option, and there is no guarantee with that either--no guarantee of success, not even a guarantee of long-term survival. So, if a person dies within a year after transplant, should we look at that as "money down the drain"?

By the way, one quick Google search tells me that the CEO of UnitedHealth Group was named by Forbes as the highest-paid CEO in 2011. His estimated compensation last year: 48.8 million dollars! Even if Kalydeco had not worked in us and was an expensive gamble, as you put it, that $600,000 is a drop in the bucket compared to the billions these companies rake in every year. Their concern is not with "saving money" to be available for other patients. It's with protecting their astronomical profits.

Unfortunately, Kalydeco is an outrageously expensive drug, and that is an issue that also complicates the matter. I'm not advocating that every CF patient should run out and try to get Kalydeco, because you're right, it won't work for every mutation. It's not a battle to be entered into lightly, but if the science points to a high likelihood of success in one's particular mutation, why shouldn't we be able to request the drug? I have a right to my life just as much as that CEO has a right to his enormous salary.

Respectfully,
Martha Weber
 

triples15

Super Moderator
How cool that it was you on the Today Show Martha! I had heard about the segment but have not seen it. I will have to watch it now for sure!

You and I have spoken (via email)before, and you helped me make my case to my doc for prescribing Kalydeco based on my having the residual function mutation S945L. I DID end up looking at the patent, and it DOES include my mutation. YAY! The in-vitro evidence is also VERY encouraging. Based on that evidence, it appears that Kalydeco may work even better for people with S945L than for those with G551D. However, none of this was enough to sway my doc. :( I agree with Jenny, a trial period would be AMAZING!!

To "hear" you say that you feel confident that within the next year or two the FDA will add residual function mutations to the labeling is music to my ears. I think about when and how I am going to get my hands on Kalydeco every single day. I tried to get into the Denver study but ended up with a poorly timed and long-lasting exacerbation and didn't make it to the screening. Ugh.

Do you think when looking at residual function they will be looking at genotype only? Or genotype and phenotype? I know to get into the study I needed not only to have the residual function mutation, but also to have sweat chloride under 80, a certain fecal elastase (can't remember the number), and certain lung function. I couldn't get in on genotype alone. I'm hopeful that if/when it is approved for those with residual function it is based on mutation only.

Thoughts?

Autumn 32 w/CF
 

cftrsplicing

New member
I don't think so.
I hope the n of 1 trial will be based on phenotype rather than on genotype. So they are focusing on discussing with FDA (And I hope for myself even with EMA) on approving this drug for other patients based on biomarkers of improvements and markers of residual function (I.E. Pancreas sufficiency).
I have some more news but I'll post it on my site next month.
 

Aboveallislove

Super Moderator
Martha,

Thank you so much for the detailed response. I came to point out today that statement only 2 mutations are proven to benefit is wrong. There is a huge group the research shows can benefit. But I also agree that not everyone should try to get and that insurance companies can and should deny some. Our son is ddf508 and has absolutely no residual function. While I wish it would work for him, it won't. So happy though that you and your sibblings can and are benefitting!!
 
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lifeisgood729

Guest
Autumn, I don't know much about what criteria they will use when they add more patients to the current FDA labeling. I'm going to defer to cftrsplicing on this one, as he seems to have more information about what Vertex is going for in their talks with the FDA. If some of you don't know, he has a great web site, cftrsplicing.com, where I found almost all of the science I submitted with my appeal. If anyone has a splicing mutation and is looking for evidence to support an appeal, that's the place to look.

cftrsplicing, I'm looking forward to whatever you post next month.

Martha
 

JENNYC

New member
While I see everyone's point. If I had not tried it. If I had not pushed to get Abby on it before we found out what her 2nd mutation was I don't even want to think about how many more surgeries we would have added to the 15 she had prior and IV treatments she would have had to endure for no reason. She is 1 of 12 that have 2585delT so I highly doubt that they will be testing anyone with that gene. I guess I just don't want anyone to be left out who it may benefit, because it's not just a small improvement. It's truly life changing. And I am so grateful for Martha and Kevin for the wonderful segment they did and am truly happy for them both.
 

2roses

New member
Go Martha! So glad you are on Kalydeco!!! Thank you for your detailed explanation above. Has Kevin started yet? Please keep us posted on how you two are doing (well three with your other brother:). Thoughts and prayers your way!

My two daughters (20 and 14) have one G551D, which immediately qualified them, and they have been on Kalydeco since 2/2012. Their CF is very symptomatic, and they have a history of multiple illnesses and hospitalizations a year (with long term lung function still very high for CF standards). While they are still symptomatic, they have been stable, fewer exacerbations, no need for constant oral antibiotics, and NO hospitalizations since starting. Still on 2-3 resp treatments a day and asthma meds, enzymes, other GI meds, etc.

Life is good :)
 
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BreathinSteven

Guest
Hi Martha…

Thank you so much for your detailed response… It is always good to get many of the details that the media always leaves out.

I did mention that I believed that insurance companies should not be for-profit, and should not answer to shareholders. And, I agree that it is unconscionable that the CEO of UnitedHealth is making the salary he is making. That is an entirely different topic and sheds a bad light on our entire corporate culture – not just health insurance organizations.

I didn’t quite imply that covering this situation was throwing money away – what I said was, “it's a $600,000.00 gamble for the first year. If it works - that's wonderful and it was money well spent. If it doesn't - that's $600,000.00 down the drain and not available for other, possibly better treatments.” You apparently provided substantial evidence that it may work for your mutation – and it worked – and that may be the “money well spent” part of my statement. It still appears that the population that Kalydeco will help is rather limited – I don’t know if you experience identical improvement to those with the “approved” G551D mutation… But I can understand a provider’s reluctance to approve an incredibly expensive treatment with little indication that it might actually work. I’m glad that it worked for you, and I’m thankful for your improvement – but, if Kalydeco had done nothing for you, I believe it would have been throwing money away, and it could have reinforced their reluctance to cover similar situations.

As for transplant survival and “throwing money down the drain” – there are similar provisions in providing transplant that work to disallow patients who would not benefit from transplant. If you are too sick – if you have certain conditions – you would not be allowed to be a transplant candidate. Dying within a year would not be a good thing – but testing and qualifying for the treatment is rigorous… Probably not unlike much of the research you had to do to show Kalydeco would likely work for your mutation versus a G551D request. I would look at it as “money down the drain” if the candidate was clearly not viable for the procedure – but proceeded anyways. But, as always, determining “clearly” is the difficult part…

Thank you for the more detailed explanation – and for enlightening me on the background of your cases. My wife and I too have had rather epic battles with my insurance companies over the years – your substantiation is impressive.

Love, Steve
 

PennyBK

New member
Hi Martha - I really appreciate your sharing this detailed information. I sent you a PM - let me know if it didn't go through. Thanks! Anna
 
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lifeisgood729

Guest
Hi Anna. Your message did go through. I just never noticed I had PMs. Sorry. I sent you a reply.

Martha
 

malcom10

New member
Hello guys,

anyone knows about user cftrsplicing who was thankfully sharing lot of important information and studies about Kalydeco and its use and possible effects on splicing mutation?

Thank you in advance to anyone who has news.

Alessandro
 

MissMe

New member
Hello guys,

anyone knows about user cftrsplicing who was thankfully sharing lot of important information and studies about Kalydeco and its use and possible effects on splicing mutation?

Thank you in advance to anyone who has news.

Alessandro
You can try to send an e-mail to the adress on the cftrsplicing.com web site!
 
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