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
