FDA Approves Triple Combo - Trikafta
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Helenlight
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Is this the same as elexecaftor or is that a different one
Great article about the triple combo, Trikafta:https://www.washingtonpost.com/heal...urn-deadly-disease-into-manageable-condition/
https://www.washingtonpost.com/heal...urn-deadly-disease-into-manageable-condition/
I really liked this article until I got to the end. The author wants to make the fact that these breakthrough modulators do not work for part of our community about race and socioeconomic status and race. That is so NOT the reality and could not be further from the truth! The reason that the rare and nonsense mutations have not been solved yet is because it is a much tougher fix scientifically!! We are not leaving these brothers and sisters behind. Science is a process. We solved the gating mutation problem first with Kalydeco because it was the easiest to fix. Next came the F508del because it was the next easiest and also the greatest population to benefit. The CFF could not be more clear!! We are not done and will not be done until we have a treatment for everyone with CF. The Washington Post does a huge disservice to our community by trying to divide us by race or economics. We stand together!!!!!
In response to above post from shay:
Unlike nonsense mutations, the science for effective drugs for many of the rare missense mutations is there, but the FDA approval process hasn’t caught up with the science.
I have a missense mutation and I’ve been on off-label Kalydeco for 5 years. My R334W mutation hadn’t been included in studies.
I was able to get it off-label because I have excellent insurance coverage and my copay is very manageable with our income.
However, this isn’t the case for many. Those on Medicaid, Medicare are at a clear disadvantage in getting approval of an off-label drug at this cost. And some of those with commercial insurance, who have been approved, can’t afford the co-pay and have had to stop taking it. Vertex co-pay assistance programs can’t be used for off-label drugs.
So, in certain cases, there is an inequity in accessibility of these breakthrough drugs based on income level.
The provider from UCSF that is quoted in the article recently did research that showed that “In cystic fibrosis (CF), the spectrum and frequency of CFTR variants differ by geography and race/ethnicity. CFTR variants in White patients are well-described compared with Latino patients. No studies of CFTR variants have been done in patients with CF in the Dominican Republic or Puerto Rico.”
https://www.genomeweb.com/sequencing/rare-cystic-fibrosis-mutations-dominate-caribbean-patients
She states that:
"If people with different mutations aren't being included in those trials, they're never going to benefit. This is one of the challenges of personalized medicine: it only works if you are including diverse groups in your studies — otherwise it just reinforces existing racial and ethnic health inequality."
Her point is well taken. It’s so difficult to be patient when there is a medication that is sitting on shelves that could be saving lives, but it’s not accessible--not because it’s not expected to be effective--but because the mutations weren’t included in studies.
The approval process will eventually catch up with science, but the reality is that lives that could have been saved, will be lost while we wait. And that is a painful truth to acknowledge for both patients and providers.
I would hate to end with that statement because for me, this article captured the significance of Trikafta, and underscored this monumental development that much of the CF community can celebrate. I shared it will all my friends and family and wanted to share it with this cysticfibrosis.com community.
So, I’ll end with what I thought was one of the most powerful statements of the article.
“Doctors who began their careers at a time when there were few adults with cystic fibrosis because patients died in their teens are now cautiously anticipating that the disease will be transformed into a chronic condition, akin to diabetes, that can be managed with a drug regimen — particularly if Trikafta is eventually approved for use in younger children and babies, before any lung damage has occurred. Patients who were unsure about whether they should bother attending college because they had always known they would die young are now being told they should think about planning for retirement."
Unlike nonsense mutations, the science for effective drugs for many of the rare missense mutations is there, but the FDA approval process hasn’t caught up with the science.
I have a missense mutation and I’ve been on off-label Kalydeco for 5 years. My R334W mutation hadn’t been included in studies.
I was able to get it off-label because I have excellent insurance coverage and my copay is very manageable with our income.
However, this isn’t the case for many. Those on Medicaid, Medicare are at a clear disadvantage in getting approval of an off-label drug at this cost. And some of those with commercial insurance, who have been approved, can’t afford the co-pay and have had to stop taking it. Vertex co-pay assistance programs can’t be used for off-label drugs.
So, in certain cases, there is an inequity in accessibility of these breakthrough drugs based on income level.
The provider from UCSF that is quoted in the article recently did research that showed that “In cystic fibrosis (CF), the spectrum and frequency of CFTR variants differ by geography and race/ethnicity. CFTR variants in White patients are well-described compared with Latino patients. No studies of CFTR variants have been done in patients with CF in the Dominican Republic or Puerto Rico.”
https://www.genomeweb.com/sequencing/rare-cystic-fibrosis-mutations-dominate-caribbean-patients
She states that:
"If people with different mutations aren't being included in those trials, they're never going to benefit. This is one of the challenges of personalized medicine: it only works if you are including diverse groups in your studies — otherwise it just reinforces existing racial and ethnic health inequality."
Her point is well taken. It’s so difficult to be patient when there is a medication that is sitting on shelves that could be saving lives, but it’s not accessible--not because it’s not expected to be effective--but because the mutations weren’t included in studies.
The approval process will eventually catch up with science, but the reality is that lives that could have been saved, will be lost while we wait. And that is a painful truth to acknowledge for both patients and providers.
I would hate to end with that statement because for me, this article captured the significance of Trikafta, and underscored this monumental development that much of the CF community can celebrate. I shared it will all my friends and family and wanted to share it with this cysticfibrosis.com community.
So, I’ll end with what I thought was one of the most powerful statements of the article.
“Doctors who began their careers at a time when there were few adults with cystic fibrosis because patients died in their teens are now cautiously anticipating that the disease will be transformed into a chronic condition, akin to diabetes, that can be managed with a drug regimen — particularly if Trikafta is eventually approved for use in younger children and babies, before any lung damage has occurred. Patients who were unsure about whether they should bother attending college because they had always known they would die young are now being told they should think about planning for retirement."
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Thanks so much for your illuminating post jricci. I am so painfully aware of the disparities of insurance plans in this country, so I did not mean to imply that there are not inequalities. I also understand that with the enormous number of rare mutations that the approval process for inclusion is not keeping up with the science. My intention, and perhaps I was less than eloquent, was to express solidarity with all people with CF and try to unite our community in moving forward from this current success to tackle all these other problems. I have seen so much divisiveness in this country and would hate to see that spill over to our community. We are a small but mighty family and I care deeply about this battle we share.
shay, I apologize if my tone was harsh. I didn’t mean to undermine your knowledge or experience. This particular topic is an extremely emotionally charged one for me. Helping those with rare mutations gain access to these breakthrough therapies has been a 5 year long plight for me. Unfortunately I’ve become somewhat jaded and hardened in the process and wish I didn’t know some of what I’ve discovered over the years.
I realize your comments were intended to unite our community. I agree that the divisiveness in our country is toxic and we that we, as a community, need to stand together.
I realize your comments were intended to unite our community. I agree that the divisiveness in our country is toxic and we that we, as a community, need to stand together.