The Cost of Staying Alive?

In the latest issue of Science, there’s an article about Trikafta (elexacaftor/ivacaftor/ tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation, which was approved by the FDA this past October.

In the U.S. alone, there are more than 30,000 people with cystic fibrosis, an inherited condition characterized by the buildup of thick, sticky mucus that results in progressive damage to the respiratory system and chronic digestive system problems, and that can have other complications as well. A generation ago, children with CF seldom lived to adulthood, and even today CF sufferers seldom live to age forty. Currently, there are more than 30,000 people in the U.S. with the disease.

In the interests of full disclosure, I have a personal interest in cystic fibrosis, because almost forty years ago one of my daughters was given a preliminary diagnosis of CF, which was indeed terrifying. Fortunately, additional tests determined that she didn’t have CF, but a combination of other factors mimicking it, which were treatable, and she recovered without lasting ill-effects. But that experience definitely made me aware of the devastating effects of cystic fibrosis.

So, the development of Trikafta struck me as a godsend for cystic fibrosis sufferers… until I saw the price tag, $311,000 a year [the “list” price], every year. That’s what it will cost to provide CF sufferers with the chance to live a close to normal and productive life.

Since Trikafta is designed to deal with the most common form of cystic fibrosis, it won’t work for all CF sufferers, but even if it only works for 60%, the annual cost to those people, or their insurors, would total nearly $6 billion.

Needless to say, some medical insurance companies have already turned down patients.

Somehow, I doubt that developing Trikafta cost anywhere near $6 billion, and it strikes me that, at least from everything I’ve seen published (and I hope I’m wrong), this is just another form of medical extortion.

5 thoughts on “The Cost of Staying Alive?”

  1. dave says:

    I saw this and started thinking about enzymes Nattokinase, Serapeptase and Lumbrokinase which are all useful for breaking down fibrin in the vascular system. I take them to dissolve biofilms from Lyme/Bartonella/Babesia/Mycotoxins/metals, natural slimy collections of bacteria/metals/toxins, in the body. It does look like these are some low cost items to help alleviate cystic fibrosis. It seems most of the US medical industry –> BigPharma — research $$ universities –> indoctrinating doctors to pharma –> patient prescriptions –> $$ spending on Pharma (insurance/subsidies/cash). The end result is that doctors, via Pharma marketing, believe that one drug fits all, when instead each animal/human is incredibly unique in their genetics and ongoing immune expression (phenotype). It’s been a long journey for me, but I’m at the point of demanding public research and data, that protects privacy, on an individual level. That threatens Pharma profits though.

    1. Wine Guy says:

      No competent doctor believes that one size fits all when it comes to medications. There are too many variables. “Incredibly unique is your redundant way of putting it.”

      As for “patient prescriptions” being part of the problem… do you take your car to the mechanic then tell that person that they cannot use their wrenches and other socket set? No. Medications are my tools. Surgery and procedures are my tools. Without them, there’s no point to modern medicine (hint: it’s in the name).

      The days of ‘Big Pharma’ flying doctors to resorts, buying expensive pieces of equipment, and such have been over since the early 1990s. For my entire hospital, there hasn’t been a ‘Drug Rep Lunch’ for the entire 15 years I’ve been working there and when reps (drug or equipment) come, it’s to familiarize us with new additions to the pharmacy or a new piece of equipment. They don’t even leave pens… just business cards. Compare that to when curriculum reps come to the little charter school I’m on the BoD for has a curriculum representative come give a presentation for the elementary language arts or mathematics…. food, pens, sometimes iPads. My neighbor who owns a construction company regularly has heavy equipment manufacturers fly him out to nice places to look over their latest tech and he leaves with lots of free nice stuff. Same for my co-worker’s husband who owns commercial dairy and beef ranches.

      Big Pharma’s latest ‘big win’ was direct to consumer marketing: Viagra, psoriatic disease treatments, inflammatory bowel treatments, etc. Doctors say no to pharma all the time. It is much harder to say no to a patient desperate for treatment of their disease, even though the medication is only marginally more efficacious than older meds.

      If you want to be serious about regulating Big Pharma, 1. make them publish all of ther data, good and bad; 2. For medications, publish absolute numbers, not relative numbers including numbers needed to treat and numbers needed to harm; 3. cap their advertising to 5% of gross income.

      And CF is just one disease. Other expensive areas of disease: Cardiovascular disease, Neoplasia (cancer), Diabetes. And there are PLENTY more. Tom’s point below is absolutely relevant… and US citizens have a very hard time saying ‘no, I’m not willing to limit my treatments or anyone else’s because they are too expensive.’

      As an aside:
      The three protein supplements you mention are unregulated food/vitamin supplements that (even in their countries of origin) have only been recently studied and there have been no long term methodical studies to look at their safety, much less their effectiveness. Nattokinase, for example, looks promising, but there have only been 10 very limited studies (both in duration and # of people).

      Same for Serrapeptase:

      Do they work? No idea. The evidence is sketchy and my personal litmus test for all of my patients is “would I use it? would I give it to my family?”

  2. Tom says:

    According to Wikipedia:

    ‘In an analysis of the drug development costs for 98 companies over a decade, the average cost per drug developed and approved by a single-drug company was $350 million.’

    The pharma companies do have a number of failures and their cost of development to the point of failure is part of the total cost of the successful drugs developed for that company. This cost could vary considerably.

    Social/Human evolution requires knowledge: the problem is how to encourage research and development and specifically who should pay for the successes and the failures.

    We return to ethics when their is a question of avarice.

  3. Daze says:

    There’s a very interesting discussion of the whole issue at this Guardian article:

  4. Angela says:

    I have Cystic Fibrosis. I am 44 and started taking TRIKAFTA in NOVEMBER 2019. Admittedly when I saw the price tag it was a shock. However, let’s analyze the cost of TRIKAFTA vs. the cost of NOT taking it. Some patients are on 4 breathing treatments a day, one treatment can cost $3-$4,000/mo, and if they are taking 4 of those, then that is $16,000. How much does it cost to go into the hospital for a week? How many weeks does the average CF patient go into the hospital every year (2-3?) What about IV antibiotics? A double lung transplant, how much is that surgery? What do the anti-rejection meds cost every month from the transplant? What about when you get cancer from your anti-rejections meds? How much does it cost to get cancer? I read in a group right when TRIKAFTA first came out from a father, something to the effect of this…. “my son is 17, had 37% lung function and he was on oxygen. We were about to go to DUKE to get evaluated for a double lung transplant, then we found TRIKAFTA. It’s been 5 weeks and his lung function went from 37% to 73%, he is no longer on oxygen, his port was removed and we are NOT going to Duke”. Admittedly I thought, “is this guy serious? …is he exaggerating?…this can’t be true”. But then I kept reading and heard more stories from parents. I had never been on a MODULATOR before because I never had the proper gene type. But I had what they needed for TRIKAFTA. Being self-employed I did have insurance, it was through ACA and to be honest it’s fabulous insurance through BCBS, and no, I don’t get a subsidy (of course, after 2020 I would probably qualify). With that said, I haven’t coughed since I started taking Trikafta. It’s been amazing. I almost think they aren’t getting paid enough…not only are they saving lives, they’ve spent a ton of money… and years and years of research. I’m pretty sure 4 weeks in the hospital for just a CF exacerbation would cost $300k. There aren’t a lot of CF patients so maybe they can’t lower the price, or shouldn’t.

    NOW…the other side of the coin. Yes, theoretically, it’s an outrageous amount of money and it should be a sin to charge that much, but the reality is, in our lifetime it won’t change and if it does, it most likely won’t be a significant change.

    With that said, I am going to the cardiologist tomorrow because ever since I started taking TRIKAFTA, my legs swell. And of course, 3 ER visits and 12 pairs of compression socks later they are still blaming it on too much salt. ( so weird, I ate salty things before TRIKFATA and never had that problem)

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