Military Significant Others and Spouse Support - MilitarySOS.com
Page 1 of 3 123 LastLast
Results 1 to 10 of 23

Thread: $300,000 Cystic Fibrosis Drug

  1. cuz i'm wonderful
    linzerfufu's Avatar
    linzerfufu is offline
    cuz i'm wonderful
    Join Date
    Aug 2010
    Location
    Oregon
    Posts
    11,833
    #1

    $300,000 Cystic Fibrosis Drug

    Advertisements
    Kalydeco is truly a wonder drug.

    Developed by Vertex Pharmaceuticals, it is the first drug that attacks not just the symptoms but the underlying cause of cystic fibrosis, a genetic lung disease that usually kills victims by the time they reach their 40s. It doesn’t work for every sufferer of the disease, but rather for a small subset — probably around 2,000 people — who have a specific genetic mutation that the drug targets. But for those it helps, it is life changing.

    “I still pinch myself every day,” says Emily Schaller, 32, who has been taking the drug since she participated in its Phase III trials five years ago. “I can take deep breaths. I can run without coughing.”

    Two years ago, as it was coming to market, Dr. Margaret Hamburg, the head of the Food and Drug Administration, described Kalydeco an “an excellent example of the promise of personalized medicine.” Personalized medicine describes drugs that treat so-called orphan diseases — that is, diseases with a small population — or subsets of people with broader diseases. This kind of targeted medicine has been the Holy Grail ever since the genome was first sequenced about a decade ago. Now it is becoming a reality.

    There is one other way that Kalydeco is an excellent example of personalized medicine: its cost. It’s more than $300,000 a year. Because patients will likely be taking the drug for the rest of their lives, it could cost millions of dollars to keep just one patient on Kalydeco. That raises another important question about the coming of personalized medicine. How are we, as a society, going to pay for it?

    What brings this question to the fore is a fight taking place in Arkansas, where the state’s Medicaid program is balking at paying for Kalydeco for a handful of young patients with cystic fibrosis. Although state officials won’t say so publicly, it is clear that cost is a key issue; The Wall Street Journal got ahold of emails that show Arkansas officials “discussing Kalydeco’s cost, and their worries about the expense of future cystic fibrosis drugs.”

    It’s likely that Arkansas will eventually fold. Most state Medicaid programs — and private insurers — are paying Kalydeco’s cost because it works so well, and because the patient population is so small.

    What happens, though, when there are 200 such drugs? Or when they are targeted not at cystic fibrosis, which has maybe 30,000 sufferers, but at diabetes or (heaven forbid) cholesterol? A drug called Sovaldi, marketed by Gilead Sciences, takes aim at hepatitis C. It is described as a “breakthrough” drug. But each pill costs $1,000 — and the full regimen costs $84,000. And the hepatitis C population isn’t 30,000 — it is over 3 million. If everyone with hepatitis C took Sovaldi, it would cost something like $300 billion, which is about what the country now pays for all prescription drugs combined.

    “This is the future of medicine,” says Barry Werth, “and there’s going to be a reckoning.” Werth is the author of “The Antidote,” a terrific book about Vertex and its race to discover and bring to market these new kinds of drugs. “Everyone wants to see these drugs succeed,” he told me. “Wall Street is all charged up. There really hasn’t been any pushback yet on cost.”

    And even when the pushback comes, as it surely will, how will we get the pharmaceutical companies to change their pricing? Brian O’Sullivan, a cystic fibrosis specialist at the University of Massachusetts Medical School, told me that he thought the price of drugs like Kalydeco was “not sustainable.” But in the next breath, he marveled at how well the drug worked and said he didn’t want to “scare companies away from doing cystic fibrosis research” by focusing too much on the cost.

    When I asked Vertex how it could possibly justify charging $300,000 for Kalydeco, a company spokesman pointed to the small patient population and “the benefit that the medicine provides.” He also said that the company had spent $6.5 billion on research in its existence, and had only two drugs approved by the F.D.A. In effect, he was saying that the Vertex drug was priced, in part, to recoup not just the research and development that led to Kalydeco but all the company’s R&D. (For cystic fibrosis sufferers with no insurance, Vertex provides the drug for free.)

    When I asked Werth how Vertex could charge $300,000, he had a much simpler answer: “Because they can.”

    Vertex has another cystic fibrosis drug that has just come through its Phase III trial and is likely to be on the market soon. It attacks a different, more common problem than Kalydeco and may broaden the number of patients who can be helped to more than 15,000. In my talks with people in the cystic fibrosis community, I got the strong sense that they are hoping this next drug will cost less.

    Dream on.
    What do you think of the price tag? I know that it's a business, and research/testing/costs are high to make new drugs, but it's also a life saving treatment that a person with CF has to take for the rest of their lives.

    My friends daughter has CF, and it's heartbreaking seeing her not be able to do the things kids should do because of her CF. Her mom doesn't even make $300,000 a year, and I'm pretty sure insurance companies are gonna freak at the cost.

    On one hand, I know it's expensive. On the other, it strikes me as fundamentally wrong that people would be dying because they can't afford treatment (for anything.)
  2. cuz i'm wonderful
    linzerfufu's Avatar
    linzerfufu is offline
    cuz i'm wonderful
    Join Date
    Aug 2010
    Location
    Oregon
    Posts
    11,833
    #2
    I'm thrown by the "if you don't have insurance it's free" part. I did the math, if 21,000 people took this drug, they'd recoop their 6.5 billion in a single year.
  3. In vino veritas
    Dr.VinoVet's Avatar
    Dr.VinoVet is offline
    In vino veritas
    Join Date
    Nov 2010
    Posts
    14,852
    #3
    Doctors, medical researchers, hell, even the janitors in the building it was developed in over years- all need to get paid. Yes, its lifesaving, but do people do my lawn for free? Or clean my teeth for free? Free service at restaurants because food is necessary for life? Or free groceries? Is my apartment free since I need shelter to survive? No. Life comes with costs, and sadly, life isnt fair, from birth to death.
  4. Team Rocket
    rocket_lizz's Avatar
    rocket_lizz is offline
    Team Rocket
    Join Date
    Mar 2012
    Location
    San Diego!!!
    Posts
    9,069
    #4
    I have mixed feelings about this. Drug development is SOO expensive that I understand companies wanted to recoup the cost. However, I don't think lifesaving care should be withheld, ever, due to cost. As the USA eventually works its way towards a single payer system (we all know it's coming), these costs won't be sustainable. No way is the gov't going to allow companies to set the price. I expect it will be more like medicaid/medicare where there is a much smaller, pre-determined payment.

    In the meantime, I think it's going to be tough for patients and doctor to navigate the issues around this. Do you ONLY get it free if you don't have insurance? What if your insurance won't cover it? Or only covers 10%? Kwim? And with the differences between states and even cities, it can get very confusing. My dad recently had a new prescription that he found out wasn't covered and cost $700 a month. He went to his PCP to complain and the guy was like "oh yeah, the Dr. who prescribed that to you just moved here from CA, where people can get rebates to cover the cost." No such rebates in Wisconsin!
    WiggleWiggle~ is my Wifey
  5. In vino veritas
    Dr.VinoVet's Avatar
    Dr.VinoVet is offline
    In vino veritas
    Join Date
    Nov 2010
    Posts
    14,852
    #5
    And I know that may seem harsh, but, honestly (no snark), where to we draw the line? How do these people (the people who made the drug) get paid to survive in their own lives if their work is expected to be given away?
  6. Pour a little salt, we were never here
    [his] lobster's Avatar
    [his] lobster is offline
    Pour a little salt, we were never here
    Join Date
    Jun 2012
    Posts
    8,737

    #6
    This is semi personal to me cause I have CF. This drug isn't for my mutation combo, but one is very near release for me and of course my friends can/are benefitting from this one.

    It reminds me of when I got malaria in Tanzania. I walked in, was tested, paid $30 for meds and left. Felt better the next day. Mean while their were parents begging for their babies and elderly people unable to pay. It was heartbreaking to know the fix was so easy and cheap and totally out of reach for them.

    Just because a substance exists, doesn't give you a right to it. Now this drug was obviously researched and created knowing it would be $$$, and we do for the most part have insurance in the US so there are differences from malaria pills and this but in the end I still don't think it's a human right to get something someone else paid for, worked on, etc...its like the hen who baked the cake. You don't just get it because you want it. Sad sometimes, yes but I still think its a value worth upholding.
  7. cuz i'm wonderful
    linzerfufu's Avatar
    linzerfufu is offline
    cuz i'm wonderful
    Join Date
    Aug 2010
    Location
    Oregon
    Posts
    11,833
    #7
    I wasn't talking given away, I was saying more sustainably priced. I realize the market is small at 30,000 CF patients, but then again, if people can't afford your treatment, well, people can't afford your treatment. You're closing off your own market further by making it (seemingly) unreasonably priced.

    I get what you're saying, Vino, and I agree. However, I would argue that for a large portion of Americans, groceries/apartments/ect. are more feasible than $300,000 a year for the rest of your life. Cancer treatments, Hepatitis drugs, those are all relatively "short term," but these folks need it forever.
  8. Pour a little salt, we were never here
    [his] lobster's Avatar
    [his] lobster is offline
    Pour a little salt, we were never here
    Join Date
    Jun 2012
    Posts
    8,737

    #8
    Well, I think all medical crap should have the inflation balloon popped but that's a massive societal undertaking not specific to this med.
  9. cuz i'm wonderful
    linzerfufu's Avatar
    linzerfufu is offline
    cuz i'm wonderful
    Join Date
    Aug 2010
    Location
    Oregon
    Posts
    11,833
    #9
    Quote Originally Posted by [his] lobster View Post
    Well, I think all medical crap should have the inflation balloon popped but that's a massive societal undertaking not specific to this med.
    Yeahhhhhhhh, that's a huge issue.
  10. Dancing Backwards in High Heels
    HisJuliet's Avatar
    HisJuliet is offline
    Dancing Backwards in High Heels
    Join Date
    Nov 2010
    Location
    Together Again!
    Posts
    9,081
    #10
    Quote Originally Posted by [his] lobster View Post
    Well, I think all medical crap should have the inflation balloon popped but that's a massive societal undertaking not specific to this med.
    Agree, but where does it start? Doctors/researchers are paid well for these labs because (generally) they went to really good schools, that cost a lot. The good schools argue that their tuition is so high because they hire outstanding faculty and have state of the art equipment, which again is costly. The state of the art equipment is arguably costly because they hire experts to develop the machines, who may have the same school tuition issues as the doctors/researchers.

    It seems like a never ending cycle wherein I can seem to find out where the "fat" needs to be trimmed.

    This drug is expensive, but I'm not sure if it is completely unreasonable.
Page 1 of 3 123 LastLast

Posting Permissions

  • You may not post new threads
  • You may not post replies
  • You may not post attachments
  • You may not edit your posts
  •