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DBL%20Hendrix%20small.png College chemistry, 1983

Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: derekb.lowe@gmail.com Twitter: Dereklowe

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July 21, 2011

Drugs for Multiple Sclerosis: Worth the Price, Or Not?

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Posted by Derek

Now this is an uncomfortable study, if you're in the business of treating multiple sclerosis. An article in Neurology looks at the cost-effectiveness of several disease-modifying therapies: the two interferon-beta-1as (Avonex and Rebif), interferon-beta-1b (Betaseron) Copaxone, Betaseron and the immune modulator Copaxone (glatiramer acetate). The authors tracked ten-year quality of life, including lost time at work, overall time without relapses, and so on, and compared that with the cost of treatment.

The final figure is in dollars per quality-adjusted life year (QALY). That's not the most exact calculation in the world, but if you're going to try to rank cost-effectiveness, no measure is going to be without controversy. There's been a lot of debate about this in the UK, where the NICE explicitly uses these figures in its recommendations, and if you haven't heard much about the concept over here, well, you're definitely going to. What's considered a good figure? To give you an idea, the NICE starts raising an eyebrow at about $40K to $50K (based on 1.62 dollars to the pound). Here, we'll stand for 100K to 150K.

And how do the MS drugs compare? Closer to $1 million/QALY than any of those figures. All of them were above $800,000/QALY. In other words, the benefits of these drugs are real (although Copaxone's were less impressive compared to the interferons), but are they real enough to justify their prices? It'll be quite interesting to see where Gilenya (fingolimod) will land once it gets more of a track record in the real world. Note that the price of all these drugs has gone up since the study's calculations (while their effectiveness has presumably not budged) and that Gilenya, I believe, costs even more than the rest of them.

This naturally brings up all the usual questions about drug pricing. In no particular order, and with no priority given to those that I agree with, we have: Who says you can put a price on quality of life? Well, if you can't, then why can't drug companies just charge whatever the market will bear? What market - drug pricing is about the worst example of a free market you could ask for! Well, what if people want to pay out of their own pockets - shouldn't they be free to? Right, sure, who does that, and how much would sales fall if everyone had to? But still, shouldn't people be able to get what therapies are available - who's the person who gets to tell patients that they can't have what's out there? OK, but since a lot of this is Medicare and the like, are we supposed to pay for everything to be done to everyone forever? And why should these drugs cost so much, anyway? Well, because insurance companies apparently will pay for them - why don't you go complain to them? And so on. I honestly have no idea what the end to these arguments might be, but studies like this one are going to force us to have them again. Here's more from the New York Times and from Bloomberg, with a hat tip to FiercePharma.

Comments (25) + TrackBacks (0) | Category: Drug Prices | The Central Nervous System


COMMENTS

1. Zachary on July 21, 2011 10:18 AM writes...

When it comes to expensive cancer drugs, say, which keep people alive for a few more months (with lousy QOL) for hundreds of thousands of dollars, I think the cost is clearly NOT indicated. I would say so for myself, and for others as well, that it's just not worth the price.

While a cool million per QALY is just as clearly unsustainable, it's a lot harder to be cool and analytical about it for otherwise healthy young adults who could lead productive happy lives.

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2. fat old man on July 21, 2011 10:24 AM writes...

I have a different point of view, having observed chemotherapy in my family. Most of the expensive cancer drugs are around $10K per month. Why would you want to cut short someone's last few months of life (please no QAL arguments here)for 10K per month? After all, many/most will not have used much health insurance up to this point in their life and have been contributing to the system for decades. Maybe it's time for them to get something paid back?

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3. DCRogers on July 21, 2011 10:34 AM writes...

Now, redo those calculations using the actual UK prices for those things -- bet the result is magically closer to the desired $100K/QALY that is about the cutoff over there?

Wonder when US health care firms will start opening up foreign "resorts" and insist you move to one to get certain high-priced drugs at the local price?

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4. DCRogers on July 21, 2011 10:36 AM writes...

Oops, meant $50K/QALY.

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5. Rick on July 21, 2011 10:54 AM writes...

Apropos (at least I think so) of the subject of the validity of QALY and its underlying philosophy, utilitarianism, Michael Sandel's "Justice" lectures (you can find them at www.justiceharvard.org/watch/) are both relevant and fascinating. For anyone really interested in the ethics and morality behind the economics and marketing of new drugs, just check out the first few lectures. You will find that most criticisms or justifications of QALY were being discussed centuries ago by Bentham, Mill, Marx, Wittgenstein and others. IMO, we're still no closer to clear, useful and satisfying principles.

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6. NoDrugsNoJobs on July 21, 2011 11:06 AM writes...

fat old man - agree with you here. If a drug extends your life a few months when all you got is a few months, it effectively doubles your remaining lifespan. if all you had was a day left on this Earth, wouldn't you want to live that last day? Its interesting with cancer drugs that when people are complaining about the cost, they give a yearly price tag (100k per year!) and then they turn around and say ("And you only die in 6 months instead of 4!)

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7. Anonymous on July 21, 2011 11:23 AM writes...

Zachary--

Try telling that to a GBM patient whose use of Avastin gives them some additional months of relatively tolerable life before the truly terrible endgame begins.

And feel free to substitute GBM for any one of a dozen similar tumor types.

You may be able to say that the patient should bear more of the cost in those situations, but to dismiss the concept of extending life by a few months in a terminal situation sounds pretty ignorant to me.


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8. Anonymous on July 21, 2011 11:47 AM writes...

Is a high $/QALY just more common with biologics.

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9. Hap on July 21, 2011 12:17 PM writes...

At some point, though, drug costs are a zero-sum game (certainly for insurance companies, who aren't going to relinquish profits for my gain, and probably for governments who don't or won't have the money or, like insurance companies, want to spend it on something else) - money spent for cancer drugs or MS can't be used for something else. I don't want to die any sooner than anyone else, but if two or three people could live full (> 20 years) productive lives with the money spent on my extra two or three months, well, I don't know that that's good for any of us.

QALY is probably a helpful figure, but it would be good to understand what else the money would be spent on - arbitrary cutoffs are useful for payers (who need to know costs), but not useful for others, who might like to know what the money would be spent on instead.

I think high costs are common with biologics because they're harder to make and are more difficult to make generics of, but Gilenya is a small molecule, as is thalidomide, and both rack up pretty big costs.

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10. Hap on July 21, 2011 12:19 PM writes...

At some point, though, drug costs are a zero-sum game (certainly for insurance companies, who aren't going to relinquish profits for my gain, and probably for governments who don't or won't have the money or, like insurance companies, want to spend it on something else) - money spent for cancer drugs or MS can't be used for something else. I don't want to die any sooner than anyone else, but if two or three people could live full (> 20 years) productive lives with the money spent on my extra two or three months, well, I don't know that that's good for any of us.

QALY is probably a helpful figure, but it would be good to understand what else the money would be spent on - arbitrary cutoffs are useful for payers (who need to know costs), but not useful for others, who might like to know what the money would be spent on instead.

I think high costs are common with biologics because they're harder to make and are more difficult to make generics of, but Gilenya is a small molecule, as is thalidomide, and both rack up pretty big costs.

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11. Kay on July 21, 2011 12:27 PM writes...

I took a look at the full article, and it seems like a large portion of the cost isn't the drug itself - it's the associated office visits, hospital admissions, home care, etc. If you live longer, you also have more general medical costs that aren't directly related to the drug itself. But it doesn't sound appealing to say, "if you die faster, it's cheaper."

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12. cynical1 on July 21, 2011 12:58 PM writes...

Facepalm! Leave it to the NMSS to fund such a heroically stupid study. What genius funded a study that anyone with a few brain cells knew would only deincentivize pharmaceutical research in the area and put a bright shiny light on the fact that the drugs above are all crap, very expensive crap? Duh! We all knew that guys! You're supposed to be funding studies that will help MS patients get better drugs not pointing out to the world that what they've come up with so far is pretty poor and maybe not worth paying for at all. Thanks guys. I have set extremely low expectations of that organization and they continue to live down to them. And I say this as the spouse of a MS patient and also a top ten fundraiser for the NMSS in my part of the state for the past ten years. I'm also a medicinal chemist with a whole lot of experience in the therapeutic area.

I wish the NMSS would just give the money directly to the patients rather than funding epically stupid "research" (and I use the word loosely) year after year after year. I could use some help paying for the $55,000 in (additional) handicap renovations currently being done on my house, all out of pocket. Maybe they should do a study on MY quality adjusted life years.

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13. Anonymous on July 21, 2011 1:02 PM writes...

80% of a persons health care cost comes in the last year, in a time over ever lower quality of life - where do you draw the line.
On average people contribute 150k to Medicare but take our >400k.

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14. Dylan on July 21, 2011 1:22 PM writes...

Just wanted to say thanks for the beautiful summary of the usual questions.

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15. NoDrugsNoJobs on July 21, 2011 1:44 PM writes...

#13 - I love that quote. People who are very sick are more likely to die within a year and they are also more likely to get treatment! People who have cancer are more likley to die in a year and they are more likely to get treatment! People who get in bad car wrecks are much more likely to die within a year and they need more treatment! If only we knew who was going to die and when, we might be able to save a whole bucket of money.

I reckon I'd probably prefer see us spending our money on health care then newer and better bombs. In fact, i think we need to make better use of the bombs we have now.

Permalink to Comment

16. Still Scared of Dinosaurs on July 21, 2011 2:05 PM writes...

I don't love QALYs (or any pharmacoeconomic analysis) but they can be useful for comparing therapies within the context of a single study. The question, though, is always "Compared to what?" In this case is the alternative going to be no treatment? You save on the drugs and associated costs but you still have the lost productivity, MD visits, hospitalizations, etc.

It's important to remember that people don't die of MS in the way they do of dangerous cancers. They tend to get more disabled in a way that gets increasingly costly over time. Delaying or denying treatment will save in the short run but whether any acceleration of other costs will ensue because of it is an important question.

Finally, all of these therapies have associated risks and many can have negative impacts on quality of life.

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17. dave w on July 21, 2011 2:18 PM writes...

Hmmm... so what's actually driving the high "cost per QALY" for the MS drugs? Is it an unusually high purchase price for the medication itself, or are they only marginally effective in relieving the condition to the extent of improving the patients' actual "quality of life"?

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18. Mike on July 21, 2011 2:37 PM writes...

I think Hap's comments (#8) really hit the nail on the head. There is only a certain percentage that we can spend on healthcare (in a sustainable way) and the question is less about each individual and more about the most efficient way to spend the limited resources.

It's a dicey issue and people are going to come away unsatisfied no matter what the final outcome is. I don't think it will be solved anytime soon.

Mike

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19. HelicalZz on July 21, 2011 3:14 PM writes...

QALY is one measure, but not the only consideration related to reimbursement. Incidence needs to be heavily factored in as well. Quite honestly, the collective 'we' can afford a high QALY cost for a low incidence disease without disrupting the system. But ... the higher the incidence the lower the QALY cost the system can sustain. What can be paid for MS could never be paid for Alzheimer's. While I have low confidence in some current MABs being evaluated, I have real fear that if they are effective, they will be the proverbial healthcare systems back breaking straws.

Zz

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20. dearieme on July 21, 2011 3:32 PM writes...

"I honestly have no idea what the end to these arguments might be": I do. Bankruptcy.

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21. Anonymous on July 21, 2011 4:46 PM writes...

This whole dilemma of sustainable costs, an adequate return on exceedingly high-risk investments, costly high capital demands, rationing of drugs to only those who can afford them, high end-of-life costs, real world limits on societal resources, ranking priorities for spending limited societal resources and an individuals' responsibilities for their own healthcare costs can be resolved by not inventing new medical technology or at least not introducing any new technology into the system that does not pay out more than it costs. For example, the FDA should not approve any treatment that does not significantly change the course of a disease. For anticancer drug that means that for a large percentage of cancer patients treated with a new drug, life will be extended decades not months, and their lives will be of high-quality. For MS patients, the disease progress would have stop or even reverse when treated with a new drug such that patient gains decades of a "normal" life. This was the case with vitamins, antibacterial drugs and HIV drugs all of which had profound outcomes for patient health, so it is possible. This should be how we deal with all disease and symptom treatments going forward. We cannot keep piling on ever more expensive clinical, safety and manufacturing requirements, demanding companion diagnostics, limiting markets to narrow focused populations and expect drug prices to go down or even stabilize. We need to do something drastic or this will never resolve. Rationing by not inventing solves the long-term cost problem and any resulting suffering is indiscriminate with respect to finances, access, class and age.

Now laying-off medicinal chemists and pharmacologists was a good first step. Moving drug discovery into federally funded universities is also very useful. The final step will be to terminate investor supported pharmaceutical commerce. Yeah, we may never see another new drug in our lifetime, but if we do, it will a step change in treatment and hopefully much less expensive for society.

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22. pharmadude on July 21, 2011 6:36 PM writes...

It makes me wince whenever I hear about huge chucks of cash being spent on extending someones life by a mere few months. Especially if that person is already middle aged or beyond. The amount of money being talked about could provide extra teachers for schools, after school programs, scholarships. I worked on a program related to FTY-720 (aka fingolimod)and would like to see it payoff. But if it doesn't it doesn't.

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23. HFM on July 23, 2011 9:38 AM writes...

@21: You're kidding, right? With the technology we have, we're already very good at keeping people alive - we can hook them up to an array of drips and machines, and stabilize them for the short term. But we can't fix what's killing them in the first place (metastatic cancer, systemic organ failure, what have you).

If you're going to die in a first-world hospital, you'll live longer all right, but it won't be pretty for anyone involved. I watched both my grandmothers go through this in the past few years...it was hard to watch them go that way. If I'd let a dog suffer as much as one of them did, I'd still be in jail. We're buying a few miserable months at the cost of hundreds of thousands of dollars, routinely. Meanwhile, my twenty-something housemates bring their rashes and scrapes to me, because they can't afford real health insurance and biologists are close enough to doctors. FFS. It ain't right.

It's hard to say that someone should have to suffer when there's an alternative available, especially when your motive is pure cost. However, there's only so much money to go around; the $800K that might have gone into MS drugs for 1 QALY could go into prevention programs, social work, heck even highway improvements, and come out with something better. And I say that as someone who would be poor enough to face the consequences.

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24. luysii on July 25, 2011 9:19 PM writes...

#23--" If you're going to die in a first-world hospital, you'll live longer all right, but it won't be pretty for anyone involved. I watched both my grandmothers go through this in the past few years...it was hard to watch them go that way."

Exactly, that's why my brother (another MD) and I made sure there were no medical heroics. They passed away peacefully at home with help from Hospice at ages 93 and 100. Forget the money, it simply isn't humane to put people through that.

However, in some cases the hand of the physician is forced, by a guilty family member who "wants everything done" to show how much they care. It happens more often than you might think.

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25. fred on August 2, 2011 10:47 PM writes...

Calculate the QALY of penicillin back in 1938. To high to make it worth using, right?

Medical progress is frequently incremental.

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