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posted by martyb on Wednesday October 23 2019, @03:01AM   Printer-friendly [Skip to comment(s)]
from the I-said-what? dept.

In an extremely rare reversal, the biotech company Biogen said that it will submit the experimental amyloid beta targeting drug aducanumab—which previously had its drug trial stopped as futile—to the Food and Drug Administration for approval.

The company said a “new analysis of a larger dataset” showed that the drug, aducanumab, reduced clinical decline in patients with early Alzheimer’s disease on multiple measures of the drug’s effectiveness. That directly contradicts a decision in March to halt studies of the therapy based on the recommendations of an independent monitoring board that was charged with protecting patients in the study.

The reversal came about because the decision to halt the study was made based on an early part of the dataset where the dosage was reduced in an effort to avoid a potential side effect. When results on patients exposed to higher doses in later portions of the study was available and factored in the results turned significant.

Biogen said that it conducted a new analysis in consultation with the FDA of a larger data set from the discontinued studies. The new analysis includes additional data that became available after the previous analysis showed the studies were “futile” — that it had no chance of succeeding. Biogen said that the new data show aducanumab is “pharmacologically and clinically active” and that it reduced patients’ clinical decline based on the results of a survey called Clinical Dementia Rating-Sum of Boxes (CDR-SB), which was the main goal of both studies.

After praising the announcement as "a testament to Biogen’s steadfast determination to follow the science and do the right thing for patients,”

Michel Vounatsos, Biogen’s chief executive, said in a statement. “We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid beta.”

There is still discussion going on and analysts are examining and questioning the results, but with tens of millions affected by the disease worldwide, there is now a glimmer of hope.


Original Submission

Related Stories

FDA Approves New Treatment for Alzheimer's Disease 20 comments

The FDA has approved a new drug for Alzheimer's disease, while not a cure it is supposed to slow the decline. Even though data is not entirely positive or straight forward in its interpretation or that it will actually even work as thought.

But if you have it then you are probably desperate enough to try almost anything that claims to work, until you get to the price tag of $56,000 per year. That will probably make it out of reach for most people, it's doubtful if any insurance will cover something like this. Perhaps you can just forget to pay the bill, they might understand due to your condition.

Biogen CEO Michel Vounatsos [...][said] he thought the drug's price was "fair" but also vowed that the company would not hike its price for four years.

https://www.cnbc.com/2021/06/07/fda-approves-biogens-alzheimers-drug-the-first-new-therapy-for-the-disease-in-nearly-two-decades.html

Previously:
In Surprise Turnaround, Biogen to Submit Previously Failed Alzheimer Drug for Approval
Disputed Alzheimer's Study Links Decrease in Amyloid Levels to Reduction in Cognitive Decline
Positive Result in Mice as Alzheimer's Drug Trials Fail and Regulatory Barriers Are Rolled Back


Original Submission

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  • (Score: -1, Troll) by Anonymous Coward on Wednesday October 23 2019, @03:09AM

    by Anonymous Coward on Wednesday October 23 2019, @03:09AM (#910652)

    niggers I've sucked off.

  • (Score: 0) by Anonymous Coward on Wednesday October 23 2019, @03:39AM

    by Anonymous Coward on Wednesday October 23 2019, @03:39AM (#910660)

    Keep looking for dos fects.

  • (Score: 5, Insightful) by JoeMerchant on Wednesday October 23 2019, @10:20AM (5 children)

    by JoeMerchant (3937) on Wednesday October 23 2019, @10:20AM (#910731)

    Is this now a good way to short-cut the costs of clinical trials? Cut them off just after the data turns toward significant "to protect the subjects" and then, when the remaining data comes in declare efficacy?

    While I applaud the decision to keep a useful drug in development, I don't think the early decision to abort the trials should allow a release to market without completion of the full trial - it should merely allow a restart of trials, probably requiring a re-do of the aborted segment of the trial.

    --
    My karma ran over your dogma.
    • (Score: 0) by Anonymous Coward on Wednesday October 23 2019, @04:44PM (4 children)

      by Anonymous Coward on Wednesday October 23 2019, @04:44PM (#910863)

      No it's not some new economy--not everything has to validate your economic beliefs--it's just that you don't understand the reasoning for cutting off trials. The principle of the idea is sound. Whether or not it was done appropriately here, I don't know (I think the drug will ultimately fail but that's a different discussion than the one you're bringing up). Should we keep a trial going for a drug with preliminary results that make it impossible for the remaining data-set to reach statistical significance?

      If I started a trial for 100 people and I had already collected 99 samples--all with no outcomes--would it be a good use of anyone's resources or time to collect the 100th? Should I expose more people to possible long-term consequences? Anything to prevent your "new economy", let's do it, sure, just let us know!

      • (Score: 2, Insightful) by khallow on Wednesday October 23 2019, @11:30PM (1 child)

        by khallow (3766) Subscriber Badge on Wednesday October 23 2019, @11:30PM (#911037) Journal

        would it be a good use of anyone's resources or time to collect the 100th?

        Depends on why the 100th didn't get collected yet. If it didn't get collected because the drug caused a horrible outcome, then that's a particularly nasty sort of observation/confirmation bias.

        • (Score: 0) by Anonymous Coward on Thursday October 24 2019, @06:27AM

          by Anonymous Coward on Thursday October 24 2019, @06:27AM (#911127)

          I agree with you. However, I think if the decision is made on statistical grounds (not on results of a single case), there is no risk of confirmation bias. As long as the full algorithm and conditionals are laid out before and is followed without deviation, all should be good.

      • (Score: 2) by JoeMerchant on Thursday October 24 2019, @01:51AM (1 child)

        by JoeMerchant (3937) on Thursday October 24 2019, @01:51AM (#911078)

        it's just that you don't understand the reasoning for cutting off trials

        Reason for cutting off the trials is irrelevant. If full trials were required for initial approval, full trials should be required for approval after "very promising post analysis revealed a different outcome..."

        If the aborted trials were overkill, doing more than was required and they had passed the actual requirement threshold, then, sure. I don't think I know of any big corporations who make a habit of going to more expense than is necessary for regulatory requirements - except perhaps in the case of drug trials where multiples are started in secret, then aborted when they go the wrong way until one finally passes the threshold of significance.

        Should we keep a trial going for a drug with preliminary results that make it impossible for the remaining data-set to reach statistical significance?

        No, but clearly that's not what happened in the story.

        Anything to prevent your "new economy"

        I think you miss my point entirely, but being AC, you're not worth the time or effort to explain.

        --
        My karma ran over your dogma.
        • (Score: 0) by Anonymous Coward on Thursday October 24 2019, @06:30AM

          by Anonymous Coward on Thursday October 24 2019, @06:30AM (#911130)

          >I think you miss my point entirely, but being AC, you're not worth the time or effort to explain.
          Fuck you too then.

  • (Score: 2) by Thexalon on Wednesday October 23 2019, @01:09PM (1 child)

    by Thexalon (636) on Wednesday October 23 2019, @01:09PM (#910781)

    If it didn't pass FDA muster before, what makes them so certain it will pass this time? It couldn't be that they made sure certain people could profit from the approval even if the drug doesn't work, could it?

    --
    The inverse of "I told you so" is "Nobody could have predicted"
  • (Score: 1, Touché) by Anonymous Coward on Wednesday October 23 2019, @03:34PM

    by Anonymous Coward on Wednesday October 23 2019, @03:34PM (#910822)

    However a Hail Mary pass is consistent with "maximizing shareholders value" as long as "conscience" is a quaint word with inconceivable meaning.

  • (Score: 0) by Anonymous Coward on Wednesday October 23 2019, @07:49PM (1 child)

    by Anonymous Coward on Wednesday October 23 2019, @07:49PM (#910963)
    • (Score: 0) by Anonymous Coward on Wednesday October 23 2019, @07:52PM

      by Anonymous Coward on Wednesday October 23 2019, @07:52PM (#910966)

      the interesting part

      I’m going to ignore all the data on those slides about amyloid level in the brain via PET scans and suchlike. I honestly do not care; what’s important are the measures of dementia. And while the new larger EMERGE data do look like there’s a dose-responsive effect in the primary measurement (the CDR-SB scale) and three secondary dementia scores, the ENGAGE data actually show worse than placebo for CDR-SB at the higher dose (see slide 19 in Biogen’s presentation). They’re also worse than placebo in the MMSE scoring, but both the ADAS-Cog13 and the ADAS-ADL-MCI scales show apparent dose-responsive improvement. I have no idea why four different dementia rating systems should give such different results, and to a first approximation, no one else seems to have a good idea, either. So we’ve got the EMERGE/ENGAGE difference to explain (you’ve seen Biogen’s explanation, higher dosing later in the trial), and the variable results within the ENGAGE trial itself.

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