a parable…

Posted on Tuesday 29 March 2016

A month ago I ran across a MEDPAGE TODAY article about Vortioxetine [Brintellix®] and a hearing at the FDA [see indications… and more vortioxetine story…]. Brintellix® had been approved by the FDA for use in Major Depressive Disorder in September of 2014. I  had previously run across it through a review article in the Journal of Clinical Psychiatry in December 2014 – a ghost-written garbled affair with multiple KOL guest authors [see the recommendation?…]. It seems that the manufacturers [Takeda and Lundbeck] had been planning for years to try to get it approved for treating the Cognitive Dysfuntion in Major Depressive Disorder. That would be a new category for FDA Approval and would’ve become a strong selling point for this late-coming antidepressant. I found a recent article by Lisa Cosgrove et al who had done a case study of this particular drug and its approval process [Under the Influence: the Interplay among Industry, Publishing, and Drug Regulation] that contained the results of both published and unpublished trials showing that in comparator studies, Brintellix® had uniformly come up short. And I later read a blog on George Dawson’s Real Psychiatry [Vortioxetine] about it.

The FDA originally looked unfavorably on approving Brintellix® for Cognitive Dysfuntion in Major Depressive Disorder, seeing it as an example of pseudospecificity. But the manufacturers kept coming. In February 2015, there was a Workshop at the Institute of Medicine entitled Enabling Discovery, Development, and Translation of Treatments for Cognitive Dysfunction in Depression: A Workshop moderated by Tom Insel [NIMH] and Thomas Laughren [formerly FDA’s director of psychiatry products]. After the workshop, the FDA agreed to reconsider, but only in a public hearing with an independent advisory committee. That happened on Feb 3, 2016 [2016 Meeting Materials, Psychopharmacologic Drugs Advisory Committee]. It was an elaborate all day affair with presenters from all parties concerned. At the end of the day, the FDA Advisory Committee voted 8:2 in favor of recommending Approval. When I looked at the papers and the presentations, I disagreed. My reasons are cataloged in my second blog [more vortioxetine story…]:

  • The NIMH presentation was thoughtful and concluded that the psychometrics being used were not a valid sole proxy for a specific Cognitive Dysfuntion designation.
  • They had one positive study [FOCUS]. But the second study [CONNECT] didn’t replicate those results. It was reported as statistically significant, but in my opinion, the statistical analysis was flawed.
  • There’s nothing in my clinical experience or reading that suggests that the affective and cognitive symptoms in depression can be cleanly parsed in this way. Frankly, this felt like a commercially driven ploy.
So I bundled up my findings into a letter [essentially the two blog posts minus the 1boringoldman·isms] and set out to figure out where to send a letter to the FDA that might get read [much thanks to Drs. Lisa Cosgrove, Bernard Carroll, and Erick Turner for their pointers]. I started with the FDA Ombudsman [who knew there was one?]. It didn’t fit their sphere of operations, but they forwarded it to someone at the generic info@fda.gov who was extremely helpful – sending it on to the reviewers herself and updating me with an email confirming it had gotten there. I was impressed. So I googled the topic last night, as I had often over the last month, and Bingo!
PRNewswire
Mar 28, 2016, 20:02 ET

OSAKA, Japan and VALBY, Denmark, March 28, 2016 /PRNewswire/ — Takeda Pharmaceutical Company Limited [Takeda] and H. Lundbeck A/S [Lundbeck] today announced that the U.S. Food and Drug Administration [FDA] issued a complete response letter [CRL] for the supplemental new drug application [sNDA] to include new data in the clinical trials section of the U.S. label of Brintellix® [vortioxetine] for treating certain aspects of cognitive dysfunction in adults with major depressive disorder [MDD]. The FDA approved Brintellix on September 30, 2013 for the treatment of MDD in adults. The CRL does not apply to the use of Brintellix in MDD.

Takeda and Lundbeck are disappointed with the response given that the U.S. FDA Psychopharmacologic Drugs Advisory Committee [PDAC] voted 8 to 2 that Takeda and Lundbeck presented substantial evidence to support a claim of effectiveness for Brintellix in treating certain aspects of cognitive dysfunction in adults with MDD. However, the companies were pleased that FDA recognized the importance of cognitive dysfunction in MDD and view it as a legitimate target for drug development…
In case you don’t speak FDA-ese, a Complete Response Letter essentially means "No" [see Complete Response Letter Final Rule]. The proceedings are here.

I don’t nor will I ever know if my letter had anything to do with the FDA’s decision. Other than the average expectable "tree" narcissism ["if a tree falls in the forest and no one hears it, does it make a sound?], I’m not sure I care. I had already gotten my reward from hearing the echo. In another way, I’d actually feel even better if the decision came out this way totally based on the FDA’s own evaluation. Takeda and Lundbeck had gone all out and to a lot of expense trying for this indication [6 cognition-specific clinical trials, bringing a lot of big KOL guns to the IOM workshop and earlier hearing, etc]. I could only guess that the 8:2 vote for Approval was in response to their zeal and presentations. But it just wasn’t in the data, and that’s what the FDA is there to evaluate. So good on them. They did the right thing after all. As for my letter…

A Parable: Two years ago, my two closest friends died in the week after Easter, and so they are much in my mind. One was a photographer whose photographic chronicles of the poverty in the Mississippi Delta and in the Appalachian Mountains will never be forgotten. But as gifted as he was with his camera, he sure had his quirks. And he was never happier than when he was talking about the intrinsic evils of politics – any politics. He claimed to have never voted, and delighted in being asked why. "It’s a trick to make you think you matter," he would begin [showing his own Appalachian roots]. This dialog was part tongue in cheek, but part deeply felt. One day, we were all floating in our little lake on a hot summer day, and he started up with his usual exposition on politics. My wife [a prime candidate for an fMRI study to locate the political regions of the cerebral cortex] announced, "Al, you can’t talk about politics anymore. You’re on political restriction. If you’re not going to vote, you have no right to complain." We were surprised at her little speech, as we were all used to Al’s diatribes and knew they would pass shortly. He didn’t say much and we went back to whatever old friends do when they’re cooling off in a lake on a hot summer day. But after that, he started voting – I think every time it came up until he died. I don’t know for sure that those things were 1:1 related, but I’d bet good money they were…
  1.  
    March 29, 2016 | 4:23 PM
     

    Exactly (re voting). Until there’s a Nihilist party, not voting is not a valid political position.

    Congratulations at finding who to contact in the FDA and getting a respectful response from them.

  2.  
    James O'Brien, M.D.
    March 30, 2016 | 12:37 PM
     

    Actually not voting is perfectly rational especially if you’re in a state where 2/3 agree with you or disagree with you. You’re more likely to die in a car accident or get hit be a meteor than produce the deciding vote.

    https://en.wikipedia.org/wiki/Paradox_of_voting

    The oligarchs have figured out being connected through K St. is FAR FAR more important than voting.

  3.  
    March 30, 2016 | 12:43 PM
     

    Shucks. I thought my parable was about sitting on the sidelines complaining versus getting truly involved…

  4.  
    Donald Klein
    March 30, 2016 | 3:17 PM
     

    Colleagues
    It seem to me that given mass voting , any individual vote is always without practical effect , except in extraordinarily unlikely situations.
    Further if you think voting expresses some general will (as I did) see the mind bending discussion in Eilenberg’s book , unfortunately entitled, ” How not to be wrong”.
    That voting is a trick to make you think you matter is one hypothesis.
    Another, it is a scheme to assure the population that social rivalries ,discriminations, major differences in income/effort ratios,etc can be attended to
    rationally, and such concerns will eventually be baseless.
    I don’t know of any data that can put such hypotheses to the test and history is debatable.
    A third statement, that is usually made as an observation , is that a democratic republic is the worst form of government , except for all the others.
    Hypothetically voting amounts to an opportunity to affirm that conviction.
    No data and is the % voting declining? .
    In any case what does it mean to be truly involved? And I don’t get a tight connection between not voting and having given up your right to complain,make speeches, write blogs etc. Please clarify.
    Eilenberg’s discussion of the Bush-Gore-Supreme Court fracas
    suggests an improvement in our voting system, that is hardly ever discussed.That’s probably for a different blog.

  5.  
    April 2, 2016 | 5:27 PM
     

    If any vote doesn’t count, there’s no point to any political campaign, is there?

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