FDA: Lapdog or Listener?

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Sociology of Health and Illness sociologist John Abraham (photo credit: eddiemcfish 2008)

BibTeX

@Manual{stream2011-55,
    title = {FDA: Lapdog or Listener?},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2011,
    month = feb,
    day = 27,
    url = {http://www.translationalethics.com/2011/02/27/fda-lapdog-or-listener/}
}

MLA

Jonathan Kimmelman. "FDA: Lapdog or Listener?" Web blog post. STREAM research. 27 Feb 2011. Web. 20 Nov 2017. <http://www.translationalethics.com/2011/02/27/fda-lapdog-or-listener/>

APA

Jonathan Kimmelman. (2011, Feb 27). FDA: Lapdog or Listener? [Web log post]. Retrieved from http://www.translationalethics.com/2011/02/27/fda-lapdog-or-listener/


The Need for Speed: GAO Reports on Accelerated Approval

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Several blog posts ago, I wrote about the policy of accelerated approval (briefly, a mechanism whereby new drugs can be approved for sale by the FDA before definitive evidence of efficacy and safety are available). In that post, I reported on a recent paper where the authors claimed that, all things considered, accelerated approval enabled patients to get quicker access to life saving drugs without major adverse impacts on patient safety.


Last week, the Government Accounting Office issued a report on the subject that took a less favorable view of the program. Rules require that companies receiving accelerated approval for new drugs complete post-marketing studies confirming their efficacy. The GAO investigated the frequency with which companies fail to submit post-marketing trial data. They found that over a third of FDA-required post-marketing studies aimed at confirming efficacy had not yet been completed. Many of these studies might be incomplete because accelerated approval was only recently granted, and it can take as long as five years to complete requested studies. Disturbingly, however, the report found that a quarter of these studies had been incomplete for over five years; other studies have been completed but not yet reviewed by the agency. The figures are worse for other types of post-marketing studies requested by the agency.

The “poster boy” drug singled out in the GAO report is the hypertension drug Proamatine, which earned Shire Pharmaceuticals $257M since it was approved under accelerated approval 13 years ago. Apparently, the drug has not been subject to adequate confirmatory testing in all this time, though FDA has issued warning letters to the company over its promotion practices.

The report saves its criticism for the FDA, which it says has not reviewed sponsors’ submissions in a timely manner, does not adequately monitor progress of post-marketing studies, and has neither specified conditions under which it would exercise its authority to withdraw drugs from market, nor has it ever exercised its authority to do so. But isn’t some criticism also warranted for companies exploiting FDA’s deficiencies? (photo credit: lindsay kay photography 2009)

BibTeX

@Manual{stream2009-81,
    title = {The Need for Speed: GAO Reports on Accelerated Approval},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2009,
    month = nov,
    day = 2,
    url = {http://www.translationalethics.com/2009/11/02/the-need-for-speed-gao-reports-on-accelerated-approval/}
}

MLA

Jonathan Kimmelman. "The Need for Speed: GAO Reports on Accelerated Approval" Web blog post. STREAM research. 02 Nov 2009. Web. 20 Nov 2017. <http://www.translationalethics.com/2009/11/02/the-need-for-speed-gao-reports-on-accelerated-approval/>

APA

Jonathan Kimmelman. (2009, Nov 02). The Need for Speed: GAO Reports on Accelerated Approval [Web log post]. Retrieved from http://www.translationalethics.com/2009/11/02/the-need-for-speed-gao-reports-on-accelerated-approval/


Accelerated Approval: Safe at Any Speed?

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Drug regulatory authorities like the FDA have a mandate to protect public health by requiring and evaluating evidence of safety and efficacy before licensing new drugs for commercial sale. But for decades now, patient advocates have argued that FDA bureaucracy kills by keeping promising drugs from the IV’s of terminal patients. In response to these criticisms, FDA and others have created new pathways for drug approval whereby drugs can be partially approved for sale on the basis of smaller, Phase 2 trials using surrogate endpoints (tumor shrinkage) instead of survival– provided drug companies confirm efficacy in subsequent trials.


This pathway, called “accelerated approval,” is controversial because it allows companies to sell drugs whose efficacy and safety is not yet well established. True- the companies are obliged to run confirmatory studies, but a) how will confirmatory trials enroll enough subjects if patients know they might be randomized to standard, ineffective drugs, and they can get the drug outside a clinical trial? b) drug companies will not have sufficient incentive to run confirmatory studies once their drug is provisionally approved. c) drug companies stand to make lots of money selling unproven drugs to desperate patients in search of a cure.

In the most recent issue of Journal of Clinical Oncology, Elizabeth Richey and coauthors put these concerns to the test in an analysis “Accelerated Approval of Cancer Drugs: Improved Access to Therapeutic Breakthroughs or Early Release of Unsafe and Ineffective Drugs.” They find that:

• a very large percentage of new cancer drugs are initially approved under “accelerated approval.” (37% approvals between 1995 and 2008)

• 63% of drugs receiving accelerated approval have their clinical benefit confirmed in subsequent studies

• drugs involving very rare cancers are often not subjected to confirmatory testing (42%); drugs for more common cancers are tested in confirmatory studies typically (71%– though the percentage I calculated from their figures is actually higher- 86%)

• drugs receiving accelerated approval are twice as likely to receive black box warnings compared with drugs approved by the standard mechanism (21% vs. 10%)

• about half of non-orphan drugs approved under accelerated approval (47%) become first line treatment regimens in the National Comprehensive Cancer Network.

The authors see the glass half full on accelerated approval: to the title question of their article, the authors answer “Improved Access to Therapeutic Breakthroughs.” (photo credit: Marxpix 2008)

BibTeX

@Manual{stream2009-85,
    title = {Accelerated Approval: Safe at Any Speed?},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2009,
    month = sep,
    day = 9,
    url = {http://www.translationalethics.com/2009/09/09/accelerated-approval-safe-at-any-speed/}
}

MLA

Jonathan Kimmelman. "Accelerated Approval: Safe at Any Speed?" Web blog post. STREAM research. 09 Sep 2009. Web. 20 Nov 2017. <http://www.translationalethics.com/2009/09/09/accelerated-approval-safe-at-any-speed/>

APA

Jonathan Kimmelman. (2009, Sep 09). Accelerated Approval: Safe at Any Speed? [Web log post]. Retrieved from http://www.translationalethics.com/2009/09/09/accelerated-approval-safe-at-any-speed/


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