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FDA Says Drug Doesn’t Work – Then Approves It Anyway!

May 25, 2012 by admin in Politics with 3 Comments

Vyndaqel—a drug that has been shown not to limit a disease’s progression and has not been compared with other drugs that treat the same symptoms—was just approved by the FDA. I think that most readers can figure out why it happened.

FDA, Janus for Modern Times

by Heidi Stevenson

The FDA has replaced the two-faced god, Janus, in these modern times. In spite of finding that Pfizer had been unable to demonstrate that their new drug, tafamidis meglumine (Vyndaqel), works, they approved it anyway!

By a vote of 13 to 4, the FDA’s Peripheral and Central Nervous Systems Drug Advisory Committee, acknowledged that Vyndaqel doesn’t work. Then, they again voted, by the same margin of 13 to 4, to approve it.

Apparently, lack of another drug to treat a rare disorder justifies approving one that simply doesn’t work. By the FDA’s thinking,  filling a gap with empty space has make sense.

Bizarre and Twisted Justification

The disorder in question is terrible. Familial amyloid polyneuropathy (FAP) is inherited and results in progressive deterioration of nerve function resulting in a wide array of miserable symptoms, including numbness and pain, and loss of function of limbs and organs.

Inadequate Trial

The justification used for approving Vyndaqel is that, though it failed to slow FAP’s progression, it does ease some symptoms. However, there are significant reasons to doubt the validity of these results:

  • Symptoms were not the primary endpoint of the study, but only secondary. The study, therefore, was not designed to provide accurate information about them.
  • The study was of the intention-to-treat variety, which means that the results for people who do not continue the treatment for any reason, including dropping out of the study, are included as if they had, thus skewing the results.
  • About 20% of the subjects dropped out of the study.
FDA Requirement to Compare with Other Drugs Not Met

Not only are the results questionable, but the testing process did not include a primary FDA requirement. Vyndaqel was not compared with any existing drugs to see if it provides better results. The FDA’s own rules require such a trial comparison before granting approval. In their industry guidance for the drug approval process, Section I(iii)(a) states:

There is no comparable or satisfactory alternative drug or other therapy available to treat that stage of the disease in the intended patient population.

No other drug was used for comparison, so a primary rule— written by the FDA itself—was broken in the rush to approve a drug that they admit wasn’t beneficial!

It makes no difference that a drug can ease a symptom unless it’s either the only drug that can do so or is compared with others to see if it does better. Without such a comparison, we have no way of knowing if the drug is worthwhile.

Why the Rush to Approval?

A factor that makes Vindaqel unique is that it’s classed as an orphan drug. Such drugs are expected to have limited use because there is limited application for them. FAP is a rare disorder, so Vindaqel was given a fast-track status under the Orphan Drug laws. Orphan Drugs are, therefore, given some leeway in the approval process. However, if there are other drugs that accomplish the same thing, then it still must be compared with existing drugs.

Apparently, Pfizer doesn’t expect Vindaqel to outperform any existing drugs for neurological symptoms, so they didn’t try to gain approval through that method. Instead, they asked for Orphan Drug status as a treatment for FAP. That gives them an easy ride to approval—and must have come as quite a surprise.

By the way, once a drug is on the market, it can be prescribed by doctors for anything they wish. Thus, you can be fairly sure that there will be some sort of pseudo science that gives the impression of Vyndaqel’s ability to benefit this disorder or that disorder. Once an Orphan Drug, there’s no law keeping it in that status.

How the sleight-of-hand that got approval in spite of not meeting the basic qualifications for approval would be an interesting tale—but without the view of a fly on the wall, it’s unlikely we’ll ever hear it.

The simple fact, though, is that Vyndaqel—a drug that has been shown not to limit the progression of FAP and has not been compared with other drugs that treat the same symptoms—was just approved by the FDA. I think that most readers can figure out why it happened.

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  • Wuzzi

    The only drug to treat my illness, interstitial cystitis, is equally dubious, with outcomes barely better than placebo and side effects that include hair loss, vomiting and bloody diarrhea.   If you refuse to take the drug as a patient you are “uncompliant” and not willing to get well (even though there was only a 6% greater improvement vs. placebo at one year and that was improvement, not back to normal).  It is called Elmiron.

    The makers are now running clinical trials where they TRIPLE THE DOSAGE for patients who don’t respond at the safe dosage (the one that causes hair loss, vomiting, bloody diarrhea, oh and liver issues…).  

    When it is an “orphan” illness, the FDA will approve almost anything they can get, are allowed to approve more dangerous drugs (that is how Remicade was approved but given a black box warning when they figured out it cured cancer; it has a “new and novel therapy approach” for an illness with “few options”).  Better yet, our lovely President (of the US) has told the NIH that they must focus on prevention instead of education or research, so funding for finding cures for our illness and letting the greater public know more about it has been cut drastically so people can be warned that smoking can cause damage to their bodies in more frightening advertisements because the other warnings are ignored by a percentage of the population (most likely the same ones that will continue to smoke anyhow).  

    They will also advertise that we should exercise more and eat less, but not tell us to avoid HFCS (because that would affect the corn growers), not tell us to avoid GMOs (because that would affect Monsanto), etc., because the corporations that now run our government (because they make tons of money by outsourcing jobs to 3rd world countries for below minimum wage where pollution standards are either much looser or don’t even exist and rarely pay a living wage to anyone but middle and upper management) take all the money they have made raiding pension funds and outsourcing jobs that used to support Americans and use it to get representatives and presidents elected and then those representatives “own” them.  

  • Seth

    Vyndaqel—a
    drug that has been shown not to limit a disease’s progression and has
    not been compared with other drugs that treat the same symptoms—was just
    approved by the FDA. I think that most readers can figure out why it
    happened. TSK TSK TSKKKKKKKK. Alll you non-naturalists
    will suffer extensively and have more money comin’ out of your
    wallets….TSKK TSKK TSKKK. SMFH. Anyways….

  • Miguel

    FAP kills after 10 years of suffering or you can make a liver transplant and submit to 20% chances of not surviving. Vyndaqel is the only hope for a lot of people.

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