FDA Versus Patients

This year, the Food and Drug Administration rejected the only medicine capable of treating the rare and fatal lung disease known as idiopathic pulmonary fibrosis. Pirfenidone, which has been available in Japan since 2008 and was just approved in Europe, was spurned by the FDA because the drug only showed efficacy in a single big trial—not the two large studies the FDA now requires. The decision to ban the drug is one of a rash of recent decisions that shows the FDA is making it more and more difficult for promising drugs to reach severely ill patients…

It wasn’t supposed to be this way. In 1997, Congress passed the FDA Modernization Act, which gave the FDA broad discretion to reduce the quantity and rigor of clinical data needed to approve drugs targeting grave illnesses. The purpose of the law was to save lives by reducing the cost and time needed to launch such medicines.

But the FDA has steadily disregarded many of the law’s provisions. Longer, larger trials that require drug makers to evaluate “hard” endpoints (like how long a cancer patient lives) rather than “surrogate” endpoints (like a drug’s ability to shrink tumors) give FDA reviewers more statistical confidence. Reviewers prefer these drawn-out trials because they insulate the FDA from critics who say that it isn’t focused enough on safety. But bigger trials increase the time needed to develop a drug, keeping it out of the hands of patients.

Read more by Scott Gottlieb writing in The Wall Street Journal.

Comments (6)

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  1. Ken says:

    Good editorial. More evidence that the FDA is not on the side of patients.

  2. Devon Herrick says:

    The FDA has been rejecting a substantial number of drug applications this year. For instance, one article I read explained the reason why it is so difficult to get a drug approved to treat obesity: because no drug can be as safe as diet and exercise.

  3. Vicki says:

    It is really hard to imagine what the rationale could be for denying drugs that even might work to terminally ill patients.

  4. Joe S. says:

    Gottlieb is always good.

  5. Erik says:

    As a cancer survivor I would say that I don’t want a drug that shows “promise” I want a treatment that is tried and true. Sounds like the FDA does not trust Big Pharma. I say they are on the right track.

  6. Linda Gorman says:

    Erik, how are you going to get to a “tried and true” treatment if you haven’t first explored drugs that show promise? Since the biology isn’t well known, a lot of drug treatment is trial and error.