The real reason Americans are missing out on life-saving drugs

May 9, 2013
May 9, 2013

The oft-repeated claim that the Federal Drug Administration (FDA) doesn’t approve drugs quickly enough is simply false. The actual approval process usually only takes six to 12 months. So why does the US lag behind Europe in the number of new drugs it brings to market?

Look no further than the clinical trials required by the FDA before the drug even reaches the government agency’s review stage. Once a drug has been developed, it can take six to ten years for three phases of clinical trials to be performed.

When the AIDS epidemic hit the US in the late ’80s, activists urged the FDA to speed up this part of the process. The result: a fast-tracking of life-saving drugs made available to the public. Under an accelerated approval process, certain drug candidates that have proven to have significant enough effects can skip phases of FDA-required clinical trials. But the fast-tracking only applies to drugs that meet certain criteria, like those needed to treat serious diseases such as AIDS or cancer.

Last night, in a debate sponsored by Intelligence Squared, Peter Huber, a senior fellow at the Manhattan Institute, and Dr. Scott Gottlieb, a resident fellow at the American Enterprise Institute, argued that the FDA’s caution is hazardous to public health.

Huber advocates for implementing Bayesian trials, where data is interpreted based on statistical probabilities. These trials remain open-ended once a drug comes to market, allowing doctors to continue analyzing performance.

According to Gottlieb, “The biggest problem is that drugs aren’t getting developed.” Pharmaceutical companies would often rather forego the sometimes decade-long process of drug trials required by the FDA, especially when certain conditions may not yield changes for decades. An example: polycystic kidney disease, where cysts in the kidneys can eventually lead to renal failure. According to FDA standards, to determine the efficacy of a drug for this disease, the trial must examine how many patients on the drug go into renal failure versus those on a placebo. The problem is that it takes 30 years for the late stage of the disease to develop, yet the FDA insists on that clinical outcome. Gottlieb says that simply looking at cyst reduction would be a logical way of determining whether a drug mitigates this condition. But that’s not happening, and drugmakers have stopped developing treatments for the condition.

A 2012 White House report on innovation in drug discovery echoed Huber and Gottlieb about the need to accelerate the process of bringing drugs to market and remedy the inefficiency of clinical trials:

Beyond organizational inefficiency, clinical trials are also expensive because they often must be extremely  large or long to provide sufficient evidence about efficacy. But in fact, it is increasingly possible to obtain  clear answers with many fewer patients and with less time….Bayesian statistical designs potentially allow for smaller trials with patients receiving, on average, better treatments. These and other modern statistical designs can improve on current protocols, which have only a very limited ability to explore multiple factors simultaneously. Such fac­tors importantly include individual patient responses to a drug, the effects of simultaneous multiple treatment interventions, and the diversity of biomarkers and disease sub­types.

Of course, there is always a cost-benefit analysis with any medicine. The FDA has to protect patients from harmful and sometimes fatal side effects. So what should consumers do when faced with the risk of taking a new drug? Says Gottlieb, “If you take a drug, you make sure you really need it.”

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