Politics & Policy

Dear FDA, Step Aside So We Might Live

Patients suffering from Lou Gehrig’s disease shouldn’t have to plead with bureaucrats to try potentially lifesaving drugs.

In the next few days, the U.S. Food and Drug Administration (FDA) is expected to decide whether to accelerate approval of a new drug that might save the lives of patients with amyotrophic lateral sclerosis (ALS), Lou Gehrig’s disease. The manufacturer, Genervon Biopharmaceuticals, requested the approval of GM6 in February after finding improvements in patients during preclinical, Phase 1 and Phase 2A trials — with no negative side effects. If the FDA grants the request, patients would gain immediate access to the drug.

Efforts to speed up approval were triggered, in part, by ALS patient Anthony Carbajal, who a little over a year ago launched a petition on Change.org urging the FDA to allow him, his mother, and others — approximately 30,000 Americans suffer from ALS — to use the new drug. To date, more than 500,000 people have signed the petition.

In a free society, of course, dying patients shouldn’t have to petition bureaucrats for permission to take promising new drugs, so long as they understand there are risks involved.

There is no known cure for ALS. Over a period of two to five years after onset, ALS patients slowly lose the ability to speak, swallow, move, and (eventually) breathe. Riluzole, approved more than 20 years ago, is the only drug the FDA has approved to treat ALS, and it extends life expectancy by only a few months.

Currently, it takes an average of twelve years for a typical new drug to move from the laboratory to the pharmacy. Without accelerated approval, GM6 faces another two and a half years of Phase 3 trials. A decision to approve or reject would come six months to two years after that.

This onerous screening process admittedly keeps some unsafe drugs off the market. But it also prevents the sick and dying from using drugs that might save their lives.

Such tradeoffs aside, the bigger question we need to consider is this: Why should government bureaucrats determine the risks that dying patients can take with their own lives? As Carbajal writes: “Thousands of ALS patients and myself want to be able to try this drug. I do not have any other options to fight. I don’t want to be just another statistic.”

Economist David R. Henderson of the Graduate School of Business and Public Policy at the Naval Postgraduate School, Monterey, Calif., argues that the FDA might have some expertise when it comes to drug safety and efficacy, “but on the only issue that matters — your trade-offs between various risks — you are the expert, and the FDA’s scientists are rank amateurs.”

The FDA approval process is not only slow; it’s also very expensive, frequently costing $1 billion or more. As a result of these prohibitively high costs, some drugs, especially those intended to combat rare diseases, are never developed; the potential market is so small that a company would never recover its investment.

The alternative to the current FDA approval regime is not to return to the days of snake-oil salesmen, or death and birth defects due to unsafe drugs.

In other areas of daily life, we routinely rely on private third parties — such as the Underwriters Laboratories, the National Fire Protection Association, and the Good Housekeeping Seal, among others — to certify product quality and safety.

Even in the medical arena, where the FDA crowds out private certifiers, doctors routinely rely on medical journals and other industry sources to evaluate whether a drug, approved by FDA for one purpose, might also help someone with a different condition. Such “off label” prescriptions are common.

There is no reason we couldn’t use similar mechanisms to provide a reasonable degree of information about the safety of medical drugs. Each patient, in consultation with a physician, could weigh this information alongside his or her medical needs and risk preferences.

In the short term, the FDA should approve GM6 for the benefit of the 30,000 Americans suffering from ALS. In the long term, the FDA should get out of the approval process, for the benefit of the rest of us.

— Benjamin Powell is a senior fellow with the Independent Institute in Oakland, Calif., and director of the Free Market Institute at the Rawls College of Business at Texas Tech University, where he teaches economics.

Benjamin Powell, Ph.D., is a senior fellow at the Independent Institute, Oakland, Calif., the executive director of the Free Market Institute, and professor of economics at Texas Tech University’s Rawls College of Business.  


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