The Public Policy

Analysis Paralysis

FDA policies can be painful for patients.

By 8.17.05

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When you have an incurable disease, as my wife does, you are forced to become informed about your options. And sometimes what your doctor would like to prescribe is simply not available here in the United States, although it may be in wide use around the globe.

That was the case with my wife when the drug she was taking to halt the progress of multiple sclerosis ceased to be effective. Her doctor wanted her to take the new drug, Rebif, that was in wide use in Europe and Canada, but was then unavailable here in the U.S. Because time was critical in stopping the advance of MS before irreversible damage was done, we took action. After consulting with Kathi's doctor and our lawyer, we imported the drug from Canada until it was eventually approved in March of 2003. We both believe the progress of the disease would have been drastic had she not had access to Rebif during this time.

The problem my wife and others had in gaining access to Rebif highlights the problem faced today by sufferers of many incurable diseases. Fortunately, drug companies have poured hundreds of millions of dollars into research to find new drugs which promise to halt or delay the progress of diseases such as MS, AIDS, arthritis, Alzheimer's, and the like. But unfortunately, the U.S. lags behind the rest of the modern world in making many of these drugs available to those suffering from these diseases.

Not only did my wife face this problem with the treatment of her disease, but more recently, my mother-in-law faced similar difficulties when the drug she was taking for arthritis was pulled from the market.

The fact is that those who suffer from incurable diseases should have the right to decide on the basis of their own individual situation if the risk of taking a new drug (that has successfully completed clinical trials and is in wide use elsewhere) should be taken. Someone who has MS or any other incurable disease must have the right to conduct their own risk analysis because it is his life that is on the line. While politicians squabble and the media fret, time is running out for those who have MS, AIDS, Alzheimer's and similar diseases.

Unfortunately, a patient at the mercy of a disease often suffers additionally from decisions made at the Food and Drug Administration (FDA).

Falling victim to bad press after the removal of COX-2 inhibitors from the market when serious side effects were reported, the FDA has grown a thin skin with regard to any press reports of complications in the use of newer drugs. What the FDA seems to ignore in its response to these public reports is the due diligence, awareness of risk and subsequent informed choices made by patients with no other hope of recovery.

Who ends up safe, and who ends up sorry?

The FDA often seems more concerned with weathering political pressure and scrutiny from a headline-obsessed media than practicing kind and prudent regulation. While the agency must remove products proved unsafe in wide use, the real tragedy is what happens when FDA reacts too quickly, and beneficial treatments end up being removed from the market without solid cause.

Such was the case with Lotronex, a treatment for Irritable Bowel Syndrome, and may also be the case with Tysabri, the only new MS treatment available in the last eight years. Manufacturers of both voluntarily pulled their product from the market in response to FDA concerns. Lotronex ultimately was allowed back on the market, albeit with additional restrictions. Tysabri currently is undergoing a safety review, and recently published data that confirm that the drug works hopefully will help FDA put it back on the market too.

MS sufferers, including my wife, have fallen victim to the culture of "analysis paralysis." Federal regulators, fearing legal action and bad press, have increasingly taken action absent sufficient cause, which limits patient access to useful and needed drugs. It forgets that this is a patient population among the most informed in the world, and in the most desperate need of new treatment options. The FDA should not confuse activity with accomplishment. Analysis paralysis has had a profound negative impact on the entire American health care system.

Although my wife was not taking Tysabri, her current medication may well become less effective over time. I believe her limited options should remain as wide as possible.

Prior to yanking widely effective drugs such as Lotronex and Tysabri off the market, the FDA must consider all factors that lead to complications, such as mixing medications or dosage deviations often made by individuals desperate for relief. Genetics also play a role in individual complications; some people can't even tolerate aspirin. Most important, consideration must be given as to whether removing a drug from the market is worse for the larger cohort of patients than its actual risk.

Patient access and informed consent are cornerstones in the treatment of serious and debilitating conditions. When the FDA removes a drug based on a minimal number of unfortunate but reasonably anticipated adverse reactions, they remove what may be the most important choice of a lifetime away from those who truly should be making it.

Nothing in life is certain -- except the results of untreated disease.

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About the Author

Bruce Eberle is president of the McLean, Virginia-based Eberle Communications Group, Inc.