Is It Safe?
|Is It Safe?
The Journal of Life Sciences
By peter pitts
Safety has become deleterious to the public health.
In the wake of genuine safety concerns, high-profile stories on counterfeiting and the successful rollout of the Medicare Part D drug benefit, the allure of foreign drug re-importation has lost much of its political allure and momentum. Most of the elected officials calling for the legalization of foreign drugs have since abandoned their incautious and dotty schemes. In their place comes the call of safety.
After all, who could be against safety? Safe is good, unsafe is bad, right? Well, not exactly. As Mark Goldberger, director of the U.S. Food and Drug Administration’s Office of Antimicrobial Products said, “It’s more complex than it seems at first glance.”
Nevertheless, it sure plays in Peoria, or perhaps, more appropriately, in Des Moines. A little harmless politicking? Hardly. Just ask the people who no longer have access to the medicines they need, or those who will suffer needlessly because of a dearth of new medicines in the pipeline.
What does safe mean, anyway? All drugs have risks as well as benefits. More often than not, the more serious the disease, the more serious the risks associated with the treatment. Consider multiple sclerosis and the new drug to treat it, Tysabri.
In February 2005, Biogen and Elan were pressured to suspend sales of Tysabri after two patients taking the drug died from the rare neurological disease progressive multifocal leukoencephalopathy, or PML. Thousands of patients with MS?taking a page from HIV/AIDS activists?descended on the FDA hearings on the matter en masse. Fourteen months later, in June 2006, Tysabri was allowed back on the market, albeit with more stringent labeling. Subsequently, an exhaustive study of all patients taking the drug found no new cases of PML. (Overall, there is a 0.1 percent risk of contracting PML if you use Tysabri. That’s half the risk of dying from aspirin. Meanwhile, patients experience about 53,000 MS relapses yearly and 5 percent of relapses cripple or kill sufferers. The point is that patients in pain?even when they are fully informed?are ready to accept risk.
Patient advocates claim that regulators too often ignore what patients face when a medicine is not available. Certainly, risks cannot be ignored, but the existing FDA approval process makes all of the information about a drug’s benefits and risks available in most cases. Further testing does little to expand on what is already known through initial certification and informed practice.
Under some legislation, even legitimate drugs?drugs that have successfully passed through the complicated, expensive and exhausting FDA obstacle course?will no longer be given the benefit of the doubt. If a drug raises the slightest concern?say, an individual or two has some extreme adverse reaction?a new safety politburo could yank the drug from the market or demand further testing. In such decisions, the thousands or even millions of patients who are deriving real benefit from the drug will have little say. A new layer of drug czars will be given nearly absolute power to shut down otherwise successful programs.
On its own initiative, the FDA already has the power to pull effective drugs from the market and has done so. Such power would be broadened and manufacturers forced to pick up the costs of postmarket monitoring and, when called for, extensive additional safety studies.
In the past, the FDA has acted aggressively and often with good cause. Fen-phen was recalled in September 1997 because it was legitimately linked to heart-valve deterioration and pulmonary hypertension. Baycol was recalled in August 2001 when cases of fatal rhabdomyolysis were reported in association with its use significantly more frequently than with other approved statins. Vioxx was withdrawn from the market in January 2006?but Vioxx had well-documented benefits for thousands of arthritis sufferers. No drug is 100 percent safe for all patients all the time.
Are there questions concerning safety after approval? Of course. And pharmaceutical companies themselves have a huge interest in monitoring on-label?and off-label?events. But hasty measures that allow shut-offs, grabbing headlines that demonize the industry more often than not have the effect of impeding new research. Consider the precautionary principle: that the introduction of a new product or process whose ultimate effects are disputed or unknown should be resisted. Prudent? No, puerile. And the unintended consequences are fatal.
Our system of drug regulation involves a careful balancing of benefits and risks based on the best possible scientific information that can be discovered about a new drug’s safety profile. Any reform should be careful. And it should not come at the cost of doing harm to public health by slowing down the availability of new and better medicines.
Our elected officials should remain mindful of these concerns when they consider how best to continue to provide the resources and oversight that make our drug approval system the world’s gold standard.
Peter J. Pitts is president of the Center for Medicine in the Public Interest (http://cmpi.org//) and a former FDA associate commissioner.
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