Academic journal article Policy Review

The FDA's Risky Risk-Aversion

Academic journal article Policy Review

The FDA's Risky Risk-Aversion

Article excerpt

DURING THE PAST half-century, the stunning pace of scientific progress has yielded powerful tools for pharmaceutical development, including rational drug design, combinatorial chemistry, gene-splicing, DNA sequencing, gene therapy, and genomics. These technologies have wrought a creative revolution fueled by massive public- and private-sector investment, enabling drug and biotechnology companies to produce innovative life-saving and life-enhancing medicines. Since 1950, new vaccines have virtually eliminated measles, rubella, poliomyelitis, mumps, and diphtheria from the United States and other industrialized countries. Because of new antibiotics, the child who once would have died from bacterial meningitis or a ruptured appendix now survives. Because of genetically engineered alfa interferon, the 50-year-old with a rare type of leukemia can be successfully treated. Thanks to new drugs based on monoclonal antibodies, cancer is now increasingly considered a chronic disease rather than a death sentence. And to patients with all kinds of potentially crippling health problems--from diabetes to multiple sclerosis to congestive heart failure--drugs can offer the gift of an all-but-normal daily life. The list of diseases and conditions that can be conquered or ameliorated by new drug therapies is long and growing.

Drugs often improve the span and quality of life in a remarkably cost-effective way, a fact of crucial significance not only to the individual patient but also to society as a whole: The responsible use of drug therapies lowers the total cost of health care. A study done by three leading health economists for the National Bureau of Economic Research found, for example, that the overall cost of therapy for heart attacks and depression--both of which are commonly treated with drugs--actually declined by an average of I percent each year from 1984 through 1991. Similarly, the costs of treatment per episode of major depression fell by 25 percent from 1991 to 1995, and studies of the impacts of a thrombolytic drug in stroke patients (1) and a new drug for migraine headache show that these treatments, too, are highly cost-effective. (2)

Another sign of progress is that in general new drugs confer an advantage over older ones in reducing mortality. In a study of patients who took drugs between January and June 2000, those who took newer medications were less likely to die by the end of 2002. The estimated mortality rates were directly related to time that had elapsed since approval of the drugs: For pre-1970 drugs, the estimated mortality rate was 4.4 percent, while the mortality rates for drugs approved during the 1970s, 1980s, and 1990s were 3.6 percent, 3.0 percent, and 2.5 percent, respectively. (3) Not surprisingly, perhaps, drugs are getting better all the time.

The same cannot be said about the regulatory climate, however. The potential of pharmaceutical development is profoundly affected by government interventions, particularly regulation. In this article, we focus on regulation in the United States, partly because the policies, procedures, and decisions of the U.S. Food and Drug Administration (FDA) are widely regarded as the "gold standard" (which means little more than they are the most stringent and risk-averse in the world) and because the United States is the leader in both the development of new drugs and in volume of sales.

Government giveth, taketh away

GOVERNMENTAL INFLUENCES ON drug development can be positive. In the United States, the research funded by the federal government (primarily the National Institutes of Health [NIH] but also the National Science Foundation [NSF] and several others) that attempts to better understand fundamental processes and to establish "proof of concept" often provides the scientific substrate that is essential to downstream product development. For example, recombinant DNA technology, which is now ubiquitous in both basic research and drug discovery, was the product of synergistic advances in several areas of government-funded research, including enzymology, microbial genetics, and physiology. …

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