The Wall Street Journal-20080215-The Other Drug War- Rx for Helping the FDA Do Its Many Tasks

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The Other Drug War: Rx for Helping the FDA Do Its Many Tasks

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Regarding "The Real FDA Scandal" (Review & Outlook, Feb. 6): You are correct that "spend[ing] more money" alone will not remedy the FDA's most significant problems, which are: mismanagement, a corporate culture that is excessively risk-averse, and lack of accountability.

Repeatedly accused, unfairly, of not being sufficiently concerned about drug safety, regulators keep raising the bar for approval, especially for innovative, high-tech products. Moreover, a regulator not sufficiently comfortable with a new technology is a fearful regulator; and a fearful regulator doggedly applies old paradigms to novel situations, slows down every phase of clinical development, and requires unnecessary testing in order to provide himself with cover should anything go wrong.

How can regulators be made more accountable? Effective management must employ sticks and carrots judiciously, but bureaucrats are now punished only for approving products that turn out to be unsafe. They are consistently rewarded for over-regulating and for delaying product development. By introducing punishment for deficient performance -- including decisions that reflect excessive risk-aversion and timidity -- a powerful, independent agency ombudsman could induce regulators to act in the public interest. The ombudsman's office would need: (1) independence from the agency and its head; (2) access to independent expertise in relevant disciplines, including medicine, medicinal chemistry, molecular biology, pharmacology, and regulation; (3) the power to impose sanctions on FDA employees found to be responsible, individually or collectively, for flawed decisions or policies. This new entity could begin to alter the FDA's culture of risk-aversion and mistreatment of innovation.

Henry I. Miller, M.D.

The Hoover Institution

Stanford, Calif.

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Dr. Miller headed the FDA's Office of Biotechnology from 1989 to 1993.

The Science Board is correct in pointing out that the FDA's "evaluation methods . . . largely unchanged over the last half- century. . . " cannot keep pace with scientific advances. Perhaps even more distressing (and a reason that the FDA is assailed from both sides of the political aisle), these antiquated evaluation methods haven't kept pace with the evolving expectations of the public and the medical community. Patients and doctors deserve to know how the FDA makes decisions on potential therapies.

As one involved in drug development, I sympathize with the regulator's need to use fixed and objective standards to adjudicate "safe and effective." All too often, however, this rigidity produces nonsensical decisions like that about Junovan, in which the compound was rejected because of statistical, not clinical, criteria.

The Science Board is correct; the FDA does have an "urgent need for developing . . . new statistical methods." This is actually an area in which we can invoke 2008's mantra of "change." Although historically, "new statistical methods" usually begin an academic search for increased complexity, why not use the Science Board's recommendation to develop an appropriate, common sense statistical methodology that will support a more transparent and rapid approach to the development of new drugs?

Jonathan Seltzer, M.D.

Director, Clinical Research

Main Line Health Heart Center

Bala Cynwyd, Pa.

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Here is a simple yet bold solution to the FDA's resource problems: Eliminate the drug efficacy requirement. As you point out, many drugs are demonstrably effective, yet do not meet the 95% statistical threshold. Tragically, these include drugs such as Junovan, which achieved "only" a 94% level of statistical certainty.

Instead of "safe and effective," let the FDA determine merely that a drug is "safe," and let doctors, patients (especially terminally ill patients), and the market determine which safe drugs are used to treat various illnesses -- as we already do with drugs that are used off- label. The vast majority of the time and expense of the FDA approval process is spent on proving "efficacy" in Phase II and III trials.

True, some ineffective drugs, quack medicines, and nostrums will make it into the market (if they are safe), but this seems to be vastly outweighed by the benefit to terminally ill patients who would be freed from the morally reprehensible situation in which they are denied potentially life-saving medications that would otherwise be unavailable.

Seth Faler

Portland, Maine