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The FDA Returns to Its Dark Ages

8:14 AM, Apr 14, 2014 • By MICHAEL ASTRUE
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Politics at its best brings people and groups together in unexpected ways. Although the Reagan administration responded sluggishly to the emergence of HIV in the 1980s, its last FDA commissioner, Frank Young, reached out to the very HIV activists who had for years made life miserable for him and other HHS officials.


Young’s outreach was an act of courage and vision for which he never received appropriate credit. Most of Young’s senior staff saw nothing in the HIV epidemic that required urgent reconsideration of the agency’s unreasonably burdensome rules and practices that had killed development of countless innovative therapies.

Young bravely overrode his staff and created a regulatory “fast track” for novel HIV therapies. His reform explicitly embraced the principle that the FDA would approve HIV drugs on the basis of “surrogate endpoints”—biological data that were likely, but not proven by past FDA standards, to measure clinical improvement. For HIV the FDA relied on T-cell counts as a measure of the strength of a patient’s immune system.

Young’s shift in policy quickly led to the approval of two HIV drugs; they were mediocre drugs by today’s standards, but they bought time for desperate patients until far superior drugs of the 1990s began to make HIV a substantially manageable disease. His initiative also gave patients an alternative to the technically illegal black-market stores called “buying clubs,” where one of their most popular products, ribavirin, was actually accelerating the death of the customers. It is worth noting that two of the start-up companies that developed HIV protease inhibitors in the 1990s after the creation of the fast track have gone on to become developers of very effective drugs for another scourge with similar biological mechanisms, hepatitis C.

In 1989 Vice President Quayle’s Competitiveness Council, led by future congressman David McIntosh, began work on a plan to extend the HIV approval rules to other life-threatening diseases. When McIntosh transmitted the draft 10-point plan to the FDA, the FDA truculently replied that it could only support minor proposed changes in one point. HHS Secretary Louis Sullivan did not want to aggravate friction with FDA commissioner David Kessler by overriding him.

I then received a quiet but emotional call from a senior White House official who expressed to me President Bush’s frustration at the FDA’s intransigence and asked me to try to broker a compromise. Accelerating breakthrough therapies was not just an abstract policy dispute for President Bush—he had lost a young daughter to a rare cancer.

Revisions responsive to some FDA concerns and meetings with senior FDA officials did not break the deadlock. I then went in to talk with Kessler, who initially defended the agency party line. For rhetorical purposes, I asked David if he wanted to revoke the HIV fast track, which he had no interest in doing. I then pressed him on the moral distinction between accelerating drugs for HIV patients but not for patients dying of cancer and other fatal diseases. Knowing David was a pediatrician, I also shamelessly included some devastating diseases of childhood.

David had no answer to my morality question. His face softened, and he relented. In 1991 the FDA approved Genzyme’s Ceredase for a very rare fatal condition called Gaucher disease in a non-blinded trial of only 12 patients; the drug has allowed almost all patients to lead almost normal lives. In 1992 HHS substantially adopted the McIntosh plan and allowed accelerated approval of drugs for all life-threatening diseases.

Unfortunately, the battle had only begun. The FDA’s lead examiner for the first drug to be approved under the accelerated approval regulations recommended against approval, but was overruled by a more senior official, Dr. David Finbloom. This pattern continued for many years with the same examiner and others putting up roadblocks against innovative therapies until Finbloom bulldozed through those roadblocks. As a result of his vision and determination, critically important new drugs for cancer, multiple sclerosis, and rare diseases became available to patients under the accelerated approval regulations. When David Finbloom died in 1999 (of the same rare brain cancer that killed Senator Ted Kennedy and my father), America lost one of its greatest patient advocates.

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