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The FDA Returns to Its Dark Ages

8:14 AM, Apr 14, 2014 • By MICHAEL ASTRUE
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In subsequent years the FDA continued to use the accelerated drug regulations for some cancer indications, but quietly retreated for most other diseases. Accelerated approval is broadly popular among patient groups, and Congress has tried to push the FDA back on the right track multiple times since the promulgation of the 1992 regulations. In 1997 and 2012 Congress codified and expanded FDA’s accelerated approval authority, but the agency has not fully implemented those statutes.

In addition to not moving forward in the ways that Congress has mandated, the FDA keeps trying to defend its stubborn march back to its Dark Ages of drug approval. The case of the most recently approved biogeneric version of Ceredase (later replaced by a recombinant form of the drug called Cerezyme) is a good example of this retreat.

Ceredase has been one of the most expensive drugs on the market, but competition is bringing that cost down. The FDA approved another version of the drug in 2010 (one I helped to develop) and recently approved a third version developed by Pfizer in 2012. To defend itself from valid criticism that the FDA is growing more rigid again, Commissioner Margaret Hamburg’s February 6, 2014, blog entry cited the 2012 approval as one of two examples of the agency’s “flexibility.” (See here.)

The FDA’s use of the Pfizer drug as an example of its “flexibility” does not withstand scrutiny; the agency required Pfizer to conduct twice the number the number of trials and enroll more than four times the number of patients that Commissioner Kessler required for the original trial of the enzyme almost a quarter of a century ago. The other example highlighted a drug for a rare cancer, the one disease area where the agency does use its accelerated drug approval authorities with less hesitation. In short, the FDA insists on looking only at the risks of taking a drug, and only rarely considers the risks of not taking a drug. That bloodless philosophy rejects the lessons taught by the HIV activists and amounts to a death sentence for tens of thousands of Americans each year.

Patients are increasingly up in arms. Over 100,000 people have signed a White House petition to approve a promising new drug for Duchenne muscular dystrophy, a debilitating disease that right now will kill one boy of every 3,600 born in this country. Despite two Phase II studies that indicate the drug is quite safe and stimulates production of a protein essential to thwarting the devastating effects of this disease, the FDA has sent the sponsor back for more long and slow trials. Under the White House’s own rules, President Obama will have to respond publicly to the petition.

It takes no imagination to picture what is happening now at HHS. Its executive secretariat is almost surely circulating for comment a brief FDA-drafted statement for the president, which tries to express heartfelt sympathy for the dying young people while also claiming that there is nothing that the FDA can do about the situation.

The second half of that statement is a lie. Many critically important drugs have been approved for desperate patients in previous administrations with far less data than the data supporting this drug’s benefits for muscular dystrophy patients. If the FDA lacks the backbone to approve it, the agency can announce that it will routinely approve all applications for compassionate-use exemptions, which will make the drug’s availability an issue between insurance companies and the sponsor. The FDA can also announce that it will allow all patients who do not meet the entry criteria for the planned Phase III trial to join a long-term open-label trial, an option that would need NIH support to become a reality because the sponsor is a small company.

Perhaps the change at the top of the HHS is an opportunity for improved leadership. President Obama should not sign that artful HHS response, nor should he plunge into the details of the application. All true presidential leadership requires is a handwritten note back to HHS that says, “I know we can do better. Please try again.”

Michael Astrue is a former commissioner of Social Security (2007-2013) and general counsel of HHS (1989-1992).

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