Regulators start thinking creatively about new approval pathway

FDA officials have won close attention for some creative thinking under way at the agency about a potential new approval pathway for a particular type of therapy. Dr. Janet Woodcock told reporters last week that new antibiotics could run through the approval process at a faster speed if they were targeted at specific drug-resistant pathogens. And the same approach might be used for other drugs as well.

"We wouldn't want such a drug to be used for non-drug-resistant organisms," said Woodcock, director of the FDA's Center for Drug Evaluation and Research, according to a report in the Wall Street Journal last week. But a developer might get a faster approval with small trials which recruited subjects in a narrow patient group. Developers would then be required to monitor the use of the treatment, making sure it was used for limited purposes and not widely prescribed by doctors on an off-label basis.

The Boston Globe points out that Woodcock discussed the possibility that surrogate endpoints--secondary indications that a drug is working, instead of the more rigorous demands made for classic primary endpoints--could be used to accelerate an approval for a variety of conditions. This discussion comes as the FDA considers expanding the more liberal approach it takes to experimental AIDS and cancer drugs. And MedPage Today notes that Woodcock discussed the FDA's contacts with the Infectious Disease Society of America, which is pushing for speedier reviews on antibiotics, as we reported on Friday.

In exchange for the swifter approvals, developers would have to be satisfied with far stricter labels specifying which patient population could benefit from the treatment. And the approach might even extend to specific groups suffering from ailments like obesity.

The FDA has been under considerable pressure in recent years to accelerate drug and device approvals. This new thinking on their part would be particularly appealing to biotechs, which might find it more feasible to keep a drug through late-stage studies if the agency could define a more efficient approval pathway. And fears about off-label safety threats might best be policed by physicians' malpractice attorneys.

"Doctors won't get in trouble with the FDA, but their malpractice lawyers might not think too highly [of doctors prescribing a limited use drug to a wider population]" Woodcock told reporters.

- here's the story from The Wall Street Journal
- here's the Boston Globe report
- here's the story from MedPage Today