FDA would like to have a new “special medical use” category for drugs that could be approved based on a small clinical development program and that would have a limited indication for specific populations.

The impetus for, and initial focus of, the program would be antibiotics for drug resistant bacteria, Commissioner Margaret Hamburg and Center for Drug Evaluation and Research Director Janet Woodcock told reporters during a briefing March 7.

“We would like to have this tradeoff, this quid pro quo, so to speak, that you could have a very small development program and then couple it with limitation of the use … some designation that it shouldn’t be used off-label,” Woodcock explained.

As to whether prescribers would refrain from off-label use, Woodcock insisted, “We think that at this point in time the health care community could honor this if it were established.”

Hamburg pointed out that hospitals, through their formularies, already are regulating the use of antibiotics, “not because of FDA, but because of both their own cost strategy and their recognition of the need to hang on to the effectiveness of certain antibiotics.”

If the new designation were put in place, Woodcock added, the agency probably would require sponsors to monitor prescriptions and determine if prescribers adhered to the limitations. “We should have a pretty rapid readout of how effective it was.”

The agency is just starting to explore the concept, which came in large part from conversations with the Infectious Disease Society of America, she noted.

Industry also has been seeking alternatives to the current approach of two large clinical trials, suggesting use of a single trial, with the limitations of the data noted in labeling Also see "Sponsors Seek One-Trial Approval Of Antibiotics For Drug-Resistant Bugs" - Pink Sheet, 3 October, 2011..

Riding The Legislative Tide

There are many issues with a special medical use category, such as patents and side effects, that would need to be discussed and “we would probably want to do it with a more formal regulatory framework” to gain input from industry, policy makers and the patient community on the pros and cons, Hamburg pointed out.

Woodcock will testify March 8 before the House Energy and Commerce Health Subcommittee on Health, whose agenda includes methods to encourage antibiotic development. The panel is looking at several issues that could be add-ons to a Prescription Drug User Fee Act reauthorization. Increasing the antibiotic pipeline is a high profile issue that has spawned the Generating Antibiotic Incentives Now (GAIN) Act in both the House and Senate to provide incentives, including five years of exclusivity for drugs designated as qualified infectious disease products Also see "Antibiotics Could Get Double Exclusivity Under House Bill; FDA Makes Its Own Moves" - Pink Sheet, 20 June, 2011..

Developing antibiotics for resistant bacteria requires a multi-faceted strategy, Hamburg said, noting that the GAIN Act is one strategy and the agency has been providing technical assistance to the sponsors.

The Commerce Subcommittee hearing will also focus on accelerated approval issues ([A#14120305001]), as well as the drug supply chain Also see "Supply Chain Safety’s Dual Visions: Pedigree Or Just Serialization" - Pink Sheet, 2 March, 2012..

FDA Exploring Limited-Use Approvals For Antibiotics Based On Small Development Programs

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