Debating 'limited use'

FDA wants public dialog on new 'limited-use' pathway inhibiting off-label use

FDA is asking the public to consider whether it should supplement the current all-or-nothing approval structure with a system that would couple approval of selected new drugs with measures designed to discourage off-label use.

The agency says it could approve drugs for conditions such as antibiotic-resistant infections and life-threatening obesity based on small, fast trials if it were confident use would be limited to well-defined populations.

There is little controversy about approving drugs based on relatively small studies that demonstrate high levels of efficacy in tightly targeted populations. But FDA's suggestion that it could work with physicians and payers to limit use of a marketed drug in the absence of documented safety concerns is controversial.

Patient and medical groups support the concept of a limited-use pathway, and the President's Council of Advisors on Science and Technology (PCAST) has endorsed the idea. PCAST's recommendation to create a Special Medical Use (SMU) pathway was drafted in close consultation with senior FDA officials and reflects the agency's thinking.

However, some drug companies are skeptical. They do not think FDA needs new authority to approve drugs for limited populations, and several biopharma executives told BioCentury they generally oppose curbs on off-label prescribing.

Whether FDA could implement a limited-use pathway on its own without new legislation is uncertain. The agency would clearly prefer the backing of Congress. But again, some stakeholders, including patient and industry representatives, don't think legislation is either necessary or wise.

Scientists at seven antibiotics developers have proposed a regulatory framework to facilitate the development of new antibiotics for limited populations that they say

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