Regulatory Focus™ > News Articles > Health Groups Applaud Legislation to Create New FDA Approval Pathway, But Offer Suggestions

Health Groups Applaud Legislation to Create New FDA Approval Pathway, But Offer Suggestions

Posted 27 February 2014 | By Alexander Gaffney, RAC

In December 2013, legislators introduced a new piece of legislation that would permit the US Food and Drug Administration (FDA) to approve antibiotic drugs for high-need sub-populations using less rigorous standards of approval.

Now critics are weighing in on that legislation, arguing that the legislation, while critical, could benefit from additional labeling requirements.


The Antibiotic Development to Advance Patient Treatment Act (ADAPT) of 2013 would modify Section 505 of the Federal Food, Drug and Cosmetic Act to create a new "limited population" pathway for certain antibacterial and antifungal drugs intended to treat serious or life-threatening diseases or conditions.

Any drug approved under the ADAPT pathway-which is itself intended to be an addition to the Generating Antibiotic Incentives Now (GAIN) pathway passed into law in 2012-is intended to be flexible.

Approvals may be based on traditional or alternative endpoints, small clinical trials (including Phase 2 studies), pharmacologic or pathophysiologic data, and "other confirmatory evidence" deemed appropriate by FDA.

The labeling of the drug would also be required to bear a prominent statement: "This drug is indicated for use in a limited and specific population of patients."

Whether or not that statement would have any effect on prescribing habits is perhaps the biggest wildcard. The current legislation would not alter physician authority to prescribe a drug of their choice, though FDA would likely approve any drug under a REMS plan to restrict access or require physician training prior to prescribing an ADAPT product. The law contains a prominent disclaimer that nothing within it is intended to "restrict, in any manner, the prescribing of antibiotics or other products by health care professionals, or to limit the practice of health care."

As with accelerated approvals based on surrogate endpoints, FDA would also be permitted to withdraw the drug or fully approve it at a later date for a larger population, thereby removing its restrictions.

Call for Labeling Changes

While the ADAPT legislation already calls for specific labeling statements, the Pew Health Group and 29 other health organizations have written to the bill's sponsors, Reps. Phil Gingrey and Gene Green, to ask for new additions to the label.

"We recommended strengthening [the labeling statement] provision by requiring the label to include a prominently placed visual element, such as a logo, to clearly indicate to the healthcare community that these drugs are approved for a limited population and must be used prudently," Pew wrote in its letter.

That recommendation notwithstanding, Pew and the other groups threw their support behind the bill, saying it confronts the considerable threat of antibiotic resistance and affords desperate patients a potentially lifesaving chance at treatment.

"We thank you for introducing the ADAPT Act," the letter concludes. "We look forward to working with you toward the establishment of a limited population approval pathway to speed patient access to new life-saving antibacterial drugs."


Pew Health Group Letter

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