Drug Development for Limited Populations: A New Proposed Pathway

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The lack of new antibiotics and the rise in drug resistance have rendered some serious and life-threatening infections untreatable, and the health care community is searching for ways to bring innovative new drugs to patients whose treatment options are limited or nonexistent.

On Jan. 31, 2013, The Pew Charitable Trusts hosted a one-day conference, "A New Pathway for Antibiotic Innovation: Exploring Drug Development for Limited Populations." Pew brought together government regulators, infectious disease physicians, public health specialists, pharmacists, pharmaceutical company representatives, and payors to weigh the merits of a proposed new Food and Drug Administration approval mechanism: a limited population regulatory pathway. The proposal promises to bring urgently needed medicines to the sickest patients by reducing some of the economic and regulatory barriers to antibiotic innovation.

Some general themes emerged from the conference:

• Payors, health care providers, and pharmaceutical companies generally supported the concept of a limited population regulatory pathway.
• The pathway could increase the feasibility and lower the costs of clinical trials for high-need antibiotics.
• Limited population antibiotics would likely command premium pricing, which could provide a reasonable return on investment for antibiotic developers. 
• Premium pricing could ensure that limited population antibiotics are not used indiscriminately, thus preserving the effectiveness of these critical drugs over time.
• A special designation or labeling alone would not curtail unnecessary use of the limited population antibiotics, but could signal that these drugs are different from other antibiotics and should be treated accordingly.
• Strict restrictions or penalties on the use of limited population antibiotics were not recommended, but all participants agreed that stewardship—the proper management and data-driven use of these drugs — would be important for success of the pathway.