A comprehensive regulatory framework to address the unmet need for new antibacterial treatments

• Published in: The Lancet infectious diseases 2013-03, Vol.13 (3), p.269-275
• Main Authors: Rex, John H, Prof, Eisenstein, Barry I, Prof, Alder, Jeff, PhD, Goldberger, Mark, MD, Meyer, Robert, MD, Dane, Aaron, MSc, Friedland, Ian, MD, Knirsch, Charles, MD, Sanhai, Wendy R, PhD, Tomayko, John, MD, Lancaster, Cindy, JD, Jackson, Jennifer, PhD
• Format: Article
• Language: English
• Subjects: Anti-Bacterial Agents - pharmacology; Antibacterial agents; Antibiotics; Antibiotics. Antiinfectious agents. Antiparasitic agents; Biological and medical sciences; Clinical Trials as Topic; Drug Approval - legislation & jurisprudence; Drug Evaluation; Drug Resistance, Bacterial; Humans; Infectious Disease; Infectious diseases; Medical sciences; Needs Assessment; Pharmacology. Drug treatments; Sustainable development; United States
• ISSN: 1473-3099; 1474-4457
• DOI: 10.1016/S1473-3099(12)70293-1

Summary To bring new antibacterial drugs to the market is challenging because discovery of new agents is difficult, two large trials per indication are needed in accordance with traditional regulatory requirements, and the economic reward is limited if the use of new antibiotics is constrained. These challenges have resulted in an alarmingly thin antibiotic pipeline, despite the rapid and continued growth in the need for new drugs. Approaches that balance the quantity of data needed for registration with the unmet medical need would encourage work in this area. Therefore, a tiered regulatory framework that allows either disease-based or pathogen-based label indications is proposed, with label wording that promotes the most appropriate use of new agents. Such a framework is within the bounds of present regulatory approaches, is amenable to international harmonisation, and would be a welcome step towards the facilitation of a robust and sustainable discovery and development infrastructure.