The framework has four tiers, which would allow for varying indications, labels and efficacy requirements. Only one of the tiers would require two randomized, controlled clinical trials, the standard requirement for new drug approvals in the U.S.
“Although historically antibiotics have not qualified for orphan drug-based registration, we propose the application of successful ideas from registration of orphan drug products and other existing successful regulatory frameworks to address the challenges of antibiotic drug development,” the executives wrote in the January issue of the Lancet.
Price is another issue that may align the orphan drug and antibiotic markets. The prices of antibiotics approved to treat a narrow subset of patients will increase markedly, Robert Guidos, vice president of public policy and government relations for the Infectious Diseases Society of America, said during a meeting convened last week by the Pew Charitable Trusts.
“Creating scenarios in which there is a pricing premium placed on antibacterials is probably the single biggest financial incentive that one could create,” Spellberg said. “That would have a tremendous impact.”
The FDA plans to hold a hearing this week to gather information on developing a new approval pathway for drugs targeting serious or life-threatening conditions that address an unmet medical need, including antibiotics.
Even as the FDA evaluates a new pathway for antibiotics, a step that is likely to improve a weak pipeline of new drugs, other questions persist about postmarket monitoring, off-label prescribing and the agency's role in stemming further antimicrobial resistance.
“The challenge for public policy is how you incentivize new drugs but in a way that you don't recreate the problem of resistance all over again,” said Ramanan Laxminarayan, director of the Center for Disease Dynamics, Economics & Policy, a Washington-based not-for-profit that seeks to improve decisionmaking in health policy. “The FDA is now seriously starting to look at how we can manage drug effectiveness while at the same time trying to get some new drugs in the pipeline.”
Physicians have the discretion to prescribe off-label uses for drugs. High prices and better education of physicians and healthcare providers could deter some off-label use of drugs approved under a new pathway, drug executives said during the Pew meeting. However, hospital officials said physicians are often unaware of a drug's indication or have not read its label.
Dr. Pranita Tamma, director of pediatric antimicrobial stewardship at Johns Hopkins Hospital in Baltimore, said during the Pew meeting that special labeling and high prices are “not enough to ensure drugs are used judiciously.”