Yesterday, the FDA clarified its expanded access policies in a series of documents including a Q&A that outlines the process of obtaining expanded access, guidance that describes how companies may charge for therapies given under expanded access programs, and a new form to request access to investigational drugs.
FDA Commissioner Robert Califf said the agency hoped to simplify and streamline the process to reduce "procedural burdens on physicians and patients."
In one document, the agency outlines requirements for physicians and companies to obtain expanded access for individual patients, or for intermediate or large-scale studies. The document notes that FDA requires IRB review of all expanded access protocols, even in cases of emergency use, as well as reporting of adverse events. Addressing concerns from drug sponsors that adverse event reports from expanded access programs could derail development efforts, the agency said that while adverse event reporting from expanded access protocols have been included in drugs' safety assessments in a "small number of cases," FDA reviewers are to interpret safety data in "the context in which the expanded access use was permitted."
In another guidance addressing the process of charging for investigational therapies, FDA said companies must provide evidence that a trial could not be conducted without charging, that the treatment has a potential clinical benefit, and that data obtained from the trial are "essential" to establishing the therapy's safety or efficacy. The company must also submit a calculation of cost recovery. In 2013 draft guidance, the agency had said companies can only charge for direct costs involved in making an investigational drug available, and only when the costs of providing the drug are "extraordinary" for the company. The new guidance reiterated both points.
FDA also finalized an Individual Patient Expanded Access Investigational New Drug Application form to speed individual requests for investigational drugs. Califf said the new, "much shorter form" should take physicians about 45 minutes to complete.
In one document, the agency outlines requirements for physicians and companies to obtain expanded access for individual patients, or for intermediate or large-scale studies. The document notes that FDA requires IRB review of all expanded access protocols, even in cases of emergency use, as well as reporting of adverse events. Addressing concerns from drug sponsors that adverse event reports from expanded access programs could derail development efforts, the agency said that while adverse event reporting from expanded access protocols have been included in drugs' safety assessments in a "small number of cases," FDA reviewers are to interpret safety data in "the context in which the expanded access use was permitted."
In another guidance addressing the process of charging for investigational therapies, FDA said companies must provide evidence that a trial could not be conducted without charging, that the treatment has a potential clinical benefit, and that data obtained from the trial are "essential" to establishing the therapy's safety or efficacy. The company must also submit a calculation of cost recovery. In 2013 draft guidance, the agency had said companies can only charge for direct costs involved in making an investigational drug available, and only when the costs of providing the drug are "extraordinary" for the company. The new guidance reiterated both points.
FDA also finalized an Individual Patient Expanded Access Investigational New Drug Application form to speed individual requests for investigational drugs. Califf said the new, "much shorter form" should take physicians about 45 minutes to complete.
