Gottlieb Outlines Changes to Expanded Access Program
FDA is making changes to ease patient access to experimental therapies under its expanded access program, according to commissioner Scott Gottlieb. Testifying before a House Energy and Commerce Health Subcommittee hearing 10/3, he noted that current regulations require physicians to obtain full IRB approval in order to treat a patient with an investigational drug. To simplify the process, Gottlieb says now just one IRB member – the chair or another appropriate person – can approve the treatment use.
Gottlieb said that over the last few years the agency has received over 1,000 applications annually for expanded access requests, noting that it authorizes 99% of these. “Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days,” he said. However, there is some “reluctance among companies to provide investigational drugs for expanded access,” he said in a related blog post. “This may have been due, in part, to uncertainty about how data for adverse events that occur during treatment under expanded access are viewed by FDA. Companies have voiced concerns that any apparent negative effects might jeopardize the product’s development.”
During the hearing, Gottlieb told subcommittee members that adverse event reports coming from expanded access use are typically not used in product review decisions because they are used outside in a clinical trial setting. A guidance has been recently revised to reflect that suspected adverse reactions must be reported “only if there is evidence to suggest a causal relationship between the drug and the adverse event.”
Gottlieb said that the agency looked at a decade of experience involving 322 drug approvals (28% had expanded access opportunities) and no adverse experiences from expanded access use prevented any approvals. He said there was one instance where adverse event information from an expanded access use was used in drug labeling and another instance that led to effectiveness assurance and approval.
During his testimony, Gottlieb also detailed FDA’s collaboration with the Reagan-Udall Foundation, patient advocacy groups, the pharmaceutical industry, and other federal agencies to launch a new online tool called the Expanded Access Navigator. “It collects for the first time, in one centralized online location, links to drug manufacturers’ expanded access policies, procedures, and points of contact. The Navigator also includes additional information to help guide patients and physicians through the steps to obtain such treatments,” he said.
The subcommittee hearing offered Gottlieb an opportunity to comment on pending right-to-try legislation being considered in Congress. He said the agency recommends narrowing the eligibility from patients who face a “life-threatening disease or condition” to “terminal illness.” Many chronic conditions (e.g. diabetes) are life-threatening, but medical and behavioral interventions make them manageable without considering unapproved therapies. In addition, the term used in one bill for “terminal illness” would benefit from a clear definition. “We recommend defining it as ‘a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months,’” he said.