The U.S. Food and Drug Administration (FDA) is easing restrictions on its compassionate-use program, also known as expanded access, which gives seriously ill patients access to experimental drugs under FDA review.
“Each year, FDA receives over 1,000 applications for the treatment of patients through expanded access … and the agency authorizes the vast majority (about 99%),” FDA Commissioner Scott Gottlieb, MD, explained. “[The] FDA recognizes that time is critical for these seriously ill patients who do not have alternative therapies and who cannot take part in a clinical trial of an investigational therapy.”
The changes will streamline the compassionate-use program. Current regulations require that a physician receive permission from all members of the institution’s internal review board to treat a patient with an experimental drug; the new process only requires approval from one designated member of an internal review board – such as the chair.
The FDA also updated its industry guidance document to clarify how adverse events in compassionate-use patients should be reported. Because expanded-access treatment takes place outside of a defined and controlled clinical trial, and the FDA cannot require a manufacturer to provide a product, a pharmaceutical company may hesitate to provide an investigational drug through the program over concerns about how adverse events would affect the drug’s regulatory review. The updated guidance document states that adverse reactions need to be reported “only if there is evidence to suggest a causal relationship between the drug and the adverse event.”
Source: FDA Voice blog, October 2, 2017.