H.R. 5247 authorizes the use of eligible investigational drugs by patients who have been diagnosed with certain illnesses, who have exhausted the approved treatment options and are not eligible to participate in a clinical trial.
The bill defines an eligible patient as those with “a stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months, or a disease or condition that would result in significant irreversible morbidity that is likely to lead to severely premature death.”
The legislation allows for investigational drugs to be used if they have completed phase 1 of clinical trials, they have not been approved or licensed for any use under the Public Health Service Act, and an application has been filed that is active under the Public Health Service Act. The legislation removes liability on the manufacturer, sponsor, physician, hospital, or clinical investigator if the above conditions are met and there are not additional factors such as willful, criminal, or reckless misconduct or gross negligence, and removes liability if such an entity makes the determination not to provide the investigational drug. The bill requires doctors for such patients to report “serious adverse events” from the drug to the manufacturer. The legislation also strengthens patients protections with clearer informed consent and real-time adverse event reporting as well as a standard to notify the Food and Drug Administration (FDA) when a patient receives an unapproved drug through the new alternative pathway. The liability protections apply to both the existing expanded access program as well as the new alternative pathway, leveling the playing field by removing a perverse incentive to choose on program over the other.