Earlier this year, Lisa Stockman Mauriello was diagnosed with a fast-moving form of ALS, a fatal neurological disease that gradually causes muscle weakness and paralysis.
So her doctor sought to enroll her in a clinical trial for an experimental drug being developed by Biogen (BIIB) called tofersen. But even though he is a clinical investigator in the late-stage study, Biogen would not admit her because enrollment had recently ended.
So the physician tried Plan B: He asked Biogen to consider compassionate use, a regulatory program designed to allow people with life-threatening illnesses to obtain access to experimental medicines. Biogen declined, citing ethical concerns for patients who were enrolled in its trial.