ALS patient who died should force Biogen, others to anticipate expanded access issues

From STAT:

A patient with ALS named Lisa Stockman Mauriello who recently battled Biogen for access to a clinical trial passed away recently, raising the issue of expanded access to trials.

Stockman Mauriello was diagnosed earlier this year with a fast-moving form of ALS caused by a genetic mutation and was told she had just months to live. Yet she was closed out of a Biogen clinical trial because enrollment had just ended.

So she did the next best thing. Along with a coterie of well-connected friends — many of whom also worked in life sciences public relations — she badgered the company to provide its drug under expanded access, a regulatory program designed to allow people with life-threatening illnesses to obtain access to experimental medicines. Biogen declined.

“Biogen should have had a plan in place from the very start about how it would handle expanded access requests that came in at all different points: before, during and after trial enrollment,” Holly Fernandez-Lynch, an assistant professor of medical ethics at the Perelman School of Medicine at the University of Pennsylvania, explained.

“The key thing is that there’s no reason Biogen should have been working these details out on the fly when Lisa made her request.”

“If you are eligible for the trial — or were eligible but declined to participate at the time — then you should be ineligible for expanded access,” she continued. “If you are ineligible for the trial — and never were eligible — and there’s no other trial coming up soon, then you should be considered for expanded access, assuming adequate supply and other logistical considerations are feasible.”