‘Remember her as the badass she was’: ALS patient who died should force Biogen, others to anticipate expanded access issues

It’s been two weeks since Lisa Stockman Mauriello passed away and a lingering question, in my mind, is whether she died in vain.

I’d like to think not.

During her final months, the 52-year-old former public relations executive battled Biogen, one of the world’s largest biotech companies, for access to an experimental treatment for ALS, a fatal neurological disease that gradually causes muscle weakness and paralysis.


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.


Sadly, Biogen declined. The company justified its decision with some reasonable explanations, but I think it went about it the wrong way.

And while it is too late for Stockman Mauriello, her advocacy prompted a renewed look at expanded access in a way that may be instructive for the pharmaceutical industry, so that other lives may be saved.

Here’s why: Initially, the company argued that providing the drug to patients outside of an ongoing study would be unfair to participants who were given a placebo. This also implied a broader concern — maybe some people would avoid future trials in hopes of getting a drug through expanded access.

That’s understandable.

In general, expanded access is a complicated and thorny issue for drug makers. Dying patients and their families are effectively pounding on doors desperate for a treatment that may or may not work. But who can blame them?

The process to obtain a treatment through expanded access actually starts with the Food and Drug Administration, which approves nearly all of the requests made by physicians treating patients in need. But then it’s left to the drug company to decide who gets its medicine.

This is where some planning is needed, though.

In recent years, a grassroots movement sprung up to argue the FDA was impeding access, which eventually led to a new federal law that gives severely ill patients the ‘right to try’ to obtain a medicine. The law doesn’t really change anything, though, because drug makers still have final say.

So the ball was in Biogen’s court when Stockman Mauriello’s physician, who has also been a clinical investigator in the company’s late-stage study for the drug, requested expanded access.

The company’s rejection came in March, and the ensuing publicity set Biogen on its heels.

Before the month was over, Al Sandrock, who oversees Biogen’s R&D, posted a note on the company’s web site to explain its position. A month later, a workaround was found, after the company spoke with patient advocates, regulators, clinical investigators and ethicists.

The plan was for Biogen to offer its drug, called tofersen, to Stockman Mauriello and others once the late-stage study was completed and participants were no longer on a placebo. The target date was mid-July. Once the biotech determined the study data showed the drug is safe and effective, the program would be opened to a broader group of patients with the gene mutation in the fall.

This sounded reasonable, but it was too late for Stockman Mauriello, who was not expected to live past the summer. Remember, she had a fast-moving form of the disease. Every day mattered.

And this pointed up a problem – rather than anticipate such a scenario could occur, Biogen seemed flatfooted.

Toby Ferguson, who heads the neuromuscular development unit at Biogen, disagreed. He told me the company had a “broader” program in place when her request was made. But it was contingent on a risk-benefit evaluation in the ongoing trial, and there weren’t any “data readouts.”

“The big impediment was our duty to the integrity of the trial and introducing delays to ALS patients who could benefit,” he maintained. “We felt it would not be reasonable to provide access while the placebo portion of trial was ongoing.”

Yet the workaround only came after harsh criticism.

“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.”

There’s another point: If Biogen wanted to finish enrolling patients in its trial before offering expanded access, then the biotech should have thought more carefully about how quickly the disease progresses and whether patients who were not eligible for the trial would survive long enough to enter the program. But this requires some forethought.

And here is a point that was not lost on many of the people who urged Biogen to make its drug available. The company worked feverishly behind-the-scenes over the past year to convince the FDA to approve its controversial Alzheimer’s drug, going as far as concocting a secret effort to win over an FDA official, despite the drug’s questionable effectiveness.

The company devoted a lot of muscle to that effort. Yet Biogen seemingly scrambled when Stockman Mauriello needed attention.

To be fair, though, there are rarely easy decisions when it comes to expanded access.

Neil Shneider, a Columbia University Medical Center neurologist who was Stockman Mauriello’s physician, acknowledged the dilemma.

“In their defense, I think they were trying to deal with this in a serious way,” he told me. “But I think there was an ethically acceptable solution to all this that would have offered Lisa at least some chance of avoiding what proved inevitable. At this point, I’m hoping what Lisa and her family did will make a difference in the lives of other people. It’s the best we can hope for.”

Whether that occurs remains to be seen. Thanks to her perch, she and her allies knew which levers to pull. Not everyone is well known or well connected, but Stockman Mauriello may have changed the conversation about expanded access, if only a little.

I’ll give the last word, though, to Luke Mauriello, one of her three sons.

At the funeral mass last week at St. Teresa of Avila Church, which looks out over the leafy suburb of Summit, N.J, the 19-year-old offered a moving eulogy that brought a rousing response from the approximately 200 people who attended.

“Let’s not remember her as a woman who died of ALS, but as the woman whose efforts advanced research and access to ALS medicines,” he implored. “Let’s remember her as the badass she was.”

Source: STAT