Fact-Checking Compassionate Use: Biogen, a Mom with ALS, and what I learned on Clubhouse

The story of Lisa Stockman Mauriello and her battle for access to the investigational medicine tofersen from Biogen has been well-covered in mainstream media. The story is very personal for this LinkedIn community -- Lisa is a peer for many as a pharma communications professional most recently as a senior executive at Syneos Health. Her battle as a mother of 3 battling a recent battle with Bulbar ALS is heart-breaking. (Edit: As some have noted, Lisa is battling a particularly aggressive form of ALS (SOD1 with A5V variant) where life expectancy is less than a year, creating tremendous urgency.)

This evening on Clubhouse I learned a tremendous amount more on this issue from some fabulous voices with great expertise and perspective.

Some in pharma have cited concerns over the impact to a timely review of a regulatory submission by FDA and other authorities. The clinical trial participants had to meet rigid eligibility criteria, whereas those seeking compassionate use may have very different disease profiles. If they were to have an adverse event while on the medicine, will that delay the regulatory review thereby delaying access to the drug for the many other patients with the disease?

Biogen's published response also pointed out the concern about respect for those who are on the placebo in the current clinical trial that has completed enrollment. Is it not unfair for them if another patient is simply granted access? Would it discourage patients who perceive they are in the placebo arm from staying in the trial, believing they could simply withdraw from the study and apply for compassionate use?

Others note the challenge of "if we respond to one, must we respond to all". If there are 15,000 patients with ALS in the US, is it the responsibility of pharma sponsor to manufacture enough drug to supply all of the patients who request access? (Edit: In the case of Lisa's particularly rare and aggressive form of ALS, the number of patients may be in the low to mid hundreds. For such a rare disorder, does the sponsor provide compassionate use for all patients?)

Experts on this call tonight point out there have been over 10,000 compassionate use requests tracked by FDA, and that none have prevented have a medicine from reaching the market.

Some also noted that Lisa's physician happens to also be an investigator in the Biogen trial, and that she would have been eligible for the study had she not been diagnosed just after enrollment closed. Could a sponsor have extended enrollment? Are there pathways for a patient to be "randomized" within an expanded access program for equity?" (Edit: It was also noted that Lisa volunteered to be randomized, demonstrating integrity and supporting equity with those who participated in the clinical trial.)

Many cited the importance of respect for those in a placebo control arm. While some expanded access programs in the past have excluded those who were study participants that withdrew from the trial to submit for compassionate use, a key message was the importance of offering all patients access to the active drug at the end of the study.

Some called the opportunity for industry and patient groups to collaborate on best practices (or better practices) in this area. In some instances, using transparency to call out those pharma that are adhering to such better practices may be an incentive to drive awareness of better approaches and change for the future.

The conclusion seems that many are still looking to Biogen for more answers. Biogen has a long-standing commitment to the ALS patient community, and has invested tremendous resources to bring new treatments forward. As the online petition for Lisa exceeds 100,000 signatures and the #tofersen4lisa hashtag spreads, many are optimistic that other creative solutions (such as randomizing her to give a fair chance) will be given fair consideration to create a solution for Lisa and all of those battling ALS.

It is not sufficient here to "be correct while also being tremendously wrong".

Many thanks to those who helped me to learn on Clubhouse this evening: Jess Rabourn, Daniel McIntyre, Erica Bersin, Farah Ahmad, Dave deBronkart, John Neal, Marc Wilenzick, Heather Manna, Helen West, Chris Benko, and Joe Dustin.