After weeks of controversy, Biogen (BIIB) has agreed to provide an experimental drug for combating ALS to a small group of very sick patients under a so-called expanded access program. But the move may come too late for the woman who pushed the company to take this step.
The decision follows sustained pressure by ALS patients, notably Lisa Stockman Mauriello, a health care communications executive who has a rare and fast-moving form of the neurological disease. This subset of patients typically has a very short life span, but no treatment options. So with help from supporters, she mounted a high-profile campaign to convince Biogen to provide access to its drug as quickly as possible.
But Biogen repeatedly refused, citing concerns that providing the drug to patients outside of an ongoing study would be unfair to participants who were given a placebo. As a result, the campaign not only cast an uncomfortable spotlight on Biogen, but also highlighted the quandaries surrounding expanded access programs in an era when innovative new medicines are spurring patients to become more assertive.
A thoughtful analysis of a tragic situation. The current environment is cruel and dysfunctional and should be unsustainable. It is a public policy problem that should be addressed as such, with transparency and participation by everyone with a stake in the outcome, not in the breach as this has been.
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