Frederick Baron, the prominent attorney dying of bone marrow cancer, has gotten his wish for a dose of Tysabri after all. The FDA and the Mayo Clinic found a legal basis to give him the drug in an emergency situation, according to a post on Dembot by his son, Andrew.
“We have every expectation of a positive result,” said Andrew Baron, the CEO of Rocketboom, in the web posting. “We cannot thank you enough for all of your thoughts and support.”
The decision by the FDA and the Mayo Clinic caps a frenzied week in which prominent friends of Baron, including former President Bill Clinton, cyclist Lance Armstrong, and U.S. Senators Ted Kennedy and John Kerry, all urged Biogen Idec to give Baron the medicine. Biogen refused, because Tysabri is unapproved for multiple myeloma, Baron’s disease, and it said that giving him the drug outside of a controlled clinical trial would create risks that the drug might be restricted for patients who need it for multiple sclerosis and Crohn’s disease.
Despite numerous phone calls from friends of Baron, a longtime Democratic fundraiser, and other concerned citizens, Biogen never re-considered its decision to provide the drug, says company spokeswoman Naomi Aoki. Yet as pressure mounted, the FDA informed Biogen late Wednesday that it was trying to resolve Baron’s situation directly with the Mayo Clinic. Aoki couldn’t be more specific on how the drug is being provided, or who is assuming liability in case something goes wrong.
Normally, with a marketed drug like Tysabri, physicians like those at the Mayo Clinic in Rochester, MN, are free to prescribe it for any disease, or any dose, however they see fit, under what is known as “off-label” use. The situation is more complicated with Tysabri, because it is currently available only under a strict prescribing and monitoring program called Touch; the program was instituted when Tysabri was brought back to the market in July 2006 after having been withdrawn in 2005 because of its link to a rare and often fatal brain infection called PML. Tysabri is now taken by an estimated 32,000 patients worldwide with multiple sclerosis and Crohn’s disease.
Baron’s physician hit upon the idea of treating him with Tysabri because samples of his cancer cells turned out to carry a molecular marker that Tysabri, an antibody drug, is designed to seek out and block, according to a letter Andrew Baron posted online Monday. Biogen has done some animal tests that suggest Tysabri might be an effective treatment for multiple myeloma, and in fact it began treating the first patient in a clinical trial of Tysabri against the condition last month, Aoki has said. Baron’s physician is an investigator on this study and well-familiar with its rules, Aoki says.
Liability has been a key question in this case all along. Andrew Baron, in an earlier comment on this site, said the FDA had assured Biogen that it would be protected from legal liability if it gave Frederick Baron the drug. “The FDA said that whatever happened in this particular case with my father, would not be counted against them. This is similar to a case which becomes expunged,” he wrote.
An FDA spokesman, in a written response to the Pharmalot blog, said it never offered Biogen a liability shield. “The FDA has provided no such assurance to the company. Our policy is to consider all available information when evaluating the safety and effectiveness of drugs,” the spokesman told Pharmalot. “We recognize and appreciate that expanded access programs involve less-controlled use of experimental treatments than the well-controlled environment of a clinical trial, and thus we would consider information that might be obtained in this instance within that context.”
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