Back in 2008, Dallas trial lawyer and high-profile Democratic Party fundraiser Fred Baron was dying of multiple myeloma. Desperate to gain access to Biogen’s Tysabri, an MS drug then being tested for myeloma in early-stage studies, his wife Lisa Blue Baron reached out to the company but ran directly into a brick wall of resistance.
“[Biogen] won’t approve for fred because he is too sick and if it fails him, it could skew the outcome of the trials(this told to me by his wife),” noted a family friend in the error-ridden post included in the batch of hacked emails from John Podesta — the chairman of Hillary Clinton’s presidential campaign — dumped by WikiLeaks on Saturday.
Incensed, Baron’s wife reached out to influential friends in the Democratic Party.
If I am unable to get drug I will take out full pg add in boston globe to ceo and board members.vFred is running out of time. He’s so sick and needs this drug. Thanks so much.
In short order, a whole lineup of high-profile officials ranging from former President Bill Clinton to Senator John Kerry and then FDA Commissioner Andrew von Eschenbach were directly lobbying former Biogen CEO Jim Mullen to provide the drug for compassionate use.
One segment of the Podesta email exchange includes:
I got through to Mullen. He had already had extensive conversations with Senator Kerry and Lance Armstrong and had heard from President Clinton, too. The answer he gave in every case was [no]. I believe that I understood it correctly that Kerry had organized a conference call with Mullen and FDA Commissioner Von Eschenbach. I hear that Von Eschenbach said it was fine with him for the drug to be used in Fred’s case. However, Biogen believes that Von E does not have that authority given the strict FDA conditions on the use of the drug.
And in the next section:
I just had a disappointing call with Dr. Von Eschenbach. FDA had a phone conference with the medical and science officers from Biogen. FDA told them there would be no prejudice to the clinical trial if this drug were used for Fred. They were appreciative but gave no indication they would change their position, which is that a corporate decision has been made they would not go outside the clinical trial. I am really upset about this, personally, since FDA has been great to help. I have not had the time today to reach out to some on the board of Biogen, but hope to do so yet today. I felt last night, after talking with Andy von Eshenbach, that with clearance from FDA, the company would be ok.
Looking back over Luke Timmerman’s coverage at the time for Xconomy, it’s interesting to note that Baron got the drug because the FDA and the Mayo Clinic provided it to him after the agency found a legal basis to do so in an emergency situation.
Biogen, though, had continued to insist that any use of Tysabri for myeloma in Baron’s case could create a situation that would cause regulators to restrict its use. This was two years after the FDA had allowed Biogen and Elan to put the drug back on the market after it was pulled following the death of several patients due to PML.
The debate over access to drugs under compassionate use policies has helped spur a whole series of “Right to Try” laws around the country. For many patients, it can be a frustrating exercise, trying to convince a biopharma company to provide a drug to them in their last dying days. For many biotechs, though, compassionate use remains a vexing issue as well. These programs can be expensive and also raises the threat that an unexpected adverse event can force a company to suspend work on a drug.
Fred Baron got the drug, but there was no happy ending. The behind-the-scenes struggle ended with his death on October 30, 2008, soon after the controversy erupted.
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