Biogen Turns Down Dying Patient’s Request For Tysabri—and Explains Why

Biogen Idec has turned down the requests of the family and high-profile friends of a dying patient at the Mayo Clinic to provide an emergency dose of Tysabri to try to combat the man’s cancer—an unapproved use of the drug. It’s a vivid example of the ethically treacherous zone that companies must traverse when patients request access to their drugs before they are approved as safe and effective by the FDA.

The case of Frederick Baron hit the blogosphere in a open letter to Biogen CEO James Mullen posted this morning by Baron’s son, Andrew. The letter grabbed instant attention (including from TechCrunch’s Michael Arrington, who wrote a scathing post about Biogen’s refusal), in part because Andrew Baron, CEO of RocketBoom, noted that celebrities including Lance Armstrong, former President Bill Clinton, U.S. Senators Ted Kennedy and John Kerry, among others, all personally called on Biogen to clear the way for their friend to get an emergency dose of Tysabri. Frederick Baron, 61, a famous trial attorney and prominent Democratic fund-raiser, has multiple myeloma, a deadly cancer of the bone marrow; Tysabri is only approved by the FDA for the treatment of multiple sclerosis and Crohn’s disease.

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called Touch; the program was instituted when Tysabri was brought back to the market in July 2006 after having been withdrawn in 2005 because of its link to a rare and often fatal brain infection called PML.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.

Frederick 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 Andrew Baron’s letter. 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, says company spokeswoman Naomi Aoki. Frederick Baron’s physician is an investigator on this study and well-familiar with its rules, but Baron isn’t eligible to participate in the trial because he is too sick, Aoki says.

In his letter, Andrew Baron wrote that FDA commissioner Andrew von Eschenbach “has granted special approval” for his father to receive Tysabri, which essentially puts the ball in Biogen’s court. Under FDA rules, the company has discretion to give out the drug for such unapproved uses—what is known as “emergency use” or “compassionate use”—but it chose not to, Aoki says. If something went wrong in an uncontrolled setting, it might lead to further restrictions on the drug’s use among existing patients with MS and Crohn’s disease, she says.

“We concluded when we re-introduced Tysabri that we cannot make it available for uses other than the FDA-approved use, or for an FDA-approved clinical trial,” Aoki says.

Still, I asked if this was a difficult decision, given the life and death nature of the matter. Andrew Baron’s letter says his father may have only a day or two left to live. “Of course, it’s a difficult decision for us. Our thoughts go out to the family. We don’t take this lightly, but we feel we have a responsibility to protect patients already on this drug who are benefiting from this drug,” Aoki says.

When asked if legal liability is part of the company’s concern, Aoki referred back to her statement about protecting the interests of patients already on the drug.

This sort of ethical quandary has come up for other companies, most recently Plainfield, NJ-based PTC Therapeutics, which has refused to give a boy with muscular dystrophy an experimental medicine outside of the protocol of a clinical trial.

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31 responses to “Biogen Turns Down Dying Patient’s Request For Tysabri—and Explains Why”

  1. This response that you have is not accurate because 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.

    The FDA said that this would not go on record against their future endevors and not apply to other cases.

    Thus, what you got was information from a PR slack.

  2. Brian says:

    Unfortunately, anyone who knows the history of Tysabri knows that at this point protecting the franchise is and should be the number one priority. The black eye that Biogen and Elan received from immuno-suppressed patients on the drug developing a brain disease and leading to the withdrawal of the drug remains a sore spot for both companies and has kept what should be a blockbuster drug with the most efficacy for MS bar none from obtaining the uptake that it deserves. Either the drug should be approved for MM or it shouldn’t, but the companies should not be subjected to compassionate use that will undoubtedly lead to negative headlines should the patient die if they cannot currently sell the drug to the thousands that currently also need it.

  3. Confused says:

    “Not apply to other cases” in which way?

    In this case, considering dozens of patients who are in the exact same condition, would it also be prudent to extend the special permission to them as well, even though they might not have Senators and Presidents as friends, granted that the Doctor who figured out in the lab that the markers match with Tysabri would be willing to perform the same test to save other lives too? Surely it is not too much effort to do this test to save other lives.

    Or should this permission not be extended to others? What do you (in the plural) think?

    This case is not as special as one might think. What is the right course of action under this situation — in general? (from an ethical viewpoint, different rules should not apply depending on if one can get Clinton to write an email or not).

  4. mike k says:

    As an MS sufferer currently on Tysabri, I agree with Biogen. I don’t think we should jeopordize the drug’s availability for currently approved conditions. As much as I want all who might benefit from Tysabri to get it, if there is a chance that further developments might take it off the shelf again, it is not worth the risk to us. They should, however, fast track studies for any conditions it may help.

  5. Jerrold says:

    Wow, the biotech companies are even more useless than I thought. They:
    * require hundreds of millions to billions of dollars of investment to produce questionable or even nonexistent results
    * offer expensive treatments with questionable efficacy
    * use pharmaceutical reps and advertising to sell people treatments they don’t need
    …and now they are cold-hearted as well!

  6. I completely agree with you Jerrold, I myself have worked for a biotech R&D, Avesthagen Biotechnologies in India and can reassure you that the condition of both Clinical Trials and manufacturing are all in tantrums and complete chaos in India as well. You people back in the U.S. or Europe are not alone. Moreover stricter FDA and Drug Development of India rules are getting potentially hazardous for patients in the advanced stage to get access to life saving drugs…

  7. paul b. says:

    This is the downside to the current high-charged legal/political/regulatory environment. Tysabri has had a rocky past, due to unfortunate PML cases coming only a couple of months into the launch. This precipitated the drug’s removal from market – a move devastating to patients and the companies alike, and one that many disagreed with. In our post-VIOXX age, it was very difficult to bring the drug back. So it’s understandable that the companies are very uptight about protecting the franchise. The FDA is not an apolitical organization.

    As someone else mentioned, this case also brings up the question of those in similar circumstances who don’t have friends in high places to write letters of appeal. The aforementioned current high-charged legal environment, while making a lot of money for the Trial Lawyers Association’s constituency, levies a tangible toll on human [quality of] life and inhibits medical progress.

  8. Those who question the legal peril of the company allowing Fred Baron to use this drug are either uninformed, naive, or full of it. I was the trial lawyer who won three of the VIoxx cases against Merck. I know the family has assured Biogen they know of the potential side effects of this drug and have offered a full and complete release from any potential liability should anything go wrong. SImilarly I have offered full legal services at no charge to Biogen, such that I would even conflict out from any future suits on this or any other drug if they would only allow Fred one single dose of the drug. THey still refuse. So, they get a free pass from the FDA, no effect on any future study or use, no potential lawsuit, and a free pass from one of the nation’s most ardent pharma attorneys, yet they still refuse.

    One must ask, is this payback? Is this industry denying lifesaving opportunities to Fred because of his lifetime role in protecting consumers against industrial wrong doing? THis is wrong, and it is shameful!

    Mark Lanier

  9. Mike K says:

    My position has changed (see above). Upon re-considering, I’ve come to the decision, that I think Biogen should give the drug to those who may benefit from it, and be sheltered from catastrophic market backlash (if this is possible). I now realize that it is selfish of me to have my earlier position, that they made the right call and withhold Tysabri from anybody that it may save/help, just to keep the dollars rolling in. I think the human race is too smart to not come up with an idea that works for Biogen AND the patients who have found a breakthrough with Tysabri.

  10. Elizabeth Z says:

    As a MS patient currently using Tysabri, I am appalled that legal and regulatory considerations are standing in the way of science and the practice of medicine. The dying cancer patient should be allowed to have a dose of Tysabri in a last ditch effort to save his life. Biogen, please reconsider your decision.

  11. Ed says:

    If Biogen was worried about PR they would let Tysabri be used. They would look like saints, but it is not about PR. Historically pharma has not done what is being asked so to assume this is some sort of payback is reaching and sad.

  12. Jeff H says:

    I agree with Biogen. While these decisions are difficult for the family and company, the undercurrent of issues cannot be ignored. With use of a drug in a patient who is so ill, the adverse outcomes/lack of effect could sway others to not participate in any current trials for MM. Thus, the potential to save 1000’s of more lives may be prohibited by this one gesture of compassion.

    Clinical trials are set up to yield and identify the most qualified and beneficial patients. Once that is determined, if physicians wish to prescribe outside of the FDA bounds, then they are free to do so. By allowing any one person to enroll, Biogen jeopardizes its ability to continue further research and the cry from others who have MM, have been denied, and are not well connected. Where’s the fairness in that?

  13. Jorel says:

    Anyone who says that they agree with Biogen must work for Biogen.

    The company has been given a free pass by EVERYONE in the country who matters, yet they still refuse to help the man out.

    That is horrible, and whoever owns stock in the company should consider dumping it.

  14. Biogen and James Mullen’s refusal to grant a compassionate use of this drug to Fred Baron under these circumstances is the most shameful and inhuman thing I have seen in a long time. Shame on them.

  15. Avon33 says:

    Baron gets what he deserves.

    He has been suing big pharmas for permitting off-lable use of drugs for all these years.

  16. NyHack says:

    People need to look at the environment that paints a company like this in to the corner to make this decision. On the surface this decision makes no sense, but in reality it is probably in the company’s best interest. Do you really think they want to play the role of villain here?

    Despite FDA’s approval of compassionate use, are we to assume that if some AE currently unassociated with Tysabri cropped up when given to Mr Well Connected there would be no impact on the trial underway in MM? I would not bet my life on that. Given the regulatory climate if I’m an MS patient I am applauding Biogen’s decision.

    Mr Lainer, while they may in fact be out of legal peril to assume there would be no regulatory peril IMO would be naive.

    Hopefully all of Mr Baron’s well connected friends will work with groups like Abigail to make real and meaningful policy change that will ulitmately allow for easier access to drugs in cases like this in the future.

  17. Robert Witte says:

    No need to repeat Mark Lanier’s comments, but rest assured that Biogen has no liability whatsoever should it grant this request. In fact, the company stands to benefit should the emergency treatment prove effective. The problem is that some refuse to put aside ideological differences and self-interest, even in circumstances that call for compassion. I may not always agree with Fred Baron’s politics, but I challenge you to find someone who gives more of their time and resources to countless charitable causes. As someone who has lost a brother to leukemia, I know the brutal battle that Fred is fighting for his life. And, I cannot imagine a person so heartless — and gutless — that they would deny him or anyone else the chance to live another day. Although Fred Baron and his family are in my prayers, I also pray that someone at Biogen will muster the wisdom and compassion to put aside the rhetoric and allow Fred Baron the treatment of tysabri that just might extend his life.

  18. NyHack says:

    Robert care to elaborate on how Biogen stands to benefit if the emergency treatment prove effective? The FDA will decide to eschew its formal review process and grant an approval for Tysabri in multiple myeloma? Biogen will no longer have to conduct clinical trials in MM? Biogen will be free to give the drug to all MM patients?

    Maybe this decision is not only about one man with friends in the right places. Where were all of these friends in high places during the AdCom to put this drug back on the market for patients where there already was extensive data on its efficacy and safety?

  19. Sam B says:

    So we have assurances from lawyers, politicians and bureaucrats that if something goes wrong, they won’t hold it against Biogen.

    I wouldn’t trust any of them further than I could throw them. Their assurances aren’t worth the bulk value of the paper they’re not printed on. Political winds change at the snap of a finger and if anything, anything went wrong to give them even one bullet of ammunition against the large, profitable corporate, they’d use it, no matter what they’d promised.

    I won’t say I agree with Biogen. But I can see why they’re doing what they’re doing.

  20. bostonsci says:

    From: Andrew Michael Baron’s site:
    + Tysabri Obtained
    Thanks to the persistence and hard work of so many friends, Frederick has received Tysabri. The Mayo Clinic working with the FDA found a legal basis for this use. We have every expectation of a positive result. We cannot thank you enough for all of your thoughts and support!

  21. Skeptics says:

    I checked with my neuro yesterday over the phone. He told me that Tysabri needs a washout period for at least 2 months before taking Tysabri. Without this washout period, Ty is such a powerful drug that it would kill the patient.

    Two months!

    But Andrew stated his father has only a few days to live. Yet, he openly solicited help from the influential Democrates so that they could pressure Biogen in OKing Tysabri. Therefore if the plead is successful and the patient gets kill instantly, he could blame Ty and Biogen for negligence. It will be lawsuit city and wealth will be transferred from the shareholders to Baron’s family again. This is a setup to milking BIG bucks from the drug company.

    Also I believe he might be associated with certain hedge funds that shorted Biogen/Elan and try to create negative news to crash these companies.

    I have little faith in lawyers.

  22. DSE says:

    How ironic for Mark Lanier to plea for a one time exception for his trial lawyer buddy Fred Baron when they are so instrumental in causing pharmaceutical companies to be so cautious. Biogen Idec and Elan almost went under due to lawsuits over serious Tysabri side effects. I guess Mr. Lanier won’t agree to talk his colleagues out of dropping lawsuits against pharama companies like Biogen Idec when unavoidable side effects like PML unfortunately strike patients once the drug goes on the market. The plaintiffs’s lawyers will all claim that the pharma comanies should have endlessly expensive clinical trials to support indications like that Baron is being treated for, or to discern adverse side effects that occur in a tiny proportion of patients.

    Meanwhile, Baron was well-connected enough to employ an army of Democratic politicans intent on regulating if not prohibiting off label use of drugs (e.g. off label use is specifically prohibited with Tysabri) to plea his case. Too bad 99.9% of patients don’t have the money or connections that would allow their doctor to do what was done for Mr. Baron, and it appears Mr. Lanier’s offer not to sue this company this one time for this product is just a one time offer. Gee, thanks

    I do wish Mr. Baron a speedy recovery. While it may be a long shot, it is amazing what American comapnies and doctors can do if allowed to practice medicine without second guessing from trial lawyers.

  23. Frederick Baron has gotten his dose of Tysabri after all. We have the update posted now here.

  24. Notalawyer says:

    Mark – there is significant legal peril for Biogen allowing Fred Baron to use this drug, and I am not naive or full of it. Regardless of the outcome, there will be a data point about the effectiveness of this drug – Andrew can claim the FDA won’t penalize the company, but that isn’t true. Who is full of it? Even if they didn’t, every trial attorney would be able to use Fred’s case to show their client someday in the future shouldn’t have been given the drug b/c of what happened to Fred.

    ” no effect on any future study or use” do you have any evidence to support that? I’ve never heard of a study that excludes relevant information when available …

    BTW, you assuring them you won’t sue them is cute – I am enough of a cynic to assume you’d use whatever information that you do gain if Fred uses the drug and pass it on to one of your partners and have them sue Biogen…

    One must ask, is this payback?

    No – it isn’t payback – it is the system you trial attorney have pushed for over the last 25 years. No risks, no informed consent, if something bad happens sue. If the only way Biogen feels safe in this case is to say no … look in the mirror.

  25. NyHack says:

    Interesting that despite Mark Lainer and Robert Witte’s claims the FDA has now in fact confirmed that anything that happens now that Mr Baron receives the drug CAN and WILL impact the FDA’s view of the drug in both the clinical trial and marketed setting???

    Please explain how you now tell potential patients in the Phase 2 trial that they may not receive study drug but if your friends are well known and famous enough we can make an exception.

    To argue that compassionate use is commonplace with drugs in phase 1 trials is just plain false. I’m happy for Mr Baron’s family but still sad for what this means to others if that makes sense. I also find it sad that doctors have provided Mr Baron’s son with “every expectation of positive results” with a drug that has barley made it in to human testing, yet in MS, patients had to fight, plead, and beg to get the drug back on the market despite plenty of data showing how effective it was as a treatment. Some world we live in.

  26. steve says:

    I don’t think Ty will help. If the report about the advance stage of MM is true, then it might be too late for any drug.

    Mayo might be a good clinic, but that outfit has been overrun by special interest. Evidence: They have been running trial for Ty in MM for months, yet they keep it a secret. Maybe only the rich and famous would be previlegd to know.

    If Fred gets Ty due to FDA’s special permission, how about Joe the plumber???

    Anyway good luck and good health Fred.

  27. Eva says:

    Get well soon Fred. We have a dinner date with you as our honor guest at Thanksgiving.

    Best of luck.

  28. evelyn munn says:

    hello…what about the use of the drug thalidomide to treat MM….side affects worse that Tsabri….I am grateful for Rush for mentioning this drug because, you see, my 52 year old son has this dreadful condition…what can I do to get this clinical trial for him….we are all so lawsuit happy and it gives companies pause…let me know..thank you…Evelyn