Fred Baron Receives Tysabri, After All
24 CommentsBy Ed Silverman // October 16th, 2008 // 7:51 pm
After several days of drama and debate over death, privilege and compassionate use, Fred Baron will receive the biologic medication to treat his multiple myeolma, according to a brief statement posted by on a blog written by his son, Andrew. The turnabout came after direct pleas by several high-profile personalities - including Hillary Clinton and Lance Armstrong - to Biogen, which sells Tysabri.
“Thanks to the persistence and hard work of so many friends, Frederick has received Tysabri,” the statement reads. “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!”
Fred Baron, you may recall, is a prominent Dallas trial lawyer who helped bankroll John Edwards’ presidential run and also paid for the former senator’s mistress to leave Chapel Hill, North Carolina, after the affair made national headlines. But the 61-year-old fundraiser was given only a few days to live and Biogen refused to make the med available to his Mayo Clinic doctors (back story).
A Biogen spokeswoman yesterday told us the biotech has a policy of not making Tysabri, which is approved only for treating multiple sclerosis and Crohn’s disease, available for any other treatment, even for compassionate use. That’s because the med was linked to a deadly brain infection - PML - which prompted a withdrawal in February 2005. Sales resumed in July 2006, but only under a closely controlled program that restricts access and usage.
The Biogen spokeswoman added that, given the drug’s complicated history, any inadvertent reactions Baron may experience could, theoretically, jeopardize its use for multiple sclerosis and Crohn’s patients if it would somehow cause labeling to change or spark another regulatory review. Biogen has similar concerns for the outcome of a Phase I clinical trial testing Tysabri for treating multiple myeloma.
UPDATE: On Friday morning, the Biogen spokeswoman called us to say “we didn’t change our minds…The FDA notified us they would be working directly with Mayo (which is one of the site for Biogen’s Phase I clinical trial)…Essentially, they cut us out of the loop. In terms of implications (for the clinical trial or any legal issues), it’s too early to say.” She declined to say whether Biogen was holding talks with the FDA to further clarify any of this.
This morning, an FDA spokeswoman wrote us to say the agency was in touch with Biogen and Baron’s doctors, but hadn’t made any promise to the biotech about implications for its clinical trial: “The FDA is working with the company and the health care professionals involved - as we would with any such request - under the provisions of FDA’s expanded access program…
“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. 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.”
Steve
Please read my comments on the same, but earlier article.
Mr. Baron will get his drugs.
Just A Thought
I am happy that Mr. Baron is getting this treatment. Hoping with all sincerity that it works miraculously.
I do have to say though, as nobody of any significance, this hurts my heart a bit. Months spent trying to bring attention to those who were and are still being harmed by a drug reformulation that wasn’t handled well by the manufacturer or the FDA. Having contacted many of the very people who threw their support behind Barton’s cause.
There are whole groups of people who are more expendable than this one man.
Regardless, I would like to extend to him my best wishes.
Dan
As a human being to another human being, I am glad he got the use.
I now encourage every dying person to call Senators Clinton, Edwards, Kerry, and Kennedy and request help. And I encourage them to post on the web if they get that help or not. I fully expect that this will prove that the special interests control these Senators and not compassion or good governance.
Kerry
I am very happy to see the computer that wore tennis shoes (FDA) make a quick and humane decision to help Mr. Baron. I sincerely applaud the FDA for acting in a reasonable and compassionate way.
Now, if we could just extend that kind of common sense and compassion to help a broader population….
May the good lord give Jesse Goodman (head of CBER division of FDA) the courage to do the right thing and help late stage prostate cancer patients. Please G_d give him the courage!
Nathan
I genuinely hope that this drug helps Fred Barron. But I agree with Dan. There were 10,790 people who died from multiple myeloma in 2007. Similar numbers are expected in 2008. Every one of those 11,000 people at death’s door should write to Senators Clinton, Edwards, Kerry, and Kennedy and see if it gets them anywhere.
I wonder if those senators would have advocated for the same treatment for Carl Rove if he was the one on death’s door.
What a crazy system.
Justice in MI
“What a crazy system.”
Totally agree.
kyoto27
Nice to have ‘our pols’ remind us that the ‘caste system America’ is functioning as it should…in spite of everything else going to hell in a handbasket. Too bad our representatives in the Capital Building don’t wave their magic wand more often to do good for the good of all who sent them there….
Doug Bremner MD
Oops, put it on the wrong post. To reiterate.
Yeah! Are they gonna give it to the welfare cheats as well?
NyHack
Steve and former PME,
Happen to read the FDA’s comments? Anything that happens now that Mr Well Connected receives the drug CAN and WILL impact the FDA’s view of the drug in both the clinical trial and marketed setting???
Glad you all think this is a great triumph over the evil drug industry. Please explain how you now tell potential patients in the Phase 2 trial that they may not receive 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 Barons 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.
Nathan
This situation made me think of the irony of the pharma buisness of late. People are busy shouting the praises of unproven (non-FDA approved) cancer medications (like this one) while the are also busy yelling and suing about all the side effects of FDA aproved medications that have been shown conclusively to be effective.
Is anyone suprised that pharma is shifting massive amounts of resources into oncology research? Part of it is certainly medical need. But much of the reason also lies simply in the fickleness of public attitutes towards drugs.
see this link:
http://www.businessweek.com/magazine/content/08_42/b4104072875569.htm?
Christopher
Nathan - well put. The triumph of hope over reality is never far away with cancer drugs and this episode demonstrates it very well.
pharma PR hack
Nathan- The pharma business isn’t shifting vast amounts of research to oncology because it is good PR but because it is good business! While Pfizer eaked out a 0.7% growth in profit margin last year- Genentech and Biogen Idec each enjoyed 20%+ growth in profit margin- not to mention that each of their drugs are all over the 70% threshold in profit margins. As even inside trade magazine pharma exec put it those are nosebleed margins at best!!!
As Mr. Baron’s case illustrates perfectly- when faced with extreme life or death issues - there is no amount of effort and payment that individuals won’t personally go to or ensure that government agencies and insurers to go as well.
You see that in the UK where individuals are suing to have Tykerb paid for- You see that in Australia where patients were picketing for Herceptin.
Globally in the most extreme government managed care market - oncology drugs still are profitable.
But pharma is always saying prices are necessary to ensure R&D continues but if everyone is chasing sky high price possibilities with oncology drugs to replace the all but gone blockbuster model and feed the insatiable wall street need - where does that really leave research and development?
MyPharmalotID
“All animals are equal, but some animals are more equal than others”
A proclamation by the pigs who control the government in the novel Animal Farm, by George Orwell. The sentence is a comment on the hypocrisy of governments that proclaim the absolute equality of their citizens but give power and privileges to a small elite.
http://www.bartleby.com/59/6/allanimalsar.html
How much more corrupt an organization can the FDA become than it already is?
Who does one have to know in the FDA or elsewhere to get Provenge?
Is that how life saving drugs are made available in the United States of America?
I’m happy that Mr. Baron, at the least, was able to receive Tysabri, but what does this say about the FDA?
About our Country?
About what the lives of the 43,000 men who have died of PCa since May, 2007, are worth?
Are their lives worth nothing? This is a travesty.
Provenge was voted 17-0 to be safe, and 13-4 to be effective by an FDA Advisory Committee on March 29, 2007. Given the events that have transpired regarding a drug that has been pulled off the market once because of safety concerns, one really does have to ask what CBER’s problem is with Provenge?!
MikeK
In regards to Fred Baron and Tysabri, did the FDA finally discover Constitutional LAW and its own POLICY?
“The Mayo Clinic working with the FDA found a legal basis for this use.”
Is this referring to our Constitution which guarantees the right to life, liberty and the pursuit of happiness?
The FDA appears to agree with CareToLive about the worth of just one single life (see “What is a Life Worth?” at http://www.caretolive.com).
In allowing Fred Baron to receive Tysabri, the FDA said its “policy is to consider all available information when evaluating the safety and effectiveness of drugs.” Is this true? They consider ALL AVAILABLE INFORMATION? How about the recent interim information that was just reported to Dendreon about Provenge for prostate cancer? The independent data monitoring committee (IDMC) reported to Dendreon a 20% reduction in the risk of death in the PROVENGE arm relative to placebo. The IDMC observed no safety concerns.
There are 96,000 men who are eligible for Provenge today. They are painfully fighting a lethal disease with no other treatment options in sight. If at least 20% survive longer with Provenge, that means 19,200 men, and their families, get to enjoy their life longer. If one life is worth it (Mr. Baron), then surely all the other lives are as precious as well.
SNAP
NyHack - Please provide the basis in fact for your statement: “To argue that compassionate use is commonplace with drugs in phase 1 trials is just plain false.” What information do you have to back this up? I don’t think you have any. I’ve seen the medical officers at the FDA bend over backwards to try to grant single patient exemptions for non-well connected patients as the rule, not the exception. They just don’t get the publicity that this case received. BTW — although commonly used, the term “compassionate use” is NOT in the IND regulations. Please do your homework before you make statements such as this.
FDA employee
Steve
SNAP
Thanks for your professional comments. Nice to see objective handling rather than one presenting what they think is right or should be.
By the way, the Constitution does not extend to the FDA as a separate entity. Equal rights does not mean equal health benefits. If there was “equality” across the board, our country would make a nice communist regime. We are where we are because of the democratic system helped by competition or can I say, capitalism!
Former pharma Marketing Exec
Well Folks, the next time you want to rant here how the little guy never gets his share, and how this “Mr. Somebody” jumped the cue, please research your facts better.
There are some prominent cases of ordinary folks petitioning drug companies and it has worked. These were for drugs in phase I and Phase II
What I disagreed with was the reason Biogen used to deny access.
That being said, I concur with Nathan and the others who suggested that anyone need access to the drug, then petition all the people who helped Mr. Barton.
I personally do not think the Pharma world is an evil empire. I think some of the decisions they make, along with the FDA are “crazy” and not well thought out. However, sometimes, and unfortunately, those times are rare, they do get it right.
Patients should beware though, if there is too much commotion made and many people are clamoring for the drug, well, the price will no doubt go up.
How can this crazy system be fixed? We shouldn’t be putting the lives of fellow human beings at the mercy of for profit businesses. But, since that is unlikely to change, this is what we get.
With regards to the comments on the constitution, if one were quite savy in the art of debate, one might find that health issues could, COULD, be covered under the 14th amendment - (hmm, it could also provide insight for preemption)
“No State shall make or enforce any law which shall abridge the privileges or immunities of citizens of the United States; nor shall any State deprive any person of life, liberty, or property, without due process of law; nor deny to any person within its jurisdiction the equal protection of the laws.”
One could argue that blocking access to potentially life saving drugs and devices, ensuring certain death, might not be in keeping with the spirit of the 14th amendment.
Not anti pharma, just wishing they would get it right more often.
Snap, thanks for your comments.
Anne PME
Glad to hear that Mr. Baron will gain access to Tysabri. Best wishes for a successful outcome.
By exposing the good, the bad and the dysfunction of a system where the medical advice and counsel of politicians and celebrities means more than a treating physician’s recommendation, Ed Silverman and Pharmalot likely helped Mr. Baron’s physicians close this deal.
I look at all of the resources that have been expended to help Mr. Baron gain (hopefully prompt) access to treatment and I can’t help but think that they would be better spent on actually providing treating physician recomended therapies and services to cancer and other patients.
All of us have had the time to post about this topic. With the exception of Ed, would our time be better spent volunteering to help a cancer (or other) patient rather than posting about it? It probably would be, but unfortunately in a wasteful and dysfuntional system like the US Health system, things don’t work that way.
Former pharma Marketing Exec
Anne PME,
I think you and others are missing a point here.
By creating this “noise” about accessing this drug, I hope more patients are now more aware of its existence.
Each of the high profile people who called on Mr. Baron’s behalf to plead his case and get access have been generous to cancer patients in many ways.
It is by shinning a light into the dark corners that Pharma would like to remain dark - sometimes, that we can be of benefit to people.
As for me, I can personally state that I spend a great deal of time advocating, working and caring for cancer patients. Providing input on issues as important as this one, is also key to helping patients.
But you are right, this is a wasteful and mostly dysfunctional health care system.
Stay healthy!
Paula Wittekind
If you didn’t watch ‘Living Proof’ on Lifetime last night, I’d recommend you try to catch it tonight. It’s on at 8:00 p.m.ET Based on a true story about a breast cancer treatment,
it’s pretty inclusive about the struggle to get a drug from pre-clinical to market and includes : time span, funding struggle, importance of celebrities, sparse info and lack of compassion from medical community, importance of patient advocates at the trial design table, impossible standards that doom trials to failure, the need for a relentless champion of the treatment…..and more.
The pharmas are the bad guys and will remain so in the public’s eye until they stop paying their CEO’s unacceptable amounts of money, with the research mafia close behind accepting money for delivering shabby and biased research results. When people are placed at the top of the prioity list, private jets may disappear and the world will be a healthier place.
Oh, do I sound cynical? I learned it all trying to get GDNF from Amgen for Parkinson’s patients.
louise
dear all,
the rules of life are try to stay well,
for the is so much greed and money to be made
upon those who are ill,
and bigpharma has no heart,
just very large wallets -
the cures are simple -
but pills and meds are magic to those who have been subliminally told - this is the cure,
eat mcdonalds, drink diet pepsi w/ aspartame,
we are the fda, we are owned by bigpharma…
we will sell own souls for bigmoney,
because the AMA doesnt make a dime if its population is healthy…
exception -
the nobel winners lose their own rights to cures…
Otto Warburg never was able to publish,
his cancer cure book -
pH balanced systems - is the key to all good health…because your body is in harmony…
drugs kill the liver, acidify the system,
the druglords of bigpharma should wake up -
because the more you drugs us with chemo,
and radiate us, feed us poison by way of the monsanto crap crops -
soon we shall all be dead, and bigpharma will have no one to suck blood money from~
sincerely,
louise
louise
PS:
Albert Einstein: “Three Rules of Work: Out of clutter find simplicity; From discord find harmony; In the middle of difficulty lies opportunity.”
Anne PME
Former pharma Marketing Exec, thanks for the post.
Let me try to respond to your post. For patients like Fred Baron and the doctors who treat them, it is about keeping the focus on the war against life threatening and/or terminal disease. This would be much easier w/out all of the secondary battles and strains on resources…all of the complications that make a patient want to stop fighting and create an incentive to stop treating physicians from advocating for their patients.
I hope that this treatment is successful for Fred Baron, but I know that it he would have a greater chance of successful outcome if he, his family and his doctors did not have to go through hoops to gain access to what his doctors and pharmacist believe may help him.
NyHack
SNAP apologies for coming back to this so late, but with Ed’s new post I took a read back through the comments.
As the first step of that single patient IND process for emergency or compassionate use, a physcian must request permission from the manufacturer. Without that permission generally the drug will not be made available.
I do not work at the FDA but can assure you having worked with numerous drug companies, that they are NOT in the business of granting approval for a single patient IND for drugs that just entered a Phase 1 trial. While most companies do not make their formal “Compassionate Use” policies public, those that do (Pfizer is one) note that its generaly considered around the beginning of Phase 3.