The Tysabri Conundrum: The Classic Risk Assessment Case
Sometimes I feel real sorry for FDA. And this is one of those times. They will be facing a grueling decision with Biogen Idec/Elan's multiple sclerosis drug natalizumab (Tysabri).
The companies announced Oct. 17 they had completed the safety evaluation of the pulled drug and found no new cases of the sometimes fatal disease, progressive multifocal leukoencephalopathy
(PML).
http://www.biogen.com/site/019_0.html
As you will recall, the drug was pulled in February after three reported cases of PML - two of which were fatal. It certainly was a doomsday scenario for the partnered firms since they - and The Street - were banking on blockbuster status for the breakthrough therapy. You see, Tysabri was shown to reduce the rate of clinical relapses by 66% in its pivotal trial study and most, including FDAers, thought that Tysabri could be a game-changer.
On a more personal note, a friend of mine, who has multiple sclerosis, was told by her doctor that a fantastic new drug had been approved for her condition and that she should consider it. Her physician also told her, in addition to his advice, to do some research on it for herself so that she could make as informed a decision as possible. She chose to hold off on Tysabri until more was known about the drug and its effects through clinical experience. It turned out to be a smart choice.
[If what FDA says and does matters to you, then make sure to attend the upcoming FDA/CMS Summit Nov. 29-Dec. 1 in Washington, D.C. A number of FDA topsiders and former FDA decision makers will be there to tell you their priorities in 2006 and help you find your way through the regulatory maze.]
Nevertheless, Biogen Idec and Elan did not give up because they had so much riding on the drug, including two separate indications already in the works. As I told you in July in my accelerated approval story, Biogen hired McKinsey & Co. to put together a very aggressive risk management program for Tysabri that would help get the drug back on the market.
The company now has data to give FDA on the drug's safety. Almost 90% of the 1,500 patients that have participated or are participating in a Tysabri study agreed to take part in a safety evaluation. Almost all of them - 98% - took a neurological exam and MRI. They found no new cases of PML.
So the question is: What will FDA do now? That's a tough one. But here's my guess: they will bring Tysabri back on the market with a black box, FDA's strongest warning. Re-entry will also come with as stringent a risk management plan as has ever been seen at the agency.
Still, I recall a conversation with one of FDA's top reviewer who had intimate knowledge of the Tysabri approval. He said that Tysabri represented the "classic" risk-benefit assessment dilemma. The PML cases shifted the argument way over to the "risk" side and the drug was withdrawn. Will the new safety data see-saw it back in the other direction? We'll have to wait and see.
Sometimes I feel real sorry for FDA. And this is one of those times. They will be facing a grueling decision with Biogen Idec/Elan's multiple sclerosis drug natalizumab (Tysabri).
The companies announced Oct. 17 they had completed the safety evaluation of the pulled drug and found no new cases of the sometimes fatal disease, progressive multifocal leukoencephalopathy
(PML).
http://www.biogen.com/site/019_0.html
As you will recall, the drug was pulled in February after three reported cases of PML - two of which were fatal. It certainly was a doomsday scenario for the partnered firms since they - and The Street - were banking on blockbuster status for the breakthrough therapy. You see, Tysabri was shown to reduce the rate of clinical relapses by 66% in its pivotal trial study and most, including FDAers, thought that Tysabri could be a game-changer.
On a more personal note, a friend of mine, who has multiple sclerosis, was told by her doctor that a fantastic new drug had been approved for her condition and that she should consider it. Her physician also told her, in addition to his advice, to do some research on it for herself so that she could make as informed a decision as possible. She chose to hold off on Tysabri until more was known about the drug and its effects through clinical experience. It turned out to be a smart choice.
[If what FDA says and does matters to you, then make sure to attend the upcoming FDA/CMS Summit Nov. 29-Dec. 1 in Washington, D.C. A number of FDA topsiders and former FDA decision makers will be there to tell you their priorities in 2006 and help you find your way through the regulatory maze.]
Nevertheless, Biogen Idec and Elan did not give up because they had so much riding on the drug, including two separate indications already in the works. As I told you in July in my accelerated approval story, Biogen hired McKinsey & Co. to put together a very aggressive risk management program for Tysabri that would help get the drug back on the market.
The company now has data to give FDA on the drug's safety. Almost 90% of the 1,500 patients that have participated or are participating in a Tysabri study agreed to take part in a safety evaluation. Almost all of them - 98% - took a neurological exam and MRI. They found no new cases of PML.
So the question is: What will FDA do now? That's a tough one. But here's my guess: they will bring Tysabri back on the market with a black box, FDA's strongest warning. Re-entry will also come with as stringent a risk management plan as has ever been seen at the agency.
Still, I recall a conversation with one of FDA's top reviewer who had intimate knowledge of the Tysabri approval. He said that Tysabri represented the "classic" risk-benefit assessment dilemma. The PML cases shifted the argument way over to the "risk" side and the drug was withdrawn. Will the new safety data see-saw it back in the other direction? We'll have to wait and see.
posted by Ramsey Baghdadi at 6:25 PM
0 Comments:
Post a Comment
<< Home