Meget god opsummering, hvis man lige vil have den korte udgave af forhistorien..
http://blogs.forbes.com/matthewherper/2010/10/29/is-provenge-the-most-important-medicine-in-biotech/?partner=yahootix
Is Provenge The Most Important Medicine In Biotech?
Oct. 29 2010 - 4:33 pm | 5,088 views | 0 recommendations | 9 comments
By MATTHEW HERPER
Provenge, a treatment that trains immune cells to attack a cancer, would be revolutionary even if there were nothing to it but cool science. But the way it has been studied, the hurdles it is still facing, and the high price that its manufacturer, Dendreon, is charging could all affect the pharmaceutical industry for years to come.
That’s why when Forbes executive editor Michael Noer asked me what biotech-related entrants should be considered for the project we’re tentatively calling Names You Need To Know in 2011, Provenge leapt to mind. But Michael doesn’t just want my opinion, he wants yours. In his words, this is an effort to “crowd-source part of the magazine” and “make media history.” Click here to read Michael’s blog post explaining this project. Is Provenge a Name You Need To Know in 2011? Leave your comments below, and on the Names You Need To Know page.
Here’s my take: Provenge is the first ‘vaccine’ proven to lengthen patients lives by teaching immune cells to attack tumors. This is an idea that has been around for years. Bone marrow transplants help fight cancer by spurring immune response, as does one of the main treatments for bladder cancer. But attempts to create treatments that harness immune response have mostly resulted in money sent down the clinical trial drain, as well as dashed hopes for patients and investors alike. But Provenge made it. It was approved in April 2010. A well-conducted clinical trial published in the New England Journal of Medicine shows that it extends the median survival of men with metastatic prostate cancer by four months.
There were some dramatic bumps along the way to the market, though. Dendreon filed for approval before that big, well-conducted trial was finished. This was a move of Captain-Kirk-like chutzpah on the part of chief executive Mitchell Gold, because the two late-stage studies the company had conducted had both, by the measures of clinical trial statistics, failed. They also appeared to show that Provenge extended patients’ lives.
A bit of explanation: clinical trials are like shooting pool. You have to call your shot (8-ball in the corner pocket!) in order for it to count. This is because any clinical trial is really only able to answer one question. If every potential outcome were measured, something would always come up positive just by chance. In neither of its first two clinical trials did Dendreon even specify that Provenge would actually increase patients’ lives. What if the results were a fluke? Dendreon’s move made some oncologists and statisticians quietly fume.
If Provenge had been reviewed by the division of the FDA that usually decides on cancer drugs, regulators might have summarily rejected it. But instead it pled its case to an easier crowd, a panel of experts in cell therapies. (In order to get an immune response, administering Provenge involves taking immune cells out of patients’ blood, altering them, and infusing them back in.)
The panel voted 13 to 4 that Provenge should be approved, causing Dendreon shares to quadruple. But some top oncologists protested and the FDA decided to reject Provenge until Dendreon’s big study – the one that was later published in the New England Journal– completed. This led to protests outside the FDA on the part of investor-patient groups and set a new tone of animosity on the part of many biotech investors toward the agency. It also meant that when the FDA finally did approve Provenge in 2010, it did so without a public meeting vetting that big clinical trial. Provenge hit the market with a price of $93,000 per patient.
Although currently Provenge sales are limited by supply constraints, there’s no doubt that it is going to be a big seller (see this video). But Medicare, which will pay for much of the treatment of men with Provenge, is pushing back, conducting a National Coverage Assessment – a review similar to what the FDA would have done if it had held its expert panel after the big, definitive study had come out. Some anonymous critics have been alleging that the study is not that definitive at all. I don’t think Medicare is looking to invalidate Provenge, but I’m sure there’s an angle behind the NCA. One possibility is that it hopes to limit how much it pays for the use of new cancer drugs. Traditionally, it will pay for treatment even in some areas that are not FDA approved.
Because the Provenge story hits so many different angles of medicine – scientific, regulatory, and economic – I think Provenge is a shoe-in as a Name You Need To Know in 2011. Am I right? Or am I right that it belongs on our list, but completely wrong in my reasoning? Are there other names we should be watching when it comes to medicine in 2011? Let us know in the comments. We are listening.
http://blogs.forbes.com/matthewherper/2010/10/29/is-provenge-the-most-important-medicine-in-biotech/?partner=yahootix
Is Provenge The Most Important Medicine In Biotech?
Oct. 29 2010 - 4:33 pm | 5,088 views | 0 recommendations | 9 comments
By MATTHEW HERPER
Provenge, a treatment that trains immune cells to attack a cancer, would be revolutionary even if there were nothing to it but cool science. But the way it has been studied, the hurdles it is still facing, and the high price that its manufacturer, Dendreon, is charging could all affect the pharmaceutical industry for years to come.
That’s why when Forbes executive editor Michael Noer asked me what biotech-related entrants should be considered for the project we’re tentatively calling Names You Need To Know in 2011, Provenge leapt to mind. But Michael doesn’t just want my opinion, he wants yours. In his words, this is an effort to “crowd-source part of the magazine” and “make media history.” Click here to read Michael’s blog post explaining this project. Is Provenge a Name You Need To Know in 2011? Leave your comments below, and on the Names You Need To Know page.
Here’s my take: Provenge is the first ‘vaccine’ proven to lengthen patients lives by teaching immune cells to attack tumors. This is an idea that has been around for years. Bone marrow transplants help fight cancer by spurring immune response, as does one of the main treatments for bladder cancer. But attempts to create treatments that harness immune response have mostly resulted in money sent down the clinical trial drain, as well as dashed hopes for patients and investors alike. But Provenge made it. It was approved in April 2010. A well-conducted clinical trial published in the New England Journal of Medicine shows that it extends the median survival of men with metastatic prostate cancer by four months.
There were some dramatic bumps along the way to the market, though. Dendreon filed for approval before that big, well-conducted trial was finished. This was a move of Captain-Kirk-like chutzpah on the part of chief executive Mitchell Gold, because the two late-stage studies the company had conducted had both, by the measures of clinical trial statistics, failed. They also appeared to show that Provenge extended patients’ lives.
A bit of explanation: clinical trials are like shooting pool. You have to call your shot (8-ball in the corner pocket!) in order for it to count. This is because any clinical trial is really only able to answer one question. If every potential outcome were measured, something would always come up positive just by chance. In neither of its first two clinical trials did Dendreon even specify that Provenge would actually increase patients’ lives. What if the results were a fluke? Dendreon’s move made some oncologists and statisticians quietly fume.
If Provenge had been reviewed by the division of the FDA that usually decides on cancer drugs, regulators might have summarily rejected it. But instead it pled its case to an easier crowd, a panel of experts in cell therapies. (In order to get an immune response, administering Provenge involves taking immune cells out of patients’ blood, altering them, and infusing them back in.)
The panel voted 13 to 4 that Provenge should be approved, causing Dendreon shares to quadruple. But some top oncologists protested and the FDA decided to reject Provenge until Dendreon’s big study – the one that was later published in the New England Journal– completed. This led to protests outside the FDA on the part of investor-patient groups and set a new tone of animosity on the part of many biotech investors toward the agency. It also meant that when the FDA finally did approve Provenge in 2010, it did so without a public meeting vetting that big clinical trial. Provenge hit the market with a price of $93,000 per patient.
Although currently Provenge sales are limited by supply constraints, there’s no doubt that it is going to be a big seller (see this video). But Medicare, which will pay for much of the treatment of men with Provenge, is pushing back, conducting a National Coverage Assessment – a review similar to what the FDA would have done if it had held its expert panel after the big, definitive study had come out. Some anonymous critics have been alleging that the study is not that definitive at all. I don’t think Medicare is looking to invalidate Provenge, but I’m sure there’s an angle behind the NCA. One possibility is that it hopes to limit how much it pays for the use of new cancer drugs. Traditionally, it will pay for treatment even in some areas that are not FDA approved.
Because the Provenge story hits so many different angles of medicine – scientific, regulatory, and economic – I think Provenge is a shoe-in as a Name You Need To Know in 2011. Am I right? Or am I right that it belongs on our list, but completely wrong in my reasoning? Are there other names we should be watching when it comes to medicine in 2011? Let us know in the comments. We are listening.
4/11 2010 16:29 1970 0
35636 og så lidt om seneste kvartalsmeddelelse:
http://www.reuters.com/article/idCNN0310518320101103?rpc=44
http://www.reuters.com/article/idCNN0310518320101103?rpc=44
