There are two sides to this discussion, (I suppose it wouldn't be much of a discussion if there weren't), demonstrated by the comments at the bottom of the page:
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Does the FDA hinder progress?
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TwistedHelix
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HarryZ
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Re: Does the FDA hinder progress?
Dom,
I don't have a problem shifting some of the focus from safety to a risk/benefit situation for new drugs but doing that relies very heavily on one particular item.....that being the honesty of the drug company providing the data to the FDA!
And from what I have seen in following MS research for over 40 years, that would be a VERY risky proposition!!
Harry
I don't have a problem shifting some of the focus from safety to a risk/benefit situation for new drugs but doing that relies very heavily on one particular item.....that being the honesty of the drug company providing the data to the FDA!
And from what I have seen in following MS research for over 40 years, that would be a VERY risky proposition!!
Harry
Well and here's another angle: is the FDA only focused on safety? Nope it also wants "efficacy" proven which is a really good thing -sort of.
Case in point, autologous stem cells for "autoimmune" diseases, which means taking cells from your hip and growing them in the lab then regiving them as a very very specialized kind of personal blood cell transfusion. No chance of rejection; it is your own cells, and in this case I am talking about getting the stem cells with NO CHEMO and no radiation. Just stem cells all by themselves.
We do this already all the time, we take people's blood filter it into specialized components and give them, for example, "packed cells" which is to say we filter out the other kinds of cells to give red cells specifically to someone.
Or we might give someone plasma specifically for other diseases.
There is no difference to filtering out red cells to regive someone or stem cells. Just different targets.
If you have leukemia, even if you are a baby, you will get a bag of these stem cells (though probably somone else's unless they saved your cord blood) although it happens in that case after some seious life threatening chemotherapy to kill off your leukemia filled bone marrow (which is why they don't get their own stem cells from marrow).
There are a number of papers out showing that giving stem cells to people with autoimmune diseases results in immunomodulation and some regeneration. These are small studies, and not yet of numbers to show efficacy.
But no one can argue it is unsafe, it isn't.
This is absolutely completely safe procedure, no question.
I asked my neuro could I get it off label if we paid for it ourselves? No way it is not proven.
Well without a clear business model that recoups costs of doing the large trials that would show efficacy it is hard to see how the "efficacy" hurdle will be crossed.....
Meanwhile it is done in China for MS.
If I was cynical I might suspect the FDA of protecting drug markets. I try to retain my innocent belief in the good of people though.
But on to what is what:
IMHO the FDA ought not stand in the way of procedures that are guaranteed safe. I can see that this might open the way for quacky stuff like maybe, let's say, Rife machines, which currently are not FDA approved and that fact is sometimes used against natural practitioners as a way to prosecute them for duping people. The key ought to be claims made; if you claim it cures something when that is not 'proven" thing then that is the litmus test
But maybe the FDA could have a special form/relase for things that are not yet proven efficacious, but are safe and have promise and this form would state clearly that the procedure while safe is not known necessarily to be helpful.
I think this should be done and I'll tell why:
The FDA currently has drugs on the market it KNOWs are unsafe, they still allow them to be marketed and sold with a "blackbox".
How absurd to have a blackbox for the lack of safety and not for lack of PROVEN efficacy when safety is not in question.
Let's have yellow box warnings: this proceudre is not yet proven helpful but is safe. A form stating such must be signed by the patient.
Then the FDA could enter you in a data base and keep track of what happens to you later on. They do this for adverse events with drugs now already.
marie
Case in point, autologous stem cells for "autoimmune" diseases, which means taking cells from your hip and growing them in the lab then regiving them as a very very specialized kind of personal blood cell transfusion. No chance of rejection; it is your own cells, and in this case I am talking about getting the stem cells with NO CHEMO and no radiation. Just stem cells all by themselves.
We do this already all the time, we take people's blood filter it into specialized components and give them, for example, "packed cells" which is to say we filter out the other kinds of cells to give red cells specifically to someone.
Or we might give someone plasma specifically for other diseases.
There is no difference to filtering out red cells to regive someone or stem cells. Just different targets.
If you have leukemia, even if you are a baby, you will get a bag of these stem cells (though probably somone else's unless they saved your cord blood) although it happens in that case after some seious life threatening chemotherapy to kill off your leukemia filled bone marrow (which is why they don't get their own stem cells from marrow).
There are a number of papers out showing that giving stem cells to people with autoimmune diseases results in immunomodulation and some regeneration. These are small studies, and not yet of numbers to show efficacy.
But no one can argue it is unsafe, it isn't.
This is absolutely completely safe procedure, no question.
I asked my neuro could I get it off label if we paid for it ourselves? No way it is not proven.
Well without a clear business model that recoups costs of doing the large trials that would show efficacy it is hard to see how the "efficacy" hurdle will be crossed.....
Meanwhile it is done in China for MS.
If I was cynical I might suspect the FDA of protecting drug markets. I try to retain my innocent belief in the good of people though.
But on to what is what:
IMHO the FDA ought not stand in the way of procedures that are guaranteed safe. I can see that this might open the way for quacky stuff like maybe, let's say, Rife machines, which currently are not FDA approved and that fact is sometimes used against natural practitioners as a way to prosecute them for duping people. The key ought to be claims made; if you claim it cures something when that is not 'proven" thing then that is the litmus test
But maybe the FDA could have a special form/relase for things that are not yet proven efficacious, but are safe and have promise and this form would state clearly that the procedure while safe is not known necessarily to be helpful.
I think this should be done and I'll tell why:
The FDA currently has drugs on the market it KNOWs are unsafe, they still allow them to be marketed and sold with a "blackbox".
How absurd to have a blackbox for the lack of safety and not for lack of PROVEN efficacy when safety is not in question.
Let's have yellow box warnings: this proceudre is not yet proven helpful but is safe. A form stating such must be signed by the patient.
Then the FDA could enter you in a data base and keep track of what happens to you later on. They do this for adverse events with drugs now already.
marie
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cheerleader
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Marie-
All really good, pertinent points.
The FDA has granted "orphan drug" status designation for the use of stem cells by Angioblast Systems, Inc....
www.angioblast.com
"New York, USA; 17 September 2008: Angioblast Systems Inc., announced today that the United States Food and Drug Administration (FDA) has granted an orphan drug designation for the use of the patented adult stem cell technology in patients undergoing bone marrow transplantation.
The FDA awarded Angioblast Systems, Inc. the right to use the proprietary
“off-the-shelf” allogeneic mesenchymal precursor cells for insufficient
haematopoietic stem cell production in patients with hematologic
malignancies who have failed treatment with conventional chemotherapy."
I find this really ironic...an orphan drug status for a patient's own stem cells! Is this just about "patenting" the procedure? I'm going to play the cynic and say that I think it's because they don't want to lose out on money they receive for licensing fees. I know that the FDA expects user fees from drug companies...will they expect fees for your own stem cells???
Arghh.
Maybe they'll be some changes with the new admin-
AC
All really good, pertinent points.
The FDA has granted "orphan drug" status designation for the use of stem cells by Angioblast Systems, Inc....
www.angioblast.com
"New York, USA; 17 September 2008: Angioblast Systems Inc., announced today that the United States Food and Drug Administration (FDA) has granted an orphan drug designation for the use of the patented adult stem cell technology in patients undergoing bone marrow transplantation.
The FDA awarded Angioblast Systems, Inc. the right to use the proprietary
“off-the-shelf” allogeneic mesenchymal precursor cells for insufficient
haematopoietic stem cell production in patients with hematologic
malignancies who have failed treatment with conventional chemotherapy."
I find this really ironic...an orphan drug status for a patient's own stem cells! Is this just about "patenting" the procedure? I'm going to play the cynic and say that I think it's because they don't want to lose out on money they receive for licensing fees. I know that the FDA expects user fees from drug companies...will they expect fees for your own stem cells???
Arghh.
Maybe they'll be some changes with the new admin-
AC
Husband dx RRMS 3/07
dx dual jugular vein stenosis (CCSVI) 4/09
http://ccsviinms.blogspot.com
dx dual jugular vein stenosis (CCSVI) 4/09
http://ccsviinms.blogspot.com
Hi Cheer,
The thought of gene patenting is really creepy to me. I'm with you on that! In fact, the guy that was profiled on "half man half tree" on TLC was treated by an American doc who took blood samples. I read that the guy's ministry of health refused to let the sample go out of the country for fear the gene responsible would be patented by the US and thus take a chance for biotech away from the indonesian country that is his home...not that they were knocking themselves out to help him mind you. End result no treatment. Can you believe it?
The key in the thing you posted I think is the word allogenic which means not from you to yourself but from someone else to you. The off the shelf thing must be a stem cell line they are keeping alive in the lab or something, it sounds like the people they want it for are those unable to get enough otherwise rather than them patenting your own stem cells.
Healthcare is a funny business. It'd be funny if it was just academic, but it is deadly serious for us.......
marie
The thought of gene patenting is really creepy to me. I'm with you on that! In fact, the guy that was profiled on "half man half tree" on TLC was treated by an American doc who took blood samples. I read that the guy's ministry of health refused to let the sample go out of the country for fear the gene responsible would be patented by the US and thus take a chance for biotech away from the indonesian country that is his home...not that they were knocking themselves out to help him mind you. End result no treatment. Can you believe it?
The key in the thing you posted I think is the word allogenic which means not from you to yourself but from someone else to you. The off the shelf thing must be a stem cell line they are keeping alive in the lab or something, it sounds like the people they want it for are those unable to get enough otherwise rather than them patenting your own stem cells.
Healthcare is a funny business. It'd be funny if it was just academic, but it is deadly serious for us.......
marie
If the FDA speeds the approval process and then problems surface once the drug is on the market they are to blame. If they take time to fully evaluate safety and efficacy and that delays a potential treatment they are to blame. It is a balancing act for the administration. I know that for sure. To speculate that they would delay a potentially life saving treatment to the market so they could collect some fees is a wild reckless speculation. Black helicopters and such.....