Stop acceptance of the FDA power

[paolocipolla]

"Risk aversion is the inherent enemy of progress."
.....
A report recently released by the Milken Institute—“The Global Biomedical Industry: Preserving US Leadership”—documents how the wheels of progress have been slowly grinding to a halt as the FDA raises the bar for drug approval.
.......

Is there an alternative?
While wholesale FDA reform is needed, doing it right would entail a monumental effort that could take years. But there are modest steps we can take in the meantime. How about carving out one or more FDA-free Enterprise Zones where doctors, scientists, and volunteer patients can make their own decisions unfettered by the heavy hand of regulators?

By Bill Frezza

http://www.bio-itworld.com/issues/20...o-the-FDA.html


Something to seriously consider IMO.

Paolo

[Dmitriy]

Thank you for this sensible post. Finally, some common sense -- government should butt out. If we want to be cured we must take risks. We already do: how many went to get useless stem cell injections. The right relationship should be: patient/ researcher/ doctor, no regulations. They drive up the costs and make treatments not feasible economically. Stop funding basic research, and get more funding towards curing chronic SCI in people. Maybe we should all move to a Native American reservation with no regulation to find the cure.

[chris arnold]

Thank you for this sensible post. Finally, some common sense -- government should butt out. If we want to be cured we must take risks. We already do: how many went to get useless stem cell injections. The right relationship should be: patient/ researcher/ doctor, no regulations. They drive up the costs and make treatments not feasible economically. Stop funding basic research, and get more funding towards curing chronic SCI in people. Maybe we should all move to a Native American reservation with no regulation to find the cure.

Much sense in what you say. In the UK, NICE has presented similar problems.

[Eileen]

The problem with lowering the bar for new drugs is that you have situations like the thalidomide babies born with congenital abnormalities, which is party why the bar was raised in the first place. Remember when they told diabetics to stop taking Avandia? Sure, it lowered glucose levels, but at the significant risk of a heart attack, making lower glucose levels a moot point.

[paolocipolla]

The problem with lowering the bar for new drugs is that you have situations like the thalidomide babies born with congenital abnormalities, which is party why the bar was raised in the first place. Remember when they told diabetics to stop taking Avandia? Sure, it lowered glucose levels, but at the significant risk of a heart attack, making lower glucose levels a moot point.

The main point IMO is that people should have the right to choose to take risk and give informed consent.
That is too simple for our complex society, I know.

Does that makes sense to you?

Paolo

[rjames]

Totally agree with your post Paolo.

The FDA seems like a huge bureaucratic slow down in the research/cure process. Like most regulatory agencies that are started with good things in mind and then turn into these huge slow moving behemoths.

The problem as I see it now is that if it's not going to be an FDA approved treatment it will be harder to get funding to develop it as it would not be able to be sold in the US.

[GRAMMY]

But, most sci therapies and lots of research being developed overseas isn't funded by the United States anyway. I'm not totally convinced their goal is to get their therapy into the US or FDA approved anyway. In fact, many seem to avoid the US quagmire entirely or take their trials overseas. Even the US biotechs say it's unrealistic to propose 24,000 pages of data to ask permission. Many of them have satellite locations overseas and plan to do trials OUT of the USA in the future. It will put US citizens on the airlines going to other countries for treatments.

[Eileen]

The main point IMO is that people should have the right to choose to take risk and give informed consent.
That is too simple for our complex society, I know.

Does that makes sense to you?

Paolo

Paolo, I agree with you entirely, and if some brave citizens wanted to volunteer for trials I think they should be allowed to, although I can see where ultimately it could end up costing taxpayers money if their health failed or if there were complications. I agree with you that this complex society probably won't let even those willing to take the risk do so, but it would hasten results if they did.

[paolocipolla]

Totally agree with your post Paolo.

The FDA seems like a huge bureaucratic slow down in the research/cure process. Like most regulatory agencies that are started with good things in mind and then turn into these huge slow moving behemoths.

The problem as I see it now is that if it's not going to be an FDA approved treatment it will be harder to get funding to develop it as it would not be able to be sold in the US.

If I had a biotech I would look for a place to run clinical trials with a rasonable regulotory agency and at reasonable cost.
Then just when I know a therapy works I would do trial in the US.

In the case of chronic SCI I think this approach is very practical as we can fly wherever the clinical trial center is.

Paolo

[StemCells&AtomBombs]

I appreciate this common sense approach to a problem and I think it would be possible to get a country to host something like this. The problem would be that any biotech wishing to participate would want to make money at it. Without access to the biggest markets which are the ones that are "overly regulated" (actually I would say poorly regulated as opposed to overly regulated) they probably would not be interested. Of course some would be able to pay out of pocket, but this would make the market even smaller as it would be comprised of the spinal cord injured who could pay.

Furthermore, the money that this would cost would be very high and could we self fund it, I don't think so.

IMO we need to do as Paolo suggests while at the same time making sure we just don't let our own governments off the hook. Aside from us, the main beneficiaries of getting out the chair will be government as they will have people working and paying taxes as opposed to the other way around.

I'm sure that I haven't stated this as well as I would have liked, but I am interested in hearing more opinions on this issue which may help me state my position better.

[Tufelhunden]

Paolo, I agree with you entirely, and if some brave citizens wanted to volunteer for trials I think they should be allowed to, although I can see where ultimately it could end up costing taxpayers money if their health failed or if there were complications. I agree with you that this complex society probably won't let even those willing to take the risk do so, but it would hasten results if they did.

I would have to agree with Eileen on this one. This post sums it up. Throw in our sue-happy entitled culture and it shouldn't be surprising as to why it takes so long to move the envelope forward.

[0xSquidy]

Paolo, I agree with you entirely, and if some brave citizens wanted to volunteer for trials I think they should be allowed to, although I can see where ultimately it could end up costing taxpayers money if their health failed or if there were complications. I agree with you that this complex society probably won't let even those willing to take the risk do so, but it would hasten results if they did.

I would have to agree with Eileen on this one. This post sums it up. Throw in our sue-happy entitled culture and it shouldn't be surprising as to why it takes so long to move the envelope forward.

The argument of risky trials costing money to taxpayers is fallacious for several reasons. One of the reasons is the way clinical trials are escalated. First 20 patients, then several hundreds in the final stage... not the other way around.

Another reason why that argument is flawed is that the people "carelessly" applying for that trial is already costing money to taxpayers. A lot of money.

So you have a million people injured costing taxpayers a ridiculous amount of money. You only try a ""dangerous"" (we know it woulnd't be that dangerous, that's why preclinical safety data is for) trial in 20 of them at a time. If it goes really bad and i mean completely messed up, then you'll have to pay their healthcare...but wait you were already paying that, and probably a similar amount. No lawsuits whatsoever as there was informed consent.
If it goes good, you save yourself the healthcare of that million...not to mention fucking curing SCI.

There are more reasons but seriously (and i don't know if i explained myself very well, been sick these days), just think about the argument and you'll find out it's completely stupid, no offense.

[lynnifer]

If I had a biotech I would look for a place to run clinical trials with a rasonable regulotory agency and at reasonable cost.
Then just when I know a therapy works I would do trial in the US.

In the case of chronic SCI I think this approach is very practical as we can fly wherever the clinical trial center is.

Paolo

Whatever happened with MacDonald and the primates in South America? Or was that just a rumor? Or an idea that never met fruition because it was there and then fell off the radar. This would have been before most of you were in your chairs - does anyone know?

[Tufelhunden]

No lawsuits whatsoever as there was informed consent.

This is what you would hope happens, but unfortunately that is not reality.

[Wise Young]

I am going to play a bit of "devil's advocate" here. The FDA is everybody's favorite punching bag but I don't think that they are the problem. The problem is the risk-averse behavior of therapeutic companies and their unwillingness to invest in clinical trials on treatments for "orphan diseases". The FDA was charged with ensuring the safety of drugs, devices, and food, as well as truth in labelling of products. In order to get approval to market products for certain indications, companies have to carry out clinical trials. Unfortunately, the cost of clinical trials have gone up probably 100x in the past two years.

Companies complain that it now costs them over $2 billion to move a therapy from discovery to market. In reality, it costs less than a billion for a successful therapy but they are counting the cost of failures. Nevertheless, even a billion is a lot of money and most companies are reluctant to invest in therapies that have are unlikely to generate billions of dollars of profits. The recent abandonment of GRPC1, the embryonic stem cell product by Geron, is a good example. The company had invested over $40 million into preclinical studies and was about to invest $20 million in phase 1 trial. Their board was looking at the future profit potential, their inability to find partners, and the likely future cost. They got cold feet and bailed.

One fo the reasons for the high costs of clinical trials for companies is because many of them don't know what they are doing and they are paying full cost for everything. Often, it is the first time that they are doing a trial for that particular condition. They spend a lot of time and money recruiting the centers, training them, and organizing them to do the trials. They proceed slowly and cautiously because they have little experience. They pay full hospital costs and have many consultants. They may encounter delays in recruiting patients. There may be glitches in delivery of product, quality control, and followup on the patients.

In contrast, a clinical trial network will already have solved many of the problems of training the doctors, recruiting the patients, doing the surgery, and collecting followup data. They will have figured out the best and most efficient ways of doing the work. They have experienced trial coordinators and efficient database management systems. NIH recognized this many years ago when they funded so-called National Cancer "Centers of Excellence" around the United States to assess chemotherapies and other therapies. By paying each center a relatively small grant of several hundred thousands dollars per year, they are maintaining a network of centers that have proven clinical trial experience, patient populations, doctors, and data collections systems. When a company has a chemotherapy they want to test, all they have to do is go to the network and offer them the data and treatment to test. If it looks good, the network can test the therapy in a year for substantially less cost that it would take for the company to set up a network, train everybody, work out all the glitches, and do the trials.

From 1985-1991, we did the first placebo-controlled double-blind randomized multicenter clinical trial of any therapy for spinal cord injury. NIH funded the study for about $6 million. Fourteen of the leading spinal cord injury centers participated and demonstrated the first therapy for human spinal cord injury. Today, this same trial would like cost $600 million. Most of it is due to the high costs of medical care, which then reflects in the high cost of clinical trials. I believe that it is possible to do clinical trials much more efficiently and for substantially less money if we use clinical trial networks to do so.

We must lower the cost of clinical trials for chronic spinal cord injury. First, it is important to understand that clinical trials can be done relatively inexpensively, if you know what you are doing and you set up clinical trial networks, which are the cheapest ways of testing therapies. Second, we have to convince companies and grant-funding agencies that it can be done for much less, so that they will start investing. We are in fact doing this in ChinaSCINet, using mostly volunteer doctors, donated therapies, and subsidized hospital care, to show that we can get a cell transplant (umbilical cord blood) and drug (lithium) combination through phase 1, 2, and 3 trials and regulatory approval for less that US$6 million. Third, we have to get the community to start investing in clinical trials, rather than waiting for some sugar daddy to come along. Bill Gates may one day fund spinal cord injury research but we should not be waiting until this happens.

The FDA and IRB (institutional review boards) will generally approve all sorts of clinical trials, even risky ones, if the right arguments and the trials are reasonably safe. FDA approval is seldom the barrier to clinical trials. Funding is usually the barrier. It is much harder to convince a company to spend hundreds of millions of dollars on a trial than to convince the FDA to approve a trial. Funding is the obstacle, not the FDA. If it only costs $6 million instead of $600 million to do clinical trials for spinal cord injury, a lot more companies will invest in trials, regardless of the FDA.

Wise.