Thursday, August 27, 2009

Panel: Naomi Kleitman, Barry McKay, Peter Gorman, and Tony Caggiano

Tony is speaking about his role as manager of the clinical trial process at Acorda.

Naomi asks if people were in clinical trials, and several explain what they were involved in.

One did the fampridine trial and is eagerly waiting for the release of the drug next fall. One used a device that was not yet approved, but the company that was manufacturing it went under. She now gets it from England at a very high cost. The panel says that it's a big issue; companies go under and there you are. A couple of people went to Gainesville for the suspended gait training, and both got benefit.

Naomi wants to know if any minds were changed today about the lack of clinical trials ~ nobody answers her question, but someone asks another one about the 4sci trials. It seems to be about repeating animal tests that seemed promising. Theyve now repeated about a dozen of these trials and so far failed to replicate what was published.

(This sucks, of course, but here's how finely tuned the spinal cord is . . .when they tried to replicate studies from Europe, one issue was that the strain of the animals being tested was a little different in the USA. Small physiological change => different outcome.)

Man in the front row is asking about off-label usage of approved drugs. They tell him that doctors are free to prescribe drugs . . . but does the NIH care about this? Naomi says that there are 26 funding centers at the NIH. The entry criteria for getting money from NIH is about a million $ a year.

Ida Cahill says that she gets phone calls from people who are thinking of going outside the USA. She knows of 2 women who went to Portugal, and 2 more who went to India. They spent $70k each plus travel to go get who knows what done to them.

Some talk about this, and how to know if a trial is legit. Basically, it's just a very risky undertaking to leave the country, especially if the therapy you're getting involves cells. Naomi gets up to talk about what red flags look like, refers people again to the iCord website where the full question set is available.

Man asks about the existence of a national database . . . if you get cancer your name automatically gets put into a giant list that keeps track of everyone; the answer is that hiipa regulations prevent this from being done -- that can't be right, can it? Then why do cancer patients do it?

A question about emergencies -- how the heck to doctors and scientists go about inviting people with new injuries (who are devastated physically and personally) to give their consent to a trial. Naomi is describing something called community consent that made it possible to include minorities, who as it turned out, were being underserved.

A question about whether or not the trials that involve cells are going to have controls. The answer is no, at least not in Phase I. What will happen in Phase II?
In some studies (ProChord) they've hidden the information from the evaluators. It's always a tension between the need to do the science right and the need to treat patients ethically.

Woman says that the VA hospitals are looking for study subjects.

Man says that the Portugal trial has been through the EU version of the FDA, and that an IRB does exist there, and at least one paper has been published about the results. He's a rehab doctor at Wayne state university. Panel member replies that the paper had flawed data, as would be clear to anybody with a statistical background. He says they also have claimed no adverse effects, but he personally knows of 6 very adverse effects. He's worked with people who got the Lisbon treatment, came back for intense PT and compared them with people who just got the PT . . . no difference. NADA.

Another panelist says that, you know, here we are with 2 qualified people looking at the same data and coming to a different conclusion. (That is what I hate.)

Woman whose daughter has been injured for 18 years says that she's seen rats walking, for heaven's sake. How on earth do you all come together? Panelist says that his favorite annual meeting is the ASIA meeting. They do learn from one another there. Also the journal Spinal Cord Injury. Also the peer review process. Also the NIH funding process itself, which is a marker to show that this work is new, promising, and original.

Woman asks the assessment measures guy what's being done with his work . . . he says that it will let small changes be documented and approaches be validated.

She says, could this assessment tool be used to convince insurance companies to pay for treatments like rehab?

He says he can't imagine that it wouldn't . . . and people should understand that to bring a group of combined treatments to the FDA -- oh, wait. Naomi says all they need is to show that their is safety in each of the individual components.

Oy, long day, people. Marilyn says it's time for a little socializing. Yay! Plus we get a buffet -- a Mexican buffet! Woo hoo, seriously.

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