rant on/ Who's life is it anyway? People claim there is such a thing as free will. Not where the FDA decides for you and your will is subservient to the bureaucrats, authorities and politicians. Our bodies are not our own. Our minds are incompetent to decide for ourselves what we want to do about something which affects nobody but us.
The individual is just a servant of the state and those in power. The individual's life is expendable. They may not try to save themselves. It is illegal to be alive. It is criminal to supply a person who wants the product, the means to stay alive.
Life, Liberty and the Pursuit of Happiness, just like "Land of the Free and Home of the Brave" in the days of segregation, makes a catchy epithet, but is ignored in reality.
With luck, some members of Congress are right now suffering prostate cancer and will be feeling the pain right there in their bones.
siliconinvestor.com
< Dear Member of Congress:
In 2002, the FDA made a very big deal about how it approved an anti-cancer chemotherapy drug in record-breaking time -- something like 47 days. While this drug may well help patients, it was not exactly a miracle cure and it had the terrible side effects associated with most chemotherapy drugs.
In 2002, the FDA turned away applications for many other oncology (anti-cancer) drugs. Some have been outright denied, some were refused because the filing was incomplete, others were sent back for more research. A disturbing number of these drugs, even though they were usually tested only on the sickest cancer patients, showed probable efficacy in at least a subset of patients AND had few, if any, serious side effects.
Problems with Thalidomide galvanized lawmakers to action in the 1960's. Prior to this time, drugs only needed to show they were safe. After the Thalidomide tragedy, lawmakers decided pharmaceuticals needed to be proven safe AND effective. I can't, and won't, argue with this decision even though the problem with Thalidomide had nothing to do with efficacy -- only safety. Given the cost of drugs today, I am comfortable with the logic behind preventing the sale of safe snake to our nation's populace.
Somewhere along the line, however, the process got out of hand
Did you know that in August the medical world learned of a drug that can help about 75% of hormone refractory prostate cancer (HRPC) sufferers fight their disease? The drug can do this with no appreciable side effects. This is excellent news for HRPC patients because there is currently no treatment for their condition. Oncologists/urologists often use radiation and/or chemotherapy, but this barely delays the spread of HRPC. Patients endure the side effects of these treatments largely in order to lessen the pain caused by the spreading cancer splitting their bones from the inside out.
Before I continue, I need to digress on two points: The first is to provide some background on HRPC. There are over a million men in the US alone who have prostate cancer, with over 200,000 new cases added each year. All men will eventually become immune to initial hormone treatments, whereupon their disease becomes known as "hormone refractory". About 100,000 patients become hormone refractory each year.
The second is to tell you the drug I am talking about is called Provenge. It is the product of a small biotech firm based in Seattle, WA called Dendreon. My company, a publisher of an investment newsletter, covers Dendreon and some of my staff members -- myself included -- own shares. I am using Provenge as an example not because of this professional connection but because it is one of the more blatant examples of a disturbing trend.
In one of its double-blinded, placebo-controlled, Phase III clinical trials, Provenge was given to a little over 70 of 130 patients with HRPC. The remainder received placebo. In an analysis of the trial, the drug helped those patients whose disease severity was less than eight on a scale of two to ten (called a 'Gleason score'). According to our best information, HRPC patients with a Gleason score less than eight comprise about 75% of all HRPC patients in the US.
The FDA won't even consider approving this drug, however. Why? It's not because of the side effects, which were slight fevers and chills that disappear quickly. There were no serious safety issues. There was no hair loss, vomiting, susceptibility to opportunistic infections, or any of the other terrible side effects associated with traditional radiation or chemotherapy treatments.
You see, when Dendreon analyzed the entire population in the study -- i.e. patients with any and all Gleason scores -- they could not tell the FDA with better than 95% confidence the treatment effects shown by Provenge were due to the drug and not random chance. All they could manage was 94% confidence.
They did manage better than 95% confidence in the subgroup of patients with Gleason scores below eight. However, since this subgroup was not predefined and controlled for in the statisticians' plans submitted to the FDA before the trial started, it doesn't count. That's right, it doesn't count.
Despite the fact the drug clearly could help tens of thousands of HRPC patients, the FDA won't approve it because a "retrospective analysis" of effectiveness like Provenge demonstrated in the Gleason less than eight subgroup doesn't count.
In news release on December 11th, we learned Provenge is also able to delay onset of bone pain in HRPC patients with a Gleason score less than eight. When HRPC becomes metastatic, it often first travels to the bone. As the tumors grow, they split the bone from the inside. This causes great pain, as you can probably imagine.
Despite the fact Provenge has proven itself safe and effective by any reasonable measure, because the Gleason sub-group was not prospectively defined in the statistical plan given to the FDA before the start of the trial, it won't be approved without additional trials. HRPC sufferers will be denied access to Provenge until Dendreon can complete the new trial.
In the required trial, Dendreon will need to enroll another 250-300 patients. That will be completed about 15 months from now. It will then take Dendreon about six months to compile the tens of thousands of pages of documents comprising a modern drug application. Even assuming the FDA grants Fast Track status and approves Provenge at their first opportunity, it will be 27 months before Provenge is available to HRPC patients.
Remember, HRPC patients have no treatments now except to lessen symptoms connected with the cancer splitting their bones from the inside. Remember also that Provenge has no significant side effects or safety issues.
Most of all, remember the existing clinical trial proved Provenge works
In the next 27 months, nearly 200,000 men will die of prostate cancer. Nearly all will have a painful time of it, even though most will choose the terrible, debilitating side effects of chemotherapy and/or radiation in an attempt to make their lives a little better.
Dear reader, if you are a member of Congress or a Congressional staffer, you'll likely show this to someone in the FDA for comment. You'll be returned a statistics-based briefing paper of why I'm full of beans. I'm guessing the brief will tell you why retrospective analyses are very dangerous, why my "94% precision" comment isn't technically correct, and a whole host of other reasons.
This is the problem. For too long, statistics have trumped common sense and people are dying painfully because of it. Provenge is just one example. Ovarian cancer patients are in a similar situation with a different drug.
I implore you to listen to common sense. The subgroup analysis done on the Provenge trial was reasonable. The drug is safe and has few side effects. The HRPC patient population has no reasonable alternatives. Step back from the statistician's mumbo jumbo and ask yourself if the FDA's decision to require a 27-month delay in the availability of Provenge is reasonable.
Perhaps the experience will make them more amenable to compromise towards common sense...
The FDA won't reform itself because it firmly believes it is acting according to the directions given to it by Congress. Therefore, only you can fix this problem. Launch hearings that invite physicians, patients, survivors, and families of those who didn't make it. Leave the statisticians at their computers for the first few days of the hearings. Better yet, make them sit through the tales from physicians and patients. Perhaps the experience will make them more amenable to compromise towards common sense and away from scientific absolutism.
You don't have to change the rules very much. Just allow -- nay, REQUIRE -- the FDA to implement common-sense conditional approval mechanisms allowing safe, apparently effective drugs like Provenge to be made available to patients with no good alternatives while confirmatory trials are underway.
Our graying nation will thank you for it.
To get The Internet Financial Connection Newsletter e-mailed to you for FREE, send a blank e-mail to ifc-subscribe@topica.com. In 1998, 225 different specific stock ideas appeared in the IFC. As of July 5, 1999, the return of the group of stocks that appeared in the IFC in 1998 rose an astounding 51%. What is even more amazing is that in 1999, 185 stocks appeared in this column and as of July 3, of 2000, that group of stocks was up 56%. In 2000, 125 stock ideas appeared in this column. As of July 2, 2001, that group of stocks was up 1.94% versus a 47% loss for the NASDAQ Composite during the same period of time.>
There's snake oil sold by the truckload in the USA now. Pharmacy shelves are full of bee pollen, St John's Wort, ginseng and any number of chemicals purporting to help one along pharmaceutically but for which there is tenuous, if any, evidence.
Supermarkets are full of dietary mayhem - aisles of sugar and fat with various flavourings, purporting to be 'food'.
"Protecting the public" against snake oil medical charlatans is worthy, but can be done in the context of similar protections as for any other product. Products must be fit for purpose, fraud is illegal, damages can be claimed. Proof in court is required if out of court settlement can't be reached.
Leave people to decide for themselves. If they want to wait for the FDA approval stamp, they can demand that. The current method is the Kremlin style central planning style which has been discredited as too slow, bureaucratic and unresponsive to individual needs.
People who suffer rare diseases have zero prospect of any help because the cost of a FDA case exceeds the economic benefit to the few who suffer. Profits would not be possible. Individuals don't matter. Free will doesn't exist.
/rant off
Mqurice |
