Tuesday, June 12, 2007

Response to NYTimes writer Gardiner Harris

Below is my letter to a writer,Gardiner Harris at the New York Times regarding his article about the FDA being to easy on allowing new medications to be approved. I disagreed.
Here is a link to his "news analysis" article:

Here is my response:
The problem at the FDA is the terrible length of time it takes for life saving drugs to get to patients. There has been a revolution in the development of medications for terrible conditions. Cancer and autoimmune disease are routinely cured in lab animals, but people cannot get these treatments.

Four years ago my youngest son, Paul, was a healthy college senior—dating, dancing, studying hard. He was a nearly straight A student at University of California, San Diego on track to graduate Cum Laude. He developed a “sprained knee” which did not get better with rest. It turned out to be the first symptom of Psoriatic Arthritis.

Today he can not get his own food or water. He cannot walk unaided. He cannot use a computer mouse. (He was once editor of his college newspaper). He cannot even talk as the arthritis has affected the joint that holds his vocal cords. Once he had a beautiful singing voice. Today his mother and I think it is a good day if he can croak/whisper ten words over the course of the entire day—mostly he kind of painfully and clumsily points to tell us of his needs. Once he was a superb athlete who ran everywhere and never took the campus shuttles. They were too slow for him.

Any of the following: HuMax CD-20, Centacor’s CNTO 1275, or Rigel’s 788 could cure him. All three work on different targets than the ineffective Enbrel he is taking now (cost $3000 a month). Any one of them could be a cure. But he will have to wait years to find out.

It will be at least another five to ten years for FDA approval of these for his particular form of autoimmune disease. He has an “orphan” autoimmune disease, not one of the Big Four—RA, Lupus, MS, or juvenile diabetes. The “orphan” autoimmune conditions get FDA approval of new medications only may years after the Big Four receive approval.

Even though every drug approved for PsA was first approved for RA, new RA drugs cannot get approved for PsA without another billion dollar clinical trial just with PsA patients. Few drug companies find it worth the cost for so few additional patients. There is no off label use of drugs either as the insurance companies will not pay for off label use and these drugs cost thousands to tens of thousands of dollars a MONTH! We might second mortgage our house and pay for a year or two but then we would be bankrupt.

Those three above are but three out of some forty plus cures I have found in the research literature on line. Paul cannot use any one of them, thanks to the FDA’s horrible rules governing experimental drugs. (Those cures are written about in previous posts on this Blog)

Researchers are terrified that if they allowed access to someone like my son and were found out, they would never get a grant again. It would be career suicide. So no mattr how promising the research or how much I beg and plead, those research cures for mice model of autoimmune disease will not get to my son.

HuMax CD-20 and CNTO 1275 are both in phase III testing. R788 is in phase II trials. Paul cannot qualify for any of those trials due to exclusion criteria. He has drug and food sensitivities. Yet the disease is destroying his joints and tendons and leaves him in terrible pain. When they are approved in five to ten years if he is still alive he will have many more crippled joints and still have food and drug sensitivities, so why can’t he try them now. R788 works on a pathway involved in drug and food sensitivities and might actually cure him of both PsA and the sensitivities. But Paul will not be able to get it.

Compassionate Use protocols are a joke. Virtually no one qualifies. If somehow you do qualify, drug companies do not want to give medications to Compassionate Use patients because if anything goes wrong with the treatment of those very sick patients, the FDA counts it against the Investigational New Drug during the FDA’s approval process. Smart companies refuse Compassionate Use Exemptions.

I have watched my son’s productive life destroyed. His disease will also end this painful crippled life he now has in the next few years because the same inflammatory process that has crippled him is destroying his arteries and putting him at great risk for a heart attack. He will die in pain, a prisoner of his own body, while we wait for the existing miracle drugs to be FDA approved.

See the Abigail Alliance website at: <http://abigail-alliance.org/> Read a few of the horror stories of dying patients desperately trying to access medication. Join with us and advocate for changes in the FDA policy.

It is not that the FDA has been snookered by Big Pharma into approving medications that should not be. It is the FDA refusing to understand the difference in patient need between a new allergy medication and a revolutionary new cure for autoimmune and cancer. A “me too” allergy antihistamine can be approved leisurely, but drugs that are novel for dire conditions are different. They should be allowed to be used by the sickest and most desperate as soon as they clear animal testing. Do you realize tens of thousands of people die needlessly each year in the United States while being denied the use of these new miracle drugs?

If Paul did qualify for a trial, he would only have a fifty/fifty chance of getting the medication. Paul would have to quit his existing meds. He would have a 50% chance of getting a nothing placebo but not be able to take any other medication. On a placebo alone he would get worse. That is great news for the study, not so good for my son. He would have more joints lost and more pain.

We know the outcome of cancers and autoimmune disease. We do not have to do trials with half the dying patients denied any treatment while we ghoulishly record when they die. It is a bit Dr. Mengele, wouldn’t you agree?

My son’s great aunt died a horrible, long, struggling to breathe, death from Multiple Myeloma two years ago. There were cancer drugs which might have cured her in clinical trials, but she could not get them. Today they are available but “darn, she just got MM too soon to have access” according to the FDA. Her last twenty four hours she could not sleep because she could not get enough air to fall asleep even with supplemental oxygen. She died finally of exhaustion when she could no longer get her breathing muscles to respond. A slow agonizing death is what the caviler attitude of the FDA condemned Aunt Lyt to.

Please consider patients like my son and my wife’s aunt in the next article you write.
Peter Welch

No comments: