FULL ACCESS TO CLINICAL TRIALS—S 3046
To: The Honorable Barbara Boxer
From: Peter Welch, father of Paul
Re: S 3046 The FULL ACCESS Bill
I am the father of a severely disabled adult son, Paul. My son has three related autoimmune diseases. He can no longer take care of himself. Once he was the editor of his college newspaper. Now his life consists of sitting in a chair in the living room, or in a wheel chair in a doctor office or in his bed packed with ice packs for the pain.
There are amazing new medications and therapies in the research and clinical trials pipeline. They offer the real possibility of immediate, life long remission of his condition. Paul would like to have access to these but has been repeatedly denied access to clinical trials. Paul has been excluded from four clinical trials because he was too sick. That's right, too sick. Not because the trials were full. Not because he did not have the condition that the investigational drug was in clinical trials for. No, he was denied four times solely because he was too sick.
Before autoimmune disease hit Paul, I had heard of clinical trials and naively thought that anyone who volunteered could get in. I believed as I had been told that the limiting factor for clinical trials was lack of volunteers. I now know that the true limiting factors are the Pharmaceutical manufacturer’s carefully constructed inclusionary and exclusionary criteria.
I have read that less than 3% of terminal cancer patients have access to clinical trial drugs. I know that my son has twice been denied participation in clinical trials due to very narrow inclusionary and exclusionary criteria. The purpose of these criteria seems to be to limit “volunteers” to the most likely candidates to have the kind of outcomes that will get the investigational drug approved. The criteria are not there for sick patients. They certainly are not written (by phama companies) to get the most important information regarding possible adverse effects of the investigational medication. These are not scientifically neutral criteria. They are biased and give less than fully valid results and worst they are patently unfair to the very sickest patients who need help the most.
I put the word, “volunteers,” in parentheses because the select few patients who can get into trials are not the sickest instead they are selected because they are poor and they are convenient. They are patients without access to health insurance and who cannot afford the thousands of dollars a month in prescription costs for approved FDA meds. They become the unwitting cannon fodder in the fraud that is clinical trials in America.
On two separate occasions in the summer of 2007, my son nearly suffocated to death due to the ‘freezing” up of his ribcage by his autoimmune diseases (psoriatic arthritis and ankylosing spondylitis). Foolishly I thought that since he had already failed at six different FDA approved meds and he was in very bad condition that he could easily get into a clinical trial or get access to a clinical trial drug under the compassionate use provisions of the FDA. I was wrong—very wrong.
Sick patients, especially very sick patients, are routinely denied access by pharmaceutical companies. The pharma companies do not want any patient using their investigational drug who might have any kind of less than optimal outcome.
Clinical trial patient selection is not random. It is not scientific. It is very selective and very biased. Everything possible is done to rig the outcome by careful selection of clinical trial patients so that an investigational medication has the best chance of approval. Any variable such as very sick patients is eliminated. Yet if these investigational drugs are approved, they are first used by the very sickest group of patients. The ones that the pharma companies do not want to allow into the clinical trials-- ones like my son Paul. Data on the very sick are absent from these sham clinical trials.
If pharma companies were forced to test all classes of sick patients, then they would have to open their trials earlier to people like my son and they would generate better data for prescribing physicians to use later on after FDA grants drug approval.
You may have heard of Compassionate Use for very sick patients as I have. But you should know that Compassionate Use FDA provisions, as currently written and enforced, are a cruel hoax. There is no compassion and there is no use.
Under current FDA regulations, the pharmaceutical company has the final say about whether a patient has access or not. There is NO outside appeal.
There is no reason for pharma companies to ever say yes. All the incentives are in direction of NO. If a pharma company allows use for individual, very sick patients, the results must be reported to FDA. These ‘very sick patient’ results will be considered when the FDA is deciding to allow approval to market. Any adverse outcomes that are encountered in use by the ‘very sickest patients’ will likely delay FDA approval.
Under current rules, if I were a drug company CEO, I would deny every request just as they do. I do not blame them. I just want the system changed. Full Access to Clinical Trials, S3046 is a good start.
Consider the case of Jacob Gunvalson of Minnesota. He is dying of a fatal form of muscular dystrophy that will kill him before age twenty. He is now 15. His mother, Cheri, helped to get federal funding for the development of the clinical trial medication PTC 124 that might save her son’s life. She was invited to stay overnight in the lead researchers home with her son. She was told her son qualified for trials. Yet when he became sicker the company denied his request to get the medication. She went to court and LOST. Her son will now die. This is the only medication that has a chance to save his life and he has NO RIGHT TO THE USE OF THIS POSSIBLY LIFE SAVING MEDICATION!
When my son was near death in the summer of 2007, after failing at the number one medication (Enbrel) for his condition (psoriatic arthritis and ankylosing spondylitis), I thought it would be easy to get access to a novel medication (Ustekinumab, CNTO1275, Stellara) that in the spring of 2007 had had “stunning” results in phase II and III human clinical trials in patients with his condition. I was so wrong.
Yet I did everything right. I contacted Frank Burroughs of Abigail Alliance, who in less than 24 hours, got me in contact with the Senior Director of Clinical Immunology at Centocor. We needed the director’s approval for Paul to get the medication. That approval never came. Even after numerous contacts.
It is now January 2009, nearly two years later. Paul still has no access to the most revolutionary new medication for psoriasis of this decade. He must have three to four hour infusions of a much older, very dangerous MOUSE PROTEIN medication every six weeks instead of the revolutionary FULLY HUMAN Ustekinumab, CNTO 1275, Stellara which is a single shot every three months.
That’s right a fully human revolutionary medication that is a single ten second shot has been denied my son by the FDA and its bureaucracy for almost two years in favor of an OLD TECHNOLOGY, mouse protein, dangerous four hour set of infusions. The infusion is so dangerous that my son must have an hour of pre-treatment with IV steroids and antihistamines before he is infused with the MOUSE PROTEIN medication (Remicade, infliximab). Even with the mandatory pre-treatment most patients develop anti-mouse antibodies within one year. My son has now had 15 months of treatment. Each new infusion is another spin of the revolver cylinder in a high stakes game of medical Russian Roulette.
Worse yet the FDA bureaucrats have for little or no reason again and again delayed the approval of ustekinumab. In June of last summer an independent panel of scientist and patient advocates UNANIMOUSLY recommended to the FDA for approval. But our friendly FDA bureaucrats decided inexplicably not to approve it last summer. They delayed their decision until December 2008, last month. We waited and hoped and prayed that Paul would not die from a reaction to his current MOUSE BASED medication during that wait. December came. We were ready for the Ustekinumab and the FDA DELAYED IT AGAIN!
Naively I thought back in the Spring of 2007, almost two years ago, when the “stunning” results of the clinical trials were announced that the FDA would speed up the medication to approval. Stupidly I thought that a brand new medication with different targets than any other one on the market which BEAT the NUMBER ONE MEDICATION in HEAD TO HEAD competition would be RUSHED to APPROVAL on a FAST TRACK. But no, the bureaucrats at the FDA just DELAY, DELAY, and DELAY.
Please help my son, others like him, and even G-d forbid maybe you or a loved one to have life saving access to revolutionary new medications and therapies. Please support as a co-signer of US Senate Bill 3046.
Father of Paul Welch