Wednesday, January 28, 2009

Major Medical Research Problems in the United States

Problems with Medical Research the FDA and current US clinical trials (DRAFT)

Good drugs take too long to get to really sick. Fewer drugs are being approved each year by the FDA. The drugs approved are taking longer and longer to go through the Byzantine clinical trials procedures wastefully required by FDA. The FDA bureaucrats only concern seems to be keeping their job protected. The safe answer is always NO! Let’s delay a little longer. No one ever got fired or sanctioned because patients died while waiting for a new therapy. Saying no means job security. No concern is shown for Americans who are sick, suffering and dying while we wait for the bureaucrats to make sure their little careers are perfectly safe. Allow these revolutionary new therapies to get to the sick and dying now!

Clinical trials are biased and give skewed results. Carefully screened selections do not give good results. They give skewed results. Screening patients to obtain those who will give you the desired outcomes is NOT SCIENCE. It is a rigged game. The whole basis of a scientific experiment is to have data inputs that are unbiased and random. Otherwise it is not science. No one can have confidence in the results.

There is no compassionate use. The concept is a hoax. Big Pharma companies have no compassion and always deny use. Under current law when Big Pharma inevitably says no to access there is no appeal and no exceptions. Not even court action can help. See the following Wall Street Journal article about a boy sentenced to death by a drug company. The courts refused to help.

All patients who wish access should be given access. That is a matter of basic fairness and decency. Good old fashioned American values. We have a right to life and right to access medications that will preserve our lives.

Placebos are mass murder.

Clinical trials cost too much. Now approximately 1.2 billion a piece. $26,000 per patient just for the phase III trials and thousands of patients are required by antiquated FDA regs

Clinical trials are needlessly long. It is now taking DECADES from DISCOVERY TO the PRESCRIPTION PAD! It should take months maybe weeks. Ustekinumab, a revolutionary new medication for psoriasis, that had “stunning results” announced after phase II trials were completed in February 2007, still has not been approved by the FDA rhymes with delay. It was UNANIMOUSLY recommended for approval by an independent panel of scientists and patient advocates in June 2008. Clinical trials head to head with Enbrel the current number one best seller for psoriasis showed it gave BETTER RESULTS. Gene studies of patients with psoriasis released in January 2009 confirmed that one of the major gene defects causing psoriasis causes an increase in the pro-inflammatory IL-23 cytokine. Guess what Ustekinumab blocks? Still the F’ing DelAy still has not allowed Ustekinumab to get to patients who need it. Approval is still blocked. Canada has approved it but not the FDA in US. We will have to go to Canada to get it? Our family lives in San Diego County near Mexico. We are thousands of miles away.

Wrong criteria measured. Instead of biometric measurements of cytokine levels and autoimmune marker molecules, our backward friends use such outmoded measuring instruments as PASI and the ACR measurement. They literally measure how big the psoriatic lesion is or how swollen the joint is. That kind of measurement is so 16th century. We live in the 21st century. Let’s use 21st century measurements. They are far more accurate.

There is a better method. It is cheaper, faster and more fair. The method is to throw it all at the wall at once see who is helped. It is called trial and error. It is a powerful method of discovery—perhaps most powerful ever discovered by humans. It is the method that traditional Oriental medicine used to discover artemisinin and (anti rejection drug based on Asian fungus I forgot name) Wait, I remember, Tacrolimus! That’s it. Both of these and MANY others have been discovered by Trial and Error and Serendipity.

Our current clinical trial method is resulting in less accurate results in more expensive trials. We are getting fewer new drugs approved ever year. We have more dead and suffering Americans for ever longer periods of times.

This is a historic tragedy because for first time in history of man, we have the tools and knowledge to rapidly to end many chronic diseases that had no chance of cures in the past. These diseases include all forms of cancer and all forms of autoimmune (RA, PsA, SLE, MS, diabetes I, etc.) All hematological disease as well like hemophilia, beta thalassemia, sickle cell anemia could fall in the next TWO YEARS. There is already a company Osiris with a stem cell product Prochymal that offers hope for cures for half of all autoimmune patients, infections, and cancers. Prochymal might not cure all of those. But the concept it is based on definitely can.

Lift all bans on testing patients and test as many as will volunteer with as many possible medications and COMBINATIONS of medication ASAP.

We are running out of time when it comes to our country’s wealth. We will not be able to afford this research for much longer. Small Pharma development companies are going out of business by the score. With their demise go the cures that they were working on.

The antibiotic super bugs are poised to kill millions of Americans. Already we are amputating limbs of injured soldiers who have compound fractures because of unstoppable infections—Iraqui-bacter and MRSA among others. We are back to CIVIL WAR medicine. In those times Civil War times not only were soldiers affected but 1/3 to ½ of all children died of infection before having children of their own. Is that what we want again?

We must have the US government DIRECT the research by setting goals and rewards by eliminating all roadblocks and forcing companies to share intellectual resources and when discoveries are made financial rewards.

Directing research can be done. Today we have the tools for directed medical research to cure virtually all chronic disease, not to mention stopping the emergency of the outbreak of the killer super bugs that today kills more people in US than AIDS.

Are the Placebo Arms of Clinical Trials a form of Mass Murder?

Another problem with clinical trials is the placebo group.

The terminal cancer patients assigned to the placebo group are being given death sentences so some clinical trial clinician can have pretty statistics. This ranks close to as evil as a Dr. Mengele experiment.

The clinician wants to be able to say, " Look in the investigational drug branch of the trial 80% of the patients were alive after six months but in the placebo group only 20% were alive in six months.

Think what this means. It means that 60% of the patients in the placebo group were sentenced to death just for pretty numbers.

These patients were terminal. We know approximately how long they will live and how they will die. We have decades of experience and data about how and when they die, yet we must watch another group of several hundred die because it makes our numbers nicer. How monstrous!

In Europe the whole idea of having placebo groups at all is being debated because it is an evil cruel thing to do to terminal patients and their families. We need that debate here, too.

Clinical Trials Results are Rigged

Paul has been denied access to a clinical trial for the FOURTH TIME. Same reason as before, he is too sick. That is correct the really sick are systematically excluded from clinical trials in the United States. Can you say RIGGED RESULTS?

Our clinical trial apologists here in the US like to pretend that allowing more access for American citizens would somehow invalidate the results of our so called "scientific" clinical trials. They tell us that the reasons that patients are denied is so that the results obtained by the trials are more scientific, more accurate, more meaningful, etc. These "fake" scientists, who excluded my son and so many other Americans from clinical trials, put on an air of mysterious scientific superiority. But they are being scientific and they are NOT TELLING THE TRUTH!


That is correct 97% of terminal cancer patients are given NO CHANCE to live by these monsters. Yet they continue to pretend that the only thing holding up clinical trials is a lack of volunteers. What they mean is that they need access to huge numbers of volunteers so that they can carefully select the 3% who will likely give them the results they are looking for.

When patients with the worst symptoms are excluded, then the clinical trial folks do not obtain accurate results for the whole spectrum of patients that will be using these drugs. If the companies running the trials can carefully select only patients with the condition that are the most likely to get the results that the company wants, then that is NOT AN UNBIASED SCIENTIFIC TRIAL! It is like a rigged middle school science fair exhibit where the probable outcomes are known in advance and the experiment/trial is set up to be pushed in a BIASED DIRECTION.

If a student in middle school comes up with a hypothesis to test that says, "all canned beverages will make a tarnished penny shinny." He must test pennies against ALL kinds of canned drinks. If the student uses only carbonated drinks, then the sstudent will "prove" the hypothesis. Yet it is not really true. The experiment is wrong because it was skewed in one direction. Carbonated drinks are a special kind of canned drink because they contain CO2 (the carbonation) which when dissolved in water makes an acid. All pennies left to sit in carbonated drinks will get shinier, it' true. But they will get shinnier in any mild acid.; however, not all bottled drinks are carbonated. So do all canned beverages put a shine on a dull penny? No, of course not! The student has rigged the test by limiting the kinds of beverages to carbonated ones. The student's shiny pennies do not prove the hypothesis.

The same false results are being obtained with these so called "scientific" clinical trials done here in the US. The inputs are carefully selected so that the correct out put or outcome is assured. Limiting patients to only a very few select patients is not an unbiased, fair clinical trial. The results obtained are not good results. The results are biased and should not be trusted.

MORE ACCESS TO CLINICAL TRIALS IS NEEDED NOW!!! Not just for the good of the very sick like my son but mostly to make the trials scientific, accurate and so the trials give good trustworthy results.

Wednesday, January 21, 2009

Letter to Honorable Barbara Boxer Re: Access 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.

Thank you,
Peter Welch
Father of Paul Welch

Tuesday, January 20, 2009

Inspector General of HHS confirms clinical trial bias problems

My son, Paul, has four times been denied access to clinical trials not because the clinical trial is full, and not because he does not have the condition that the clinical trial investigational drug is being tested on. No, he has been denied over and over because he is too sick with the condition that the investigational drug is supposed to help. He was most recently excluded just this week.

You might think that he is being excluded for good scientific reasons. But you would be wrong. The sickest patients should be the first ones signed up in the clinical trials. Once medications are FDA approved for sale to the public, the sickest patients are first in line to try the new meds as nothing else has worked for them. The doctors of these very sick patients will prescribe these meds as soon as they are allowed. However, FDA and more importantly the prescribing doctor will have NO INFORMATION about the effects of the new medication on these very sick patients because these patients were systematically excluded from the clinical trials.

Why are the clinical trial investigators so compelled to rid the trials of the very ill? Could it be money? Some people think so. Here is what one scientist said in the article below, "...bias, whether intended or not, can slip into the process when money is involved."

Here are the views of another prominent American physician and researcher quoted in the article below: "If a study is funded by a drug company to investigate its product and if the investigator also has relationships with that company, you can be darn sure that if the study makes it to publication, it's going to favor that product."

Clinical Trials in the United States have inclusionary and exclusionary criteria to carefully select only very particular patients with the intent of getting the FDA approval. No other purpose comes close--not scientific accuracy--not getting answers to the tough questions--not finding out new information, not for helping the suffering volunteers, and never for any kind of compassionate use. I have read that less than 3% of DYING cancer patients are given access to clinical trials by Big Pharma.

Why do three prominent American physicians and researchers (see below) declare that "lax or absent (FDA) regulatory oversight implies a lack of concern and a tacit acknowledgment of a lack of (FDA) enforcement of existing rules" ?

We are often told that Big Pharma really needs volunteers for clinical trials. If more people would just volunteer,clinical trials could move faster and drugs would be approved sooner. The impression given by Big Pharma is that it is really difficult to get people to volunteer. The opposite is true. There are huge number of volunteers but most are rejected. Big Pharma encourages volunteers because it needs huge numbers so it can carefully cherry pick just the right ones so the "right" results can be obtained. This process of course is the opposite of good science. It is in fact a rigged game.

It is not just me criticizing the FDA, here is another quote from the article below :"...nobody cares and clearly that is the message the FDA is sending out"

There is no question that clinical trials in the US volunteers are picked to get fixed results. It is one thing for me to notice but now I am not the only one saying it. Now the BUSH appointed secretary General of Health and Human Services confirm that the game is rigged. This fact soon becomes apparent to every patient who tries to get into a clinical trial or harbors the false hope that ther eis the possibility of qualifying for compassionate use of an investigational drug. Here is the URL of the article:

and here is the article:

FDA Not Effectively Monitoring Investigator Conflicts of Interest, HHS Watchdog Says
Neil Osterweil
Medscape Medical News 2009. © 2009 Medscape

January 16, 2009 — More than one third of new drug marketing applications approved by the US Food and Drug Administration (FDA) were missing information about potential conflicts of interest for clinical trial investigators, which could allow bias to creep into the approvals process, a government report released this week has found. The FDA has said that it agrees with most of the report's findings.

Federal regulations require clinical trial sponsors to collect financial information from investigators at the outset of trials, and to report on their efforts to minimize the possibility that financial self-interest could color trial results, notes the report from the office of Daniel R. Levinson, Inspector General of the Department of Health and Human Services (HHS).
Trial sponsors — usually the company developing or licensing the drug or device in question — only have to disclose this information when they apply for marketing approval, and even then there's a loophole: they can claim that they "acted with due diligence" to collect financial information from each investigator to satisfy regulations.

Report Found Few Financial Disclosures

Yet 42% of new drug marketing applications filed in fiscal year 2007 were short on investigator financial disclosures, and in fully one fifth of cases where such information was disclosed, FDA reviewers took no action, the watchdogs reported.

"In FY 2007, only 1% of clinical investigators disclosed a financial interest," the report's authors write. "By way of comparison, the Journal of the American Medical Association reported that between 23% and 28% of academic researchers had financial interests in medical companies. Further, we found a number of limitations in FDA's oversight, leaving FDA unable to determine whether sponsors submit financial information for all clinical investigators."

In addition, the HHS inspector general's staff found that FDA can't verify whether relevant information has been collected on all investigators because of incomplete data and the lack of on-site verification procedures.

Collection and review of such information should be routine, as should appropriate action when required, and all of it should occur before a trial ever gets off the ground, contends a former FDA reviewer interviewed by Medscape Medical News.

"You want to intervene at a time when there's an ability to do something about it," said John H. Powers III, MD, now an assistant clinical professor of medicine at the George Washington University (GWU) and University of Maryland Schools of Medicine.

"To have a situation where the sponsor has already done the trial, submits the information, and then the FDA says 'you have conflicts of interest with your investigators,' puts the sponsor in a bad spot, the investigator in a bad spot, and it puts the FDA in a bad spot. It seems to me that if you're going to regulate here that you should do it a proactive manner," he said.

"Management Failure"

Federal regulations concerning drug marketing approvals require that "adequate measures are taken to minimize bias on the parts of the subject, observers, and analysts of the data," Dr. Powers noted, yet one of the most common sources of potential bias — investigator relationships with industry — are overlooked or ignored.

"This is a management failure," said David B. Ross, MD, PhD, a former FDA reviewer who is now a clinical assistant professor at GWU. "The managers who have set this policy have done nothing to enforce it and there are no consequences, and the reason is that nobody thinks it matters; they're kind of doing this for show."

When Dr. Ross was at the FDA, he and other reviewers warned agency management that a study used to support approval of the antibiotic telithromycin (Ketek, sanofi aventis) was marred by per-patient payments to investigators of up to $400, and, in at least one case, by fraud on the part of the investigator who enrolled the most patients (she was later sentenced to 57 months in federal prison), and by "serious violations in trial conduct" in 9 other trial sites, leading to 4 referrals for criminal investigation.

Despite the warning, and despite ongoing concerns about reported hepatotoxicity from the drug, FDA management failed to take substantive action in response to reviewer concerns, a failure that Dr. Ross said is emblematic of the agency's apparent indifference to the possible influence of investigator financial relationships with industry.

"...CDER [the Center for Drug Evaluation and Research] is not doing that good of a job, and with what they do track, what are they doing with it? It's like having laws against speeding and not writing any tickets," Dr. Ross said.

Relationships Can Have Profound Effect

Eric G. Campbell, PhD, an associate professor at the Institute for Health Policy and the Department of Medicine at Massachusetts General Hospital and Harvard Medical School in Boston, said that financial considerations — whether honoraria, consulting fees, stock options, or direct payments — can have a profound effect on which trials get published or promoted.
"The important thing to remember is that relationships with industry — they may not always be conflicts — are ubiquitous in all aspects of medicine, medical research, and medical regulatory affairs," Dr. Campbell said. "If a study is funded by a drug company to investigate its product and if the investigator also has relationships with that company, you can be darn sure that if the study makes it to publication, it's going to favor that product," he said.

Like Dr. Ross and Dr. Powers, Dr. Campbell pointed out that lax or absent regulatory oversight implies a lack of concern and a tacit acknowledgment of a lack of enforcement of existing rules.

"The bottom line is that if you don't report stuff and nobody pays attention to whether it's reported or not, you're just signaling that it's not important and that nobody cares, and clearly that's the message that FDA is sending out," he told Medscape Medical News.

Key Findings

The report, prepared by staff from the Chicago regional Office of Evaluations and Inspections in the HHS Inspector General's Office, was based on reviews of financial forms, attachments, and accompanying FDA review notes for all 118 marketing applications approved by the agency in fiscal year 2007. The watchdogs also reviewed FDA regulations and guidance, conducted structured interviews with FDA officials, and surveyed FDA reviewers.

The key findings were:

1% of clinical investigators (206 of 29,691) disclosed a financial interest.

FDA does not have a complete list of clinical investigators and does not use on-site inspections to confirm that submitted financial information is complete, meaning that reviewers cannot determine whether sponsors have submitted financial information for all clinical investigators

42% of FDA-approved marketing applications were missing financial information,

23% of approved marketing applications were missing a certification or disclosure form or required attachments, and

in 28% of applications, sponsors used the due-diligence exemption to indicate that they were unable to provide complete financial information.

FDA did not document a review of any financial information for 31% of marketing applications.

Reviewers who followed a review template were more likely to include financial information than those who did not.

In 20% of applications with disclosed financial conflicts, neither the FDA nor sponsors took action.

The Inspector General's recommendations for the FDA were as follows:
Ensure that sponsors submit complete financial information for all clinical investigators.
Use a complete list of clinical investigators to check that sponsors have submitted financial information for all.
Require that sponsors submit financial information for clinical investigators as part of the pretrial application process.
Check that sponsors have submitted all required attachments to financial forms.
Update guidance to sponsors regarding the due-diligence exemption.
Add a review of financial information to the on-site inspection protocol.
Ensure that reviewers consistently review financial information and take action in response to disclosed financial interests.
Require that all centers consistently use a template that includes a prompt to document a review of financial information.
Provide additional guidance and training to reviewers.

FDA Replies

In response to a request from Medscape Medical News for comment, FDA spokesperson Karen Riley sent a statement noting that the agency agreed with all of the HHS recommendations except the one stating that the FDA should request information from clinical investigators prior to trial initiation.

"The intent of clinical investigator financial disclosure is not to discourage investigators from being included in the study," the FDA response says. "Rather, it allows sponsors to identify and manage potential conflicts throughout the development of a regulated product, from design through the conduct of the clinical trials."

The statement goes on to say that this recommendation for financial disclosures before trial initiation could needlessly add to "the complexity and cost of the clinical trial enterprise with no commensurate gain in the protection of human subjects or the quality of the data."

This pretrial disclosure recommendation is not meant to be punitive or burdensome, Dr. Powers said, but it recognizes the fact that bias, whether intended or not, can slip into the process whenever money is involved.

"When people bristle at regulations, I always think of James Madison's quote," he remarked. "Madison said that 'If all men were angels, no government would be necessary.' The reasons why we do these things is not because everybody is inconsistent or immoral: it's because some people are. It seems onerous to people who have no intention of doing the wrong thing, but unfortunately these rules are written for people who aren't going to do the right thing."

Monday, January 19, 2009

On-Line Petition for Access

I came across an on-line petition site urging the FDA to hurry the cures of type I diabetes, an autoimmune disease like psoriasis. My son has two cousins with Type I diabetes as well as several more with psoriasis. The tendency for autoimmune disease runs in families. While I wish the petition on line would advocate for all autoimmune disease and not just diabetes Type I, it did seem like a good idea to sign. At least it cannot hurt. Whether it will help or not I do not know. I urge you to go to the site and sign as well. Perhaps someone will listen.

I included the following statement with my signature. The statement below represents my frustration with the FDA.

FDA delays are not just a diabetes problem. My son has psoriasis, psoriatic arthritis, and ankylosing spondylitis. The FDA is grossly incompetent shows a complete lack of concern for all sick Americans. A NOVEL medication (Ustekinumab) showed "stunning results" for conditions like my son has in phase II clinical trial results released in February of 2007.

Naively back in the spring of 2007, I thought this brand new kind of biologic medication (anti IL 12/23) would be rushed to patients in weeks or at most a few months. I was sure that once the FDA officials in charge read about the results that they would do ever thing possible to get it to suffering patients. But NO! Here we are TWO YEARS LATER and the FDA still has not approved the medication.

Last June Ustekinumab was recommended for FDA approval by 100% of the outside experts who were brought in to evaluate it. That was SEVEN MONTHS AGO!

In September results of head to head clinical trial were announced. The trial had pitted ustekinumab against the number one prescribed biologic for psoriasis, Enbrel. Ustekinumab out performed Enbrel. The new investigational medication was more efficacious than the number one prescribed medicine. It has to be approved immediately, right? NO, that was FIVE MONTHS ago and there still is no approval.

Ustekinumab is ONLY a bridge to a future cure. But it is a dramatically better medication than anything now on the market. Its approval should be an EASY DECISION.

My son has nearly died twice form the severe inflammation in his ribcage due to the PsA and AS. In the summer of 2007, my wife and I watched him struggling to breathe, wondering which would be his last breath. We desperately tried to get Ustekinumab from the manufacturer and were TURNED DOWN! There is no appeal not even a court appeal (google Jacob Gunvalson to see how useless the courts are). The FDA gives TOTAL decision making power to the company. The dying patient HAS NO RIGHTS!

We have tried unsuccessfully for last two years to get compassionate use for our son to use ustekinumab. He has been denied over and over and over.

We have four times tried to get him into clinical trials. Four times he has been TOO SICK. He was excluded.

The myth that sick Americans can get access to investigational medication is a cruel hoax. The truly sick are denied due to narrow exclusionary and inclusionary criteria meant to get drug approved. The clinical trial and its criteria are NEVER set up to help patients. They are not even to get complete scientific results.

The current clinical trials system is rigged. There is no dearth of volunteers as the drug companies say. Instead there is a dearth of “just right” patients that are carefully pre-screened to give drug best possible chance of approval. Less than 10% of dying cancer patients are given access to clinical trials. THIS IS NOT A SCIENTIFIC SYSTEM, nor is it compassionate.

The sickest Americans are given NO access.

Today there is a chance for a cure for approximately 50% of autoimmune and perhaps 95% of blood disorder patients (beta thalessemia, hemophilia etc) who are “missing something.” The new treatment is called Prochymal by Osiris. Prochymal is an infusion of blood and immune forming stem cells from many donors of blood. It does not need to be matched for blood type. By providing the "missing factor" it turns off autoimmune disease in as little as a few days in some patients. Hemophiliac patients who are all missing clotting factor could be cured in a week or less. Diabetes could be turned off and insulin levels normalized in a month. Prochymal is just one of the MAJOR breakthroughs in cures that is being delayed by the FDA,s career protecting bureaucrats and by the FDA's Byzantine clinical trials protocols.

Prochymal should be tried on every autoimmune disease and blood disorder TODAY not two decades from now. Ustekinumab a big step forward but not even close to a cure yet even this relatively small step has been delayed for two years. Two years of dying and suffering patients for no reason. Still it should be approved today. It also should be tested on ALL other autoimmune diseases especially on ones with similar inflammatory pathways--diabetes type I , psoriasis, psoriatic arthritis, ankylosing spondylitis, rheumatoid arthritis and others.

Prochymal, various autoimmune vaccines and other procedures offer the chance for LIFE TIME CURES. Why isn’t the FDA jumping on board and speeding up the approval of these revolutionary advances?

Ustekinumab is just one example of TOTAL INCOMPETENCE and feet dragging by FDA bureaucrats afraid to say "yes" to anything. There need to be a new kind of “affirmative action” hires for FDA bureaucratic jobs. These affirmative action folks would either have chronic disease themselves or have a family member with one. If the FDA decision makers had something to care about besides their protecting their jobs, then perhaps, they would allow these revolutionary new drugs and technologies come to market, QUICKLY!

Enough of industry insiders running the FDA! Enough of career bureaucrats protecting their back sides! APPROVE THE CURES NOW!