FDA CONDEMNATION OF THE TERMINALLY ILL
One out of every two Americans will likely be stricken with some form of cancer. FDA approved cancer treatments are ordinarily not curative. Those treatments, consisting principally of radiation and chemotherapy, offer little hope and much misery for the vast majority of cancer patients. The FDA approved treatments rarely result in longevity greater than if the patient received no treatment at all, yet access to unapproved, experimental treatments depends on FDA allowance. While FDA allows access to experimental drugs sponsored by favored regulatees (large drug companies with a long history of agency drug approvals), it often denies access to experimental drugs sponsored by innovative companies that lack a cozy relationship with the agency. FDA’s control of access is rife with conflict of interest, bias, arbitrariness, and subjective discrimination. Assuming near Godlike power, FDA ultimately determines who may have access to unapproved cancer treatments that hold out hope for a cure and who may not.
A recent brilliant documentary by Nehst Out entitled Cut Poison Burn (available at cutpoisonburn.com) reveals the desperate and horrible circumstances befalling those who must not only struggle to fight cancer afflicting their loved ones but also a Food and Drug Administration that denies them access to their treatment of choice and condemns them to FDA approved therapies that even their conventional oncologists have deemed incapable of arresting the cancer’s progression. The documentary focuses on the Navarro family’s struggle to save a beautiful young boy, Thomas Navarro, from the ravages of glioblastoma (an aggressive brain tumor). As if the torture endured through rounds of chemotherapy and radiation were not enough, the Navarros must also struggle with an FDA that in their most urgent hour denies them access to a promising experimental cancer treatment and consigns Thomas to a horrible death.
Despite its abysmal track record since President Nixon declared war on cancer in 1971, conventional cancer treatment has become very big business. In 2010, cancer care cost the American public an estimated $125 billion, yet even with expenditures of that enormous size almost all have died either from the treatments administered or from cancer progression, or both. The ugly truth is that the federal government has lost the war on cancer yet compels Americans to purchase the same failed treatments for the disease by locking out promising experimental alternatives. FDA jealously guards the interests of the largest drug manufacturers, commonly allowing access to clinical trials of their cancer drugs, while disallowing access to clinical trials sponsored by individuals and companies that lack a cozy relationship with agency regulators. The consequence is a horrific destruction of innovation in medicine and of hope for terminally ill cancer patients. Federal law allows FDA regulators to possess the power to determine the fate of the terminally ill, yet those regulators have an inherent conflict of interest that influences their decisions. If they leave government service, they will do well financially if they secure positions of employment with major drug companies, but they will limit prospects for such employment if they offend those regulatees by allowing competing innovators to expand treatment uses of experimental cancer drugs.
Because half the American population is bound to contract cancer and most cancers are incurable, a just government would do everything in its power to ensure that those diagnosed with cancer would be informed of and encourage to explore alternatives, provided they were informed of the potential risks and known benefits of each option as compared to the FDA approved treatment. That is not the case, however. For those seeking an alternative to FDA approved treatment and to FDA favored drug manufacturers, the agency has an all too common answer: No (and often “no” without any rational explanation).
FDA jealously guards its gate-keeper role, whereby drugs are only allowed to be marketed if they have been given FDA approval. The system is one FDA Associate Director of the Office of Drug Safety David Graham has described as corrupt, favoring incumbent drug company regulatees even to the extent of approving unsafe drugs like Vioxx and Avandia. Economists schooled in public choice theory regard FDA as a quintessential example of industry capture (whereby the regulators become servants of the agency’s principal regulatees). FDA argues that it must paternalistically police who has access to experimental drugs because to do otherwise would permit unapproved cancer therapies that may be harmful to proliferate, making a mockery of the agency’s costly drug approval process. Companies and individuals would avoid the expense and burden of seeking FDA approval for drugs, the agency contends, choosing instead to make the unapproved drugs available to large segments of the population without seeking agency approval.
That argument is quite fickle, and it rests on a series of false assumptions. At the outset, because FDA approves or disapproves clinical trials, the universe of trials is limited to those trials which the FDA believes hold out some promise of efficacy. In addition, the number of people who are terminally ill with cancer for whom conventional treatments are inefficacious is a set figure and involves people for whom FDA has failed (in other words, despite their dire need there is no FDA approved treatment capable of curing their terminal illnesses). Demand for cancer treatment at any particular moment is inelastic and, so, the notion that an unlimited expansion in patient sales would occur in the market is fictive. Moreover, when conventional treatments are inefficacious, hope for life depends entirely on access to experimental drugs which, by definition, are not FDA approved, regardless of who sponsors them. Finally, in practice FDA does not deny cancer patients access to experimental drugs in toto, regardless of the sponsor. Rather, FDA acts selectively, granting access based on the exercise of subjective, politically influenced opinion. The very same patient denied access to a clinical trial whose sponsor is disfavored by FDA can be admitted to a clinical trial for a different drug by a sponsor favored by the agency.
The system for allowing access is corrupt, heavily subject to political influence, and biased in favor of drug company sponsors favored by the FDA, namely those that have a cozy relationship with the FDA from years of seeking and obtaining approval of drugs. Congressmen Dan Burton and Peter DeFazio know well the corruption within the agency. Each has confronted FDA Commissioners who condemned without recourse patients seeking access to experimental drugs by sponsors disfavored by the agency. Each has demanded reversals of those decisions and in several instances (despite FDA asserting to sponsors and patients that the decisions were medically based and final), the Commissioners have ordered reversals bowing to political pressure, sometimes too late for the patients concerned.
When a patient seeks an experimental drug for a serious or life-threatening condition, an ordeal of extraordinary proportions may confront the person when he or she is least able, physically and emotionally, to endure it. The drug trial sponsor must be contacted and convinced to seek a “compassionate use” exemption from the FDA. That request for exemption comes in the form of either a “single patient investigational new drug” submission by the sponsor or, if the patient may die imminently, an “emergency investigational new drug” submission (which may be made by phone). FDA political appointees exercise enormous subjective discretion in determining if a patient with cancer will be permitted to have access to a clinical trial of an experimental cancer drug. FDA ordinarily allows afflicted patients access to an experimental drug, provided that it is recommended by a physician and is acceptable to the drug company sponsor. In a significant number of cases, however, the FDA refuses access to a clinical trial. Although the agency is loath to admit it, denial of the patient’s choice occurs in those instances where the FDA harbors a bias against the sponsor or the treatment. Sometimes that bias is born of good evidence that the clinical trial is fraudulent or that the experimental drug is too dangerous, but it may also be born of an agency effort to ensure that favored regulatees are protected against new or novel cancer treatments of promise from an individual or company not among those having close ties to the agency.
The decision to deny a dying patient access to an experimental drug is an extraordinary exercise of federal power. That horrific decision is made daily by the directors of FDA’s Division of Oncology Products (Drs. Robert Justice and Patricia Keegan) under the direct supervision of the FDA Commissioner Margaret Hamburg. The FDA has criteria in 21 C.F.R. § 312.305 that limit access, but it exercises considerable discretion in interpreting the criteria, resulting in inconsistent decision making. Repeatedly members of Congress, most notably Congressman Dan Burton from Indiana and Congressman Peter DeFazio from Oregon, have pressured FDA Commissioners to reverse decisions denying access that the Oncology Products Division Directors declared final. While FDA Commissioners protest that their decisions are wholly science based, in fact the subjective criteria and the inconsistencies in decision prove that bias is the norm and that those with political access to the powerful can achieve reversals.
When determining if a patient will be given access to a clinical trial, FDA considers a few subjective factors. First, it requires that the patient have a “serious or immediately life-threatening disease” that is essentially not treatable with FDA approved drugs and devices. In 21 C.F.R. § 312.300(b), FDA reveals the inherent subjectivity in this determination: “Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such key factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one.” Next, FDA evaluates the treatment, again on largely subjective grounds, assessing whether “[t]he potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.” FDA then insinuates its anti-competitive bias into the process, deeming even a drug for which clinicians recommend access for the terminally ill be kept from those patients on the basis that allowing the use would “interfere with” potential FDA market approval for the drug or because FDA deems other drugs under another IND or FDA approved drug protocol available for treatment use.
When the drug sponsor is a large pharmaceutical company having a portfolio of several FDA approved drugs seeks a compassionate use exemption, it is ordinarily granted. The subjective factors are all resolved in the sponsor’s favor. When the drug sponsor lacks that cozy relationship, FDA often subjectively concludes the treatment to be one with potential risks that are unreasonable or concludes the condition to be one for which FDA approved treatments remain available. It is, of course, always the case that an FDA approved treatment is available for cancer or that another IND by a favored regulatee is available for treatment use. The factor is thus entirely fungible, depending on the political preference of the regulator. Chemotherapy, radiation, and surgery are conventionally approved to one extent or another for every cancer. So FDA may always conclude that an experimental treatment is unwarranted because the patient could receive treatments that are FDA approved instead or could participate in some other IND for the cancer.
The FDA’s political manipulation of the compassionate use process is one of the greatest examples of arbitrary and capricious agency action. It carries with it consequences acceptable to no one but the FDA bureaucrats who administer the program: destruction of patient hope and life. When a director of the Division of Oncology Products decides to deny a terminally ill patient access to an experimental treatment, that interposition of federal power between doctor and patient has profound consequences. Invariably the patient’s last, best hope for cure is removed by force of law, compelling the patient either to leave the country in search of the same or comparable treatments, return to horrific chemotherapy and radiation treatments that will make life unbearable and may hasten death, or resign to die.
On June 17, 2011, Patricia Clarkson was diagnosed with Stage III multiple myeloma. Although predicting an individual’s life expectancy is in fact impossible, Patricia’s doctor, like many oncologists and hematologists, make those unscientific predictions. Her doctor told her that she had no more than 4 to 5 years of life left. This common practice is not only unscientific but also cruel in the extreme and contrary to medical ethics because it inflicts injury, often causing the patient diagnosed with cancer to experience a profound loss of hope and a feeling of utter helplessness, conditioning them to accept with resignation whatever treatment regimen is recommended even treatments with lethal side effects. Often patients diagnosed with cancer and given an estimate of time left suffer a loss of the will to live and a diminution in their immune system that hastens death.
Patricia underwent a battery of tests, two MRIs, bone scans, and sophisticated laboratory analyses, all confirming the diagnosis of Stage III multiple myeloma. On June 18, 2011, she was hospitalized for sudden acute kidney failure, but she responded well to treatment and was released on June 21. While hospitalized, she was placed on Velcade, a chemotheraphy drug. The Velcade reduced her plasma tumors from 80% to 15%, but she began to experience gastroenterological reactions to the drug and severe pain in her lower back.
She was then given radiation therapy. Her reactions to the Velcade became so severe that her treatment regimen was halted, pending resolution of the reactions. In the interim, one of her oncologists advised that genetic testing revealed that she was missing chromosome 13 and gene p53 (a condition common among 50% of those afflicted with multiple myeloma), which he said would reduce her life expectancy to a year or two. Again, this heartless, unscientific pronouncement only further injured Patricia’s psyche atop the enormous physical suffering she had already experienced.
This oncologist recommended a bone marrow transplant which he said would extend her life one to two years (another unscientific prediction because bone marrow transplants in these circumstances are notoriously unsuccessful). Fortunately Patricia did her own research on bone marrow transplantation and decided that the length of hospital stay, risk of complications, and likely need for more than one transplantation surgery made it a foolish option.
In late November 2011, convinced that there was no conventional option that offered her any hope and that each option given her would come with greater physical disability and pain, decreasing, not increasing, her life expectancy, Patricia began looking for non-conventional alternatives. Having discussed the matter with trusted friends, she became convinced that she might benefit from receiving antineoplastons, a non-toxic, experimental drug discovered over thirty years ago by medical researcher Dr. Stanislaus Burzynski. One of those friends, Mary Jo Siegel, was diagnosed twenty years before with non-Hodgkins lymphoma and was pronounced cured following receipt of the antineoplastons treatment. Patricia traveled to Houston to meet Dr. Burzynski. Dr. Burzynski regretfully informed Patricia that the FDA had ruled that it would not grant any additional compassionate use exemptions for patients to receive his antineoplatons, although FDA had approved numerous exemptions in 2011. After conducting a physical exam and evaluating the test results, Dr. Burzynski recommended that Patricia take sodium phenylbutyrate along with the chemotherapy drug Revlimid until such time as a better option became available. Her local oncologist acknowledged Patricia’s decision to work with Dr. Burzynski, but refused to affiliate with him or provide local medical support, apparently for fear of FDA retaliation against her and her clinic.
Informed that the FDA could only be persuaded to lift its ban on Burzynski’s treatment if enough political pressure were brought to bear, Patricia contacted her member of Congress, Jerry McNerney. In December McNerney’s aides sent Erik Laughner, a consumer safety officer in the FDA’s Division of Oncology Products, a letter requesting a compassionate exemption for Patricia. The request was denied. FDA offered no explanation for its denial. Patricia then turned to Senator Diane Feinstein for help. She wrote to the Senator and asked her to urge FDA to permit participation in the Burzynski clinical trial. Senator Feinstein’s staff sent a letter to the FDA on December 14. On January 6, 2012, the FDA responded with a conclusory denial, reciting that it was aware of no data supporting “the use of antineoplaston therapy as a potentially safe and effective treatment for multiple myeloma.”
Patricia then met with Senator Feinstein’s aides and provided them with more details supporting her request for access to the Burzynski treatment. Dan Morrison of Senator Feinstein’s office later informed Patricia that he did not receive any response to his last inquiry to the FDA. He had no options to give Patricia other than encouraging her to contact the FDA directly by phone (a dead end given that FDA refuses to speak with patients about its compassionate use decisions; it only speaks to its regulatees, the clinical trial sponsors) or send a letter to the White House.
The war on cancer has become a war dominated by “friendly fire,” where the medical troops fighting the battle more often than not kill the civilian patients they are supposed to protect. It is a war we have lost yet continue to wage using the same failed munitions. It is a war the federal government allows to be waged by those with political influence but not by those without that influence who nevertheless have discovered treatments that hold out promise. It is a very corrupt and inhumane war.
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For Patricia Clarkson, the need for access to Dr. Burzynski’s antineoplastons is acute. Because political influence plays such an important part in this sordid business of convincing the FDA to permit a dying person freedom of choice, I urge those who read these words to correspond with the FDA and with their members of Congress to demand that FDA Commissioner Hamburg act now to reverse FDA’s decision to deny Patricia Clarkson antineoplastons treatment. You may register your complaint with Commissioner Margaret Hamburg at the following email address: Margaret.Hamburg@fda.hhs.gov. You should also ask Congressmen Darrell Issa, Chairman of the House Committee on Oversight and Government Reform, to demand that Commissioner Hamburg reverse her the denial of treatment. You can reach him on Twitter (@DarrellIssa). You should also ask Congressman Dan Burton, member of the House Committee on Oversight and Government Reform, to intervene on Patricia’s behalf. You can reach him on Twitter (@RepDanBurton).
� 2012 Jonathan W. Emord - All Rights Reserved