HON. RON PAUL
OF TEXAS
IN THE HOUSE OF REPRESENTATIVES
Thursday, August 2, 2012
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Mr. PAUL.
Mr. Speaker, I rise to introduce the “Compassionate Freedom of Choice Act.” This legislation allows terminally ill patients to use drugs, treatments and devices that have not yet been approved by the Food and Drug Administration (FDA) if their physicians certify: (i) such patients have no other treatment options; and (ii) the patient executes written, informed consent that they are aware of any potential risks from the drug, device, or treatment.
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It is important to remember that this legislation only applies to otherwise terminally ill patients. Denying these patents a possible opportunity to cure their illness—or at least reduce their suffering—is nonsensical and cruel. The FDA’s approval process for drugs, devices, and treatments is costly and time consuming. Yet, time is the luxury terminally patients do not enjoy. So why should the FDA deny terminally-ill patients access to drugs, devices, and treatments that the patient’s physicians have determined represents the patent’s only possible chance for survival?
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For example, the FDA refused to allow Abigail Burroughs (who was diagnosed with head and neck cancer at the age of 19) access to the cancer drugs Iressa and Erbitux by the FDA. Never mind that a renowned oncologist at Johns Hopkins had determined there was a significant chance of saving her life if she could use these new drugs. With her only chance of survival denied by the federal government, Abigail passed away on June 9, 2001, at the age of twenty-one.
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Another example of why this bill is necessary is the case of thirteen-year old Anna Tomalis, who enjoyed horseback riding and soccer until she was diagnosed with embryonal sarcoma. Chemotherapy and surgery failed to reverse the cancer, so Anna’s parents decided to try experimental drugs. They petitioned the FDA for approval to use Deforolimus, developed by Merk and ARIAD. Unfortunately, the FDA decided Ana was too sick to be admitted in Deforolimus’s clinical trials and did not grant her a “compassionate use” exemption until three weeks before she died.
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Mr. Speaker, I have attached a list of other patients who were denied access to treatments by the FDA even though their doctors believed these treatments where the only option left to potentially save their lives. I ask my colleagues to help make sure that no more Americans with terminal disease are denied treatments simply because the FDA has decide these Americans are better off facing certain death than using an “unapproved” drug, treatment, or device. Please cosponsor the Compassionate Freedom of Choice Act.
PERSONS DENIED ACCESS TO COMPASSIONATE DRUG USE BY THE FDA
(I) ABIGAIL BURROUGHS
Abigail Burroughs learned at the age of nineteen that she had head and neck cancer. http://abigail-alliance.org/WLFl FDAlLawsuit.pdf (last accessed May 21, 2012). For the next eighteen months, Abigail fought the cancer by undergoing painful chemotherapy and radiation treatments, to no avail. Id. Abigail was told in March of 2001 that she had run out of FDA-approved options. Id. Abigail’s cancer cells had very high EGFR (Epidermal Growth Factor Receptors) expression. Her renowned oncologist at Johns Hopkins knew there was a significant chance of saving her life if she could get the new EGFR cancer drugs Ire ssa and Erbitux. Id. Abigail could not get Iressa, however, because the clinical trials were very limited as to the number and type of patients who could qualify—as is usual for clinical drug trials. Id. The Erbitux clinical trials were for colon cancer patients only. Id. Abigail never obtained Iressa or Erbitux, and thus a chance to live, and so she died on June 9, 2001, at the age of twenty-one. Id. The Abigail Alliance, created by her father Frank Burroughs shortly after her death, is a 501(c) non-profit organization and can be accessed through http://abigail-alliance.org. The Abigail Alliance is now dedicated to expanding access to experimental drugs through the compassionate use exemption.
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(II) DAVID BAXTER
High school student David Baxter was diagnosed with colorectal cancer in the spring of 2001. http://abigail-alliance.org/WLF FDA Lawsuit.pdf (last accessed May 21, 2012). David was unable to participate in clinical trials of promising new cancer drugs because clinical trials are usually open only to patients eighteen and older. Id. In the following months he endured various types of chemotherapy. Id. Of one of his hospital stays, he wrote, “You hear a lot of scary stories about cancer patients, and let me tell you right now that they are true—every single one of them.” Id. From the stories of nurses coming in at two in the morning to take your vitals for some awful reason, to the noises from the room across the hall—either screams or moans of who knows what.” David died in his sleep at home on October 6, 2001, shortly after his seventeenth birthday. Id.
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(III) ALITA RANDAZZO
Alita Randazzo, age thirty-five, was diagnosed with colorectal cancer in the spring of 2000. Id. Alita responded well at first to Eloxatin (Oxaliplatin), but she had to endure the expense and physical demands of traveling to France to get the drug. Id. She did not qualify for the clinical trial of Eloxatin in the U.S. and was not fortunate enough to get into the drug’s limited compassionate use program. Id. (Before finally being approved in the U.S. in May of 2003, Eloxatin had been approved in Europe six years earlier.) After eight months, Eloxatin stopped helping Alita and her doctors believed her last chance was Erbitux. Id. Alita was unable to obtain Erbitux, and died on July 20, 2002. Id.
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(IV) JOEL OPPENHEIM
Joel Oppenheim was first diagnosed with
multiple myloma in 1995 but the disease did not become active until 1999. Id. At that time, he was treated with dexamethadrone (“dex”), which had unpleasant side effects and was only minimally effective. Id. Thalidomide, which is not approved for multiple myeloma, was added to the dex by Joel’s doctors. This off-label use of thalidomide was possible because thalidomide had been approved for leprosy, and is thus available for
doctors to prescribe for other conditions as they see fit. Thalidomide has become the first line of treatment for multiple myeloma. Id.
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As Joel’s disease worsened in 2000, his oncologists recommended that he seek to participate in clinical trials of Revamid or PS–341 Velcade. Id. Revamid is a derivative of thalidomide that avoids thalidomide’s side effects (which extend well beyond its notorious effect on pregnant women). Id. Joel was unable to obtain a place in the Revamid trials or Velcade trials because his prior treatment with dex put him outside the narrow protocols of the trial. Id. The massive number of patients who applied for the trials would have rendered it unlikely for Joel to win a place, in any event. Thus, Joel was prevented from using Revamid, which was safer and more effective than his thalidomide treatment. Id.
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In light of Joel’s inability to obtain Revamid or Velcade, his oncologists recommended an autologous bone marrow transplant, which he underwent on April 15, 2001. Id. This is a dangerous and damaging medical procedure. Id. Joel survived the transplant, but was disabled from working and still suffers from an impaired immune system. A disease such as the West Nile virus that would typically have mild effects on other people would probably kill Joel. Id.
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Approximately a year and a half after the bone marrow transplant, Joel’s cancer worsened again. Id. He again attempted to enter numerous trials for Velcade, but was rejected. He was disqualified from some trials on account of his prior dex treatment and from others on account of his transplant (which had been made necessary by his lack of access to Velcade or Revamid). Id. To increase his chances of acceptance into a trial, on his doctors’ advice, Joel stopped taking dex or any other treatment; one of the criteria of the trials was no dex or other drugs within the prior six months. The trials were repeatedly delayed. Id. Without medication, Joel’s cancer grew much worse. Id. Finally, in June of 2003, through the efforts of one doctor, Joel was admitted to a trial of Revamid. Over the last three years, FDA restrictions on investigational drugs have caused countless patients like Joel to die or suffer from bone marrow procedure. Id.
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(V) GIDEON SOFFER
Gideon was active in the Los Angeles Jewish community and a student at U.C. Berkeley. http://abigail-alliance.org/WSJ%20Sen% 20Kennedy%20Gideon.pdf (last checked May 21, 2012). He passed away January 11, 2012. http://www.tributes.com/show/Gideon-Joseph -Sofer90709467 (last checked May 21, 2012). In 2003 he was 22 years old; he stood five foot six inches tall and weighed just over 100 pounds. Id. He suffered from Crohn’s disease which caused him to remove half of his intestine. Crohn’s disease is an inflammatory disease of the bowels, which can cause breakage and perforations in the intestines. 1.5 million Americans suffer from it, including a disproportionate number of Ashkenazi Jews. Id. In 2007 Gideon enrolled in a clinical trial for a treatment that could save his life: an adult stem cell therapy that helps damaged intestinal tissue regenerate from the inflammation caused by Crohn’s. The sponsor, Osiris Therapeutics, reported that Crohn’s patients in the therapy’s Phase II trial all experienced clinical improvement after receiving the cells. Id. A Phase III trial for the treatment was nearing completion at the time of the petition, but the FDA approval was years away. He was placed in the trial but was withheld the life-saving drug and given a placebo instead. http://abigail-alliance.org/ WSJ%20Sen%20Kennedy% 20Gideon.pdf (last checked May 21, 2012).
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(VI) KIANNA KARNES
44 year old Kianna Karnes was a mother of four and grandmother of one when doctors told her she had kidney cancer that was spreading throughout her body. http://abigail-alliance.org/3_WSJ_Editorials_Kiannas_ Law.pdf (last checked May 21, 2012). Two different developmental drugs, BAY 43–9006 and SU 11248, showed great promise against this once untreatable disease, but the FDA did not move to approve the drugs, and instead began imposing new testing requirements that make it all but impossible for their developers—Bayer and Pfizer—to provide them to terminal patients on a “compassionate use” basis by forcing a placebo. Id. Karnes died in 2005, the very same day that the FDA granted her compassionate use exemption. Id.
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(VII) ANNA TOMALIS
13 year old Ana Tomalis liked horseback riding and soccer. http://online.wsj.com/article/SB1219447890_05365195.html?mod = opinion _main_commentaries (last checked May 21, 2012). From 2005 to 2008 Ana fought embryonal sarcoma, a rare form of liver cancer. Id. After chemotherapy and surgery did not work her parents turned to experimental drugs. Id. One drug that they petitioned the FDA for was Deforolimus, developed by Merk and ARIAD. Unfortunately Ana was too sick to be admitted in the clinical trials and the FDA did not grant her a “compassionate use” exemption until three weeks before she died, August 15, 2008. It was too little too late. Id.
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(VIII) JACOB GUNVALSON
Jacob, since the age of eight, had Duchennemuscular dystrophy, a rare and devastating disease, which has confined him to a wheelchair. http://www.huffingtonpost .com/2008/12/17/jacob-gunvalson-terminall _n _151650.html (last visited May 21, 2012). His mother, Cheri Gunvalson, with a master’s degree in nursing, was instrumental in getting federal legislation passed to provide more research money for the disease. Id. Jacob’s only hope was PTC Therapeutics’ PTC124, an experimental drug given to only 165 in a clinical trial. Id. Jacob was ineligible because his disease was too far advanced already rendering him unable to walk. Id. In December of 2008 the Third Circuit ruled against the Gunvalson’s reversing a lower court decision stating that a pharmaceutical company does not have to provide an experimental drug to terminally ill person. Id.
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(IX) BRANDON RYAN
Brandon, at 22 years old, had severe melanoma skin cancer.http://www.nytimes.com/ 2010/09/19/health/research/19trial.html?page wanted=5&_r=3&partner=rss&emc=rss&src =ig (last visited May 21, 2012), http:// www.legacy.com/obituaries/ bakersfield/obituary.aspx?n=brandon -p-ryan&pid=143628760 (last visited May 21, 2012). He shared that skin cancer and the shared slim chance of survival with his cousin Thomas McLaughlin. Id. A new drug issued by Roche called PLX 4032 was being offered in clinical trials at the University of Los Angeles Medical Center which was near their homes. Id. Thomas who was in the trial urged his cousin to join lauding that the tumors stopped growing after only two months of taking the pills. Ryan was admitted to the trial but was assigned by the lottery to be the control arm—instead of taking the pills; he was to get infusions of a chemotherapy that had been notoriously ineffective in treating melanoma. Id. The standard chemotherapy used in melanoma, decarbazine, slowed tumor growth in 15 percent of patients for an average of two months. Id. By contrast, PLX 4032 had halted tumor growth
in 81 percent of patients for an average of eight months. Brandon died in 2010. Id.
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(XI) LIDDY SHRIVER
In April 2002, Elizabeth Shriver was diagnosed with Ewing’s sarcoma. http:// sarcomahelp.org/liddy.html. She died January 15, 2004 at the age of 37 after the cancer metastasized to her right thigh, lungs, brain and abdomen. Id. Liddy was a computer scientist and sought to approach the cancer as “just another problem to solve reading as much as she could about cancer, various treatments, and the results of clinical trials. Id. More information can be reached by going to http://sarcomahelp.org/index.html. (XI) LORRAINE HEIDKE MCCARTIN
Lorraine raised four children and has been battling a rare form of breast cancer since 2006. http://www.boston.com/business/health care/articles/2011/01/05/testinglruleslforce lpatientsltolwaitlforlnew drugs/?page=2 (last viewed May 21, 2012). In 2010 her hopes were buoyed when doctors said she was a good candidate to take a promising experimental drug called T–DM1 which had reduced patient’s tumors with few side effects in clinical trials. Id. But before the treatments were to start under an expanded access program the drug’s maker Roche shut down the program after the FDA refused to speed the approval process. Id. She is still alive, but must drive about 500 miles to get the treatment.
