[Congressional Record Volume 158, Number 118 (Friday, August 3, 2012)]
[Extensions of Remarks]
[Pages E1407-E1409]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




          INTRODUCTION OF COMPASSIONATE FREEDOM OF CHOICE ACT

                                 ______
                                 

                             HON. RON PAUL

                                of texas

                    in the house of representatives

                        Thursday, August 2, 2012

  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.
  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?
  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.
  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.
  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.

[[Page E1408]]

       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/WLF_ 
     FDA_Lawsuit.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.


                           (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.


                          (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.


                          (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.
       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.
       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.
       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.


                           (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).


                           (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.


                           (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.


                         (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.


                           (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

[[Page E1409]]

     in 81 percent of patients for an average of eight months. 
     Brandon died in 2010. Id.


                           (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/testing_rules_force 
     _patients_to_wait_for_new 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.

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