Abigail’s 16-Year Senate Victory Over FDA Bureaucrats - The American Spectator | USA News and Politics
Abigail’s 16-Year Senate Victory Over FDA Bureaucrats

It was early March 2001 when 21-year-old Abigail Burroughs was told by her oncologist that conventional options for her cancer had been exhausted. The good news was that a new drug Erbitux had a good chance to save her life.

The problem was that the Food and Drug Administration had not approved its use.

Even though weakened, Abigail, with her father Frank, and family and friends, began lobbying the drug companies; but they could do nothing without FDA approval. They petitioned Congress but even widespread and sympathetic media coverage could not budge FDA.

Like all government bureaucracies FDA had expertise but its procedures emphasized caution, with its monopoly powers allowing it to proceed at its own unhurried pace and ignore outsiders.

So Abigail died on June 9, 2001, a week and a half after doing an interview with WJLA-TV when she told host Dale Solly, “This is not just about me. I am trying to help so many others.”

Frank recounted that just hours “after the most horrible tragedy I could have ever imagined, I decided that I would continue with my efforts.” He organized Abigail Alliance for Better Access to Developmental Drugs with few resources to lobby for others with fatal diagnoses who could not obtain treatment with drugs from government experts claiming their four stage/two-year approval process was for patients’ own good even if they expired before it was completed.

Frank and the Alliance spent the next several years trying to persuade government and media, achieving partial reforms from the FDA, some of which became standard care for seriously ill patients. The Alliance targeted drugs that had cleared preliminary Phase 1 testing and even more-advanced Phase 2 or Phase 3 trials which had proven that the drugs had worked at least for some test participants but it still was not able to substantially expand access to needed drugs.

In 2003 the Alliance and the Washington Legal Foundation went to Federal court to argue that terminal patients with no options left but death have a constitutional right to therapy if under the care of a qualified physician. They lost in an appeals court in 2007, but as Ronald L. Trowbridge and Steven Walker wrote in the Wall Street Journal at the time, the following examples given in testimony by the Alliance demonstrated the “tragic standard for loss of life at the hands of FDA bureaucrats” over this period.

  • Coming out of Phase I testing in 1998, the experimental drug Gleevec was “known beyond any reasonable doubt to be safe and effective.” The Alliance started requesting access to the drug for chronic myelogenous leukemia in June 2001. FDA did not approve Gleevec until March 2003, during which period approximately 3,600 patients had been denied access to the drug, with many dying. Of the few granted exceptions in small clinical trials by the FDA, more than 80% who received Gleevec were still alive at the time of the court case.
  • The FDA rejected the drug Eloxatin, for advanced colorectal cancer, in March 2000 despite its being approved in at least 29 other countries. In January 2002, the Alliance asked for reconsideration but the agency delayed approval until August, during which period about 40,000 such cancer sufferers died.
  • Erbitux was proposed to mitigate colorectal and head and neck cancers and was rejected by FDA in December 2001 after a six-month appeal by the Alliance. The FDA finally approved it in February 2004, leaving almost 179,000 people to die from colorectal and head and neck cancer in the meantime.
  • The Alliance began working for access to Revlimid, for multiple myeloma and myelodysplastic syndrome, in June 2002. Patients had to wait until December 2005 for FDA approval. Nearly 74,000 patients with these terminal cancers died without ever getting Revlimid.
  • Patient access to Velcade to treat multiple myeloma was begun by the Alliance in June 2002, which the FDA did not approve until May 2003. Today the FDA points to this drug as proof it can work in a timely fashion but they only approved it for 25% of sufferers (since shown to be too low), but even with that limitation, about 2,600 patients died without ever getting Velcade.
  • In June 2004, the Alliance started pushing the FDA to make Nexavar and Sutent, available for kidney cancer treatment. FDA approval for both drugs required dying kidney cancer patients be given the choice of death from their cancer or a 50/50 chance of being blindly randomized to a sugar pill. The evidence of efficacy was so compelling for Nexavar that the Bayer drug company stopped the test for ethical reasons and allowed the placebo patients to get the drug. The agency approved Nexavar in December 2005 and Sutent in January 2006, with about 20,000 kidney cancer patients dying waiting for both.
  • Avastin for colorectal and lung cancers was petitioned for approval in June 2002. FDA finally approved this obviously effective cancer drug successfully used off label in February 2004. Almost 360,000 patients died in the meantime.
  • Approval for lung cancer treatment drug Tarceva was requested in June 2001 but the FDA did not approve it until November 2004. In the interim, 531,000 people with lung cancer died. Tarceva also extended the effectiveness of an existing drug for pancreatic cancer, and about 102,000 patients died from that disease during the FDA’s delay.
  • Lobbying for approval of Bexxar for non-Hodgkin’s lymphoma began in June 2002 and was not approved until after intense pressure from oncologists in June 2003. About 26,000 died during the delay. The Alliance called the FDA’s “intransigent and incompetent” opposition during the test period a “regulatory hatchet job” that caused the drug to be “dramatically underused” thereafter.
  • Attempts to gain access to Alimta for lung cancer began in June 2002. It was not approved by FDA until February 2004, during which period approximately 249,000 lung cancer patients died.
  • The Alliance began efforts for access to Tykerb for breast cancer in June 2004. About 25% of breast cancers include the biomarker predictive of benefit from Tykerb. Nearly 28,000 women who had this marker died from their cancer waiting for Tykerb until it was approved in March 2007.

Trowbridge and Walker summarized the FDA treatment of these 12 drugs that “had they been available to people denied entry to clinical trials — it might have helped more than one million mothers, fathers, sons and daughters live longer, better lives. We have actually underestimated the number of ‘life-years’ lost at more than 520,000, because we have not included other safe and effective uses of these drugs that the FDA has yet to approve.”

The FDA responded by allowing a relatively limited number of terminal patients into small clinical trials and what they labeled an “expanded access/compassionate use” program covering a few thousand per year. Patient rights activists persevered by promoting what they called “Right to Try” bills granting right to treatment for all terminally ill patients under proper medical supervision. Such bills were adopted in 38 states with the support of the Goldwater Institute, including by Vice President Mike Pence when he was governor of Indiana.

The FDA opposed each state bill as well as a national version as not necessary because their programs had already expanded access, claiming that drug companies being private companies would still be able to refuse treatment, and that such a policy would undermine FDAs ability to guarantee drug safety for others. Matthew Bellina, a Navy veteran with ALS, responded in 2016 through the Washington Post PowerPost that FDA “disincentivizes” doctors and companies from participating in their own programs “by making the process onerous and by refusing to put in writing that the agency will not use any adverse events that happen outside of clinical trials to shut down a trial or delay approval.”

“If a patient is willing to try a promising treatment — even when he or she understands the risks, a doctor thinks the treatment may help the patient more than anything else on the market today, and a company agrees and is willing to provide the treatment — the federal government should not get a veto stamp,” Bellina argued. The FDA claimed it approved almost all requests for compassionate treatment, about 1,200 a year. But  Dr. Razelle Kurzrock of the University of California, San Diego, who ran clinical trials at MD Anderson Cancer Center, told the Goldwater Institute she would “spend hours on the phone with the FDA trying to get a verbal commitment” beforehand. “It’s almost a self-fulfilling prophecy for the FDA to say they approve everything, because you don’t even put in the application before you sort of get a verbal approval from the FDA that it’s worth doing.”

“In 2014, nearly 25,000 people in France were using investigative treatments through that government’s equivalent program,” Bellina continued. “If a country with one-fifth the population of the U.S. can help 2,000 percent more people, we clearly have a problem. No one advocating for the state or federal Right to Try laws thinks it is a magic pill. But it does open up new options for people like me whose time is running out.”

“What is the downside of creating new pathways for the terminally ill to access promising treatments? Maybe the law won’t help millions of people, or even many — but for those that it does help, it’s a game-changer. Just ask the 78 terminal cancer patients who are still with us today because of a state version of this law.”

In response to Bellina’s testimony to Congress and years of public advocacy, Wisconsin Republican Sen. Ron Johnson introduced a Federal Right to Try bill with Democratic Sens. Joe Donnelly, D-Ind.; Joe Manchin, D-W.V.; Angus King, I-Maine; and 40 Republicans as co-sponsors. President Trump supported it too. The bill passed the Senate on August 1, 2017 and was referred to the House of Representatives for final action.

It took 16 years after Abigail Burrough’s death for her inspiration to overcome the regulatory insensitivity of the government’s monopolistic drug approval process — and it is still not law today, with the FDA still opposed but hopefully less intransigent about implementing the law than it was for Bexxar and the others.

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