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.
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.