Instead, often the main hurdles, as the Forbes family noted, are whether the drug company has an expanded access program for a specific drug and, if so, whether to approve the application. There is no requirement for companies to report how many orders they accept, although some large pharmaceutical companies make this information public.
Perhaps more importantly, it is not known to what extent family media campaigns affect these decisions. That’s what worries Arthur Caplan, a professor of bioethics at the Grossman School of Medicine at New York University. “If you have the resources, hire PR people, build your own website, and you have a good chance of blaming or shaming the company for providing something for you,” he told me. “I understand why people do this, but it’s hardly an equitable system.”
Dr. Caplan pioneered a possible solution. Working with Janssen Pharmaceuticals, a part of Johnson & Johnson, he leads an advisory committee of physicians, ethicists and community members that evaluate these requests and then develop a recommendation for the pharmaceutical company. As well as the American organ allocation system, which Dr. Caplan also helped to develop, this committee aims to ensure a fair allocation of a limited resource. Pharmaceutical companies should not be swayed to say yes by the nicest pictures or pressured into saying no out of concern that a negative result could affect consumer confidence in a drug. Instead, the committee applies consistent criteria to each anonymous potential recipient and makes a decision based on medical factors rather than the pathos of a personal story, wealth, or media prominence.
This is a start, especially when combined with better outcome tracking and better education for clinicians on how to enroll their patients in clinical trials and navigate expanded access if such trials are not a possibility. A lawyer by training, Ms. Forbes has done most of this browsing and advocacy on his own, but there are many families for whom this would not be possible. And when doctors’ time is a limited resource, how much of that process can they realistically take on for a drug that hasn’t yet been proven to work?
That’s the question behind much of this conversation. How far do we go when the alternative is death? And what is sacrificed in the process? “You’re hoping that you can be so rare, that if you can stay alive long enough for the next drug, it could change things,” Forbes said, reflecting on those last few weeks.
Even though her husband never received the expanded access drug, she doesn’t regret putting so much time and effort into getting it. They believed this drug would work, she said, but they were also able to consider a reality In which Forbes did not live to see it.
“Despite how it all ended, it was good for Michael to feel positive and hopeful,” she said. “It was really good for me, because there was no stone unturned.” This is not the kind of victory they wanted, but it is something.