Art Caplan, professor of Bioethics at NYU Langone Medical Center, says the scatter-shot way in which individual patients are left to petitioning drug companies for access to experimental drugs creates an unfair process.
"One problem in this country is we haven't set up a system. Right now what we've got is a squeaky-wheel lottery; if you can figure out what your options are, if you know how to use social media, if you know how to have a bake sale, if you can hire a PR firm -- you can have a shot at an unapproved drug," says Caplan.
"But that's all obviously inequitable relative to the number of people who might want to seek compassionate use."
Caplan says that without more safety and efficacy data on BioMarin's drug, it's too early in the process for a compassionate use exception for a different form of cancer than the one BioMarin is targeting. There's still a big risk that the drug could harm Sloan, speeding her death, as much as it helps her, he says.
Sloan's doctor agrees the system needs changing.
"My own belief is that the drug companies, the manufacturers, the insurance companies, the providers like my hospital M.D. Anderson, the individual medical professionals, the investigators, the patients -- everybody wants the same things. Safe, effect, novel therapies for as many people as it's appropriate for," says Levenback.
"Even BioMarin, I believe. I don't think this is like 'Let the masses eat cake.' I think they're on the same mission. But the whole system is giving this dysfunctional result."
Levenback says the window for Sloan is closing. If it's going to be effective, she will need the new drug soon. Knowledge of that fact has put Sloan on a mission, and she says she's not giving up.