Pathways for desperate patients to get unapproved drugs have been around for decades, and have sometimes prompted fierce battles over how quickly, and for whom, the drugs should be made available. The process was formalized in the 1980s, when dying AIDS patients pushed for early access to treatments.
In 2021, patients with amyotrophic lateral sclerosis, or A.L.S., pressured Biogen, the company developing a drug for a rare genetic form of the neurodegenerative disease, to offer it through expanded access. The patient at the center of the campaign, Lisa Stockman Mauriello, who was shut out of a clinical trial because of when she was diagnosed, ultimately received the drug via expanded access just days before she died and nearly two years before it won F.D.A. approval.
Today, the case of daraxonrasib has all the ingredients — a deadly disease, a lack of decent treatment options for patients and clear data indicating the drug works — that “bode in favor of an ethical default for the company to set up one of these programs,” said Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania.
The F.D.A.’s sign-off only applies domestically; requests for patients must be placed by a doctor licensed in the United States. At least for now, that restriction will prohibit most desperate patients in other countries from getting the drug.
Cynthia Ataefe, 66, of Buenos Aires, has metastatic pancreatic cancer and wants to try the drug. But she is too sick to travel for a clinical trial, even if she could get into one, according to her daughter, Cecilia Gerson, who lives in New York.
Ms. Gerson said earlier this week that the family is pinning its hopes on expanded access. “I still hope that somehow we will get the drug in time,” she said.