Mayo Clinic has a really good writeup of the trade offs associated with compassionate use: https://www.mayoclinic.org/healthy-lifestyle/consumer-health/expert-a

Mayo Clinic has a really good writeup of the trade offs associated with compassionate use: https://www.mayoclinic.org/healthy-lifestyle/consumer-health/expert-answers/compassionate-use/faq-20058036 https://www.mayoclinic.org/healthy-lifestyle/consumer-health... Essentially, if a patient has no other options and might die, the FDA allows the use of unproven drugs.