In a time when evidence-based prescribing and clinical guidelines are hot topics in medicine, trying to access a not-yet-approved drug to use in a patient can feel like navigating uncharted waters. Many physicians are unaware that the US Food and Drug Administration (FDA) allows the use of unapproved drugs outside of clinical trials and — even if they did know it is possible — have no idea how to access such investigational drugs for their patients.
This knowledge is largely sequestered into certain clinical specialties, such as oncology or rare disease, and it is not taught in medical school or during residency: instead, is largely self-taught. Thus, while some physicians have become very accustomed to requesting pre-approval access to drugs, the majority lack this knowledge. In this essay, I use a fictional case to trace the process for requesting access to an unapproved drug. I hope to explode several myths about the process, such as the notion that the FDA is the primary actor in granting access to unapproved drugs and the belief that physicians must spend 100 hours or more completing paperwork for pre-approval access.
Imagine you are a physician, and you have a pregnant patient who has tested positive for Zika. While she is only mildly ill, she’s terrified that her unborn child may be impacted by the virus, which has been provisionally linked with microcephaly and other abnormalities. She’s so concerned that she is contemplating an abortion, even though she and her husband have been trying to have a child and were overjoyed to learn she was pregnant.