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Ethics Consult: OK to Give Babies Experimental Drug Not in Trial?

— You make the call

Last Updated January 17, 2020
MedpageToday

Welcome to Ethics Consult -- an opportunity to discuss, debate (respectfully), and learn together. We present an ethical dilemma in patient care; you vote on your decision in the case. Next week, we'll reveal how you all made the call. And stay tuned -- bioethical experts Alyssa Burgart, MD, MA, and Alison Bateman-House, MPH, PhD, will weigh in next week with an ethical framework to help you learn and prepare.

will also be hosting a Twitter Chat with Alyssa Burgart () and Alison Bateman-House () on this case on Wednesday, January 15 at 4:00 p.m. ET. To join, follow on Twitter and join the discussion live on our feed by clicking . Use the hashtag on all of your tweets; we encourage you to comment, answer questions, ask questions, and react during the chat.

A woman is pregnant with twin boys. A previous son was diagnosed with Menkes disease, an X-linked copper transport disorder, at around 3 months of age and died before his second birthday. The disease causes slowed physical growth, intellectual disability, and brittle, colorless hair. Unfortunately, prenatal genetic testing reveals both twins also have Menkes. There is no FDA approved treatment for the condition. The mother's deceased son was enrolled in a clinical trial for an investigational drug using subcutaneous copper histidine injections. Unfortunately, treatment was not effective, though it's unclear if this was because the drug was started too late (one study showed it was more effective when started before symptoms appeared) or that his mutation was too severe. The mother works with her physician to determine if there are any current clinical trials that the boys can be enrolled in at birth before they become symptomatic.

However, all trials with the drug are now closed. Instead, after the boys are born, the medical team seeks to obtain it outside of a clinical trial via non-trial preapproval access (historically known as compassionate use). The physicians attempt to convince the company to provide the therapy and the FDA to allow its use in these two patients. Then they will approach the hospital's IRB for final approval to use the drug outside a trial.

Case based on:

See the results and what an expert has to say.

, is assistant professor of anesthesia at the Stanford University School of Medicine and core faculty in the . She is a bioethicist at Stanford Healthcare and Lucile Packard Children's Hospital.

is an assistant professor in the Division of Medical Ethics at NYU Langone Health's Grossman School of Medicine. She is co-chair of the Working Group on Compassionate Use and Preapproval Access (CUPA).

And check out some of our past Ethics Consult cases: Euthanize Resistant Dementia Patient?, 'Freeze' Little Girl in 6-Year-Old Body?, and Deaf Couple Only Wants Deaf Baby.