Several of the families I write about say that it takes about $3-5 million to fund a phase 1 clinical trial for a handful of patients to test a gene transfer protocol. A phase 1 trial assesses safety and possibly efficacy (any measurable improvement or slowing of the disease course) in a few patients. The small patient pool may reflect the rarity of the disorder and/or the high cost – a viral vector alone can run $1 million.
So I thought I’d put the cost to launch a phase 1 clinical trial for gene therapy into perspective for Rare Disease Day 2016, February 29. Here are 14 random things that cost ~$5 million:
• A 30-second Super Bowl commercial
• 1/5 of Leonardo DiCaprio’s fee per film
• 1/3 of Jennifer Lawrence’s fee per film
• 1/1000th of a presidential political campaign or of Donald Trump’s net worth
• 5 years of Chris Christie’s travel budget
• Annual salary for a judge on The Voice
About the Author
Ricki Lewis is a science writer with a PhD in genetics. The author of several textbooks and thousands of articles in scientific, medical, and consumer publications, Ricki’s first narrative nonfiction book, “The Forever Fix: Gene Therapy and the Boy Who Saved It,” was published by St. Martin’s Press in March 2012. In addition to writing, Ricki provides genetic counseling for parents-to-be at CareNet Medical Group in Schenectady, NY and teaches “Genethics” an online course for master’s degree students at the Alden March Bioethics Institute of Albany Medical Center.