14 Things that Cost the Same as a Gene Therapy Clinical Trial

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
• Cheapest apartment on East End Avenue, Manhattan
• 2 nights of earnings from a Bruce Springsteen or Coldplay tour
• A federally-funded study, “Help a Hipster,” to empower the nicotine-addicted to quit via parties at bars and nightclubs
• 1/12 of a Republican or Democratic national convention in 2016
• Kendall Jenner’s annual earnings as a fashion model
• Average yearly salary of NBA players
• 20 lectures by Hillary Rodham Clinton or Donald Trump
• A Labmorghini Veneno built to honor the automaker’s 50th birthday

Apples and oranges, I know, but it all makes me kind of sick.

I asked Kristin Smedley, President of the Curing Retinal Blindness Foundation and subject of several posts at DNA Science because she is so inspiring, and because she is better at math than I am, to estimate what it would take for her organization’s fundraising efforts to hit the $5 million mark:

• COINS FOR THE CURE – my daughter’s soccer team raised $1079 in their coin collection for us in 2015. We’d need 4,634 teams to collect that many coins to hit $5 million.
• LEMONADE STANDS – lots of kiddos host lemonade stands for us. The average stand makes about $57. We would need 87,719 lemonade stands to hit $5 million.
• BIKE THE BASIN – $20 per bike rider… we’d need 250,000 bike riders!
• CASUAL/DRESS DOWN DAY – school staff and businesses host dress down or casual days for us where employees pay $5 to wear jeans for the day. We’d need a million people in jeans to raise $5 million.
• CARS FOR THE CURE (vehicle donation program) – here in the Philadelphia area we work with Aspite Auto Auction to coordinate people donating their used car/boat to Aspite, who then auctions it and gives us the proceeds. The average donation per vehicle is $600. We’d need 8,333 vehicles donated to raise $5 million.
• COCKTAILS-FOR-A-CURE – A couple of families in our organization host cocktail events to raise money. Our event costs $55 per person, so we would need 90,909 people to attend to reach $5 million.

Best of all would wed the two parts of this post – if Kristin’s film treatment about raising two boys with inherited blindness is picked up. “I wonder how many donations our “Feel Good Movie of the Year” will generate??????”

For Rare Disease Day I’d like to honor all the family members who raise awareness and funds that, ultimately, will help the entire community. I’ll be back on the official Rare Disease Day, Monday, with a special-request post here. I had too many ideas this week, so for my take on the scientifically embarrassing finale of the X-Files, see Rare Disease Reports.

The truth is not out there!