• Home
  • Latest
  • Fortune 500
  • Finance
  • Tech
  • Leadership
  • Lifestyle
  • Rankings
  • Multimedia

Trendingnow

1

Corporate America has been draining the world's water. Matt Damon's new campaign calls on Gap, Starbucks, and Amazon to help give it back

2

Analysts expected oil to surge above $200 but China has quietly kept prices half of that—and can’t for much longer

3

When SpaceX starts trading, some 'shareholders' will discover they own nothing at all

1

Corporate America has been draining the world's water. Matt Damon's new campaign calls on Gap, Starbucks, and Amazon to help give it back

2

Analysts expected oil to surge above $200 but China has quietly kept prices half of that—and can’t for much longer

3

When SpaceX starts trading, some 'shareholders' will discover they own nothing at all
Healthgene therapy

Gene Therapy Is Revolutionary Science That Can Cure Blindness. Is It Worth $1 Million a Pop?

By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
November 16, 2017, 4:17 PM ET

2017 has been a marquee year for gene therapy and medical technologies which harness—and manipulate—the body’s very biological building blocks to fight disease. But with great innovation comes profound questions about cost. And the expected sky-high price tags for new treatments in the cell-based therapeutics field will be an important factor for patients with deadly rare diseases.

A new report by the U.S. Institute for Clinical and Economic Review (ICER) suggests that the potential $1 million list price for Spark Therapeutics’ gene therapy Luxturna—which recently received a unanimous FDA advisory committee recommendation to treat a rare, inherited form of blindness, considerably bolstering its chances for regulatory clearance—isn’t validated by its expected benefit. (To be clear, no price has actually been set for Spark’s treatment since it hasn’t actually been approved yet.)

“[A]t a placeholder price of $1,000,000, the high cost makes this unlikely to be a cost-effective intervention at commonly used cost-effectiveness thresholds,” said the group.

ICER is a controversial entity among drug makers who argue it gives short shrift to life-changing medical innovation; Spark’s therapy, for instance, would only require a single dose for a potentially long-lasting benefit that gives sight to the blind. From an outside perspective, the notion of a $1 million drug might give many pause; for a patient who desperately wants to reverse his or her condition, the calculus could be more nuanced.

This debate isn’t limited to Spark, whose drug could be approved by mid-January or even sooner. Swiss pharma giant Novartis and U.S. biotech Gilead Sciences unit Kite Pharma both won landmark FDA approvals this year for cancer drugs that re-engineer the body’s immune system cells to kill cancerous cells, for instance. Novartis’ drug, Kymriah, comes with a $475,000 list price. However, that cost only kicks in if the treatment shows signs of working within the first month. Creative reimbursement arrangements like that may become the wave of the future in an age when medical technology and prices alike stun the mind.

This essay appears in today’s edition of the Fortune Brainstorm Health Daily. Get it delivered straight to your inbox.

About the Author
By Sy Mukherjee
See full bioRight Arrow Button Icon

Latest in Health

Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025

Most Popular

Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Fortune Secondary Logo
Rankings
  • 100 Best Companies
  • Fortune 500
  • Global 500
  • Fortune 500 Europe
  • Most Powerful Women
  • World's Most Admired Companies
  • See All Rankings
  • Lists Calendar
Sections
  • Finance
  • Fortune Crypto
  • Features
  • Leadership
  • Health
  • Commentary
  • Success
  • Retail
  • Mpw
  • Tech
  • Lifestyle
  • CEO Initiative
  • Asia
  • Politics
  • Conferences
  • Europe
  • Newsletters
  • Personal Finance
  • Environment
  • Magazine
  • Education
Customer Support
  • Frequently Asked Questions
  • Customer Service Portal
  • Privacy Policy
  • Terms Of Use
  • Single Issues For Purchase
  • International Print
Commercial Services
  • Advertising
  • Fortune Brand Studio
  • Fortune Analytics
  • Fortune Conferences
  • Business Development
  • Group Subscriptions
About Us
  • About Us
  • Press Center
  • Work At Fortune
  • Terms And Conditions
  • Site Map
  • About Us
  • Press Center
  • Work At Fortune
  • Terms And Conditions
  • Site Map
  • Facebook icon
  • Twitter icon
  • LinkedIn icon
  • Instagram icon
  • Pinterest icon

Latest in Health

Redwood Outdoors Duo Sauna Review (2026): Our Honest Experience
HealthDietary Supplements
Redwood Outdoors Duo Sauna Review (2026): Our Honest Experience
By Emily PharesJune 12, 2026
2 hours ago
A person holds an iPhone as someone next to them points at the screen.
EconomyApple iPhone
You can blame America’s plummeting fertility rate on the iPhone, study finds: ‘People are all depressed and alone and doomscrolling’
By Sasha RogelbergJune 12, 2026
9 hours ago
fda
HealthTobacco
Why is the FDA approving kid-friendly, fruit-flavored e-cigarettes?
By Matthew Perrone and The Associated PressJune 12, 2026
10 hours ago
Testosil Review (2026): Testosterone Supplement Reviewed
HealthDietary Supplements
Testosil Review (2026): Testosterone Supplement Reviewed
By Christina SnyderJune 11, 2026
20 hours ago
BrickHouse Nutrition Field of Greens Review (2026)
HealthDietary Supplements
BrickHouse Nutrition Field of Greens Review (2026)
By Emily PharesJune 11, 2026
20 hours ago
worm
HealthFood and drink
The pest that could devastate the American cattle industry was in Texas, but now it’s in New Mexico, too
By Jeffrey Collins and The Associated PressJune 9, 2026
3 days ago

Most Popular

Corporate America has been draining the world's water. Matt Damon's new campaign calls on Gap, Starbucks, and Amazon to help give it back
Environment
Corporate America has been draining the world's water. Matt Damon's new campaign calls on Gap, Starbucks, and Amazon to help give it back
By Catherina GioinoJune 9, 2026
3 days ago
Analysts expected oil to surge above $200 but China has quietly kept prices half of that—and can’t for much longer
Energy
Analysts expected oil to surge above $200 but China has quietly kept prices half of that—and can’t for much longer
By Sasha RogelbergJune 10, 2026
2 days ago
When SpaceX starts trading, some 'shareholders' will discover they own nothing at all
Investing
When SpaceX starts trading, some 'shareholders' will discover they own nothing at all
By Jim EdwardsJune 12, 2026
6 hours ago
Current price of oil as of June 11, 2026
Personal Finance
Current price of oil as of June 11, 2026
By Joseph HostetlerJune 11, 2026
1 day ago
Marc Lore’s robots make 500 burrito bowls an hour. A human can make 45
Innovation
Marc Lore’s robots make 500 burrito bowls an hour. A human can make 45
By Amanda GerutJune 9, 2026
3 days ago
Costco CEO Ron Vachris rose from forklift driver to the C-suite without a college degree: ‘Don’t chase a title’ is the career advice that got him there
Success
Costco CEO Ron Vachris rose from forklift driver to the C-suite without a college degree: ‘Don’t chase a title’ is the career advice that got him there
By Preston ForeJune 8, 2026
4 days ago

© 2026 Fortune Media IP Limited. All Rights Reserved. Use of this site constitutes acceptance of our Terms of Use and Privacy Policy | CA Notice at Collection and Privacy Notice | Do Not Sell/Share My Personal Information
FORTUNE is a trademark of Fortune Media IP Limited, registered in the U.S. and other countries. FORTUNE may receive compensation for some links to products and services on this website. Offers may be subject to change without notice.