Novartis believes its new gene therapy is worth more than $4 million for a one-time dose, and the company has some data to back that up. But, with a global spotlight on the escalating cost of medicine, is it politically viable to set a new record for the world’s most expensive drug?
The treatment, called AVXS-101, has demonstrated dramatic effects in spinal muscular atrophy, a rare genetic disorder whose most severe form is fatal for almost all patients before age 2. In a 15-patient trial, infants with SMA who got AVXS-101 had a 100 percent survival rate after 24 months, data that convinced Novartis (NVS) to pay $8.7 billion for the gene therapy’s inventor.
Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!
GET STARTEDWhat is it?
STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.
What's included?
- Daily reporting and analysis
- The most comprehensive industry coverage from a powerhouse team of reporters
- Subscriber-only newsletters
- Daily newsletters to brief you on the most important industry news of the day
- Online intelligence briefings
- Frequent opportunities to engage with veteran beat reporters and industry experts
- Exclusive industry events
- Premium access to subscriber-only networking events around the country
- The best reporters in the industry
- The most trusted and well-connected newsroom in the health care industry
- And much more
- Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
About the Author
Something fundamentally wrong with the argument that high price for a gene therapy is justified because it is a “one-time treatment”. If that argument would have been applied 50 years ago when antibiotics were emerging, that also were “one-time treatment” for the patient with an (usually lethal) infection, we would not have antibiotics today. Most probably within few years from now most non communicable diseases will have a “one-time” gene based therapy, therefore, the pricing/marketing strategy should not be based on the “one-time” dose or regimen but rather on the size (number) of the patients that would benefit from the gene based technology, that probably would be similar independently of the disease.