The staff of the Institute for Clinical and Economic Review, or ICER, are known as the nerds of the drug industry: bespectacled killjoys who emerge a few times a year to scold drugmakers for pricing their latest cancer or MS advance far beyond reason.
But last year, they sat down and concluded a forthcoming treatment was worth up to $3.9 million — more than any medicine in history, more than a 45-year supply of Humira, the autoimmune drug often held up as an emblem of America’s runaway drug spending.
advertisement
It was a testament to the power of a new class of gene therapies to deliver something pharma so rarely does: Genuine cures. The treatment, approved last week as Lenmeldy, may allow some babies born with an ultra-rare neurodegenerative disease called metachromatic leukodystrophy, or MLD, to grow up and live essentially normal lives.
Get unlimited access to award-winning journalism and exclusive events.
Subscribe Log In
Previous article：
The woman behind the Dana Farber
Next article： Jerome Adams on the need for new antivirals for Covid
Next article： Jerome Adams on the need for new antivirals for Covid
