Is the world’s most expensive drug worth the price tag?

The staff at the Institute for Clinical and Economic Review, also known as ICER, are often seen as the nerds of the drug industry. They are seen as bespectacled individuals who appear a few times a year to criticize drugmakers for pricing their latest cancer or MS treatments at exorbitant prices. However, last year, the ICER staff surprised everyone by concluding that a forthcoming treatment was worth up to $3.9 million. This valuation made it the most expensive medicine in history, surpassing the cost of 45 years’ worth of Humira, a commonly used autoimmune drug associated with high drug prices in America.

This valuation represented the tremendous potential of a new class of gene therapies to achieve something that pharmaceutical companies rarely do: provide genuine cures. The treatment, now approved as Lenmeldy, has the capability to allow babies born with the rare neurodegenerative disease known as metachromatic leukodystrophy (MLD) to live essentially normal lives. This breakthrough demonstrates the power of gene therapies to deliver life-changing treatments for rare and debilitating conditions, offering hope to patients and their families facing these challenges.