Breakthrough in Medical Science: ICER Approves Gene Therapy to Cure Rare Disease at Record-Setting Price

The Institute for Clinical and Economic Review, commonly referred to as ICER, has long been viewed as the “nerds” of the drug industry. They are typically seen as bespectacled killjoys who emerge from their seclusion only a few times a year to criticize pharmaceutical companies for overpricing their latest cancer or MS treatments.

However, last year, ICER shocked many with its decision that a new treatment was worth up to $3.9 million – more than any other medicine in history and equivalent to a 45-year supply of Humira, a commonly used autoimmune drug. This decision demonstrated the extraordinary potential of a new class of gene therapies to provide genuine cures, something that the pharmaceutical industry rarely accomplishes.

The treatment, now approved as Lenmeldy, offers hope to babies born with metachromatic leukodystrophy (MLD), an ultra-rare neurodegenerative disease. Lenmeldy may enable these infants to grow up and lead essentially normal lives, representing a significant breakthrough in medical science.