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

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

The Institute for Clinical and Economic Review, also known as ICER, has a reputation for being the “nerds” of the drug industry. They are seen as bespectacled killjoys who only come out a few times a year to criticize drugmakers for pricing their latest cancer or MS treatments at exorbitant prices. However, last year, ICER shocked many when they determined 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.

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