Why the world’s most expensive drug might be worth the price

The Institute for Clinical and Economic Review (ICER) is known for critiquing drug prices, but they recently valued a new treatment at $3.9 million, the highest in history. This treatment, now approved as Lenmeldy, has the potential to cure babies with metachromatic leukodystrophy, allowing them to lead normal lives. This showcases the power of gene therapies to provide actual cures, a rarity in the pharmaceutical industry. The staff at ICER, often seen as the “nerds” of the industry, have recognized the value of this groundbreaking treatment and its potential impact on patients with rare diseases.

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