The Institute for Clinical and Economic Review (ICER) has earned a reputation as the “nerds” of the drug industry, known for their critical approach towards drugmakers when it comes to pricing their latest treatments. However, last year, ICER shocked many with a decision that marked a significant milestone in the pharmaceutical industry. They determined that a new treatment was worth up to $3.9 million, making it the most expensive medicine in history and equivalent to a 45-year supply of Humira, a commonly used autoimmune drug.

The decision by ICER demonstrated the extraordinary potential of gene therapies to provide genuine cures. 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.

ICER’s decision marks an important turning point in the way we think about drug pricing and what constitutes value in healthcare. It shows that while cost is always an important consideration, sometimes it’s not enough to determine whether a treatment is worthwhile or not. With Lenmeldy’s success story, we can see how gene therapies have immense potential to transform people’s lives and pave the way for even more groundbreaking medical discoveries in the future.