New Gene Therapy Treatment: A Breakthrough in Medical Science and Drug Pricing

The Institute for Clinical and Economic Review, also known as ICER, is renowned for its expertise in the drug industry. They are often perceived as analytical and somewhat detached, but their work has had a significant impact on drug pricing. In recent years, ICER has become increasingly recognized for its ability to identify the potential of new treatments and drugs.

In 2018, 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. This decision marks a turning point in how we approach medical treatments and could pave the way for even more groundbreaking discoveries in the future.