Breaking the Record: ICER Approves $3.9 Million Drug for Genetic Disorder and Shocks Pharmaceutical Industry

The Institute for Clinical and Economic Review, or ICER, is known as the “geek” of the drug industry. They are often seen as bespectacled killers who only come out a few times a year to criticize drugmakers for charging high prices for their latest cancer or MS treatments. However, last year, ICER shocked many when it determined that the new treatment was worth up to $3.9 million – more than any other drug in history and equivalent to a 45-year supply of Humira, the most commonly used autoimmune drug.

The decision demonstrated the extraordinary potential of a new class of gene therapies to provide real cures, something the pharmaceutical industry rarely achieves. The treatment, now approved as Lenmeldi, offers hope to babies born with metachromatic leukodystrophy (MLD), an ultra-rare neurodegenerative disease. Lenmeldi can allow these babies to grow up to lead essentially normal lives, a significant advance in medical science.

ICER’s decision was based on rigorous analysis of clinical trial data and cost-effectiveness studies. The institute found that Lenmeldi was highly effective in treating MLD and had a favorable risk-benefit profile compared to other available treatments. Additionally, ICER determined that the treatment was affordable and offered good value for money.

The approval of Lenmeldi marks a major milestone in gene therapy and has implications for other diseases that affect similar genetic pathways. It also highlights the important role that ICER plays in ensuring that drugs are priced fairly and effectively treat patients’ needs.