The most expensive medicine in the world

How do you safeguard your health plan for the introduction of expensive and medically covered, new gene therapies? Recent gene therapy approvals have garnered news headlines because of their revolutionary capabilities and extremely high cost. In September, we posted about Hemgenix, a gene therapy for adults with hemophilia B, as a therapy to watch. When the therapy received FDA approval for eligible persons in November, the price was listed as $3.5 million – making it the most expensive medicine in the world today. Clearly, it is imperative to have a partner watching how drug and therapy innovations might affect your medical and pharmacy plans.

Hemgenix is a gene therapy for adults with hemophilia B (congenital factor IX [FIX] deficiency) who currently use FIX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. Remarkably, 94% of participants in the supporting study who received Hemgenix were able to discontinue use of intravenous FIX infusions. This is an important consideration since FIX infusion therapy can cost of approximately $500,000–$750,000 per year.

These products would very likely be covered under the medical benefit, and self-funded payers should discuss utilization management strategies and other options with their medical carrier. Payers need to carefully consider their capacity to absorb such costs and confirm they have adequate safeguards in place to protect plan solvency. Strategies may vary from stop loss coverage to benefit carve-outs to address these ultra-high cost therapies.