What is it?
Ultra high-cost drugs (UHCDs) are potentially expensive, durable, or curative therapies and serve to treat conditions that are often orphan and rare diseases. UHCDs, which are (exclusively at the moment) comprised of gene and cell therapies, promise durable effects – at least 15 years. Unlike other expensive specialty drugs that help patients manage chronic conditions, ultra-high-cost drugs promise long-term amelioration of the condition after a single application. Up to now, these gene and cell therapies have largely targeted orphan and ultra-orphan diseases. But over the next decade a wave of these drugs focused on more common conditions will enter the market with the potential to impact millions of Americans.
UHCDs are typically extremely expensive, ranging from thousands to millions of dollars. The cost of UHCDs is often justified as they typically require very few applications and could displace other longitudinal care. However, while they promise long-term savings by alleviating much of the physical burden of the illness, their high upfront cost can hit purchasers and consumers hard.
To mitigate against the financial risk for novel therapies, purchasers of these products are exploring new financing models with manufacturers. Performance-based agreements and orphan reinsurance benefit managers are gaining traction.
Some of the better-known examples of UHCDs currently on the market are Luxturna and Zolgensma, which treat Leber Congenital Amaurosis (LCA) and Spinal Muscular Atrophy (SMA) respectively, Danyelza to treat neuroblastoma, Solris to treat atypical hemolytic uremic syndrome, and Yescarta CART-cell therapy for non-Hodgkin lymphoma. In addition, it is also likely that gene therapies for Hemophilia and Sickle Cell will enter the market in the next three years. These treatments are indicated for much larger populations than LCA and SMA.