Priced at a staggering $2.125 million per patient, Zolgensma is now the world’s most expensive medication. Nevertheless, this one-dose gene therapy is a potential life-saver for children with spinal muscular atrophy (SMA), the leading genetic cause of death in infants. These are ultra-orphan disease patients, which means that this drug will only go to a handful of children who desperately need it to survive.
Ultra-orphan is the term given to drugs that are used to treat extremely rare diseases that are chronically debilitating or life-threatening. They affect very small numbers of patients—fewer than 20 patients per million and usually as few as one per million or less.
The impact an ultra-rare disease can have on patients, their families, and society, however, is profound. The diseases are often severe, chronic, and progressive, with high mortality rates. Ultra-orphan drugs are often expensive on a per patient basis because patient numbers—and sales—are so low. That said, if Novartis, the manufacturer of Zolgensma, pushes this drug for expansion to the entire U.S. population, the price tag would become problematic.
At the moment, the drug is approved only for a very small population of patients, so its cost will have no economic impact on the U.S. healthcare system. Furthermore, while payers will get sticker shock, the overall cost is negated by the humanitarian and economic benefits. This is often the case with patients/plan members with orphan diseases, who typically generate high claims costs related to frequent episodes of acute care—office visits, regular visits to the emergency department, hospital admissions, at-home care, ventilators, and so on.
This means that even a highly expensive new therapy could cost less in the long run especially—if it helps to reduce episodes of acute care. In fact, it may be in the best interest of the payer to encourage physicians to prescribe these new medications, despite the price tag.