Undeniably, exorbitant prices can indicate a treatment is not economically viable. For instance, the gene therapy Glybera, previously known as the costliest drug, was only acquired once before it was withdrawn from the market due to its inadequate effectiveness that failed to warrant its $1 million price tag.
Until recently, the title of the most expensive treatment was held by a $3.5 million hemophilia treatment, Hemegenix, another gene therapy. Contrary to expectations, the sales of these high-cost therapies have not surged as anticipated.
Orchard, a company that dealt with gene therapy, had to abandon another DNA solution, Strimvelis, which was a breakthrough cure for a certain immune deficiency. Despite its potential, it had to be discontinued in 2022 due to a small patient population and the availability of alternative treatments. Even a money-back guarantee was not able to salvage Strimvelis.
Afterwards, Orchard was acquired by Japanese company Kyowa Kirin, and is now a subsidiary of it. Despite their success in trials, gene therapies seem to be falling short overall. In the case of Lenmeldy, early diagnosis of the disease is crucial as symptoms may develop too late in children. Currently, many kids are identified only when an older sibling has already been affected by the inherited condition.
In 2016, an article by MIT Technology Review described the profound impacts of the MLD gene therapy, but it also underscored the tragedy for parents who lose one child, enabling others to be saved. Orchard aims to address this predicament by advocating for MLD to be added to the list of diseases screened for at birth, potentially securing their market and saving countless more lives.
A decision regarding the inclusion of this disease in newborn screening could be made after a committee meeting on the issue in May. Among the advocates of this approach is Amy Price who runs her own consultancy, Rarralel, in Denver. Price, a mother to three children affected by MLD, lost one child but managed to save two thanks to the MLD gene therapy that they received in 2011 during its testing phase.
These children, now tweens and teenagers, are leading normal lives. According to Price, this outcome is worth the high cost of the treatment. She argues that the economic implications of not treating the child far outweigh the costs of gene therapies. However, she admits that this is a difficult reality to grasp for those focused solely on the price.
