Ciji Green’s heart-wrenching journey began when her daughter, Maisie Green, was diagnosed with spinal muscular atrophy (SMA), a life-threatening condition that leads to progressive muscle degeneration. Without treatment, SMA often proves fatal before the age of two. In 2019, the Food and Drug Administration approved Zolgensma, a single-dose gene therapy that could potentially save Maisie’s life, but the staggering cost of $2 million left Green grappling with despair.
Initially, Green’s insurance provider refused to cover the costly treatment. “I became very angry to know that there was something that could help her. And I knew without a shadow of a doubt I was burying my daughter before she was 2,” Green shared. The urgency of the situation highlights a growing crisis within the U.S. healthcare system, particularly as high-cost therapies expand beyond rare diseases to more prevalent conditions.
With over 300 high-cost genetic therapies currently in clinical trials, the implications for employer-sponsored insurance plans are significant. Economist Jonathan Gruber, who played a role in the development of the Affordable Care Act, described the situation as “a coming tsunami” that may overwhelm the existing insurance framework. Many employers, especially those that are self-insured, face a tough choice: cover these expensive treatments or risk financial instability.
The staggering costs of gene therapies often stem from the complexity of their development. Doug Ingram, CEO of Sarepta Therapeutics, which markets one of the highest-priced treatments, noted that their drug, Elevidys, costs $3.2 million but could be the only option to slow the progression of Duchenne muscular dystrophy, another devastating muscle-wasting disease. Ingram explained the challenges of manufacturing such therapies, emphasizing the need for significant investment and infrastructure that was lacking at the time of their inception.
Despite the high costs, the urgency for effective treatments has compelled healthcare providers to reconsider their options. For example, Mike Poore, CEO of Mosaic Life Care, faced a personal crisis when an employee’s twins were diagnosed with SMA. Initially, the organization opted not to cover genetic therapies due to the financial burden it would impose on employee premiums. However, when the twins’ treatment cost reached $4.2 million, the situation prompted Poore to seek alternative funding channels, ultimately leading to Medicaid covering the treatment costs.
As these therapies continue to break pricing records, Gruber stated that neither employers nor pharmaceutical companies are to blame. He emphasized the need for collective acknowledgment of this new reality and the necessity for societal action to manage costs. He advocated for government intervention and negotiated drug pricing to alleviate the burden on employers.
Ingram echoed these sentiments, suggesting that prices could decrease as manufacturing processes improve and regulatory frameworks are refined. “It costs nearly $3 billion on average to develop a therapy, and the probability of success is nearly zero at the beginning,” he explained. Streamlining regulations could ultimately lead to lower costs and increased accessibility of life-saving treatments.
In a desperate bid to secure treatment for her daughter, Green turned to philanthropy and demanded a meeting with her insurance provider. “I wanted them to look her in the eye and say we’re the reason you are going to die,” she recalled. Ultimately, the insurance appeals board reversed its decision, allowing Maisie to receive Zolgensma. The impact of the therapy has been transformative; although Maisie faces challenges and does not walk, she is thriving academically.
Reflecting on the ordeal, Green described her daughter as her “very own miracle.” As Maisie Green grows older, her mother’s fight serves as a poignant reminder of the complexities and moral dilemmas surrounding high-cost medical treatments. The landscape of gene therapies is evolving rapidly, and as the need for innovative solutions becomes more pressing, the healthcare system must adapt to ensure that no child is left behind.
