Ciji Green faced an unimaginable situation when her daughter, Maisie, was diagnosed with spinal muscular atrophy (SMA), a condition that leads to severe muscle wasting and often results in death before the age of two without intervention. In March 2019, the U.S. Food and Drug Administration approved a groundbreaking gene therapy called Zolgensma, designed to treat SMA. However, the cost of this single-dose treatment is a staggering $2 million, and Green’s insurance provider refused to cover it.
The emotional toll of this denial was profound. Green expressed her feelings of anger and desperation, stating, “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.” Zolgensma is part of a new class of gene therapies that aim to address rare diseases, where treatment options are minimal. As these high-cost drugs expand into more common diseases, they threaten to significantly impact the U.S. healthcare system.
Economist Jonathan Gruber, who contributed to the development of the Affordable Care Act, warned of a potential crisis: “I liken it to a coming tsunami, which is basically gonna overwhelm the employer-sponsored insurance system.” Currently, over 300 high-cost genetic therapies are in clinical trials, with some targeting diseases that affect millions. Many employers, particularly those who are self-insured, face difficult financial choices. Gruber noted that about two-thirds of the insured population in the U.S. operates under self-insured arrangements, leaving companies to decide whether to cover these expensive treatments, potentially risking bankruptcy in the process.
The high price of these therapies raises questions about their affordability. Doug Ingram, CEO of Sarepta Therapeutics, acknowledged the challenges associated with developing gene therapies, stating that significant manufacturing capacity and funding are required. For instance, Sarepta’s Elevidys therapy, which costs $3.2 million, is crucial for children suffering from Duchenne muscular dystrophy, another life-threatening condition. Ingram explained that the complexity of creating these therapies necessitates raising billions of dollars, which can be a daunting task for any company.
The financial burden of such treatments has led some institutions to limit coverage. Mike Poore, CEO of Mosaic Life Care, shared his experience with the impact of high-cost therapies on his organization. After initially deciding not to cover genetic treatments to avoid raising employee premiums, he faced a personal crisis when an employee’s twins were diagnosed with SMA. The estimated cost for their gene therapy stood at $4.2 million. Public outcry ensued when the family was denied coverage, leading to threats against Poore and his family, underscoring the emotional and ethical complexities surrounding healthcare decisions.
As the healthcare landscape evolves with the introduction of expensive gene therapies, solutions are urgently needed. Gruber emphasized that neither employers nor drug manufacturers are at fault; rather, society must acknowledge the shift in treatment paradigms and find collaborative ways to manage these costs. He advocates for government support and negotiated pricing as essential steps forward.
Ingram supports the idea that therapy prices may decrease as manufacturing processes improve and regulations are streamlined. “Today, on average, it takes more than 10 years to develop a therapy, costing nearly $3 billion,” he pointed out. He believes that reducing these costs requires a reevaluation of regulations that may be outdated in light of modern scientific advancements.
For Ciji Green, the battle to secure treatment for Maisie involved relentless advocacy. After initially being denied coverage, she sought philanthropic support and demanded a meeting with the insurance company. “I wanted them to look her in the eye and say we’re the reason you are going to die,” Green recounted. Ultimately, the insurance appeals board approved coverage, allowing Maisie to receive Zolgensma in 2019. This single treatment proved transformative for her life.
Reflecting on the outcome, Green stated, “It changed our life. It was what she needed.” Initially told that her daughter would likely not survive past age two, Green now celebrates Maisie’s progress. At the time of a recent feature by 60 Minutes, Maisie, now six years old, excels in school, achieving straight A’s. While the effects of her condition remain, Green considers her daughter a “miracle,” illustrating the profound impact of medical advancements on individual lives.
