Mother Celebrates Life-Saving Drug, Faces $2 Million Cost Challenge
Ciji Green faced a daunting challenge when her daughter Maisie was diagnosed with spinal muscular atrophy (SMA). Likely to be fatal before age two without treatment, this condition severely impacts muscle strength. In 2019, the FDA approved Zolgensma, a gene therapy designed to treat SMA, but its staggering price tag of $2 million posed significant hurdles for families seeking the treatment.
Insurance Denial and Advocacy
When Green’s insurance company declined to cover Zolgensma, she was filled with anger and despair. “I knew without a shadow of a doubt I was burying my daughter before she was 2,” she expressed. This situation is not unique; it highlights a widespread issue regarding high-cost genetic therapies that can save lives but often remain inaccessible.
The Rising Cost of Gene Therapies
The approval of Zolgensma symbolizes a breakthrough in gene therapies. While the treatment showcases high potential for SMA, it raises concerns about affordability as more genetic therapies emerge. Jonathan Gruber, an economist, warns that this influx of expensive treatments could overwhelm employer-sponsored insurance systems. Today, over 300 genetic therapies are undergoing clinical trials, potentially reshaping healthcare costs across the United States.
Self-Insured Challenges
Many American companies operate on self-insured models, paying their own medical bills. Gruber notes that about two-thirds of insured individuals fall into this category. The burden of covering new, costly medications presents a dilemma for employers: either risk financial hardship or deny critically needed treatments to employees.
Testimonies from Healthcare Leaders
Doug Ingram, CEO of Sarepta Therapeutics, offers insight into the complexities of developing and pricing gene therapies. Sarepta’s Elevidys is priced at $3.2 million but could significantly improve conditions like Duchenne muscular dystrophy. Ingram emphasizes the urgency for therapies, as many children face dire prognoses. “The first question is could you even do this and do this safely?” he asked, pointing out the significant financial and logistical challenges involved in gene therapy development.
Real-World Struggles
Mike Poore, CEO of Mosaic Life Care, illustrates the tough decisions hospitals face regarding high-cost treatments. Their decision not to cover genetic therapies was reconsidered when staff experienced personal challenges. After learning of twins diagnosed with SMA and the exorbitant cost for treatment, Poore took steps to seek assistance for their care from various sources, ultimately leading to Medicaid covering the costs.
Confronting the Future of Gene Therapy Costs
Gruber advocates for collective societal action to tackle these costs. He proposes government involvement and price negotiations as potential solutions. Ingram suggests that efficient manufacturing practices and regulatory adjustments could help bring down Prices over time. Significant refinements are necessary to reduce the average $3 billion cost and extensive development timeline for new therapies. This shift could foster a more sustainable healthcare framework.
Maisie’s Journey
Despite numerous barriers, Ciji Green’s persistence paid off. After demanding a meeting with the insurance company, Maisie finally received Zolgensma. The impact was transformative. Green described this moment as life-changing. “It was what she needed,” she said, sharing the joy that followed Maisie’s treatment.
At 6 years old, despite her challenges, Maisie is thriving academically. Ciji Green refers to her as “my very own miracle,” defining a future filled with hope despite the adversity faced. The increasing availability and pricing of gene therapies raise questions about the ethics of healthcare decisions in America and highlight the need for urgent and systemic solutions.
