Ciji Green faced an unimaginable dilemma when she learned that her daughter, Maisie, required a life-saving drug priced at an astounding $2 million. Diagnosed with spinal muscular atrophy (SMA), a condition that causes severe muscle degeneration, Maisie’s survival hinged on receiving the gene therapy known as Zolgensma. Without intervention, the prognosis indicated that she would not live past her second birthday.
In March 2019, the U.S. Food and Drug Administration approved Zolgensma, a groundbreaking treatment that could potentially alter the course of SMA. Despite its promise, the exorbitant cost presented a significant barrier. Green’s insurance provider refused to cover the drug, leading her to express profound frustration. “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 stated.
The case of Maisie Green highlights a growing concern regarding the financial implications of high-cost gene therapies within the U.S. healthcare system. Economists warn that such therapies, while revolutionary, could overwhelm employer-sponsored insurance frameworks. Jonathan Gruber, an economist who played a key role in developing the Affordable Care Act, remarked, “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 undergoing clinical trials, with some intended for more common diseases. This expansion raises questions about how employers will be able to shoulder these expenses. Gruber emphasized that many companies in America are self-insured, meaning they pay their own medical bills. “About two-thirds of the insured in America are in such arrangements,” he noted. This model could lead to difficult financial decisions for employers: “Do I cover this drug and potentially go bankrupt? Or do I not help my unlucky employee?”
The reasons behind the high costs of these drugs are multifaceted. Doug Ingram, CEO of Sarepta Therapeutics, which markets a gene therapy priced at $3.2 million for Duchenne muscular dystrophy, explained the complexities involved. “The first question is could you even do this and do this safely? The second question that was obvious is if you could, you have to manufacture this stuff,” he said. Ingram highlighted the challenges of scaling production to meet the demand for these innovative treatments.
Mike Poore, CEO of Mosaic Life Care, a nonprofit hospital system in Missouri, experienced the challenges of funding high-cost therapies firsthand. His organization initially opted not to cover genetic therapies due to the significant financial burden it would impose, raising employee premiums by $125 per month. However, when a family within his organization faced a crisis—twins diagnosed with SMA—the situation became personal. Faced with public backlash, Poore sought alternative funding sources, ultimately resulting in Medicaid covering the twins’ treatment. “I got death threats,” he recalled, underscoring the intense pressure that accompanies such financial decisions.
As the healthcare landscape evolves, both employers and pharmaceutical companies find themselves navigating complex moral and financial dilemmas. Gruber posited that neither side is at fault but rather that society must collectively address the implications of these high-cost treatments. “We just have to recognize as a society that something’s changed,” he said.
Ingram suggested that prices may eventually decrease as manufacturing processes improve and regulatory frameworks are streamlined. He pointed out that the average timeframe to develop a therapy spans over 10 years and costs nearly $3 billion. He advocates for a reassessment of regulatory requirements, arguing that many could be modernized to expedite the development of affordable therapies.
Ciji Green’s fight for Maisie illustrates the lengths to which parents will go to secure treatment for their children. After pursuing philanthropic avenues and demanding a meeting with the insurance company that denied coverage, Green succeeded in overturning the initial decision. Maisie received Zolgensma, and the impact was transformative. “It changed our life. It was what she needed,” Green reflected.
Today, at the age of 6, Maisie is thriving, demonstrating the potential for gene therapy to reshape the lives of children with serious conditions. Although she experiences challenges, including not walking, she excels academically, making straight A’s in school. Green describes her daughter as “my very own miracle,” a testament to the life-changing power of medical advancements and the ongoing struggle to make such treatments accessible to all families in need.
