When Ciji Green learned that her daughter, Maisie, needed a drug costing $2 million to treat a life-threatening condition, she faced a heartbreaking dilemma. Maisie was diagnosed with spinal muscular atrophy (SMA), a severe genetic disorder that leads to muscle deterioration and often results in death before the age of two without treatment. The only available therapy, Zolgensma, received approval from the Food and Drug Administration in 2019 but comes with a staggering price tag.
Initially, Green’s insurance provider refused to cover the cost of this critical medication, prompting her to voice her outrage. “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. Zolgensma is part of a new class of gene therapies designed to address rare diseases like SMA, where treatment options are limited.
The situation highlights a broader issue within the U.S. healthcare system, which is grappling with the implications of high-cost genetic therapies. Economist Jonathan Gruber, a key figure in the development of the Affordable Care Act, foresees a “coming tsunami” of these therapies that could overwhelm employer-sponsored insurance plans. More than 300 high-cost genetic therapies are currently in clinical trials, many targeting diseases that affect larger populations.
Gruber explains that many American companies are self-insured, meaning they directly pay for their employees’ medical bills. “About two-thirds of the insured in America are in such arrangements,” he said. This model leads to difficult financial choices for employers, who must weigh the potential bankruptcy that could result from covering these expensive treatments against their responsibility to support their employees.
The high costs of gene therapies are particularly evident in the case of Doug Ingram, CEO of Sarepta Therapeutics, which markets therapies priced in the millions. His company’s drug, Elevidys, costs $3.2 million for a single-dose treatment aimed at Duchenne muscular dystrophy, a condition that also results in muscle wasting. Ingram pointed out the urgent need for treatments in this area, stating that parents have been advised to cherish their time with children diagnosed with Duchenne because the disease is ultimately fatal.
Ingram described the challenges of developing such therapies, emphasizing the need for advanced manufacturing capabilities and substantial financial backing. “We have to raise billions of dollars to do this,” he noted, highlighting the complexities of bringing a new therapy to market.
The financial pressures extend beyond the drug companies to healthcare providers. Mike Poore, CEO of Mosaic Life Care, a nonprofit hospital system in Missouri, faced backlash after his organization decided not to cover genetic therapies to avoid raising employee premiums by $125 per month. When a staff member’s twins were diagnosed with SMA, the public outcry following their denied coverage forced Poore to seek alternative solutions, ultimately resulting in Medicaid covering the children’s treatment.
“This situation is a bellwether of what is to come,” Poore remarked, emphasizing that the frequency of high-cost therapies is only expected to increase.
Addressing the issue of exorbitant drug prices is crucial, especially as many stakeholders share the blame. Gruber asserts that there are no “bad guys” in this scenario; rather, society must recognize the changes brought about by these new, expensive treatments. “We have to act jointly to absorb those costs,” he stated, suggesting that government intervention and negotiated pricing may be necessary.
Ingram also believes that prices could decrease as manufacturing processes improve and regulations become more efficient. Currently, developing a new therapy takes over 10 years and costs nearly $3 billion on average, with early-stage success rates being very low. He called for a reevaluation of some regulations to facilitate more affordable and successful therapy development.
For parents like Ciji Green, navigating the high costs of gene therapies often requires resourcefulness. After pursuing philanthropic avenues and demanding a meeting with her insurer, Green’s persistence paid off when the appeals board agreed to cover the cost for Maisie. In 2019, Maisie received Zolgensma, a treatment that proved transformative.
“It changed our life. It was what she needed,” Green said, reflecting on the journey. Initially given a grim prognosis, Maisie is now thriving at age six, although she still faces challenges, such as not being able to walk. Green describes her daughter as a “miracle” and highlights the importance of continued advocacy for access to these life-saving treatments.
While the landscape of genetic therapies evolves, the financial implications for families, employers, and the healthcare system at large will require careful navigation and cooperation among all parties involved.
