A Million Dollar Cure That Became a Commodity Instead of A Lifeline

At 20 years old, Zoe Davis, a college student, was rushed to the hospital for severe pain in her abdomen and legs. At a young age, she was diagnosed with sickle cell disease (SCD) and suffered from severe pain. SCD is notorious for hospitalizing patients with chronic pain that often leaves them incapable of completing basic tasks. Zoe is one of the approximately 100,000 Americans living with the inherited blood disorder that causes red blood cells to be malformed.

Historically, patients have been limited to the same drugs that mitigate pain but fail to address SCD’s root cause. Approximately two years ago, that changed when the FDA approved two gene therapy treatments for SCD, Casgevy and Lyfgenia.

Both treatments address SCD by modifying the hemoglobin genes in stem cells to create healthier blood cells that are less likely to sickle. After the clinical trials were completed, 96.7% of patients reported no major crises in the following year.

When doctors first approached Zoe with the new treatment option, she was not entirely on board. What was a scientific parachute for the disease was immediately disregarded due to its financial burdens. The costs: 

Casgevy - $2.2 million.

Lyfgenia - $3.1 million. 

While this would not have been the cost incurred by Zoe herself after insurance and payment plans, the financial burden was still enough to create a deterrent in her mind. This is not an isolated incident. The science says that gene therapy treatments are effective, yet clinical application is scarce, and regular Americans face far too many obstacles to obtain them.

Traditional drugs are repeatedly manufactured and are able to become profitable through their mass volume and market infiltration. Gene therapies, on the other hand, are created to be a one-time curative treatment. This is the reasoning used by manufacturers for their price points. They argue that they are conducting these pricing models based on perceived value and save the individual from additional medical costs. 

The issue with this is that the pricing model does not consider the fact that much of the research behind CRISPR-based therapies and stem cell engineering is all publicly funded or supported by other academic institutions. Hence, when you truly consider where the funding originates from, it is actually the citizens’ taxes that pay for the research grants. The private manufacturers are able to sit and rake in the profits. This is not just the case for SCD drugs:

Hemgenix (Hemophilia B): $3.5 million

Zolgensma (Spinal Muscular Atrophy): $2.1 million

Luxturna (Retinal Dystrophy): $850K

While the diseases and the actual price points are different, it does not change the fact that the treatments are sold based on what the companies believe the ultrawealthy will pay.

In some cases, people will argue that the high prices of the cure are justified as the patient is not actually paying that total price. However, insurance companies are not lining up to cover these costs. In fact, for patients struggling with SCD, 60% rely on Medicaid. While Medicaid offers a drug rebate program and the Gene Therapy (CGT) Access Model to mitigate drug costs, we can estimate hundreds of thousands of dollars in out-of-pocket costs due to extraneous expenditures.

The chemotherapy drugs used within the SCD treatment regimen are known to cause infertility among other side effects, which require an additional $20,000+ a year to manage. Prior to gene therapy, patients were already struggling to find appropriate specialists and affordable care. With those added costs, gene therapy is simply unattainable for most.

Even for patients who decide they are interested in receiving treatment, it is not easy to obtain. Of those who are typically hospitalized, 93.4% are African American and 4.8% Hispanic. Patients from these demographics tend to lack access to specialized treatment centers and Medicaid-covered services. This points to the reality of American healthcare. The providers will only present themselves in locations that are inherently profitable. 

The larger patterns of society showcase that curative medicine is structurally incompatible with a health system that is focused on profitability. It also showcases the fundamental contradictions of medical innovation. The public funds the science. The private firms are able to patent it. The patients then end up paying top dollar based on the market.

How Can We Tackle This?

While it may feel as though this concept of gene therapy is a failure, that’s far from the case. A cure exists, and when people receive it, it works. If we create the infrastructure to deliver it widely, it has the potential to change tens of thousands of lives.

Pilot programs of Medicaid negotiate care prices and help reimburse hospitals to minimize patients' costs and increase coverage; however, this is not enough to reach equity. The current CMS Cell and Gene Therapy (CGT) Access Model utilizes outcomes-based payment structures that allow states to pay based on whether the patients are genuinely improving. This program provides some relief; however, it fails to cover other expenses such as traveling or loss of wages. Medicaid’s pilot program must be expanded into a permanent initiative that covers not only the total costs of treatment but also any miscellaneous expenses, which would lift pressure off patients.

New federal incentives need to prioritize hospital accreditation in high-incidence regions, rather than directing resources towards high-income areas. These resources need to come in the form of government subsidies. This would begin to create stable infrastructure and bring care closer to the communities that need it.

Increasing gene therapy access would reduce suffering from a disease that diminishes and takes the lives of so many unnecessarily. For Zoe, it might have meant no more visits to the hospital.

Reviewed by: Sehar Mahesh

Designed by: Sebastian Mardales

References:

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