Decoding the high costs and potential impact of emerging gene therapies

Gene therapies treat genetic mutations that cause diseases or hinder the body’s ability to fight them. Rather than addressing the symptoms of the genetic mutation, gene therapies attempt to fix the root cause by modifying the expression of those genes.
 

Types of gene therapy

• Gene addition: Replacing a disease-causing gene with a healthy copy of the gene
• Gene silencing: Deactivating a disease-causing or malfunctioning gene
• Gene editing: Introducing a new or modified gene into the body to help treat a disease
   

The U.S. Food and Drug Administration (FDA) approved the first gene therapy in 2017 and has approved 19 as of June 2024.⁴ They’ve also approved several cell therapies, a closely related treatment of genetic disease. One study estimates there will be between 54 and 74 approved cell and gene therapies by 2030.⁵

Thus far, clinical trials of gene therapies have shown encouraging results in treating a variety of genetic diseases, including:

• Hemophilia
• Sickle cell disease
• Inherited neurological disorders
• Severe combined immunodeficiency
   

Many of the diseases that gene therapies treat are rare

The FDA has approved 2 gene therapies to treat sickle cell disease (SCD), which affects approximately 100,000 people in the U.S.⁶ That figure represents less than 0.03% of the U.S. population. Most diseases treated with approved gene therapies, including those with the highest price tags, are significantly rarer than SCD.

The high cost of specific gene therapies may seem more justified when you consider how transformative they can be. Gene therapies for MLD and SCD, for instance, have radically improved patients’ lives.

Children with the genetic disease MLD lack an enzyme needed to break down fatty substances known as sulfatides that can build up and become toxic to their nerves. Typically, MLD kills affected children before they turn 7. The first MLD patients treated with Lenmeldy, however, have continued to make this missing enzyme for more than 12 years after their treatment.¹¹ Instead of living a short and painful life, they’re developing like other children thanks to gene therapy.

Patients living with SCD have also seen marked improvements from gene therapy. Without gene therapy, they can regularly experience vaso-occlusive crises (VOC), where sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, causing severe pain and organ damage. In one multi-center trial of SCD gene therapy Casgevy, over 93% of patients had improved outcomes for at least 12 consecutive months.¹²
 

Of course, other gene therapies have fallen short. Elevidys is the first gene therapy for pediatric patients between 4 and 5 years of age with Duchenne muscular dystrophy (DMD), which can strip children of their ability to walk by their teens and be fatal by age 30. A recent study showed that one year after receiving the one-time treatment, patients on Elevidys improved by 2.6 points on the North Star Ambulatory Assessment (NSAA) scale, while those in the placebo group improved by 1.9 points.¹³ The difference was statistically insignificant, and the study failed to meet its goal. While this is unfortunate news for DMD patients in the short-term, the results reaffirm why the United States Food and Drug Administration requires rigorous clinical trials of gene therapies: to ensure each gene therapy is safe and effective before it’s approved.

Gene therapies are expensive because of the high costs of research and development, clinical trials, and manufacturing. “Gene therapies treat rare diseases, which means fewer people taking the drugs — so the price of each gene therapy must make up for the cost of research and development,” explains Bryce Mendelsohn, MD PhD, a Kaiser Permanente physician specializing in genetics.

Given the rarity of the diseases gene therapies treat, it’s unlikely that a high percentage of any business’s employee population would be prescribed gene therapy by their doctor. Gene therapies are also just one of many treatments for genetic disease.

“Before we select someone for gene therapy, we consider the natural course of the disease, the option of alternative treatments, and the efficacy and safety of the gene therapy,” Dr. Mendelsohn explains. “Even if you have a million employees, you're only going to have a couple people a year, at most, who would be considered for gene therapy.”

That said, the cost of a single employee or an employee’s family member who needs gene therapy could be substantial. There is no question that employers must consider strategies around gene therapies that can protect their employees and their bottom line.

You can play a vital role in safeguarding the health of your employees and their families while helping them and your business mitigate the high cost of gene therapies.

While the high cost and life-changing potential of advancements in gene therapies makes a splash in the media, it’s important to arm your organization with the facts to determine the best approach for your employees and bottom line. Keep in mind that:

1. Few of your employees will qualify for gene therapies in the immediate future.
   
2. When prescribed to the ideal candidate, gene therapies can have remarkable outcomes.
3. The gene therapy market will expand rapidly by 2030. It’s critical to begin planning for this expansion now.

The number of approved gene therapies and U.S. citizens who need them is going to grow. There are currently over 3,600 cell and gene therapies in the approval pipeline.¹⁵ And one study estimates the number of expected people treated with gene therapy or stem cell therapy will amount to over 340,000 by 2030.¹⁶
 

While the expanding field of costly gene therapies may seem like more cause for concern, remember that most of these treatments will be for rare genetic diseases. If you take a proactive role in educating yourself about gene therapies and speaking with your health care partner, you can help prepare your organization for the future of gene therapies.