Rural Americans May Face Challenges Accessing Gene Therapy

Limited access to gene therapies may impact Americans with rare cancers who live in rural areas.

The newly-approved gene therapies—tisagenlecleucel (Kymriah) for refractory B-cell precursor acute lymphoblastic leukemia and axicabtagene ciloleucel (Yescarta) for diffuse large B-cell lymphoma—offer significant benefits to patients with cancer; however, patients living in rural areas may have to travel hundreds of miles to receive treatment, according to an article from MIT Technology Review.

Tisagenleclucel comes with a $475,000 price tag and axicabtagene ciloleucel is only slightly less costly, at $373,000 for a single treatment. Not only can these high costs put treatment out of reach for some patients, but they are currently only offered in certain areas of the country, according to the article.

A handful of rural states—such as Montana, Wyoming, North Dakota, and New Mexico—do not have any hospitals or clinics that are able to administer gene therapy.

The lack of access to gene therapy may have significant consequences in rural states, in which cancer mortality is already high, according to MIT. Since gene therapy is typically a last resort type of treatment, patients are usually very ill and may have difficulty traveling long distances to receive treatment.

The authors noted that gene therapy is still very new, so additional facilities may receive the necessary training to administer the treatment. In the short-term, patients may be unable to receive gene therapy due to geographical constraints, according to the article.

However, other factors could limit access to gene therapy in the long-term as well. The authors wrote that patients can experience potentially life-threatening adverse events, which only certain medical teams are equipped to address. If many patients experience these adverse events or die, the uptake of gene therapy at additional sites may be slowed, according to the article.

CAR-T cell therapies also treat rare cancers with small patient populations, which may discourage clinics from receiving the required training.

Another complicating factor is how insurance will pay for the costly therapies. The authors said that hundreds of patients are currently waiting to receive treatment due to payment delays. If insurers won’t cover the costs, manufacturers likely won’t expand the number of treatment sites, according to the article.

“We need to watch out for a situation in which these therapies only become available to urban elites who live near academic medical centers,” said Aaron Levine, PhD, Georgia Tech School of Public Policy. “It’s still early enough for things to change and evolve.”

Since the administration and management of adverse events requires a large and specially trained staff, it may be best that gene therapy is only available at facilities with a wealth of resources.

However, these issues may have a greater impact once gene therapies are approved for more common types of cancer, according to the article.

“At that point, I think it’s justified to expand the number of centers, and hopefully that expansion includes smaller cities and more rural states,” said Peter Emanuel, director of the Winthrop P. Rockefeller Cancer Institute in Little Rock, Arkansas.