Aug 05th 2021

Gene Therapy Is Here. Are You Ready for the Impact?

The future is here—gene therapy is on the cusp of becoming a reality for individuals with previously untreatable conditions. From sickle cell disease to hemophilia to cystic fibrosis, the pipeline for groundbreaking gene therapies is growing.

 

The potential impact on healthcare is huge, but so is the cost hurdle. With some gene therapies costing millions of dollars for a single treatment, one major question is how to pay for them. Insurance plans need to start seriously considering the implications, from the perspective of both cost and quality.

 

The need for gene therapy

 

Gene therapy is an experimental technique that transplants normal genes into cells that have defective or missing genes. The goal is to correct the underlying problem at the genetic level in order to reduce or prevent the symptoms of the disease.

 

An estimated 25-30 million Americans have a rare disease, according to the U.S. Department of Health and Human Services. While approximately 7,000 rare diseases have been identified, only a few hundred approved treatments are available.

 

Because more than 80 percent of rare diseases are caused by a single gene, gene therapy has the potential to cure a host of different conditions. Many of those conditions have no other viable cures. Others currently require a lifetime regimen of managing symptoms—whereas it may only take one dose of gene therapy to treat them.

“Gene therapy is fundamentally a different prospect for many patients," Philip Gregory, chief scientific officer of gene therapy company bluebird bio, told the NewStatesman magazine. “The goal is to address the underlying cause of disease through a one-time treatment."

 

Current developments

 

Over the last four decades, researchers have made slow but steady progress in gene therapy as they built the necessary foundation for success. In the last few years, however, the pace has accelerated, and the FDA has approved several gene therapy products since 2017. The approved treatments include:

·      Luxturna (voretigene neparvovec-rzyl) for an inherited form of vision loss that may result in blindness in children or adults.

·      Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy (a genetic disease impacting muscle movements, worsening with age) in infants under 2 years old.

 

When the FDA announced the approval of Luxturna in 2017, Dr. Scott Gottlieb, FDA commissioner at the time, said, “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses. We're at a turning point when it comes to this novel form of therapy."

 

Currently, hundreds of clinical trials are under way. Some are for rare diseases and various forms of cancers, but others address inherited, life-threatening disorders such as cystic fibrosis.

Healthcare leaders expect the pipeline to continue to expand this year. And, according to the Insurance Journal, the FDA anticipates approving as many as 20 gene and cell therapies each year by 2025.

 

Who will pay?

 

Gene therapy treatments are among the costliest therapies, not only in the United States but around the world. Luxturna, for example, costs $850,000, while Zolgensma is $2.125 million.

 

The health insurance community is examining various cost models and programs for gene therapy, focusing on the question of how to make these products financially sustainable options for future generations.

 

As the potential of these treatments continues to move beyond experimental—and more and more innovative research generates approved products—insurance plans will need to continue to closely monitor these developments. Without a doubt, they will add further complexities to designing health plan benefits, and innovative approaches will almost certainly be necessary for this technology to become widely available.