How to have a data-driven discussion on gene and cellular therapies

Gene and cellular therapies have life-changing potential, but they are costly and can be complicated to understand. As the pipeline expands and as plans strive to make proactive, informed decisions for their organizations, we’re offering guidance to help you navigate this complex space.

Educate yourself about gene and cellular therapies.

Gene therapies are highly targeted medications that introduce genetic material into a person’s DNA to “edit” and replace faulty or missing genetic material that leads to disease. Gene therapies are intended to be administered in a single dose provide long-term benefit, and in some cases, a cure.

Cellular therapies are created by a process that involves removing cells from the patient’s body and modifying the genes of those cells in a laboratory, essentially programming them to treat or fight a specific disease. These modified cells are then introduced back into the patient’s body. 

More than 900 gene and cellular therapies are in the pipeline. The six therapies approved to date by the Food Drug Administration (FDA) treat conditions that affect a relative few Americans, such as spinal muscular atrophy, with a patient population of about 4,000. In the near future, we expect to see gene and cell therapies available for much more prevalent conditions, such as Duchenne muscular dystrophy, which affects 17,000 patients; hemophilia A and B, with at least 30,000 patients, and even prostate cancer, which has a patient population of nearly 250,000.

While not all patients will be appropriate candidates for these costly therapies, plan sponsors should begin to prepare for this pipeline now. By 2025, the FDA estimates it will approve 10 to 20 gene and cellular therapies each year.

These key resources can help you begin to understand the rapidly evolving landscape of gene therapy:

• Gene Therapies 101
• A Brief History of Gene Therapy
• Gene Therapy vs. Cell Therapy
• Recognizing Patient Challenges

Understand your coverage options

Plans have a spectrum of coverage options to consider, from not covering gene and cellular therapies at all to stop-loss based solutions, to programs that ensure no copays to members.

Utilize sophisticated modeling tools to understand the pipeline and its impact to your plan.

Some forecasting methods are more helpful than others as plans prepare for the potential impact of gene therapies. Many tools use only disease prevalence and incidence data. However, the evaluation of each individual drug in the pipeline should also consider patient demographics, disease severity, characteristics of the patient/prescriber population, and clinical trial data. 

Download our discussion guide to learn more about the gene and cellular therapy pipeline and how you can begin to prepare a data-supported recommendation for your organization.