Cell and Gene Therapies

Biopharmaceutical innovation is ushering in the next generation of advanced medicines. Cell and gene therapies fight diseases at their source - the cellular or genetic level - through one-time administration, resulting in long-term, potentially curative benefits. These transformational therapies are revolutionizing health care and should be accessible to all patients who need them.

Cell Therapy

Introducing new cells into a patient’s body to help treat or cure a disease

A variety of different types of cells can be used in cell therapy and can come from the patient or a donor. In some cases, such as CAR-T, cells are genetically modified before being (re)introduced into the patient.

Gene Therapy

Modifying or introducing new or corrected genes into a patient’s body with the goal of treating, preventing or potentially curing a disease

Examples of gene therapy approaches include replacing a mutated gene that causes disease with a healthy copy or introducing a new or modified gene into the body.

2017

The year the U.S. FDA approved the first ever directly administered gene therapy

6

The number of FDA-approved CAR-T cell therapies

(as of September 2023)

9

The number of FDA-approved gene therapies

(as of December 2023)

480+

The number of cell and gene therapies in development

(as of June 2022)

The Potential to Treat and Cure Some of the Most Devastating Diseases

With cell and gene therapies, we can now cure inherited childhood blindness, for example, and enable children with spinal muscular atrophy to live past the age of two. And CAR-T therapies now arm the body’s own immune system to fight blood cancers.

Some transformative therapies have the potential to lower overall health care costs by reducing health care utilization and the cost of managing these devastating illnesses over a patient’s lifetime. For example, the current standard of care for hemophilia requires patients to receive blood factor infusions multiple times a week. In the year following administration, gene therapies have the potential to reduce health care costs by as much as $730,000 per patient.

Despite this impressive pipeline, cell and gene therapies are projected to remain a small share of U.S. health care spending over the next five to eight years, totaling less than 0.5% of future health care spending. What’s more, we can make these treatments accessible and affordable by moving toward a value-driven health care system.

Chemists examining test tubes in a laboratory

Stories from the lab: Researchers discuss innovative treatments for cancers of the blood

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Ensuring Access to Transformative Therapies

We must help ensure patients who need these transformative therapies can access them, and that our payment system keeps pace with innovation. Cell and gene therapies are tailored treatments based on a patient’s genetic makeup and use specialized manufacturing and delivery processes. And while stakeholders across the health care system may benefit over the long-term from cell and gene therapies, one payer may ultimately face one up-front payment for these complex therapies but not receive the benefit from their potential savings over the long term. That’s why innovative approaches to finance and payment may be needed.

Biopharmaceutical companies are working with payers to develop new ways to pay for medicines, like innovative contracts. These flexible payment arrangements may lower costs through voluntary, market-based arrangements between manufacturers and payers.

To date, insurers and biopharmaceutical companies have announced nearly 100 such partnerships – covering nearly 70 treatments including many cell and gene therapies – and their continued use promises to deliver even greater value to the health care system in the future. These innovative contracts can improve outcomes, add more treatment options and lower out-of-pocket costs for patients while providing savings for the health system.