US Pharm. 2024;49(10):14.

Research and development of cell and  gene therapies (CGTs) in the United States have increased substantially over the past 20 years. These products, which provide novel approaches to treat, cure, or prevent rare and severe diseases, include immunotherapies, cancer vaccines, and autologous and allogenic cells (e.g., hematopoietic, embryonic, and adult stem cells). Cell therapy utilizes living cells that may be unmodified (native) and/or gene-edited (engineered). Gene therapy involves the insertion of functional genes into malfunctioning genes of cells using vectors (e.g., viruses) to yield gene augmentation therapy, gene inhibition therapy, suicide gene insertion, and other targeted treatment modalities. CGTs for human use are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER).


FDA-Approved Products: FDA approval of biologics has increased from 14% of all new drug approvals in 2004 to 48% in 2023. As of August 2024, a total of 38 CGTs have been FDA approved for use in the U.S. Most of these products (21%) are umbilical cord blood derivatives (Allocord, Clevecord, Ducord, Hemacord, University of Colorado Cord Blood Bank, MD Anderson Cord Blood Bank, LifeSouth, and Bloodworks). Chimeric antigen receptor T-cell therapeutic products, which represent the next largest group of approved CGTs (16%), include the following cancer immunotherapy products: Abecma, Breyanzi, Carvykti, Kymriah, Tecartus, and Yescarta. The newly established Office of Therapeutic Products of the CBER maintains a complete list of FDA-approved CGTs at www.fda.gov/vaccines-blood-biologics.

Pipeline: Last year was considered a breakthrough year for CGTs, as seven gene therapies were approved by the FDA. This growth is largely due to the 3.44-fold increase in clinical trials involving CGTs between 2004 and 2023 (102 vs. 351 trials, respectively). Five hundred products are currently in the U.S. pipeline, with 10 to 20 new CGT approvals expected in 2025. Advances in the areas of oncology (B-cell leukemia and lymphoma), rare diseases (hematologic, neurologic, and ophthalmologic conditions), and highly prevalent diseases (age-related macular degeneration and osteoarthritis) are most anticipated based on studies forecasted through 2026.

Considerations: The introduction of transformative CGTs for numerous potentially life-threatening conditions, degenerative diseases, genetic disorders, and cancers has revolutionized healthcare and offers great promise to countless patients. However, affordability poses a significant challenge for many individuals. CGT costs can range from thousands to millions of dollars per dose or treatment. Therefore, health-plan sponsors must explore financial strategies to support sustainable reimbursements and patient access to these therapies (e.g., performance-based contracting, subscription models, payment installments) as well as appropriate coverage criteria (e.g., use for FDA-approved indications only).

The content contained in this article is for informational purposes only. The content is not intended to be a substitute for professional advice. Reliance on any information provided in this article is solely at your own risk.

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