Cell Therapy

Key facts

Licensed products regulated as

Biologics under PHS Act Section 351, reviewed by CBER

Lower-risk tissue products

HCT/Ps under PHS Act Section 361 and 21 CFR Part 1271

Cell sources

Autologous (the patient) or allogeneic (a donor)

First CAR-T approval

Kymriah (tisagenlecleucel), August 2017

Medicare inpatient grouping

MS-DRG 018, CAR T-cell and other immunotherapies

What is cell therapy?

Cell therapies use whole living cells as the medicine. Examples range from cord blood cell products used in transplantation to chimeric antigen receptor T-cell (CAR-T) therapies, in which a patient's T cells are engineered to recognize and attack cancer cells.

Many cell therapies are also gene therapies, because the cells are genetically modified before infusion. Others, such as cord blood products, are used without genetic modification.

The term is also used loosely by clinics marketing unapproved stem cell treatments. The FDA has warned that many such products require FDA approval and are not legally marketed.

How cell therapies are regulated

The regulatory path depends on how the cells are processed and used:

• Section 351 products: cells that are, for example, more than minimally manipulated or intended for a non-homologous use need an Investigational New Drug application for trials and a Biologics License Application (BLA) for marketing.
• Section 361 HCT/Ps: human cells, tissues and cellular and tissue-based products that meet criteria such as minimal manipulation and homologous use are regulated under 21 CFR Part 1271, which focuses on preventing communicable disease, without premarket approval.
• Manufacturing: autologous products are made one patient at a time, so cell collection, manufacturing and infusion must be tightly scheduled.
• Administration: CAR-T therapies are given at specialized treatment centers equipped to manage serious side effects such as cytokine release syndrome.

Why cell therapy matters

Cell therapies concentrate care in a small set of sites, which shapes analysis:

• Treatment center mapping: use is concentrated at academic medical centers and cancer centers able to collect cells and manage toxicities, which drives targeting and referral network analysis.
• Payment: inpatient CAR-T cases in Medicare group to MS-DRG 018, while outpatient administration and commercial contracts vary, so cost analysis must follow the claim type.
• Trial site selection: cell therapy trials need apheresis capacity, cell processing and intensive care support, which narrows the pool of qualified sites.
• Competitive tracking: the FDA list of approved cellular and gene therapy products and ClinicalTrials.gov records show which targets, such as CD19 and BCMA, are crowded.