Immunological Cellular Therapies and Gene Therapies

Policy No: 112
Originally Created: 04/01/2018
Section: Medicine
Last Reviewed: 05/01/2019
Last Revised: 05/01/2019
Approved: 05/16/2019
Effective: 06/01/2019

The policy applies to all facilities (hospitals, surgery centers, kidney centers, etc.)


Immunological cellular therapies
Methods of treating cancer and other diseases. The spontaneous regression of certain cancers (e.g., renal cell carcinoma, melanoma) supports the idea that a patient's immune system can delay tumor progression and, on rare occasions, can eliminate tumors altogether. These observations have led to research into various immunologic therapies designed to stimulate a patient's own immune system.

Adoptive cellular therapies
A type of immunological cellular therapy. A treatment used to help the immune system fight diseases, such as cancer and infections with certain viruses. T cells are collected from the patient, processed, and returned to the patient. Therapies of this type include, but are not limited to CAR-T therapies.

Gene therapies
Gene therapy is proposed to treat or prevent certain diseases by inserting foreign genetic information into a person's cells. Gene therapies can work by several mechanisms:

  • Replacing a disease-causing gene with a healthy copy of the gene
  • Inactivating a disease-causing gene that is not functioning properly
  • Introducing a new or modified gene into the body to help treat a disease

There are a variety of types of gene therapy products, including but not limited to:

  • plasmid DNA,
  • viral vectors,
  • bacterial vectors,
  • human gene editing technology and
  • patient-derived cellular gene therapy products.

Gene therapies include, but are not limited to voretigene neparovove-rzyl.

Drug component (active Ingredients)

Any component of a drug product intended to furnish pharmacological activity or other direct effect in the diagnosis, cure, mitigation, treatment, or prevention of disease, or to affect the structure or any function of the body of humans or other animals. Active ingredients include those components of the product that may undergo chemical change during the manufacture of the drug product and be present in the drug product in a modified form intended to furnish the specified activity or effect.

National Drug Code (NDC)
Each listed drug product is assigned a unique 11 digit, 3-segment number. This number, known as the NDC, identifies the labeler, product, and trade package size. The first segment, the labeler code, is assigned by the FDA. A labeler is any firm that manufactures (including repackers or relabelers) or distributes (under its own name) the drug. The second segment, the product code, identifies a specific strength, dosage form, and formulation of a drug for a particular firm. Different formulations or different strengths of the same formulation should be assigned different product codes. The third segment, the package code, identifies package sizes and types. Different package codes only differentiate between different quantitative and qualitative attributes of the product packaging.

Policy Statement

For the purpose of the Immunological Cellular Therapies and Gene Therapies reimbursement policy, drug components and NDC applies to FDA approved agents/drugs administered in an outpatient facility, inpatient hospital or ASC settings. Examples of FDA approved agents/drugs includes, but are not limited to: tisagenlecleucel, axicabtagene ciloleucel, or voretigene neparvovec-rzyl.

Supplier or manufacturer invoice and NDC of the agents/drugs are required to be billed for the drug component(s). Drug component charges must be uniquely identified from other charges associated with the inpatient or outpatient services in a separate claim. Drug component charges must be the facility's actual acquisition cost by invoice. Claims will be denied if the health plan is unable to identify the charges.

Reimbursement for the drug component(s) will be at the supplier or manufacturer invoice price. If supplier or manufacturer invoice is not provided, reimbursement will be 50% of wholesale acquisition cost (WAC) published by MediSpan.

Any off-invoice discount, rebate or refund for the drug component received by the facility must be returned in full to the health plan. Additionally, the health plan must not be billed if no payment has been made by the provider for the drug component.

Immunological cellular therapies and gene therapies are only eligible for coverage in certain clinical conditions subject to pre-authorization and as specified in the health plan's applicable medical and medication policies. Please see the cross-reference section.


National Cancer Institute (NIH)

Registrar Corp, U.S. FDA Drug Definition

United States Food and Drug Administration, Vaccines, Blood & Biologics, Cellular and Gene Therapy Products, What is Gene Therapy

United States Food and Drug Administration, Drug Approvals and Databases, National Drug Code Directory

Cross References

Discarded Drugs and Biologicals

Medication Policy Manual (Note: Do a (Ctrl+F) and enter drug name in the find bar to locate the appropriate policy.)

Immunological Cellar Therapies and Gene Therapies, Medical Policy Manual, Medicine, Policy No. 42

Immunological Cellar Therapies and Gene Therapies, Medicare Advantage Medical Policy Manual, Medicine, Policy No. 42


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