Medical policy: Expanded Access

Policy number: MP 2.386

Effective date: 11/1/2026

Policy

Expanded access for non-investigational interventions

Expanded access to interventions that are approved, yet not typically preferred per medical policy may be covered when an agreement exists between a provider and Capital (or other Blue plan) and at least ONE of the following criteria is met:

1. The intervention meets the Capital medical policy requirements for necessity, including cost-effectiveness; OR
2. The agreement specifies criteria for coverage with evidence development.
   1. This may include, but does not require, enrollment by the member in a clinical trial.

Expanded access for investigational interventions (“Compassionate Use”)

The medical necessity and appropriateness for routine patient costs associated with an intervention authorized by the FDA as an investigational intervention, yet used outside of a clinical trial (compassionate use), will be reviewed on an individual basis, and must meet all the following criteria:

• The member has a serious or immediately life-threatening disease or condition and
• No comparable or satisfactory therapeutic alternatives are available; and
• The product manufacturer and the patient’s doctor have made special arrangements to obtain the product for the patient; and
• These arrangements are documented and authorized by the FDA; and
• Use is consistent with FDA authorization and indications and
• Member benefit justifies the potential risks of treatment and
• Member enrollment in a clinical trial is not possible and
• Member is a medically appropriate candidate and
• Use is consistent with peer-reviewed published medical literature or other appropriately recognized resources

Routine patient costs related to expanded access to investigational interventions do not include any of the following:

• The investigational drug, biological product, device, medical treatment or procedure itself;
• Items and services that are not used in the direct clinical management of the patient;
• Any service that is clearly inconsistent with widely accepted and established standards of care for a particular diagnosis;
• Services and supplies provided by the sponsor;
• Member travel expenses.

Cross-references

• MP 4.003 Medical Necessity
• MP 2.383 Orphan Drugs, Humanitarian Use Device (HUD)
• MP 2.103 Off-Label Use of Medications and Other Interventions

Product variations

This policy is only applicable to certain programs and products administered by Capital Blue Cross and subject to benefit variations. Please see additional information below.

FEP PPO - Refer to Section VI of the FEP Medical Policy Manual. The FEP Medical Policy Manual can be found at: FEP Medical Policy Manual.

Description/Background

Expanded access for investigational interventions

Per the FDA, expanded access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product (i.e., one that has not been approved by Food and Drug Administration). The Food and Drug Administration (FDA) is committed to increasing awareness of and knowledge about its expanded access programs and the procedures for obtaining access to human investigational drugs (including biologics) and medical devices.

Wherever possible, use of an investigational medical product by a patient as part of a clinical trial is preferable. However, when patient enrollment in a clinical trial is not possible (e.g., that there are no ongoing trials, a patient may not have access to a clinical trial or may not be eligible for the clinical trials, distance to get to a trial prevents access), patients may be able to receive the product, when appropriate, through expanded access.

Investigational medical products have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

Ensuring patient safety is a priority. FDA must determine that the potential patient benefit justifies the potential risk of the expanded access use of the investigational drug, and that the potential risk is not unreasonable in the context of the disease or condition to be treated. Even with safeguards, there may be significant unknowns about safety and effectiveness.

Rationale

N/A

Definitions/Background

Expanded access: Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

Disclaimer

Capital Blue Cross’ medical policies are used to determine coverage for specific medical technologies, procedures, equipment, and services. These medical policies do not constitute medical advice and are subject to change as permitted by law or applicable clinical evidence from independent treatment guidelines. Treating providers are solely responsible for medical advice and treatment of members. These polices are not a guarantee of coverage or payment. Payment of claims is subject to a determination regarding the member’s benefit program and eligibility on the date of service, and a determination that the services are medically necessary and appropriate. Final processing of a claim is based upon the terms of contract that applies to the members’ benefit program, including benefit limitations and exclusions. If a provider or a member has a question concerning this medical policy, please contact Capital Blue Cross’ Provider Services or Member Services.

Coding information

Note: This list of codes may not be all-inclusive, and codes are subject to change at any time. The identification of a code in this section does not denote coverage as coverage is determined by the terms of member benefit information. In addition, not all covered services are eligible for separate reimbursement.

Specific Procedure Coding does not apply to this policy

References

1. U.S. Food and Drug Administration (FDA). IDE Institutional Review Boards (IRB). Updated May 13, 2024.
2. U.S. Food and Drug Administration (FDA). News and Events. Expanded Access. Updated September 8, 2025.
3. U.S. Food and Drug Administration (FDA). Expanded Access for Medical Devices. Updated June 21, 2019.
4. Electronic Code of Federal Regulations. Title 21 Food and Drugs, Chapter I Food and Drug Administration, Department of Health and Human Services, Subchapter D Drug for Human Use, Part 312 Investigational New Drug Application, Subpart I. Expanded Access to Investigational Drugs for Treatment Use. Aug. 13, 2009.