Medical policy: Expanded Access

Número de política: MP 2.386

Fecha de entrada en vigor: 11/1/2026

Política

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.

Referencias cruzadas

• 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

Variaciones del producto

Esta política solo se aplica a ciertos programas y productos administrados por Capital Blue Cross y está sujeta a variaciones en los beneficios. Consulte la información adicional a continuación.

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.

Descripción/Antecedentes

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.

Fundamento

N/D

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.

Exención de responsabilidad

Las políticas médicas de Capital Blue Cross se utilizan para determinar la cobertura de tecnologías, procedimientos, equipos y servicios médicos específicos. Estas políticas médicas no constituyen un consejo médico y están sujetas a cambios según lo permita la ley o la evidencia clínica aplicable de las pautas de tratamiento independientes. Los proveedores que brindan tratamiento son individualmente responsables de los consejos médicos y el tratamiento de los miembros. Estas pólizas no son una garantía de cobertura o pago. El pago de las reclamaciones está sujeto a la determinación del programa de beneficios del miembro y la elegibilidad en la fecha del servicio, y a la determinación de que los servicios son médicamente necesarios y apropiados. El procesamiento final de una reclamación se basa en los términos del contrato que se aplican al programa de beneficios de los miembros, incluidas las limitaciones y exclusiones de beneficios. Si un proveedor o miembro tiene alguna pregunta sobre esta política médica, debe comunicarse con Servicios para proveedores o Servicios para miembros de Capital Blue Cross.

Información de codificación

Nota: esta lista de códigos puede no ser exhaustiva y los códigos están sujetos a cambios en cualquier momento. La identificación de un código en esta sección no denota cobertura, ya que la cobertura está determinada por los términos de la información de beneficios del miembro. Además, no todos los servicios cubiertos son elegibles para un reembolso por separado.

Specific Procedure Coding does not apply to this policy

Referencias

1. Administración de Medicamentos y Alimentos (FDA) de los EE. UU. IDE Institutional Review Boards (IRB). Updated May 13, 2024.
2. Administración de Medicamentos y Alimentos (FDA) de los EE. UU. News and Events. Expanded Access. Updated September 8, 2025.
3. Administración de Medicamentos y Alimentos (FDA) de los EE. UU. 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.