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| Medical Policy: | I-9-016 |
| Topic: | Treatment of Gaucher Disease |
| Section: | Injections |
| Effective Date: | May 28, 2018 |
| Issue Date: | May 28, 2018 |
| Last Reviewed: | February 2018 |
Gaucher disease is a genetic disorder resulting in the malfunction or absence of the enzyme glucocerebroside. The deficiency of this enzyme causes accumulation of glucosylceramide in the liver, spleen, bone marrow, and lymph nodes. Treatment for this disease focuses on either replacement of the missing or ineffective enzyme (enzyme replacement therapy [ERT]), or reduction of the substrate upon which it acts (substrate reduction therapy [SRT]). |
This policy is designed to address medical guidelines that are appropriate for the majority of individuals with a particular disease, illness, or condition. Each person's unique clinical circumstances may warrant individual consideration, based on review of applicable medical records.
| Policy Position Coverage is subject to the specific terms of the member’s benefit plan. |
Imiglucerase (Cerezyme), taliglucerase alfa (Elelyso), and velaglucerase alfa (VPRIV) are indicated for use as a long-term enzyme replacement therapy for pediatric and adult individuals with a confirmed diagnosis of Type I Gaucher disease.
Imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), are indicated for treatment when ONE or more of the following conditions exists in individuals with confirmed Type I Gaucher disease:
- Anemia with hemoglobin of:
- Less than or equal to 11.5 g/dL for females; or
- Less than or equal to 12.5 g/dL for males; or
- Less than or equal to 1.0 g/dL or more below the lower limit of normal for age and sex; or
- Thrombocytopenia with platelet count less than or equal to 120,000/mm³; or
- Bone disease (e.g., osteonecrosis, osteopenia, secondary pathologic fractures); or
- Clinically significant hepatomegaly (liver size 1.25 or more times normal) or splenomegaly (spleen size five (5) or more times normal); or
- Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia.
The use of imiglucerase (Cerezyme), velaglucerase alfa (VPRIV) and taliglucerase alfa (Elelyso), for any other indication, including but not limited to the following, is considered experimental/investigational and, therefore, not covered. Scientific evidence of safety and efficacy has not been proven.
- Type II Gaucher disease
- Type III Gaucher disease
| Place of Service: Outpatient |
Experimental/Investigational (E/I) services are not covered regardless of place of service.
Treatment of Gaucher disease is typically an outpatient procedure which is only eligible for coverage as an inpatient procedure in special circumstances, including, but not limited to, the presence of a co-morbid condition that would require monitoring in a more controlled environment such as the inpatient setting.
| The policy position applies to all commercial lines of business |
| Denial Statements |
Services that do not meet the criteria of this policy will be considered experimental/investigational (E/I). A network provider can bill the member for the experimental/investigational service. The provider must give advance written notice informing the member that the service has been deemed E/I. The member must be provided with an estimate of the cost and the member must agree in writing to assume financial responsibility in advance of receiving the service. The signed agreement must be maintained in the provider’s records.
| Links |
- Link to Provider Resource Center for the Medical Policy Update
03/2018, Coverage Criteria Revised for the Treatment of Gaucher Disease
 |
Medical policies do not constitute medical advice, nor are they intended to govern the practice of medicine. They are intended to reflect Highmark's reimbursement and coverage guidelines. Coverage for services may vary for individual members, based on the terms of the benefit contract.
Discrimination is Against the Law
The Claims Administrator/Insurer complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability, or sex. The Claims Administrator/Insurer does not exclude people or treat them differently because of race, color, national origin, age, disability, or sex. The Claims Administrator/ Insurer:
- Provides free aids and services to people with disabilities to communicate effectively with us, such as:
- Qualified sign language interpreters
- Written information in other formats (large print, audio, accessible electronic formats, other formats)
- Provides free language services to people whose primary language is not English, such as:
- Qualified interpreters
- Information written in other languages
If you believe that the Claims Administrator/Insurer has failed to provide these services or discriminated in another way on the basis of race, color, national origin, age, disability, or sex, you can file a grievance with: Civil Rights Coordinator, P.O. Box 22492, Pittsburgh, PA 15222, Phone: 1-866-286-8295, TTY: 711, Fax: 412-544-2475, email: CivilRightsCoordinator@highmarkhealth.org. You can file a grievance in person or by mail, fax, or email. If you need help filing a grievance, the Civil Rights Coordinator is available to help you.
You can also file a civil rights complaint with the U.S. Department of Health and Human Services, Office for Civil Rights electronically through the Office for Civil Rights Complaint Portal, available at https://ocrportal.hhs.gov/ocr/portal/lobby.jsf, or by mail or phone at:
U.S. Department of Health and Human Services
200 Independence Avenue, SW
Room 509F, HHH Building
Washington, D.C. 20201
1-800-368-1019, 800-537-7697 (TDD)
Complaint forms are available at http://www.hhs.gov/ocr/office/file/index.html.
Insurance or benefit/claims administration may be provided by Highmark, Highmark Choice Company, Highmark Coverage Advantage, Highmark Health Insurance Company, First Priority Life Insurance Company, First Priority Health, Highmark Benefits Group, Highmark Select Resources, Highmark Senior Solutions Company or Highmark Senior Health Company, all of which are independent licensees of the Blue Cross and Blue Shield Association, an association of independent Blue Cross and Blue Shield plans.
Highmark retains the right to review and update its medical policy guidelines at its sole discretion. These guidelines are the proprietary information of Highmark. Any sale, copying or dissemination of the medical policies is prohibited; however, limited copying of medical policies is permitted for individual use.
