C1 Esterase Inhibitor (recombinant) (Ruconest®)
IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
POLICY
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.
FDA-Approved Indication
Ruconest is indicated for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).
Limitation of Use
Effectiveness was not established in HAE patients with laryngeal attacks.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review:
A. For initial authorization, the following should be documented:
1. C1 inhibitor functional and antigenic protein levels
2. F12, angiopoietin-1, plasminogen, kininogen-1 (KNG1), heparan sulfate-glucosamine 3-O-sulfotransferase 6 (HS3ST6), or myoferlin (MYOF) gene mutation testing, if applicable
3. Chart notes confirming family history of angioedema and the angioedema was refractory to a trial of high-dose antihistamine therapy, if applicable
B. For continuation of therapy, chart notes demonstrating a reduction in severity and/or duration of attacks
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a prescriber who specializes in the management of HAE.
IV. CRITERIA FOR INITIAL APPROVAL
Hereditary angioedema (HAE)
Authorization of 6 months may be granted for treatment of acute HAE attacks when the requested medication will not be used in combination with any other medication used for the treatment of acute HAE attacks and either of the following criteria is met at the time of diagnosis:
A. Member has C1 inhibitor deficiency or dysfunction as confirmed by laboratory testing and meets one of the following criteria.
1. C1 inhibitor (C1-INH) antigenic level below the lower limit of normal as defined by the laboratory performing the test; or
2. Normal C1-INH antigenic level and a low C1-INH functional level (functional C1-INH less than 50% or C1-INH functional level below the lower limit of normal as defined by the laboratory performing the test)
B. Member has normal C1 inhibitor as confirmed by laboratory testing and meets one of the following criteria:
1. Member has an F12, angiopoietin-1, plasminogen, kininogen-1 (KNG1), heparan sulfate-glucosamine 3-O-sulfotransferase 6 (HS3ST6), or myoferlin (MYOF) gene mutation as confirmed by genetic testing, or
2. Member has a documented family history of angioedema and the angioedema was refractory to a trial of high-dose antihistamine therapy (i.e., cetirizine at 40 mg per day or the equivalent) for at least one month.
V. CONTINUATION OF THERAPY
Authorization of 6 months may be granted for continuation of therapy when all of the following criteria are met:
A. Member meets the criteria for initial approval.
B. Member has experienced a reduction in severity and/or duration of acute attacks.
C. Prophylaxis should be considered based on the attack frequency, attack severity, comorbid conditions, and member’s quality of life.
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Ruconest [package insert]. Warren, NJ: Pharming Healthcare Inc.; April 2020.
2. Bowen T, Cicardi M, Farkas H, et al. 2010 International consensus algorithm for the diagnosis, therapy, and management of hereditary angioedema. Allergy Asthma Clin Immunol. 2010;6(1):24.
3. Cicardi M, Bork K, Caballero T, et al. Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012;67:147-157.
4. Busse PJ, Christiansen, SC, Riedl MA, et al. US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema. J Allergy Clin Immunol: In Practice. 2021 Jan;9(1):132-150.e3.
5. Zuraw BL, Bork K, Binkley KE, et al. Hereditary angioedema with normal C1 inhibitor function: consensus of an international expert panel. Allergy Asthma Proc. 2012; 33(6):S145-S156.
6. Maurer M, Magerl M, Ansotegui I, et al. The international WAO/EAACI guideline for the management of hereditary angioedema – the 2021 revision and update. Allergy. 2022 Jan 10. doi: 10.1111/all. 15214. Online ahead of print.
7. Lang DM, Aberer W, Bernstein JA, et al. International consensus on hereditary and acquired angioedema. Ann Allergy Asthma Immunol. 2012;109:395-402.
8. Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis, and approach to treatment for angioedema: consensus report from the Hereditary Angioedema International Working Group. Allergy. 2014;69:602-616.
9. Bowen T. Hereditary angioedema: beyond international consensus – circa December 2010 – The Canadian Society of Allergy and Clinical Immunology Dr. David McCourtie Lecture. Allergy Asthma Clin Immunol. 2011;7(1):1.
10. Bernstein JA. Update on angioedema: Evaluation, diagnosis, and treatment. Allergy and Asthma Proceedings. 2011;32(6):408-412.
11. Longhurst H, Cicardi M. Hereditary angio-edema. Lancet. 2012;379:474-481.
12. Farkas H, Martinez-Saguer I, Bork K, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017;72(2):300-313.
13. Henao MP, Kraschnewski J, Kelbel T, Craig T. Diagnosis and screening of patients with hereditary angioedema in primary care. Therapeutics and Clin Risk Management. 2016;12:701-711.
14. Bernstein J. Severity of hereditary angioedema, prevalence, and diagnostic considerations. Am J Med. 2018;24:292-298.
15. Bork K, Aygören-Pürsün E, Bas M, et al. Guideline: Hereditary angioedema due to C1 inhibitor deficiency. Allergo J Int. 2019;28:16–29.
16. Craig T, Busse P, Gower RG, et al. Long-term prophylaxis therapy in patients with hereditary angioedema with C1 inhibitor deficiency. Ann Allergy Asthma Immunol. 2018;121(6):673-679.
17. Sharma J, Jindal AK, Banday AZ, et al. Pathophysiology of Hereditary Angioedema (HAE) Beyond the SERPING1 Gene [published online ahead of print, 2021 Jan 14] [published correction appears in Clin Rev Allergy Immunol. 2021 Feb 17]. Clin Rev Allergy Immunol. 2021;10.1007/s12016-021-08835-8. Doi:10.1007/s12016-021-08835-8.
18. Kanani, A., Schellenberg, R. & Warrington, R. Urticaria and angioedema. All Asth Clin Immun 7, S9 (2011), Table 2.
19. Veronez CL, Csuka D, Sheik FR, et al. The expanding spectrum of mutations in hereditary angioedema. J Allergy Clin Immunol Pract. 2021;S2213-2198(21)00312-3.
ORIGINAL EFFECTIVE DATE: 6/13/2009
MOST RECENT REVIEW DATE: 4/9/2024
ID_CHS
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
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