CurrentMass General Brigham Health PlanPolicy N/A

Ruconest (C1 esterase inhibitor [recombinant])

Pharmacy benefit prior authorization and coverage policy for Ruconest for treatment of acute hereditary angioedema (HAE) attacks in adults and adolescents, including specialty pharmacy designation, initial and continuation criteria, limits, and exclusions.

Policy Summary

PayerMass General Brigham Health Plan

PolicyRuconest (C1 esterase inhibitor [recombinant])

Policy CodePolicy N/A

Change TypeAdministrative/formatting

Effective DateJan 1, 2024

Next Review Date

Key ActionPrior authorization is required for Ruconest; approvals may be granted for new members currently receiving therapy and for acute HAE attacks meeting lab/genetic criteria; approvals limited to 6 months.

SourceLink

POLICY UPDATE CHANGES

Reviewed at Dec P&T, switched from SGM to Custom: Effective 1/1/2024

1Covered Indication (acute HAE attacks)

1Approval Duration (months)

6Approval Interval (months)

Coverage Summary

This policy covers Ruconest (C1 esterase inhibitor [recombinant]) under the pharmacy benefit with prior authorization for the FDA-approved indication: treatment of acute attacks in adults and adolescent patients with hereditary angioedema (HAE). Authorization is limited to use for acute HAE attacks and requires that Ruconest not be used in combination with other medications for acute HAE attacks. All other indications are considered experimental/investigational and not medically necessary.

Members New to the Plan

Authorization may be granted for members new to the plan who are currently receiving treatment with the requested medication, excluding when the product is obtained as samples or via manufacturer's patient assistance programs.

Member is currently receiving treatment with the requested medication (excluding samples or manufacturer's patient assistance programs)

Provider Actions

Prior Authorization

Prior authorization required

Prior authorization is required for Ruconest. Approvals may be granted for members new to the plan who are currently receiving the medication and for treatment of acute hereditary angioedema (HAE) attacks that meet the laboratory/genetic criteria. All approvals will be granted for a maximum of 6 months.

Documentation Required

Laboratory and genetic documentation required

Document diagnostic testing demonstrating HAE or HAE-related mutation: C4 level below the laboratory lower limit of normal; C1‑INH antigenic level below the laboratory lower limit OR a low C1‑INH functional level (functional C1‑INH < 50% or below the laboratory lower limit of normal). If C1‑INH is normal, document genetic testing showing an F12, angiopoietin‑1, plasminogen, KNG1, HS3ST6, or MYOF gene mutation, or provide a documented family history of angioedema plus a trial of high‑dose antihistamine (e.g., cetirizine 40 mg/day or equivalent) for at least one month demonstrating refractoriness.

Billing Rule

Specialty pharmacy dispensing

This medication has been designated specialty and must be filled at a contracted specialty pharmacy (designated specialty medication dispensing requirement).

Clinical Evidence & References

Evidence sources referenced in this policy include the Ruconest package insert (Pharming Healthcare Inc.; April 2020) and multiple international consensus/guideline publications on HAE diagnosis and management (2010–2021). Specific guideline citations referenced include international consensus algorithms and guideline updates from 2010 through 2021 addressing diagnosis, therapy, and management of hereditary angioedema.

Background & Definitions

Ruconest is a recombinant C1 esterase inhibitor FDA‑approved for treatment of acute HAE attacks in adults and adolescents. The policy restricts use to acute attacks, requires prior authorization (with specialty pharmacy dispensing), and specifies laboratory/genetic documentation to confirm C1‑INH deficiency or relevant mutations when applicable. Effectiveness was not established for laryngeal attacks, approvals are granted for 6 months, and all other indications are considered experimental/investigational and not medically necessary.

Definitions

HAEHereditary angioedema

Revision History

2023-12-13administrativeLatest

Reviewed at Dec P&T, switched from SGM to Custom: Effective 1/1/2024 (administrative/formatting change; no clinical policy statement changes)

2024-01-01effective_date

Policy effective date: Ruconest (C1 esterase inhibitor [recombinant]) Effective 01/01/2024