Hemophilia Gene Therapies
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Defines medical benefit coverage, prior-authorization requirements, and eligibility criteria for Hemgenix and Roctavian gene therapies for adults with hemophilia A or B across Univera Healthcare lines of business (excluding Medicare Part D).
No material clinical or coverage changes in this revision.
Coverage Criteria for Gene Therapies
inv-01: Hemgenix Initial Therapy
Covered when ALL of the following are met
Approval duration: 6 months to allow one-time administration
inv-02: Roctavian Initial Therapy
Covered when ALL of the following are met
Approval duration: 6 months to allow one-time administration
Patients with any of the following conditions are not eligible for coverage. Documentation, including laboratory results obtained within the past 3 months, is required to verify exclusions: active Hepatitis B or C infection; uncontrolled human immunodeficiency virus (HIV) defined as CD4+ count ≤200/μL; thrombocytopenia defined as a platelet count below 50 × 10^9/L; advanced liver disease (suggestive of or equal to METAVIR Stage 3 disease — e.g., a FibroScan score ≥9 kPa is considered equivalent); or a known coagulation disorder other than the specific hemophilia diagnosis under consideration.
Retreatment after prior administration of a gene therapy product is considered investigational and not eligible for coverage. The policy specifies that retreatment with an FDA‑approved gene therapy or any other gene therapy under investigation has not been proven to be safe and effective and therefore will be treated as investigational.
Use of Hemgenix or Roctavian solely for patient or provider convenience is explicitly not medically necessary and will not be authorized. The policy requires clinical indications (e.g., documented life‑threatening hemorrhages or repeated serious spontaneous bleeding despite continuous routine prophylaxis) and does not permit convenience as a justification for coverage.
Initial Therapy Authorization
inv-20: Initial Therapy — One-time administration; approval granted for 6 months to allow administration
Initial authorization is for one-time administration; approval granted for 6 months to allow administration.
Approval granted for 6 months to allow one-time administration
Continuation and Recertification Criteria
inv-21: Continuation Therapy — Continued approval and recertification requirements
Continued approval and recertification requirements
Coding and Diagnostic Thresholds
Provider Requirements and Documentation
Prior authorization required (contract dependent)
Prior authorization is required and is contract dependent; clinical documentation must be submitted for each request unless otherwise specified. HCPCS codes J1411 and J1412 apply to the therapies covered by this policy.
- Prior-authorization is contract dependent (policy guideline).
- Clinical documentation required for initial and recertification requests unless otherwise specified.
Step therapy may be required in addition to LCD/NCD
Step therapy requirements may be imposed in addition to any applicable Medicare NCD/LCD criteria; providers must document prior use of and inadequate control on routine prophylactic factor replacement therapy where specified (e.g., repeated serious spontaneous bleeding episodes or life‑threatening hemorrhages despite continuous prophylaxis).
- Providers must document prior use of and inadequate control on routine prophylactic factor replacement therapy where specified.
- Step therapy may be applied alongside NCD/LCD requirements for Medicare Advantage members.
Required clinical documentation for initial and recertification requests
Submit clinical documentation for each initial and recertification request to demonstrate eligibility and ongoing benefit. Supporting documentation should include progress notes, previous treatment history, diagnostic testing, laboratory results, genetic/biomarker results, and imaging as applicable.
- Progress notes documenting previous treatments and treatment history.
- Diagnostic testing and imaging reports as applicable.
- Laboratory test results and genetic/biomarker results.
Lab and screening documentation required (NAbs, inhibitors, exclusion labs)
Provide documentation confirming screening for neutralizing antibodies (NAbs) to AAV5 and documentation confirming absence of active or historical factor VIII or IX inhibitors; required laboratory results for exclusion conditions must be recent (within the past 3 months).
- Confirmation of NAb screening against AAV5 (Hemgenix and Roctavian require NAb documentation).
- Documentation confirming no active factor inhibitors or history of inhibitors (drug‑specific).
- Laboratory results for exclusion criteria (e.g., hepatitis, HIV, platelets, liver disease) taken within the past 3 months.
Triggers for denial — failure to meet age, diagnosis, lab, or exclusion criteria
Requests will be denied if the patient does not meet age, diagnosis, laboratory, or exclusion criteria specified in the policy (for example: age <18, insufficient factor activity, active hepatitis B/C, uncontrolled HIV, thrombocytopenia, advanced liver disease, or other disqualifying coagulation disorders).
- Age under 18 years.
- Factor IX activity >2% for Hemgenix or FVIII activity ≥1 IU/dL for Roctavian (does not meet severity thresholds).
- Active Hepatitis B or C infection.
- Uncontrolled HIV (CD4+ count ≤200/μL).
- Thrombocytopenia (platelet count below 50 × 10^9/L).
- Advanced liver disease (METAVIR Stage 3 or FibroScan ≥9 kPa).
- Known coagulation disorder other than the specific hemophilia indication.
- Retreatment after prior gene therapy (considered investigational).
Step Therapy Requirements
| Requirement | Details |
|---|---|
| Prior use of routine prophylactic factor replacement therapy | Provider must document prior and continuous use of routine prophylactic factor replacement therapy (factor VIII for hemophilia A; factor IX for hemophilia B) before considering gene therapy as specified in the applicable drug-specific criteria. |
| Evidence of inadequate control while on prophylaxis | Documentation must show life‑threatening hemorrhages or repeated serious spontaneous bleeding episodes despite continuous routine prophylaxis. For Hemgenix, if on prophylactic factor IX therapy, a minimum of 150 exposure days must be documented when applicable. |
| Required supporting documentation | Submit clinical documentation including progress notes detailing prior treatments and treatment history, diagnostic testing, laboratory results (including recent factor activity levels), and genetic/biomarker testing as applicable to demonstrate prior prophylactic use and inadequate control. |
| Step therapy application | Step therapy requirements may be imposed in addition to LCD/NCD requirements; providers must follow any contract‑specific step therapy rules when applicable. |
| Prior authorization | Prior authorization is required and is contract dependent; clinical documentation must be provided for initial and recertification requests. |
Site of Care Considerations
Site of care (infusion center) may impact approval timeframe
Requested site of care (e.g., infusion center) may affect the approval timeframe and is subject to review.
- Providers should note the intended administration site on the authorization request; approvals may be contingent on site review.
Definitions
Background
Hemophilia is an inherited bleeding disorder caused by deficiency of coagulation factor VIII (Hemophilia A) or factor IX (Hemophilia B). Severity is classified by factor activity levels (severe disease is typically defined as <1% of normal). Gene therapies addressed in this policy — Hemgenix (etranacogene dezaparvovec-drlb) for hemophilia B and Roctavian (valoctocogene roxaparvovec-rvox) for hemophilia A — are single intravenous AAV5‑based infusions intended to increase circulating factor activity. Both therapies require treatment planning with a hematologist at a Hemophilia Treatment Center, pre‑treatment screening for neutralizing antibodies to AAV5, and confirmation of absence of factor inhibitors; initial authorization is for a one‑time administration with approval duration provided to allow administration.
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