CurrentHealth NetPolicy CP PHAR.64

Clinical Policy: Topotecan (Hycamtin)

Defines medical necessity criteria, approvals, dosing limits, continuation criteria, covered indications (FDA and NCCN-recommended off-label), provider documentation and generic-first directive, and approval durations for topotecan (Hycamtin) across Commercial, HIM and Medicaid lines of business.

Policy Summary

PayerHealth Net

PolicyClinical Policy: Topotecan (Hycamtin)

Policy CodePolicy CP PHAR.64

Change TypeAnnual reviews and revisions (2021-2025)

Effective Date06.01.11

Next Review Date

Key ActionProvider must submit documentation (office chart notes, lab results, other clinical information) supporting that member has met all approval criteria.

SourceLink

POLICY UPDATE CHANGES

2Q 2025 annual review: added pediatric medulloblastoma to off-label NCCN recommendations and updated vaginal cancer criteria; references reviewed and updated.

2Q 2024 annual review: revised ovarian mucinous carcinoma wording to mucinous neoplasms; added neuroblastoma and revised Merkel cell carcinoma criteria; added redirection to generic topotecan if available.

2Q 2022 annual review: expanded coverable ovarian diagnoses to include fallopian tube and primary peritoneal cancer; added requirement for single agent use or in combination with bevacizumab or sorafenib; modified cervical and off-label criteria; changed commercial capsule approval duration to 12 months or duration of request.

2Q 2021 annual review: updated reference for HIM off-label use policy to HIM.PA.154 and reviewed references; no significant coverage changes.

3FDA-approved primary indications listed (ovarian, small cell lung, cervical)

8NCCN off-label indications referenced

30Days minimum on therapy for continuation

Coverage Summary

Policy Number: CP PHAR.64. Status: covered_with_criteria. Effective Date: 06.01.11; Last Review Date / Approval Date: 05.25. Scope summary: defines medical necessity criteria, approved indications (FDA-approved and NCCN-recommended off-label), dosing limits, continuation/renewal criteria, required provider documentation and oncologist involvement, a generic-first directive for topotecan (Hycamtin injection), and approval durations across Commercial, HIM (marketplace), and Medicaid lines of business.

Initial Therapy Criteria

Initial Approval Criteria

Topotecan (Hycamtin) is medically necessary when ALL criteria below are met for the specified diagnostic category.

A. Ovarian cancer / fallopian tube / primary peritoneal cancer (must meet all)

Diagnosis of ovarian cancer (including serous epithelial carcinoma, mucinous neoplasms, clear cell carcinoma, endometrioid carcinoma, low-grade serous carcinoma, and carcinosarcoma), fallopian tube cancer, or primary peritoneal cancer
Request is for topotecan for injection
Prescribed by or in consultation with an oncologist
Age ≥ 18 years
Disease progression on or after initial or subsequent chemotherapy (see Appendix B for examples of therapies)
Administration/regimen
- As a single agent
- In combination with bevacizumab or sorafenib (off-label) and dose is supported by practice guidelines or peer-reviewed literature; prescriber must submit supporting evidence
Dose limits
- Dose does not exceed 1.5 mg/m2 per day (IV) for 5 consecutive days every 21 days1.5 mg/m2/day IV x5 q21 days
  Maximum IV single dose = 4 mg/dose per dosing section
- Or dose is supported by practice guidelines or peer-reviewed literature for relevant off-label use (prescriber must submit evidence)
Generic-first injection: For Hycamtin injection requests, member must use generic topotecan, unless contraindicated or clinically significant adverse effects are experienced

B. Small cell lung cancer (must meet all)

Diagnosis of small cell lung cancer
Prescribed by or in consultation with an oncologist
Age > 18 years
Member has received prior chemotherapy (see Appendix B)
Generic-first: For Hycamtin requests, member must use generic topotecan if available, unless contraindicated or clinically significant adverse effects are experienced
Dose limits (select a or b)

C. Cervical cancer (must meet all)

Diagnosis of cervical cancer
Request is for topotecan for injection
Prescribed by or in consultation with an oncologist
Age > 18 years
Permitted regimens
- In combination with cisplatin or paclitaxel
- As a single agent as second-line or subsequent therapy

D. Off-label NCCN Compendium Recommended Indications (must meet all)

Request is for topotecan for injection and diagnosis is one of the following per NCCN compendium: Ewing sarcoma (as second-line with cyclophosphamide), osteosarcoma (as second-line with cyclophosphamide), non-pleomorphic rhabdomyosarcoma (single agent or with cyclophosphamide), neuroblastoma (as induction with cyclophosphamide), endometrial carcinoma (as second-line or subsequent therapy, single agent), pediatric medulloblastoma (in combination with temozolomide), leptomeningeal metastases (route = intrathecal), Merkel cell carcinoma (progressed on checkpoint immunotherapy), vaginal cancer (single agent or with paclitaxel or cisplatin)
Prescribed by or in consultation with an oncologist
Age ≥ 18 years for all indications except pediatric medulloblastoma (pediatric age allowed per NCCN recommendation)
Generic-first injection: For Hycamtin requests, member must use generic topotecan if available, unless contraindicated or clinically significant adverse effects are experienced

E. Other diagnoses/indications (must meet 1 or 2)

Formulary on/PDL: If drug is on formulary/PDL: follow no coverage criteria policy for relevant line of business (CP.CPA.190 for commercial, HIM.PA.33 for marketplace, CP.PMN.255 for Medicaid)
Formulary off: If drug NOT on formulary/PDL: follow non-formulary policy for relevant line of business (CP.CPA.190 for commercial, HIM.PA.103 for marketplace, CP.PMN.16 for Medicaid)
Off-label policy: If use not listed in section III and criterion above does not apply, refer to off-label use policy for relevant line of business (CP.CPA.09 commercial, HIM.PA.154 marketplace, CP.PMN.53 Medicaid)

Continuation Therapy Criteria

Continuation (Renewal) Therapy

Criteria to authorize continued therapy (must meet all):

Continued therapy (must meet all)

Member is currently receiving medication via Centene benefit; documentation supports member has received this medication for at least 30 days≥ 30 days on therapy
Member is responding positively to therapy
Generic-first: For Hycamtin requests, member must use generic topotecan if available, unless contraindicated or clinically significant adverse effects are experienced
Dose increase requirements

Diagnoses / Indications Not Authorized

Diagnoses/Indications Not Authorized

Not authorized diagnoses/indications: Non-FDA approved indications which are not addressed in this policy are not covered unless sufficient documentation of efficacy and safety is provided per off-label use policies or evidence of coverage documents

Refer to CP.CPA.09 (commercial), HIM.PA.154 (marketplace), CP.PMN.53 (Medicaid)

Applicable Codes

Pharmacy J-codesHCPCS

J8705	Topotecan, oral, 0.25 mg
J9351	Injection, topotecan, 0.1 mg

Provider Actions

Prior Authorization

Oncologist involvement and PA requirement

Requests for topotecan (Hycamtin) must be prescribed by an oncologist or written in consultation with an oncologist. For off-label uses or dosing outside standard FDA regimens, the prescriber must submit supporting evidence and justification with the prior authorization request.

Documentation Required

Support clinical criteria

Provider must submit documentation such as office chart notes, laboratory results, and other clinical information to support that the member meets all approval criteria.

Clinical Evidence & References

References include Hycamtin capsule and injection prescribing information (2018, 2019) and the NCCN Drugs & Biologics Compendium and disease-specific NCCN guidelines (2024-2025). The policy aligns coverage to FDA labels and NCCN compendium recommendations and redirects to generic topotecan when available.

Background

Background: Topotecan (Hycamtin) is a topoisomerase inhibitor. FDA-approved indications include relapsed small cell lung cancer (capsules), metastatic ovarian cancer (injection) after progression following prior chemotherapy, and use in combination with cisplatin for advanced/recurrent cervical cancer. The policy aligns coverage to FDA labels and NCCN compendium recommendations and includes a generic-first redirection to generic topotecan for injection when available.

Revision History

05.25revisedLatest

2Q 2025 annual review: added pediatric medulloblastoma to off-label NCCN recommendations and updated vaginal cancer criteria; references reviewed and updated. is_material: true

05.24revised

05.22revised

Policy Summary

PayerHealth Net

PolicyClinical Policy: Topotecan (Hycamtin)

Policy CodePolicy CP PHAR.64

Change TypeAnnual reviews and revisions (2021-2025)

Effective Date06.01.11

Next Review Date

Key ActionProvider must submit documentation (office chart notes, lab results, other clinical information) supporting that member has met all approval criteria.

SourceLink

On This Page

Capsule: 2.3 mg/m2 orally once daily for 5 consecutive days every 21 days

2.3 mg/m2/day orally x5 q21 days

Injection: 1.5 mg/m2 per day IV for 5 consecutive days every 21 days1.5 mg/m2/day IV x5 q21 days

Maximum IV single dose = 4 mg/dose

Dose is supported by practice guidelines or peer-reviewed literature for the relevant off-label use (prescriber must submit supporting evidence)

Generic-first injection: For Hycamtin injection requests, member must use generic topotecan, unless contraindicated or clinically significant adverse effects are experienced

Dose limits

Dose does not exceed 0.75 mg/m2 per day IV on days 1-3 every 21 days0.75 mg/m2/day IV days 1-3 q21 days
Maximum IV single dose = 4 mg/dose
Dose is supported by practice guidelines or peer-reviewed literature for the relevant off-label use (prescriber must submit supporting evidence)

Dose is within FDA maximum limit for any FDA approved indication or is supported by practice guidelines or peer-reviewed literature for the relevant off-label use (prescriber must submit supporting evidence)

1.5 mg/m2/day IV x5 q21 days or guideline-supported

Maximum IV single dose = 4 mg/dose

New dose does not exceed established dose limits: Ovarian cancer IV 1.5 mg/m2/day for 5 consecutive days every 21 days; Small cell lung cancer oral 2.3 mg/m2/day for 5 days every 21 days or IV 1.5 mg/m2/day for 5 days every 21 days; Cervical cancer IV 0.75 mg/m2/day on days 1-3 every 21 days1.5 mg/m2/day IV x5 q21; 2.3 mg/m2/day PO x5 q21; 0.75 mg/m2/day IV days1-3 q21

Maximum IV single dose = 4 mg/dose

Or new dose is supported by practice guidelines or peer-reviewed literature for the relevant off-label use (prescriber must submit supporting evidence)

Billing Rule

Generic-first for injection

For Hycamtin injection requests, the member must use generic topotecan if available unless the generic is contraindicated or clinically significant adverse effects are experienced. Document any contraindication or adverse reaction in the medical record.

Prior Authorization

Dose increase evidence

If requesting a dose increase, the provider must document that the new dose is within the policy’s specified dose limits or submit practice guidelines or peer‑reviewed literature supporting the requested dose.

05.21clarified

2Q 2021 annual review: updated reference for HIM off-label use policy to HIM.PA.154 and reviewed references; no significant coverage changes. is_material: false