CurrentNeighborhood Health Plan of Rhode IslandPolicy 2089-A

Cystagon (cysteamine bitartrate) coverage for nephropathic cystinosis

Defines prior authorization, coverage criteria, documentation, prescriber specialty, and authorization durations for Cystagon (cysteamine bitartrate) for treatment of nephropathic cystinosis for members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCystagon (cysteamine bitartrate) coverage for nephropathic cystinosis

Policy CodePolicy 2089-A

Change TypeClarified indications

Effective Date

Next Review Date

Key ActionSubmit required documentation to initiate prior authorization: initial requests must include assay detecting increased leukocyte cystine concentration or genetic testing results; continuation requests must include lab results or chart notes documenting response to therapy.

POLICY UPDATE CHANGES

Indications limited to nephropathic cystinosis; all other indications considered experimental/investigational and not medically necessary.

1Covered Indications

All other indicationsNot covered (experimental/investigational)

Coverage Summary

Coverage stance: covered_with_criteria. Policy number: 2089-A. Subject: Cystagon (cysteamine bitartrate) coverage for nephropathic cystinosis. Scope summary: Defines prior authorization, coverage criteria, documentation, prescriber specialty, and authorization durations for Cystagon (cysteamine bitartrate) for treatment of nephropathic cystinosis for members of Neighborhood Health Plan of Rhode Island. Indications are limited to nephropathic cystinosis; all other indications are considered experimental/investigational and not medically necessary. Authorization duration: 12 months.

Initial Therapy Criteria

FDA‑approved Indication: Nephropathic cystinosis - Initial Authorization

Authorization of 12 months may be granted when ALL of the following criteria are met:

ALL of the following

Diagnosis of cystinosis confirmed by the presence of increased cystine concentration in leukocytes or by genetic testing
Member will not use Cystagon in combination with Procysbi

Continuation Therapy Criteria (Reauthorization)

Continuation of Therapy (Reauthorization)

Authorization of 12 months may be granted for continued treatment for members responding to therapy when ALL of the following are met:

ALL of the following

Indication is listed in the Coverage Criteria section (nephropathic cystinosis)
Member is responding to therapy demonstrated by lab results or chart notes documenting improvement, stabilization, or slowing of disease progression
Examples of response metrics: serum creatinine, calculated creatinine clearance, leukocyte cystine concentration, or maintained growth (height)

Unproven / Not Medically Necessary

Policy change/clarification: Indications limited to nephropathic cystinosis; all other indications considered experimental/investigational and not medically necessary. Policy language: authorization may be granted for 12 months when criteria are met. The policy excludes concomitant use with Procysbi; members must not use Cystagon in combination with Procysbi as referenced in the coverage criteria and documentation requirements.

Provider Actions & Requirements

Prior Authorization

Prior authorization required for initial and continuation requests

Submit required documentation to initiate prior authorization. For initial requests include an assay detecting increased leukine (leukocyte) cystine concentration or genetic testing results supporting the diagnosis. For continuation (reauthorization) requests include lab results or chart notes documenting a positive response to therapy (for example: improvement, stabilization, or slowing of disease progression as shown by serum creatinine, calculated creatinine clearance, leukocyte cystine concentration, or maintained growth).

Initial requests must include leukocyte cystine assay results or genetic test results.
Continuation requests must include lab results or chart notes documenting response to therapy (e.g., serum creatinine, creatinine clearance, leukocyte cystine concentration, or maintained growth).

Documentation Required

Prescriber specialty requirement

This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders.

Denial Risk

Concomitant therapy exclusion

Authorization will not be granted if the member will use Cystagon in combination with Procysbi.

Clinical Evidence & References

Definition — Nephropathic cystinosis: A lysosomal storage disorder characterized by cystine accumulation; diagnosis is confirmed by increased leukocyte cystine concentration or genetic testing. Background linkage: Cystagon is indicated for management of nephropathic cystinosis in children and adults per the FDA; the package insert is cited as evidence for the indication.

Cystagon [package insert], Mylan Pharmaceuticals Inc.; August 2021.

Background

Cystagon (cysteamine bitartrate) is indicated for management of nephropathic cystinosis in children and adults per FDA. The policy requires objective confirmation of diagnosis (increased leukocyte cystine concentration or genetic testing) for initial authorization and documentation of response (lab results or chart notes showing improvement, stabilization, or slowing of disease progression) for reauthorization. Concomitant use with Procysbi is excluded and will prevent authorization.