CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Cholbam (cholic acid) — Coverage Criteria

Policy governing authorization criteria for Cholbam (cholic acid) for treatment of bile acid synthesis disorders due to single enzyme defects and as adjunctive therapy for peroxisomal disorders (including Zellweger spectrum disorders) for Neighborhood Health Plan of Rhode Island members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyCholbam (cholic acid) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateApr 1, 2022

Next Review DateN/A

Key ActionObtain prior authorization and provide diagnostic confirmation and specialist involvement for initial 6-month approvals.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsinitial authorization period

12 monthsreauthorization period

2 indicationsFDA-approved indications listed

Medicaiddocument scope

Coverage Criteria for Cholbam (cholic acid)

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Indications

Indications: Cholbam is indicated for treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption. The safety and effectiveness for extrahepatic manifestations have not been established; all other indications are considered experimental/investigational and not medically necessary.

Initial therapy — SEDs

Authorization of 6 months may be granted for treatment of bile acid synthesis disorders due to single enzyme defects when ALL of the following are met:

SED initial criteria: 1. Diagnosis confirmed by mass spectrometry or other biochemical testing, genetic testing, or enzyme assay; 2. Medication prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist; 3. Documentation that the member has liver dysfunction (e.g., elevated transaminases, bilirubin, presence of cholestasis) at baseline.

Initial therapy — Peroxisomal disorders

Authorization of 6 months may be granted for adjunctive treatment of peroxisomal disorders when ALL of the following are met:

PD initial criteria: 1. Diagnosis confirmed by mass spectrometry or other biochemical testing or genetic testing; 2. Medication prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist; 3. Documentation that the member exhibits manifestations of liver disease.

Reauthorization — SEDs

Authorization of 12 months may be granted for continued treatment in members with documentation requesting reauthorization who have achieved and maintained improvement in liver function:

SED reauthorization criteria: Documentation of maintained improvement in liver function (reduced transaminases, reduced bilirubin, no evidence of cholestasis on liver biopsy).

Reauthorization — Peroxisomal disorders

Authorization of 12 months may be granted for continued adjunctive treatment in members with documentation requesting reauthorization who have achieved and maintained improvement in liver function:

PD reauthorization criteria: Documentation of maintained improvement in liver function (reduced transaminases, reduced bilirubin, no evidence of cholestasis on liver biopsy).

Use of Cholbam for extrahepatic manifestations of bile acid synthesis disorders or peroxisomal disorders is not supported by established safety or effectiveness data. The policy limits coverage to the FDA‑approved hepatic indications: treatment of bile acid synthesis disorders due to single enzyme defects and adjunctive treatment of peroxisomal disorders (including Zellweger spectrum disorders) in patients with liver‑related manifestations, steatorrhea, or complications from decreased fat‑soluble vitamin absorption.

Prior Authorization, Documentation, and Reauthorization

Prior Authorization

Prior Authorization Required — Initial (6 months)

Initial authorization (6 months) — Prior authorization required: initial authorizations may be granted for 6 months when all specified diagnostic, prescriber, and baseline criteria are met.

SEDs: Diagnosis confirmed by mass spectrometry or other biochemical testing, genetic testing, or enzyme assay; Prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist; Documentation of baseline liver dysfunction (elevated transaminases, bilirubin, or cholestasis).
PDs (including Zellweger spectrum): Diagnosis confirmed by mass spectrometry, other biochemical testing, or genetic testing; Prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist; Documentation that the member exhibits manifestations of liver disease.

Prior Authorization

Reauthorization — Continued Therapy (12 months)

Reauthorization (12 months) — Reauthorization may be granted for 12 months when documentation shows sustained clinical benefit.

SEDs: Authorization of 12 months may be granted for continued treatment when documentation requesting reauthorization shows the member has achieved and maintained improvement in liver function.
PDs: Authorization of 12 months may be granted for continued adjunctive treatment when documentation shows the member has achieved and maintained improvement in liver function (reduced transaminases, reduced bilirubin, no evidence of cholestasis on liver biopsy).

Note

Documentation Required

Required Documentation

Required documentation — For initial authorization provide diagnostic confirmation (mass spectrometry, other biochemical testing, genetic testing, or enzyme assay), documentation of baseline liver dysfunction or manifestations of liver disease as applicable, and evidence that the medication is prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist.

Diagnostic confirmation: mass spectrometry, other biochemical testing, genetic testing, or enzyme assay.
Prescriber: hepatologist, metabolic specialist, or gastroenterologist (prescribed by or in consultation).
Baseline findings: For SEDs — liver dysfunction (elevated transaminases, bilirubin, or cholestasis). For PDs — manifestations of liver disease.

Denial Risk

Not Medically Necessary / Experimental Indications

Non‑approved indications — All other indications outside FDA-approved indications and compendial uses are considered experimental/investigational and not medically necessary.

Key Definitions

Confirmed diagnosis

Accepted diagnostic methodsDiagnosis confirmed by mass spectrometry, other biochemical testing, genetic testing, or enzyme assay.

Prescriber requirementMedication prescribed by or in consultation with a hepatologist, metabolic specialist, or gastroenterologist.

Context of useRequired for FDA‑approved indications: bile acid synthesis disorders due to single enzyme defects and adjunctive therapy for peroxisomal disorders.

LimitationSafety and effectiveness for extrahepatic manifestations have not been established; other indications are experimental/investigational and not medically necessary.

Improvement in liver function

Criteria defining improvementReduced transaminases, reduced bilirubin, and no evidence of cholestasis on liver biopsy.

Use for reauthorizationDocumentation of maintained improvement in liver function is required for reauthorization of continued treatment (12 months).

Baseline vs follow‑upBaseline documentation must show liver dysfunction (e.g., elevated transaminases, bilirubin, presence of cholestasis); improvement is relative to baseline measures.

Background

Cholbam (cholic acid) is FDA‑approved for two indications: 1) treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and 2) adjunctive treatment of peroxisomal disorders (including Zellweger spectrum disorders) in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat‑soluble vitamin absorption. The approval includes a limitation of use noting that the safety and effectiveness for extrahepatic manifestations of these disorders have not been established.