CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Wilson's disease pharmacologic treatment authorization

Policy governing prior authorization and continuation criteria for pharmacologic treatment of Wilson's disease for Neighborhood Health Plan of Rhode Island Medicaid members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyWilson's disease pharmacologic treatment authorization

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJul 1, 2024

Next Review DateN/A

Key ActionObtain prior authorization for up to 6 months when diagnostic and prescribing criteria are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsauthorization period

Penicillamine 250 mgquantity-limited product

8/dayquantity limit

Coverage Criteria for Wilson's Disease Pharmacologic Treatment

inv-01: Initial Therapy

Authorization for 6 months may be granted when ALL of the following are met:

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Initial Approval Criteria: 1) Documented diagnosis of Wilson's disease confirmed by genetic testing showing biallelic pathogenic ATP7B mutations OR confirmation of at least two of the following clinical features: presence of Kayser-Fleischer rings; compatible liver biopsy (liver copper >250 mcg/g); serum ceruloplasmin <20 mg/dL; 24-hour urinary copper >40 mcg/24 hours; 2) Patient has experienced an inadequate response, intolerance, or contraindication to zinc therapy, or zinc would not be an appropriate therapy; 3) Medication is prescribed by, or in consultation with, a gastroenterologist, hepatologist, or liver transplant physician.

From policy initial therapy section

inv-02: Continuation Therapy

Authorization for 6 months may be granted for continuation when ALL of the following are met:

Continuation Criteria: 1) Patient continues to meet initial approval criteria; 2) Documentation of a positive response to treatment as indicated by improvement in neurologic condition or improved liver findings.

From policy continuation section

This section summarizes the policy's coverage criteria for pharmacologic treatment of Wilson's disease. Authorization for treatment is time-limited to 6 months and is contingent on meeting the diagnostic and prescribing requirements specified below. Key requirements include documented confirmation of Wilson's disease, evidence regarding prior or contraindicated zinc therapy when applicable, and that the medication be prescribed by or in consultation with an appropriate specialist (gastroenterologist, hepatologist, or liver transplant physician).

Key Laboratory and Coding Values

inv-05: Serum ceruloplasmin

Serum ceruloplasmin threshold< 20 mg/dL

Role in diagnosisOne of the clinical features used to confirm Wilson's disease when genetic testing is not available

Measurement contextReported as serum ceruloplasmin level in mg/dL

inv-06: 24-hour urinary copper

24-hour urinary copper threshold> 40 mcg/24 hours

Role in diagnosisOne of the clinical features used to confirm Wilson's disease when genetic testing is not available

Measurement contextReported as total copper excreted in a 24-hour urine collection

Provider Requirements, Documentation, and Denial Triggers

Prior Authorization

Prior Authorization Required

Prior authorization required for up to 6 months for treatment of Wilson's disease. Authorization may be granted when diagnostic and prescribing criteria are met.

Authorization duration: up to 6 months
Medication must be prescribed by or in consultation with a gastroenterologist, hepatologist, or liver transplant physician

Step Therapy

Zinc Therapy Step

Requirement that patient has experienced inadequate response, intolerance, or contraindication to zinc therapy, or that zinc would not be an appropriate therapy. Zinc therapy is the preferred initial step before other agents unless contraindicated or not tolerated.

Step therapy: trial of zinc therapy required unless documented inadequate response, intolerance, or contraindication
If zinc is not appropriate, document clinical rationale

Documentation Required

Required Documentation

Documented confirmation of Wilson's disease is required. Acceptable documentation: genetic testing showing biallelic pathogenic ATP7B mutations OR documentation of at least two supporting clinical features. Include evidence of treatment response for continuation requests.

Diagnostic confirmation: biallelic pathogenic ATP7B mutations via genetic testing OR at least two of the following: Kayser-Fleischer rings; liver biopsy with compatible copper content; serum ceruloplasmin < 20 mg/dL; 24-hour urinary copper > 40 mcg/24 hours
Prescriber documentation: medication prescribed by or in consultation with a gastroenterologist, hepatologist, or liver transplant physician
For continuation: documentation of positive response (improved neurologic condition or improved liver findings)

Denial Risk

Denial Triggers / Common Documentation Deficiencies

Potential triggers for denial include lack of diagnostic confirmation, missing documentation of zinc trial or justification for foregoing zinc, and absence of appropriate prescriber or treatment response for continuation.

No genetic confirmation or fewer than two documented clinical features supporting Wilson's disease
No documentation of trial of zinc therapy and no documented rationale for why zinc is contraindicated or inappropriate
Prescriber is not a specialist and no documentation of specialist consultation
For continuation requests: no documentation of clinical improvement or ongoing benefit

Background

Wilson's disease is a genetic disorder of copper metabolism that requires lifelong pharmacologic management to reduce copper accumulation and prevent hepatic and neurologic injury. Diagnostic confirmation for policy purposes can be by genetic testing demonstrating biallelic pathogenic ATP7B mutations or by at least two characteristic clinical findings such as Kayser-Fleischer rings, compatible liver biopsy (liver copper >250 mcg/g), serum ceruloplasmin < 20 mg/dL, or 24-hour urinary copper > 40 mcg/24 hours. Treatment authorization under this policy requires documentation of diagnosis and appropriate specialist involvement, and consideration of zinc therapy response, intolerance, or contraindication when applicable.

Definitions and Diagnostic Confirmation

inv-12: Confirmed Wilson's disease

Diagnostic confirmation by geneticsGenetic testing showing biallelic pathogenic ATP7B mutations

Alternative clinical confirmationPresence of at least two clinical features (e.g., Kayser-Fleischer rings; compatible liver biopsy with liver copper >250 mcg/g; serum ceruloplasmin <20 mg/dL; 24-hour urinary copper >40 mcg/24 hours)

Required documentationDocumentation of either genetic confirmation or at least two supporting clinical features must be provided for authorization