By Counterforce Health Team in Orfadin (nitisinone) —

How to Get Orfadin (nitisinone) Covered by Aetna CVS Health in North Carolina: Complete PA Guide with Appeals Timeline

Answer Box: Getting Orfadin (nitisinone) Covered by Aetna CVS Health in North Carolina

Aetna CVS Health requires prior authorization for Orfadin (nitisinone) with approval contingent on confirmed hereditary tyrosinemia type 1 (HT-1) diagnosis, elevated succinylacetone levels, and documented dietary management. Standard decisions take 72 hours; expedited requests get 24-hour responses. If denied, you have 180 days to appeal internally, then can request external review through North Carolina's Smart NC program within 120 days.

First step today: Have your provider gather HT-1 confirmation (genetic testing or elevated succinylacetone >2 μmol/L), recent liver function tests, and dietary restriction documentation, then submit PA via Aetna provider portal or fax to 1-866-249-6155.

Table of Contents

  1. Coverage Basics
  2. Prior Authorization Process
  3. Decision Timelines
  4. Medical Necessity Criteria
  5. Costs and Specialty Pharmacy
  6. Denials and Appeals
  7. North Carolina External Review
  8. Renewal Requirements
  9. Common Questions (FAQ)
  10. Sources and Further Reading

Coverage Basics

Is Orfadin (nitisinone) Covered by Aetna CVS Health?

Yes, but with restrictions. Orfadin is classified as a specialty tier, non-preferred medication across Aetna plans and requires prior authorization for all members. The drug is dispensed exclusively through CVS Specialty Pharmacy due to its limited distribution status.

Which Plans Cover Orfadin?

  • Commercial Aetna plans (individual, employer-sponsored)
  • Aetna Medicare Advantage (available in select North Carolina counties including Durham, Wake, Orange)
  • North Carolina State Health Plan (for state employees with Aetna coverage)

Medicaid plans in North Carolina may have different processes and should be verified separately.

Prior Authorization Process

Step-by-Step: Fastest Path to Approval

  1. Gather Required Documentation (Patient/Family)
    • Insurance card and member ID
    • Complete medical records from metabolic specialist
    • Recent lab results showing elevated succinylacetone
    • Dietary management plan from registered dietitian
  2. Provider Submits Prior Authorization (Healthcare Provider)
    • Submit via Aetna provider portal (Availity)
    • Alternative: Fax to specialty PA line at 1-866-249-6155
    • Include all supporting documentation and medical necessity letter
  3. CVS Specialty Enrollment (Concurrent)
    • Provider or patient calls CVS Specialty at 1-866-814-5506
    • Verify benefits and begin enrollment process
    • CVS may assist with PA submission
  4. Decision and Dispensing (72 hours standard)
    • Aetna responds within required timeframe
    • If approved, CVS Specialty contacts patient within 24-48 hours
    • First shipment arrives 3-5 business days after approval

Decision Timelines

Request Type Decision Timeline Auto-Approval if No Response
Standard PA 72 hours Yes, automatically approved
Expedited/Urgent 24 hours Yes, automatically approved
Appeal (Internal) 72 hours standard, 24 hours expedited N/A
Tip: Request expedited review if delays could jeopardize liver function or if patient is currently on Orfadin and facing interruption.

Medical Necessity Criteria

Required Documentation for Approval

Confirmed HT-1 Diagnosis:

  • Genetic testing showing two pathogenic FAH gene variants, OR
  • Metabolic specialist confirmation with ICD-10 code E70.21
  • Elevated succinylacetone levels exceeding 2 μmol/L in blood or urine

Clinical Support:

  • Recent liver function tests (AST, ALT, GGT, albumin, bilirubin)
  • Age-appropriate dosing plan (typically 1 mg/kg twice daily starting dose)
  • Active dietary restriction of tyrosine and phenylalanine with registered dietitian support
  • Documented monitoring plan for succinylacetone levels

Weight-Based Dosing Limits:

  • Pediatric patients: 0.5-1 mg/kg/day initially, maximum 2 mg/kg/day
  • Adult patients: Same weight-based limits apply
  • Quantity limits enforced based on patient weight and FDA-approved dosing

Costs and Specialty Pharmacy

CVS Specialty Pharmacy Process

Once PA is approved, Orfadin is dispensed exclusively through CVS Specialty:

  • Enrollment: CVS contacts patient to verify insurance and copay assistance
  • Shipping: 1-month supply shipped to home or clinic
  • Refills: Automatic coordination with prescriber for refills
  • Monitoring: Annual renewal requires adherence >85% and continued medical necessity

Copay Assistance Options

  • Insurance-specific programs: CVS Specialty verifies and enrolls during setup
  • Financial hardship waivers: Available for qualifying patients
  • Manufacturer support: Contact CVS Specialty for current programs

For complex coverage situations, Counterforce Health helps patients navigate prior authorization requirements and appeal denials by creating targeted, evidence-backed submissions that align with Aetna's specific criteria.

Denials and Appeals

Common Denial Reasons and Solutions

Denial Reason How to Overturn Required Documentation
No HT-1 diagnosis Submit genetic testing or specialist confirmation FAH gene variants or metabolic specialist letter
Lack of dietary management Provide dietitian documentation Registered dietitian treatment plan
Off-label use Clarify FDA-approved indication FDA labeling for HT-1 indication
Non-formulary status Request formulary exception Medical necessity letter explaining why alternatives inadequate

Internal Appeal Process

If your PA is denied:

  1. File appeal within 180 days of denial notice
  2. Submit additional documentation addressing specific denial reasons
  3. Request peer-to-peer review if clinical judgment is questioned
  4. Consider expedited appeal if urgent medical need exists

Appeal submission methods:

  • Aetna member portal
  • Fax: Check denial letter for specific appeals fax number
  • Mail: Include all supporting documentation and member ID

North Carolina External Review

Smart NC Program Overview

North Carolina offers robust external review rights through Smart NC, administered by the Department of Insurance. This applies to fully insured Aetna plans (not self-funded employer plans unless they opt in).

When You Can Use External Review

  • After exhausting internal appeals with Aetna
  • Medical necessity denials for treatments over $500
  • Experimental/investigational determinations
  • Within 120 days of final internal denial

How to Request External Review

  1. Contact Smart NC: Call 1-855-408-1212 or submit request online
  2. Free service: No cost to patients; Smart NC provides guidance
  3. Timeline: Standard reviews decided within 45 days; expedited within 72 hours
  4. Binding decision: If approved, Aetna must provide coverage within 3 business days
Note: Smart NC staff can help you complete the external review request and gather supporting documentation, though they cannot serve as your official representative.

Success Factors for External Review

  • Well-documented medical necessity with specialist support
  • Peer-reviewed studies supporting Orfadin use in HT-1
  • Evidence of failed alternatives or contraindications
  • Clear treatment goals and monitoring plan

Renewal Requirements

Annual Reauthorization

Orfadin requires yearly renewal with documentation of:

  • Continued medical necessity for HT-1 treatment
  • Adherence >85% to prescribed therapy
  • Updated lab results showing biochemical response
  • Ongoing dietary management with professional support

Monitoring Requirements

  • Succinylacetone levels: Regular monitoring to assess treatment response
  • Liver function tests: Periodic evaluation of hepatic status
  • Plasma tyrosine: Monitoring to prevent toxicity from dietary restrictions
  • Growth and development: In pediatric patients

Common Questions (FAQ)

How long does Aetna CVS Health PA take in North Carolina? Standard requests: 72 hours. Expedited requests: 24 hours. If Aetna doesn't respond within these timeframes, the request is automatically approved.

What if Orfadin is non-formulary on my plan? Request a formulary exception with medical necessity documentation. Emphasize FDA approval for HT-1 and lack of therapeutic alternatives.

Can I request an expedited appeal? Yes, if delays would seriously jeopardize your health or ability to regain function. Your physician must certify the urgent medical need.

Does step therapy apply to Orfadin? Some plans may require trial of generic nitisinone first. Document any failures, intolerances, or contraindications to generic formulations.

What happens if I move within North Carolina? Coverage should continue if you remain on the same Aetna plan. Notify CVS Specialty of address changes for shipping.

Can I appeal if I disagree with quantity limits? Yes, if your physician documents medical necessity for higher doses within FDA limits (maximum 2 mg/kg/day for HT-1).

From Our Advocates

We've seen many Orfadin denials overturned when families work closely with their metabolic specialist to document the progressive nature of HT-1 and the critical role of early intervention. The key is presenting a comprehensive picture that includes genetic confirmation, biochemical markers, and a clear monitoring plan. This composite guidance reflects successful strategies across multiple cases, though individual outcomes vary.

Sources and Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical advice. Always consult with your healthcare provider and insurance plan for specific coverage decisions. Coverage policies and requirements may change; verify current information with official sources.

When navigating complex insurance requirements for rare disease medications like Orfadin, specialized support can make the difference between approval and denial. Counterforce Health transforms insurance denials into targeted appeals by analyzing denial letters, plan policies, and clinical notes to create evidence-backed submissions that speak directly to payer requirements.

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