By Counterforce Health Team in Orfadin (nitisinone) —

Do You Qualify for Orfadin (Nitisinone) Coverage by Aetna CVS Health in California? Decision Tree & Next Steps

Answer Box: Quick Qualification Check

Most likely eligible: Confirmed hereditary tyrosinemia type 1 (HT-1) with elevated succinylacetone (>2 μmol/L) and supporting genetic testing. Fastest path: Submit Aetna's prior authorization form through your specialist with HT-1 diagnosis, lab results, and dietary management plan. First step today: Contact your metabolic specialist to gather documentation and confirm CVS Specialty Pharmacy enrollment. Appeals through California's DMHC Independent Medical Review have favorable outcomes for medically necessary rare disease treatments.

Table of Contents

  1. How to Use This Decision Tree
  2. Eligibility Triage: Do You Qualify?
  3. If "Likely Eligible": Document Checklist & Submission
  4. If "Possibly Eligible": Tests to Request
  5. If "Not Yet": Alternative Options
  6. If Denied: California Appeal Path
  7. Coverage Requirements at a Glance
  8. Common Denial Reasons & Solutions
  9. Frequently Asked Questions

How to Use This Decision Tree

This guide helps you determine if you qualify for Orfadin (nitisinone) coverage through Aetna CVS Health in California and provides your next steps based on your specific situation.

Start here: Answer the questions in the eligibility triage section below. Based on your answers, you'll be directed to the appropriate action plan—whether that's gathering documentation for immediate submission, requesting additional testing, exploring alternatives, or preparing for appeals.

Each path includes specific forms, deadlines, and contact information verified through official Aetna CVS Health policies and California insurance regulations.

Eligibility Triage: Do You Qualify?

Primary Qualification Questions

1. Do you have a confirmed HT-1 diagnosis?

  • ✅ Yes: Diagnosed by metabolic specialist with genetic testing → Likely Eligible
  • 🔍 Suspected: Abnormal newborn screening or symptoms but no genetic confirmation → Possibly Eligible
  • ❌ No: No HT-1 diagnosis or considering off-label use → Not Yet

2. Do you have elevated succinylacetone levels?

  • ✅ Yes: Blood or urine succinylacetone >2 μmol/L documented → Continue to question 3
  • 🔍 Unknown: Never tested or results pending → Possibly Eligible
  • ❌ Normal: Consistently normal levels → Not Yet

3. Are you following a low-tyrosine/phenylalanine diet?

  • ✅ Yes: Working with metabolic dietitian → Likely Eligible
  • 🔍 Starting: Diet plan in development → Possibly Eligible
  • ❌ No: No dietary management → May need dietary consultation first

4. Have you tried generic nitisinone (if required by your plan)?

  • ✅ Yes, failed/intolerant: Documented adverse effects or inadequate response → Likely Eligible
  • 🔍 Never tried: Plan may require step therapy → Check plan requirements
  • ❌ Stable on generic: May not qualify for brand Orfadin → Discuss with specialist

If "Likely Eligible": Document Checklist & Submission

You appear to meet Aetna CVS Health's medical necessity criteria for Orfadin. Here's your action plan:

Required Documentation Checklist

  • HT-1 diagnosis confirmation with FAH gene testing results (2 pathogenic variants)
  • Recent lab results showing elevated succinylacetone (>2 μmol/L)
  • Metabolic specialist consultation notes documenting diagnosis and treatment plan
  • Dietary management plan from registered dietitian specializing in metabolic disorders
  • Prior therapy documentation (if generic nitisinone was tried and failed)
  • Current liver function tests (AST, ALT, GGT, albumin, bilirubin)
  • Weight-based dosing rationale (typically 1 mg/kg twice daily)

Submission Path

Step 1: Contact CVS Specialty Pharmacy

  • Call: 1-866-814-5506 (Aetna Specialty Precertification)
  • Confirm enrollment and benefits verification
  • Request prior authorization packet

Step 2: Provider Submission

Step 3: Track Decision

  • Standard timeline: 72 hours for specialty medications
  • Expedited timeline: 24 hours if medically urgent
  • Follow up if no response within timeframe (auto-approval may apply)
Tip: Aetna cannot deny a request that wasn't responded to within their published timeframes. If you don't hear back within 72 hours for a standard request, contact member services to confirm auto-approval status.

If "Possibly Eligible": Tests to Request

You may qualify but need additional testing or documentation. Work with your healthcare team to obtain:

Diagnostic Tests to Request

Immediate Priority:

  1. Succinylacetone measurement (blood and/or urine)
  2. FAH gene sequencing through NHS Genomic Medicine Service or equivalent
  3. Comprehensive metabolic panel including liver function tests
  4. Alpha-fetoprotein (AFP) level

Supporting Tests:

  • Plasma amino acid analysis (elevated tyrosine)
  • Urine organic acids (succinylacetone, hydroxyketoheptanoate)
  • Coagulation studies (PT/INR, PTT)

Timeline to Re-apply

  • Test results typically available: 1-2 weeks for biochemical tests, 2-4 weeks for genetic testing
  • Resubmit application: Once HT-1 diagnosis is confirmed with supporting lab values
  • Track progress: Schedule follow-up with metabolic specialist to review results

What to Monitor

Keep detailed records of:

  • All lab values and dates
  • Symptoms or clinical changes
  • Dietary adherence and challenges
  • Communication with healthcare providers

If "Not Yet": Alternative Options

If you don't currently meet criteria, explore these pathways:

Immediate Alternatives

Generic Nitisinone:

  • May be preferred by Aetna CVS Health as first-line therapy
  • Significantly lower cost than brand Orfadin
  • Same active ingredient and mechanism of action
  • Available from multiple manufacturers

Formulary Exception Request:

  • If Orfadin is non-formulary, request exception with clinical justification
  • Document why generic alternatives are inappropriate
  • Include specialist letter explaining medical necessity for brand formulation

Preparing for Exception Requests

Build Your Case:

  1. Document treatment history with other therapies and outcomes
  2. Gather specialist support from metabolic disease experts
  3. Research clinical guidelines supporting Orfadin use in your situation
  4. Consider second opinion from another metabolic specialist if appropriate

Off-Label Considerations:

  • Aetna evaluates off-label use based on medical literature and compendia
  • Requires stronger evidence of medical necessity
  • May need peer-to-peer review with medical director

If Denied: California Appeal Path

California offers robust appeal rights with high success rates for medically necessary treatments. Choose your path:

Level 1: Internal Appeal with Aetna CVS Health

Timeline: File within 180 days of denial Decision: 30 days for standard, 72 hours for expedited Process: Submit appeal form with additional clinical documentation

Level 2: California DMHC Independent Medical Review

When to use: After internal appeal denial or immediately for experimental/investigational denials Timeline: File within 180 days of final denial Decision: 30-45 days standard, 7 days expedited Cost: Free to patients Success rate: Favorable for rare disease treatments with strong medical evidence

To file IMR:

  • Contact DMHC Help Center: 888-466-2219
  • Submit application online at healthhelp.ca.gov
  • Include all denial letters and complete medical records
Note: California's IMR process is binding on the insurer. If the independent medical review overturns the denial, Aetna CVS Health must authorize coverage.

When to Request Expedited Review

Request expedited appeals if:

  • Your health is in immediate danger without treatment
  • Delay could cause permanent disability or loss of function
  • Current symptoms are rapidly worsening

Coverage Requirements at a Glance

Requirement Details Documentation Needed Source
Prior Authorization Required for all strengths PA form + clinical documentation Aetna PA List
Diagnosis Hereditary tyrosinemia type 1 FAH gene testing + elevated succinylacetone FDA Label Requirements
Pharmacy Network CVS Specialty only Enrollment verification CVS Specialty: 1-866-814-5506
Step Therapy May require generic trial Documentation of failure/intolerance if applicable Plan-specific
Dietary Management Low tyrosine/phenylalanine diet Dietitian consultation notes Medical necessity criteria
Monitoring Plan Regular lab surveillance Succinylacetone, liver function tracking Treatment guidelines

Common Denial Reasons & Solutions

Denial Reason How to Overturn Required Documentation
"No HT-1 diagnosis" Submit genetic testing results FAH gene sequencing showing 2 pathogenic variants
"Lack of dietary management" Provide dietitian consultation Metabolic dietitian treatment plan and progress notes
"Generic available" Document brand medical necessity Specialist letter explaining why generic inappropriate
"Not medically necessary" Strengthen clinical justification Updated specialist letter with guideline citations
"Experimental/investigational" Challenge with FDA approval status FDA label and approval documentation

Frequently Asked Questions

How long does Aetna CVS Health prior authorization take in California? Standard requests are decided within 72 hours for specialty medications. Expedited requests for urgent situations are decided within 24 hours. If Aetna doesn't respond within these timeframes, the request may be automatically approved.

What if Orfadin is non-formulary on my plan? You can request a formulary exception with documentation that preferred alternatives are medically inappropriate. This requires specialist support explaining why the brand formulation is necessary over generic options.

Can I request an expedited appeal in California? Yes. California allows expedited appeals when your health is in immediate danger or when standard timelines could cause permanent harm. Expedited internal appeals are decided within 72 hours, and expedited DMHC reviews within 7 days.

Does step therapy apply if I've previously failed generic nitisinone? No. If you have documented failure, intolerance, or contraindication to generic nitisinone, you can typically bypass step therapy requirements with proper documentation from your specialist.

What's the difference between Aetna CVS Health and CVS Caremark? CVS Caremark is Aetna's pharmacy benefit manager that handles prescription drug coverage and prior authorizations. CVS Specialty specifically manages specialty medications like Orfadin.

How much does Orfadin cost without insurance? Costs vary by strength and formulation. Contact Sobi (the manufacturer) for current pricing or explore patient assistance programs. Manufacturer support may be available for eligible patients.

Can I continue current treatment during appeals? California's "Aid Paid Pending" protection may allow you to continue receiving services during the appeal process if you file within 10 days of the denial and are currently receiving the treatment.

When navigating complex prior authorizations and appeals for specialty medications like Orfadin, having expert support can make the difference between approval and denial. Counterforce Health helps patients, clinicians, and specialty pharmacies turn insurance denials into targeted, evidence-backed appeals by analyzing denial letters, plan policies, and clinical notes to craft point-by-point rebuttals aligned with each payer's specific requirements.

From our advocates: We've seen many HT-1 patients initially denied for Orfadin due to incomplete documentation of their dietary management plan. In one recent case, adding detailed dietitian notes and succinylacetone monitoring logs to the appeal led to approval within the standard review period. The key was demonstrating comprehensive metabolic care, not just the diagnosis alone.

Sources & Further Reading

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Coverage decisions depend on individual circumstances and plan details. Always consult with your healthcare provider and insurance plan for personalized guidance. For assistance with appeals and coverage issues, contact the California DMHC Help Center at 888-466-2219 or visit healthhelp.ca.gov.

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