By Counterforce Health Team in Orfadin (nitisinone) —

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

Answer Box: Your Path to Orfadin Coverage

Aetna CVS Health requires prior authorization for Orfadin (nitisinone) in New York, with approval based on confirmed hereditary tyrosinemia type 1 (HT-1) diagnosis, elevated succinylacetone levels (>2 μmol/L), and dietary management plan. Standard decisions take 72 hours; expedited cases 24 hours. If denied, New York's external appeal process through the Department of Financial Services offers binding review within 72 hours for formulary denials.

First step today: Call CVS Specialty at 1-866-814-5506 to verify benefits and request the precertification packet while your metabolic specialist gathers required documentation.

Table of Contents

  1. How to Use This Decision Tree
  2. Eligibility Triage: Do You Qualify?
  3. If "Likely Eligible" - Your Action Plan
  4. If "Possibly Eligible" - Tests to Request
  5. If "Not Yet" - Alternative Paths
  6. If Denied - New York Appeal Options
  7. Coverage at a Glance
  8. Common Denial Reasons & Solutions
  9. Frequently Asked Questions

How to Use This Decision Tree

This guide helps patients with hereditary tyrosinemia type 1 (HT-1) navigate Aetna CVS Health's prior authorization process for Orfadin in New York. Work through each section with your metabolic specialist or treating physician to determine your eligibility and next steps.

Before you start, gather:

  • Insurance card with member/group ID
  • Recent lab results (succinylacetone, liver function tests)
  • Genetic testing results (FAH gene)
  • Documentation of dietary management
  • Records of any prior nitisinone therapy

Eligibility Triage: Do You Qualify?

✅ Likely Eligible if you have:

  • Confirmed HT-1 diagnosis via genetic testing (2 pathogenic FAH gene variants) OR metabolic specialist confirmation
  • Elevated succinylacetone in blood/urine (>2 μmol/L)
  • Active dietary restriction of tyrosine and phenylalanine with registered dietitian support
  • Recent liver function tests showing need for treatment
  • Age-appropriate dosing plan (typically 1 mg/kg twice daily starting dose)

⚠️ Possibly Eligible if you have:

  • HT-1 diagnosis but missing recent succinylacetone levels
  • Dietary management but no formal dietitian documentation
  • Prior generic nitisinone trial without documented failure/intolerance
  • Incomplete genetic testing results

❌ Not Yet Eligible if you have:

  • No confirmed HT-1 diagnosis
  • Normal succinylacetone levels
  • No dietary management plan
  • Seeking coverage for off-label uses

If "Likely Eligible" - Your Action Plan

Document Checklist

Ensure your metabolic specialist has:

Required Document Specific Details Source
HT-1 Diagnosis FAH genetic testing (2 pathogenic variants) + specialist notes Aetna PA Criteria
Lab Results Succinylacetone >2 μmol/L in blood/urine; liver function tests (AST, ALT, GGT, albumin, bilirubin) FDA Prescribing Information
Treatment Plan Weight-based dosing rationale, monitoring plan for succinylacetone levels EMA Product Information
Dietary Management Registered dietitian notes on tyrosine/phenylalanine restriction Aetna PA Requirements

Submission Path (3-Step Process)

Step 1: Enroll with CVS Specialty

  • Call 1-866-814-5506 for Aetna members
  • Verify benefits and request precertification packet
  • Confirm dispensing requirements

Step 2: Provider Submits PA

  • Complete 2024 Aetna PA form via Availity provider portal
  • Alternative: Fax to specialty PA line at 1-866-249-6155
  • Attach all required documentation

Step 3: Track Decision

  • Standard timeline: 72 hours
  • Expedited (if urgent): 24 hours
  • No response within timeline = automatic approval
Tip: Request expedited review if delaying treatment could worsen liver function or cause clinical deterioration.

If "Possibly Eligible" - Tests to Request

Missing HT-1 Confirmation

  • Request FAH gene sequencing if not completed
  • Obtain metabolic specialist consultation for clinical diagnosis
  • Timeline: 2-4 weeks for genetic results

Incomplete Lab Work

  • Order succinylacetone testing (blood and/or urine)
  • Update liver function panel (AST, ALT, GGT, albumin, bilirubin, PT/INR)
  • Consider alpha-fetoprotein if elevated previously
  • Timeline: 3-5 business days for results

Dietary Documentation Gaps

  • Schedule appointment with registered dietitian
  • Document current tyrosine/phenylalanine intake
  • Establish formal dietary management plan
  • Timeline: 1-2 weeks to complete

If "Not Yet" - Alternative Paths

For Unconfirmed HT-1 Diagnosis

  • Discuss newborn screening results with pediatric metabolic specialist
  • Consider genetic counseling for family history assessment
  • Explore differential diagnoses if clinical picture unclear

For Off-Label Requests

  • Prepare formulary exception request with:
    • Peer-reviewed literature supporting off-label use
    • Documentation of standard therapy failures
    • Specialist letter explaining medical necessity
  • Consider clinical trial options if available

Cost Considerations

  • Generic nitisinone (Nityr) may be preferred formulary option
  • Sobi patient assistance programs for eligible patients
  • State pharmaceutical assistance programs in New York

If Denied - New York Appeal Options

New York offers robust appeal protections through the Department of Financial Services (DFS) external review process.

Internal Appeal (Required First Step)

Timeline Process Required Documents
File within 180 days of denial Submit appeal form via Aetna member portal or mail Original denial letter, new clinical evidence, specialist letter
Decision in 30 days (72 hours expedited) Aetna medical review team evaluates Consider peer-to-peer review with medical director

New York External Appeal

When to File: After receiving Aetna's final internal denial (or waiving second-level internal appeal)

Timeline: 4 months from final adverse determination to file

Process:

  1. Download NY External Appeal Application
  2. Complete physician attestation forms
  3. Submit via fax: (800) 332-2729 or mail to: NY DFS, 99 Washington Avenue, Box 177, Albany, NY 12210

Decision Timelines:

  • Standard: 30 days
  • Non-formulary drug: 72 hours
  • Expedited medical necessity: 24 hours

Cost: Maximum $25 (waived for Medicaid enrollees or financial hardship)

Note: External appeal decisions are binding on Aetna and have strong success rates for rare disease medications with proper documentation.

Getting Help with Appeals

Community Health Advocates: Free counseling on insurance denials

  • Helpline: 888-614-5400
  • Assists New Yorkers with filing appeals and understanding rights

DFS Consumer Assistance:

Coverage at a Glance

Requirement What It Means Where to Find It
Prior Authorization Required for all Aetna plans 2024 Precertification List
Formulary Tier Specialty tier, non-preferred Aetna Drug Guides
Step Therapy May require generic nitisinone trial Plan-specific; exceptions available
Dispensing CVS Specialty Pharmacy only 1-866-814-5506
Age Limits None for HT-1 indication FDA-approved for all ages

Common Denial Reasons & Solutions

Denial Reason How to Overturn Required Documentation
"No HT-1 diagnosis confirmed" Submit genetic testing results FAH gene sequencing showing 2 pathogenic variants
"Missing biochemical evidence" Provide recent succinylacetone levels Lab results showing >2 μmol/L in blood/urine
"Lack of dietary management" Document restriction plan Registered dietitian notes on tyrosine/phenylalanine limits
"Step therapy not completed" Request exception or document failure Generic nitisinone intolerance/inadequate response records
"Not medically necessary" Appeal with specialist support Metabolic specialist letter citing FDA indication

Frequently Asked Questions

How long does Aetna CVS Health PA take in New York? Standard decisions: 72 hours. Expedited (urgent medical need): 24 hours. No response within timeline equals automatic approval.

What if Orfadin is non-formulary on my plan? Request formulary exception with medical necessity documentation. New York external appeals for non-formulary drugs receive 72-hour decisions.

Can I request an expedited appeal? Yes, if delaying treatment would jeopardize your health. Document clinical urgency and request expedited review at both internal and external appeal levels.

Does step therapy apply if I've tried generics outside New York? Document prior therapy attempts with clinical notes showing failure, intolerance, or inadequate response. Out-of-state records are acceptable with proper documentation.

How much does Orfadin cost with Aetna coverage? Costs vary by plan. Specialty tier copays typically range from $40-100+ per month. Contact CVS Specialty at 1-866-814-5506 for specific benefit verification.

What happens if my external appeal is approved? Aetna must cover the medication and refund your appeal filing fee. Coverage continues for the prescribed duration with appropriate monitoring.

About Counterforce Health: Counterforce Health helps patients, clinicians, and specialty pharmacies turn insurance denials into successful, evidence-backed appeals. Our platform identifies denial reasons and drafts targeted rebuttals using payer-specific criteria, FDA labeling, and peer-reviewed studies to improve approval rates for specialty medications like Orfadin.

When navigating complex prior authorization requirements, having expert support can make the difference between approval and denial. Counterforce Health's specialized approach to appeals has helped countless patients access critical medications by addressing each payer's unique requirements and procedural standards.

Sources & Further Reading

Disclaimer: This guide provides general information about insurance coverage and is not medical advice. Coverage decisions depend on individual circumstances, plan benefits, and clinical factors. Consult your healthcare provider and insurance plan for specific guidance. For assistance with New York insurance issues, contact the Department of Financial Services at 1-800-400-8882.

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