By Counterforce Health Team in Orfadin (nitisinone) —

Get Orfadin (Nitisinone) Covered by UnitedHealthcare in California: Decision Tree & Appeal Guide 2025

Quick Answer: Do You Qualify for Orfadin Coverage?

Yes, if you have: Confirmed hereditary tyrosinemia type 1 (HT-1) diagnosis with elevated succinylacetone levels, prescribed by a metabolic specialist, and following dietary restrictions. Fastest path: Submit prior authorization via UnitedHealthcare Provider Portal with HT-1 lab results, specialist letter, and dietary plan. Start today: Gather your newborn screening results or genetic testing, recent succinylacetone levels, and current prescription. If denied, California's Independent Medical Review has strong success rates for medically necessary rare disease treatments.

Table of Contents

  1. How to Use This Guide
  2. Eligibility Decision Tree
  3. If You're Likely Eligible
  4. If You're Possibly Eligible
  5. If You're Not Yet Eligible
  6. If Your Request is Denied
  7. Appeals Playbook for California
  8. Common Denial Reasons & Solutions
  9. Costs & Patient Assistance
  10. FAQ

How to Use This Guide

This decision tree helps patients and clinicians navigate UnitedHealthcare's prior authorization process for Orfadin (nitisinone) in California. Work through each section in order—the eligibility triage determines your next steps, whether that's immediate submission, gathering additional evidence, or preparing for appeals.

Note: This guide covers commercial UnitedHealthcare plans and Medicare Advantage. Medicaid (Medi-Cal) managed by UnitedHealthcare may have different requirements.

Eligibility Decision Tree

Step 1: Confirmed HT-1 Diagnosis?

✅ YES - You have:

  • Positive newborn screening with elevated succinylacetone
  • Genetic testing showing FAH gene mutations
  • Clinical diagnosis by metabolic specialist
  • Elevated tyrosine metabolites in urine

→ Go to Step 2

❌ NO - You need:

→ Go to If You're Not Yet Eligible

Step 2: Specialist Involvement?

✅ YES - You have:

  • Prescription from metabolic specialist or pediatric geneticist
  • Regular monitoring plan established
  • Dietary management with registered dietitian

→ Go to Step 3

❌ NO - You need:

  • Referral to metabolic specialist
  • Comprehensive treatment plan
  • Dietary consultation

→ Go to If You're Possibly Eligible

Step 3: Current Monitoring & Management?

✅ YES - You have:

  • Recent succinylacetone levels
  • Liver function monitoring
  • Tyrosine/phenylalanine dietary restrictions
  • Regular follow-up appointments

→ Go to If You're Likely Eligible

❌ NO - You need:

  • Updated lab work
  • Dietary plan documentation
  • Monitoring schedule

→ Go to If You're Possibly Eligible

If You're Likely Eligible

Document Checklist

Required for Prior Authorization:

  • UnitedHealthcare member ID and policy information
  • Prescription from metabolic specialist
  • HT-1 diagnosis confirmation (lab results or genetic testing)
  • Recent succinylacetone levels (blood/urine)
  • Dietary management plan from registered dietitian
  • Liver function tests (AST, ALT, bilirubin)
  • Alpha-fetoprotein levels
  • Medical necessity letter from prescriber

Submission Path

1. Provider Portal Submission (Fastest)

2. Phone Submission

  • Call OptumRx: 888-397-8129
  • Have all documents ready for fax submission
  • Request expedited review if clinically urgent

3. Medical Necessity Letter Template

Your prescriber should include:

  • Patient's confirmed HT-1 diagnosis with supporting labs
  • Clinical rationale for Orfadin therapy
  • Dosing plan (typically 0.5 mg/kg twice daily)
  • Monitoring schedule for succinylacetone and liver function
  • Dietary management plan
  • Expected clinical outcomes
Clinician Corner: Reference FDA prescribing information for dosing guidelines and monitoring requirements. Include specific succinylacetone levels and cite that Orfadin is the only FDA-approved treatment for HT-1.

If You're Possibly Eligible

Tests to Request

From Your Metabolic Specialist:

  • Comprehensive metabolic panel including succinylacetone
  • Genetic testing for FAH gene mutations if not done
  • Liver function assessment
  • Growth and development evaluation (pediatric patients)

From Your Dietitian:

  • Formal dietary assessment
  • Tyrosine and phenylalanine restriction plan
  • Nutritional monitoring schedule

Timeline to Re-Apply

  • Gather evidence: 2-4 weeks
  • Schedule specialist visit: 1-2 weeks
  • Lab results: 1 week
  • Resubmit PA: Immediately after documentation complete

What to Track

Document all symptoms, dietary compliance, and any clinical changes. Take photos of any skin or eye irritation that might indicate tyrosine toxicity, as this supports the need for continued dietary restrictions alongside Orfadin.

If You're Not Yet Eligible

Alternatives to Discuss

Immediate Options:

  • Strict dietary management while pursuing diagnosis
  • Liver transplant evaluation if severe disease
  • Clinical trial participation (rare)

Diagnostic Pathway:

  • Newborn screening review if available
  • Comprehensive metabolic testing
  • Genetic counseling and testing
  • Specialist consultation

Preparing for Exception Requests

Even without complete HT-1 confirmation, you may request a formulary exception if:

  • Clinical presentation strongly suggests HT-1
  • Urgent need for treatment while awaiting test results
  • Prior liver complications consistent with tyrosinemia

Counterforce Health specializes in turning insurance denials into targeted, evidence-backed appeals. Their platform can help identify the specific denial basis and draft point-by-point rebuttals aligned to UnitedHealthcare's own coverage policies, pulling the right medical citations and weaving them into appeals that meet procedural requirements.

If Your Request is Denied

Understanding Your Denial Letter

UnitedHealthcare denial letters typically cite:

  • "Not medically necessary" - Need stronger clinical justification
  • "Experimental/investigational" - Need FDA approval documentation
  • "Step therapy required" - Need proof alternatives failed/contraindicated
  • "Quantity limits exceeded" - Need dosing justification

Immediate Next Steps

1. Request Peer-to-Peer Review

  • Call number on denial letter within 3 business days
  • Have prescriber available for clinical discussion
  • Success rate: ~40% of denials resolved at this level

2. Gather Additional Evidence

  • Updated lab results showing disease progression
  • Literature supporting medical necessity
  • Documentation of dietary compliance
  • Specialist consultation notes

Appeals Playbook for California

Level 1: Internal Appeal with UnitedHealthcare

Timeline: Must file within 180 days of denial Process:

  • Submit via member portal or mail to address on denial letter
  • Include updated medical records and specialist letter
  • Decision within 30 days (expedited: 72 hours if urgent)

Required Documents:

  • Completed appeal form
  • Original denial letter
  • Medical necessity documentation
  • Prescriber attestation

Level 2: California Independent Medical Review (IMR)

When to Use: After internal appeal denial or 30 days without response Success Rate: ~10-12% overturn rate for prescription medications Timeline: 30 days for standard, 3-7 days for expedited

How to File:

Required Documents:

  • Internal appeal denial letter
  • Complete medical records
  • Independent medical opinion supporting necessity
  • Dietary management documentation

Expedited Appeals

Qualify if:

  • Delay could seriously jeopardize health
  • Current HT-1 symptoms worsening
  • Risk of liver complications

Timeline: 72 hours for UnitedHealthcare, 3 days for California IMR

Common Denial Reasons & Solutions

Denial Reason Solution Documents Needed
No HT-1 diagnosis confirmed Submit genetic testing or newborn screening Lab results, genetic counselor report
Not medically necessary Stronger clinical justification Specialist letter, FDA labeling, disease progression
Experimental treatment Prove FDA approval FDA prescribing information
Dietary management not attempted Document dietary compliance Dietitian reports, food logs
Quantity limits exceeded Justify dosing based on weight/response Dosing calculations, monitoring results

Costs & Patient Assistance

Manufacturer Support

  • Sobi Cares: Patient assistance program
  • Copay assistance: May reduce out-of-pocket costs
  • Contact: Check Sobi website for current programs

Cost Estimates

  • 30-day supply (10 kg child): ~$12,500-$13,600
  • Annual cost: $150,000-$165,000
  • Most patients pay significantly less with insurance coverage

Additional Resources

  • California Rare Disease Network: State-specific support
  • National Organization for Rare Disorders (NORD): Patient assistance funds
  • HealthWell Foundation: Copay assistance for rare diseases

Frequently Asked Questions

How long does UnitedHealthcare prior authorization take? Standard: 5-7 business days. Expedited (urgent): 24-72 hours. Missing documentation can extend timelines.

What if Orfadin isn't on my formulary? Request a formulary exception with medical necessity documentation. UnitedHealthcare must cover medically necessary treatments even if non-formulary.

Can I get a short-term supply while waiting? Yes, most pharmacies can provide a 5-day emergency supply at full cost. Ask your prescriber about samples if available.

Does step therapy apply to Orfadin? Rarely for HT-1, as Orfadin is the only FDA-approved treatment. Document if dietary management alone has failed.

What if I move from another state to California? California's IMR process may be more favorable than your previous state. Gather all previous denial and appeal documentation.

How do I know if my plan is regulated by DMHC or CDI? Most HMOs and managed care plans are under DMHC. Check your member handbook or call the DMHC Help Center at 888-466-2219.

From our advocates: We've seen cases where initial denials for rare disease medications like Orfadin were overturned after providing comprehensive dietary management documentation alongside genetic testing results. The key is demonstrating that both pharmaceutical and dietary interventions are medically necessary components of HT-1 treatment. While outcomes vary, thorough documentation significantly improves approval chances.

Disclaimer: This information is for educational purposes only and does not constitute medical or legal advice. Always consult with your healthcare provider and insurance plan for specific coverage details. For additional help with insurance appeals in California, contact the DMHC Help Center at 888-466-2219.

Sources & Further Reading

Powered by Counterforce Health—AI that turns drug denials into evidence-based appeals patients and clinicians can submit today.