Challenge

Families encountered:

• Insurance roadblocks, including state-to-state Medicaid denials.
  
• International coordination hurdles for non-U.S. patients.
  
• Confusing, time-sensitive eligibility and documentation steps.
  
• Limited, inconsistent communication between families, providers, and researchers.

United4Rare’s Role

Kirsten acted as navigator and advocate, working directly with parents, clinicians, payers, and the research team to keep each family moving forward. She ensured requirements were clear, timelines were realistic, and decision-makers stayed engaged.

Key Actions

• Care navigation: Guided families through eligibility, enrollment steps, and document prep.
  
• Medicaid advocacy: Reversed two denials across states—including one case escalated to the Office of the Governor of Alaska—demonstrating that persistence and patient advocacy can overcome entrenched barriers.
  
• International coordination: Connected non-U.S. families with trial sponsors and aligned cross-border requirements.
  
• Research liaison: Maintained direct communication with the lead researcher so family questions were answered and patient voice informed operations.
  
• Trial readiness: Organized paperwork, insurance confirmations, and logistics to prevent avoidable delays at treatment start.

Impact

• First-in trial: Devyn became the first child enrolled, opening the door for others to follow.
  
• Access unlocked: Families previously denied—especially due to Medicaid issues—secured enrollment.
  
• System shift: United4Rare showed how focused patient advocacy can bridge families and research and influence state-level decision-making.

What’s Next

• Expand this navigation model to additional sites and families.
  
• Formalize payer escalation pathways for rare-disease trials.
  
• Create a reusable “trial readiness” toolkit to shorten time from referral to first dose.