Abstract
Abstract
Background
ANCA-associated vasculitis, encompassing Granulomatosis with Polyangiitis (GPA), Microscopic Polyangiitis (MPA), and Eosinophilic Granulomatosis with Polyangiitis (EGPA), represents a group of rare, systemic autoimmune diseases associated with significant morbidity and mortality if not treated promptly. Despite the availability of clinical guidelines and lengthy patient resources, no concise, patient-accessible navigational tool existed to guide newly diagnosed individuals or people still navigating their care through immediate and critical decisions. Delays between symptom onset, diagnosis, and initiation of appropriate specialty care remain a persistent and preventable source of organ damage in this population.
[1, 2]
Objective
This paper describes the development, rationale, and program framework of the
Decision Tree: Best Practice Suggestions
, a free patient navigation tool developed by the Eosinophilic & Rare Disease Cooperative (ERDC) in collaboration with rheumatologists, pulmonologists, nephrologists, pharmacists, insurance specialists, other nonprofit organizations, and people living with ANCA vasculitis (patients and care partners). We examine the unmet need driving its development, the collaborative methodology used to create it, and the multi-component program designed to support its adoption and impact.
Methods
The Decision Tree was developed through a structured, multi-stakeholder consensus process involving clinical specialists across rheumatology, nephrology, and pulmonology; pharmacists and insurance billing specialists; primary care providers; nonprofit partner organizations; and patients and care partners with direct disease experience. Content domains were identified through a gap analysis of existing patient resources and community-level care barriers. The resulting tool was iteratively validated prior to launch to ensure actionability, clinical precision, applicability across disease subtypes and plain-language accessibility at or below an eighth grade reading level.
Results
The Decision Tree addresses nine important relevant care navigation domains: emergency and hospital care access; securing a maintenance provider; Center of Excellence consultation; securing key specialists; insurance and medication access; labs, tests, and monitoring; treatment options; quality-of-life assessment; and a glossary of terms. The tool was integrated into ERDC's Hospital Emergency Advocacy & Treatment (HEAT) Kit and will be supported by a multi-platform education program including livestream sessions, podcast content, and web-based resources. It is freely available through ERDC, RareDecisionTree.org, and partner organizations if they select to share it.
[3]
The Decision Tree is designed to meet patients’ needs throughout their journey, not simply upon diagnosis. The Decision Tree integrates action-oriented navigation steps with a plain-language glossary, empowering patients and care partners to navigate key decision points across their disease continuum.
Conclusions
Patient-facing navigation tools designed specifically for rare disease populations represent a scalable, low-cost strategy to reduce care delays, streamline care team assembly, and mitigate preventable organ damage. The Rare Disease Decision Tree model developed for ANCA-associated vasculitis provides an open-access, replicable framework to overcome complex diagnostic and therapeutic hurdles across high-acuity rare autoimmune conditions.