Table 1.

Rare Disease Stakeholders - Opportunities to Address Equity

Stakeholders	Opportunities to Address Equity
Government funding agencies	• Critically review funding allocations across rare diseases for potential inequities in allocation.  • Invest in integrated approaches to research to understand variation in outcomes within and across rare diseases.
Federal and state policymakers	• Promote regulatory supports for rare disease therapy development and coverage for new therapeutics.  • Review targeted variants and cut-off levels used in NBS for racially and ethnically diverse communities.  • Invest in systemic supports for robust short- and long-term follow-up of positive NBS screening results.
Industry partners	• Focus philanthropic donations on diverse patient communities  • Invest in financial support and navigation programs for patients  • Engage with diverse patient partners at all stages of research  • Consider value-based payment arrangements for high-cost drugs
Researchers	• Engage with diverse patient partners at all stages of research  • Evaluate research protocols to reduce burden on patient participants
Healthcare systems	• Work with existing pediatric complex care providers to support families  • Develop parallel navigation programs for adults with rare diseases
Healthcare providers	• Develop educational materials for community providers around rare diseases, implicit bias, and care needs  • Adopt patient-centered models of care coordination and communication
Public and private payers	• Develop internal programs to improve care coordination and support navigation  • Explore pilot programs to fund complex care and coordination for rare disease patients
Patient and family groups	• Actively seek to diversify representation, leadership and decision-making within disease groups  • Consider the potential downstream benefits and drawbacks of various funding priorities for socioeconomically and phenotypically diverse members of the patient community