Patient advocacy
Patient Advocacy at the Harriet Joyce Foundation: Championing Children with Rare Diseases
The Harriet Joyce Foundation is deeply committed to advancing equity and access for children affected by rare and complex pediatric diseases. Through strategic advocacy, hands-on support, and cross-sector partnerships, the Foundation works on multiple fronts to address the urgent needs of families navigating the challenges of rare disease care. Key areas of advocacy include:
01
Advocating for Surgical and Specialized Procedure Coverage
Children with inborn errors of metabolism require specialized medical formulas and dietary management to survive and thrive. The Foundation advocates directly with manufacturers of metabolic formulas to ensure consistent access, affordability, and distribution of life-sustaining nutritional products. It also works to address supply chain issues and regulatory barriers, particularly in countries where access is unreliable or non-existent
02
Supporting Cross-Border Medical Care and Care Management
When local health systems cannot meet the complex needs of a child with a rare disease, the Harriet Joyce Foundation facilitates medical travel to the United States for highly specialized treatment and care coordination. This includes logistical and financial support, care navigation, and integration with top-tier U.S. hospitals and multidisciplinary teams. The goal is not just urgent treatment, but the establishment of long-term care strategies families can continue back home.
03
Connecting Families to Clinical Trials and Pharmaceutical Research
Innovation in rare disease treatment depends on clinical trials, but access is often opaque or geographically limited. The Foundation assists families in identifying and navigating clinical trials—connecting them to pharmaceutical companies and research networks developing targeted therapies. This includes helping with eligibility screening, application processes, and ongoing engagement to ensure children are not left behind in the drug development landscape.
04
Insurance Advocacy:
Rare diseases often require high-cost, life-altering surgeries or interventions, which can be denied or delayed by insurance systems. The Foundation provides direct advocacy for families to secure health coverage, working with public insurers, private plans, and hospital financial assistance programs. These efforts ensure that children are not excluded from critical procedures due to bureaucratic or financial obstacles.
05
A Holistic, Family-Centered Approach
Beyond logistics and policy, the Foundation serves as a trusted ally and voice for families, offering emotional support, education, and connection to a wider community of caregivers and clinicians. Every action is grounded in the belief that no family should face a rare disease diagnosis alone—and that meaningful access to care is a right, not a privilege.