Unquestionably, 2020 was a year like no other. Everyone has had to pivot enormously. The National Gaucher Foundation has continued to focus on how to best provide information and support to our community during these extraordinary times.
We are proud that the NGF has continued to fulfill our mission to provide financial support and services to patients and families; offer educational and diagnostic programming; and collaborate with medical professionals.
This blog post presents a snapshot of how our work evolved in 2020. We invite you to review our complete 2020 Impact Report, which includes meaningful testimonials from real patients about our work.
Direct Financial Support for Gaucher Patients
Our CARE Programs offer two types of financial support to people in need who live with Gaucher disease. Ours is the only federally approved patient-assistance program that is disease-specific. During 2020, we provided $2.45 million in individual grants.
Patients and families who meet the eligibility criteria may apply for financial assistance with:
- Eligible insurance premiums (CARE Program)
- Eligible out-of-pocket expenses (CARE+ Program)
Supporting Optimal Health With Gaucher Disease
Living with Gaucher disease requires lifelong follow-up and care. Today, early diagnosis and disease management can help Gaucher patients live well and avoid irreversible bone disease.
We employ licensed social workers as Optimal Health Advisors (OHA’s) to guide people to helpful resources. For instance, we connect patients with Gaucher specialists for the expert disease management they need to maintain good health. At other times, we help individuals keep continuous health insurance. Learn more about optimizing your health.
Promoting Timely Diagnosis of Gaucher Disease
Throughout the year, our Gaucher Diagnosis Accelerator (GDA), directed by our Clinical Advisor in concert with our Optimal Health Advisors, continued to support people on their diagnostic journeys.
Our marketing efforts reach out to communities with a higher likelihood of Gaucher to create awareness and action. When people contact us, an OHA speaks with them. If they determine that Gaucher disease may be a possible diagnosis, we arrange for the individual to have the enzyme assay blood test and further testing as needed.
If someone is identified as a Gaucher carrier or person with Gaucher, we guide them to appropriate resources. We support them in getting answers to their questions and connect them with relevant expertise and care providers. Through GDA, we have helped 24 people get a diagnosis—and solutions.
Advocacy for Gaucher Patients During the Pandemic
The spring brought anxiety as we wondered how the coronavirus pandemic might affect people with Gaucher disease. We lobbied Congress to provide access to care even when sheltering at home. While we were just one of many voices doing this work, more voices make a louder call to action so that legislators can better represent our community.
We also worked closely with physicians, including a physicians’ working group, to look into the impact of COVID-19 on Gaucher patients. They shared their direct observations with us, along with case presentations and analysis. This information helped our community feel more confident that the virus poses no greater risk to people living with Gaucher than to anyone else.
Still, that “average” risk to our loved ones, communities, and economies has been substantial. Current events directed the tone of one of our major initiatives in patient education.
At the time of this blog’s publication, the U.S. Food and Drug Administration (FDA) has approved emergency use authorization (EUA) for one COVID-19 vaccine and is on the cusp of approving a second vaccine EUA. We provided details earlier this fall for the Gaucher community about forthcoming COVID-19 vaccines and continue to update our Coronavirus Tips for Patients with important information as our understanding of the virus evolves.
Education and Digital Engagement for the Gaucher Community
Throughout the year, we chose educational topics for this blog that we believed would empower our community. Specifically, we believe that, especially when chaos seems to surround us, the best response is to take action on what we can control. For many of us, that is our day-to-day well-being.
Actionable blog posts on wellness and health
To help you, our community members, take care of yourselves, we published relevant blog posts all year. Topics included managing inflammation to boost the immune system and improve gut health, and bone-strengthening exercises. We also addressed the importance of self-care and emotional resilience.
Of course, health insurance coverage is a cornerstone of life with a chronic disease. We looked into the importance of the Affordable Care Act and what might happen should it be repealed. You can search all of our blog posts online.
Member education: Gene therapy and financial literacy
In the first and last quarters of 2020, we addressed education topics that are vital to the Gaucher community. Our February webinar hosted a panel of scientists who discussed new and upcoming developments in gene therapy for Gaucher disease.
Later in the year, we hosted a series of webinars about financial literacy (funded by a Financial Advocacy RARE Patient Impact Grant from Global Genes). All too often, we don’t learn financial literacy along with other essential life skills. While this topic isn’t specifically health-related, living with a rare disease brings more than its share of financial challenges. We followed up the webinar series with a blog post about financial tips for people living with Gaucher disease.
Our Continuing Role—and How You Can Help
Our data shows that our work to provide current, relevant information is serving the Gaucher community. We are a global leader in providing answers to patients and families living with Gaucher disease, as our web search rankings (outlined in the impact report) demonstrate.
But we can’t make that impact alone. Our community helps ensure we can do this essential work, and this year’s report is a celebration of success for us all.
To ensure that our yearly impact remains equally strong in 2021, please consider making a gift. We are deeply appreciative of your involvement, engagement, and contribution to our community.
- National Gaucher Foundation Impact Report: https://yearly.report/from/#/national-gaucher-foundation/2020-impact-report
- Dec 2019 Blog: https://www.gaucherdisease.org/blog/2019-impact-report/
- Dec 2018 Blog: https://www.gaucherdisease.org/blog/national-gaucher-foundation-2018-year-in-review/