GFPD is Advocacy!
Beginning in 2017, the GFPD started training our families and professionals to advocate on behalf of legislation that impacts both the peroxisomal disorder community and the rare disease community.
To learn more about the GFPD’s advocacy efforts, email: Ryan@thegfpd.org
What are we advocating for
1. Interagency Coordinating Committee
The Need: Federal rare disease programs and projects occur across the entire government aimed to address challenges within the rare community such as the diagnostic odyssey, a limited rare disease therapy pipeline, and access to care and treatments.
The Proposed Solution: Interagency coordinating committees have been successful in assessing existing federal initiatives and identifying opportunities for improvements, ensuring one agency initiative can have maximum impact through coordination with other agencies, tracking metrics that can show progress or other areas where resources are needed.
The creation of an interagency coordinating committee will provide a central location for sustained coordination of the federal government’s rare disease initiatives.
Importance of Newborn Screening for the GFPD Community
Newborn screening is an incredible tool to get families closer to receiving the support they need to give their loved one the best quality of life possible.
Receiving an accurate diagnosis can help eliminate a difficult and expensive diagnostic odyssey.
The earlier GFPD families can learn about peroxisomal disorders, the earlier they can focus on beneficial therapeutic interventions and getting the best treatment available.
In December 2021, the Journal of the American Medical Association Network Open (JAMA Network Open) published a first of its kind study, Expert Evaluation of Strategies to Modernize Newborn Screening in the United States. This wide-ranging study delved into gaps in the current system and provided solutions for the future“The pace of therapeutic discovery is accelerating, a future for which newborn screening must be prepared. The strategies identified in this study offer the first steps toward a modern newborn screening system where all babies in the U.S. have access to a timely diagnosis, thus reducing the risk of preventable injury and death.”
Broad adoption of newborn screening is crucial for our GFPD Community, and we are grateful for continued research and support from the scientific and advocacy communities to make this a reality. Newborn screening programs vary depending on where you live. To learn more about newborn screening and legislation in different US states, please visit The EveryLife Foundation’s Newborn Screening page.