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: 

What are we advocating for

1. Accelerating Kids Access to Care Ask

The Need: Currently, if a patient needs to see a provider outside of their state, the provider must become a part of the patient’s state’s Medicaid program, which is a long, time-consuming process.

The Proposed Solution: This bill would change this process by making a new, faster process for doctors to enroll in another state’s Medicaid program by:

  • Establishing a federal streamlined screeening and enrollment pathway
    • Voluntary, meaning providers are not required to do this
    • If providers opt to be screened through this pathway, they will be eligible to enroll in other state’s Medicaid programs for a 5-year period, assuming they remain in good standing
  • Eligible providers must present the most limited risk of fraud and abuse, be enrolled in Medicare or their home state Medicaid program, and not be excluded from participating in any federal health programs.
2. Safe Step Act

The Need: Step Therapy, also known as “fail first”, is a practice used by many health insurers that forces patients to try medications preferred by the insurer before being approved for the medication being prescribed by the patient’s healthcare professional. In the rare disease community, this can mean irreversible damage.

Proposed Solution: This bill would ensure that employer plans / Pharmacy Benefit Managers offer an expedited and medically reasonable step therapy exception process.

  • 72 hours for non-emergencies
  • 24 hours for emergencies

This bill also requires a group health plan to implement and make readily available a clear process for an individual to request an exception to the protocol.

3. 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.

4. Join the Rare Disease Congressional Caucus 

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.