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. Improving Health Outcomes Through Increased Access to Innovative Treatment
The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act. (S.526/H.R.  1092)

The Need: The Prescription Drug User Fee Act (PDUFA) provides updates in 2012 and provisions in the 21st Century Cures Act in 2016, the FDA with programs and  policies to ensure that patient perspectives are gathered and made available throughout the risk- benefit analysis of potential therapies. However, there is currently no requirement that the FDA use this data in their analysis or report on how the patient perspective factored into the risk- benefit analysis.

The Proposed Solution: The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act would amend the Food, Drug and Cosmetic Act to ensure that patient experience, patient engagement, and patient-focused drug development data be included in the risk-benefit assessment. Additionally, the BENEFIT Act would require the FDA to report on how these data were used throughout the risk-benefit assessment.

2. Ensuring Access to Essential Nutrition For All: The Medical Nutrition Equity Act (H.R. 3783/S. 2013)

The Need: Thousands of children and adults in the United States live with inborn errors of metabolism (IEM) that prevent their bodies from properly metabolizing and absorbing normal, everyday food. For these patients, medical nutrition is the primary treatment for the effective management of these conditions. Unfortunately, many health insurance plans in the United States do not provide reimbursement for medical nutrition despite their proven efficacy in the treatment of IEMs, causing medical nutrition therapy to be cost- prohibitive for many patients. Without access to treatment, IEM patients suffer severe negative health outcomes including deleterious cognitive effects, mental health issues, and in some cases, death.

Proposed Solution: The Medical Nutrition Equity Act (116th Congress- S. 3657/H.R. 2501) would provide for the coverage of medically necessary food and vitamins and individual amino acids for digestive and inherited metabolic disorders, already covered with Tricare under Federal health programs and private health.

3. Increase Access to Healthcare Services: Accelerating Kids Access To Care Act

The Need: When children enrolled in Medicaid or CHIP (Children’s Health Insurance Program) require access medically necessary care out of state there are often lengthy delays caused by the process to screen and enroll out of state physicians as eligible providers in the child’s home state’s Medicaid or CHIP program. The process varies by state and requires providers to spend significant amounts of time to complete the requirements.

The Proposed Solution: The Accelerating Kids’ Access to Care Act seeks to address this problem in a commonsense way that balances access to care with program integrity needs. The proposal will establish a screening pathway that children’s hospitals and related providers can utilize on a voluntary basis. If providers opt to use this pathway and are screened successfully, they can be enrolled in other state Medicaid programs if called upon to provide care to children. Eligible providers will be limited to those providing care for children or, in limited cases, to people over the age of 18 who are being treated for a condition that developed prior to age 18. The legislation builds upon existing CMS activities, including an ongoing multi-state pilot in which CMS is screening providers via the Advanced Provider Screening System and is also consistent with ongoing efforts to reduce regulatory burdens in healthcare. It is consistent with actions CMS has taken via the COVID-19 public health emergency (PHE) waivers to permit states to waive such requirements during the emergency. Finally, the legislation pertains only to provider screening and enrollment and does not change the authority states have to authorize out-of- state care and negotiate payment with providers who accept such cases.

4. Unlocking the Potential of Precision Medicine for our Children: The Precision Medicine Answers for Kids Today Act (H.R. 5989) 

The Need: Nearly 80% of rare diseases are genetic and 70% begin in childhood. Currently, barriers such as lack of insurance coverage and the inability to see relevant health professionals, prevent patients from receiving timely diagnosis.

The Proposed Solution: This legislation will help eliminate these barriers to allow better access to genetic and genomic testing. Genetic and genomic testing will help us understand the individual needs of patients and ensure answers can be provided for millions of families with children struggling with unknown illnesses. Making these tests more accessible will save lives, revolutionize our health care industry, and ease the burden on our system through prevision instead of treatment. This bill would create a program at the Department of Health and Human Services (HHS) for states to provide coverage for genetic and genomics testing for three years, and to study how this coverage improves the diagnosis of pediatric health conditions.

5. Sign the Congressional Sign On Letter for FDA Task Force on Rare Disease Activities 

6. 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.