GFPD Advocacy Initiatives to Improve the Lives of Individuals with Peroxisomal Disorders

The GFPD is bringing a team of 20 individuals to advocate for rare disease initiatives that affect  the care and research of our warriors.  Learn more about the five GFPD asks, along with three additional rare disease day asks below. 

GFPD Rare Disease Day Asks:

Improving health outcomes through increased access to innovative treatments:

The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act.

The Need: Through the Prescription Drug User Fee Act updates in 2012 and provisions in the               21st Century Cures Act in 2016, the FDA has programs and policies in place 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.

Increase access to health care services: Accelerating Kids Access to Care Act

(H.R 3089/S. 1544)

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.

Ensuring access to essential nutrition for all: 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 under Federal health programs and private health insurance.

Unlocking the potential of precision medicine for our children:

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.

Additional Rare Disease Day Asks:

  • Sign the Congressional sign on letter for FDA Task Force on Rare Disease Activities
  • Join the Rare Disease Congressional Caucus
  • Support the Appropriations Request for FY 2024 for Rare Disease Programs