POLICY INTIATIVES

Newborn Screenings Saves Lives Reauthorization Act H.R. 482 / S. 350

Newborn screening allows for early detection of rare genetic conditions at birth. The Newborn Screening Saves Lives Act governs newborn screening at the federal level. Legal statutes and administrative regulations allow for each state to determine how their newborn screening program is run. This Act reauthorizes existing federal programs that:

  • assist states in improving and expanding programs
  • support parent and provider education
  • ensure laboratory quality and effective surveillance
  • facilitate adding of conditions to the Recommended Uniform Screening Panel (RUSP)

The current authorization expired September 30, 2019.


STAT (Speeding Therapy Access Today) Act H.R. 1730 / S. 670

The STAT Act is a bipartisan, bicameral, community-led bill aimed at improving the development of and access to therapies for the rare disease community. The goal is to enact targeted policy reforms at the Food and Drug Administration (FDA) to accelerate development of therapies while also facilitating patient access to those therapies.

The STAT Act will:

  • Accelerate rare disease therapy development
  • Optimize interagency coordination
  • Advance science-based regulatory policies
  • Facilitate access to therapies

Learn more www.statact.org.


Access to Genetic Counselor Services Act H.R. 2144 / S. 1450

Medicare beneficiaries face significant barriers in accessing genetic counselor services, and these barriers worsened during the COVID-19 pandemic. Recent evidence also finds disparities to accessing genetic counseling services based on social determinants of health and geographic location. The Access to Genetic Counselors Services Act will:

  • Provide beneficiaries direct access to genetic counselors through direct access and billing,
  • Improve care for all patients seeking genetic counseling by facilitating the ability of additional physician practices and hospitals to employ genetic counselors,
  • Enable genetic counselors to bill Medicare directly and be reimbursed for services delivered to Medicare beneficiaries at 85% of physician payment levels,
  • Enhance team-based care coordination for all patients, facilitate education and communication for patients and providers, and increase patient and provider satisfaction, and
  • Help to address health disparities and inequities that affect those with lower incomes, improving care for vulnerable, dual eligible Medicare-Medicaid beneficiaries.

Improving Medicare beneficiary access to genetic counselors could also improve care for non-Medicare patients, including family members who could benefit from genetic testing.


Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act S. 373

This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including information developed by a product sponsor or a third
party such as a patient advocacy organization or academic institution – be considered as part of the
risk-benefit assessment. This action will send an important signal to all stakeholders that patient
experience and PFDD data will be fully incorporated into the agency’s review process and will
encourage such entities to develop scientifically rigorous and meaningful tools and data.

The BENEFIT Act will also enhance an important transparency and accountability provision
included in the 21st Century Cures Act by requiring the FDA to share how such patient experience
and PFDD data was considered within the risk-benefit assessment for any approved therapies. This
will provide additional learnings to all stakeholders, particularly patients, and help further refine and
develop such tools going forward.


Ensuring Lasting Smiles Act H.R. 1916/S. 754

According to the Centers for Disease Control and Prevention (CDC), one in 33 newborns suffer from a congenital anomaly, but these patients often struggle to get their needed services covered by their health insurer. The Ensuring Lasting Smiles Act would require all private insurance group and individual health care plans to cover medically necessary services for all congenital anomalies and birth defects. 


Medicaid Expansion

Medicaid expansion would give more low-income adults access to health care services, resulting in improved health outcomes.  In addition, Medicaid expansion would reduce the health coverage gap for individuals living below poverty level and reduce the cost of uncompensated care for emergency room use by individuals without health insurance. Medicaid expansion would increase state economic growth by creating new health care sector jobs to treat the influx of newly enrolled Medicaid beneficiaries. 

Incentivizing Medicaid Expansion Act

This bill provides the enhanced federal medical assistance percentage (FMAP) to every state that expands Medicaid coverage for individuals who are newly eligible under the Patient Protection and Affordable Care Act, regardless of when such expansion takes place. The bill applies retroactively.


The National Economic Burden of Rare Disease Study

The EveryLife Foundation of Rare Diseases conducted The National Economic Burden of Rare Disease Study providing the most comprehensive assessment of the total economic burden of rare diseases (RDs) in a single year. Through this research, the estimated economic cost of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019. To generate the data for this study, EveryLife identified both direct medical costs, via an analysis of claims data, and indirect and non-medical costs, via a survey (The Rare Disease Impact Survey) of 1,399 members of the rare disease community.

The study identifies an urgent need to fund research, enhance awareness, and improve access to diagnoses, care, and treatment of rare disease. Congressional support of rare disease appropriations priorities would advance critical rare disease research and therapy development at the National Institutes of Health, the Centers for Disease Control and Prevention, and the Food and Drug Administration.

Source: Cover Alabama. www.coveralabama.org | EveryLife Foundation for Rare Diseases. www.everylifefoundation.org | National Organization for Rare Diseases. www.rarediseases.org.