Hill Day Issue Information
Hill Day Issue Information Legislative Ask #3
Legislative Ask #2
Cosponsor the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 373
Cosponsor the Newborn Screening Saves Lives Reauthorization Act, S. 350/H.R. 482
TALKING POINTS
TALKING POINTS
Of the four million babies born in the U.S. each year, 1 in 300 are found to have a potentially devastating
While much progress has been made in driving forward policies and procedures to ensure the patient
condition through newborn screening. 20,000 newborns benefit from the early detection and delivery of life-saving treatments. Newborn screening is the practice of testing every newborn for certain genetic, metabolic, hormonal, and functional conditions that are not otherwise apparent at birth. Diagnosis through newborn screening saves lives, improves healthcare outcomes, and reduces long-term healthcare costs by allowing for detection and intervention at the earliest moment possible. Newborn screening is the most successful public health program in the history of our country.
perspective is considered by FDA, reviewers evaluating candidate drugs and other medical products, some significant gaps remain. One such gap is the lack of any requirement in law today that the FDA include patient experience or patient-focused drug development (PFDD) data as a part of its risk-benefit framework. This means that the agency’s signature tool for evaluating risk-benefit does not have data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, decision on whether or not to approve a product.
THE BENEFIT ACT The BENEFIT Act will enhance an important transparency and accountability provision included in the
KEY BILL PROVISIONS
Reauthorizes the Health Resources and Services Administration (HRSA) state grants to expand and improve screening programs, provide educational resources to parents and health care providers, and improve follow-up care for infants with a detected condition. Reauthorizes the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children which advises the Secretary of Health and Human Services on newborn and childhood screening policies and priorities to enhance state health agencies to ensure screening is available to every eligible infant. Includes the evidence-based federal Recommended Uniform Screening Panel (RUSP).
BACKGROUND Federal newborn screening programs expired on September 30, 2019. The House and Senate both introduced legislation that used the same language to re-authorize
and build upon the current federal newborn screening program. The House passed the legislation on June 23rd and the Senate legislation is still pending.
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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. 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. Many significant statutory and regulatory advances have been made in the past decade to ensued that patient experience is meaningfully incorporated into product development and regulatory review processes. The BENEFIT Act represents the evolution, following on critical provisions coming out of PDUFA V, VI, and the 21st Century Cures Act that have been implemented by the FDA and embraced by stakeholders.
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