Legislative Conference Agenda

Hill Day Issue Information

Legislative Ask #2

Cosponsor the Newborn Screening Saves Lives Reauthorization Act, S. 350/H.R. 482 Cosponsor the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 373

TALKING POINTS

 Of the four million babies born in the U.S. each year, 1 in 300 are found to have a potentially devastating 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.

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.

Hill Day Issue Information

Legislative Ask #3

TALKING POINTS

 While much progress has been made in driving forward policies and procedures to ensure the patient 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 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.

Hill Day Issue Information

Legislative Ask #4

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

TALKING POINTS

 Genetic counselors are experts with advanced training in medical genetics and counseling, uniquely equipped to support patients, families, and providers throughout the genetic testing process.  Genetic counselors improve patient care and healthcare costs. The services of the more than 5,000 certified genetic counselors in the U.S. are increasingly important to patients and providers given advances in genetic testing, with approximately ten new genetic tests becoming available daily, and as genetic testing is becoming more complex.  Limited access to genetic counseling services can result in increased wait-times for genetic testing and subsequent delays in diagnosis.  Current Medicare policy denies Medicare beneficiaries direct access to highly trained, board-certified genetic counselors to navigate complex factors affecting their health.

ACCESS TO GENETIC COUNSELOR SERVICES ACT

 Enact S. 1450/H.R. 2144 to update Medicare law to improve the lives of up to 60 million Americans, including those with rare diseases.  Provide beneficiaries direct access to genetic counselors through direct access and billing.  S. 1450/H.R. 2144 will enable genetic counselors to bill Medicare directly and be reimbursed for services delivered to Medicare beneficiaries at 85% of physician payment levels.  Improve care for all patients seeking genetic counseling by facilitating the ability of additional physician practices and hospitals to employ genetic counselors.  Help to address health disparities and inequities that affect those with lower incomes, improving care for vulnerable, dual eligible Medicare-Medicaid beneficiaries.

Hill Day Issue Information

About the Rare Disease Congressional Caucus

•Co-Chairs: Representatives G.K. Butterfield (NC) and Gus Bilirakis (FL) and Senators Roger Wicker (MS) and Amy Klobuchar (MN)

TALKING POINTS

 The Rare Disease Caucus is a bipartisan, bicameral caucus that works to raise awareness of rare diseases.  Rare diseases affect more than 30 million Americans and their families.  There are more than 7,000 known rare diseases but unfortunately, 93-95% do not yet have a treatment approved by the FDA.  Rare or orphan diseases are defined as diseases affecting fewer than 200,000 people in the U.S.  More than 80% of rare diseases are considered ultra-rare, affecting fewer than 6,000 people. Some diseases affect fewer than 100.  Rare diseases include rare cancers, tropical or neglected diseases, genetic diseases and many pediatric diseases including cancers. Many of these diseases are life-threatening and have no treatment options.  The Rare Disease Congressional Caucus helps bring public and Congressional awareness to the unique needs of the rare disease community (including patients, physicians, scientists, and industry), and creates opportunities to address barriers to the development of and access to life-altering treatments.  The Caucus gives a permanent voice to the rare disease community on Capitol Hill. Working together we can find solutions that transform hope into therapies and cures.