Remarks to the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children

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U.S. Department of Health & Human Services
Health Resources and Services Administration
HRSA Press Office: (301) 443-3376


by HRSA Administrator Mary K. Wakefield, PhD, RN

January 26, 2012
Washington, D.C.

Let me begin by recognizing Dr. Joseph Bocchini on his appointment as the Chairman, and thank him for his willingness to serve in this capacity.  I also extend a welcome to the new Advisory Committee members in attendance today.  You are joining the ranks of a committee with a record of substantive contributions.

I offer my sincere gratitude for your work in guiding and influencing the national dialogue as it pertains to infants and families affected by heritable disorders.

Your impact is clearly real and growing. 

This Advisory Committee offers consumers, families, grassroots organizations, advocacy organizations, health care providers, researchers and state health officials a vehicle through which they can join forces to reduce mortality and morbidity associated with genetic disorders.  The utility and relevance of your work is apparent in a number of ways.

For example, the systematic reviews that the Committee has coordinated have provided new mechanisms for evidence-based reviews of rare conditions.

Additionally, you offer a unifying voice for the consideration of modifications or addition of conditions to the Newborn Screening Program. 

You are also the link between the dynamic and expanding field of genetics and public health, and you provide a framework for the discussion of these interactions in public health genomics.

To illustrate, consider what followed your 2010 recommendation to begin screening for Severe Combined Immunodeficiency Disorder.  Pilot studies in various states currently cover approximately 25 percent of births in the United States. 

According to your May 2011 report, nearly 962,000 newborns have been screened and 60 infants, or about one in 16,000, have been identified with some form of immune deficiency.  Fourteen infants with SCID have been diagnosed and received treatment.  And your committee report concluded that screening for SCID has almost certainly saved lives.

In many ways, this committee is helping to level the playing field for infants across the nation.  No longer are some states only screening for 3 or 4 conditions, and others screening for more than 30 conditions.  Now all states screen for a minimum of 29 disorders, so infants may no longer be missed for a life-threatening disorder simply by virtue of where they live.

Related to this, CDC has recognized the advances in newborn screening and your role in guiding the field and setting standards with a uniform screening panel.

CDC found that “improvements in technology and endorsement of a uniform newborn screening panel of diseases have led to earlier life-saving treatment and intervention for at least 3,400 additional newborns each year with selected genetic and endocrine disorders."

Furthermore, the percentage of infants who receive hearing tests has risen from less than half of all babies born in the U.S. in 1999 to 97 percent today.

Simply put, the efforts of this committee have improved children’s health.

Five years ago, you advised that newborn screening was one component of a larger process.  Your 2007 ‘Roadmap’ Report envisioned “…a broad enterprise, of which (long-term follow-up) is an essential part, and an enterprise that involves many players who need to work in partnership.”

Moreover, in 2009 your Advisory Committee recommendations counted primary care professionals among those key players:

 As you noted, “Primary care providers should recognize that genetics and genomic medicine will be an integral component of care and should develop an effective strategy for incorporating emerging clinical genetic medicine recommendations into their clinical practice.

HHS and HRSA are proud to have the committee as a partner and as an important resource to inform our work. 

In addition to reflecting back, I also want to use the invitation to be with here today to talk about the Committee’s work going forward, offer a word about how you do it and consider the environment in which you are doing it. 

As you are aware, legislation governing the scope of action for this Committee is up for renewal in 2013, so this is an ideal time for you to examine how the Committee functions. 

Just as all of us at HRSA, across HHS and the entire Federal government are responding to the twin  realities of decreasing budgets and increasing demand for effectiveness and efficiency, so, too, must your Committee.

Your ability to streamline operations and improve efficiency to meet your statutory expectations is especially important in the run-up to reauthorization.

And this is a good time to consider how the Committee can best assist the Secretary in her decision-making process.  Along these very lines, HRSA and MCHB have been reviewing our own efficiency as part of our agency’s strategic planning process.

This review – this recalibration of how we do business – is something we’ve asked all of our bureaus and offices to do, and it’s something we’re asking of all of our Advisory Committees to consider as well.

HRSA is certainly here to support and partner as you consider how to best engage your work.    We’re prepared to assist you in navigating the process and help you to maximize your contributions to the Secretary and the country.

Let me also briefly comment on the environment, or the broader backdrop for your efforts.  While your work is important to HRSA, it is also highly relevant to achieving the aims of this Administration.

U.S. infant mortality rates remain a harsh reminder that we still have unfinished business that carries over from the 20th century – and that, for all its strengths, health care in this country too frequently fails to protect the smallest and most fragile members of society.

Improving the health of our nation’s children is of paramount importance to the President and Secretary Sebelius, and they’ve committed resources accordingly.

For example, the expansion of the Children’s Health Insurance Program under President Obama has boosted insurance coverage for children from low-income families from 7 million in 2008 to 11 million children now.

And the Affordable Care Act has guided U.S. health care into a new focus on expanding access, improving quality, and promoting wellness and prevention for all Americans by breaking down barriers to screening services and primary and preventive care -- especially for the young.

A lot has been said and written about the Act, but let me take a moment to highlight some of the provisions that that have an immediate impact for all families, especially those parents raising children with heritable disorders:

  • The law bans co-payments for basic preventive health services and screenings, and ends the practice of denying coverage for pre-existing conditions, a provision with important, concrete benefits for infants and children with heritable disorders.
  • The ACA provision that allows dependents to remain on their parents’ health insurance plans until age 26 has already helped insure about a million young adults nationwide, including many with heritable disorders who were previously uninsurable as they entered young adulthood.
  • It has established high-risk insurance pools, providing a safety net for people with pre-existing conditions who were previously denied coverage.
  • And the ACA eliminated lifetime caps on benefits, which relieves a particularly cruel burden on those with such disorders as Gaucher, Fabry, or other Lysosomal Storage Diseases that require regular costly therapies.
  • The law also reauthorized until 2015 of Family-to-Family Health Information Centers.  These 41 centers, staffed by parents of kids with special health needs, have linked more than 170,000 families to federal and state programs, clinics, special insurance pools and rehabilitation services to give children with heritable disorders and other disabilities a better chance in life.

Also very important: The Affordable Care Act created and supports the Home Visiting program administered by HRSA, in partnership with Administration on Children and Families.  Under the program, nurses, social workers and others visit expectant mothers and their families in high-risk communities.  There, they provide counseling and intervention services designed to improve health outcomes for mothers and infants, school readiness for children, and economic self-sufficiency - the research underpinnings of this new program.  Evidence behind the program clearly indicates that providing these interventions sooner decreases the need for more costly clinical care and social services later. 

Of course, the Act also funds a major expansion of the community health center system.

Currently, health centers provide prenatal care to a half-million expectant mothers every year – 67 percent of them in the first trimester – while tending to 178,000 births.

Health centers also immunize 233,000 kids under the age of 2; administer lead tests to 300,000; manage 540,000 cases of asthma; and provide early-detection screening that identifies 775,000 children per year who have hearing, developmental or perinatal disorders and conditions.

As the system expands, so, too, will the populations it serves.  Over time, prenatal care will become available to tens of thousands of additional mothers, and immunizations and screenings will be offered to many more infants.  And these patients are, more and more, cared for in “health homes,” an orientation health centers across the country increasingly use to coordinate care and boost care quality.

In addition to the ACA, we’re driving innovation on other fronts.    

Many of you, I hope, have heard of HRSA’s partnership with the National Healthy Mothers, Healthy Babies Coalition and others to provide expectant and new moms with free text messages reminding them to take care of their health and give their babies the best start in life.

Called Text4Baby, the service sends brief text messages three times a week to young and expectant mothers who might otherwise be hard to reach – but who always have their cell phones handy.  These messages include at least two texts that are broadcast in the second and third trimester to remind expectant mothers about the importance of newborn screening.  Text4Baby – a product of this White House, CDC, HRSA and key private sector partners – is an ingenious use of resources and technology.

So that provides you with just a brief snapshot of some of the efforts we have underway that – coupled with yours – are designed to markedly strengthen the health care and health of our nation’s infants and children.

Your work fits into this broader set of activities, but as you know Federal Advisory Committees have a different role.  You’re not delivering services; you are asked to join your expertise to specified federal work

It is not an easy charge, but it is what lawmakers expect of your efforts.

Thank you for listening.  I’ll be happy to answer any questions you may have.  

Date Last Reviewed:  April 2017