Health and Human Services Secretary Alex Azar today approved the recommendation that newborn screening for spinal muscular atrophy be implemented nationwide. This recommendation was issued on February 8, 2018, by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). With Secretary Azar’s signature, SMA becomes part of the Recommended Uniform Screening Panel (RUSP), a list of 35 core conditions that all newborns born in the US are recommended to be screened for.
“The approval of this recommendation is an important step toward our goal of having every baby born in the United States screened for SMA,” said Jill Jarecki, PhD, chief scientific officer at Cure SMA. “Newborn screening and early treatment will save the lives of babies born with SMA. SMA is currently the leading genetic cause of death for infants under two years of age, but we now have an historic opportunity to change that.”
Early last year, following the approval of Spinraza, Cure SMA and the SMA Newborn Screening Coalition prepared and submitted a nomination of SMA to the ACHDNC. The nomination was accepted into evidence review in May 2017. The nine-month evidence review process culminated in February’s vote by the committee, and today’s approval of the recommendation by Secretary Azar.
What Happens Next
Each state must determine whether they will implement SMA screening, and how quickly they will implement it. While this RUSP decision is not required for states begin screening, many states will wait until the decision is made. This means that the next several months will be a critical period of determined advocacy by our community, in order to ensure that as many states as possible will implement screening as quickly as possible.
Four states—Indiana, Minnesota, Missouri, and Utah—have already adopted permanent SMA screening. In addition, five states—Georgia, Massachusetts, New York North Carolina, and Wisconsin—have begun or will soon begin pilot screening. Pilot screening is temporary, but is often a precursor to the adoption of permanent screening. If you are interested in leading advocacy efforts in your state, email us at [email protected]. Or, sign up to be an advocate. When you sign up, we’ll notify you of opportunities to advocate in your state or district.
We would specifically like to thank and acknowledge the families who have testified in support of this nomination. Thank you to Kristen Lasko, Amy Medina, Beth Moore, Debra Schaefer, and Cheryl Yoder for sharing your stories. Thank you to Drs. Tom Crawford, Darryl DeVivo, and Kathy Swoboda, who also testified in support of the nomination.
We thank the committee for their careful consideration of the SMA nomination, and Secretary Azar for his timely approval. We also thank the working group who assembled the nomination packet.
We thank our partners in the SMA Newborn Screening Coalition—Biogen, AveXis, and Genentech/Roche—for their support of this important issue.