November 14, 2024 Meeting

Agenda (PDF - 247 KB)

Meeting Minutes – Pending

Webcast

  • November 14, 2024 – Pending

Transcripts

  • November 14, 2024 – Pending

Presentations

Newborn Screening Health Information Center Overview (PPTX - 8 MB)

Session Purpose: To provide an overview of the NBSIC website (Home | Newborn Screening Information Center (NBSIC) (hrsa.gov) that provides up-to-date information and resources about newborn screening.

Molly Lynch, M.P.H.
Project Director, RTI International

Research Funding Opportunities to Document Lived Experience of Patients and Families

Session Purpose: To provide information on funding opportunities for research on rare diseases that includes family and/or lived experience perspectives. It is essential that the family and youth voice be included in the research literature so that the ACHDNC has as complete information as possible through the evidence review process.

Alternative Funding Opportunities for Newborn Screening Research (PPTX - 1 MB)

Zhanzhi (Mike) Hu, Ph.D.
Cofounder, Project Guardian

Funding Opportunities for Rare Disease Research at the Patient-Centered Outcomes Research Institute (PPTX - 39 MB)

Nora McGhee, Ph.D.
Associate Director, Clinical Comparative Effectiveness Research Patient Centered Outcomes Research Institute, PCORI

AHRQ Funding Opportunities (PPTX - 2 MB)

Robyn Sagatov, Ph.D., MHS, RDN
Division of Priority Populations, Office of Extramural Research, Education, and Priority Populations Agency Health Research and Quality

NIH Research Funding Opportunities to Document Lived Experience of Patients and Families (PPTX - 3 MB)

Melissa Parisi, M.D., Ph.D.
Chief, Intellectual and Developmental Disabilities Branch Eunice Kennedy Shriver National Institute of Child Health and Human Development

Including the lived experience in public health activities (PPTX - 7 MB)

Catharine Riley, Ph.D., M.P.H.
National Center on Birth Defects and Developmental Disabilities Centers for Disease Control and Prevention

Family Outcomes of Newborn Screening: Project Update (PPTX - 3 MB)

Session Purpose: To provide an update on the development of an assessment tool to document family outcomes of newborn screening. This is part of the larger work to implement the Blueprint for Change by measuring what matters to youth and families.

Don Bailey, Jr., Ph.D., Med
RTI International

Elizabeth Reynolds, Ph.D.
Research Public Health Analyst, Genomics, Ethics, and Translational Research (GET) program 
RTI International

Metachromatic Leukodystrophy (MLD) Evidence-Based Review Update (Presentation Forthcoming)

Alex R. Kemper, M.D., M.P.H., M.S.
Lead, Evidence-Based Review Group

Laboratory Developed Tests

Session Purpose: To provide information on the impact of the Food and Drug Administration's final rule and phaseout policy to increase oversight of laboratory developed tests (LDTs).

APHL LDT Final Rule Comments, Concerns, Proposed Solutions and Actions (PPTX - 3 MB)
Peter Kyriacopoulos
Chief Policy Officer
Association of Public Health Laboratories

Laboratory Developed Tests in Newborn Screening (PPTX - 550 KB)
Scott Shone, Ph.D., HCLD (ABB)
Public Health Laboratory Director
North Carolina Department of Health and Human Services

Newborn Screening Higher-Tier Screening (PPTX - 3 MB)

Session Purpose: To share updates and information on proposed next steps for the higher-tier testing ad hoc topic group.

Shawn Moloney, M.P.H., MLS(ASCP)CM
Newborn Screening Laboratory Manager
Michigan Department of Health and Human Services

Chanika Phornphutkul, M.D., FACMG
Committee Member

New Business Session

A proposed NIH Pilot Project to Assess the Feasibility of Adding a WGS Component into the US NBS Program. Led by NCATS, NICHD, and NHGRI

Newborn Screening by Whole Genome Sequencing (PPTX - 2 MB)

Melissa Parisi, M.D., Ph.D.
Chief, Intellectual and Developmental Disabilities Branch Eunice Kennedy Shriver National Institute of Child Health and Human Development

Date Last Reviewed: