A Review of the Committee Nomination and Review Process Nancy S. Green, MD Associate Dean for Clinical Research Operations Associate Professor of Clinical.

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Presentation transcript:

A Review of the Committee Nomination and Review Process Nancy S. Green, MD Associate Dean for Clinical Research Operations Associate Professor of Clinical Pediatrics, Division of Hematology Associate Director, Irving Institute for Clinical Translational Research Columbia University Medical Center ACHDNC

ACHDNC Evidence Review Process: Overview Nomination Form HRSA Administrative Review Advisory Committee External Evidence Review Workgroup (ERW) Possible Recommendations: Universal NBS Targeted screening Pilot study Critical studies needed No recommendation Recommend against NBS Possible Further Study(ies ) Nomination Review and Prioritization Workgroup (NRPW)

ACHDNC Evidence Review Process: Subset Nomination Form HRSA Administrative Review Advisory Committee * External Evidence Review Workgroup (ERW) [Dr. Perrin] Nomination Review and Prioritization Workgroup (NRPW) An AC sub-group Task: Readiness for EBR? - Criteria - Recommendation to AC * Only the AC may make binding decisions An Independent group Tasks: Formal EBR - Define terms and criteria - EBR report Committee Chair Criteria Workgroup: - Nomination process and form

Nomination Review and Prioritization Workgroup: Is a Nominated Condition Ready for EBR? 1) The nominated condition(s) is medically serious. 2) Prospective pilot data (U.S. and/or international) from population-based assessment are available for this disorder. 3) The spectrum of this disorder is well described, to help predict the phenotypic range of those children who will be identified based on population-based NBS. 4) There is a screening test that is capable of identifying the condition. 5) If the spectrum of disease is broad, those who are most likely to benefit from treatment are identifiable, especially if treatment is onerous or risky. 6) There are defined treatment protocols, FDA approval/clearance (if applicable) and availability of treatment. Overall recommendation to the Advisory Committee by the NRPW: 2 options: Ready for EBR or Not Ready for EBR, with rationale

SCID – Ready for EBR – under review Pompe - Ready for EBR – under review Krabbe – Ready Fabry – Not ready Niemann Pick – NRPW review pending Nomination Review and Prioritization Workgroup: Nominated Conditions Considered to Date for EBR