May 9 – 10, 2024 Meeting

Agenda (PDF - 164 KB)

Meeting Minutes (PDF - 224 KB)

Webcast

• May 9, 2024 - Pending
• May 10, 2024 - Pending

Transcripts

• May 9, 2024 (PDF - 494 KB)
• May 10, 2024 (PDF - 402 KB)

Presentations

Day 1

Regulatory Process for the Review of Drugs for Rare Diseases (PDF - 1 MB)

Anita Zaidi, MD, Lead Physician
Center for Drug Evaluation and Research (CDER) –
U.S. Food and Drug Administration

Duchenne Muscular Dystrophy Evidence-Based Review: Update (PDF - 451 KB)

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

*Nominator (Parent Project Muscular Dystrophy) letter to the ACHDNC Committee (PDF - 237 KB)

At the request of the Nominators, the Committee unanimously voted during the May 2024 meeting to pause the evidence review for Duchenne Muscular Dystrophy until additional information is received by May 2025.

ACHDNC Decision Matrix Tool: Public Health System Assessment (PDF - 179 KB)

Ned Calonge, M.D., M.P.H.
Committee Chair

Day 2

ACHDNC Nomination and Evidence Review Process (PDF - 169 KB)

Ned Calonge, M.D., M.P.H.
Committee Chair

Qualitative Evidence Synthesis: GRADE-CERQual Approach For Assessing the Confidence in Synthesized Findings (PDF - 2 MB)

Jane Noyes, M.D., PhD
Professor in Health and Social Services Research and Child Health
Bangor University

Newborn Screening Ad Hoc Topic Groups: Updates and Committee Discussion (PDF - 1 MB)

Jelili Ojodu, M.P.H.
Association of Public Health Laboratories