Skip to main content

Advisory Committee on Heritable Disorders in Newborns and Children

May 9-10, 2024 | In-Person with Virtual Option

Agenda

At the request of the DMD nominators, there will not be a vote for inclusion of Duchenne Muscular Dystrophy to the RUSP during the May meeting. Instead, the Committee will decide during the Thursday DMD agenda time what action to take, such as a vote to postpone a RUSP recommendation to a future meeting.

Thursday, May 9, 2024 from 10:00 AM – 3:45 PM

(All times shown are in Eastern Time)

TIME TOPIC
10:00 AM – 10:15 AM
  • Welcome
  • Roll Call
  • Opening Remarks
  • Committee Business
10:15 AM – 11:00 AM Public Comments
11:00 AM – 11:10 AM Break
11:10 AM – 11:30 AM Regulatory Process for the Review of Drugs for Rare Diseases
11:30 AM – 12:00 PM Committee Discussion
12:00 PM – 1:00 PM Lunch
1:00 PM – 1:30 PM Duchenne Muscular Dystrophy Evidence-Based Review: Update
1:30 PM – 1:50 PM Committee Discussion
1:50 PM – 2:00 PM

Committee Liaison Update

  • Committee Discussion and Vote
2:00 PM – 2:20 PM ACHDNC Decision Matrix Tool:  Public Health System Assessment
2:20 PM – 2:45 PM Committee Discussion
2:45 PM – 2:55 PM Break
2:55 PM – 3:10 PM ACHDNC Nomination and Evidence Review Process
3:10 PM – 3:30 PM Committee Discussion
3:30 PM – 3:45 PM Awards and Acknowledgements
3:45 PM End Day 1

Friday, May 10, 2024, from 10:00 AM – 2:00 PM

(All times shown are in Eastern Time)

TIME TOPIC
10:00 AM – 10:15 AM
  • Welcome
  • Roll Call
  • Opening Remarks
  • Committee Business
10:15 AM – 10:35 AM Qualitative Evidence Synthesis: GRADE-CERQual Approach For Assessing the Confidence in Synthesized Findings
10:35 AM – 11:00 AM Committee Discussion
11:00 AM – 11:15 AM Outcomes Research Criteria Update
11:15 AM – 11:35 AM Committee Discussion
11:35 AM – 12:30 PM Lunch
12:30 PM – 1:45 PM Newborn Screening Ad Hoc Topic Groups:  Updates and Committee Discussion
1:45 PM – 1:55 PM New Business
1:55 PM – 2:00 PM Adjourn