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  4. FDA Oncology Center of Excellence-American Society of Hematology (ASH) Public Workshop: Sickle Cell Disease (SCD) Clinical Endpoints - 12/06/2018
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Workshop | In Person

Event Title
FDA Oncology Center of Excellence-American Society of Hematology (ASH) Public Workshop: Sickle Cell Disease (SCD) Clinical Endpoints
December 6, 2018

Date:
December 6, 2018

Co-sponsored by the:
U.S. Food & Drug Administration (FDA) and
the American Society of Hematology (ASH)
Twitter: #OCESickleCell

Co-Chairs:   

Julie Panepinto, MD, MSPH – Medical College of Wisconsin
Ann Farrell, MD – FDA/CDER

Date: Oct 17-18, 2018

Time: 8:00 am to 5:00 pm

Location: Hilton Washington DC/Rockville Hotel & Executive Meeting Center 1750 Rockville Pike
Rockville, Maryland 20852

Registration:

For in-person or webcast participation in this public workshop, please register at the following link by Wednesday October 10, 2018: http://www.hematology.org/Meetings/SCD/8842.aspx

Background:

For regulatory approval of therapeutics for the treatment of SCD, the FDA requires clinical endpoints that show direct evidence of clinical benefit or improvement in an established surrogate for clinical benefit. Since the original hydroxyurea approval, science and technology have evolved, and medical care delivery for patients with sickle cell disease has changed. The robust SCD drug development pipeline is poised to deliver new therapies to patients; however, there is general agreement that a timely discussion about endpoints is needed. To facilitate this discussion, FDA and ASH are convening this public workshop focused on clinical endpoints for SCD.  

Workshop Objectives:

The goals of the Workshop are to identify opportunities to bring uniformity and standards to existing SCD endpoints, identify gaps, and propose development of new endpoints as a focus for future research. The workshop will include seven panels on the following topics:

  1. Patient reported outcomes (PRO)
  2. Pain (non-PRO)
  3. Brain
  4. Biomarkers
  5. End organ considerations
  6. Endpoints related to curative therapies
  7. Considerations of endpoints for low-resource settings

Webcast Information:

ASH will provide a free-of-charge, live webcast of this workshop. Further information regarding the webcast, including the web link for the webcast, will be made available closer to the workshop date.

FDA and ASH plan to post archived webcasts after the meeting, however, in cases where transmission was not successful, archived webcasts will not be available.

Meeting Information: 

  • Agenda and speakers list coming soon

Contact:

FDA/CDER

American Society of Hematology (ASH)

 

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