Public Workshop – Complex Issues in Developing Drug and Biological Products for Rare Diseases

The Food and Drug Administration (FDA) is announcing a public Workshop entitled “Complex Issues in Developing Drug and Biological Products for Rare Diseases.”

The purpose of the workshop is to discuss complex issues in clinical trials for developing drug and biological products (“drugs”) for rare diseases, including endpoint development and selection, use of surrogate endpoints and the accelerated approval pathway, clinical trial design, conduct and analysis, safety considerations, and dose selections; and to discuss ways to encourage and accelerate the development of new therapies for pediatric rare diseases. FDA is seeking input on these topics from academic, clinical, and treating communities; patients and advocacy groups; industry; and government agencies.

FDA is also holding a separate public workshop entitled “Complex Issues in Developing Medical Devices for Pediatric Patients Affected by Rare Diseases” that will be held on January 8, 2014. (Separate registration is required). The purpose of this workshop is to discuss issues related to medical devices for the diagnosis and treatment of pediatric patients affected by rare diseases. (Contact: Carol Krueger; 301-796-3241; carol.krueger@fda.hhs.gov; see web page.

Date:            January 6-7, 2014

Time:            8:00 a.m. to 5:00 p.m.

Location:     FDA White Oak Campus
                      10903 New Hampshire Avenue,
                      Bldg. 31, Room 1503 (Great Room)
                      Silver Spring, Maryland 20993

FDA Campus Information
Public Meetings at the FDA White Oak Campus

Registration to Attend the Workshop:

If you wish to attend this Workshop, you must register by submitting a written request to CDERrarediseaseworkshop@fda.hhs.gov before close of business on December 20, 2013:

  1. Name
  2. Title
  3. Affiliation, Company or Organization
  4. E-mail address
  5. Telephone number 


January 6

January 7

For questions regarding workshop content please contact:

Tomeka Arnett, Office of Medical Policy, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Bldg. 51, Room 6338, Silver Spring, MD 20993, 301-796-2500, FAX: 301-847-3529, tomeka.arnett@fda.hhs.gov.

Estimated* attendance for Days 1 and 2 was approximately 650-700 attendances (in-person and via webcast) on Day 1, and approximately 400-450 on Day 2.

*The number of attendees was estimated based on sign-in registration for in-person attendance and webcast log-ins via the internet counted separately for each day. Registration is voluntary, and not all attendees elected to sign the in-person register for one or both days. There was no registration for the webcast. Hence, these numbers should be seen as estimates only.

Page Last Updated: 12/11/2014
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
Language Assistance Available: Español | 繁體中文 | Tiếng Việt | 한국어 | Tagalog | Русский | العربية | Kreyòl Ayisyen | Français | Polski | Português | Italiano | Deutsch | 日本語 | فارسی | English