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U.S. Department of Health and Human Services

About FDA

A. Rare Diseases Program – Internal Training

Description: Provide internal training

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

1. FDA staff training course

2/15/2011

Completed 2/15/2011

B. Rare Diseases Program – Outreach FY 2011

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

1. Gaucher biomarker workshop

9/20/2010

Completed 9/20/2010

2. Duchenne Muscular Dystrophy workshop

9/29/2010

Completed 9/29/2010

3. Investigator training course

10/18/2010

Completed 10/18/2010

4. Website development

3/31/2011

Completed 11/15/2010

5. Central Nervous System drug delivery workshop

8/31/2011

Completed 7/31/2011

C. Rare Diseases Program – Outreach FY 2012

Description: Outreach efforts include the development of staff training related to the development, review and approval of drugs for rare diseases. The training will be provided to CDER and CBER review staff to familiarize them with the challenges associated with rare disease applications, to promote best practices in review and regulation of these applications, and to encourage flexibility and scientific judgment in the review of products for rare diseases. Other workshops and trainings are intended to facilitate the development of the science of drug development for rare diseases including disease specific as well as more generally applicable topics.

Briefing Status: ON TRACK

Prior Briefing Status: ON TRACK

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

1. NORD/DIA Investigator Training Course

10/22/2012 – 10/24/2012

Completed 10/24/2012

2. NIH/ FDA Natural History Studies Workshop

5/16/2012 - 5/18/2012

Completed 5/18/12

3. FDA Rare Disease Patient Advocacy Day – March 2012

3/1/2012

Completed 3/1/2012

4. “Characteristics of Marketing Applications for RD Indications” publication – FY 12 Q1

FY12 Q1 (Exact Date TBD)

Completed Published in Drug Discovery Today 5/1/2012

5. Rare Diseases Guidance Development

FY12 Q1 (Exact Date TBD)

On Hold In Clearance

6. NORD Corporate Council FY12 Q2

5/15/2012

Completed 5/15/2012

Key Projects Legend

Milestone StatusDefinition
Not Yet StartedWork for specific milestone has not yet been started.
CompletedMilestone and/or overall project is completed.
On TrackMilestone - On track for completion by milestone deadline. Quarter status - Project is on track for completion based on overall milestone status.
On HoldMilestone - On hold, but deadline for completion has not passed. Quarter status - Project is on hold, based on overall milestone status.
DelayedMilestone - Delayed as it has not been completed and deadline has passed. Quarter status - Project is delayed based on overall milestone status.

Glossary

Note: The data provided on this website is produced on an ongoing basis for performance management purposes and is subject to change due to updates of preliminary estimates, corrections, or other reasons. In addition, FDA may change the type or amount of data provided on this website at any time. Information marked as "Completed" may include measures and/or key projects for which activities are ongoing but no longer tracked as part of FDA-TRACK.