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U.S. Department of Health and Human Services

About FDA

2012 Small Clinical Trials Course

Description: FDA's Office of Orphan Product Development (OOPD), in collaboration with NIH's Office of Rare Diseases Research (ORDR), is conducting a 2-day course entitled, "The Science of Small Clinical Trials," which deals with the design and analysis of clinical trials with small study populations. Small clinical trials may be appropriate and even a necessity in various contexts that include, but not limited, to rare diseases. The course, which will be held at the FDA White Oak campus, will draw upon the talents of many subject matter experts and stakeholders to identify when such trials should be conducted, along with strategies and trial designs that are conducive to overcoming the challenges they present. The course will also be viewable via webcast to FDA employees on the White Oak campus. The typical student is a Federal scientist, clinician, academic or industry representative with experience in human subject research. The course is offered for free. It is expected that there will be about 160 registered on-site students.

Public Health Outcome: This training will facilitate greater efficiency in research and regulatory review and ultimately benefit the public health as it helps to bring new medical therapies to market. This is particularly critical for rare diseases and other areas in which unmet medical needs have been identified for small populations.

Accomplishment: The Office of Orphan Products Development along with the National Institutes of Health (NIH), Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), held the Science of Small Clinical Trials Course on November 27 – 28, 2012 at the FDA White Oak Campus in Silver Spring, MD. The course covered techniques for clinical studies with limited sample sizes including a focus on the study of rare diseases, and provided examples of how such methods have been implemented. There were over 80 attendees from government, academia, industry, and patient advocacy groups and more than 200 additional participants who joined the event via live webcast. The first day focused on general principles and included six case studies of small drug, biologic and device trials that supported marketing approvals. Various techniques including Enrichment, Adaptive design and Bayesian statistical methods were highlighted. The second day provided an opportunity to focus on special considerations in the rare disease space, as well as topics including historical controls, the role of patient organizations, and the industry perspective. All sessions were enhanced by active learning methods and panel discussions with audience participation.

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

A. Establish planning committee and identify working assignments; schedule and establish recurring meeting dates and times to discuss the content and format of the course.


Completed 2/26/2012

B. Finalize draft agenda for the course.


Completed 3/31/2012

C. Finalize agenda topics for the course and identify/contact speakers to participate.



Completed 4/19/2012

D. Finalize speaker list.



Completed 6/14/2012

E. Draft Federal Register notice and send to Regulatory Staff for publishing.



Completed 9/20/2012

F. Complete requests to Speakers to provide draft presentations to the course director.



Completed 11/21/2012

G. Finalize speaker travel and accommodations.



Completed 11/14/2012

H. Hold 2-day training course.



Completed 11/28/2012

I. Post speaker presentations online.



Completed 12/18/2012


  • The dates in italics under the milestone due dates are modified milestone due dates which had to be updated due to real-time delays. The milestone status reflects the revised dates.

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.


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.