The Science of Small Clinical Trials (2009-2011)


Introduction | Curriculum | Venues | Who May Attend | Web Site | Enrollment | Continuing Education Credits | Schedule | Contact Us

Introduction

"The Science of Small Clinical Trials," is a training course created jointly by the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA), and the Office of Rare Diseases Research (ORDR) of the U.S. National Institutes of Health (NIH), dealing with the scientific aspects of designing and analyzing clinical trials based on small study populations. While small clinical trials are a necessity in the context of rare diseases, being able to conduct small trials with scientific rigor is of increasing importance in other contexts, particularly as advances in genomic science and device design provide increasing opportunity for individualized therapy. The three editions of the course have received a total of 2256 registrations.

The course was held three times: in 2009, 2010, and 2011. For the last two editions, it was made freely available to any interested participant, and lectures could be viewed in person or online via live or delayed streaming video, or by means of downloading podcast files.

OOPD/ORDR are considering whether or not to hold the course again in 2012, and, if so, what format it will take. If you are interested in participating if such a course occurs, you may send email to be added to a mailing list.



Nature of the Course Curriculum

This course provides a broad overview of its subject, and does not require a strong mathematical background. It is not a high-level statistical seminar. Nor is it an introductory statistics course, assuming no prior knowledge whatsoever. The target audience is professionals with some awareness of basic stastical concepts who are interested in drug/device evaluation and regulatory affairs, in the context of small study populations (as for rare diseases, or personalized medicine). The primary mission of the course has been to increase awareness that FDA does accept and evaluate clinical trials based on small populations, approving them when appropriate.

Prior versions of the course have featured integrated case studies, and expert lecturers from within and outside of FDA/NIH. Attendees were encouraged to take a self-administered, open-book, confidential web-based online examination at the end of the course, to qualify for a certificate from OOPD/ORDR.


Course Venues

Onsite:

The 2009 and 2011 versions of the course were held at the FDA's White Oak Campus in Sliver Spring, MD. The 2010 course was held on the NIH campus in Bethesda, MD; if the historical pattern continues, the next course would be held at NIH.

Online:

The 2010 course was made available via the Internet by means of RealPlayer. The 2011 course was made available via the Internet by means of Adobe Connect Pro


Who Has Attended

The 2009 edition of the course was open only to FDA and NIH employees. The 2011 and 2011 versions were open to all interested parties, and included participants representing a wide variety of organizations, including academic institutions, the pharmactuical industry, government (civilian and military), non-profit organizations, and patient support groups, as well as individuals.


Course Web Site & Teaching Materials

The course has been supported by an extensively developed web site, based upon the open-source course management system known as moodle, which is widely used by many institutions of higher learning. The web site has contained everything needed for the course, including: the course schedule; information about the course venue; a calendar system; access to the course textbook; downloadable lecture materials and supplementary resources; instructions about how to view the course live or by delayed on-demand video streaming or podcast file; discussion forums for each presentation; a live text-chat room; course evaluation questionnaires; and, the self-administered course certification examination.


Enrollment

Enrollment has been required, for all participants (attending in-person or via the Internet). If the course is offered again, this web site will afford an opportunity to register online.


Continuing Education Credits

Regrettably, we were not able to provide continuing education credits for the 2009-2011 courses, but we will endeavor to do so for a future version of the course.


Course Schedule & Lecturers

The 2011 course pursued the following schedule. The course comprised eight ninety-minute lectures, occuring on Tuesdays and Thursdays from 19 April through 12 May, 2011. Short case studies were integrated into most presentations, and each session ended with a question-and-answer period.


Contact Us

For further questions, contact the course administrator.


The Science of Small Clinical Trials (small-trials.keenminds.org) / October 2011