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New FDA/NIH Course Aims to Improve How Gov’t Designs, Evaluates ‘Smaller’ Trials

The US Food and Drug Administration and the National Institutes of Health will hold a course in January designed to improve how their employees design and evaluate small-scale clinical trials for rare diseases.
“Although no part of the course addresses genomics directly, the design/analysis methods [that will be] discussed are of use in a wide variety of circumstances, genomics included,” a spokesperson from the Office of Orphan Products told Pharmacogenomics Reporter this week.
The FDA said in a description of the course that overcoming the issues that arise in designing and analyzing clinical trials in small populations “will become of increasing importance in achieving efficiency in other contexts, particularly as genomic science provides opportunities for individualized pharmacology.”
The course, called “The Science of Small Clinical Trials,” will be conducted by the FDA's Office of Orphan Products Development and NIH's Office of Rare Diseases, and will be held at the FDA’s White Oak campus in Bethesda, Md. The course comprises eight one-hour lectures to be held on Tuesdays and Thursdays throughout the month of January.
It is intended to “explore issues that arise when designing, conducting and evaluating small-scale clinical trials” and to “address an important concern within rare disease communities, where clinical studies have to rely on much smaller study groups,” according to a description of the course. 
Calling smaller trials “an inescapable reality,” the FDA said that as advances in organ transplantation, cellular therapy, and genomic medicine continue to grow, “understanding how to conduct and evaluate” them “is of growing importance.”

“Small clinical trials are an inescapable reality.”

Lawrence Lesko, director of FDA’s Office of Clinical Pharmacology, has previously said that the agency’s requirements for large-scale randomized clinical trials are not always feasible or affordable to accommodate the comparatively smaller patient populations that typify orphan or gene-related diseases. As a result, these requirements must change for pharmacogenomic-based drugs and diagnostics to come to market [see PGx Reporter 02-28-2007].
According to the OOP spokesperson, because of recent advances in “genomic medicine, we thought it was a good time to make our peers more aware of the considerable work that has been done to develop statistically/scientifically rigorous methods for use in small trials.”
To be sure, the focus of the course will not be specifically on running smaller clinical trials for genomic medicine. Rather, it emphasize that “genomics is here, and no longer just a tantalizing future development, and that this is one of many reasons why we need to pay more attention to the science of small clinical trials,” the spokesperson added.
The course was inspired by a 2001 Institute of Medicine report entitled “Small Clinical Trials: Issues and Challenges,” which identified several small clinical trial designs for rare disease populations, including n-of-1 design, sequential design, decision analysis-based design, ranking and selection design, adaptive design, and risk-based allocation design.  
These and others models will be discussed during the course. “We will continue to try to find examples of small studies wherever we can find them within FDA,” said the spokesperson, who added that the final two lectures will comprise a series of case studies of specific trials. “Our problem thus far has been to find sufficient time to present the material we already have on hand.”
Each participant in the course will receive a copy of the IoM monograph and study materials from a website. There will be eight course lectures delivered by officials within and outside of the FDA and NIH. At the conclusion of the course, participants will take a certification examination.
“This is the first attempt at the course, but interest has been gratifyingly high so it will almost certainly be repeated,” the spokesperson said. In the future, the FDA hopes to expand the course’s content to include greater discussion of genomic issues and perhaps even open the course for broader participation.

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