Recombinant DNA Advisory Committee - 2/10-11/92 
Metabolic Information Network 
Dr. Ledley gave a presentation about the Metabolic Information Network by stating four 
points: (1) given the public nature of gene therapy, a thorough and verifiable follow-up is 
essential; (2) many patients will be lost to follow-up by clinical investigators; (3) different 
investigators will bring in different information; and (4) the number of patients 
participating in clinical trials will increase exponentially. The collection, handling, and 
analysis of clinical data is time-consuming and expensive. If an investigator has incomplete 
and/or duplicative reporting, the value of the collected data is limited. These studies need 
experts in the process of data collection, follow-up, networking, and case reporting. He 
introduced Ms. Susan Mize, Director of the Metabolic Information Network. 
Ms. Mize described the Metabolic Information Network of North America, in which 
physicians and cases from the U.S., Canada, and Mexico are currently being collected. 
The Network's primary functions are to serve as a resource for research investigators and 
to improve patient access to treatment. The system has a 98% accuracy level regarding 
unduplicated cases on the database. Tracking cases is important, as is physician follow-up. 
The number of physicians using the system is currently 325, with 220 physicians committed 
to submit cases upon diagnosis of the selected disorders. 
Dr. Ledley discussed the importance of the RAC receiving information from the 
investigators to identify what is safe, what needs to be reviewed, and what does not. He 
emphasized his support for Dr. Leventhal's concerns regarding reporting procedures. The 
reporting problem is substantial, and the time has come to implement this program. Dr. 
Leventhal stated that the network is an interesting idea, but it does not address the 
protocol reporting issue. The RAC does not receive enough information about the 
protocols to make informed decisions regarding safety. Dr. H. Miller asked the RAC to 
reconsider accepting the investigators' FDA reports to see whether the reports were 
adequate. 
Discussion 
Dr. Mclvor stated that the RAC needs to see reports on an individual basis for these 
reasons: (1) the RAC needs to determine whether their review process is addressing 
appropriate questions; (2) the RAC needs to identify which protocols can be dropped 
from the process; and (3) the RAC needs to use this information to determine the correct 
questions to ask about new protocols. Dr. Ledley commented that investigators need to 
examine all existing data before initiating a new gene therapy protocol. 
Ms. Buc suggested that the RAC adopt Dr. Leventhal's forms; however, if investigators 
choose to submit their FDA reports to the RAC, those reports should be accepted. If the 
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Recombinant DNA Research, Volume 15 
