Adeline Vanderver, MD, Program Director of the Leukodystrophy Center in the Division of Neurology at Children’s Hospital of Philadelphia (CHOP), and Co-Principal Investigator of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), provides some tips for research groups interested in launching their own Rare Diseases Clinical Research Network (RDCRN) consortium.
Leukodystrophies are a group of rare, progressive, metabolic, genetic diseases that affect the central and peripheral nervous system. Symptoms vary with each type of leukodystrophy but can dramatically limit movement, sensory abilities, and overall development. Although there are currently no treatments for leukodystrophies, RDCRN-funded research consortia like the GLIA-CTN, provide researchers, biotech leaders, and patient advocacy leaders around the world with infrastructure support, funding opportunities, and/or other critical resources needed to collaborate more efficiently.
Dr. Vanderver advises groups interested in launching their own research consortium to scrutinize the design of their natural history studies and/or registries with the same level of rigor that they would any other scientific study or clinical trial. Dr. Vanderver stated that a successful application will generally include a high level of detail around the design of the natural history study, including an inventory of outcomes measures, and how those outcomes measures will be used to inform additional studies. Further, the application should include input from experts in statistics and study design. “You want to partner with data management experts, statisticians, and outcomes experts who can help you in how you construct your natural history study,” Dr. Vanderver noted, adding that “the RDCRN is a very collaborative group, so when I applied, I asked for examples, and I reached out to others who were existing leads of RDCRN projects and asked them.” The RDCRN provides an ideal framework to standardize methodologies across research consortia to ensure that the design of natural history studies, along with the data collected, can be shared and applied across different rare diseases.
To learn more about leukodystrophies, visit theglia.org.
To learn more about RDCRN, visit rarediseasesnetwork.org/.
To see more interviews with RDCRN Consortium researchers, visit checkrare.com/rare-diseases-clinical-research-network/
