The National Center for Advancing Translational Science has released a Notice indicating that an RFA will be released to re-compete the Rare Disease Clinical Research Network (RDCRN), NOT-TR-18-002.
The Notice indicates that the RFA for RDCRN re-competition will likely come out in March 2018, with applications due in August. This is one of the few NIH funding vehicles dedicated to addressing natural history studies, biomarker and endpoint development, and other studies under the clinical trial readiness category. Clinical trials also can be supported.
Following the language used in prior RDCRN releases, NIH notes: "Each RDCRC must include a consortium of clinical investigators, institutions, and relevant organizations, including active engagement of patient organizations, for the study of a group of a minimum of three rare diseases, or a therapeutic area or concept (e.g., genetic mutation, organ or organelle)." Adequately addressing this language with a tightly integrated network of investigators and compelling clinical studies is key to devising competitive applications. Targeting genetically distinct disorders appears to be the defining criterion for many of the consortia, but not all. Examining the organization of the existing RDCRNs may be the best way to explore potential application strategy models (see: https://ncats.nih.gov/rdcrn).
RDCRN applicants must demonstrate strong interactions with patient advocacy groups. MDF would be pleased to partner with applicants addressing myotonic dystrophy.