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serves as a key opportunity for strengthened collaborations and working together across different
organizations, because patient advocacy groups can submit resources to be evaluated for inclusion in the
NCATS Toolkit. NCATS Toolkit puts these resources into context with explanations about how the
research process works and how patient advocacy groups can collaborate with researchers in academia,
industry, and government agencies. In short, NCATS Toolkit educates patient advocacy groups about
translation and empowers them to engage in research.

Currently, the NCATS Toolkit is undergoing a redesign to better serve the needs of the rare disease
community. To make information more accessible, it is transitioning from being a standalone site to
becoming a part of the NCATS site. As a result, NCATS Toolkit will be positioned as a key NCATS resource
for patient advocacy groups. To make information more understandable, relevant, and actionable, the
redesign relies on the principles of user experience and scientific communication. Ultimately, NCATS
Toolkit seeks to explain translation systematically to early and intermediate patient advocacy group leaders,
prepare them to have effective conversations with their research partners, and show them how they can
catalyze the therapy development process. NCATS Toolkit illustrates the many ways through which patient
advocacy groups can promote research, including maintaining patient registries, which are a key aspect of
patient-focused therapy development.

Research projects
Specific information on all NIH-funded research, including the programs listed below, can be found in the
NIH RePORTER.

The rare diseases clinical research network
The Rare Diseases Act of 2002 (Public Law 107-280) established the Rare Diseases Clinical Research
Network (RDCRN). The National Institutes of Health was directed by this legislation to establish and
support Research Centers of Excellence that would facilitate the diagnosis, management, and treatment of
rare diseases. The RDCRN consists of multiple clinical research consortia and a Data Management and
Coordinating Center (DMCC), and each consortium actively partners with patient advocacy groups. Since
its initial funding cycle, the RDCRN has included 33 individual consortia [Table 2], studying over 325 rare
diseases. DRDRI NCATS leads this initiative, partnering with multiple other institutes and centers (ICs)
across the NIH. (see Home|Rare Diseases Clinical Research Network (rarediseasesnetwork.org) for an up-
to-date list). Rare diseases are often multisystemic and do not always fit neatly into the mission of specific
NIH ICs. To overcome this challenge, the RDCRN program brings together multiple ICs to collaborate and
support different areas of science within a single consortium.

Each consortium focuses on at least three related rare diseases and promotes collaborative and multi-site
programs and emphasizes a patient-centric approach focusing on clinical research and translational science.
The current cycle emphasizes clinical trial readiness to lessen the risk often faced when a rare disease is
targeted in a clinical trial. Actions taken to prepare for trials include conducting natural history studies,
identifying biomarkers, developing or modifying existing outcome measures, and establishing cohesive
networks of clinicians and patients.

The RDCRN activities are supported by the DMCC. The DMCC provides infrastructure support to
individual consortium as well as network-wide activities. Support includes: (1) administrative support to
facilitate network operations; (2) clinical research support, including best practices and protocol
development; (3) Data management support that builds and maintains a robust and secure data
infrastructure; and (4) engagement and dissemination that promotes patient engagement and broad