Core services focus on six general areas: (a) participant recruitment through registry use, development and collaborations, (b) IRB-related consultation, (c) clinical trials development and implementation, (d) consultation on product development, (e) biostatistics support, and (f) coordination of translational activities with the functions of other IDDRC and Hopkins core resources.
Registries and Recruitment of Research Participants
The core assists investigators with their recruitment through the use of multiple resources. First, several registries already exist within our Institution. Examples include IAN, CARD, assessment results from patients seen in the Department of Neuropsychology, a tissue repository and its associated clinical documentation within the IDDRC Genetics Core, the CADDE in the School of Public Health, and a Rett syndrome registry. The core will coordinate access to these resources for supported investigators.
Some projects require development of pseudo-registries of their own. In these cases, the core assists in coordinating selection of data elements with existing registries to maximize compatibility and opportunities for data merges. In this way, investigators will not be forced to “reinvent the wheel” for each new effort. Along these same lines, there is increasing interest in establishing national registries for conditions like Fragile X syndrome, Down syndrome, etc., and the core coordinates local activities with national initiatives.
In addition, the core provides overviews of all KKI and Hopkins clinics providing services to people with developmental disabilities and their families. These clinics see over 24,000 patients annually, representing an enormous potential resource. In addition, core faculty are very familiar with ways to identify and recruit potential participants through communication with care providers as well as through connections with advocacy and parent groups.
Development and Implementation of Clinical Trials
A major priority for this core, and of the IDDRC overall, involves efforts to accelerate the clinical translation process, moving new agents, devices and intervention methods into clinical trials and eventually to the marketplace. The Clinical Translational Core will complement other programs focused on “translation” through coordination and facilitating access, directly informing IDDRC investigators about the process of moving from pre-clinical to clinical research, the structuring of clinical trials, and the mechanics of conducting those trials within the specific populations of interest (e.g., addressing consent issues when procedures entail “more than minimal risk” and the ability of research participants to consent/assent is uncertain).
Regulatory Approvals by IRB and FDA
The core provides guidance in the preparation of materials for IRB review. Investigators with “human subjects” protocols can consult with highly experienced colleagues in the preparation of materials for FDA and IRB reviews. This “pre-review” system also serves an educational function for PIs and project coordinators, speeds up the overall process of IRB review and approval, and reduces needs for subsequent revisions.
Guidance in applying for FDA review and approval of INDs and IDEs regarding the FDA process is available primarily through the CTU and the JHU Institute for Clinical and Translational Research. Core support is available for IDDRC investigators needing guidance on how to access these resources and for supplementing them as needed.
Navigation to Move Ideas from Inception through Translation
Core faculty are available to guide core users in navigating the process of moving ideas from conception or bench into translational trials. Users can meet with core faculty as well as experienced consultants to discuss potential projects or to “brainstorm” about potential projects and applications.
Because the majority of IDDRC-supported investigators have little or no clinical trials experience, the core provides a seminar series on Clinical Trials and Translational Medicine as a forum for both experienced and less experienced clinical investigators to meet to learn about current projects and opportunities for translation, coordinated with other KKI and IDDRC-sponsored presentations and events. In addition, consultations are arranged with expert consultants from the Department of Bioengineering, the Office of Technology Transfer and the Business School to provide advice on how best to develop potential treatments and medical devices that might be useful for individuals with IDD.
Consultation on Biostatistics and Clinical Trials Design
The core provides consultations in biostatistics to Center investigators encompassing the entire scope of IDDRC-supported research. Core biostatistics support primarily focuses on consultation and training, with investigators maintaining responsibility for executing their own databasing and analyses. Additional resources can be identified within the Hopkins ICTR for providing the actual conduct of more labor-intensive activities. These are provided as fee for services at greatly subsidized costs, with the first several hours of consultation/services provided free of charge to IDDRC investigators. Whenever IDDRC investigators require this level of support, referrals to the ICTR are provided.
In the vast majority of cases, biostatistics support involves relatively straightforward services but on occasion the biostatistics faculty and investigators find that their interests overlap to such a degree that true partnerships develop. Both situations are encouraged, and in the past we have seen long-term collaborations emerge through this process.
Coordination with Other IDDRC, KKI and Hopkins Resources
Our institutional environment includes many programs supporting translational research. However, the mission of these other programs is not explicitly focused on IDD, and an important function of this core is to coordinate utilization of the resources of these other programs in a way that enhances the productivity of IDDRC-supported projects. This is accomplished through the interpersonal and programmatic relationships that have been established between core faculty and colleagues directing those complementary programs. Thus, the core facilitates access to these resources for IDDRC investigators.