T1D-RAID is a cooperative program designed to facilitate translation to the clinic of novel, scientifically meritorious therapeutic interventions. It does this by making available, on a competitive basis, NCI resources for the pre-clinical development of drugs, natural products, and biologics. A partial listing of those services includes: high-throughput screening, studies in animal models, formulation, pharmacology and toxicology studies, and bulk substances acquisition.
In some cases, additional preclinical testing is needed to validate potential therapies under disease-specific conditions and in multiple animal models before therapeutics can enter the T1D-RAID development pipeline. In recognition of this need, NIDDK recently established the Type 1 Diabetes Preclinical Testing Program (T1D-PTP) to provide investigators with access to the established facilities and expertise needed to extend, enhance and validate preclinical studies of promising new therapeutics. The T1D-PTP program currently supports two contracts, which are separate from each other and from the T1D-RAID NCI contract resources mentioned above, to assist in preclinical development of therapeutics for T1D:
The T1D-RAID and T1D-PTP are programs intended to remove the most common barriers to progress in identification and development of new therapies for Type 1 Diabetes. The common goal of these programs is to support and provide for the preclinical work necessary to obtain "proof of principle" establishing that a new molecule or novel approach will be a viable candidate for expanded clinical evaluation.
The T1D-RAID and T1D-PTP programs are not intended to be a pipeline for materials for NIH-held INDs. It is assumed that most of the products will be studied clinically under investigator-held INDs within the originating (or a collaborating) institution.
Acceptance into the NIDDK T1D-PTP program does not imply any future commitment for services under T1D-RAID. Future T1D-RAID service requests should be submitted separately and will be reviewed according to T1D-RAID program guidelines.
Like T1D-RAID, NIDDK T1D-PTP does not award grants to a particular laboratory. Support will be provided in the form of access to NIDDK-supported contract facilities that will assist investigators by providing expertise and facilities needed for preclinical testing in multiple disease-relevant animal models. It may happen that some assays are best carried out in the originating laboratory, in which case NIDDK will advise on other mechanisms available for support of this work. The focus will be on using NIDDK staff and contractor expertise to define the most effective and cost-efficient means of accomplishing the necessary tasks to establish preclinical efficacy of new therapeutics.
NIDDK’s Type 1 Diabetes Preclinical Testing Program is designed to assist in accomplishing tasks that are rate-limiting in bringing discoveries from the laboratory to the clinic. Once a project has been approved, NIDDK staff interact directly with the Principal Investigator (PI) and T1D-PTP contractors to design, perform and oversee execution of approved tasks. The necessary tasks will vary from project to project, and will vary depending on the specific T1D-PTP Contract required to perform services (see NOT-DK-09-006 or NOT-DK-09-009).
The output of T1D-PTP activities will be data and information that will be made fully available to the originating investigator, as well as to the public, for support of further preclinical development from an IND application to clinical trials.
The following are acceptable types of organizations that can request program resources:
It is anticipated that the majority of Requests submitted will involve a potential therapeutic that either already has protected intellectual protection or that is in the public domain. Please contact representatives of the T1D-PTP program for more information about intellectual property rights. Under the T1D-PTP program, NIH Materials Transfer Agreements will form the basis for sharing novel reagents or confidential information with NIDDK, its contractors and subcontractors [see example MTA: NIDDK T1D-PTP MTA (pdf, 30kb)].
Requests will be reviewed for support by a specially convened T1D-PTP Review Panel consisting of outside experts from academia and industry. An internal NIDDK Review Panel will also discuss the submitted requests. Review Panel members are bound by confidentiality agreements customary for review of NIH grants. The review criteria will reflect an emphasis on fostering development of T1D therapeutics and relevant preclinical testing. Acceptance into the program will be based on scientific merit, programmatic relevance, perceived translational potential and availability of resources.
A specially convened ad hoc Review Panel of experts from academia and industry will initially evaluate the Requests in a confidential manner for scientific and technical merit. The Panel will review and prioritize each Request on the basis of the following evaluation criteria:
Requests will receive a secondary level of review by the NIDDK internal oversight committee. NIDDK projects will be compared with tasks currently assigned to NIH contractors and a final prioritization of projects will be made taking into consideration the following additional factors:
T1D-PTP Review Committees have the authority to scale back an investigator's request based on development or feasibility concerns and to recommend approval of only a portion of the requested tasks. The number of projects to be supported in any review cycle will be a function of the level of merit and availability of funds and resources.
The NIDDK expects to convene an oversight committee consisting of outside advisers and a subgroup of its own members. This group will periodically review the progress and direction of the T1D-RAID and T1D-PTP programs and explore opportunities for program development and enhancement.