Josephine Briggs

February 2005 marks the beginning of an NIH Roadmap pilot program to expedite the transition of novel therapeutic discoveries to early-phase clinical trials.

A component of the Roadmap’s clinical research re-engineering initiative, the NIH-RAID (rapid access to interventional development) pilot will make available, on a competitive basis, the means to address regulatory and/or technical barriers to moving a promising idea from the bench to bedside testing.

Process

According to Josephine Briggs, director of the Division of Kidney, Urologic and Hematologic Diseases, NIDDK, and co-chair of the implementation group for Translational Research Core Services, this pilot program will provide services for academic investigators, as well as for some intramural researchers, "of the sort that are more available in industry settings than in the academic world."

Such services include the technical resources to synthesize small molecules, peptides, and oligonucleotides for early-phase clinical studies; ADME testing (absorption, distribution, metabolism, and excretion) to monitor how a drug behaves in people; and the development of pharmacology assays on blood and urine samples.

The NIH-RAID pilot is modeled after the NCI Developmental Therapeutics Program and will rely on contractors who have established relationships with NCI—a resource "too valuable," Briggs commented, not to apply to this Roadmap initiative.

The new program will, however, have a separate review setting and administration, and the projects will not be limited to those related to cancer. It’s anticipated that availability to the broader research community will accelerate discoveries for other major public health challenges.

Co-chaired by NCCAM Director Stephen Straus and including representatives from 13 different institutes, the implementation group will appoint a review board to select those projects to be awarded grants. Once the projects are actually approved, however, the oversight will largely go back to the co-sponsoring institute(s).

Funding to support individual projects will come from both the Roadmap and individual institutes, with the institutes assuming the bulk (three-fourths) of support in the specific disease areas akin to their mission. Briggs points out that institute co-sponsorship is critical to ensure disease-specific expertise in the oversight of the projects.

Applications for NIH-RAID awards will undergo a two-stage evaluation process (see chart below). The first stage will establish that the resources requested are within the scope of the pilot program and that there is institute co-sponsorship. Applicants selected from this pool will then be invited to submit a full proposal for assessment by an external panel of experts.

Projections

Asked what sort of projects NICHD might be interested in co-sponsoring, Phyllis Leppert, Reproductive Sciences Branch chief, cited research on the prevention of preterm labor and new leads in contraception as examples of areas that have not been adequately addressed by the private sector and would likely encounter translational barriers without NIH resources.

Traci Mondoro, health science administrator of the NHLBI Blood Resources Program, observed that high-risk ideas or therapies for uncommon disorders that frequently do not attract private sector investment, such as hematologic diseases, would be among NHLBI target projects.

NIEHS RAID priorities, according to Dennis Lang, deputy director of extramural research and training, include environmental components of childhood asthma, allergies, and cardiovascular diseases, as well as research related to countering potential terrorist activities or, as described at the NIH-RAID website: "development of new therapeutic approaches for treating or preventing injury and disease caused by the intentional release of chemical agents by terrorists."

For an overview of each institute’s RAID research priorities, visit this website and click "Role of the NIH institutes and centers."

Overall, Briggs commented, "projects will usually not be therapies for very common disease[s] because, by and large, these topics are being addressed by private industry." But she added that there could well be exceptions to that generalization, such as research involving a therapy that cannot attract private sector support because property protection is considered inadequate.

"The focus will not just be on rare diseases but on conditions where we think there is a roadblock," she said.

While the number and kind of proposals remain to be seen, "there has been a discussion about antibiotics for unusual infections and protein-folding chaperone molecules that might treat genetic diseases that result in impaired protein processing, as two possible examples," Briggs said.

The two-year pilot RAID program will fund about 8 to 10 projects a year. "One measure of the success of the program," Briggs noted, "will be whether it can get the projects into early clinical testing."

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