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Based on the wide spread failure rate of CNS clinical trials a renewed focus on early drug development is necessary. Novel study designs and technologies to enhance Phase I-II studies support and de-risk Proof of Concept trials, using a ‘go no-go’ strategy. The growing application of a Research Domain Criteria (NIMH initiative) approach to drug development and patient ‘diagnosis’, along with the prioritization of behavioral, biomarker, and other neurophysiological measures demonstrating engagement in neurocircuitry of relevance might be the ‘path forward’ to successful development of new therapies for our patients with brain disorders.

Drs. Brett English and Larry Ereshefskywill focus at this year’s annual meeting on Early Drug Development describing novel approaches and technologies to enhance Phase I studies to inform & de-risk drug development to support Proof of Concept trials. An exciting new approach is the growing adoption of a Research Domain Criteria (NIMH initiative) influenced approach to drug development. The prioritization of behavioral, biomarker, and other neurophysiological measures that demonstrate engagement in neurocircuitry of relevance, can bring greater sensitivity to detect signal than typical behavioral measures (i.e., rating scales).

The implications for drug development will be discussed in the context of a data driven presentation demonstrating these new technologies (quantitative EEG, Event Related Potential, fMRI, and cerebral spinal fluid sampling). Supplemental information from the NIMH RDoC background document are available here.

During this informative session the challenges of traditional drug development for new therapies of CNS disorders will be discussed. A new, ‘neurocircuitry domain’ approach for CNS development will be outlined, incorporating the bases for a shift to the use of Research Domain Criteria. Also, a development plan which incorporates pre-clinical strategies and biomarkers neurocircuitry assessments in both healthy volunteers and patients will be discussed.

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