Critical Path Institute (C-Path) and CHDI Foundation, Inc., are collaborating to set up a broad-based consortium that will more clearly define regulatory pathways leading to the approval of Huntington’s disease (HD) therapeutics. The HD Regulatory Science Consortium (HD-RSC) will bring together participants from within and without the HD community to advance drug development tools (DDTs, such as biomarkers and clinical outcome assessments) for regulatory endorsement and to facilitate clinical data standardization and collaboration. These DDTs will be made publicly available to help expedite and de-risk the drug-development pathway, speed the time to drug approval, and offer further incentive to drug developers to enter the HD sphere.
This regulatory focus is in response to the US Food and Drug Administration’s (FDA) Guidance for Industry and FDA Staff – Qualification Process for Drug Development Tools. Such tools would potentially accelerate the drug approval process. Among the consortium’s specific projects will be the regulatory qualification of biomarkers and outcome measures, and the development of a clinical trial simulator. The HD-RSC will also work in partnership with the Clinical Data Interchange Standards Consortium (CDISC) to develop therapeutic area data standards, and then centrally warehouse shared data as the foundation for these and other activities.
C-Path will manage the day-to-day activities of the HD-RSC, which is expected to first meet in Q2 2017. CHDI has provided planning and start-up funds.
“We support and share C-Path’s model of collaborative initiatives to accelerate drug development,” says Robi Blumenstein, President of CHDI Management, Inc. “The need for C-Path’s expertise in consortium building and regulatory science has become increasingly apparent as our community sees a number of high-quality therapeutic programs approach the clinic.”
Through the pre-competitive consortium approach, CHDI, C-Path, and other stakeholders can share data and knowledge, avoid duplication of effort, and collaborate in innovative ways so that scientific advances can be translated into therapeutics as efficiently as possible.
“One of the fundamental challenges to drug development for a genetically predetermined disease such as HD is shifting the paradigm for drug approval, so that we can intervene as early as possible; ideally, this would eventually be before any overt symptom onset,” says Martha Brumfield, PhD, C-Path president and CEO. “For quantitative analytical approaches to address this problem, data sharing, standardization, and aggregation through a rigorously defined procedure are paramount. In this regard, C-Path’s core competencies in clinical data standards development, as well as secure data management, standardization, and curation, will be tapped. These skills, combined with C-Path’s track record of success in consortium building and regulatory science, and CHDI’s extensive data resources and research network, provide a solid foundation for the HD-RSC’s success.”
Source: Critical Path Institute