Objective: To establish a broad-based consortium, the Huntington’s Disease Regulatory Science Consortium (HD-RSC), which will advance disease specific data standards and drug development tools, as well as define regulatory pathways to speed the approval of HD therapeutics.

Background: There are few approved medications for the treatment of HD, so there is a significant and urgent need for effective new therapies. Recent progress in understanding disease pathophysiology, identification of new therapeutic targets, and insights derived from technological developments are driving new pharmacotherapeutic approaches in HD. This is evident in the significant increase in pharmaceutical companies now advancing therapies for HD. Therefore, Critical Path Institute (C-Path) and the CHDI Foundation have formed a collaboration to bring together the HD community and build a foundation for HD-RSC.

Methods: HD-RSC will bring together partners from industry, academia, government, and patient advocacy organizations to share data and expertise to achieve regulatory goals in support of HD drug development.  Ongoing work, including creating an inventory of clinical data from observational cohorts and clinical trials and existing computational models, will serve as a foundation for HD-RSC regulatory science initiatives. C-Path’s core competencies are being leveraged for the development of Clinical Data Interchange Standards Consortium (CDISC) data standards for HD, and development of a secure, online database to house standardized clinical data from new and existing trials and observational cohorts.

Results: Data sharing among consortium members will enable the development of quantitative simulation platforms that integrate disease, drug, and clinical trial features as tools to select optimal trial designs, as well as efforts to qualify biomarkers and clinical outcome assessments for HD trials.

Conclusions: As a mechanism to support these regulatory strategies, HD-RSC will officially launch activities with a membership meeting in mid-2017. The goal of the first HD-RSC meeting will be to bring together the HD community, report progress on the foundational goals of the C-Path and CHDI collaboration, and plan the way forward for HD-RSC.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/huntingtons-disease-regulatory-science-consortium-hd-rsc-enabling-pathways-to-effective-treatments-through-regulatory-science-and-innovation/