HD Regulatory Science Consortium (HD-RSC) A consortium aimed at accelerating treatments for Huntington s disease
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1 HD Regulatory Science Consortium (HD-RSC) A consortium aimed at accelerating treatments for Huntington s disease Critical Path Institute / CHDI Foundation
2 Huntington s Disease Regulatory Science Consortium The overall goal of this initiative is to create a regulatory science strategy for HD, offering additional incentive and de-risking for HD therapeutic development by all stakeholders The HD Regulatory Science Consortium (HD-RSC) will provide a forum and structure to bring together the necessary participants from the HD community for data contribution and tool development, leading to efficiencies in development of new therapies 2
3 HD-RSC Proposed Governance Structure Academic Industry 3
4 C-Path Consortia Fifteen global consortia collaborating with 1,450+ scientists and 84 organizations Coalition Against Major Diseases Focusing on diseases of the brain Coalition For Accelerating Standards and Therapies Data standards Critical Path for Parkinson s Consortium Enabling clinical trials in Parkinson s Disease Critical Path to TB Drug Regimens Accelerating the development of TB drug regimens and diagnostics Duchenne Regulatory Science Consortium Duchenne Muscular Dystrophy Huntington's Disease Regulatory Science Consortium Expediting approval of Huntington's therapeutics International Neonatal Consortium Neonatal clinical trials Multiple Sclerosis Outcome Assessments Consortium Drug Effectiveness in MS Biomarkers Clinical outcome assessment instruments Clinical trial simulation tools Data standards In vitro tools Polycystic Kidney Disease Outcomes Consortium New imaging biomarker for PKD Patient-Reported Outcome Consortium Assessing treatment benefit Electronic Patient-Reported Outcome Consortium Electronic capture of treatment benefit Predictive Safety Testing Consortium Drug safety Pediatric Trials Consortium Developing effective therapies for children Type 1 Diabetes Consortium Qualifying biomarkers for type 1 diabetes Transplant Therapeutics Consortium New drug development tools for transplantation
5 Back up slides 5
6 HD-RSC Governance Founding Principles This HD initiative is uniquely positioned to address the current gaps in HD drug development and assist industry as they deliver new therapies to patients in need The goals of the consortium are to achieve consensus in advancement of HD drug development tools; data sharing is key to success Members are expected to provide in kind contributions in terms of sharing data and knowledge/expertise Confidentiality is fundamental to the principles of members when signing onto the HD-RSC membership agreement 6
7 Roles & Responsibilities of CPP Coordinating Committee Recommend subject matter expertise and contribute input as to the prioritization of consortia activities Review progress of workgroups, including recommending participants Review and provide input on manuscripts for publication and regulatory submissions (e.g. white papers, briefing packages, etc.) Assist with communication and coordination with relevant HD initiatives (e.g. NINDS HD biomarkers) Recommendation of HD-RSC Co-Chairs for the various working groups CONFIDENTIAL 7
8 Proposed Initial Focus Areas and Working Groups CDISC data standards for HD. Identify and acquire key HD datasets Aggregate HD clinical data into C-Path Online Data Repository (CODR) Enable stakeholder access to the integrated HD Database as defined by data contributors HD disease progression model in manifest HD Model-based clinical trial enrichment tool for clinical trial design EMA and FDA regulatory acceptance paths HD-RSC modeling publication strategy Prioritize leading candidate HD biomarkers Seek input from FDA and EMA on HD candidate biomarkers Develop and deliver on a HD- RSC biomarkers publication strategy Identify expectations for reliable and clinicallymeaningful outcome measures acceptable for use in HD clinical trials. Prepare points to consider white paper focused on advancing therapies in premanifest HD 8
9 All Proposed HD-RSC Working Groups are Data-Driven Working Group 2 HD Modeling (Model-based clinical trial enrichment platform) Working Group 4 HD Clinical Outcomes Assessments Working Group 1 HD Data (CDISC Standards, data management and integration) Working Group 5 HD Science Forum (White papers, points to consider, etc.) Working Group 3 HD Biomarkers 9
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