Objective: To align with regulators on the utility and development strategy of novel clinical outcome assessments for use in Huntington’s disease (HD) clinical trials.

Background: The HD-RSC is a joint activity of the Critical Path Institute and CHDI Foundation and is creating and pursuing a regulatory science strategy for HD. Emerging therapeutics targeting interventions in early disease, in some cases prior to formal HD motor diagnosis, pose a significant challenge for outcome measures in the pre-diagnosis period. Specifically, the field needs to align on clinical outcome assessments (COAs) that are sensitive to the early non-motor symptoms present in early HD. There are several domains of interest in these populations in addition to motor outcomes, including functional, cognitive, and behavioral assessments. Appropriate tools are needed to optimize outcome strategies to evaluate potential beneficial therapeutic effect.

Method: Launched in 2018, the HD-RSC includes 15 industry members in addition to scientific academic advisors and advocacy organization representatives. Consortium working groups (WGs) focus on different aspects of clinical trial design optimization and standardization of efficacy assessments in HD. Members of the COA WG within the HD-RSC met with the FDA in April 2019 to discuss existing data and to gain feedback on the prospective study designs for the validation and use of a functional assessment and a cognitive assessment in HD clinical trials.

Results: Feedback from the discussion with the FDA was incorporated into outcome measurement development processes and is being used to guide additional studies.

Conclusion: To advance novel treatments lacking regulatory precedent, early alignment with regulators is essential to the development of tools that will be used in clinical programs. Further, alignment during tool development helps accelerate the regulatory review and ultimate approval of emerging therapeutics. Public-private partnerships and consortium-based approaches provide an ideal platform to pursue informal interactions with regulators and engage directly with review divisions (Maxfield 2017).

References: Maxfield KE, Buckman-Garner S, Parekh A. The Role of Public-Private Partnerships in Catalyzing the Critical Path. Clin Transl Sci. 2017 Nov;10(6):431-442. doi: 10.1111/cts.12488. Epub 2017 Aug 3.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/seeking-regulatory-feedback-on-novel-clinical-outcome-assessments-a-case-example-from-the-huntingtons-disease-regulatory-science-consortium-hd-rsc/