Affiliations: [a]
Critical Path Institute, Tucson, AZ, USA
| [b]
CHDI Management/CHDI Foundation, New York, NY, USA
Correspondence:
[*]
Correspondence to: Emily C. Turner, Huntington’s Disease Regulatory Science Consortium, Critical Path Institute, 1730 E. River Road, Suite 200, Tucson, AZ 85718, USA. Tel.: +1 520 547 3440; E-mail: eturner@c-path.org.
Abstract: Huntington’s disease (HD) is a devastating neurodegenerative disorder that urgently needs disease-modifying therapeutics. To this end, collaboration to standardize clinical research practices in the field and drive progress in addressing drug development challenges is paramount. At a meeting in 2017 organized by CHDI Foundation and the Critical Path Institute, stakeholders across the pharmaceutical industry, academia, regulatory agencies, and patient advocacy groups discussed the need for and potential impact of a consortium dedicated to HD regulatory science. Consequently, the Huntington’s Disease Regulatory Science Consortium (HD-RSC) was formed, a precompetitive consortium that is dedicated to building a regulatory strategy to expedite the approval of HD therapeutics.
Keywords: Huntington’s disease, neurodegenerative, biomarkers, clinical outcome assessment, mutant huntingtin, model-informed drug development, Critical Path Institute (C-Path)
