Regulatory-accepted drug development tools are needed to accelerate innovative CNS disease treatments

Biochem Pharmacol. 2018 May;151:291-306. doi: 10.1016/j.bcp.2018.01.043. Epub 2018 Feb 1.


Central Nervous System (CNS) diseases represent one of the most challenging therapeutic areas for successful drug approvals. Developing quantitative biomarkers as Drug Development Tools (DDTs) can catalyze the path to innovative treatments, and improve the chances of drug approvals. Drug development and healthcare management requires sensitive, reliable, validated, and regulatory accepted biomarkers and endpoints. This review highlights the regulatory paths and considerations for developing DDTs required to advance biomarker and endpoint use in clinical development (e.g., consensus CDISC [Clinical Data Interchange Standards Consortium] data standards, precompetitive sharing of anonymized patient-level data, and continual alignment with regulators). Summarized is the current landscape of biomarkers in a range of CNS diseases including Alzheimer disease, Parkinson Disease, Amyotrophic Lateral Sclerosis, Autism Spectrum Disorders, Depression, Huntington's disease, Multiple Sclerosis and Traumatic Brain Injury. Advancing DDTs for these devastating diseases that are both validated and qualified will require an integrated, cross-consortium approach to accelerate the delivery of innovative CNS therapeutics.

Keywords: Biomarker qualification; CDISC standards; Drug development tools; Neurodegeneration; Regulatory science.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Biomarkers / analysis*
  • Central Nervous System Diseases / drug therapy*
  • Central Nervous System Diseases / metabolism
  • Drug Development* / legislation & jurisprudence
  • Drug Development* / methods
  • Drug Discovery* / legislation & jurisprudence
  • Drug Discovery* / methods
  • Guidelines as Topic
  • Humans
  • United States
  • United States Food and Drug Administration


  • Biomarkers