Disease Modification in Alzheimer's Disease: Current Thinking

Ther Innov Regul Sci. 2020 Mar;54(2):396-403. doi: 10.1007/s43441-019-00068-4. Epub 2020 Jan 6.

Abstract

Alzheimer's disease (AD) has increasingly been recognized as a huge unmet medical need. Currently, there is no approved drug to cure, prevent, or even slow down the disease. It is imperative to develop disease-modifying treatments for AD to alter the underlying disease progression. This paper reviews the most up-to-date regulatory guidance on how to demonstrate disease modification and provides an overview of available methodologies and applications to clinical trials. The intent is to assist the field with future clinical trials designed to demonstrate disease-modifying effect in AD. The methodologies may be generalizable to broader neurodegenerative diseases.

Keywords: Alzheimer’s disease; biomarker; delayed start; disease modification; persistent effect; regulatory guidance.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease* / drug therapy
  • Humans
  • Primary Prevention