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Table 3 Five “rights” implemented across the spectrum of drug development

From: The “rights” of precision drug development for Alzheimer’s disease

Right element

Target identification

Drug candidate optimization

Non-clinical assessment

Phase 1

Phase 2

Phase 3

Target

Druggable target identified in AD biology

 

PD effect supported

PD effect may be assessed with biomarkers

PD effect supported by biomarkers

PD effect supported by biomarkers and clinical outcomes

Drug

 

Chemical properties

ADME; toxicity; efficacy in animals

PK, ADME in healthy volunteers; MTD established; BBB penetration established

PK, PD in AD

PD in AD

Biomarker

  

Development of biomarkers useful in trials

Toxicity biomarkers

Patient selection; target engagement biomarkers

Patient selection; DM; toxicity; predictive biomarkers

Patient

   

Healthy volunteers; AD for immuuno-therapy trials

Prodromal AD, AD dementia

High-risk normal subjects; prodromal AD; AD dementia

Trial

   

Single ascending dose; multiple ascending dose

Drug-placebo difference at endpoint; adaptive designs

Drug-placebo difference at endpoint; adaptive designs; delay to milestone

  1. AD Alzheimer’s disease; ADME absorption, distribution, metabolism, excretion; DM disease modification; PK pharmacokinetics; PD pharmacodynamic