From precision interventions to precision health

Area of Science:

• Pharmacogenomics
• Clinical Pharmacology
• Translational Medicine

Background:

• Precision medicines, tailored to individual profiles, are rapidly advancing.
• Current preclinical and clinical trial methods struggle to predict intervention precision.
• Understanding mechanisms of action (MOAs), clinical effects, and individual utility is challenging.

Purpose of the Study:

• To highlight the need for improved strategies in precision medicine research.
• To emphasize the necessity of collecting human data for better prediction.
• To address the limitations in current preclinical and clinical trial designs.

Main Methods:

• Review of current precision medicine development and evaluation strategies.
• Identification of data gaps in understanding intervention impact.
• Proposal for built-for-purpose clinical trials.

Main Results:

• Current methods are insufficient for anticipating the precise MOAs, clinical effects, and utility of precision medicines.
• Data on living human beings is crucial for accurate assessment.
• Need for tailored clinical trials to gather specific data.

Conclusions:

• Enhanced clinical trial designs are essential for advancing precision medicine.
• Collecting comprehensive human data will improve the prediction of treatment efficacy and patient benefit.
• Future research must focus on built-for-purpose trials to realize the full potential of precision medicine.