Improving Translational Paradigms in Drug Discovery and Development

Area of Science:

• Biomedical Research
• Drug Development
• Translational Science

Background:

• High attrition rates (90%) persist in clinical drug development.
• Failures stem from T1 (pre-clinical to human) and T2 (clinical phases) translation issues.
• T3 and T4 translation address population and real-world health outcomes.

Purpose of the Study:

• To provide an overview of translational science concepts.
• To suggest improvements in biomedical research to overcome 'valleys of death'.
• To discuss tools like Translational Scoring and the FDA Accelerated Approval Pathway.

Main Methods:

• Review of translational science principles.
• Analysis of factors contributing to drug compound attrition.
• Discussion of current initiatives and regulatory pathways.

Main Results:

• Identified key factors for T1/T2 attrition: lack of biomarkers, siloed expertise, industrialization focus, poor governance, and flawed data.
• Highlighted the role of NIH NCATS in promoting unbiased translation.
• Noted the potential impact of Translational Scoring and FDA Accelerated Approval.

Conclusions:

• Addressing translational failures requires integrated approaches and improved data quality.
• Implementing tools like Translational Scoring can mitigate attrition.
• Regulatory pathways like Accelerated Approval may lower drug approval hurdles.