Bringing rigour to translational medicine

Area of Science:

• Neuroscience
• Translational Research
• Drug Development

Background:

• The field of translational neuroscience is experiencing stagnation.
• Research practices in stroke studies reveal systemic issues like bias and lack of generalizability.
• These problems contribute to the repeated failure of drug candidates in clinical trials.

Purpose of the Study:

• To review evidence of systemic issues in stroke research and other translational domains.
• To propose solutions for improving the efficiency and success rate of drug development.
• To advocate for enhanced rigor in preclinical studies.

Main Methods:

• Literature review of evidence in stroke research and other translational domains.
• Analysis of how research motivations can lead to bias and reduced generalizability.
• Examination of current funding, assessment, and publication practices in research.

Main Results:

• Systemic issues, including bias and lack of rigor, are prevalent in translational research.
• These issues contribute significantly to the high failure rate of drug candidates in clinical trials.
• Preclinical studies often lack the necessary detail, rigor, and statistical power.

Conclusions:

• Better scientific training and revised research funding, assessment, and publication models are crucial.
• Implementing rigorous standards in preclinical (phase III) studies is essential for successful clinical trials.
• Addressing these issues can reduce wasted investment and accelerate drug development.