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Translational Scoring of Candidate Treatments for Alzheimer's Disease: A Systematic Approach
1Department of Brain Health, School of Integrated Health Sciences, University of Nevada, Las Vegas, Nevada, USA, cumminj@ccf.org.
Background:
There are many failures in treatment development for Alzheimer's disease (AD). Some of these failures are the result of development programs that lacked critical information about candidate drugs as these were advanced from one phase of development to the next. Translational scoring (TS) has been proposed as a means of increasing the rigor with which treatment development programs are executed. Previously, these approaches were not specific to AD or to the phase of drug development. Detailed information on the characteristics needed to advance a candidate agent from one phase to the next is the basis for success in subsequent phases.
Summary:
The TS approach is presented with a score range of 0-25 for agents entering phases 1, 2, and 3 of development and those that have completed phase 3 and are being considered for regulatory review. Each phase has 5 essential categories scored from 0-5 indicating the completeness of the data available when the agent is being considered for promotion to the next phase. Lower scores suggest that the development program should be reexamined for missing information while higher scores increase the confidence that the agent has the potential to succeed in the next phase. Scoring guidelines are provided and examples of scores for drugs in recent development programs are provided to illustrate the principles of TS. Key Messages: Successful development of drugs for AD treatment requires disciplined informed decision-making at each phase of development. TS is a methodology for more rigorous drug development to help ensure that inadequately characterized drugs are not advanced and that the development platform at each phase is optimal to support success at the next phase.
Insights
Translational scoring (TS) enhances Alzheimer's disease (AD) drug development rigor. This scoring system ensures critical data informs decisions, improving the likelihood of successful AD treatments.
Area of Science:
- Neuroscience
- Pharmacology
- Drug Development
Background:
- Alzheimer's disease (AD) drug development faces high failure rates.
- Failures often stem from insufficient data during candidate drug progression.
- Existing methods lack specificity for AD and development phases.
Purpose of the Study:
- Introduce Translational Scoring (TS) to increase rigor in AD drug development.
- Provide a phase-specific scoring system (0-25) for AD drug candidates.
- Enhance decision-making for advancing drugs through development phases.
Main Methods:
- Developed a TS system with a 0-25 score range.
- Defined 5 essential scoring categories (0-5) for each development phase (1, 2, 3, regulatory review).
- Provided scoring guidelines and illustrative examples from recent AD drug programs.
Main Results:
- Lower TS scores indicate a need to re-evaluate development programs for missing information.
- Higher TS scores increase confidence in a drug candidate's potential for success.
- The TS approach facilitates informed decision-making at critical junctures.
Conclusions:
- Disciplined decision-making is crucial for successful AD drug development.
- TS is a methodology for rigorous drug development, optimizing each phase.
- Ensures inadequately characterized drugs are not advanced, supporting subsequent phase success.
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