Author: Ken Rubenstein, PhD
Only about 20% of compounds that advance to clinical trials reach the market. Given this high rate of attrition and ever-increasing costs, companies need fallback plans as an alternative to canceling projects and writing off significant investments. Planning optimal backup compound strategies is often difficult. This Insight Pharma Report:
- Describes how companies are addressing strategic and tactical issues concerning backup compounds and predicts how they are likely to do so in the future
- Provides perspective on best practices to adopt in addressing relevant backup compound issues and how evolution in de-risking technologies may affect those practices
- Presents case histories that illustrate the use of backup compounds in practice
- Examines strategic issues in backup compound selection and utilization, as well as tactical considerations, and investigates trends in the field
- Covers business-related matters that bear significantly on backup strategies and tactics
- Analyzes the results of an extensive Insight Pharma Reports survey conducted among individuals active in commercial small-molecule drug discovery
- Includes complete transcripts of interviews with industry veterans who are highly knowledgeable about backup compound-related issues
Backup Compound Strategies: Commercial and Tactical Considerations begins by considering definitions for backup compounds, reviewing key literature references that present expert views on the topic, and presenting case studies that describe how medicinal chemists approach design and selection of backup compounds. Backup strategies currently in effect, and the types of issues companies consider when deciding whether to designate and advance backup compounds once a drug candidate is selected, are examined. These issues include the necessity for backup compounds; whether backup strategies are ad hoc or policy-based; whether to advance backups based on the same or different chemical scaffold as the prototype; how to best deal with addressing backup compound-related questions before a prototype has advanced to the stage where information needed for such decision-making becomes available; and the role of biomarkers and backup compounds in drug repositioning.
Backup Compound Strategies: Commercial and Tactical Considerations then considers tactical aspects affecting backup compound design and selection, focusing first on descriptive tools that are used to assess and compare how close candidate compounds come to meeting requirements for product viability: the target product profile and the clinical utility index. We then focus on the use of biomarkers to help predict how compounds will fare downstream in the pipeline. This includes the effects of biomarkers for predictive toxicology, predictive efficacy, and mechanism of action.
Backup compound strategies are next viewed from the business and commercial perspectives, and the effects of evolutionary changes in drug discovery R&D on backup compound strategies and tactics are considered. Decision and risk analysis tools that are employed, and their utility in dealing with backup compound-related issues, are described before turning to a discussion of backup compounds as viewed from the perspective of the project portfolio as well as with respect to in-licensing deals.
Results, conclusions, and observations from Insight Pharma Reports’ survey of individuals involved with commercial small-molecule R&D on practices, attitudes, and opinions concerning backup compounds are presented. Also examined is the impact of the changing role of medicinal chemistry in pharma, and trends suggesting how backup compound strategies and tactics may evolve in coming years.
About the Author
Ken Rubenstein, PhD, a biochemist and molecular biologist, received his PhD at the University of Wisconsin and postdoctoral training at the University Of Pennsylvania School Of Medicine. He was a key innovator and research manager for Syva Company, the diagnostics branch of Syntex Corporation. During his 13 years with Syva, Dr. Rubenstein became vice president, scientific affairs, and a function that included strategic planning. Since 1983, he has served as a technology and marketing consultant to biomedical companies and an industry analyst, with more than 40 published studies to his credit.