Backup Compound Strategies: Best Practices for Reducing Phase II Risk
By Ken Rubenstein, PhD
Backup compound planning has risen in importance as the percentage of R&D projects focused on unprecedented targets has skyrocketed. Heads of R&D and chemistry, therapeutic area heads, portfolio analysts, and project managers all recognize that there is no surer way to minimize Phase II risk than a well thought-out backup plan.
This report drives home real world experiences and best practices in backup compound strategies with:
- Case studies of actual backup programs at large and small pharma companies
- Expert interviews with senior R&D managers who discuss the nuts and bolts of backup planning, and offer best practices and cautionary examples
- A proprietary survey of the views, practices, and plans of individuals in R&D involved with backup decision making.
Backup Compound Strategies: Best Practices for Reducing Phase II Risk details the strategic and tactical aspects of backup planning, examining organizational issues and business considerations such as:
- What organizational design ― pure functional vs pure project, with varying matrix forms in between ― is best suited to backup planning
- What to do with backups once the prototype has entered the market
- Determining the level of resources available to a backup program
How Consistent Are Backup Strategies within Your Company?
Source: CHI Insight Pharma, Backup Compound Survey, June 2007 (N=36)
Despite the paramount importance of having well-thought-out SOPs for backup compounds, CHI’s proprietary research shows that nearly 40% of companies surveyed (N=36) do not have a clearly articulated way of thinking about backup compounds, and an additional 14% employs a backup plan only at "some sites." This is not surprising in view of the daunting number of factors to be taken into account in the backup calculation. Such key decisions as the number of backups, when to begin development, and the level of investment required are, in turn, determined by numerous strategic and tactical factors, such as:
- The type of company
- The importance of the therapeutic area to the company
- The importance of the project to the therapeutic area
- The risk of the prototype molecule’s attrition for chemical or biological reasons
- Whether the project strongly meets the target product profile criteria Etc.
The most complete backup strategies include one or more backup programs in addition to the backup or follow-on compounds for the primary compound. These additional backup programs may be based on the same target in the same biological pathway, or on a target in a different biological pathway.
This report is a trove of proven experience and sophisticated thinking about a critical but often neglected R&D decision.
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 Medical School. He was a key innovator and research manager for Syva Company, the diagnostics branch of Syntex Corporation. During his 13 years at Syva, Dr. Rubenstein became Vice President, Scientific Affairs, a function that included strategic planning responsibility. During his 21 years of consulting experience, Dr. Rubenstein has served more than 50 companies and venture capital firms in the biotechnology, diagnostics, and bioanalytical sectors with the emphasis on technology and market assessment. Recent projects have focused on cancer diagnostics, biomarkers, DNA microarrays, homogeneous immunoassays, SNP detection, biochips, genetic testing, point-of-use diagnostics, and biosensors. Dr. Rubenstein has authored more than 40 widely read industry analysis reports and served on the advisory boards of Nugen Technologies, DiscoveRx, and LJL Biosystems (now Molecular Devices).