Life Science Leader Magazine Supplements

CMO 2017

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Page 29 of 81

By D. Casebier TIMELINE AND DEVELOPMENTAL ACTIVITIES WHEN RESOURCES ARE LIMITED LIFESCIENCELEADER.COM THE CMO LEADERSHIP AWARDS 2017 28 DRUG DEVELOPMENT Outsourcing MANAGING EXPECTATIONS AND MAKING CHOICES Finally, I'd like to address the concept and great chal- lenge of designing, performing, and particularly accept- ing the results of the "killer experiments." These are the critical experiments that companies undertake and which will determine "in black and white" the viability of a program. Unfortunately, and perhaps too often, when negative results are indeed received, attempts to salvage the asset begin, thus seriously compromis- ing the entire premise of the experiments. Everyone should be aware that while early failure of a drug saves money for the company and investors – and is, in a way, planned for – those running the business have a vested interest in keeping the program (and their positions) alive. Therefore, setting expectations, adhering to prin- ciples, and maintaining original targets are tough. It takes a good deal of discipline to face the failure of a program and shut it down rather than continue in the hope of finding a lifeline. Even short of results that could end development, virtuals, startups, and biotechs particularly should prepare the members of their organization for the unexpected. Material can suddenly fail on stability, prompting a reformulation, limiting storage or packag- ing conditions, and shortening expiry. Issues may arise in scale-up that change the impurity profile enough to require a bridging safety study. A good policy is to list the potential ways that things could go wrong and provide guidance to executive management regarding their cost should those contingencies arise. Working with your partners — CROs, CDMOs, consultants — can help ameliorate negative impacts. Exact costs are almost impossible to project, but even "greater than 'X' and smaller than 'Y'" can help to get folks' attention as to the magnitude of any risk. All stakeholders participating in a small biotech need to be in agreement as to plans, activities, tim- ing, possible risks and remediation, and all known and potential costs. With everyone on board, have confidence in your plan and stick with it. Very few organizations have the challenge of wondering what to do with all the cash and free days on their hands. Smaller drug developers especially need to make the best of all their resources. L set of common-purpose studies, as an individual unit (e.g., Ames, micronucleus, and caco-2; receptor binding/ inhibition panels; physico-chemical studies and prefor- mulation), then build these out with dependencies and timing, with best estimates for expense; and (2) place more flexible activities in gaps that typically occur in development (repeat dosing studies or report genera- tion from service providers). Once these are interlocked, the budgeting becomes significantly easier to project. I like to clearly identify activities that are flexible but still must be completed by certain filing, publication, presentation, or commercialization target dates. These hard-target dates need to be monitored in order to prevent a seemingly small developmental activity from delaying the advancement of an entire program. A major advantage of an integrated plan is the coor- dination of internal and external studies and activities. If external study plans are synthesized with internal activities, the resulting plan can eliminate material shortages and at the same time help avoid redundant activities and expenses (not to mention maximizing safety and data acquisition). These plans may also help project needs for subsequent manufacturing and supply demands, something every contract manufac- turing organization and sponsor knows is difficult to anticipate at best. I typically advocate starting to work with potential commercial API, drug substance, and drug product CMO partners as soon as possible. Late-stage tech- nology transfer is much more challenging, and often more expensive overall, than selecting a partner that can provide services for process optimization into clinical trial supply and through to commercialization. Generally, if a company has helped commercialize a compound, it is also able to contribute that experience to your advancement. DAVE CASEBIER is principal at DSC Consulting LLC. I typically advocate starting to work with potential commercial API, drug substance, and drug product CMO partners as soon as possible.

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