“Strategy” is defined as a course of activities designed to lead to an overall aim. Thus, the CMC regulatory compliance strategy is the course of CMC activities that lead to a defined regulatory objective like initiating Phase 1 first-in-human clinical studies. Defining that CMC regulatory compliance strategy is always the first step; but reaching a consensus on what that strategy should be can be a major challenge for a start-up.
In a preclinical company willing to move to human as fast as possible, two major forces shape the CMC regulatory compliance strategy:
- tolerance of risk
- resource allocation and cost
It is also key for small Biotechs to monitor the CMC strategy implementation and progress to ensure that the limited resource of the company are not wasted but focused on critical aspects of the drug development strategy.
When establishing the CMC strategy 5 key elements should be addresses:
- Align CMC activities with company objective
- Address the specific challenges of the Bioprocess
- Address quickly the potential safety product issue
- Follow the ICH guidance
- Set-up a risk-based CMC approach
Big Pharma companies have done well with CMC over the years, as they move away from a focus on R&D to one that emphasizes acquisition strategies and licensing.
Small Biotech companies do not always understand the importance of the Big Pharma to have a clear CMC strategy. Some take the approach of « Let’s run as fast as we can with whatever works, and if there’s a problem, we’ll deal with it later ». Small biotechs and investors should have in mind that establishing the proper CMC strategy is crucial to protecting the investment. Big Pharma will be reluctant to buy a process that cannot be scaled up; has dozens of impurities that were previously undetected by Small Biotech CMC quality control group; may not have sufficient stability; or may have been created with an uncharacterized cell bank.