A Maturing Market

January 11, 2023 / by Larry Lockwood, Ph.D.

Expectations, oversight and transparency

From the early days of therapeutics development, a common refrain has been that GMP manufacturing takes longer and costs more than expected. This can be traced back to when programs often started in academic laboratories, which then needed substantial analytical and process development as a program moved towards clinical stages.

The disconnect between the academic endeavor and the requirements to move a program into clinical trials could give the impression that working with a CDMO is problematic. The obstacle is more likely a lack of transparency and information sharing as the parties work towards an Investigational New Drug (IND) filing. As the entire industry matures, there are new considerations regarding client experience.

A change in the market
Now, programs are being developed with the understanding of the importance of analytical assays, including potency assays, and reasonable consistency from the manufacturing process. The focus for innovators and therapeutic developers is now on:

• Cost of goods
• Patient affordability
• Durability of the therapeutic treatment with a higher percentage of products being developed for BLA from the beginning

Supporting these changes is a trend toward the use of platform processes, platform assays and regionalized supply chains. While this has always been a desirable state for the industry to achieve, the recent announcement that the Center for Biologics Evaluation and Research (CBER) will "spin off" a group to focus specifically on the review and approval of new cell and gene therapies has highlighted the need for standardization.

Standardizing processes, from critical raw materials procurement through production of drug product such as plasmids, allow development time and IND review time to be systematically reduced because the expectations for these platform processes will be widely known and understood.

With improvements to platform processes and platform assays, regionalized supply chains will be better able to address the needs of the autologous therapeutic community, providing confidence that the process is well controlled, robust and suitable to maintain patient safety.

The need for transparency between innovators and their vendors is critically important. The Innovators are expecting modern solutions to this age-old problem. Developers are seeking out vendors that have electronic notebooks, electronic batch records and electronic clinical data that can be reviewed close to real-time.

This can mean that, in many cases, vendors are scrambling to catch up with these new expectations. We need to remember that, from the innovator’s perspective, these are essential tools that can keep their programs on track and allow them to have the necessary oversight of their manufacturing partners.

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