Discussing the key responsibilities and features that pharmaceutical CMC Regulatory functions must possess in order to succeed while applying these characteristics to an emerging topic of your choice impacting industry.

Background Information:

In the already complex field of pharmaceuticals, CMC has become more intricate.  For example, new formulations have changed CMC requirements, dictated by the need to characterize products of increased complexity and the expectation of regulatory authorities that companies will keep pace with the latest technologies to minimize quality risks.

The industry has progressed from synthesizing small peptides to generating full-fledged protein therapeutics routinely on a large scale.  It is familiar with recombinant proteins that mimic endogenous agonists (e.g. interferon, insulin) and is equally familiar with the wave of monoclonal antibodies. Recombinant proteins have become common science in CMC’s standardized development processes with
the introduction of platforms such as optimized expression systems and vectors.

These emerging categories of therapeutics will place new demands on CMC RA, for example, greater cross-functional cooperation and focus on relationships with regulatory agencies.  These trends will demand an appraisal of CMC Regulatory capabilities based on a company’s product pipeline.

Prompt:

Examples of emerging CMC-related topics are listed below.  Choose one from the list or a separate topic of interest.  Summarize the relevance and impact of your topic in relation to key responsibilities and characteristics of a CMC Regulatory function necessary for effectively meeting regulatory requirements throughout drug development.

• Manufacturing processes may now involve transgenic plant cell cultures and transgenic animals
• In addition to commercial recombinant proteins and monoclonal antibodies, there are now commercial cell-based medicines and DNA-based medicines (gene therapy)
• Strategic international regulatory guidances have been adopted that are driving the entire industry to a higher standard of performance, including Quality by Design and Quality Risk Management
• Biosimilars are growing in number with high-revenue biopharma molecules going off patent
• Advanced-formulation products, which have a complex drug delivery formulation and are intended to improve pharmacodynamics and target the drug substance to specific areas
• Antibody-drug conjugates are gaining importance; in this case, the covalent modification redefines the mechanism of action of the mAb, creating the opportunity for a new level of drug potency