The CMC Essentials Training course is running full steam ahead!
Some key take-aways from the first 3 sessions:
We discussed how the Risk-Benefit paradigm evolves during product development, and how it is an important guide for companies to balance their CMC development efforts- doing too little can harm the patient or get regulatory pushback, and doing too much can burn the company's budget, to the point of extinction.
We saw how every product is a unique snowflake, with its unique chemical / biological features, and how they are the basis for the rest of the work in CMC.
The nuances that make our lives easier with small molecules (be it their ADME profile, stability and manufacturing) are the points we struggle with as the product becomes more complex (e.g. recombinant proteins, monoclonal antibodies, gene therapies and the (current) peak of Cell therapies).
We swished through the scientific principles behind key manufacturing technologies: from the deterministic nature of Organic Chemistry, through the fussier (probabilistic) nature of Molecular biology and cell cultures, and a brief intro to the engineering principles of purification technologies...
And last week: We rolled our sleeves and started working the daily events of the CMC life.
And what a better place to start, then the actual manufacturing floor?
We saw what is so special about Pharmaceutical Manufacturing facilities, and what it takes to build one (TL;DR: Lots of requirements, translating to lots of Money).
We discussed the Pros and Cons of manufacturing in-house vs. outsourcing manufacturing to a CMO (Contract Manufacturing Organization).
We saw how for most start-up companies, the lowest risk approach is to start development in a CMO, that already has the facilities and the know-hows, and complies with GMP.
The closer companies get to Commercial phase, the risk-benefit balance flips towards building their own commercial plant, and that's what many companies choose to do.
In between, we mentioned there are many other types of arrangements, since its not black and white.
We spent a great deal on understanding the practical meaning of GMP, because we saw it doesn’t matter where you manufacture your drug: in-house, or outsourced, it is you, the Pharma company who is “ultimately responsible to ensure processes are in place to assure the control of outsourced activities and quality of purchased materials.” – ICH Q10.
We discussed the typical workflow: from CMO Selection to first GMP batch, and shared some tips and tricks to make life easier.
Next week, we will look into the Heart of CMC: Manufacturing process and Pharmaceutical Development. We will start from scratch, but also see the end goal, with what ICH Q11 and Q8 tell us, and understand the concepts of Comparability and Technology Transfers.