One of the challenges on the way to commercialization is the transition from preclinical development to the clinical stage. This transition involves, amongst others, key considerations regarding the raw materials that are used in the manufacturing process. Whereas the safety and quality of raw materials often have a lower priority in the initial preclinical phase, they are crucial in the late clinical phases and commercial production.
To enable a seamless transition we recommend identifying the appropriate GMP grade raw materials and reliable suppliers already during early stage preclinical research and prior to clinical development of an ATMP. Changing raw materials at a late stage in clinical development in order to meet the increased regulatory requirements often creates significant additional costs and loss of precious time.
These additional costs are only to a small extend caused by slightly higher costs of GMP grade raw materials. They are primarily driven by the need to perform time-consuming clinical comparability studies to prove the raw material changes do not alter the final ATMP. Switching to the required GMP grade raw materials at an early stage, i.e. directly after pre-clinical development, will prevent the need for such studies and thereby brings a significant economic benefit.
A study performed by the Tufts Center for the Study of Drug Development (CSDD) estimated that the costs of an amendment for a phase III trial costs more than three times as much as an amendment for a phase II trial (read more). They estimated the average direct cost to make changes to the protocols of phase III trials tops $1 million per study.