By Kevin Kane, PhD, Senior Staff Scientist, Research and Development, Thermo Fisher Scientific, and Audrey Kelleman, PhD, Senior Manager, Business Management, Thermo Fisher Scientific
Small molecule drug development has changed substantially in recent years. With the heightened focus on molecularly targeted therapies, small molecule active pharmaceutical ingredients (APIs) and drug products are more complex and potent than ever, requiring increasingly specialized manufacturing processes and drug delivery solutions. At the same time, the competitive demand for rapid entry into clinical development—combined with accelerated review pathways—translate into compressed manufacturing and delivery timelines.
These dynamics challenge sponsors to balance the need for speed with optimal execution of chemistry, manufacturing, and control (CMC) activities to ensure the quality of the finished product during all phases of development. Achieving this balance is particularly challenging for new and emerging biopharma companies who are dominating the pipeline and opting to hold onto their molecules possibly through commercialization. Without the resources, expertise, and bandwidth of large pharma, these companies rely on strategic partnerships with consultants and contract development and manufacturing organizations (CDMOs) to support their development programs.
The more distributed the development team, however, the more risk there is in advancing molecules to initial clinical studies. In many cases, drug substance, drug product, and clinical strategy activities are spread across multiple companies or consultants. This translates into multiple technology and process transfers across the development lifecycle and introduces multiple points of potential vulnerability. Minimizing risk while accelerating progress to clinic requires a collaborative approach to project execution built on an understanding of the process and science involved in each phase of development and the impact that phase-specific decisions have down the line.
In this white paper, we discuss key strategies for efficiently advancing small molecules from API to pre-clinical data and clinical supply.