When developing a biologic, great science does not always translate into a great product. Doing the rights studies, the right way, is paramount to realizing the product's potential. Knowing which studies to conduct, when to conduct them and interpreting the data in the context of the product's development, can make the difference between success and failure.
Biological medicines, including therapeutic proteins, DNA vaccines, monoclonal antibodies, and fusion proteins are large, complex molecules that cannot be fully defined by physicochemical analytical methods. They are manufactured from genetically modified living cells using processes that are usually complex. Biological medicines are often 200 to 1,000 times the size of small molecule drugs, and because of the biological nature of the starting materials, the manufacturing processes have inherent variability and product heterogeneity.
For biologics "the product is the process," as the properties of the biologic often depend directly on the nature of the manufacturing process. Manufacturers must ensure product consistency, quality, and purity by ensuring that the manufacturing process remains substantially the same over time. When process changes occur, manufacturers need to demonstrate comparability-that the process changes have not changed the quality of the molecule, and therefore, the safety and efficacy profile. To this end, understanding which of the biological medicine's quality attributes are important, as they are critical in determining whether additional in vitro or in vivo studies are needed to ensure the generated data is supportive for the product's future development.
Early decisions in the large-molecule development process can significantly impact the time to the clinic, progression to the market, development costs, and the overall technical product development risk. If product development aspects are not considered early enough, key studies may need to be repeated, and/or unexpected results explained, both of which can add significant delays and costs.
Sponsors can accelerate their product's development and minimize costs by avoiding common pitfalls of large-molecule drug development.
Given the nature and complexity of biological medicine development, choosing a development partner with proven expertise in biologics is critical to market success. In fact, choosing the right partner with the right experience can help streamline development processes and timelines and reduce your development costs.
Overall, planning early and considering the end goal throughout the development process is critical to realizing the potential value of the biological medicines you are developing and bringing innovative medicines to patients.