Development of a new chemical entity (NCE) as an active pharmaceutical ingredient (API) is a complex, expensive and risky business. The development path needs to be meticulously planned and executed to minimize challenges and roadblocks. To ensure speedy development to clinical phase, it's crucial to ask the right questions from the start. It helps make the best possible decisions about how to progress a molecule through the development process and how to move forward as quickly and as cost-effectively as possible.
When evaluating project needs, there are some key questions to be considered when looking to accelerate a program to clinical trial:
First and foremost, you need to ask yourself: What is my end goal? Invariably, the answer will be to commercialize the molecule as soon as possible, but this is a vague and open-ended statement, and much more granular details are needed. Leading to additional sub-questions like: Do I have the sufficient financial resources to progress the molecule? Do I want an ideal, robust and scalable process developed immediately, or can it to be done as quickly and cheaply as possible at first, and improved later?
Next, there are questions about timing. What do you actually need immediately and what is on the list of things that you would like to have, but are less time-critical? You may want everything right away, but it might not be necessary to move the project forward. If it’s just going to add time and cost, and not add value or answer critical questions, then it might be better to delay it. Of course, if it’s clear it will be needed at some point in the future, then set out a clear plan for how it will be achieved.
Another important question to ask is how the molecule can be made if the volume requirement is increased? If it’s a new molecule, your medicinal chemists might only have made a few milligrams of the material, and the synthetic route used is unlikely to remain viable as volumes increase. For example, discovery routes often include reagents that are challenging to work with on a large scale or may require an expensive purification process. Designing a new manufacturing process will be an important factor driving future success and uninterrupted supply of material.
A few other examples could include a natural product extracted from a rare plant or natural source for which a synthesis might not even exist, and if it does and is patented, can it be licensed? In either case, you would be unable to make the kilogram or more of material you need for preclinical studies without first developing a suitable synthetic route, hence knowing your limitations is of utmost importance.
Of course, there are questions about the molecule itself: Does it pose any clear safety risks? If it is deemed highly potent, for example, then the appropriate level of containment will be required while it is being manufactured and handled, adding further cost and complexity. And what about the raw materials, intermediates and reagents used during the synthesis? Do they pose any handling or environmental risks? How might these be mitigated?
Further questions cover the supply chain, such as whether you are seeking a single source. Where do your raw materials come from, and if they are only from one part of the world, do you want to diversify the supply chain? Do you want to make them in house, or do you want an external vendor to do it for them?
Then, looking to the future, there are questions about what teams are already in place and what additional teams will need to be developed. This will almost certainly include analytical and regulatory groups if they do not yet exist. Once the material is made, a range of analytical work will be needed to meet the CMC requirements of regulatory filings. And, of course, there’s intellectual property – do they have this in place, or are they infringing someone else’s IP, creating a potential legal issue in future?
The Siegfried Acceleration Hub in Grafton, Wisconsin USA offers early-phase development and manufacturing services. Our skilled team has deep experience in all aspects of progressing a project from the med chem discovery lab through to clinical trials and commercialization.
The Acceleration Hub is part of Siegfried's global network of 13 sites globally. Siegfried is a leading CDMO offering development and manufacturing of pharmaceutical ingredients and finished dosage forms as an integrated supplier. Access to Siegfried's extensive development experience provides a strong foundation for customer programs, ensuring high-quality processes and development efficiency. The possibility to seamlessly transfer to Siegfried's global network of pilot plants and launch plants also enables smooth scale-up all the way to commercial manufacturing.
In conclusion, the Siegfried Acceleration Hub team is committed to providing the support you need – collaborating with you by asking the right questions from the start and successfully achieving the milestones. We at Acceleration Hub work in close partnership with clients of all sizes. Our experienced and seasoned team can help you to set your program on a speedy and successful commercial launch by asking appropriate questions at the right time.