Drug development can be expensive and risky. Less than one-tenth of candidates that leave discovery reach commercialization, and the cost of development to market may reach billions of dollars. At the same time, candidate molecules are extremely complex, the need for product differentiation is greater than ever, and developers more frequently have ambitions to take their program further along the path to market so that they can realize greater value when it is sold or licensed.
For these reasons, gaining a solid understanding of a molecule’s characteristics is vital to a program starting well.
Ask the right questions from the beginning
At the beginning of a program, when money and materials are often in short supply, it pays to collaborate with a partner whose team has deep experience in early phases of development. This experience ensures the right questions are being asked and answered from the start. Asking the right questions will help guide customers to early milestones, ensuring informed decisions can be made with minimal investment.
For example, despite the advent of in silico techniques and the now ubiquitous AI, a program that screens and selects the most promising preclinical candidates needs to be formulated in a way that is both practical and iterative. More small molecules exhibit sub-optimal physicochemical characteristics, and it should be considered early on how ongoing development will address issues such as poor solubility and bioavailability.
Attention to chemistry, manufacturing and controls (CMC) is also important at this stage. CMC activities include the establishment of manufacturing processes along with defining product testing methods to ensure that the product is safe, effective, and consistent.
By taking a planned approach and prioritizing those tests and decisions that will have the greatest impact, a program can be progressed with less waste and reach the clinic faster.
Make phase-appropriate decisions
Given that materials may be in short supply, development programs should be designed around each phase. An experienced contract development and manufacturing organization (CDMO) will counsel clients to start with only the material they will need to reach their goal, maximizing budget and time for them to demonstrate that their candidate molecule meets expectations. For smaller or early-stage innovators, this may even help them gain further funding.
Through a phase-appropriate approach, various aspects of pharmaceutical development can be considered early on and future needs that will become important later, such as efficient manufacturing, tech transfer, and dose-scaling, can be better anticipated.
A strong partner balances phase-appropriate program needs with a holistic understanding of the customer's program, including consideration of what will be needed as development progresses. By taking this approach, waste can be avoided and a program’s journey to the clinic, and ultimately to market, can be accelerated.
Consider scalability
Innovators often seek partners with end-to-end capabilities, from early-stage development to commercial supply. Early-stage companies typically lack the in-house expertise needed to bring a molecule from bench to market, and larger companies may wish to avoid the complexity of multiple suppliers. Partnering with a single provider conserves resources and reduces risks associated with tech transferring a program between organizations as its scale increases, especially when managing cultural differences. A reduction of suppliers also means a reduction of complexity. Having a partner that can support your program from early development to commercial production under a single roof enables secure supply chains that will ensure reliable materials as demand grows.
Decisions made at the early development stage may also have a profound effect on the program as it matures. It is therefore important to work with a partner that is able to consider and advise on factors such as how program risk will be managed, if a formulation can be developed that is both scalable and stable, and even on strategies for how intellectual property and regulatory considerations will be handled.
A responsible early development partner will also establish an OPEX mindset starting in early phase to ensure scalability. At an early stage, programs may only need as little as 50-grammes of material, but it is vital the client can assure themselves of a reliable supply of materials, made under GMP conditions, at scales of 100-kilograms and beyond.
For all these reasons, it's very important for scalability to be considered when choosing a partner to ensure accelerated speed to clinical trial.
The Siegfried Acceleration Hub
The Siegfried Acceleration Hub based in Grafton, Wisconsin has expertise and experience across all of these success factors. It helps its customers to maximize speed to clinical by:
- Bringing the project on the right path right from the start
- Ensuring phase-appropriate development
- Making sure that scalability is considered from the start
This site is part of the global Siegfried network, which offers development and manufacturing services from early phase all the way to commercial production and drug formulation under one roof. With this experience, it is well positioned to deliver strategic insights and start a program on the road to speedy and successful commercialization.
If you are interested in learning more about the Siegfried Acceleration Hub and its capabilities, contact us.
