Small and medium-sized companies generate greater than two-thirds of the clinical candidates in the pharma and biotech industry drug pipeline. They often, however, lack the knowledge and resources required to take a new medicine from the lab to the market and need support from CDMOs that offer integrated, tailored, customized solutions and personalized technical support.
New Growth Engine or a Changing Market
The pharmaceutical industry looks very little as it did even a decade ago. Not only has the focus shifted from the development of blockbusters to drugs aimed at indications for smaller patient populations, the majority of new clinical candidates now come from medium-sized and smaller companies, including those that consist of two employees and a patent.
Notable is the increased interest in the development of drugs for the treatment of rare diseases. In 2014 and 2015 respectively, approximately 47% 1 and 41% 2 of the novel new drugs approved each year by FDA were destined to treat rare or orphan diseases. In 2016 (through November 10), 32.5% of the 19 FDAapproved drugs received orphan drug designations.3
One driver for this growing interest is the lower phase III development costs for orphan drugs, which according to market research firm EvaluatePharma, are generally half those for non-orphan drugs. 4 In addition, according to a recent study of nearly 10,000 clinical and regulatory phase transitions by the Biotechnology Industry Association, Biomedtracker and Amplion, rare disease programs have higher success rates at each phase of development than the average for all programs. 5
It has been reported by the Tufts Center for the Study of Drug Development that smaller firms developing small-molecule drugs have higher clinical approval success rates than large companies. 6 However, it was also noted that “this advantage was offset to some degree by lower returns on approved drugs, suggesting different strategic objectives with regard to risk and reward by firm size.” 7
To complicate matters, a growing percentage of drugs are being developed under accelerated timelines through the use of four FDA programs designed to reduce drug approval times: Fast Track, Breakthrough Therapy, Accelerated Approval and Priority Review. 6 In 2014 1 and 2015 2 respectively, 60% and 66% of the new FDA-approved drugs fell into one or more of these expedited categories. As of November 10, just over 50% of the new drugs approved in 2016 received special designations from the FDA. 3
Alcami has identified approximately 2,800 pharmaceutical companies and universities in the U.S. alone with revenues up to $500 million. Of those, approximately 50% are located in “neglected” states, or states that have little or no outsourcing options. These companies often have limited knowledge of the drug development and approval process and in many cases are unsure what their next steps should be. As a result, they need extensive support and require more than just contract service providers — they need partners.
New Outsourcing Paradigm
The historical outsourcing paradigm involving RFP processes managed by procurement personnel at pharmaceutical companies is not relevant for these small and medium-sized firms. They have limited resources and time; the longer a project takes, the more their minimal capital is consumed. A new approach for rapidly selecting service partners and implementing projects is essential. These companies also need solutions that are tailored and customized for their specific circumstances and include personalized technical support.
Alcami has elected to take an educational approach, showing small and medium-sized pharmaceutical companies what they need to know when they are still potential customers. Rather than these firms needing to seek and find an outsourcing partner, Alcami is actively providing information about the entire drug development process, including regulatory compliance requirements.
In addition to a playbook on drug development and commercialization, Alcami is creating educational events designed specifically for different regions of the country where there are high concentrations of biotech startups and university-based companies. This “create your own forum” approach will serve as a way to connect companies in need of information with experts on all aspects of the pharmaceutical development process — both Alcami scientists and external specialists, such as university professors actively involved in joint programs with the industry.
A second component to Alcami’s approach involves a departure from commission-based sales. This conventional system encourages short-term thinking, which is inappropriate for small and medium-sized businesses that require on-going support. Indeed, our sales approach is tailored to educating customers, and our salespeople are intimately involved in the establishment of educational relationships. They have technical knowledge and can understand all aspects of drug development and manufacturing, and thus are able to help customers identify the technical and operational support they need to progress to the next stage.
Operational Flexibility Is Paramount
Such an educational approach has, as a prerequisite, operational flexibility. Often small and medium-sized companies do not know what they need until shortly before they need it — and many times only after receiving information from our technical sales staff. As a CDMO, Alcami must be extremely flexible and responsive in order to rapidly accommodate these changing requirements.
Continued growth of the company (expectations of portfolio expansion by 3-4 times) with increasing emphasis on the support of small and medium-sized customers translates to the simultaneous pursuit of numerous clinical programs. In order to remain flexible and responsive under these conditions, Alcami is implementing work cells.
Every company has its own set of unique requirements for development and manufacturing projects. Consistent among our clients, however, is the need for project certainty. In response, Alcami is implementing teams of dedicated resources, referred to as work cells, that are fully accountable for the end-to-end lifecycle of management of client projects.
Alcami’s work cells are designed such that the cell either already has or will develop specialized knowledge of client product(s) and processes. The work cell will remain with the project in all phases for the entirety of its duration, as well as with a particular client over multiple projects and years. This level of specialization creates strategic advantage for our clients in the form of change readiness, complexity reduction and elimination of waste.
Close collaboration with our clients along the way take lessons learned and transform them into best practices to ensure future projects benefit from the experiences and knowledge already gained. With Alcami’s global team executing under the same standards and best practices, our clients will experience unrivaled levels of project performance, quality and certainty.
Integration Is Crucial Too
Customers of all sizes, from “two employees and a patent” to small to mid-sized also benefit strongly from partnerships with CDMOs that can provide end-to-end solutions from development to commercialization. Alcami addresses this need by incorporating three components in all of its offerings to support projects at different stages: concept-to-clinic, approval and post-approval. Technical governance, including well laid-out plans from the bench to the pilot and plant scales, is another important aspect of the support offered to smaller sponsor firms. A single program manager also oversees each project — acting as a mentor — listening to and providing the customer with valuable guidance.
Anticipating customer needs and meeting them will ultimately drive business growth – and it is this belief that is driving alcami to implement the “customer comes first” concept in a number of different ways.
Putting the Customer First
The key concept behind Alcami’s proactive, educational approach is that the needs of the customer come before those of the CDMO. This concept isn’t new in many industry sectors, but is a novel approach in pharmaceutical outsourcing. Anticipating customer needs and meeting them will ultimately drive business growth — and it is this belief that is driving Alcami to implement the “customer comes first” concept in a number of different ways.
Our Protect Your Brand™ offering is one example. This innovative solution supports pharma and biotech companies looking to implement a dual sourcing strategy but that lack the resources to secure a permanent second sourcing option. It enables customers to quickly bridge unexpected gaps in a given supply chain without any requirement for a long-term commitment. Customers can secure their supply chains by being proactively prepared on an annual basis for potential future needs.
Under the program, Alcami will validate a product (API or formulated product) in advance of potential manufacturing needs (for clinical or commercial supply) without any long-term commitment and no minimum volume — just validation. We will maintain the required capacity for our partners at an agreed-upon level and be ready on quick notice to accommodate the start of production in accordance with U.S. and international regulatory compliance requirements. As with other offerings, the Protect Your Brand service comprises three distinct supply chain solutions to prevent disruptions from occurring at the earliest concept through to post-approval commercial supply. Customers can also choose their desired state of readiness via our propriety Alcami State of Readiness Ranking system. By responding quickly, Alcami can minimize the effects of a supply disruption, helping to prevent shortages and delays. We do this by using our proprietary state of Alcami Readiness™ scoring system and cover it in our annual state of readiness meeting.
Meanwhile, the ProForm Select™ integrated offering, by addressing both API process and formulation development in parallel, ensures that customers avoid process instabilities and missed clinical milestones. Under the guidance of a single project manager, solid-state chemistry and formulation development occur together, allowing seamless technical and quality alignment and a focus on proactive solutions. As a result, projects move more quickly with improved process stability, mitigated risk and no need for tech transfer.
Conclusion
Small and medium-sized drug companies are the growth engine of the pharmaceutical industry but lack easy access to service providers with the knowledge and capabilities they need to take their promising candidates from the clinic to the market. These innovative firms desire to partner with fully integrated science and technology CDMOs like Alcami that are easy to work with and committed to helping them move rapidly through the drug development process.
At Alcami, we continue to invest in our capabilities to ensure that we can meet the needs of this important customer base, including the development of educational initiatives specifically tailored to meet their needs. Simultaneously, we are working to achieve operational and commercial excellence in all aspects of our business (people, processes and systems) and implementing programs that redefine the role of a CDMO by considering the customer first.
References
- Novel New Drugs 2014 Summary. Rep. U.S. Food and Drug Administration Center for Drug Evaluation and Research. Jan. 2015. Web.
- Novel New Drugs 2015 Summary. Rep. U.S. Food and Drug Administration Center for Drug Evaluation and Research. Jan. 2015. Web.
- Novel Drug Approvals for 2016. Rep. U.S. Food and Drug Administration Center for Drug Evaluation and Research. Jan. 2017. Web.
- New Report Shows Orphan Drug Market to Reach $176 Billion by 2020. EvaluatePharma. 29 Oct. 2014. Web.
- Thomas, David W., Justin Burns, Adam Carroll, John Audette, Michael Hay, et al. Clinical Development Success Rates 2006-2015. Rep. Biotechnology Innovation Organization (BIO). June 2016. Web.
- DiMasi, Joseph A. “Pharmaceutical R&D performance by firm size: approval success rates and economic returns.” American Journal of Therapeutics 21.1 (2014): 26-34. Web.
- “Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review.” U.S. Food & Drug Administration. Web.