CDMOs to benefit from rising BTDs, orphan drugs and fast track status therapies

But personalized medicines will require new logistics and manufacturing systems.

Bradbury explores how the changing drugs pipeline and increasing diversity of innovators are creating opportunities for the CDMO sector. He asserts that the increase in drug approvals and priority reviews are likely to benefit the CMO industry in the next few years, as manufacturing of these products is often outsourced.

“In 2018, 34 orphan-designed drug NMEs were approved, the highest between 2009 and 2018; this represented 53% of all NME approvals. Of these approvals, 65% were outsourced - the strongest outsourcing propensity for orphan NMEs since 2014. Mega cap companies sponsored five orphan NMEs; only one of these had commercial dose outsourced.”

Priority Review therapies and other accelerated drugs are more likely to be contract manufactured than products that go through standard review. Both small and mid-sized cap companies are more likely to outsource their dose manufacturing capabilities and/or expertise. Therefore, small and medium-sized pharma companies with priority review therapies represent a likely strong stream of new business for the industry in the near term.

According to Bradbury’s analysis, 23 NMEs were approved with Fast Track status in 2018. This was significantly more than in 2017. Of these products, 70% of them were outsourced, which represents a 5-year increase average of 58%. Due to this, the rising number of priority review designations and small-cap companies gaining FDA approvals can only be a positive sign for dose CMOs. Similarly, outsourcing percentages for CDMOs of Breakthrough Therapy Designation (BTD) has never been so high. Demand also remains for high potency APIs, of which around 60% are developed for oncology – and we continue to see rising demand for CDMOs with containment facilities.

In other new implications for the contract services sector, Vetter’s Peter Soelkner predicts that new drugs will have to be produced using a more patient-centric methodology. Soelkner emphasizes that the use of personalized medicines will mean increasingly individualized dosages and packaging. But he forewarns that this will necessitate that contract providers and the industry adapt to help reduce the costs per patient.

Soelkner states that personalized medicines will require new logistics and manufacturing systems, as each therapy is tailored specifically to an individual’s own genetic profile. Looked at more broadly, nearly 40% of the new drug pipeline already involves injectables, and often for smaller patient cohorts. This will continue to present manufacturing challenges, as companies switch from systems designed for larger batches.

“Such systems will need to allow for the creation of APIs in a small scale and possibly, be directly integrated at the filling site. However, we believe that for the main disease indications today’s existing types of therapies will remain standard procedure. For many diseases individualized personalized medicine is still someway off from becoming clinical routine,” added Peter Soelkner.

Personalized medicine helps avoid unwanted costs for insurance payers, by only prescribing these expensive medications for people who are going to respond to it. But these smaller volume therapies are also simultaneously driving up manufacturing costs per unit for each affected patient. To counter these costs, Soelkner suggests that the use of Artificial Intelligence might be solution to help lower the total cost of production.

“Digitization and Artificial Intelligence innovations may help manufacturers and their partners enhancing overall productivity and improve cost effectiveness in the future” added Peter Soelkner.

Stuart Needleman, Chief Commercial Officer, Piramal Pharma Solutions, explores how the patient-centric journey will operate through the pharmaceutical supply chain. He states that there will be a progressive trend across the pharmaceutical industry, with a shift towards lower volume targeted therapies. This has been driven by an increasing amount of research into more complex drugs and compounds.

Needleman argues that one approach (advocated by Piramal Pharma Solutions) is to deliver new ways of motivating the workers so that they have a real understanding of the importance of their work and the effect on real-world patients.

At Piramal Pharma Solutions, Patient Awareness Councils are being introduced across global sites in order to deliver true patient centricity. According to Needleman, “These new bodies comprise cross-functional executives and employees, and they act as the patients advocates and ambassadors for patient centricity though development and commercialization. Moving forward in the future, they will have an extremely important role to play in every project, and are tasked with creating, managing and monitoring the best practices for applying patient-centricity to the entire organization.”

In another emerging patient-focused trend on the rise, Needleman also reports that patients are seeking a greater deal of information, calling them the “globally informed patient”. Consequently, to help improve transparency, we may even see license holders share and celebrate the manufacturing records of CDMO partners.

Orhan Caglayan, Brand Director added: “These results mirror what we are seeing at ICSE – the world’s largest contract services exhibition – where our exhibitors are reporting robust growth in demand, especially for smaller and medium pharma customers who need specialist partners to help discover, develop and manufacture therapies. It is, of course, also why our event is crucial, as we bring together the contract services community so that attendees can meet multiple potential contract services partners, whilst also learning about the main trends in the industry. In the next few years, we anticipate extremely good growth for our contract service specialists and we will continue to adapt our content to showcase the newest technologies that help pharma customers advance products more quickly to patients.”