When queried about the areas of most importance to them, their companies, and the biopharma outsourcing industry over the next 12 months, experienced professionals from around the world mostly came back with this: cells, cells, and more cells.
For example, Takuya Seko, Corporate Officer and Executive Director, CMC Production, Ono Pharmaceutical Company, Ltd., put it this way: “The key to what transpired in 2017 and looking to 2018, can be summed up by the word ‘modality.’ Specifically in terms of biologics, the substances companies will need to handle will continue to spread from proteins – such as antibodies – to various cells. The raw materials themselves are changing dramatically from chemicals to animal cells, such as CHO [Chinese Hamster Ovary Cells – an epithelial cell line used in the production of therapeutic proteins], and of course human cells. This will continue as the fastest change we all face as drug developers.”
Tina Larson, Vice President Technical Operations, Achaogen, looks forward to what this future holds. “For 2018, I am particularly excited to see how the cell-based therapy companies continue to mature and implement production technologies, product quality testing, and distribution systems suited to one batch per patient. I expect the new approaches required for such quick turnaround and fast assessment of product quality will also inspire the more traditional manufacturers to become more agile.”
Larson says as a part of this development, health authorities will partner with companies “to create regulatory guidance that makes it all possible.”
“It feels very similar to the '90s,” she continues, “when biopharma production using recombinant DNA technology was maturing and blazing new trails. And look at how that transformed the pharma industry over time!”
Julia O’Neill, Principal, Tunnell Consulting, Inc., agrees with all of the above. She says the most important development of 2017 for her work has been “the emergence of gene and cell therapies as viable commercial ventures.”
“This revolution in the way we attack disease is going to be a huge disruptor,” she says. “In the ‘old days’ we were satisfied to find a chemical entity that could reduce severity of symptoms. Now we are precisely understanding the underlying cause of a disease, and going after it at its root, with the potential to cure with a single treatment. And accelerated pathways just amplify the disruptive effect.”
However, O’Neill believes these disruptive technologies cannot move forward fast enough if sponsored by splintered, small start-up organizations. “We need to drive a platform approach,” she says. “CMOs and CTLs [Clinical Trial Leaders] are in a unique position to champion this, and regulators and health authorities can facilitate more rapid adoption of platform technology approaches.”
Taken all together, we see that these three legs – scientific and technological advancements for advanced therapeutic targeting (think cells), engaged regulators and regulatory bodies, and evolving and enabling outsourcing of all kinds – form the drug development stool upon which sits an exciting future for our industry.
An Eye On Outsourcing
The industry, though, must be careful that this is indeed a stable stool. And perhaps particularly keep an eye on that outsourcing leg.
Anand Ekambaram, Executive Director, Global Technical Operations, Biologics and Sterile Products, Merck & Co., Inc., is one who offers some perspective.
“Throughout the last year,” he says, “proactive risk management has become more visible and important in our company, and I believe this is an area of interest for the entire industry. With the growth in volume and criticality of outsourced products, there is a realization of the increasing amount of risk that is now embedded in our external supply chains. These risks can impact the critical path of launch of key new products, and if not properly managed, can have a significant impact on revenue generation. I see a lot more emphasis placed on proactive, end-to-end risk management. At Merck, internal review meetings have been reconfigured or reshaped to give visibility to on-going risks, and to tracking progress of remediation activities.”
Along those lines, Vincent Kosewski, SVP Manufacturing and Supply Chain Management, Kala Pharmaceuticals, Inc., predicts that in 2018, “We will continue to see more integrated and strategic supplier/sponsor relationships to ensure supply continuity.”
Some of this is coming from regulatory requirements that in effect are pushing drug sponsors and service providers closer together. “FDA granted some enforcement relief to manufacturers relative to the 2017 product serialization requirements, but as we move into 2018, work is proceeding at a furious pace to meet regulatory – and customer – requirements. Manufacturers, CMO’s, 3PL’s, wholesalers, and software solution providers are making significant progress against challenging deadlines. It will be very interesting to see the state of the industry at the end of 2018.”
Peter Bigelow, President at xCell Strategic Consulting, knows what he saw by the end of 2017. “I was reminded a number of times just how important it is to select high quality suppliers, develop strong partnerships, and create relationships that motivate and benefit all parties. Unless both sides [and we can insert the regulatory arm as well] of a partnership are able to meet their business goals and carry out their vision, the commitment won’t be strong, and the results will be compromised. We are in a complicated business, and there is much competition for the best resources. Companies that value their partners will come out ahead.”
“I am concerned,” continues Bigelow, “that the incredible changes ongoing in our business will actually create indecision and stall progress. There is an unprecedented amount of consolidation in the industry, product portfolios are under incredible pressure, development timelines are decreasing, and the compliance bar continues to be raised. Executives need to deal with all this commotion, yet continue to get on with business and make good decisions quickly.”
It appears we have our work cut out for us. The entire industry – drug owners, outsourcing service providers, and global regulatory bodies – has a lot of science and technology, engineering, partnering and platforming … and yes, so many cells to pursue in our most immediate future. Patients wait eagerly for us to succeed.