PBOA president, Gil Roth, sets out the challenges and opportunities ahead for companies operating in the drug manufacturing sector
New drugs being developed for smaller patient populations are having a large impact on the contracts that contract development and manufacturing organisations are forging with their pharma clients, particularly as many of these products are cures rather than treatments, according to Gil Roth, president of the Pharma & Biopharma Outsourcing Association.
Delivering a keynote address sponsored by Samsung Biologics at the CPhI Festival of Pharma virtual event, Roth said that while there is certainly room for large-scale manufacturing in the overall pharma/biopharma outsourcing sector, products for small patient groups create a different set of manufacturing demands and business models with the shorter runs and facilities and operations that must go “patient to vein” in short order.
“While there’s a great deal of pipeline work in the gene and cell therapy space, there are challenges: many of these products are cures rather than treatments; this implies a frontloading of patient population with diminishing volumes required over time,” he told the event. “Such a model means that CDMO contracts have to mirror this approach; a significant change from the ongoing volumes typically treated in contract relationships.”
He added that one important question going forward is how CDMOs and customers will develop contracts that reflect this new model of cure versus treatment.
The emerging focus on narrower patient populations from specialty drugs, orphan indications or personalised medicines is also influencing pharma companies’ decisions to work with CDMOs as well as the investments that CDMOs make, Roth argued.
“These things shift production away from the large batch/large volume products and head toward smaller batches or individualised treatments and that requires a different way of working with CDMOs which historically were built around large-scale production,” he said.
Roth also identified the current wave of new modalities for treatment as one of the major trends in drug R&D and ultimately drug manufacturing: “We see this in the gene and cell therapy space where revolutionary advances in patient treatment will require new manufacturing models. GCT represent a major change in drug development and patient outcomes and these modalities apply to many different therapeutic areas.”
He said new modalities create challenges and opportunities for pharma companies and CDMOs, such as challenging production, and “scarce” manufacturing facilities and expertise.
“For a start-up or virtual company in this space it makes much more sense to work with a CDMO for development and manufacturing than to invest in internal capacity,” he continued. “At the same time, large pharma companies are making that internal investment but are bridging those efforts by working with CDMOs as they develop new products, and in some instances they may learn that working with CDMOs on these sorts of thing is better than having the internal capacity and change direction partway through.”
Turning to new technologies, Roth said that the US Food and Drug Administration and US Congress have been promoting continuous manufacturing (CM) as a means of onshoring drug manufacturing while simultaneously retaining flexibility for search capacity, reducing the environmental and personnel footprint, increasing quality and reducing the need for regulatory inspection.
He said that while CM is sometimes considered as a magic wand to solve manufacturing infrastructure issues that otherwise are being unattended, “in reality, there’s been discreet adoption of it and it’s almost exclusively been for new products, which makes sense. Changing the manufacturing process for existing products from batch to continuous can be time consuming and create regulatory headaches in multiple regions. Beyond that, the cost involved with the changeover likely wouldn’t be justified for low-margin products.”
Within his presentation, Roth also touched on supply chain disruption such as drug shortages, which were in evidence pre-pandemic, and had led to a call from some quarters to “onshore” drug manufacturing in the US.
“Where drugs will get manufactured and what incentives and mandates will drive those geographic decisions are going to be key questions for the biopharma industry in the years ahead,” he concluded.
Roth had opened his presentation with one very significant caveat; that it is nigh on impossible to predict the foreseeable future, as the spread of the coronavirus pandemic in 2020 has shown.
“There isn’t a single person who was at CPhI [Worldwide] last year who would have foreseen the circumstances we’re currently in,” he said. “At the same time no one would have predicted that vaccine R&D would have made such fast progress nor that contract manufacturing capacity could be ramped up for the projected number of doses that will be needed to get us through to the other side.”