The latest Horizons: Life Sciences report, clocking in just short of 100 pages in length, relies on feedback from approximately 500 respondents from North America and Europe. A significant number of those respondents are managers, directors, vice presidents and CEOs.
“This issue has primarily focused on research and development,” said Peter Walters, director, advanced therapies at CRB.
Walters said a handful of strong trends resounded across the market as a whole. “The first is this idea of companies diversifying to chase discovery,” he added.
The pandemic helped catalyze innovation across the biopharma industry, but other factors, such as the mapping of the human genome, continue to help drive the field forward. In addition, an improved understanding of genetic-based disease has helped spark a massive rise in cell and gene therapies.
“Now, RNAs are taking a spotlight as a result of the global pandemic that put them into the world view,” Walters said.
Here, we highlight a number of trends affecting the biopharma industry that were featured in CRB’s latest Horizons report.
1. Multimodal buildings help drive innovation
As many pharmaceutical companies pursue diverse therapeutic approaches, the biopharma industry is seeing a rise in multimodal buildings. “So, 5–10 years ago, you might see one building dedicated to R&D and one building dedicated to another therapy’s GMP,” Walters said.
But now, biopharma industry is focused on creating more collaborative spaces — both in R&D and the GMP environments. “We’re seeing a new building model that embraces all of that,” Walters said. “One building could offer multifunction capability in terms of R&D and GMP manufacturing.”
More than 90% of the respondents to the CRB survey said they were pursuing a multimodal initiative of some kind.
2. Rising use of contract manufacturing
The pandemic helped drive reliance on contract manufacturing organizations (CMOs)/contract development and manufacturing organizations (CDMOs).
Roughly five to ten years ago, CMOs had a place in the industry, but “it was definitely more suppressed” than currently, Walters said. Then, companies tended to use partners to help fill production lots or accelerate the timelines at an early clinical development phase.
During the pandemic, CMOs helped pharma and biotech companies with supply chain management and clinical development at large. Staffing shortages (see below) have also driven the industry’s reliance on CMOs. “Now, over half of respondents are planning to use or are using CMOs to support their pipeline,” Walters added. “CMOs have essentially cemented themselves into the bedrock of how biotech exists.”
3. Staffing shortages persist
Even before the pandemic, many companies focused on science and technology struggled to find skilled workers.
The problem has only persisted in the pharmaceutical industry. “Staffing shortages are a massive weak point in the industry right now,” Walters said.
The shortage of skilled workers is also fueling partnerships across the biopharma industry.
The growth of the cell-and-gene therapy market also contributes to the staffing shortages. Cell and gene therapies often rely on manual processes. Companies developing cell and gene therapies can’t, for example, hire a worker straight out of high school to perform routine operations. They must rely on highly-trained technicians with significant knowledge. “The industry is hiring doctors, in some cases, to do these processes,” Walters said. “So The available talent pool is not only small to begin with but is being consumed by the industry as fast as the workers are available.”
4. Pharma 4.0 adoption ticking up
While the pharmaceutical industry’s interest in Pharma 4.0 initiatives has gained momentum during the pandemic, the limited talent pool is one factor slowing their adoption. In addition, the sector must contend with shortages of both skilled workers and a lack of labor capacity in general.
There are, however, a considerable number of workers with experience transforming paper-driven facilities to adopt islands of automation or begin to implement automated facilities. “That’s a model that’s been looked at over the past 10–20 years,” Walters said.
Conversely, finding experts who can help implement a fully integrated adaptive facility with predictive, AI-driven capabilities is more challenging. “Unless you have somebody who knows how to implement it, you’re stuck at the planning stages,” Walters said.
CRB uses a digital plant maturity model popularized by BioPhorum and the International Society of Automation. Level 1 is predigital. Level 2 has digital silos, while Level 3 involves a connected plant. Levels 4 and 5 involve predictive and adaptive capabilities, respectively.
“At L2, you have some islands of automation,” Walters said, referring to Level 2. “L3 is the system standardization phase. At L4, you’re getting into networks and real-time predictions and analysis. And then at L5, you have a fully 4.0 plant with AI-driven, self-optimizing autonomous systems.”
Most companies at present peg themselves at Level 3. Outfitting a plant with Level 3 capabilities is relatively straightforward with programmable logic controllers (PLCs) and other connected systems.
Most companies with Level 4 capabilities are Big Pharma firms with deep pockets. Some of the more advanced companies are working on outfitting isolated systems with Level 5–inspired capabilities.
CRB found that CMOs generally lagged behind Big Pharma companies in Pharma 4.0 adoption. Similarly, smaller biotech companies generally trailed larger biotech companies in their technological maturity.
Making the leap from Level 3 to 4 is no small feat. “Maybe people don’t realize that it’s a significant change to how you do business and that change can cause a fair amount of disruption,” Walters said. For example, moving from a paper batch record to a fully automated digital batch record system could affect manufacturing workers and a company’s process-developing group.
“We are seeing some facilities that are putting in some of the placeholders for some of these technologies so that when the companies are ready to take that jump, it’s kind of been pre-enabled,” Walters said.
5. Cloud and edge computing gaining ground
In terms of emerging technologies, cloud and edge computing were the furthest in adoption. “Internet of Things was fairly high,” Walters said.
Technologies such as quantum computing, digital twins and augmented reality were at the bottom of the list in adoption.
Augmented reality, however, is gaining popularity but also remains at an early phase of deployment. “I’ve seen a lot of demos around the power of augmented reality — having someone from manufacturing be able to essentially train on the fly with equipment,” Walters said. For example, a worker could put on AR goggles to access an SOP. Or they could use AR to scan a code on a piece of equipment to read a user manual or contact a remote technician.”
But while such AR applications are powerful, they require significant infrastructure.
6. Cell and gene therapies and RNA technologies offer promise and pitfalls
More than 300 respondents to the CRB report had cell and gene therapies in their pipeline. The segment is one of the “most dynamic—and challenging” in the Horizons survey.
While cell and gene therapy adoption is ramping up, regulators are keeping a close eye on them. As a result, many cell and gene therapy companies closely monitor patients for long periods. “Some of these patients are reporting back for years after receiving therapy,” Walters said.
In October 2021, FDA placed a clinical trial (NCT04416984) on hold for Allogen’s AlloCAR T therapy after identifying a chromosomal abnormality in a patient enrolled in the trial. The incident caused the company’s stock price to fall nearly 50% at that time. FDA later concluded that the abnormality was unrelated to Allogene’s AlloCAR T therapy.
Interest in RNA technologies is also surging thanks to their potential to accelerate development timelines and reduce costs. The CRB report lumped RNA therapies together, including non-coding and coding RNA. In addition, the report uncovered a range of delivery systems that reflects the diversity of applications and subtypes in the RNA market.
Given the widespread use of messenger RNA–based COVID-19 vaccines, the industry has seen a “huge uptick in the use of lipid nanoparticles and protein nanoparticles and lysosomes,” Walters said.
While vaccines helped put RNA on the map, growing interest in RNA drug products could lead to an ever-more-diverse RNA landscape in the coming years. Interest is growing, for instance, in naked administration where RNA is injected locally without a carrier.
In 2021, FDA approved a novel siRNA from Novartis that could reduce cholesterol with two annual doses, offering a potential alternative to statin therapy.
Filed Under: Cell & gene therapy
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