As is the case in the industry, outsourcing of therapeutics is big business. This is true for small molecules, biotherapeutics, and Cell and Gene Therapies (CGTs). Generally-speaking, no matter the type of molecule or therapy, outsourcing presents a host of benefits for Sponsors looking to optimize their manufacturability and de-risk their internal processes.

Cell and gene therapy considerations

Cell therapies, such as CAR-Ts, and gene therapies are extraordinarily sensitive to timing, initial conditions, and other attributes like donor status. All of these antecedents lead to a product profile whose Critical Quality Attributes (CQAs) are paramount to ensuring the greatest viability and chance of therapeutic success with the patient to whom they’re administered.

Outsourcing companies who specialize in this are generally very fluent in minimizing contamination risks and other early-stage production issues that can lead to abject failure. Smaller companies can leverage the knowledge and expertise of CDMOs to produce their products and not have to build their own infrastructure for manufacturing, as well as taking advantage of platforms which exist from some outsourcing vendors. Many CDMOs have great expertise with tech transfers and scale-ups, among other activities, which allow Sponsor companies to bring their designs to reality much more effectively than they could otherwise do themselves—and in many cases, would be so cost-prohibitive, they otherwise probably would never make it past discovery or early development without an outsourcing partner.

Advanced therapies such as CGTs suffer from their own idiosyncratic attributes and challenges that other medicinal therapies do not. Some of these ultimately manifest in the potential for greater Quality and Regulatory challenges, for which CDMOs are often very well-equipped to help navigate as well. While the Regulatory requirements still call for ‘Sponsor oversight’ of each vendor relationship, outsourced production can help tie the whole development and manufacturing package together in a way that makes it much more amenable to successfully entering an approval pathway with a greater probability of success.

Due to their discrete production processes, and current novelty, CGTs haven’t evolved to take full advantage of economies of scale yet, to the degree that they will in the future. Cell therapies average a cost of about $400,000 per patient, and gene therapies average about $2.3 million per patient. Though in some cases, these treatments bring the patient into the realm of a “cure” for certain conditions—so there aren’t years to decades of recurrent prescription charges accruing as in the case of other legacy treatment options. On the payer side, insurance premiums and actuarial calculations are still evolving, based on the treatment options available, initial price tag, reimbursable conditions, effectiveness, and treatment durability, among other considerations. The reason this is important is because the nexus of CGTs necessarily includes the economics of the modality, and the need for profitability of production to render the treatments viable.

Not only does outsourcing make sense from a production and quality standpoint, but it tends to make strong financial sense for Sponsors as well. For this reason, it’s apropos to consider that CDMOs are actually creating the ability for many CGTs to become reality and reach the market. You can look at the situation then as outsourcing being not just a nice-to-have option, but an enabler in large part of CGT treatment options themselves. We may find that CDMOs begin to become constrained with a large number of disparate CGT programs in the coming years, but measuring total capacity-to-program bottlenecks is a difficult task in the CGT world. Especially when economies of scale are brought to bear, and CDMOs can arrange and rearrange their portfolios and production methods in a variety of ways to suit more customers differently as time goes on.

As is the case with many elements in the industry (supply chain, for example), the interwoven nature leads to a situation of interdependence—each element dependent upon the other. And one in which capacity constraints, manufacturing challenges, regulatory changes, and material quality are inextricably tied into the overall outcomes. This is not so different from, for example, the automotive or technology sectors where designers, material suppliers, and manufacturers are in a delicate balance of relying on each other for continued growth and success. And like those industries, the more we do it, the better we get through repeated iteration together.