When to outsource manufacturing? Advice for cell and gene therapy developers

This fast-growing area in the biopharmaceutical industry is creating a crowded pipeline of products in need of manufacturing and delivery to patients in need. Like the personalized treatments they offer, there is also no one-size-fits-all answer for cell and gene therapy developers looking to determine why, or when, to outsource manufacturing or process development to a Contract Development and Manufacturing Organization (CDMO)[1].

Depending on the stage of their technology, each developer will have specific needs: for example, a pre-clinical company will be more interested in Good Manufacturing Practice (GMP) and Chemistry and Manufacturing Control (CMC) processes; while, a Phase III clinical trials sponsor, will probably need more advice on Regulatory Strategy Guidance and Supply Chain solutions. As such, a reflective analysis of what should be outsourced and how is an important task for players in this field. CDMOs should also take a deep look at their portfolios and what can they offer to their new cell and gene therapy clients, as the demand for more biologics-related services increases.

This is an extract taken from the whitepaper Insourcing vs outsourcing in cell and gene therapy manufacturing. Click here to read the full paper.

Patient population: a key consideration

The patient population being treated is one of the key considerations when deciding between in-house versus outsourced manufacturing to a CDMO. In conventional medicine, the ratio of incidence-to-prevalence of patients for a given disease is expected to be high; so, capital expenses, such as those required to build a manufacturing facility, can be repaid over a number of years[2].

In contrast, for genetic diseases, the ratio of incidence-to-prevalence population can be quite low, especially when it relates to the treatment of rare diseases[2]. This means that the capital invested into building a manufacturing facility for a single gene therapy has to repay over just a couple of years after product launch, which can make the business case for building an own facility unattractive[2]. Also, in order to guide a proper planning of the needed facility, cell and gene therapy companies must be able to establish the design/scale requirements of the manufacture with limited knowledge on the final scale required, future product launch quantities, or even, a correct market penetration. In this case, outsourcing can be an effective and economical bridge, until the real demand for the product is known.

Insourcing: build your “Lego lab”

Nevertheless, for certain cell and gene therapy companies who have difficulty finding a CDMO partner, or even securing production slots in the timeframe needed to deliver the treatment to patients; it might make sense to build a small, early phase GMP facility[2]. Not only that, but the key to success in a fast-moving market as that of cell and /gene therapies, is the ability to access new technologies to improve manufacturing operations and/or a rapid implementation of process improvements. So, being in control of these activities, by in-house capabilities, might be the way to go[3].

Exyte, a construction company for the life sciences industry, has recently launched a brand-new, pre-fabricated cell/gene therapy laboratory facility called RITA, built to work much like Lego blocks with standard highly customizable components that can fit together smoothly in whatever way a company needs[4]. These facilities are designed so that the customer’s unique product features and technologies can easily fit into any space, with a notion of “rather than adapting the process to the facility — it’s a facility that adapts to the process”[5].

This extract is taken from the whitepaper Insourcing vs outsourcing in cell and gene therapy manufacturing. Click here to read the full paper.

References

1. Centre for Commercialization of Regenerative Medicine C. How Can a Contract Development and Manufacturing Organization Advance a Cell & Gene Therapy Product? 2019;26th December 2019.
2. CellCultureDish. Key Considerations in Gene Therapy Manufacturing for Commercialization 2018:33.
3. Harris E. Adaptimmunes’s deep dive into manufacturing & process development. 2019;29th December 2019.
4. Exyte. Design, engineering, construction and fit-out of life sciences & specialty chemical facilities. 2019;29th December 2019.
5. Parrish M. Gene therapies: on the rise. 2019;29th December 2019.