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
- 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.
- CellCultureDish. Key Considerations in Gene Therapy Manufacturing for Commercialization 2018:33.
- Harris E. Adaptimmunes’s deep dive into manufacturing & process development. 2019;29th December 2019.
- Exyte. Design, engineering, construction and fit-out of life sciences & specialty chemical facilities. 2019;29th December 2019.
- Parrish M. Gene therapies: on the rise. 2019;29th December 2019.