Outsourcing Also Starts With the Patient

By Louis Garguilo, Chief Editor, Outsourced Pharma

Whether you develop and manufacture your drug or therapy in-house, or go out to CDMOs for those activities, starts with patients.

Is this axiomatic?

If so, like all self-evident statements, there’s the risk familiarity works against attaining the real attention it’s due. If not, the statement needs further proliferation.

In either case, starting outsourcing decisions with the patient requires decorative detail.

Particularly, perhaps, if you set out as a cell or gene therapy developer. This from an article co-authored by four gene-therapy industry professionals (listed below):

“In conventional medicine, the ratio of the incidence to prevalence of patients for a given disease is expected to be high so that capital expenditures, such as those required to build a manufacturing facility, can be amortized over a number of years.

“In contrast, for genetic diseases, the ratio of incidence to prevalence population can be quite low.” This, says the authors, suggests the “prevalence population” gets treated relatively quickly after launch, and thus a manufacturing facility is needed only to treat the “incidence population,” which will be particularly limited for rare and other targeted diseases.

The logic holds water for a cell-therapy, orphan-market and other emerging developers – and casts significant ramifications across major components of precision (or personalized) medicine pursuits.

For all these, any capital allocated to an in-house manufacturing facility has to amortize at an accelerated pace post initial product launch to realize an optimal outcome – financially, and ultimately for the targeted patient population.

“This can make the business case for building your own facility quite high,” conclude the authors.

Patient-Centric Outsourcing (PCO)

In most wide-lens biopharma discussion, or conversely in specific clinical-trial communique, “patient centricity” suggests conscious designing of potential drug or therapy solutions, resulting trial implementation, and in fact a total health-care system, around the patient’s optimal experiences and outcomes.

In other words, do what’s best for the patient. Another axiom that frequently needs revisiting.  

Focused specifically as we are in these pages on advancing drug development and manufacturing partnerships, patient centricity is defined by those whole patient populations, as our authors suggest above.

If the patient component of the equation (there will be others, of course) lends itself to buying over building to some degree or in totality, then those patients (and the nature and number of your programs) should factor into related decisions such as:

• In which specific segments of our development and manufacturing continuums do we need labs/facilities and partners?
• Which partners (or partner) should we select – and by what criteria?
• How can we (with help from our selected CDMO) design new or utilize existing platforms, technologies, and equipment?
• How do we best ensure quality and supply?

Can You Outsource

The article I quote from above is from (and shares the title of) the ebook: “Insights on Successful Gene Therapy Manufacturing and Commercialization.” The article includes this estimate:

Over 65% of cell and gene therapy manufacturing is outsourced to contract manufacturers.

However, “gene therapy developers may need to wait up to 18 to 24 months before accessing a CDMO’s production capacity.”

Particularly, viral-vector manufacturing capacity is “estimated to be 1–2 orders of magnitude lower than what is needed to support current and future commercial supply requirements” for gene therapy manufacturing.

As reported here, more capacity is coming online, but our authors believe for immediate (and long-term relief, “the most durable capacity increase would come from improving manufacturing practices to increase process productivity.”

They name advancements including engineering cell lines for increased productivity, improving plasmids, and constructs and boosting process recovery are all areas currently being explored. “For example, current process recovery is quite low, less than 20%. If you increase recovery to 50%, manufacturing requirements can be cut by two and a half.”

All the above, then, makes it “critical for developers to establish a manufacturing plan early in the development timeline by securing either in-house or outsourced manufacturing resources.”

And regarding that early plan: The patient population being treated serves as a contributing key consideration.

So patients are key, production capacity a concern … and one other familiar factor: the anguish we’ve reported on for many months over a lack of workers. 

This challenge, needless to say, applies to your hiring your own professionals needed to serve your patient population if you remain internal, and to the current quantity of expertise at your CDMO – as well as plans for future hiring as programs proceed and multiply.

For example, our authors point to a recent report on the current and forecasted skills demand for the cell and gene therapy industry in the UK, which indicates a 112% increase in related jobs from 2019 to 2024.

“There will be a 126% increase in bioprocessing roles including manufacturing, supply chain and logistics, process development and total quality. This increase in the need for skilled employees raises concerns about recruitment or retention required for industry growth. Companies involved in the report also expressed concern that academic courses are not producing industry ready graduates and postgraduates.”

Back To Patients

I’ll end here where we began, with patients and how that vital component relates to us as outsourcers.

We’ve heard in our Outsourced Pharma conferences (and online events), and I’ve related comments from professionals over the years regarding a practice they regard as essential to increasing the attention – and indeed affection – of workers at CDMOs for the programs they are partnering with:

Go to the CDMO, gather the employees, show them videos and presentations describing the actual people relying on the drugs and therapies they are working on. Make it equally about patients as it is the newly analytical data, or sudden deviation, or titer troubles.

Even more in practice today, when possible, bring a patient or patients to the CDMO. Let them speak about their lives and hopes – some of which lay in the hands of the professionals you’ve chosen to work with.

Because if you have decided correctly, you are there at the CDMO facility because you’ve determined that’s the best place for your patients.

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Authors of “Insights on Successful Gene Therapy Manufacturing and Commercialization”:
Brandy Sargent, Editor in-chief, Cell Culture Dish & Downstream
Jane F. Barlow, MD, MBA, MPH, Chief Clinical Officer, Real Endpoints and Senior Advisor MIT Center for Biomedical Innovation
Clive Glover, PhD, Director, Gene Therapy Strategy, Pall Corporation
Tony Hitchcock, Technical Director, Cobra Biologics