ICON_Jan2021_CellandGeneTherapy - 14

The Critical Role of a CRO in Cell and Gene Trials

Insight to Patient Specimen Tracking and Data, " page

of any trial as sponsors make their case to regulators

28). According to Allenson, instead of a steady stream

to get the therapy buttoned down and approved.

of data throughout a study, the bulk of data in a CGT

When you consider that many of the CGT trials have

comes all at once, early in the trial.

been granted accelerated approval pathways, a

" It is a challenge to manage the volume of data

CRO with deep experience in CGT trials and a strong

at the beginning of these trials, " Allenson notes. " In

bench of regulatory experts can help navigate the

most of these trials you come in and give a one-time

complex terrain of gaining approval for this relatively

infusion of the genetically modified cells and then

new class of therapeutics.

you wait for the cells to do their thing. But, while

" You want a CRO that is extremely well-versed

that is happening, we are monitoring toxicities and

in the regulatory process specific to cell and gene

monitoring the patients' labs, and it is generating a

therapies. Because we're working with genetically

large bolus of patient data. If you don't get on top of

modified organisms, it really throws these trials into

that data from the beginning, and stay on top of it,

a much more complicated bucket, and the require-

it can snowball out of control, and you could spend

ments continue to change and vary by region, "

an entire year cleaning up data on a study that's not

says Fletcher. Regulatory requirements and filing

enrolling any more patients. This presents an extreme

timelines for cell and gene therapies vary by product

burden on the sites-especially noticeable during

definition and by country/region.

COVID. We've developed tools that help predict data

According to the American Society of Gene and

volumes that enable us to proactively plan for the

Cell Therapy (ASGCT), gene therapy involves the

resources we'll need. "

transfer of genetic material, usually in a carrier or

As an example, Allenson says that her recent

vector, and the uptake of the gene into the appro-

work with one sponsor had them monitoring and

priate cells of the body. Cell therapy involves the

collecting data from the patients every day once

transfer of cells with the relevant function into the

they had received their therapy. " That's a lot of

patient. Some protocols utilize both gene therapy

bandwidth the pharmaceutical company needs,

and cell therapy. They may use ex vivo expanded or

including boots on the ground medical monitors

selected antigen-speciﬁc T cells, tumor-inﬁltrating

who are also available to pick up the phone, answer

lymphocytes (TILs), or genetically modiﬁed immune

the emails, and provide the information in a timely

cells, including T cells or natural killer (NK) cells,

manner. It has changed the paradigm for how we

expressing novel TCRs or synthetic chimeric antigen

monitor these types of trials from a CRA monitoring

receptors (CARs).

perspective, to a CRO perspective, and even from the
pharmaceutical perspective. "
And, of course, good, clean data are the lifeblood

14 |

ClinicalOMICs.com

The regulatory pathways are subtly different for
each. We can use gene therapies in the EU as an
example of the level of complication. For products
http://www.ClinicalOMICs.com
ICON_Jan2021_CellandGeneTherapy

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