ICON_Jan2021_CellandGeneTherapy - 8

The Critical Role of a CRO in Cell and Gene Trials

right along with our sponsors. With no handbooks

not only an ethics review but will be reviewed

and very few standards, we've had to figure out the

by other departments, four or five even, before

unique nuances of CGT trials and build our own tools

being adopted. This can have an impact on project

and business processes. Because of this, ICON built

timelines which most sponsors do not foresee.

its own CGT Center of Excellence that is a separate

" One of the stumbling blocks we often see is

operating division. Think of us as a CRO within a

when a sponsor develops a timeline they commu-

much larger CRO, if you will. And, by the way, we

nicate to their board or their guiding hierarchy with

continue to learn every day! "

promises that are impossible to manage in cell and
gene therapy, " Fletcher says. " We are very transparent

Moving into a clinical trial

about this and we do it as quickly as anyone can, but

Before a potential new therapy is even administered

if you are not planning your timelines correctly, you

to the first patient in a clinical trial, there are

need to speak to your board to manage expectations.

numerous areas that can increase how long it will

We break down CGT trial execution into journeys-

take from beginning to
end. From writing and

" The 'blindspots' we
find in the product journey are
in the site procedures prior to
shipment and after delivery. "
	

-Tamie Joeckel,

		 CGT Center of Excellence

re-writing protocols to setting

the regulatory journey, the patient journey, the site
journey, the product journey, and the data journey.
They all present unique challenges. " She adds,
" Multiple study stops and starts are much more

up an array of

common in CGT trials. Site activation and recruitment

committees,

plans can be impacted by pauses in the early stages

sponsors that

of the study due to protocol amendments and/or

don't have a

safety issues. In our analysis of 13 studies we were

guiding hand can

running, we found the median pre-FSI amendments

get bogged down.

in CGT trials is three. This is the type of foresight an

" There are so many

experienced CRO should be able to provide during

layers, so many more reviews,
so many more processes that you have to get ahead

planning. "
Surprisingly, even the very structure of a CGT trial

of, or it will draw out and drag out forever, " Fletcher

is a challenge for both large pharma companies and

notes. " It all starts with protocol writing. If you don't

emerging biotechs. Typically, cell and gene therapy

have a well-written and well-thought-through

clinical trials are adaptive studies, a design that is not

protocol, you are doomed from the beginning. "

commonly used due to their complexity. But because

Even the review of the protocol itself is much

today most CGT trials are first-in-human studies,

more involved and time consuming than a typical

sponsors and their CRO partners will need to have a

clinical trial, Fletcher points out, as it will need

game plan for different scenarios that may arise and

8|

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ICON_Jan2021_CellandGeneTherapy

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