ICON_Jan2021_CellandGeneTherapy - 21

Cell and Gene Therapy Clinical Trials - The Promise and Complexity of Living Therapies

CO: Do the services ICON provides differ in

because the population is so reduced and

trials for rare genetic diseases, Parkinson's,

scattered geographically. This is where patient

and others versus those for lymphoma or

advocacy interaction can be critical in " heat

leukemia using live cells?

mapping " patient populations, sites, and KOL's.
A lot of focus is on organizing awareness, educa-

TJ: ICON is fortunate to have a rare disease center

tion, and offsetting safety concerns-building

of excellence with significant experience in the

trust-in the trials. These

global regulatory and clinical operations path-

patient-centric trials

ways. We have successfully filed over 60 orphan

also involve high-

drug designations. When you combine their

touch, concierge

therapeutic area expertise with our support-

services for both

ive cell and gene therapy experience, it's truly

the patients and

a rare combination (if you'll excuse the pun).

their caretakers.

The challenges in programs for rare and orphan

Home health ser-

diseases-patient recruitment and education,

vices can be a huge

finding the right investigators and KOL's, and

value add to these

recruiting the right sites-don't change that

trials to alleviate the

dramatically from an operational standpoint

burden of site visits for simple tests and assess-

until you add the CGT component. I think the

ments. We've found this to be especially useful

area of rare and orphan diseases really exempli-

during the pandemic-bringing the trial to the

fies the value ICON brings to our sponsors. We

patient whenever possible.

" We've found [home
health services] to be
especially useful during
the pandemic-bringing
the trial to the patient
whenever possible. "

offer the therapeutic area expertise that is supported by an overarching and complementary
cell and gene therapy center of excellence.

CO: Is the competition for clinical trial sites for
these diseases and for PIs as intense as they
seem to be for oncology CGT trials?

CO: Is patient recruitment a bit easier for
rare diseases due to existing research

TJ: The competition for the " most desirable " sites

organizations focused on some of these

is indeed an issue. Especially in cell and gene

diseases?

therapy. We often encounter sites that are
overburdened and may not have the resources

TJ: Let's face it, one of the major challenges in any

to dedicate to smaller programs. ICON has an

clinical trial is finding patients-and it's even

extensive global site intelligence database that

more complicated in rare and orphan diseases

we use to look at suggested sites. With rare and
ClinicalOMICs.com

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http://www.ClinicalOMICs.com

ICON_Jan2021_CellandGeneTherapy

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