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Clinical Spotlight

The Future of Clinical Trials:
Connected and Virtual Approaches for rare diseases

Scott Schliebner, Senior Vice President, Center for Rare Diseases
at PRA Health Sciences talks about how a more patient- centred
approach is needed to increase participation and access, and

how technology may hold the key

for Rare Diseases

Clinical drug development is an incredibly
difficult and challenging endeavour in most
disease states; developing new medicines and
therapies for patients with rare diseases poses
an additional set of hurdles and complexities to
overcome. By definition rare disease patients are
few and far between and so often geographically
dispersed and not located near major academic
medical centres. For many rare diseases there
may be only one specialty treatment centre in a
given country, creating physical challenges to
connect a patient to a clinical site and to a
traditional clinical trial. To participate in a clinical
trial, patients and families must often travel great
distances, sometimes to other countries, for
extended periods of time. This is not a realistic
paradigm for most patients and ultimately this
traditional approach is unsustainable for the
future development of new treatments for rare
In addition to geographic challenges, many rare
diseases are multi-disciplinary in nature,
requiring the coordinated care of several
different medical specialists. It is not uncommon
for a rare genetic disorder to require the
involvement of a neurologist, a geneticist, an
endocrinologist, a paediatrician, a psychiatrist,

Coordinating care amongst these many different
specialists, within the context of a regulated and
controlled clinical trial, is nearly impossible in our
current model.
Wit h m any rare diseases affect ing children,
a pat ient ?s part icipat ion in a clinical trial
oft en m eans t heir ent ire fam ily is enrolled
in a clinical trial. Coordinat ing parent work
schedules, sibling school schedules, t im e
away from work, and juggling dom est ic
obligat ions ? all on t op of already having a
child wit h a rare disease ? is overwhelm ing!
Further, the lack of awareness and unclear
diagnostic criteria for many rare diseases results
in patients often embarking upon a ?diagnostic
journey?: visiting countless physicians and
hospitals to better understand their condition
and to seek the appropriate diagnosis and
eventual treatment.

The need for a patient-focused, forward-
thinking paradigm

These challenges and complexities require a new
approach to rare disease drug development,
where clinical trials are adapted to the daily life
and experience of the patients we serve.


Rare Revolution (

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