Rare Disease Day
March 3, 2014
Keynote speaker of the
GLOBALHealthPR annual meeting,
which concluded on Rare Disease
Day, Alexion Pharmaceuticals
managing director David Kwasha
questioned why patients should
not have access to treatments for
rare diseases.
Kwasha said that considering the
majority of very rare diseases were
often the most life-threatening,
and commonly affected children
and young adults, greater
collaboration was needed between
government, industry and the
public.
“Why should any human be
discriminated against access to
life-saving therapy simply because
their disease is ultra-rare?”
GLOBALHealthPR chair John
Seng said effective communication
would encourage healthcare
authorities to put more funding
into treatment access.
MEANWHILE the US Food and
Drug Administration (FDA) said in
recognition of Rare Disease Day,
that it was focusing on paediatric
rare diseases.
The FDA had been directed
by Congress to issue a report
and strategic plan focused on
accelerating and encouraging
the development of therapies for
paediatric rare diseases, Office of
Orphan Products Development
director Gayatri R. Rao said.
Several agencies were working
together to develop this, and
a web-based educational tool
for patients with a rare disease,
advocacy groups, researchers and
industry had been launched as well,
she said.
“We are committed to working
with the paediatric rare disease
community to face those challenges
head-on and to accelerate the
development safe and effective
therapies for these diseases.”
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