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I think it's a partnership
I think that Europe
paved the way in many
ways
for the COMP (Committee of Orphan Medicinal Products at the European Medicines Agency) to have patient organizations
on the Committee of Orphan Medicinal Products
I think that companies
are increasingly realizing
that the patients know their diseases better than anyone else does
For many of these very rare diseases we do not know much
about the natural history of the disease
and so it's the patients
that certainly know that better than anyone else
consequently
uh I think that
we will see more and more patient involvement
with
the companies.
Patients also put pressure
good pressue
you know please don't
stop the development of this
our patients need it
and so
listening to that
companies will look at
a drug that they may have been considering dropping
either because they didn't think the interest was enough or there were some questions
about
safety or something
They'll look at it long and hard and see if they can figure out
a better way to develop the drug
and
everyone US and Europe is looking at better ways for drug development
our current drug development process
is what it is but we need to
develop a faster more efficacious
less traumatic
less expensive way to do this we can't afford it otherwise.
I think that
I'm sure they're green shoots yet but they are buds
hoping to break through the spring soil
but
I think we're going to be looking more at surrogates and biomarkers
I think we have to be certain
that they are real
and that's always a concern
and safety remains such a concern
that we have to be ever vigilant a lot more post marketing
surveillance because that's when you find out the real safety
and not being
afraid
to say
look we approved this based on the best information possible
we have to re-look at this now
I think that as the genome project
the genome initiatives
go further down the road we'll learn more about
how to better target therapies and target
safety issues
we're on the threshold of some really nice things
we're just not quite there yet.
I didn't know much about intellectual property and with
Orphan Drugs and Rare Diseases
it is a strange
basket
because on the one hand one needs to protect
intellectual property so that you know that the project is yours
on the other hand if you have fifty patients in a country
who's gonna, the competition isn't there
the scales don't move quite appropriately to know where they are at any one
time
here's where I think the patient groups will be very very helpful
because
pharmaceutical companies investigating a disease looking at a drug for a disease
their natural inclination is to go toward what's
the fastest and the easiest
because they have a slightly not completely
but a slightly different
view
they to make money for their shareholders
uh they have to show that they're productive
yes they are are interested in the patients without the patients they don't exist
uh they're not nasty uh people
but they have to make a success
so that they can continue to help people
but with patients saying but
but but
but we need we need we need I think progress will be made
whether opening everything to everyones scrutiny
would do anything I to know
uh I just don't know enough about it and I can't comment.