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That particular paper & the thesis actually came out of the idea that we in The Netherlands
we think that we have very
good basic medical research that we have very good doctors we have
highly ranked hospitals
and then we do a lot of research on the field of orphan drugs and rare diseases
but then finally out of all of these orphan designations that were out there in Europe
I think when I started doing my
PhD work only
six of five or six or seven
orphan designations were
originating from The Netherlands from doctors, physicians, companies, institutions in The Netherlands
and we thought well that's that's very strange all this high
biomedical knowledge, all these clever doctors
then only six orphan designations out of three hundred or four hundred or something
and then we thought well maybe it's about
it's not only about
biomedical science but also about innovation so we started looking at some European Innovation Indexes
But these were much too broad you can't see any correlation between general innovation indexes
and orphan drug development because they include
everything like broadband internet connections to the number of cars, the number of
I don't know alot of indicators
So we thought
we should focus on those
indicators of
innovation that are particularly
related to orphan drug development
one of the first things we thought about was
the amount for money that's being spent in a country
on a private level so by companies and institutions not only by the government but on a private
level
on the development
of pharmaceuticals, pharmaceutical
R&D expenditures.
And then the second one was SME's
Small to medium sized enterprises maybe the motor of innovation in a country
because they're willing to take high risks
they're
close to the clinics and a sort of intermediary between the clinics
and the big pharmaceutical companies.
So we
wanted to find us the number of SME's in each country.
And then the third one was a member of patents because of course patents are an indicator
of innovation.
So basically we grouped these three together; the number of patents
private R&D expenditure on pharmaceuticals and
the number of SME's
involved in pharmaceutical business
And then what you see
for each of these
individual indicators is quite a close correlation between
the indicator
and the number of orphan designations
that are originating from a specific country in Europe
and that's basically
yeah it's basically the finding that we found.
Oh well that that's a difficult question
because
what it really means is
getting the pharmaceutical companies in the country the small ones, the large ones
let them spend money and apply for patents that's basically the idea
and I think that
when we start with patents then there's more and more of the feeling in a number of
countries especially in The Netherlands that if you're doing
good biomedical research
then doctors will
apply for a patent before
for example submitting the manuscripts because that's always..
are you going for a good manuscript in a good journal
or are you going for a patent and then maybe miss the good journal?
and then more and more
patents are being applied for so
maybe if patients are working together with the doctors
Then patients may of course
stimulate this environment but I think that on the other hand it's difficult to
let the patient organisation play a pivotal role in
getting more pharmaceutical companies into Europe
that's something that
must be more on a European level through lobby work ..
I think when we look at the drug development and discovery process for orphan drugs
then a couple of things are really important of course
first the
diagnosis and identification of to patients
and that's something I think that's a classical role for patient organisations
identification of the patients where are the patients what kind of
subtypes do we see throughout Europe
and without
the identification of the patient groups of course
there's no possibility to conduct clinical trials
You've seen that my thesis is built around translation & discover the first chapter
and I think that's the role of the patient organisation in that [phase] is especially
in creating awareness of the disease because
if there's no awareness there's no disease in the eyes of the doctors
identification of the patients
everything around that particular part
and then the second part going from of the designation
towards an approved orphan drug
you see the patients can play a
very big role I think in the design of the trial
Not only the design of the trial from the patient's perspective like
questions like is it
not too much of a burden for us to to do
that's more like the classical things that patients can be involved
but also what kind of a endpoints are relevant
which target population to involve in clinical trials and
which,how to conduct a clinical trial what kind of comparator do we use
I think of course that's the role of the the doctors and the scientists
but patients can have a very large role in that as well
so there patients play a pivotal role I think.
That's something we wanted to include that question in the thesisas well but
it's very difficult to quantify so we can't use that to do an analysis
that's something that's still
from the beginning of the thesis until nowadays
I'm always wondering how to quantify
patients involvement into this
analysis actually
because we're doing one
additional analysis at the moment so ...
Yeah if we can quantify that I'm sure that
there'll be
some influence of patient's organisations
both on the translation of rare disease research into orphan designations
as well as
approved Orphan Medicinal Product.
I'm sure about that but I can't
I'm not able say more without figures
I really think that orphan drugs are the future and
we see
companies more and more companies even the big companies
getting into the orphan drug arena nowadays
and changing their whole pattern
of drug development
..(how do you say that)..
the way that they think
Into getting into
niche markets, orphan drugs at the forefront
and that's I think a very good thing
that should give an enormous boost to orphan drug development in the next ten years
because orphan drug
development was fostered I think the past ten years by a couple of companies who are very
interested like Genzyme and Amgen and these companies,
by a lot of small and medium-sized companies
and a couple of companies that's had drugs in development and they discovered Oh wow
it's for an orphan disease let's try to get an orphan designation for that.
And nowadays you see a real interest into orphan drugs and a real
focus into this orphan drugs arena I hope that that sparks innovation in the next years.
[Comment: They're already talking of the mini-buster as in the blockbuster is gone]
Yeah "niche-busters", "mini-busters" right
and yeah that's maybe the next best works for 2020!