|
Ask the Healthcare
Council - Archive
Q.
How long does it take
new biotech medicines to
reach patients?
A. It
depends on where to live in Europe as some Member
States are delaying patient access to biotech medicines.
All
biotech medicines must
go through the EMEA (European
Medicines Agency) for
marketing authorisation.
These medicines are
among the most
innovative treatments in
the world and are
treating common diseases
like cancer and heart
disease but also
generating a whole new
range of targeted
therapies for rare
disorders like cystic
fibrosis.
According to the EFPIA
Patient W.A.I.T.
indicator, many new
medicines do not even
reach the patient.
There are only two EU
countries (Germany and
the UK) where patients
can expect to benefit
from all EMEA approved
new medicines.
Below
you can see the average
time delay between
marketing authorisation
and market access for
EMEA medicines with
marketing authorisation
from 1 January 2003 to
31 December 2006
|
Country |
Number
of
medicines
with
Marketing
Authorisation |
Number of
medicines
accessible to
patients |
Average time
to access (days) |
Minimum delay (days) |
Maximum delay (days) |
|
Austria |
69 |
36 |
370 |
0 |
1111 |
|
Belgium |
69 |
38 |
503 |
28 |
1054 |
|
CzechRepublic |
55 |
39 |
289 |
0 |
1131 |
|
Denmark |
69 |
21 |
81 |
0 |
1043 |
|
Estonia |
43 |
6 |
289 |
118 |
433 |
|
Finland |
69 |
43 |
151 |
0 |
940 |
|
France |
69 |
40 |
312 |
69 |
636 |
|
Germany |
69 |
69 |
0 |
0 |
0 |
|
Greece |
69 |
51 |
243 |
20 |
867 |
|
Hungary |
69 |
26 |
351 |
0 |
791 |
|
Ireland |
69 |
55 |
83 |
0 |
384 |
|
Italy |
69 |
47 |
333 |
59 |
817 |
|
Netherlands |
69 |
51 |
180 |
0 |
721 |
|
Norway |
69 |
33 |
132 |
0 |
766 |
|
Portugal |
69 |
21 |
137 |
0 |
969 |
|
Slovakia |
60 |
36 |
426 |
153 |
1308 |
|
Slovenia |
60 |
25 |
297 |
0 |
579 |
|
Spain |
69 |
34 |
271 |
24 |
742 |
|
Sweden |
69 |
47 |
140 |
0 |
421 |
|
Switzerland |
66 |
40 |
204 |
23 |
1292 |
|
UK |
69 |
69 |
0 |
0 |
0 |
Table: EFPIA
Patient W.A.I.T. indicator
Reasons behind these
delays vary from Member
State to Member State,
but at EuropaBio we feel
that patients would get
greater access to
biotech treatments if
there was more value
placed on healthcare
innovations when health
technologies are being
assessed in national
medicines agencies.
To learn more about
health technology
assessments and our
campaign to get the true
value of these
treatments acknowledged,
see our
Value of Innovation page.
To learn more about the
Patient W.A.I.T.
Indicator
Q.
What is the potential
impact of genetic
research on new
medicines in Europe
today?
A. Genetic
research is critical to the development of biotechnology-based
diagnostics to better diagnose disease and provide new ways to match
medicine doses and treatments to the individual.
In
addition to providing
effective methods for
appropriate diagnosis,
the results of genetic
research can offer new
medicines and treatments
that target more
effectively the cause of
the disease, rather than
just the symptoms,
leading to a more
effective way of
treating a disease.
Potentially, the use of
genetic research could
revolutionise the
effectiveness of
treatments, allowing for
a more specific
diagnosis, treated with
medicines that are
tailored to respond to
the disease. This
revolution will be most
effective against
diseases that are
genetic in origin, such
as cancer, multiple
sclerosis, diabetes,
cardio vascular diseases
etc.
A
great example of how
genetic research is
making a difference is
in a surgery to reverse
blindness in young
people. Genetic
research that began in
1985 has resulted in a
process by which a
healthy copy of a
defective gene is placed
directly on the retina,
allowing for the
regaining of sight in
the blind. To
learn more about this
treatment, pleas see the
report "Gene
therapy gives hope to
the blind".
In
Europe today, biotech
medicines are estimated
to account for
approximately 20% of all
marketed medicines and
represent 50% of all
medicines in the
pipeline. As more
genetic information is
made available targeted
treatments are the
expected result.
For
more information on how
biotech treatments are
already making a
difference, please see
www.bioimpact.org
Q. What are “advanced therapies”?
A. “Advanced therapies” is an umbrella term that covers the three new
techniques that are revolutionising modern medicine: cell therapy,
gene therapy and tissue engineering. These techniques are already
producing new treatments, and in the future are likely to produce
many more.
In cell therapy, new cells are introduced into tissue to treat a
disease – either from the patient’s own body (autologous), from
another person (allogenic) or from an animal (xenogenic). One
current example is the use of human adult stem cells in treating leukaemia. Around the world, researchers have been making progress
towards using stem cells to treat conditions ranging from heart
disease, spinal cord injury and brain damage to muscle damage, low
blood supply and even deafness and baldness.
In gene therapy, genes are introduced to replace, repair or switch
off defective ones. It has been used, so far with varying degrees of
success, in severe combined immunodeficiency (SCID), cystic
fibrosis, muscular dystrophy and sickle-cell anaemia, among others.
Tissue engineering is aimed at replacing or repairing defective
tissue, and is generally seen as relating to bone, cartilage and
blood vessels such as veins and arteries. This expanding field
covers the techniques of bioengineering and cell biology (and often
cell therapy itself). It is already being used to repair or replace
burnt skin, for example.
More on Advanced
Therapies
Q.
What is an “orphan
medicine”?
A.
Thousands
of diseases and
conditions affect so few
people that without
special support it would
be unlikely that any
company could afford to
develop a treatment or
cure for them. Lacking
a company to sponsor
development, drugs for
these diseases or
conditions are called
“orphan medicines”.
Each “orphan” disease or
condition may affect
only a few thousand
patients, but there are
some 8,000 rare
diseases, most of them
lacking effective
medical treatments.
Although individually
rare, collectively they
affect some 20 to 30
million Europeans and
their families.
The term
“orphan medicine” is
very specific in that it
refers to a diagnosis,
prevention or treatment
of a disease that is
both life-threatening or
seriously debilitating
and which is also rare.
The
European Union has
defined an orphan
medicine as one that is
intended for a
life-threatening or
seriously debilitating
condition that has no
satisfactory method of
treatment and also:
or
The
estimated average
development cost of a
new medicine is
estimated to cost some
US$ 1.2 billion. Orphan
medicines are often no
different from medicines
developed for larger
patient populations.
It’s a risky business:
it takes between 7 and
12 years to develop a
new drug, and just 1 in
every 5,000 research
compounds goes to
market. And, in the
case of
biotechnology-driven
products, development
and manufacturing costs
can also be substantial
investments.
Between
70 to 80 per cent of
these rare diseases have
a genetic origin, so
biotechnology is an
important tool for
developing treatments
for them.
To learn more about
orphan medicines
Back to Ask the
Healthcare Council
|
