| Statement
Patient organisations on
"Better Medicines for Children" |
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Patient organisations are very concerned about the situation
in the EU where 50 to 90 (in neonates) percent of the have
not been tested for use in children. This means that existing
medicines have no information on safe and effective use in
children leading to the use of unauthorised medicines which
may result in significant risks, lack of efficacy, unexpected
adverse effects and even death. This also means non-availability
of therapeutic advancesfor children.
"Children deserve as much attention as do adults"
Children represent a vulnerable population with developmental,
metabolic, physiological and psychological differences from
adults. There are no regulations for testing new drugs for
children.
A large coherent effort of all parties concerned (clinical,
research, industry, regulatory and financial community and
of course patient organisations) will be necessary to address
the present, unacceptable situation for existing medicines
as well as to address the situation for new medicines.
The target group is seventy-five million children in the EU,
all aged under sixteen.
Patient organisations are supportive of resolutions, statements
and activities of various stakeholders such as the Council
of the European Union, the European Federation of Pharmaceutical
Industries (EFPIA), the European Forum for Good Clinical Practice
(EFGCP) and the Confederation of European Specialists in Paediatrics
(and its ethical and operational guidelines for good clinical
practice in paediatric research).
Patient organisations welcome the EC- consultation document
from DG Enterprise on "Better medicines for children"
and the invitation for comments.
Patient organisations highly favour the proposed incentives,
regulatory requirements, research funding suggestions and
the attention for greater transparency and expert regulatory,
ethical and clinical supervision.
Patient organisations request attention for
1. Specific issues as the rare disorders, which have been
adopted as a special field of interest and require a special
and separate supportive approach, but also since many of the
future diseases are genetic (65% of all COMP opinions concern
medical conditions affecting children).
2. Collaboration beyond Europe
| a. |
on the use of information and research findings from
countries such as USA and Japan. This is of special importance
for the proposed central database on information on medicinal
products for children including information on newly approved
indications and negative trials |
| b. |
in the field of rare diseases to obtain more statistical
relevant data more easily. |
| c. |
the time before the effective implementation of the
new regulation, which will probably only be in 2004 with
likely results in 2006. Could the existing US data already
be brought to Europe ? |
3. The high ethical and scientific requirements for conducting
research in children. We refer to the great work of the European
Forum for Good Clinical Practice. This forum works, since
January this year, together now with patient organisations.
A strong and adequate European ethical framework specialised
in the ethical aspects of trials with children is desirable.
This also in order to protect the dignity and rights of children
and to see that paediatric patients are not overexposed to
research
4. The need for increasing awareness and education on the
subject to contribute to alertness in the prescription of
medicines for children as well as to motivate governmental
officials, researchers and industry towards a synergistic
approach and investment on the national as well as on the
European level.5. The role patient organisations play and
can play in this field. Many patient organisations have good
relationships with (paediatric) specialist groups at national
and European levels, which would facilitate trials.
Patient organisations favour
1. The creation of an EU-level fund that would support additional
research on existing medications regarding their use in children.
Due to the backlog this fund would need an amount of 200 million
euros which is about the same as the US- Congress provided
for this purpose. Public and private sectors could invest
in this fund. It would be advisable that the proposed regulation
more clearly indicates and defines possible resources for
the fund.
2. Market exclusivity /one year patent extension for suitable
pediatric programmes; and "kid marketing authorisation"
or period of data protection for studies on medicinal products
where no protection of intellectual property exists will be
necessary; these are all providing the necessary incentives
for industry.
Especially in the field of rare disorders, caution should
be paid to the "pediatric rule", which sets the
system as an obligation. One should not add hurdles to an
already complex field.
3 The creation of a central database, the formation of an
EMEA paediatric expert advisory group and the building of
a pan-European network of clinical excellence that would link
existing national initiatives and develop European capacity
and knowledge
Patient organisations are worried about the considerable
backlog. Many medicines have to be re-tested and a large research
investment will be needed. Therefore action and measures have
to be taken in the short term to address this unacceptable,
urgent situation.
Broadly speaking there is a common view on this urgent and
vital subject.
Europe's children will greatly benefit from a well co-ordinated,
well supported, well funded approach.
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