Medicines for
children in the European Union
The patient perspective |
|
By Y.S. Poortman
Seventy million children at risk
There are seventy-five million children in the European Union
all under the age of sixteen, who are at risk when taking
medicines. With the enlargement of the EU this will soon be
over hundred million children.
Fifty to ninety (in neonates) percent of the available medicines
have not been tested for safe use in children and consequently
lack information on safe and effective use. "Children
do at least deserve as much attention and carefulness as do
adults" (Byrne)
Children represent a truly vulnerable population with developmental,
metabolic, pharmaco dynamic and kinetic, physiological and
psychological differences from adults. There is a high level
of concern among Patient Organisations in the Europe Union
about the safety and efficacy issues, the limited amount of
age-appropriate research and the lack of regulation accorded
to medications that are currently being used in the treatment
of children. This situation results in significant and unnecessary
risks, lack of efficacy, unavailability of therapeutic advances,
unexpected adverse effects (chloramphenicol,thalidimide, diethylstilbestrol,
tetra cycline, valproate, asperin &) and even death among
this age group.
The importance of the subject "children and medicines"
will considerably grow in the near future because of increasing
insights from genetic and molecular research and the increasing
possibilities for early detection of disease . Consequently
medicines will play a more vital role for children in prevention,
suppression and/or delay of disease expression.
A large joint and coherent effort
A large coherent effort of all parties - clinical, research/academia,
industry, regulatory and financial community and last but
not least the patient organisations - is necessary to address
the present unacceptable situation for existing medicines
as well as for applying new knowledge and technologies for
the development and production of new effective and safe drugs.
Patient Organisations greatly welcome the EC-DG Enterprise
action on "Better medicines for children" and the
invitation for comments and input. They 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 are supportive of resolutions, statements
and activities of those various stakeholders in this proposal
- 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.
Special Initiatives
Patient Organisations would like to see specific initiatives
developed in the areas of:
| 1. |
Rare Genetic Disorders that require a special and separate
supportive approach and have been adopted as a special
field of interest by Patient Organisations. New technologies
using molecular diagnostic tests will be used more and
more to detect genetic mutations linked to various diseases.
Sixty five percent of all of the opinions of the Committee
for Orphan Medicinal Products (COMP) concern those medical
conditions (usually Rare Diseases) affecting children.
In respect to rare disorders special attention is asked
for the communication on the consultation paper by the
European Organisation for Rare Disorders (EURORDIS) which
is fully supported. |
| 2. |
Collaboration Beyond Europe:
 |
By 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 about medicinal products for children
that will include information on newly approved
indications and negative trials. |
|
By obtaining more statistically relevant data
more easily In the field of Rare Diseases. |
|
By shortening the time period between now and
the effective implementation of the proposed new
regulation (approval by 2004 with implementation
in 2006?). "Importing" the already existing
data from the United States could be a contribution.
President Bush signed in January this year the "Best
Pharmaceuticals for Children Act |
|
Over 400 studies are registered and for over 20
medicines information regarding the use in children
changed. (pharmacos.eudra.org) |
|
| 3. |
Improvements in the ethical and scientific requirements
for conducting research in children (also including clinical
trials with children on pharmaco genetic properties of
new medicines). A strong and adequate European framework
specialised in the ethical aspects of trials with children
is necessary in order to protect the dignity and the rights
of children. The European Forum for Good Clinical Practice
(EFGCP) is working, and since recently in partnership
with patient organisations, on these issues also to see
that paediatric patients are not overexposed to research
and not over or under treated. |
| 4. |
Increased awareness and education concerning the required
activities for the availability and prescription of safe
and effective medicines for children. Dialogue on the
benefits and risks of paediatric research for individual
children participating in trials as well as for public
health in general, is important. Awareness of the great
potential pressure and burden which is laid on parents
and children participating in trials. Government officials,
researchers and the pharmaceutical industry must be motivated
towards a synergistic approach with investments at national
levels and the European level. |
| 5. |
Priority for (preventive) treatment of serious and life
threatening diseases |
| 6. |
The active role that Patient Organisations play and
could play in this field. Many Patient Organisations have
excellent relationships with paediatric specialist groups
at both national and European levels. The involvement
of patient organisations on the European policy and regulatory
level as well on the projectbound and individual level
should be recognised by making available the required
financial resources.
Moreover patient organisations could play an important
role in the set up of clinical trials, in recruitment
of participants and in communication. |
| 7. |
Long term follow up of the effects of paediatric medicines.
|
Patient Organisations favour:
| 1. |
The creation of a paedriatic fund at the European level
that would support additional research on existing medications
regarding their use in children. Due to the backlog this
fund would initially require an amount of 200 million
Euros with investment opportunities for both the public
and private sectors. This amount is equivalent to what
the United States Congress provided for this purpose.
It would be advisable that the proposed regulation more
clearly indicates and defines other possible resources
for the fund. |
| 2. |
Extension of Market exclusivity for suitable pediatric
programmes and
"Kid Marketing Authorisation"
that gives a period of data protection for studies on
medicinal products where no protection of intellectual
property exists. These are necessary incentives for industry
whose efforts in generating paedriatic scientific studies
deserve recognition and which are of special relevance
in the field of rare disorders.
Unnecessary hurdles in an already complex field should
be avoided. |
| 3. |
The development of a central database, the formation
of an EMEA pediatric expert advisory group and the building
of a pan-European network of clinical paedriatic and pharmacological
excellence and specific expertise in the performance of
trials on paediatric populations and that could link existing
national and European initiatives. These initiatives can
avoid unnecessary trials and contribute to efficient and
adequate use of already available expertise, experience
and data. |
Patient organisations are worried about the considerable
backlog and the gap between the present and required situation.
Many existing medicines have to be re-tested which means a
large research investment. Regulation and incentives to test
new medicines are lacking so far. Therefore, it is essential
that measures be taken in the short term to address this urgent
and unacceptable situation.
Europe's children will greatly benefit from a well co-ordinated,
well supported, and a well-funded approach.
Finally it should be realised that the subject "better
medicines for children" is part of a huge research effort
and worldwide process running from diagnosis to treatment
and from gene to cure (see "pathways to treatment").
This effort greatly benefit from new genetic and biotech knowledge
and suffers from fragmentation, lack of coherence and of structural
funding. Patient organisations play an increasing role by
providing data, motivating patients/families, facilitating
and supporting research, communication with media and politicians,
influencing health policy on the national and EU-level. For
this reasons they have united in European Alliances and started
the European Patient Forum. In the European Platform Patient
Organisations Science and Industry (EPPOSI) patient organisations
work together with scientific groups and industries to influence
healthcare policy.
Although this paper may be considered as representative for
the opinion of many parents and patient organisations, it
does reflect explicitly the opinion of 7 European patient
alliances who discussed the subject extensively during a meeting
in Brussels (Gamian, EAGS, EAMDA, ECFPN, IDF, EPV, EAIH).
The author is chairman of the European Platform Patient Organisations,
Science and Industry (EPPOSI), past vice president of the
European Alliance of Genetic Support Groups), former director
of the Dutch Alliance of Parent & Patient organisations.
|
