Opinion of the European Economic and Social Committee on the Proposal for a Regulation of the European Parliament and of the Council on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/83/EC and Regulation (EC) No 726/2004 (COM(2004) 599 final — 2004/0217 COD)
Date: 2005-05-11
Institutions: European Economic and Social Committee
Subjects: European Medicines Agency, child, drug surveillance, market approval, medical research, medicinal product, paediatrics, pharmaceutical legislation, testing

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Official Journal of the European Union

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II

(Preparatory Acts)

EUROPEAN ECONOMIC AND SOCIAL COMMITTEE

417th PLENARY SESSION (MEETING OF 11 AND 12 MAY 2005)

Opinion of the European Economic and Social Committee on the Proposal for a Regulation of the
European Parliament and of the Council on medicinal products for paediatric use and amending
Regulation (EEC) No 1768/92, Directive 2001/83/EC and Regulation (EC) No 726/2004

(COM(2004) 599 final — 2004/0217 COD)

(2005/C 267/01)

On 12 November 2004 the Council decided to consult the European Economic and Social Committee,
under Article 251 of the Treaty establishing the European Community, on the abovementioned proposal.

The Section for the Single Market, Production and Consumption, which was responsible for preparing the
Committee's work on the subject, adopted its opinion on 20 April 2005. The rapporteur was Mr Braghin.

At its 417th plenary session (meeting of 11/12 May 2005), the European Economic and Social Committee
adopted the following opinion unanimously.

1. Summary of the Committee's recommendations

entrusted with the scientific management of
the Medicines
Investigation for the Children of Europe (MICE) programme
that the Commission proposes to set up under an appropriate
initiative.

The EESC considers protecting the paediatric population
1.1
to be a top priority since it is a vulnerable group with specific
physiological psychological, and developmental characteristics.
For this reason, it believes that the decision to conduct paedia-
tric studies should be based on clearly identified, scientifically
researched needs and that compliance with the ethical condi-
tions for the trials themselves should be ensured.

1.2
The EESC approves of the proposal to set up a Paediatric
Committee within the EMEA, believing that it is an appropriate
instrument
for ensuring quality paediatric studies based on
scientific and ethical principles. It recommends that it should
include a broader spectrum of specific paediatric competences
with respect to the development and use of paediatric medi-
cines, and that the number of experts assigned to it by the
Commission should be increased.

The EESC welcomes the proposed authorisation proce-
1.4
dure and especially supports the new PUMA procedure (the
Paediatric Use Marketing Authorisation)
for pharmaceuticals
with existing market authorisation.
It would further recom-
mend introducing an abridged centralised procedure for cases
where this is justified by the safety data, especially if gathered
through the periodic safety update reports.
It also suggests
specifying that
in cases where grounds are established for
adopting orphan medicine procedures for a specific subcate-
gory of the paediatric population, the market authorisation
holder may opt for either of the two procedures.

The EESC considers that

1.3
the Paediatric Committee's
responsibilities should be broadened at the outset. In particular,
it recommends that its role be strengthened in the context of
the European network of researchers and research centres for
It further recommends that it should be
paediatric studies.

1.5
In view of the time and resources required for paediatric
studies as well as sensitive ethical and compliance issues
relating to paediatric patients,
the EESC agrees with the
proposal to set up a system of incentives and rewards but
would suggest strengthening them in certain specific situations.

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1.6
The EESC supports the proposal to increase the avail-
ability of information on the use of medicines in the paediatric
sector to medical and health practitioners,
including greater
access to information on the EudraCT Database (1). It further
recommends adopting a broader communication strategy that
facilitates a safer and more effective use of medicines in chil-
dren.

— to increase the development of medicines for use in chil-

dren;

— to ensure that medicines used to treat children undergo

high quality research;

1.7
The EESC considers it necessary to conduct a detailed
study of the epidemiological situation for infants, therapeutic
approaches and existing shortcomings in the availability of
paediatric medicines, as well as a more detailed study of the
paediatric use of so-called off-label prescriptions.

The EESC would therefore

1.8
the
Commission play an active role in setting up a network linking
the relevant authorities and specialised research centres,
in
order to further our understanding of demand mechanisms for
medicines and better therapeutic practice.

recommend that

1.9
Finally, the EESC hopes that cooperation with the WHO
and the dialogue with the relevant international authorities can
be stepped up in order to speed up authorisation procedures
for paediatric medicines and avoid any duplication or futile
repetition of clinical studies.

2. Introduction

— to ensure that medicines used to treat children are appro-

priately authorised for use in children;

— to improve the information available on the use of medi-

cines in children, and;

— to achieve these objectives without subjecting children to
unnecessary clinical trials and in full compliance with the
EU Clinical Trials Directive.

The proposal includes a number of measures to achieve

2.4
these objectives. The most significant are the following:

2.4.1
Setting up a Paediatric Committee within the Euro-
pean Medicines Agency (EMEA). The Paediatric Committee
should be responsible for the assessment and agreement of
paediatric investigation plans and for the relevant system of
waivers and deferrals. It should also be responsible for assessing
the compliance of dossiers with approved paediatric investiga-
tion plans and existing Community legislation; adopting an
inventory of therapeutic needs in the paediatric population,
and improving the information available on the safe use of
medicines in various paediatric fields, in order,
inter alia, to
avoid duplicating or conducting unnecessary studies.

and psychological

The paediatric population is a vulnerable group that
2.1
differs from the adult population because of its specific devel-
opmental, physiological
characteristics,
which makes age and development related research of medi-
cines particularly important.
In contrast to the situation in
adults, more than 50 % of the medicines used to treat the chil-
dren of Europe have not been tested and are not authorised for
use in children: the health and therefore quality of life of the
children of Europe may suffer from a lack of
testing and
authorisation of medicines for their use.

2.4.2
The studies in children are to be based on a paediatric
investigation plan approved by the Paediatric Committee.
When assessing such plans the Paediatric Committee will take
that studies
into consideration two overarching principles:
should only be performed if there is a potential therapeutic
benefit to children (and avoiding duplication of studies). The
requirements for studies in children should not delay the
authorisation of medicines for other populations.

voiced

concerns

Although there may be

2.2
about
conducting trials in the paediatric population, this has to be
balanced by the ethical issues related to giving medicines to a
population in which they have not been tested and therefore
their effects, positive or negative, are unknown. In order to
address the concerns about trials in children, the EU Directive
on clinical trials (2) lays down specific requirements to protect
children who take part in clinical trials in the EU.

2.4.3
All studies performed in accordance with a completed,
agreed paediatric investigation plan are to be presented at the
time of application for authorisation for new active ingredients,
new indications, new pharmaceutical forms or new routes of
administration for an authorised medicine, unless a waiver or a
deferral has been granted by the Paediatric Committee.

2.3

The general objectives of the proposal are:

(1) European Clinical Trials Database
(2) OJ L 121, 1.5.2001

2.4.4
In order to establish a vehicle for providing incentives
for off-patent medicines, a new type of marketing authorisa-
It will utilise existing marketing
tion, PUMA,
authorisation procedures but
specifically for medicinal
products developed exclusively for use in children.

is proposed.

is

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2.4.5
To increase the availability of medicines for children
across the Community, because the requirements in the propo-
sals are linked to Community-wide rewards and to prevent the
distortion of free trade within the Community, it is proposed
that an application for a marketing authorisation including at
least one paediatric indication based on the results of an agreed
paediatric investigation plan will have access to the centralised
Community procedure.

For new medicines and for products covered by a
2.4.6
patent or a Supplementary Protection Certificate (SPC), if all the
measures included in the agreed paediatric investigation plan
are complied with, if the product is authorised in all Member
States and if relevant information on the results of studies is
included in product information, the six-month SPC extension
will be granted.

2.4.7
Similar incentives are proposed for orphan medicinal
products, for which, provided that the requirements for data on
use in children are fully met, the usual ten-year market exclu-
sivity is extended by two years.

2.4.8
benefit
marketing authorisation (PUMA).

Products with existing marketing authorisation, will
from the data protection associated with a new

2.5
The Clinical Trials Directive establishes a Community
database of clinical trials (EudraCT). It is proposed to build
onto this database an information resource of all ongoing and
terminated paediatric studies conducted both in the Com-
munity and in third countries.

2.6
The Commission intends to examine the possibility of
setting up a paediatric study programme: Medicines Investiga-
tion for the Children of Europe (MICE), taking into considera-
tion existing Community Programmes.

2.7
Establishing a Community network has also been
proposed. The network would link together national networks
and clinical trial centres in order to build up the necessary
competences at a European level and to facilitate the imple-
mentation of studies, to increase cooperation and avoid dupli-
cation of studies.

2.8
The proposal is based on Article 95 of the EC Treaty.
Article 95, which prescribes the codecision procedure described

in Article 251, is the legal basis for achieving the aims set out
in Article 14 of the Treaty, which includes the free movement
of goods
in this case human medicinal
products.

(Article 14(2)),

3. General comments

3.1 Safeguarding paediatric health and clinical trials in children

The Committee considers the protection of the paedia-
3.1.1
tric population to be a top priority, insofar as it is a vulnerable
group with specific developmental, physiological and psycholo-
gical characteristics.
this fundamental objective is to be
pursued in the field of paediatric pharmaceuticals, the following
conditions must be fulfilled:

If

— only necessary clinical

in children are
conducted, and futile duplication should be avoided;

trials

to be

— clinical tests must be adequately controlled, monitored, and
imperative to

conducted in accordance with the ethical
provide maximum protection for the paediatric patient;

— adequate information and communication processes must
guarantee a deeper understanding of recommended thera-
peutic approaches for this group;

— active pharmacovigilance mechanisms should facilitate the
continual and scientifically grounded updating of paediatric
therapeutic practice.

The EESC therefore considers that

3.1.2
the decision to
require and conduct clinical studies should be based on clearly
defined needs supported by research.
It should therefore be
verified that:

— existing information on the pharmaceutical product does
not adequately ensure safe and effective use in children (3);

— the level of (current or potential) use in children is substan-

tial (4);

— the medicine is likely to have benefits;

— the additional scientific and medical information acquired
through the use of a medicine with existing authorisation
implies benefits for paediatric use.

(3) In the USA, the FDA may require trials for paediatric use in cases
where the inadequate labelling of a medicine on the market could
expose patients to significant risk.

(4) In the USA, the FDA defines ‘substantial’ as at least 50,000 patients.
At this level, a company may be required to conduct clinical paedia-
tric trials.

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3.1.3
In view of the above, the EESC believes that it would
be appropriate for the ethical standards and specific regulations
for protecting minors laid down in the Directive on the imple-
mentation of good clinical practice in the conduct of clinical
trials (5) to be mentioned in the Directive's articles and not only
in the recitals. The Paediatric Committee's general criteria for
approving the paediatric investigation plan (PIP) should take
account of the recommendations of the relevant International
Conference on Harmonisation (6), and should comply with
directive 2001/20/EC on clinical
trials in order to ensure
compliance with the ethical conditions for the trials themselves.

3.1.4
The EESC therefore insists that the proposal's true
focus should be the paediatric patient and his health needs. It is
from this perspective that we should deal with issues relating
to the medical approach and consequently the clinical and ther-
apeutic information that
should be available to medical
personnel and, for the purposes of their specific competences,
to other health care professionals to enable them to treat a
specific patient requiring their care.

3.2 Basic information gaps affecting the use of medicines

The EESC considers the assessment of

3.2.1
the present
situation, causes and risks to be insufficient. The EIA dedicates
only a few pages to them and the explanatory memorandum of
the proposal makes no reference to them whatsoever.

It would have been appropriate to conduct a study
3.2.1.1
on the epidemiological situation for infants and gaps in the
existing therapeutic arsenal, thereby orientating research trends
appropriately in order to identify research priorities to be
supported through Community funding (within the framework
of ongoing discussions on the 7th Research Framework
Programme). In addition, such a study would have showcased
the work of the CHMP Paediatric Expert Group, which has
drawn up a list of 65 unpatented active ingredients to be
treated as research and development priorities in paediatrics. It
would also have facilitated and accelerated ongoing efforts to
implement
study
abovementioned MICE
programme.

paediatric

the

Since the return on investment

3.2.1.2
the
market for medicines to treat paediatric diseases with high inci-
dence rates is substantial enough to motivate the pharmaceu-
tical industry to develop new paediatric indications and adapt
formulations for the paediatric population. However the cost of

is probable,

(5) OJ L 121 of 1 May 2001
(6) With particular reference to guideline ICH E 11, which states: ‘The
ethical imperative to obtain knowledge of the effects of the medic-
inal products in paediatric patients has to be balanced against the
ethical imperative to protect each paediatric patient in clinical trials’.

development for rarer diseases or diseases affecting specific sub
age groups outweighs the return on investment. The industry
(especially small/medium-sized European companies) cannot
afford these costs without adequate incentives or research
funding. It is precisely for these rarer diseases affecting specific
sub groups that additional tools must be provided to compen-
sate for the high cost, in terms of human, time and financial
resources, of investing in paediatric research.

3.2.2
The Committee believes that it would also have been
appropriate to conduct a more detailed study on the paediatric
use of so-called off-label prescriptions. Such a study would
have established the extent of the practice and the precise nega-
tive effects associated with the inappropriate use of medicinal
products. A better insight into the situation could have facili-
tated a more substantiated analysis of possible remedies and
incentives to be applied.

3.2.2.1
The EESC realises that the relevant information is
heterogeneous, and has been gathered in the Member States by
different bodies with extremely diverse, incomplete, and distor-
tive operational procedures. As a consequence, it is doubtful
whether the data will lend itself to comparison or the extrapo-
lation of general observations that are scientifically grounded.
Despite the limitations, a study of prescriptions and the use of
medicines would provide a preliminary, albeit schematic, over-
view of obvious discrepancies in terms of the scope and use of
therapeutic classes and the active ingredients used, sometimes
without scientifically grounded therapeutic justification.

3.2.2.2
Another discernible shortcoming lies in the analysis
of differences in medical practice in Member States, which is
undoubtedly relevant on the basis of the data gathered on the
classes of medicines prescribed for various diseases. The EESC
not only considers that such a study can no longer be post-
poned, it also believes that it would be particularly effective in
safeguarding public health, which is a primary asset. Bearing in
mind that vocational training, health care procedures, and the
administration of treatment and medicine are Member State
competences, the EESC hopes that the open method of coordi-
nation will also be applied to pharmaceuticals. It also hopes
that, in the interests of public health, a set of well-formulated
and coordinated guidelines on best medical practice in various
therapeutic fields and patient population strata, including the
paediatric population, will be drafted in good time, with the
active support of medical and patient associations.

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3.2.3
A parallel study should have been conducted on the
findings of monitoring and pharmacovigilance mechanisms, an
area where European legislation is undoubtedly in the
vanguard. Clearly, pharmacovigilance networks should have
already identified the presence or absence of cases of inap-
therapeutic shortcomings,
propriate use and,
for
which, the EU authorities,
in cooperation with the relevant
national authorities, could already have established an appro-
priate information policy.

indirectly,

Given the widespread use of off-label prescriptions, we
3.2.4
need to question the relative efficacy of an approach based on
authorisation procedures (as recommended in the proposal
under consideration). The Committee believes that it would
have been advisable to adopt parallel actions to encourage
good practice in the use of paediatric pharmaceuticals by
doctors, health operators in general, and parents, whose under-
standable anxieties to alleviate their children's suffering often
put the doctor under pressure to prescribe short-term remedies
that do not always meet the young patient's real needs.

3.2.5
Another aspect that has not been taken into proper
account is the importance of the pharmacist's role in purchase
decisions and in providing advice on the appropriate use of
medicines. This category of health professional could provide
valuable support for an active education and pharmacovigilance
policy.

3.2.6
It would also be worth deepening the analysis of avail-
able data on safe use, especially pharmacovigilance, in order to
assess whether the different prescriptive approaches applied in
various EU Member States and the different pharmaceutical
classifications have different impacts in terms of inappropriate
use and adverse reactions.

3.2.7
The EESC realises that these matters go beyond the
primary scope of the proposal under consideration but would
nevertheless recommend that the Commission play an active
role in establishing a network linking the authorities to specia-
list research centres in order to increase our understanding of
the mechanisms that influence demand for pharmaceuticals,
their rational use, best therapeutic practice, and other similar
aspects, thereby facilitating the harmonisation of the internal
market for pharmaceuticals too.

3.3 The paediatric committee and clinical trials

The EESC agrees with the proposal

3.3.1
to establish a
Paediatric Committee within the EMEA. The responsibilities of
this committee are extremely diverse and range from the assess-
ment of the content and modalities of all paediatric investiga-
tion plans to the preventive assessment of the potential benefits
for
for
drafting such plans in compliance with good clinical trials prac-

the paediatric population;

from scientific support

tice to providing a therapeutic inventory, and support and
consultancy services for setting up a European network of
researchers,
in
conducting studies in the paediatric population. In addition to
the abovementioned responsibilities, the committee will also be
responsible for avoiding the duplication of studies.

and centres with specific

competencies

3.3.2
In view of the broad range of the Paediatric Commit-
tee's responsibilities, the EESC does not consider the compe-
tences set out in Article 4(1) to be sufficient, especially with
regard to pre-clinical and clinical development methodology (in
particular, experts in pharmacology, toxicology, pharmacoci-
netics, biometrics,
(including
and biostatistics),
in the paediatric fields corresponding to the
neonatologists)
most significant therapeutic groups, and experts in pharmacoe-
pidemiology. Furthermore, the EESC would recommend that
the number of experts designated by the Commission be
increased to include the representatives of health care facilities
for children.

specialists

The EESC notes that

3.3.3
the paediatric population is
defined as ‘that part of the population aged between birth and
18 years’ (Article 2) and realises that, to date, not even a stand-
ard ICH definition has been agreed upon. The EESC hopes that
in conducting specific studies for each subpopulation,
the
Paediatric Committee will avoid subjecting to unnecessary
studies a population whose constitution and age do not expose
them to risk.

3.3.4
The EESC approves of the principle that paediatric
investigation plans should be submitted during the develop-
ment phase of a new pharmaceutical product and welcomes
the possibility of continued dialogue between the proposer and
the Paediatric Committee. The EESC is nevertheless concerned
by the request to submit them ‘unless otherwise justified, not
later than upon completion of the human pharmaco-kinetic
studies’ (Article 17(1)). In fact, during this phase, safety trials in
the adult patient population will not have been concluded, and
consequently the safety profile will not be clearly defined. It
would therefore not be possible to draw up a comprehensive,
well-formulated paediatric investigation plan (especially for the
various subcategories of the paediatric population). This would
incur the risk of starting unnecessary studies or repeating
studies with different dosages from those initially foreseen.

3.3.5
The EESC is also concerned that the request will delay
the development of new medicines for the adult population,
whereas at a more advanced stage of development it would be
easier to identify at-risk populations, including the paediatric
population, focus research efforts on important information
gaps and put forward better targeted plans for active pharma-
covigilance.

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3.3.6
The EESC also expresses concern at the proposal that
‘any studies completed before this proposed legislation is
adopted will not be eligible for the rewards and incentives
proposed for the EU. They will, however, be taken into account
for the requirements contained in the proposals and it will be
mandatory for companies to submit the studies to the compe-
tent authorities once this proposed legislation is adopted’ (7).
This proposal risks slowing down or reducing the number of
ongoing or projected studies by companies, while waiting for
the
implemented
throughout the European Union.

regulation to be

final version of

the

3.4 Incentive measures

3.4.1
The EESC agrees that there is a need to create appro-
priate incentives to ensure that paediatric clinical trials are
conducted in accordance with principles of best practice and
ethical standards, and that paediatricians, paediatric clinics and
wards are provided with an enhanced therapeutic arsenal,
including safe, effective, high quality pharmaceuticals that have
been conceived and designed for the paediatric population,
following the logic of the terms of the Council Resolution of
14 December 2000 and bearing in mind experience gained in
the United States in the light of specific legislation (8) adopted
in that country.

3.4.2
The time and resources required for studies of this
type, as well as sensitive ethical and compliance issues relating
to paediatric patients, explain why market forces have not been
to develop pharmaceutical products that may be
sufficient
specifically categorised as ‘paediatric’. In view of this fact, the
EESC considers that
the incentives and rewards granted in
certain situations are not always sufficient.

specifically assessed in the context of the proposed general
report on the experience gained from the application of the
regulation.

3.4.3
The new procedure outlined in Title III Chapter II
(PUMA) for medicines with marketing authorisation that are
not protected by a patent or supplementary protection certifi-
cate constitutes an important and viable innovation for avail-
able paediatric use marketing authorisation procedures. The
possibility of following centralised procedures even if the initial
authorisation for a pharmaceutical product for adults has been
obtained through national procedures constitutes a genuine
opportunity.

3.4.4 Welcome progress has been achieved in terms of
procedural flexibility, in particular, the possibility of referring
to existing data in an authorised medicine's dossier (Article
31(4)) and the possibility of using a known brand name by
simply adding the letter ‘P’
in superscript (Article 31(5)). In
such instances, the EESC recommends that the pharmaceutical
form and dosage should also be prominently displayed on the
packaging if they have been adapted for paediatric use.

for

3.4.5
However, the EESC notes that this flexibility is coun-
tered by a certain rigidity which could act as a disincentive to
paediatric research,
the obligation to obtain
instance,
authorisation in all Member States in order to benefit from the
extension of the Supplementary Protection Certificate (SPC).
The Committee considers that this provision is excessive, espe-
cially in an enlarged Union. It believes that only large multina-
tionals producing pharmaceuticals of guaranteed success will
actually benefit.

3.4.2.1
In particular, the six-month extension of the Supple-
mentary Protection Certificate does not appear to adequately
compensate for the higher costs, risks and delays in completing
the dossier and obtaining authorisation that paediatric studies
could imply for a new product. Admittedly, appropriate
waivers and deferrals have been foreseen. Nevertheless,
if
paediatric research were made compulsory, the commitment
would become particularly expensive and time-consuming.

3.4.6
The assertion that all data relating to development
should be disclosed is also cause for concern since it changes
existing legislation on the disclosure of information and data
relating to marketing authorisation dossiers. This approach
would also appear to act as a disincentive to launching research
into new types of drugs and the appropriate dosage for paedia-
tric use of established medicinal products that are already
marketed.

3.4.2.2
The EESC notes with concern the current tendency
to focus research and development efforts on active ingredients
with broad market potential, which absorb a growing propor-
tion of investment in research and development, whereas ingre-
dients with smaller or niche market potential are secondary
priorities. If this mechanism were applied to new paediatric
it would be impossible to fulfil the objective of
medicines,
obtaining a genuinely innovative and sufficiently diverse
arsenal of paediatric medicines within a reasonable timeframe.
The EESC advocates that such risks be carefully monitored and

(7) See the explanatory memorandum under Information on the use of

medicines for children, p. 7

(8) Best Pharmaceuticals for Children Act, 1 April 2002, Public Law n.

107-109.

3.5 Information on the use of medicines for children

One of the proposal's objectives is to increase the
3.5.1
availability of
information on the use of medicines in the
paediatric sector. The EESC agrees that the increased availability
of information could facilitate the safe and effective use of
medicines in children and thereby promote public health.
Furthermore, the availability of information could help to avoid
duplicating studies or carrying out unnecessary studies on chil-
dren.

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3.5.2
For this reason, the EESC also supports the proposal
to use the Community clinical trials database (EudraCT), estab-
lished by the Clinical Trials Directive, as a foundation for an
information resource on all ongoing and terminated paediatric
studies conducted in the Community and third countries.

3.5.2.1
Nevertheless, the arrangements for using this data-
base are not sufficiently clear: who should have access, what
data should be disclosed or withheld on grounds of individual
privacy protection, or the need to protect sensitive or confiden-
tial industrial information.

3.5.2.2
Similarly, no clear line has been drawn between
information (available to health profes-
available technical
sionals) and information to be made available to the general
public in the package leaflet. In this segment of the paediatric
market, comprehensible and transparent package leaflets play a
particularly important role in preventing behaviour that could
potentially harm the paediatric patient.

Title VI on Communication and Coordination outlines
3.5.3
a series of actions and obligations (for instance, the fact that
available data on all existing uses of medicinal products in the
paediatric population must be collected by the Member States
within two years of the entry into force of the Regulation —
it does not tackle the issue of under-
Article 41). However,
standing the proper use of pharmaceutical products in the
paediatric sector and the policies to be adopted vis-à-vis health
professionals and the general public.

4. Concluding comments

The EESC reiterates its fundamental agreement with the
4.1
proposed regulation, but wonders whether its legal basis, more
specifically, Article 95 of
the EC Treaty for implementing
objectives established under Article 14(2) (free circulation of
goods), is the most appropriate basis in an area of implementa-
tion with significant public health implications. Although all
legislation adopted for the pharmaceutical sector is based on
the abovementioned article, it should be borne in mind that, in
the case under consideration, the fundamental objective is the
health and protection of the paediatric population.

4.2
The EESC hopes the Commission will soon draw up
another proposal that focuses on the demand for pharmaceuti-
cals, rather than on supply. The objective would be to create an
operational tool that facilitates and encourages data collection
and dissemination on the availability and use of medicines;
setting up epidemiological and prescriptive use data bases; as
well as establishing guidelines through the increased involve-
ment of health professionals and patient associations, thereby
simultaneously extending the application of the open method
of coordination to this sector.

4.3
The communication and coordination process in Title
VI seems somewhat restrictive. The EESC recommends that a
broader communication strategy resulting in a more rational
use of medicine in paediatrics should be prepared and imple-
mented. Furthermore, doctors and health care professionals
should be supplied with all necessary information tools for
their purposes. Following the same line of thought, we should
reconsider whether, and under what procedures, scientific
researchers and doctors should have access to the information
on clinical trials that is available on the European Clinical Trials
Database (EudraCT).

4.4
The EESC welcomes the proposal to set up a paediatric
study programme, Medicines Investigation for Children in
Europe (MICE), to provide Community funding for research
carried out by groups, companies, and paediatric hospital
networks on the paediatric use of unpatented medicines, or
observational or cohort studies in their post-registration phase.
The EESC would, however, have preferred orientation guide-
lines and a more precise definition of the Paediatric Commit-
tee's role in this respect. This would avoid lengthy discussions
as to who should identify priority therapeutic fields requiring
further
the assessment of
priority needs and the specific studies to be conducted, particu-
larly in view of the considerable differences in current medical
practice in Member States.

information on paediatric use,

4.5
The EESC therefore recommends that these competences
be specifically attributed to the Paediatric Committee under
Article 7 of the regulation, in order to facilitate speedy imple-
mentation and ensure better coordination of all the Paediatric
Committee's institutional activities.

the

4.6
At the same time, the EESC hopes that, in establishing
and implementing a European network of researchers and
centres with specific roles to play in carrying out studies on the
paediatric population under Article 43,
Paediatric
Committee will not merely assume a supportive and advisory
role for the agency. It should play an active part, with the
possible support of a forum that brings experts from all
Member States together, be they academics or paediatric sub-
specialists. Furthermore,
including,
should it be necessary to define specific research study proto-
cols, researchers from companies involved in the protocol
through their own products, insofar as they are best placed to
know the specific features of these products.

the EESC recommends

4.7
The fact that the Paediatric Committee's primary role is
to approve paediatric investigation plans (PIP), which is at the
very heart of the proposal
leads the EESC to fear that the
tendency to formalise clinical paediatric studies will prevail
over the pursuit of some of the objectives,
including ethical
objectives, cited above, such as avoiding futile duplication or
paediatric studies that are not genuinely necessary.

C 267/8

EN

Official Journal of the European Union

27.10.2005

4.8
The EESC suggests that the need to analyse information
on the EudraCT database and conduct a detailed assessment of
the periodic safety update reports (established under the most
recent legislative amendments) should be specifically included
amongst the Committee's competences. The reports include
epidemiological data, prescription surveys, and the results of
published studies, thereby reducing the magnitude and duration
of clinical studies, or in some cases making them redundant.

From a procedural point of view, it should be assumed
4.9
that, should such documentation permit
the assessment of
safety data for existing medicines (obtained through pharma-
covigilance, information reports and the periodic safety update
reports) regarding formulations and dosage for paediatric use, it
will be possible to adopt a shorter, simpler centralised proce-
dure to amend appropriately the technical information in the
package leaflet,
than the PUMA procedure, which
remains lengthy and expensive (9).

rather

Also from the procedural point of view,

4.10
the EESC
considers that it is necessary to specify that in cases where
grounds are established for adopting orphan medicine proce-

dures for a subcategory of
the
market authorisation holder may opt for either of the two
procedures.

the paediatric population,

4.11
The EESC emphasises the importance of publishing
research results and approved changes to the package leaflet,
and including information for paediatric use for all unpatented
medicines with the same active ingredients.

4.12
The EU is already the regulatory authority for the regis-
tration of pharmaceuticals in developing countries, and the
WHO already consults it when assessing medicines that can be
registered in such countries. It is to be hoped that an expedi-
tious application of this regulation in the EU will also have a
positive impact on paediatric therapies available in the least
developed countries. The EESC hopes that constructive coop-
eration with the WHO will be further strengthened and that
the Commission will pursue regular dialogue with all interna-
tional authorities in order to speed up approval procedures for
new substances and indications, dosages and formulations that
are more appropriate for paediatric use, thereby avoiding any
unnecessary duplication and repetition of clinical studies.

Brussels, 11 May 2005

The President

of the European Economic and Social Committee

Anne-Marie SIGMUND

(9) A simplified mechanism of this type has already been adopted in
the United States, where 33 products now include paediatric infor-
mation in their leaflet as a result of post-registration clinical studies
(since periodic safety update reports do not exist in the United
States such studies were required), whereas 53 are authorised for
exclusive paediatric use on the basis of a complete clinical study
plan.