EN    EN  COMMISSION OF THE E UROPEAN COMMUNITIES 
Brussels, 29.9.2004 
COM(2004) 599 final 
2004/0217 (COD)   
Proposal for a 
REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL 
on medicinal products for paediatric use and amending Regulati on (EEC) No 1768/92, 
Directive 2001/83/EC and Regulation (EC) No 726/2004 
(presented by the Commission)  
     
{SEC(2004) 1144} 
 
EN 2   EN EXPLANATORY MEMORANDUM  
1. I NTRODUCTION AND BACKGROUND  
The public health concern and its causes The paediatric population is a vulnerable gr oup with developmental, physiological and 
psychological differences from adults, which ma kes age and development related research of 
medicines particularly important. In contrast to the situation in  adults, more than 50% of the 
medicines used to treat the children of Europe ha ve not been tested and are not authorised for 
use in children: the health and therefore quality  of life of the childre n of Europe may suffer 
from a lack of testing and authoris ation of medicines for their use. 
Although there may be concerns voiced about c onducting trials in the p aediatric population, 
this has to be balanced by th e ethical issues related to gi ving 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
1 
lays down specific requirements to protect children who take part in clinic al trials in the EU. 
Related initiatives: EU Orphan Regulation and US legislation on medicines for children 
The absence of research into treatments for rare diseases led the Commission to propose the 
Regulation on orphan medicinal products, subs equently adopted in  December 1999. This 
Regulation has proved successful in stimulating research leading to the authorisation of 
medicines to treat rare diseases. 
In the US, specific legislation to encourage the pe rformance of clinical trials in children has 
been introduced, containing combined measures  of incentives and obligations which have 
proven successful in stimulating the developmen t of medicinal products for paediatric use. 
Council Resolution 
The Council Resolution of 14 December 2000 called on the Commission to make proposals in 
the form of incentives, regulatory measures or ot her supporting measures in  respect of clinical 
research and development to ensure that new medicinal products for children and medicinal 
products already on the market are fully ad apted to the specifi c needs of children. 
2. J USTIFICATION  
Objective 
The overall policy objective is to improve the he alth of the children of  Europe by increasing 
the research, development and authorisa tion of medicines for use in children. 
General objectives are: to increase the developmen t of medicines for use in children; to ensure 
that medicines used to treat children are subject  to high quality research; to ensure that 
medicines used to treat children are appropriately authorised for use in children; to improve 
                                                 
1 OJ L 121, 1.5.2001, p. 34.  
 
EN 3   EN the information available on the use of medicines in children, and; to achieve these objectives 
without subjecting children to unnecessary clini cal trials and in full compliance with the 
EU Clinical Trials Directive. 
Scope, legal basis and procedure 
The proposed system covers medicinal produ cts for human use within the meaning of 
Directive 2001/83/EC. 
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 fr ee movement of goods (Art icle 14(2)), in this 
case human medicinal products. While taking account  of the fact that any regulations on the 
manufacture and distribution of medicinal products must be fundamentally aimed at 
safeguarding public health, this aim must be achieved by means that do not impede the free 
movement of medicinal products within the Community. Since the Amsterdam Treaty came 
into force, all legislative provisions adopted by the European Parliament and the Council in 
this field have been adopted on the basis of that Article, since the differences between the 
national legislative, regulato ry and administrative provisions  on medicinal products tend to 
hinder intra-Community trade a nd therefore directly affect the operation of the internal 
market. Any action to promote the development and authorisation of medicinal products for 
paediatric use is justified at a European level with a view to preventing or eliminating these 
obstacles. 
Subsidiarity and proportionality 
The proposal builds on the expe rience gained with the existi ng regulatory framework for 
medicines in Europe and learns from the requi rements and incentives for paediatric medicines 
in the US and the EU orphan regulation. On the basis of the available evidence it is concluded 
that it is unlikely that the current public heal th issue regarding medicines for children will be 
resolved in the EU until a specific le gislative system is put in place. 
Community action allows the best possible use of the instruments set up in the pharmaceutical 
sector to complete the internal market. In addi tion, authorisation of medicines for children is a 
Europe-wide issue. However, Member States will have an important role in the fulfilment of the objectives of the proposal. 
Legislative and administ rative simplification 
All the key measures in the proposal build on or strengthen the existing framework for the 
regulation of medicinal products. This proposal  directly interfaces with five existing 
Community legislative texts: Directive 2001/83/EC of the European Parliament and of the 
Council of 6 November 2001
2 which sets the framework fo r the regulation of medicinal 
products; Regulation (EC) No 726/2004 of the European Parliament and of the Council of 
31 March 20043 which establishes the European Medi cines Agency (EMEA) and created the 
centralised authorisation pr ocedure for medicinal products ; Directive 2001/20/EC of the 
European Parliament and of the Council of 4 April 2001 which provides a framework for the 
regulation and conduct of clinic al trials in the Community; Regulation (EC) No 141/2000 of 
                                                 
2 OJ L 311, 28.11.2001 p. 67. 
3 OJ L 136, 30.4.2004 p. 1.  
 
EN 4   EN the European Parliament and of the Council which establishes a Comm unity system for the 
designation of medicinal products as orphan medicinal products and incentives to stimulate 
their development and authorisation4 and; Council Regulation (EEC) No 1768/92 of 
18 June 19925 which created the Supplement ary Protection Certificate. 
This proposal for a regulation establishes a precise legal framework, however, where more 
detailed implementing provisions are required, a Commission regulation is foreseen and it is 
proposed that further provisions be adopted by the Commission as guidelines, in consultation 
with the Member States, the EMEA and the parties concerned. 
Consistency with other Community policies 
Consistency will be sought with activities in th e areas of research and development and health 
and consumer protection. 
Outside consultation 
Interested parties have been widely consulte d on this proposal. Deta il on the consultation 
conducted by the Commission is included in the Extended Impact Assessment that 
accompanies this proposal. 
Evaluation of the proposal: Extended impact assessment 
This proposal has been the subject of a Commissi on extended impact assessment, attached to 
this proposal, which is based on data colle cted by an independent contracted study. 
3. P RESENTATION  
A brief description of the main elements of  the proposal is hereby provided. For a more 
detailed description, we refer to the Commi ssion’s explanatory document attached to this 
proposal. 
Key measures included in the proposal 
The Paediatric Committee A committee with expertise in all aspects related to medicines for children is central to the 
proposal and its operation. The Paediatric Comm ittee will be responsible primarily for the 
assessment and agreement of p aediatric investigation plans and requests for waivers and 
deferrals described below. In addition it may assess compliance with paediatric investigation plans and be asked to assess the results of studies . In all its work it will consider the potential 
significant therapeutic benefits of studies in children including the ne ed to avoid unnecessary 
studies, it will follow existing Community requi rements and will avoid any delay in the 
authorisation of medicines for other populations as a result of the requirements for studies in 
children. 
                                                
 
4 OJ L 18, 22.1.2000, p. 1. 
5 OJ L 182, 2.7.1992, p. 1. 
 
EN 5   EN Marketing authorisation requirements 
The paediatric investigation plan will be the document upon which the st udies in children are 
based and will have to be agreed by the Paedia tric Committee. When assessing such plans the 
Paediatric Committee will have to take into consideration two overarching principles: that studies should only be performed if there is a potential therapeutic benefit to children (and 
avoiding duplication of studies) and that the requirements for studies in children should not 
delay the authorisation of me dicines for other populations. 
A core measure is a new require ment for the results of all st udies performed in accordance 
with a completed, agreed paediatric investigation plan to be presented at the time of applications, unless a waiver or a deferral has been granted. This core requirement has been 
included to ensure that medicines are develope d for children based on their therapeutic needs. 
The paediatric investigation plan will be the basis upon which compliance with this 
requirement is judged. 
Waivers from the requirements 
Not all medicines being de veloped for adults will be suitable for children or will be needed to 
treat children and unnecessary st udies in children should be a voided. To deal with such 
situations a system of waivers from the requ irements described above is proposed. The 
Paediatric Committee will start work as soon as it is set up, on lists of waivers of specific 
medicinal products and classes of medicina l product. For products not included in the 
published lists, a simple pro cedure is proposed for companies to request waivers.  
Deferrals from the timing of initiation or completion of studies in children 
Sometimes studies in children will be more appropriate when there exists some initial 
experience on use of a product in adults or studies in  children might take longer than studies 
in adults. This might apply to the entire paedia tric population or just a subset. Therefore, to 
deal with this situation, a system of deferra ls is proposed together with a procedure for 
agreeing them with the Paediatric Committee. 
Marketing authorisation procedures 
The procedures set out in existing pharmaceutical legislation are not altered by the proposals. 
The requirements above will require the Competen t Authorities to check compliance with the 
agreed paediatric investigation plan at the ex isting validation step for marketing authorisation 
applications. The assessment of safety, quality and efficacy of medicines for children and the 
granting of marketing authorisat ions remain the remit of the Competent Authorities. To 
increase the availability of medicines fo r children across the Community, because the 
requirements in the proposals 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 p aediatric indication based on the results of an 
agreed paediatric investigation plan will have access to the centralised Community procedure. 
The Paediatric Use Marketing Authorisation (PUMA) 
In order to establish a vehicle for providing ince ntives for off-patent medicines, a new type of 
marketing authorisation, the Paediatric Use Marketing Authorisation (PUMA) is proposed. A 
 
EN 6   EN PUMA will utilise existing marketing authoris ation procedures but is specifically for 
medicinal products developed excl usively for use in children. 
The name of the medicinal product granted a PU MA can utilise the existing brand name of 
the corresponding product authorised for adults but the product names of all medicines 
granted a PUMA will include a superscript of the letter “P” to aid recognition and prescribing. Thus, pharmaceutical companies will be able to  capitalise on existing brand recognition while 
benefiting from the data protectio n associated with a new marke ting authorisation. The data 
protection period associated with  the PUMA may prove more valu able in light of the recent 
case law of the European Court of Justice con cerning the interpretati on of data protection 
rules
6. 
An application for a PUMA will require the submission of data necessary to establish safety, 
quality and efficacy specifically in children, coll ected in accordance with an agreed paediatric 
investigation plan. However, an application fo r a PUMA may refer to data contained in the 
dossier of a medicinal product which is or  has been authorised in the Community. 
Extension of the duration of the su pplementary protection certificate 
For new medicines and for products covered by a 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 in formation, the six-month SPC extension will be 
granted. The mechanism for this will be the inclusion of a statement in the marketing 
authorisation that these measures have been me t. Companies will then be able to present the 
marketing authorisation to patent offices that will then award the SPC extension. The need to 
have a marketing authorisation in all Member States is to prevent a Community-wide reward 
without Community-wide benefits to child-h ealth. Because the reward is for conducting 
studies in children and not for de monstrating that a product is sa fe and effective in children, 
the reward will be granted even when a paediatr ic indication is not granted. However, relevant 
information on use in paediatric populations wi ll have to be included in authorised product 
information. 
Extended market exclusivity for orphan medicinal products 
Under the EU orphan regulation, medicinal prod ucts designated as or phan medicinal products 
gain ten-years of market exclus ivity on the granting of a marketing authorisation in the orphan 
indication. As such products are frequently not patent protecte d the reward of SPC extension 
can not be applied and when they are patent -protected, SPC extension would provide a double 
incentive. Therefore it is proposed to extend the ten-year period of orpha n market exclusivity 
to twelve-years if the requirements for data on use in children are fully met. 
Paediatric study programme: Medicines Invest igation for the Children of Europe (MICE) 
An additional tool for promoting high qualit y, ethical research that may lead to the 
development and authorisation of medicines fo r children should be the provision of funding 
for studies into the paediatric use of medici nes not covered by a patent or a supplementary 
protection certificate. The Commission intends to examine the possibility of setting up a 
                                                 
6 Case C-106/01, Novartis Phar maceuticals UK, judgment of 29 April 2004, not yet reviewed. 
 
EN 7   EN paediatric study programme: Medi cines Investigation for the Ch ildren of Europe, taking into 
consideration existing Community Programmes.  
Information on the use of medicines for children 
One of the objectives of this proposal is to in crease the information available on the use of 
medicines for children. Through increased availabi lity of information, the safe and effective 
use of medicines for children can be increas ed so promoting public health. In addition, 
availability of this information with help prev ent the duplication of studies in children and the 
conduct of unnecessary studies in children. 
The Clinical Trials Directive establishes a Commun ity 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 th e Community and in third countries. 
In addition, based on a survey of existing us e of medicines in Europe, an inventory of 
therapeutic needs of children will be established by the Paediatric Committee. 
It is also proposed to create a Community network to link t ogether national networks and 
clinical trial centres in order to build up the necessary competences at a European level and to 
facilitate the conduct of studies , to increase co-operation and avoid duplication of studies. 
Pharmaceutical companies have, in some cases, al ready conducted clinical trials in children. 
However, frequently, the results of these st udies have not been submitted to Competent 
Authorities and have not resulted  in updates to product information. To deal with this issue, it 
is proposed that any studies completed before th is proposed legislation is adopted will not be 
eligible for the rewards and incentives proposed fo r 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 competen t authorities on ce this proposed legislation is 
adopted. 
Other measures 
The interface between the Committee for Medici nal Products for Human Use, its Scientific 
Advice Working Group and other Community co mmittees and working groups on medicines 
and the Paediatric Committee will be manage d by the EMEA. In addi tion, free scientific 
advice from the EMEA to sponsors devel oping medicines for children is proposed. 
The proposals presented will place demands on co mpetent authorities and particularly on the 
EMEA. It is proposed to increase the Community  subsidy, to be allocated to the EMEA to 
take account of the new tasks. A financ ial statement accompanies this proposal. 
 
EN 8   EN 2004/0217 (COD) 
Proposal for a 
REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL 
on medicinal products for paediatric use and amending Regulati on (EEC) No 1768/92, 
Directive 2001/83/EC and Regulation (EC) No 726/2004 
(Text with EEA relevance) 
THE EUROPEAN PARLIAMENT AND THE CO UNCIL OF THE EUROPEAN UNION, 
Having regard to the Treaty establishing th e European Community, and in particular 
Article 95 thereof, 
Having regard to the proposal from the Commission1, 
Having regard to the Opinion of the European Economic and Social Committee2, 
Having regard to the Opinion of the Committee of the Regions3, 
In accordance with the procedure refe rred to in Article  251 of the Treaty4, 
Whereas: (1) Before a medicinal product for human us e is placed on the market of one or more 
Member States, it generally has to have  undergone extensive studies, including 
pre-clinical tests and clinical trials, to en sure that it is safe, of high quality and 
effective for use in the target population. 
(2) Such studies may not have been done fo r use in children and many of the medicines 
currently used to treat children have not b een studied or authorised for such use. 
Market forces alone have proven insuffi cient to stimulate adequate research, 
development and authorisation of medicines for children. 
(3) Problems resulting from the absence of suitably adapted medicines for children 
include inadequate dosing info rmation leading to increased risks of adverse reactions 
including death, ineffectiv e treatment through under-dos ing, non-availability to 
children of therapeutic advances, as well as the use of extemporaneous formulations to 
treat children which may be of poor quality. 
                                                
 
1 OJ C […], […], p. […]. 
2 OJ C […], […], p. […]. 
3 OJ C […], […], p. […]. 
4 OJ C […], […], p. […]. 
 
EN 9   EN (4) The aim of this Regulation is to increas e the development of medicines for use in 
children, to ensure that medicines used to  treat children are s ubject to high quality, 
ethical research and are appropriately aut horised for use in ch ildren, and to improve 
the information available on th e use of medicines in the va rious paediatr ic populations. 
These objectives should be achieved without subjecting children to unnecessary clinical trials and without delaying the auth orisation of medicinal products for other 
populations. 
(5) While taking into account the fact that a ny regulations on medicinal products must be 
fundamentally aimed at safeguarding public health, this aim must be achieved by 
means that do not impede the free moveme nt of medicinal products within the 
Community. The differences between the national legislative, regulatory and 
administrative provisions on me dicinal products tend to hinder intra-Community trade 
and therefore directly affect the operati on of the internal market. Any action to 
promote the development and authorisation of  medicinal products for paediatric use is 
therefore justified with a view to preventi ng or eliminating these obstacles. Article 95 
of the Treaty is therefore the proper legal basis. 
(6) The establishment of a sy stem of both obligations and rewards and incentives has 
proved necessary to achieve th ese objectives. The precise na ture of the obligations and 
rewards and incentives should take account of the status of the particular medicinal 
product concerned. This Regulation should apply to all the me dicines required by 
children and therefore its scope should cover products in development and yet to be authorised, authorised products still cove red by intellectual pr operty rights and 
authorised products no longer covere d by intellectual property rights. 
(7) Any concerns about conducting trials in th e paediatric population should be balanced 
by the ethical concerns about giving medicines to a populati on in which they have not 
been tested. The public health threat from  the use of untested medicines in children 
can be safely addressed through the study of  medicines for children, which should be 
carefully controlled and m onitored through the specific re quirements for the protection 
of children who take part in clinical trials in the Community laid down in Directive 2001/20/EC of the European Parl iament and of the Council of 4 April 2001 
on the approximation of the laws, regulati ons and administrative provisions of the 
Member States relating to the implementati on of good clinical prac tice in the conduct 
of clinical trials on medicinal products for human use
5. 
(8) It is appropriate to create a scientific committee, the Paediatric Committee, within the 
European Medicines Agency, hereinafter ‘the Agency’, with expertise and competence in the development and assessment of all aspects of medicinal products to treat 
paediatric populations. The Paediatric Comm ittee should be primarily responsible for 
the assessment and agreement of paediatric investigation plans and for the system of 
waivers and deferrals thereof, and it should al so be central to various support measures 
contained in this Regulation. In all its work  the Paediatric Committee should consider 
the potential significant therapeutic benefits of  studies in children including the need 
to avoid unnecessary studies. It should follow existing Community requirements, 
including Directive 2001/20/EC, as we ll as Internati onal Conference on 
Harmonisation (ICH) guideline E11 on the de velopment of medici nes for children, and 
                                                
 
5 OJ L 121, 1.5.2001, p. 34.  
 
EN 10   EN it should avoid any delay in the authorisation of medicine s for other populations as a 
result of the requirements for studies in children. 
(9) Procedures should be established for the Agency to agree and modify a paediatric 
investigation plan, the document upon whic h the development and authorisation of 
medicines for children should be based. Th e paediatric investigation plan should 
include details of the timing and the meas ures proposed to dem onstrate the quality, 
safety and efficacy of the medicinal product in the paediatric population. Because the 
paediatric population is in fact made up of a number of subpopulations, the paediatric 
investigation plan should sp ecify which subpopulations n eed to be studied, by what 
means and by when. 
(10) The introduction of the paediatric investig ation plan in the lega l framework concerning 
medicinal products for human use aims at en suring that development of medicines for 
children becomes an integral part of the de velopment of medicinal products, integrated 
into the development programme for adults . Thus, paediatric investigation plans 
should be submitted early during product development, in time for studies to be conducted in children before marketing authorisation applications are submitted. 
(11) It is necessary to introduce a requ irement for new medicinal products and for 
authorised medicinal products covered by a patent or a supplem entary protection 
certificate to present the results of studies in children in accordance with an agreed 
paediatric investigation plan in order to obtain validation of a marketing authorisation 
application or an application for a new indication, new pharmaceutical form or new 
route of administration. The paediatric i nvestigation plan shoul d be the basis upon 
which compliance with that requirement is judged. However, that requirement should 
not apply to generics or similar biological  medicinal products and medicinal products 
authorised through the well-es tablished medicinal use proc edure, or to homeopathic 
medicinal products and traditional herbal medicinal products authorised through the 
simplified registration procedures of Directive 2001/83/EC of the European 
Parliament and of the Council of 6 Novemb er 2001 on the Community code relating to 
medicinal products for human use
6. 
(12) In order to ensure that research in ch ildren is only conducted to meet their therapeutic 
needs, there is a need to establish procedures for the Agency to waive that requirement for specific products or for classes or part of  classes of medicinal products, to be then 
published by the Agency. As knowledge on science and medicine evolves over time, provision should be made for the lists of waiv ers to be amended. However, if a waiver 
is revoked, the requirement should not apply for a given period in order to allow time for at least a paediatric investigation plan to be agreed and studies in children to be 
initiated prior to marketi ng authorisation application. 
(13) In certain cases, the Agency should defer the initiation or completi on of some or all of 
the measures contained in a paediatric inves tigation plan, with a vi ew to ensuring that 
research is done only when it is safe and et hical to do so and that the requirement for 
study data in children does not block or dela y the authorisation of medicines for other 
populations. 
                                                
 
6 OJ L 311, 28.11.2001, p. 67. Directive as last amended by Directive 2004/27/EC (OJ L 136, 30.4.2004, 
p. 34.) 
 
EN 11   EN (14) Free scientific advice should be provide d by the Agency as an incentive to sponsors 
developing medicines for children. To ensu re scientific consistency, the Agency 
should manage the interface between the Paediatric Committee and the Scientific Advice Working Group of the Committee for Medicinal Products for Human Use , as 
well as the interaction between the Paed iatric Committee and the other Community 
committees and working groups concerning medicinal products. 
(15) The existing procedures for the market ing authorisation of medicinal products for 
human use should not be changed. However,  it follows from the requirement for the 
results of studies in children to be presen ted in accordance with an agreed paediatric 
investigation plan that competent authoritie s should check compliance with the agreed 
paediatric investigation plan  and any waivers and deferrals  at the existing validation 
step for marketing authorisation applicati ons. The assessment of  safety, quality and 
efficacy of medicines for children and the gr anting of marketing authorisations should 
remain the remit of the competent author ities. Provision should be made for the 
possibility of asking the Paediatric Co mmittee for an opinion on compliance and for 
an opinion on the safety, quality and efficacy of a medicine in children. 
(16) To provide healthcare professionals a nd patients with information on the safe and 
effective use of medicines in  children and as a transparency measure, information 
regarding the results of  studies in children, as well as on  the status of the paediatric 
investigation plans, waivers and deferrals, should be included in product information. 
When all the measures in the paediatric i nvestigation plan have been complied with, 
that fact should be recorded in the mark eting authorisation, and should then be the 
basis upon which companies can obta in the rewards for compliance. 
(17) To distinguish medicines authorised for use in childre n following completion of an 
agreed paediatric investigation plan and enab le their prescription , provision should be 
made for the name of medicinal products granted an indication for use in children 
following an agreed paediatric investigation plan to include the letter “P” in blue 
lettering surrounded by the outline  of a star, also in blue. 
(18) In order to establis h incentives for authorised products no longer covered by 
intellectual property rights, it is necessary to establish a new type of marketing authorisation, the Paediatric Use Mark eting Authorisation. A Paediatric Use 
Marketing Authorisation shoul d be granted through existin g marketing authorisation 
procedures but should apply specifically fo r medicinal products developed exclusively 
for use in children. It should be possible fo r the name of the medicinal product granted 
a Paediatric Use Marketing Authorisation to utilise the existing brand name of the corresponding product authorised for adults, in order to capita lise on existing brand 
recognition while benefiting from the data exclusivity associated with a new marketing authorisation. 
(19) An application for a Paediatric Use Marketing Authorisation should include the 
submission of data concerning use of the product in the paediatric population, 
collected in accordance with an  agreed paediatric investigation plan. These data may 
be derived from the publishe d literature or from new st udies. An application for a 
Paediatric Use Marketing Authorisation should also be able to refer to data contained 
in the dossier of a medicinal product wh ich is or has been authorised in the 
Community. That is intended to provide an additional incentive to attract small and 
 
EN 12   EN medium sized enterprises, including gene ric companies, to develop off-patent 
medicines for children. 
(20) This Regulation should include meas ures to maximise access of the Community 
population to new medicinal pr oducts tested and adapted for paediatric use, and 
minimise the chance of Community-wide rewards and incentives being granted 
without sections of the Community p aediatric population benefiting from the 
availability of a newly au thorised medicine. An a pplication for a marketing 
authorisation, including an application for a Paediatric Use Marketing Authorisation, 
which contains the results of studies conducted in co mpliance with an agreed 
paediatric investigation plan should be eligible for the Community centralised 
procedure set out in Ar ticles 5 to 15 of Regul ation (EC) No 726/2004  of the European 
Parliament and of the Council  of 31 March 2004  laying down Community procedures 
for the authorisation and supe rvision of medicina l products for human and veterinary 
use and establishing a European Medicines Agency7. 
(21) When an agreed paediatric investiga tion plan has led to th e authorisation of a 
paediatric indication for a product alr eady marketed for other indications, the 
marketing authorisation holder should be obliged to place the product on the market 
taking into account the paediatric information within tw o years following the date of 
approval of the indication. That requirement  should relate only to products already 
authorised, but not to medicines author ised via a Paediatric Use Marketing 
Authorisation. 
(22) An optional procedure should be establis hed to make it possible to obtain a single 
Community-wide opinion for a nationally aut horised medicinal product when data on 
children following an agreed paediatric investigation plan form part of the marketing authorisation application. To achieve this, th e procedure set out in Articles 32 to 34 of 
Directive 2001/83/EC may be used. This will allow the adoption of a Community 
harmonised Decision on use of the medicina l product in children and its introduction 
in all national product information. 
(23) It is essential to ensure that pharmac ovigilance mechanisms are adapted to meet the 
specific challenges of collecting safety da ta in children, incl uding data on possible 
long-term effects. Efficacy in children may also need additional study following 
authorisation. Therefore, an  additional requirement fo r applying for a marketing 
authorisation that includes the results of  studies conducted in compliance with an 
agreed paediatric investigat ion plan should be an oblig ation for the applicant to 
indicate how he pr oposes to ensure the long-term follow-up of possible adverse 
reactions to the use of the medicinal produc t and efficacy in the paediatric population. 
Additionally, where there is a particular cause for concer n, provision is made for the 
possibility of requiring the applicant to submit and implement a risk management 
system and/or perform specific post-marketi ng studies as a condition of the marketing 
authorisation. 
(24) For products falling within the scope of the requirement to submit paediatric data, if 
all the measures included in the agreed paed iatric investigation plan are complied with, 
if the product is authorised in all Member  States and if relevant information on the 
                                                
 
7 OJ L 136, 30.4.2004, p. 1. 
 
EN 13   EN results of studies is included in product information, a rewa rd should be granted in the 
form of a 6-month extension of the Supplem entary Protection Certificate created by 
Council Regulation (EEC) No 1768/928. 
(25) Because the reward is for conducting studies  in children and not for demonstrating that 
a product is safe and effectiv e in children, the reward s hould be granted even when a 
paediatric indication is not authorised. Howe ver, to improve the information available 
on the use of medicines in the paediatric  population, relevant information on use in 
paediatric populations should be include d in authorised product information. 
(26) Under Regulation (EC) No  141/2000 of the European Parlia ment and of the Council of 
16 December 1999 on orphan medicinal products9, medicinal products designated as 
orphan medicinal products gain ten years of  market exclusivity on the granting of a 
marketing authorisation for the orphan indi cation. As such products  are frequently not 
patent-protected, the reward of Supplementary Protection Ce rtificate extension cannot 
be applied and when they are patent-prote cted, such an extension would provide a 
double incentive. Therefore, for orphan medi cinal products, instead of extension of the 
Supplementary Protection Certificate, the te n-year period of orphan market exclusivity 
should be extended to twelve ye ars if the requirement for da ta on use in children is 
fully met. 
(27) The measures provided for in this Regulation should not pr eclude the operation of 
other incentives or rewards. To ensure transparency on the different measures 
available at Community and Member State levels, the Co mmission should draw up a 
detailed list of all the incentives availabl e, on the basis of information provided by the 
Member States. The measures set out in this Regulation, including the agreement of 
paediatric investigation plans, should  not be grounds for obtaining any other 
Community incentives to suppor t research, such as the f unding of research projects 
under the multi-annual Community Framework Programmes for Research, 
Technological Development and Demonstration Activities. 
(28) In order to increase the av ailability of information on the use of medicine s in children, 
and to avoid the repetition of studies in children which do not add to the collective 
knowledge, the European databa se provided for in Articl e 11 of Directive 2001/20/EC 
should include an information resource of  all ongoing, prematurely terminated, and 
completed paediatric studies conducted both in the Community and in third countries. 
(29) An inventory of therap eutic needs of children should be adopted by the Paediatric 
Committee after consultation with the Commis sion, the Member Stat es and interested 
parties, and regularly updated. The invent ory should identify the existing medicines 
used by children and highlight the therapeutic  needs of children and the priorities for 
research and development. In this way, co mpanies should be able to identify easily 
opportunities for business development; the Paediatric Committee should be able to better judge the need for me dicines and studies when as sessing draft paediatric 
investigation plans, waivers and deferrals ; and healthcare professionals and patients 
should have an information source availabl e to support their decisions as to which 
medicines to choose. 
                                                
 
8 OJ L 182, 2.7.1992, p. 1. Regulation as la st amended by the Act of Accession 2003. 
9 OJ L 18, 22.1.2000, p. 1. 
 
EN 14   EN (30) Clinical trials in the paediatric popul ation may require specifi c expertise, specific 
methodology and, in some cases, specific f acilities and should be carried out by 
appropriately trained investigators. A netw ork linking together existing national and 
European initiatives and study centres in or der to build up the necessary competences 
at a European level, would help facilitating co-operation and avoiding duplication of 
studies. This network should contribute to the work of strengthening the foundations 
of the European Research Area in the c ontext of Community Framework Programmes 
for Research, Technological Development a nd Demonstration Activities, benefit the 
paediatric population and provi de a source of information and expertise for industry.  
(31) For certain authorised products, pharm aceutical companies may already hold data on 
safety or efficacy in children. To improve  the information available on the use of 
medicines in the paediatric populations, companies holding such data should be 
required to submit them to all competent au thorities where the product is authorised. 
In this way the data can be assessed a nd, if appropriate, information should be 
included in the authorised product informati on aimed at healthcare professionals and 
patients. 
(32) Community funding should be  envisaged to cover all as pects of the work of the 
Paediatric Committee and of the Agency resulting from the implementation of this Regulation, such as the assessment of paedia tric investigation plans, fee waivers for 
scientific advice, and information and tran sparency measures, including the database 
of paediatric studies  and the network. 
(33) The measures necessary for the implemen tation of this Regulation should be adopted 
in accordance with Council Decision 1999/468/EC of 28 June 1999 laying down the procedures for the exercise of impl ementing powers conferred on the Commission
10. 
(34) Regulation (EEC) No 1768/92, Dir ective 2001/83/EC and Regulation (EC) 
No 726/2004 should therefore be amended accordingly, 
HAVE ADOPTED THIS REGULATION: 
TITLE I 
Introductory provisions 
CHAPTER 1 
SUBJECT -MATTER AND DEFINITIONS  
Article 1 
This Regulation lays down rule s concerning the development of medicinal products for 
human use in order to meet the specific therapeu tic needs of the paediatric population, without 
subjecting children to unnecessary clinical trials and in compliance with Directive 2001/20/EC. 
                                                
 
10 OJ L 184, 17.7.1999, p. 23. 
 
EN 15   EN Article  2 
In addition to the definitions laid down in Article 1 of Directive 2001/83/EC, the following 
definitions shall apply for the purposes of this Regulation: 
(1) paediatric population  means that part of the population aged between birth and 
18 years; 
(2) paediatric investigation plan means a research and development programme aimed 
at ensuring that the necessary  data are generated determining the conditions in which 
a medicinal product may be authorised to treat the paediatric population. 
(3) medicinal product authorised for a paediatric indication  means a medicinal product 
which is authorised for use in part or all of the paediatric population and in respect of which the details of the authorised indica tion are specified in the summary of the 
product characteristics drawn up in  accordance with Article 11 of 
Directive 2001/83/EC.  
CHAPTER 2 
PAEDIATRIC COMMITTEE  
Article 3 
1. A Paediatric Committee is established within the European Medicines Agency set up 
under Regulation (EC) No 726/2004, hereinafter “the Agency”. 
The Agency shall act as the secretariat of  the Paediatric Committee and shall provide 
it with technical and scientific support. 
2. Save where otherwise provided in this  Regulation, Regulati on (EC) No 726/2004 
shall apply to the Paediatric Committee. 
3. The Executive Director of the Agency shall ensure appropriate co-ordination 
between the Paediatric Committee and th e Committee for Medicinal Products for 
Human Use, the Committee for Orphan Medicinal Products, their working parties and any other scientif ic advisory groups. 
The Agency shall draw up specific proce dures for possible consultations between 
them. 
Article 4 
1. The Paediatric Committee shall be co mposed of the following members: 
(a) five members of the Committee fo r Medicinal Products for Human Use, 
appointed by the latter; 
 
EN 16   EN (b) one person appointed by each Member State whose national competent 
authority is not represented through the members appointed by the Committee 
for Medicinal Products for Human Use; 
(c) six persons appointed by the Comm ission, on the basis of a public call for 
expressions of interest, in order to represent paediatricians and the interests of 
patient associations. 
For the purposes of point  (b), the Member States  shall cooperate, under the 
coordination of the Executive Dir ector of the Agency, in orde r to ensure that the final 
composition of the Paediatric Committee covers the scientific areas relevant to paediatric medicinal products, and includi ng at least: pharmaceut ical development, 
paediatric medicine, paediatric pharm acy, paediatric pharmacology, paediatric 
research, pharmacovigilance and ethics. 
2. The members of the Paediatric Committee sh all be appointed for a renewable period 
of three years. At meetings of the Paediatric Committee, they may be accompanied by experts. 
3. The Paediatric Committee shall elect its  Chairman from among its members for a 
term of three years renewable once. 
4. The names and scientific qualifications of the members shall be published by the 
Agency. 
Article 5 
1. When preparing its opinions, the Paediatric  Committee shall use its best endeavours 
to reach a scientific consensus. If such a consensus cannot be reached, the opinion shall consist of the position of the majority  of members and dive rgent positions, with 
the grounds on which they are based.  
2. The Paediatric Committee shall draw up its own rules of procedure for the 
implementation of its tasks. The rules of procedure shall enter into force after 
receiving a favourable opinion from the Management Board of the Agency and, 
subsequently, from the Commission. 
3. All meetings of the Paediatric Committee may be attended by representatives of the 
Commission, the Executive Director of th e Agency or his representatives. 
Article 6 
Members of the Paediatric Committee and its experts shall undertake to act in the public 
interest and in an independent manner. They sha ll not have financial or other interests in the 
pharmaceutical industry that coul d affect their impartiality. 
All indirect interests that coul d relate to the pharmaceutical industry shall be entered in a 
register held by the Agency which the public may consult. The register shall be updated 
annually. 
 
EN 17   EN Members of the Paediatric Committee and its experts shall declare at each meeting any 
specific interests which could be considered to  be prejudicial to th eir independence with 
respect to the points on the agenda. 
Members of the Paediatric Committee and its expe rts shall be required, ev en after their duties 
have ceased, not to disclose any information of the kind covered by the obligation of professional secrecy. 
Article 7 
1. The tasks of the Paediatric Committee shall include the following: 
(a) to assess the content of any paediatr ic investigation plan for a medicinal 
product submitted to it in accordance with this Regulation and formulate an opinion thereon; 
(b) to assess waivers and deferrals  and formulate an opinion thereon; 
(c) at the request of the Committee fo r Medicinal Products for Human Use, a 
competent authority or the applicant, to  assess compliance of the application 
for a Marketing Authorisation with the concerned agreed paediatric 
investigation plan and form ulate an opinion thereon; 
(d) at the request of th e Committee for Medicinal Pr oducts for Human Use or a 
competent authority, to assess any data generated in accordance with an agreed paediatric investigation plan and form ulate an opinion on the quality, safety or 
efficacy of the medicinal product for use in the paediatric population; 
(e) to advise on the content and format of data to be collected for the survey 
referred to in Article 41 and to adopt an inventory of therapeutic needs as 
referred to in Article 42; 
(f) to support and advise the Agency on establishing the European network 
referred to in Article 43; 
(g) to assist scientifically in the elab oration of any documents related to the 
fulfilment of the objectives  of this Regulation; 
(h) to provide advice on any question related to medicina l products for paediatric 
use, at the request of the Executive Dire ctor of the Agency or the Commission. 
2. When carrying out its tasks, the Paediatric Committee shall consider whether or not 
any proposed studies can be expected to be  of significant therapeutic benefit to the 
paediatric population. 
 
EN 18   EN TITLE II 
Marketing authorisation requirements 
CHAPTER 1 
GENERAL AUTHORISATION REQUIREMENTS  
Article 8 
1. An application for marketing authorisat ion under Article 6 of Directive 2001/83/EC 
in respect of a medicinal product for human  use which is not authorised in the 
Community at the time of entry into force of this Regulation sh all be regarded as 
valid only if it includes, in addition to the particulars and documents referred to in Article 8(3) of Directive 2001/ 83/EC, one of the following: 
(a) the results of all studies performed a nd details of all information collected in 
compliance with an agreed p aediatric investigation plan; 
(b) a decision of the Agency granting a product-specific waiver; (c) a decision of the Agency granting a class waiver; (d) a decision of the Agency granting a deferral. For the purposes of point (a), the decisi on of the Agency agreeing the paediatric 
investigation plan concerned shall al so be included in the application. 
2. The documents submitted pursuant to pa ragraph 1 shall, cu mulatively, cover all 
subsets of the p aediatric population. 
Article 9 
In the case of authorised medicinal products which are protected either by a supplementary 
protection certificate under Re gulation (EEC) No 1768/92, or by a patent which qualifies for 
the granting of the supplementary protection ce rtificate, Article 8 of this Regulation shall 
apply to applications for auth orisation of new indications, in cluding paediatr ic indications, 
new pharmaceutical forms and ne w routes of administration. 
Article  10 
Articles 8 and 9 shall not apply to products au thorised under Articles 10, 10a, 13 to 16 or 16a 
to 16i of Directive 2001/83/EC. 
 
EN 19   EN Article 11 
In consultation with the Member States, the Agency and other interested parties, the 
Commission shall draw up the modalities c oncerning the format and content which 
applications for agreement or modification of a paediatric investigation plan and requests for 
waivers or deferrals must follow in order to be considered valid. 
CHAPTER 2 
WAIVERS  
Article 12 
1. Production of the information referred to in point (a) of Article 8(1) shall be waived 
for specific medicinal products or for cla sses of medicinal products, if there is 
evidence showing any of the following: 
(a) that the specific medicinal product or cla ss of medicinal produc ts is likely to be 
ineffective or unsafe in part or all of the paediatric population; 
(b) that the disease or condition for which the specific medicinal product or class is 
intended occurs only in adult populations; 
(c) that the specific medici nal product does not represen t a significant therapeutic 
benefit over existing treatments for paediatric patients. 
2. The waiver provided for in paragraph 1 may be issued with reference only to one or 
more specified subsets of the paediatric  population, with reference only to one or 
more specified therapeutic indications, or with reference to a combination of both. 
Article 13 
The Paediatric Committee may of its own motion adopt an opinion, on the grounds set out in 
Article 12(1), to the effect th at a class or a product-specifi c waiver, as referred to in 
Article 12(1), should be granted. 
As soon as the Paediatric Committee adopts an opinion, the procedure laid down in Chapter 4 
shall apply. In the case of a class waiver, only Article 26(4) shall apply. 
Article 14 
1. The applicant may, on the grounds set out in Article 12(1), apply to the Agency for a 
product-specific waiver. 
2. Within 60 days of receipt of the applica tion, the Paediatric Committee shall adopt an 
opinion as to whether or not a product -specific waiver s hould be granted. 
 
EN 20   EN Either the applicant or the Paediatric Committee may request a meeting during that 
60-day period. 
Whenever appropriate, the Paediatric Co mmittee may request the applicant to 
supplement the particulars and documen ts submitted. Where the Paediatric 
Committee avails itself of this option, th e 60-day time-limit shall be suspended until 
such time as the supplementary inform ation requested has been provided. 
3. As soon as the Paediatric Committee adopt s an opinion, the procedure laid down in 
Chapter 4 shall apply. The Agency shall in form the applicant accordingly without 
delay. The applicant shall be informed of  the reasons for the conclusion reached. 
Article 15 
1. The Agency shall maintain a list of all waivers. 
2. The Paediatric Committee may, at any time, adopt an opinion advocating the review 
of a granted waiver. 
In the case of a change affecting a product- specific waiver, the procedure laid down 
in Chapter 4 shall apply. 
In the case of a change affecting a cl ass waiver, Article 26(5) shall apply. 
3. If a particular product-specific or class wa iver is revoked, the requirement set out in 
Articles 8 and 9 shall not apply for 36 months  from the date of its removal from the 
list of waivers. 
CHAPTER 3 
PAEDIATRIC INVESTIGATION PLAN  
SECTION 1 
REQUESTS FOR AGREEMENT  
Article 16 
1. Where the intention is to apply in accordan ce with points (a) or (d) of Article 8(1), a 
paediatric investigation plan shall be dr awn up and submitted to  the Agency with a 
request for agreement. 
2. The paediatric investigation plan shall specify the timing and the measures proposed 
to assess the quality, safety and efficacy of the medicinal product in all subsets of the 
paediatric population that may be concer ned. In addition, it shall describe any 
measures to adapt the formulation of the medicinal product so as to make its use 
more acceptable, easier, safer or more effec tive for different subsets of the paediatric 
population. 
 
EN 21   EN Article 17 
1. In the case of applications as referred to in Articles 8 and 9, the paediatric 
investigation plan shall be submitted with a request for agreement, unless otherwise 
justified, not later than upon completion of the human pharmaco-kinetic studies in adults specified in Section 5.2.3 of Part I of Annex I to Direc tive 2001/83/EC, so as 
to ensure that an opinion on use in the paediatric population of the medicinal product 
concerned can be given at the time of th e assessment of the ma rketing authorisation 
or other application concerned. 
2. Within 30 days of receiving the request referred to in paragraph 1, the Agency shall 
verify the validity of th e request and shall prepar e a summary report for the 
Paediatric Committee. 
3. Whenever appropriate, the Agency may ask the applicant to  submit additional 
particulars and documents, in which case  the time-limit of 30 days shall be 
suspended until such time as the supplem entary information requested has been 
provided. 
Article 18 
1. Within 60 days of receiving a proposed paedia tric investigation plan which is valid, 
the Paediatric Committee shall adopt an op inion as to whether or not the proposed 
studies will ensure the gene ration of the necessa ry data determining the conditions in 
which the medicinal product may be used to  treat the paediatric  population or subsets 
thereof, and as to whether or not the expect ed therapeutic benefits justify the studies 
proposed. 
Within the same period, either the applicant or the Paediatric Committee may request 
a meeting. 
2. Within the 60-day period referred to in paragraph 1, the Paediatric Committee may 
request the applicant to propose modifica tions to the plan, in which case the 
time-limit referred to in paragraph 1 for th e adoption of the final opinion shall be 
extended for a maximum of 60 days. In such  cases, the applicant or the Paediatric 
Committee may request an additional meeting during this period. The time-limit shall be suspended until such time as th e supplementary information requested has 
been provided. 
Article 19 
As soon as the Paediatric Committee adopts an opinion, whether positive or negative, the 
procedure laid down in Chapter 4 shall apply. 
Article 20 
If, having considered a paediatr ic investigation plan, the Paediatric Committee concludes that 
Article 12(1)(a), (b) or (c) applies to the medicinal product concerned, it shall adopt a 
negative opinion under Article 18(1). 
 
EN 22   EN In such cases, the Paediatric Committee shal l adopt an opinion in favour of a waiver under 
Article 13, whereupon the procedure laid  down in Chapter 4 shall apply. 
SECTION 2 
DEFERRALS  
Article 21 
1. At the same time as the paediatric investigation plan is submitted under 
Article 17(1), a request may be made for de ferral of the initiation or completion of 
some or all of the measures set out in that plan. Such deferral shall be justified on 
scientific and technical grounds or on  grounds related to public health.  
In any case, a deferral shall be granted wh en it is appropriate to conduct studies in 
adults prior to initiating studies in the paediatric population or when studies in the 
paediatric population will take longer to conduct than st udies in adults. 
2. On the basis of the experience acquired as a result of the operation of this Article, the 
Commission may adopt provisions in accordan ce with the procedure referred to in 
Article 51(2) to define further the grounds for granting a deferral. 
Article 22 
1. At the same time as the Paediatric  Committee adopts a positive opinion under 
Article 18(1), it shall, of its own motion or following a request submitted by the applicant under Article 21, adopt an opinion, if the conditions specified in Article 21 
are met, in favour of deferring the initiati on or completion of some or all of the 
measures in the paediatric investigation plan. 
An opinion in favour of a deferral shall spec ify the time-limits for initiating or 
completing the measures concerned. 
2. As soon as the Paediatric Committee adopts an opinion in favour of deferral, as 
referred to in paragraph 1, the procedur e laid down in Chapter 4 shall apply. 
SECTION 3 
MODIFICATION OF A PAEDIATRIC INVESTIGATION PLAN  
Article 23 
If, after the decision agreeing the paediatric investigation plan, the applicant encounters 
difficulties with its implementation such as to render the plan unworkable or no longer 
appropriate, the applicant may propose changes or request a deferral or a waiver, based on 
detailed grounds, to the Paediatric Committee.  The Paediatric Committee shall review these 
changes and adopt an opinion proposing their refu sal or acceptance. As s oon as the Paediatric 
Committee adopts an opinion, whet her positive or negative, the procedure laid down in 
Chapter 4 shall apply. 
 
EN 23   EN SECTION 4 
COMPLIANCE WITH THE PAEDIATRIC INVESTIGATION PLAN  
Article 24 
The Paediatric Committee may, in the following cas es, be requested to gi ve its opinion as to 
whether studies conducted by the applicant are in compliance with the agreed paediatric 
investigation plan: 
(a) by the applicant, prior to submitting an application for marketing authorisation or 
variation as referred to in Articles 8 and 9, respectively; 
(b) by the Agency, or the competent author ity, when validating an application, as 
referred to in point (a), which does not  include an opinion concerning compliance 
adopted following a request under point (a); 
(c) by the Committee for Medicinal Products  for Human Use, or the competent 
authority, when assessing an application, as referred to in point (a), where there is 
doubt concerning compliance and an opinion has not been given already following a 
request under points (a) or (b). 
Member States shall take account of such an opinion. If the Paediatric Committee is asked to give an opinion under the first paragraph, it must do so 
within 60 days of receiving the request. 
Article 25 
If, when conducting the scientific assessment of  a valid application, the competent authority 
concludes that the studies are not  compliant with the agreed paediatric investigation plan, the 
product shall not be eligible for the re wards provided for in Articles 36 and 37. 
CHAPTER 4 
PROCEDURE  
Article 26 
1. Within 30 days of receiving the opinion of  the Paediatric Committee, the applicant 
may submit to the Agency a written request, on detailed grounds, for a 
re-examination of the opinion. 
2. Within 30 days of receiving a request for re-examination pursuant to paragraph 1, the 
Paediatric Committee, having appointed a new rapporteur, shall issue a new opinion 
confirming or revising its previous opini on. The opinion shall be duly reasoned and a 
statement of reasons for the conclusion r eached shall be annexed to the new opinion, 
which shall become definitive. 
 
EN 24   EN 3. If, within the 30-day period referred to in paragraph 1, th e applicant does  not request 
re-examination, the opinion of the Paediatric Committee shall become definitive. 
4. The Agency shall adopt a decision w ithout delay. This decision shall be 
communicated to the applicant. 
5. In the case of a cl ass waiver as referred to in Article 13, the Agency shall adopt a 
decision, which shall be published. 
CHAPTER 5 
MISCELLANEOUS PROVISIONS  
Article 27 
The sponsor of a medicinal product intended for paediatric use may, prior to the submission 
of a paediatric inves tigation plan and during its implem entation, request advice from the 
Agency on the design and conduct of the various tests and studies necessary to demonstrate 
the quality, safety and efficacy of the medi cinal product in the paediatric population in 
accordance with Article 57(1)(n) of Regulation (EC) No 726/2004. 
In addition, the sponsor may request advice on the design and conduc t of pharmacovigilance 
and risk management systems as referred to in Article 35. 
The Agency shall provide advice under this Article free of charge. 
TITLE III 
Marketing authorisation procedures 
Article 28 
Save where otherwise provided in  this Title, marketing authorisation procedures for the 
marketing authorisations covered by this Titl e shall be governed by the provisions laid down 
in Regulation (EC) No 726/2004 or in Directive 2001/83/EC. 
CHAPTER 1 
MARKETING AUTHORISATION PROCEDURES FOR APPLICATIONS FALLING 
WITHIN THE SCOPE OF ARTICLES 8 AND 9 
Article 29 
1. Applications may be submitted in accord ance with the procedure laid down in 
Articles 5 to 15 of Regulation (EC) No 726/2004 for a marketing authorisation as 
referred to in Article 8(1) of this Regulat ion which includes one or more paediatric 
 
EN 25   EN indications selected on the ba sis of studies cond ucted in compliance with an agreed 
paediatric investigation plan. 
Where authorisation is granted, the results of those studies shall be included in the 
summary of product characteristics and, if a ppropriate, in the p ackage leaflet of the 
medicinal product, whether or not all the paediatric indications concerned were 
approved. 
2. Where a marketing authorisa tion is granted or varied, a ny waiver or deferral which 
has been granted pursuant to  this Regulation shall be recorded in the summary of 
product characteristics and if  appropriate, the package leaflet of the medicinal 
product concerned. 
3. If the application complies with all the m easures contained in the agreed completed 
paediatric investigation plan  and if the summary of pr oduct characteristics reflects 
the results of studies conducted in comp liance with that ag reed paediatric 
investigation plan, the competent author ity shall include within the marketing 
authorisation a statement indicating complia nce of the application with the agreed 
completed paediatric investigation plan. 
Article 30 
In the case of medicinal products authorised  under Directive 2001/83/EC, an application as 
referred to in Article 9 of this Regulation may be submitted, in accordance with the procedure laid down in Articles 32, 33 and 34 of Dir ective 2001/83/EC, for authorisation of a new 
indication, including the extension of an author isation for use in the paediatric population, a 
new pharmaceutical form or a new route of administration. 
That application shall comply with the require ment laid down in point  (a) of Article 8(1). 
The procedure shall be limited to the assessment of the specific sections of the summary of 
product characteristic s to be varied. 
CHAPTER 2 
PAEDIATRIC USE MARKETING AUTHORISATION  
Article 31 
1. For the purposes of this Regulation, a paed iatric use marketing authorisation shall 
mean a marketing authorisa tion granted in respect of a medicinal product for human 
use which is not protected by a supplem entary protection certificate under 
Regulation (EEC) No 1768/92 or by a patent which qualifies for the granting of the 
supplementary protection certificate, cove ring exclusively ther apeutic indications 
which are relevant for use in  the paediatric population, or  subsets thereof, including 
the appropriate strength, pharmaceutical form  or route of administration for that 
product. 
 
EN 26   EN 2. Submission of an applicati on for a paediatric use marke ting authorisation shall in no 
way preclude the right to apply for a mark eting authorisation for other indications. 
3. An application for a paediatric use marke ting authorisation shal l be accompanied by 
the particulars and documents necessary to establish safety, quality and efficacy in children, including any specific data needed  to support an appropriate strength, 
pharmaceutical form or route of administrati on of the product, in accordance with an 
agreed paediatric investigation plan. 
The application shall also include the decision of the Agency agreeing the paediatric 
investigation plan concerned. 
4. Where a medicinal product is or has been authorised in a Member State or in the 
Community, data contained in the dossier on that product may, where appropriate, be 
referred to, in accordance with Article 14 (11) of Regulation (EC) No 726/2004 or 
Article 10 of Directive 2001/83/EC, in an a pplication for a paediatric use marketing 
authorisation. 
5. The medicinal product in resp ect of which a paediatric us e marketing authorisation is 
granted may retain the name of any me dicinal product which contains the same 
active substance and in respect of wh ich the same holder has been granted 
authorisation for use in adults. 
Article 32 
Without prejudice to Article 3(2) of Regul ation (EC) No 726/2004, an application for a 
paediatric use marketing authorisation may be  made in accordance with the procedure laid 
down in Articles 5 to 15 of Regulation (EC) No 726/2004. 
CHAPTER 3 
IDENTIFICATION 
Article 33 
Where a medicinal product is granted a marke ting authorisation for a paediatric indication 
based on the results of studi es conducted in compliance w ith an agreed paediatric 
investigation plan, the label shall display the name of th e medicinal product followed by a 
superscript of the letter “P” in blue lettering surrounded by the outli ne of a star, also in blue, 
for any paediatric presentation. 
The first paragraph shall apply whether the na me of the medicinal product is an invented 
name or a common name as referred to in Article 1(20) and (21) , respectively, of 
Directive 2001/83/EC. 
 
EN 27   EN TITLE IV 
Post-authorisation requirements 
Article 34 
Where medicinal products are authorised for a paediatric indication following completion of 
an agreed paediatric investigation plan and t hose products have already been marketed with 
other indications, the marketing authorisation ho lder shall, within two years of the date on 
which the paediatric indication is authoris ed, place the product on the market taking into 
account the paediatric indication. 
Article 35 
1. In the following cases, the applicant shall detail, in addition to the normal 
requirements for post-marketing monitoring, th e measures to ensure the follow-up of 
efficacy and of possible adverse reactions to the paediatric us e of the medicinal 
product: 
(a) applications for a marketing authorisa tion that includes a paediatric indication; 
(b) applications to include a paediatric indication in an existing marketing 
authorisation; 
(c) applications for a paediatr ic use marketing authorisation. 
2. Where there is particular cause for con cern, the competent authority may require, as 
a condition for granting marketing authorisat ion, that a risk management system be 
set up or that specific post-marketing studies be performed and submitted for review. The risk management system  shall comprise a set of ac tivities and interventions 
designed to prevent or minimise risks re lating to medicinal products, including the 
assessment of the effectiveness of those interventions. 
Assessment of the effectiveness of any risk  management system and the results of 
any studies performed shall be included in the periodic sa fety update reports referred 
to in Article 104(6) of Directive 2001/83/ EC and Article 24(3) of Regulation (EC) 
No 726/2004. 
In addition, the competent authority may request submission of additional reports 
assessing the effectiveness of any risk mi nimisation system and the results of any 
such studies performed. 
3. In the case of a deferral, the marketing authorisation holder shall submit an annual 
report to the Agency providing an update on progress with paediatric studies in 
accordance with the decision of Agency agr eeing the paediatric investigation plan 
and granting a deferral. 
 
EN 28   EN The Agency shall inform the competent auth ority if it is found that the marketing 
authorisation holder has failed to comply with the decision of Agency agreeing the 
paediatric investigation pl an and granting a deferral. 
4. The Agency shall draw up detailed guidelin es relating to the application of this 
Article. 
TITLE V 
Rewards and incentives 
Article 36 
1. Where an application under Articles 8 or  9 includes the results of all studies 
conducted in compliance with an agreed paed iatric investigation plan, the holder of 
the patent or supplementary protection cer tificate shall be entit led to a six-month 
extension of the period referred to in Ar ticles 13(1) and 13(2) of Regulation (EEC) 
No 1768/92. 
The first subparagraph shall also apply where completion of the agreed paediatric 
investigation plan fails to l ead to the authorisat ion of a paediatric indication, but the 
results of the studies conducted are re flected in the summary of product 
characteristics, and if appropriate, in th e package leaflet of the medicinal product 
concerned. 
2. The inclusion in a marketing authoris ation of the statement referred to in 
Article 29(3) shall be used for the purposes  of applying paragraph 1 of this Article. 
3. Where the procedures laid down in Directive 2001/83/EC have been used, the 
six-month extension of the period referred to  in paragraph 1 shall be granted only if 
the product is authorised in all Member States. 
4. Paragraphs 1, 2 and 3 shall apply to products that are protected by a supplementary 
protection certificate under Regulation (EEC) No 1768/92, or under a patent which 
qualifies for the granting of the supplement ary protection certifi cate.They shall not 
apply to medicinal products  designated as orphan medi cinal products pursuant to 
Regulation (EC) No 141/2000.  
Article 37 
Where an application for a marketing authoris ation is submitted in respect of a medicinal 
product designated as an orphan medicinal pr oduct pursuant to Regulation (EC) No 141/2000 
and that application includes the results of all studies conducted in comp liance with an agreed 
paediatric investigation plan, and the statement referred to in Article 29(3) of this Regulation 
is subsequently included in the marketing auth orisation granted, the te n-year period referred 
to in Article 8(1) of Regul ation (EC) No 141/2000 shall be extended to twelve years. 
 
EN 29   EN The first paragraph shall also apply where comp letion of the agreed paediatric investigation 
plan fails to lead to the author isation of a paediatric indicati on, but the results of the studies 
conducted are reflected in the su mmary of product characteristic s, and if appropriate, in the 
package leaflet of the me dicinal product concerned. 
Article 38 
1. Where a paediatric use marketing author isation is granted in accordance with 
Articles 5 to 15 of Regulation (EC) No 726/2004, the data and marketing protection 
periods referred to in Article 14(11) of that  Regulation shall apply. 
2. Where a paediatric use marketing authoris ation is granted in accordance with the 
procedures laid down in Directive 2001/83/ EC, the data and ma rketing protection 
periods referred to in Article 10 (1) of that Directive shall apply. 
Article 39 
1. In addition to the rewards and incentiv es provided for in Articles 36, 37 and 38, 
medicinal products for paediatric use may be  eligible for incentives provided by the 
Community or by the Member States to s upport research into, and the development 
and availability of, medicinal products for paediatric use. 
2. Within one year of the entry into force of  this Regulation, the Member States shall 
communicate to the Commission detailed info rmation concerning any measures they 
have enacted to support res earch into, and the developm ent and availa bility of, 
medicinal products for paediatric use. This  information shall be updated regularly at 
the request of the Commission. 
3. Within 18 months of the entry into force of this Regulation, the Commission shall 
publish a detailed inventory of all in centives provided by the Community and 
Member States to support research into, and the development and availability of, 
medicinal products for paediatric use. Th is inventory shall be updated regularly. 
TITLE VI 
Communication and coordination 
Article 40 
1. Appropriate details of trials  that are contained within agreed paediatric investigation 
plans, including those that ar e carried out in third countries , shall be entered into the 
European database created by Ar ticle 11 of Directive 2001/20/EC. 
 
EN 30   EN 2. The Commission shall, on a proposal by the Agency and in consultation with 
Member States and interested parties,  draw up guidance on the nature of the 
information referred to in paragraph 1 to be  entered in the European database created 
by Article 11 of Directive 2001/20/EC. 
Article 41 
Member States shall collect available data on all existing uses of medicinal products in the 
paediatric population and, within  2 years of the entry into fo rce of this Regulation, shall 
communicate these data to the Agency. 
The Paediatric Committee shall provide guidance on the content and the format of the data to 
be collected. 
Article 42  
1. The Agency shall assess the data referred to in Article 41, in partic ular with a view to 
identifying research priorities. 
2. On the basis of the assessment under paragraph 1 and other information available, 
and following consultation with the Commi ssion, the Member States and interested 
parties, the Paediatric Committee shall esta blish an inventory of therapeutic needs. 
The Agency shall publish the i nventory within 3 years of th e entry into force of this 
Regulation, and shall update  it on a regular basis. 
3. In establishing the inventory of therapeutic needs, account shall be taken of the 
prevalence of the conditions in the paediatric populati on, the seriousness of the 
conditions to be treated, the availability a nd suitability of altern ative treatments for 
the conditions in the paediatric population, including the efficacy and the adverse reaction profile of those tr eatments, including any unique paediatric safety issues. 
Article 43 
1. The Agency shall, with the scientific s upport of the Paediatric Committee, develop a 
European network of existing national a nd European networks, investigators and 
centres with specific expertise in the pe rformance of studies in the paediatric 
population. 
2. The objectives of the European network shall be, inter alia , to coordinate studies 
relating to paediatric medicinal products, to build up the necessa ry scientific and 
administrative competences at European le vel, and to avoid duplication of studies 
and testing in children. 
3. Within one year of the entry into force of this Regulation, the Management Board of 
the Agency shall, on a proposal from  the Executive Director and following 
consultation with the Commission, the Member  States and interest ed parties, adopt 
an implementing strategy for the launchi ng and operation of the European network. 
This network must, where appropriate, be co mpatible with the work of strengthening 
 
EN 31   EN the foundations of the European Research Area in the context of the Community 
Framework Programmes for Researc h, Technological Development and 
Demonstration Activities. 
Article 44 
1. Within one year of the entry into force of this Regulation, a ny paediatric studies 
already completed, by the date of entry into  force, in respect of products authorised 
in the Community shall be submitted for assessment to the competent authority. 
The competent authority shall, as appropriate, update the summary of product 
characteristics and package leaflet, and shall vary the marketing authorisation 
accordingly. Competent authorities shall ex change information regarding the studies 
submitted and their implications for any marketing authorisations concerned. 
The Agency shall coordinate the exchange of information. 
2. All existing paediatric studies, as referre d to in paragraph 1, shall be taken into 
consideration by the Paediatric Committee when assessing applications for paediatric investigation plans, waivers and deferra ls and by competent authorities when 
assessing applications submitted pursuant to Articles 8, 9 or 31. 
3. No paediatric studies, as referred to in pa ragraph 1, which have at the date of entry 
into force of this Regulation already been submitted for assessment in a third country, shall be taken into consideration for the rewards and incentives provided for 
in Articles 36, 37 and 38. 
Article 45 
Any other marketing authorisation holder spons ored studies which involve the use in the 
paediatric population of a medicinal product co vered by a marketing authorisation, whether or 
not they are conducted in complia nce with an agreed paediatric investiga tion plan, shall be 
submitted to the competent authority within six months of the completion of the studies 
concerned. 
The first paragraph shall apply whether or not the marketing authorisa tion holder intends to 
apply for a paediatric indication. 
The competent authority shall, as appropriate , update the summary of  product characteristics 
and package leaflet, and shall vary the marketing authorisation accordingly. 
Competent authorities shall exchange information regarding the studies submitted and their 
implications for any marketi ng authorisations concerned. 
The Agency shall coordinate the exchange of information. 
 
EN 32   EN TITLE VII 
General and fina l provisions 
CHAPTER 1 
GENERAL  
SECTION 1 
FEES, COMMUNITY FUNDING , PENALTIES AND REPORTS  
Article 46 
1. Where an application for a paediatric us e marketing authorisation is submitted in 
accordance with the procedure laid dow n in Regulation (EC) No 726/2004, the 
amount of the reduced fees for the examination of the application and the maintenance of the marketing authorisa tion shall be fixed in accordance with 
Article 70 of Regulation (EC) No 726/2004. 
2. Council Regulation (EC) No 297/95
11 shall apply. 
3. Assessments of the following by the Paedia tric Committee shall be free of charge: 
(a) applications for waiver; (b) applications for deferral; (c) paediatric investigation plans; (d) compliance with the agreed paediatric investigation plan. 
Article 47 
The Community contribution provided for in Article 67 of Regulation (EC) No 726/2004 shall 
cover all aspects of the work of the Paedia tric Committee, including scientific support 
provided by experts, and of the Agency, includ ing the assessment of paediatric investigation 
plans, scientific advice and any fee waivers provided for in th is Regulation, and shall support 
the Agency’s activities under Arti cles 40 and 43 of this Regulation. 
Article 48  
1. Without prejudice to the Protocol on the Privileges and Immuniti es of the European 
Communities, each Member State shall determine the penalties to be applied for 
                                                
 
11 OJ L 35, 15.2.1995, p. 1. 
 
EN 33   EN infringement of the provisions of this Regulation or the implementing measures 
adopted pursuant to it in relation to medicinal produc ts authorised through the 
procedures laid down in Directive 2001/83/EC  and shall take all measures necessary 
for their implementation. The penalties sh all be effective, proportionate and 
dissuasive. 
Member States shall inform the Commission of these provisions no later than […]. 
They shall notify any subsequent alterations as soon as possible. 
2. Member States shall inform the Commissi on immediately of any litigation instituted 
for infringement of this Regulation. 
3. At the Agency's request, the Commission may impose financial penalties for 
infringement of the provisions of this Regulation or the implementing measures 
adopted pursuant to it in relation to medicinal produc ts authorised through the 
procedure laid down in Regulation (EC) No 726/2004. The maximum amounts as well as the conditions and methods for coll ection of these penalties shall be laid 
down in accordance with the procedure referred to in Article 51(2) of this Regulation. 
4. The Commission shall publish the names of  the marketing aut horisation holders 
involved and the amounts of and reasons  for the financial penalties imposed. 
Article 49 
1. On the basis of a report from the Agency and at least on an annual basis, the 
Commission shall publish a list of the companies that have benefited from any of the 
rewards and incentives in this Regulation and the companies that have failed to comply with any of the obligations in this Regulation. The Member States shall 
provide this information to the Agency. 
2. Within six years of entry into force of this Regulation, the Commission shall publish 
a general report on experience acquired as a result of its application, including in 
particular a detailed inventor y of all medicinal products au thorised for p aediatric use 
since its entry into force. 
SECTION 2 
COMMITTEE  
Article 50 
The Commission shall, after consulting the Agency, adopt appropriate provisions for the 
performance of the tasks of the Paediatric Com mittee referred to in Articl e 7 in the form of a 
Regulation in accordance with the pro cedure referred to in Article 51(2). 
 
EN 34   EN Article 51 
1. The Commission shall be assisted by th e Standing Committee on Medicinal Products 
for Human Use set up by Article  121 of Directive 2001/83/EC. 
2. Where reference is made to this  paragraph, Articles 5 and 7 of 
Decision 1999/468/EC shall apply, having re gard to the provisions of Article 8 
thereof. 
The period laid down in Article 5(6) of Decision 1999/468/EC shall be set at three 
months. 
CHAPTER 2 
AMENDMENTS  
Article 52 
Regulation (EEC) No 1768/92 is amended as follows: 
(1) In Article 7, the following paragraph 3 is added: 
“3. The application for an extension of th e duration of a certificate already granted 
in application of Article 13(3) of this Regulation and of Article 36 of 
Regulation (EC) No […/… of the Eur opean Parliament and of the Council 
(Paediatric Regulation)*] sh all be lodged not later than two years before the 
expiry of the certificate. 
_____________________ 
* OJ L […], […], p. […].” 
(2) Article 8 shall be amended as follows: 
(a) in paragraph 1, the following point (d) is added: 
“(d) where the application for a certificat e includes a request for an extended 
duration in application of Article 13( 3) of this Regulation and Article 36 
of Regulation (EC) No […/… (Paediatric Regulation)]: 
(i) a copy of the statement indicat ing compliance with an agreed 
completed paediatric investigation plan as referred to in Article 36(3) of Regulation (EC) No […/…(Paediatric Regulation)]; 
(ii) where necessary, in addition to the copy of the authorisations to 
place the product on the market as refe rred to in point (b), copies of 
the authorisations to place the pr oduct on the market of all other 
Member States, as referred to in Article 36(4) of Regulation (EC) 
No […/… (Paediatric Regulation)] .” 
 
EN 35   EN (b) the following paragraph 1a is inserted: 
“1a. The application for an extension of  the duration of a certificate already 
granted shall contain: 
(a) a copy of the certific ate already granted; 
(b) a copy of the statement indicat ing compliance with an agreed 
completed paediatric investigation plan as referred to in Article 36(3) of Regulation (EC) No […/… (Paediatric 
Regulation)]; 
(c) copies of the authorisations to  place the product on the market of 
all Member States.” 
(c) paragraph 2 is replaced by the following: 
“2. Member States may provide that a fee is to be payable upon application 
for a certificate and upon application for the extension of the duration of 
a certificate.” 
(3) Article 9 is amended as follows: 
(a) in paragraph 1, the follo wing subparagraph is added: 
“The application for an extension of  the duration of a certificate already 
granted shall be lodged with the compet ent industrial property office of the 
Member State which granted the certificate.” 
(b) the following paragraph 3 is added: 
“3. Paragraph 2 shall apply to the no tification of the application for an 
extension of the duration a certificat e already granted. The notification 
shall additionally contain the request for an extended duration of the certificate in application of Ar ticle 36 of Regulation (EC) 
No […/… (Paediatric Regulation)].” 
(4) In Article 11, the follo wing paragraph 3 is added: 
“3. Paragraphs 1 and 2 shall apply to the no tification of the fact that an extension 
of the duration of a certifi cate already granted has been  granted or rejected.” 
(5) In Article 13, the follo wing paragraph 3 is added: 
“3. The periods laid down in paragraphs 1 and 2 shall be exte nded by six months 
in the case of application of Article 36 of Regulation (EC) No […/… (Paediatric Regulation)]. In th at case, the duration of the period laid 
down in paragraph 1 of this Article may be extended only once.” 
 
EN 36   EN Article 53 
In Article 6 of Directive 2001/83/EC, the first subparagraph of paragraph 1 is replaced by the 
following: 
“No medicinal product may be placed on the mark et of a Member State unless a marketing 
authorisation has been issued by the competent au thorities of that Member  State in accordance 
with this Directive or an authorisation has been granted in accordan ce with Regulation (EC) 
No. 726/2004, read in conjunction with Regulation (EC) No […/of the European Parliament and of the Council (Paed iatric Regulation)*]. 
___________________ 
* OJ L […], […], p. […].” 
Article 54 
Regulation (EC) No.726/2004 is amended as follows: 
(1) Article 56(1) is replaced by the following: : 
“1. The Agency shall comprise: 
(a) the Committee for Medicinal Products for Human Use, which shall be 
responsible for preparing the opini on of the Agency on any question 
relating to the evalua tion of medicinal produc ts for human use; 
(b) the Committee for Medicinal Products for Veterinary Use, which shall be 
responsible for preparing the opini on of the Agency on any question 
relating to the evaluati on of medicinal products for veterinary use; 
(c) the Committee on Orphan Medicinal Products; (d) the Committee on Herbal Medicinal Products; (e) the Paediatric Committee; (f) a Secretariat, which shall provide te chnical, scientific and administrative 
support for the committees and ensure  appropriate coordination between 
them; 
(g) an Executive Director, who shall exercise the re sponsibilities set out in 
Article 64; 
(h) a Management Board, which shall ex ercise the responsibilities set out in 
Articles 65, 66 and 67.” 
 
EN 37   EN (2) The following Article 73a is inserted: 
“Article 73a 
Decisions taken by the Agency under Regula tion (EC) No […/… of the European 
Parliament and of the Council (Paediatric Re gulation)*] may form the subject of an 
action before the Court of Justice of th e European Communities under the conditions 
laid down in Article  230 of the Treaty. 
___________________ 
* OJ L […], […], p. […].” 
CHAPTER 3 
FINAL PROVISIONS  
Article 55 
The requirement laid down in Article 8(1) shal l not apply to valid app lications pending at the 
time of entry into force of this Regulation. 
Article 56 
1. This Regulation shall enter into force on the thirtieth day following that of its 
publication in the Official Journal of the European Union . 
2. Article 8 shall apply from … [18 m onths following the entry into force]. 
Article 9 shall apply from … [24 mont hs following the entry into force]. 
Articles 31 and 32 shall apply from … [6 months following the entry into force]. 
This Regulation shall be binding in  its entirety and directly applicable in all Member States. 
Done at Brussels, […] 
For the European Parliament For the Council 
The President The President […] […] 
 
EN 38   EN LEGISLATIVE FINANCIAL STATEMENT  
Policy area(s): Internal market  
Activities: The activities of the European Medicines Agen cy are included in the following policies: 
- Support for the development of paediatric medicines ; 
- Improvement in the protection of public hea lth and for consumers across the Community 
- Maintaining a reliable and independent source of scientific advice and information, and 
- Support and achievement of the internal market for the pharmaceutical sector. 
 
TITLE OF ACTION : 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 
 
1. BUDGET  LINE(S)  + HEADING(S) 
02.040201 – European Agency for the Evaluati on of Medicinal Products — Subsidy under 
Titles 1 and 2 
02.040202 – European Agency for the Evaluati on of Medicinal Products — Subsidy under 
Title 3 
2. OVERALL  FIGURES   
2.1. Total allocation for action (Part B): € million for commitment 
EUR 21 282 million 
2.2. Period of application: 
2007 to 2012 
2.3. Overall multiannual esti mate of expenditure: 
(a) Schedule of commitment appropriati ons/payment appropria tions (financial 
intervention) (see point 6.1.1) 
 EUR million ( to three decimal places) 
 
  
Year 
2007  
2008  
2009  
2010  
2011 2012 
and 
subs. 
Years  
Total 
Commitments 1,000 3,039 3,377 3,598 5,053 5,215 21,282  
Payments 1,000 3,039 3,377 3,598 5,053 5,215 21,282 
 
EN 39   EN (b) Technical and administrative a ssistance and support expenditure (see point 6.1.2)  
C o m m i t m e n t s         
P a y m e n t s         
 
Subtotal a+b        
Commitments 1,000 3,039 3,377 3,598 5,053 5,215 21,282  
Payments 1,000 3,039 3,377 3,598 5,053 5,215 21,282 
(c) Overall financial impact of human res ources and other admini strative expenditure 
(see points 7.2 and 7.3) 
Commitments/ 
payments        
 
TOTAL a+b+c        
Commitments 1,000 3,039 3,377 3,598 5,053 5,215 21,282  
Payments 1,000 3,039 3,377 3,598 5,053 5,215 21,282 
2.4. Compatibility with financial progr amming and financial perspective 
[X] Proposal will entail reprogramming of the relevant heading in the financial 
perspective. The proposal is compatib le with the new financial framework 
(2007-2013) proposed by the Commi ssion (Communication from the 
Commission to the Council and the Eu ropean Parliament COM(2004) 101). 
2.5. Financial impact on revenue1: 
[X] Proposal has no financial implications  (involves technical aspects regarding 
implementation of a measure) 
3. BUDGET  CHARACTERISTICS 
Type of expenditure Budget 
line New EFTA 
contrib
ution Contributions 
from 
applicant 
countries Heading in 
financial 
perspective
Non-comp Non-diff 02.040201 NO YES NO 1 a  
Non-comp Non-diff 02.040202 NO YES NO 1 a  
                                                 
1 For further information, see separate explanatory note. 
 
EN 40   EN 4. LEGAL  BASIS   
– Treaty establishing the European Community and notably article 235. 
– Draft Regulation of the Europ ean Parliament and of the Council on medicinal products for 
paediatric use (to support the Agency’s wo rk required for the operation of the draft 
Regulation including all work of  the Paediatric Committee, scientific advice and any fee 
waivers provided for by virtue of the draft Regulation). 
– Regulation (EC) No 726/2004 of the European parliament and of th e Council of 30 May 
2004, establishing the community  procedures for the aut horisation and follow up of 
medicines for human and veterinary use, and establishing the European Agency for the 
Evaluation of Medicinal Products (OJ L 136, 30.4.2004, p. 1). 
– Council Regulation (EC) No 297/95 of 10 February 1995 modified by Council 
Regulation (EC) No 2743/98 of 14 December 1998 concerning fees payable to the European Agency for the Evaluation of Medicinal Products (OJ L 345, 19.12.1998, p. 3). 
5. DESCRIPTION
 AND  GROUNDS 
5.1. Need for Community intervention  
5.1.1. Objectives pursued It is estimated that between 50 and 90% of medicinal products used in the paediatric 
population have never been specifi cally studied or authorised (l icensed) for use in that age 
group. This leaves no alternative to  the prescriber than to use pr oducts “off-label” (i.e. use of 
product authorised for adu lts - products that have  not been tested or au thorised for paediatric 
use) or use of completely unaut horised products with the associat ed risks of inefficacy and/or 
adverse reactions (side effects). 
The overall policy objective is to improve the he alth of the children of  Europe by increasing 
the research, development and authorisa tion of medicines for use in children. 
General objectives are to: 
• increase the development of me dicines for use in children; 
• ensure that medicines used to treat child ren are subject to hi gh quality research; 
• ensure that medicines used to treat childr en are appropriately authorised for use in 
children; 
• improve the information available on the use of medicines in children; 
• achieve these objectives without  subjecting children to unnecessa ry clinical trials and in 
full compliance with the EU Clinical Trials Directive. 
 
EN 41   EN 5.1.2. Measures taken in connection with ex ante  evaluation  
The draft Regulation of the Eur opean Parliament and of the C ouncil on medicinal products for 
paediatric use was the subject of a Commissi on Extended Impact Assessment (EIA). The EIA 
accompanies this Financial Statement. The Commission’s EIA is based on an independent, 
externally contracted study, specifically desi gned to estimate the economic, social and 
environmental impacts of the proposal. The EI A also draws on experience with the existing 
EU pharmaceutical market and regulatory fr amework, experience w ith legislation on 
paediatric medicines in the US, experience with orphan medicines in the EU, extensive 
consultation with stakeholders, and the published literature.  
5.1.3. Measures taken following ex post  evaluation 
The draft Regulation of the Eur opean Parliament and of the C ouncil on medicinal products for 
paediatric use is a new legislative proposal an d no interim or ex post evaluation has been 
conducted. 
5.2. Action envisaged and budget intervention arrangements 
The key measures included in the draft paediatric regulation are:  
• the establishment of an expert committee, the Paediatric Committee within the EMEA;  
• a requirement at the time of marketing author isation applications for new medicines and 
line-extensions for existing patent-protected me dicines for data on the use of the medicine 
in children resulting from an agr eed paediatric investigation plan;  
• a system of waivers from the requirement fo r medicines unlikely to benefit children;  
• a system of deferrals of the requirement to ensure medicines are te sted in children only 
when it is safe to do so and to prevent th e requirements delaying the authorisation of 
medicines for adults;  
• excluding orphan medicines, a mixed reward and incentive for compliance with the 
requirement in the form of a six-month extension to the supplementary protection 
certificate (in effect, a six-month patent  extension on the active moiety);  
• for orphan medicines, a mixed reward and in centive for compliance with the requirement 
in the form of an additional tw o-years of market exclusivity added to the existing ten years 
awarded under the EU orphan regulation; 
• a new type of marketing authorisation, th e PUMA, which allows ten years of data 
protection for innovation (new studies) on off-patent products;  
• amended data requirements for PUMA applic ations to attract SMEs including generics 
companies; 
• a reference in the explanatory memorandum to the establishment, via separate legislation 
of an EU paediatric study programme to f und research leading to the development and 
authorisation of off-patent  medicine for children;  
 
EN 42   EN • access to an optional centralised procedure via the community referral procedure for 
existing nationally authorised medicines to gain an EU-wide Commission Decision on use 
in children;  
• measures to increase the robustness of pha rmacovigilance for medicines for children;  
• a requirement for industry to s ubmit to the authorities study reports they already hold on 
use of their medicine in children, to maximise  the utility of existing data and knowledge;  
• an EU inventory of the therapeutic needs of  children to focus research, development and 
authorisation of medicines;  
• an EU network of investigator s and trial centres to conduct the research and development 
required;  
• a system of free scientific advice for the industry, provide d by the EMEA;  
• a database of paediatric studies (based on th e existing database set up by the EU Directive 
on clinical trials (OJ L 121, 1.5.2001, p. 34). 
Populations affected by the activity: 
• more than 100 million children in the newly en larged EU stand to benefit from better 
medicines for children. Children will al so be enrolled into clinical trials; 
• healthcare professionals will benefit throug h the supply of medicines specifically 
developed for children and may take part in clinical research on medicines for children; 
• all pharmaceutical companies seeking to access th e EU market will be affected by the draft 
Regulation; 
• the EMEA and all National competent author ities will have to change their working 
practices as a result of the draft Regulation; 
Expense type 
Article  47 of the draft Regulation on medicinal pr oducts for paediatric use foresees a 
contribution from the Community to cover the work resulting from the draft Regulation on 
medicinal products for paediatric use, incorp orated into the contribution provided for in 
Article 67 of Regulation (EC) No 726/2004 and in  Article 7 of Regulation (EC) No 141/2000 
to be allocated to the European Medicines Agen cy. This contribution should cover all aspects 
of the work of the European Medicines Ag ency to implement and operate the draft 
Regulation, in particular: the operation of the Paediatric Co mmittee including a ssessment of 
paediatric investigation plans, requests for waivers and deferrals, a ssessment of compliance 
with paediatric investigation plans and assessment of the safety, quality and efficacy of medicinal products for paediatric use; an EU i nventory of the therapeutic needs of children; 
an EU network of investigators and trial centres  to conduct the research ; free scientific advice 
for the industry; a database  of paediatric studies. 
The explanatory memorandum of the draft paed iatric regulation makes a reference to the 
possible creation of a paediatric study program me: Medicines Investigation for the Children 
 
EN 43   EN of Europe (MICE)2. The creation of the funding and its operation would be included in a 
separate Commission initiative. A detailed assess ment of the impacts of  the programme will 
accompany that separate initiative. However, given the interface between legislation on a 
paediatric study programme and the draft p aediatric Regulation assessed here, some 
consideration is required. An EU paediatric study programme , focussed on funding or part 
funding studies on off-patent medicines will be important if research and authorisation for 
children of off-patent products are to occur for the majority of products needed by children. It 
is envisaged that the paediatr ic study programme may be funde d, at least in part, from the 
Community budget. The paediatric study programme would also need to take account of other 
relevant Community funding, including the 6th and 7th Framework Programmes operated by 
the Commission Directorate Gene ral Research. Community funding for studies into off-patent 
medicines for children (which may lead to the authorisation of an off-patent medicine for 
children) may only be partial,  e.g. 50% funding: the remainde r of the funding may need to 
come from industry, Member State g overnments or medical charities.  
An EU paediatric study programme has the potentia l to stimulate research  and development of 
off-patent medicines for children and coul d have a major beneficial impact on EU 
pharmaceutical companies, including SMEs, and a major impact on clinical trials conducted 
in the EU including strengthening pharmaceutical R&D in Europe. 
Estimated resources and costs of the paediatric  Regulation, based on the draft proposal 
released for consultation by the European Commission on 8 March 2004 
The increased contribution will cover: incr eased administration co sts of the European 
Medicines Agency relating to all tasks of the Paediatric Committee; the costs of free scientific advice and fee reductions for paediatr ic use marketing authorisations. 
Justifications of the reso urces implications based on its coming into force in 2007 
As of 2006, the EMEA would have to set up a task force to prepare for the work of the 
Paediatric Committee and the procedures as laid out in the Regulation. It is estimated that the 
task force would require 1 A grade full time and 1 C grade half time. This will be covered by an internal redeployment. 
In 2007 
Activities planned for the first year. All activ ities are based on the EMEA’s experience of 
Committee activities, and in particular the experien ce gained in the last 3 years of activities on 
orphan medicinal products and the Committee for orphan medicinal products. Activities will 
start in full as soon as the Regulation is implem ented due to the legal ob ligations created by it. 
                                                
 
2 The impact of the referenced paediatric study programme will critically depend on its funding, size and 
awarding rules. A fund, set up un der the United States Best Pharm aceuticals for Children Act 2002, is 
of $ 200 000 000 for fiscal year 2000 and such su ms as are necessary for each of the succeeding five 
years for the study of the use in the paediatric po pulation of medicinal products for which there is no 
patent protection or market exclusivity. The CHMP Paediatric Expert Group has produced a 
preliminary list of sixty-five off-patent active substa nces considered to be priorities for research and 
development for children in the EU. 
 
EN 44   EN A. Paediatric Committee  
Functioning of the Paediatric Committee  
• Meeting costs 
A monthly meeting of 2-3 days is necessary. Eleven meetings a year with 31 members are 
envisaged, representing 682-1023 expert days. In addition, it is anticipated that additional 
experts will be needed on an ad-hoc  basis by the Paediatric Committee.  
• Meeting Management and Conference services  
Eleven meetings a year of 31 members plus additional experts will have heavy implications 
on the Meeting Management & Conferences Sector  of the EMEA which will have to organise 
travel and accommodation and meetings, as  well as on the meeting room occupation.  
• Secretariat costs 
The secretariat of the Paediatric Committee re presents a full time position all year round, 
therefore taking into considera tion the need for a back-up, this  represents 1.5 A grade and 
1.5 C grade positions. 
• Expert costs 
Estimated at 5-10 experts per Paediatric Committ ee meeting, in addition to members of the 
Paediatric Committee (i.e. 55-110 experts per year). 
Activities of the Paediatric Committee  
– Paediatric Investigation Plans 
– Deferrals 
– Waiver of Paediatric Investigation Plans  
– Paediatric needs 
– Paediatric priorities  
– Compliance  
– Expert work 
In the draft paediatric Regulation, there is an  obligation to submit the results of studies 
performed according to an agreed Paediatric In vestigation Plan for applications for marketing 
authorisations of new products  (Marketing Authorisation App lications) and variations for 
patented products. The best estimate of the numb er of Paediatric Inve stigation Plans to be 
submitted per year to the Agency in the first years is about 235-285.  
The activities related to the submission of Paediatric Investigation Plans are rather similar to 
the work done for orphan drug designation. Howeve r the level of scientific involvement to 
 
EN 45   EN judge the submitted plan is considered higher, more complex, and the number of procedures is 
2.5 times more than the current number of orphan applications. 
– Agreed Paediatric Inves tigation Plans revisions 
– Procedures 
It is not expected that applications for the revision of Paediatric Investigation Plans would 
occur in the first year. Only proced ures would have to be established. 
B. Other activities created by the Regulation 
– Paediatric scientific advice 
There will be an increase in scientific advice for paediatric development. It is expected that up 
to 60% of companies may seek advice (the current situation is about 30% for products 
submitted for Marketing Authorisation). This repr esents about three times the current number 
of Scientific Advice requests (currently 100 pe r year). See section 6.2 for details of the 
financial implications of fee waivers for paediatric scientific advice. 
– Information publication and management 
This has implications on the current developm ent of the databases at the EMEA and on other 
forms of EMEA communication.  
– Survey of paediatric use and inventory of research priorities 
These activities will be performed by the staff in  charge of other paediatric activities but will 
represent a significant pa rt of the workload. 
– Establishment of a paediatric research network 
This is a new type of activity for the EMEA, which will require at least a full time position for 
an A and a C grade. 
C. Impact on the Agency 
In addition to involving specific staff all activit ies have direct implications on other sectors 
such as Meeting Management and Conference, IT and administration. 
The activities will generate the need for regular training, workshops and will involve missions 
outside the Agency (for example for the estab lishment of a network of paediatric clinical 
research). 
D. Need for Experts in Secondment 
To strengthen the collaboration between EMEA a nd Member States in part icular in relation to 
paediatric activities on national products, auth orisations and pharmac ovigilance, the EMEA 
will invite Experts in Secondment to join the Ag ency to facilitate the work. This will be done 
also at the stage of the preparatory work. 
 
EN 46   EN A typical stabilised year  
It has been considered that year 2009 would represent a typi cal year, when the number of 
applications per year would be stable, and a ll activities provided fo r by the Regulation would 
be developed. 
A. Paediatric Committee 
Functioning of the Paediatric Committee 
– Meeting costs 
No major changes in acti vities are anticipated. 
– Meeting Management and Conference services  
No major changes in acti vities are anticipated. 
– Secretariat costs 
No major changes in acti vities are anticipated. 
– Experts costs 
Changes in activities may be needed. Estimates  are however given for the same numbers. 
Activities of the Paediatric Committee  
Figures for new products (on patent) should remain  stable. Variations capturing products that 
never included a Paediatric Investigation Plan  should slightly decreas e, as some products 
would have been captured at the stage of mark eting authorisation applications. This would 
however not be the case of variation applicatio ns in a new indication (new therapeutic area) 
for which a new Paediatric Investigation Plan may have to be submitted. 
There should not be any more products undergoing purely nationa l procedures in respect of 
the obligation to submit a Paed iatric Investigation Plan. 
The ‘stable’ number of Paediatric Use Mark eting Authorisation pr ocedures cannot be 
estimated. It is judged that  the initial figure of 15 pe r year should be kept.  
Overall the level of activ ities should remain around 235-285 procedures per year.  
The additional (fully developed) tasks will include in particular the Annual Reports on 
deferrals, and the revision of agreed Paediatr ic Investigation Plans. Once a Paediatric 
Investigation Plan is agreed, th e draft Regulation offers the possi bility to amend it as often as 
needed on request from the sponsor. It is estimat ed that 30% of the P aediatric Investigation 
Plans may need revision at some point in time. This may represent a minimum of 80 
additional applications a year. 
 
EN 47   EN B. Activities created by the Regulation 
– Scientific Advice 
Paediatric Scientific Advice and follow up pr ocedures would increase progressively over 
time. 
– Pharmacovigilance and risk management 
This activity will be fully developed. 
– Information publication and management 
Modifications or developments of the current st ructures will take place over several years.  
– Inventory of research priorities 
Regular updates are forecas ted for in the Regulation. 
– Establishment of a paediatric research network 
The implementation and running of the network should be in place.  
C. Impact on the Agency  
In addition to involving specific staff all activ ities and their related increases have direct 
implications on other sectors. 
5.3. Methods of implementation 
The draft Regulation will be implemented and operated primarily by th e existing European 
Medicines Agency. Certain aspects will also be operated by the National Competent Authorities. The Commission will be responsible for an implementing regulation and a number of supporting guidelines. 
 
EN 48   EN 6. FINANCIAL  IMPACT 
6.1. Total financial impact on Part B - (over the entire pr ogramming period) 
(The method of calculating the total amounts set out in the table below must be explained by 
the breakdown in Table 6.2. ) 
6.1.1. Financial intervention 
Commitments (in EUR million to three decimal places) 
Breakdown 2007  2008  2009  2010  2011 2012 
and 
subs. 
Years  Total 
02.040201 – European 
Agency for the Evaluation of 
Medicinal Products — 
Subsidy under Titles 1 and 2 
 
02.040202 – European 
Agency for the Evaluation of 
Medicinal Products — 
Subsidy under Title 3 
  
 
0,800 
   
0,200 2,397
0,6422,688
0,6892,881
0,717 
 
4,280 
   
0,773 4,409
0,80617,455
3,827
A c t i o n  2         
e t c .         
TOTAL 1,000 3,039 3,377 3,598 5,053 5,215 21,282
 
EN 49   EN 6.1.2. Technical and administrative assistance,  support expenditure  and IT expenditure 
(commitment appropriations) 
 2007  2008  2009  2010  2011 2012 
and 
subs. 
Years  Total 
1) Technical and 
administrative assistance N.A.       
a) Technical assistance 
offices       
b) Other technical and 
administrative assistance: 
- intra muros: 
- extra muros: of which for construction 
and maintenance of 
computerised management systems        
Subtotal 1        
2) Support expenditure        
a) Studies        
b) Meetings of experts        
c) Information and 
publications        
Subtotal 2        
TOTAL         
 
EN 50   EN 6.2. Calculation of costs by measure en visaged in Part B (over the entire 
programming period)3 
(Where there is more than one action, give suffici ent detail of the specific measures to be 
taken for each one to allow the volume and costs of the outputs to be estimated.) 
Commitments (in EUR million to three decimal places) 
Breakdown  Type  
of outputs 
(projects, files ) Number of 
outputs 
(total for years 
2007-2012)  Average unit 
cost Total cost 
(total for years 
2007-2012)  
 
 1 2 3 4=(2X3) 
Paediatric medicines management  
- Measure 1 
 
  
  
Paediatric 
activities costs 
for the EMEA general subsidy 
Staff 
Expenditure 
other. 
  
 
 
  
 
  
 
 
  
  
 
 
  
17,455 
3,827 
 
TOTAL COST    21,282 
These costs are mainly due to: 1. the supplementa ry staff needed to perform the tasks induced 
by the new regulation on medicinal products for p aediatric use, 2. scientific advice being 
given without a fee, and, 3. fee reductions for marketing authoris ation applications.  
Staff will be required to: provide the secretariat of the new expert committee the Paediatric 
Committee, administer requests for opinions from the Paediatric Committee, create and 
maintain an inventory of the therapeutic needs of the children of Europe, create and maintain an EU network of clinical trial centres to  conduct tests of medi cines for children, and, 
collation and publica tion of information about medicine s for children. Projections for 2011 
foresee that 24 people (14,5 A and 9,5 C) will be necessary to support the EMEA work related 
to the paediatric regulation. Support st aff will bring the overall figure to 26. 
Regarding scientific advice, currently, reque sts for such advice command a fee from the 
EMEA. This fee is used mainly to pay expert s from the National agencies who conduct the 
scientific evaluation of the requests (with thei r accompanying dossiers). The draft paediatric 
regulation will lead to such scientific advi ce being given without the payment of fees. 
Therefore the EMEA will have to pay money to th e National agencies and this will have to be 
                                                
 
3 For further information, see separate explanatory note. 
 
EN 51   EN covered. Furthermore, the total number of reque sts for scientific advice is predicted to 
increase dramatically as a result of the p aediatric regulation. The current average fee for 
scientific advice is abou t 40 000 € and it is predicted that, Fo r the period of six years starting 
in 2007, about 330 free pieces of scie ntific advice will be given.  
Regarding fee reductions for marketing author isation applications, the current fee is 
approximately 200 000 €. This pays mainly for th e scientific evaluation conducted by experts 
from the National agencies. The fee reduction fore seen in the paediatric regulation is 50% and 
this will apply to a small proportion of all paed iatric marketing authorisations (the so called 
Paediatric Use Marketing Authorisations – PU MAs). For the period of six-years starting in 
2007 it is estimated that about 30 paediatric use marketing authorisation applications will be 
made that will attract the 50% fee reduction. Hence the EMEA will have to pay the National 
agencies but this will not be covered by adequate fees. 
Staff requirement  2007  2008  2009  2010  2011 2012 
and 
subs. 
Years  
Secretariat Paediatric 
Committee Paediatric 
Investigation Plan 
applications 
Paediatric Research Network 
Funding of studies 
Support staff  1 
 
1 
  
1 
 3
10
1
23
10
1123
10
1223 
 
14 
3 2 4 
 3
14
324
TOTAL 3 16 17 18 26 26
Expenditure costs will mostly cover the reimburs ement of the experts in relation with the new 
committee ‘Paediatric Committee’, as well as  other missions and trainings. Some IT 
developments will also be necessary in order to include this new category of medicinal products in the several existing databases. 
 
EN 52   EN Expenditure Other  2007  2008  2009  2010  2011 2012 
and 
subs. 
Years  
Meetings Paediatric 
Committee  
31 members and 5 experts 
11 x 2-day meetings 
Workshops, trainings and 
missions 
IT development and web 
publication 
  
 
0,050 
 
0,100 
 
0,050 
 0,413
0,119
0,1100,452
0,127
0,1100,474
0,133
0,110 
 
0,498 
 
0,165 
 
0,110 
 0,523
0,173
0,110
TOTAL 0,200 0,642 0,689 0,717 0,773 0,806
7 IMPACT  ON  STAFF  AND  ADMINISTRATIVE  EXPENDITURE   
7.1. Impact on human resources 
Staff to be assigned to management of the 
action using existing resources Description of tasks deriving from the 
action 
Types of post 
Number of 
permanent posts Number of 
temporary posts Total 
 
Officials or 
temporary staff A 
B 
C N.A.  
   
If necessary, a fuller description of the 
tasks may be annexed. 
Other human resources     
Total     
 
EN 53   EN 7.2. Overall financial impact of human resources 
Type of human resources Amount (€) Method of calculation *  
Officials 
Temporary staff N.A.  
Other human resources 
(specify budget line)   
Total   
The amounts are total expend iture for twelve months. 
7.3. Other administrative expenditu re deriving from the action 
Budget line 
(number and heading) Amount € Method of calculation 
Overall allocation (Title A7) 
ex A0701 – Missions 
ex A07030 – Meetings 
ex A07031 – Compulsory committees 1 
Paediatric Committee  
A07032 – Non-compulsory committees 1 
A07040 – Conferences ex A0705 – Studies and consultations 
Other expenditure (specify) 
Training  
N.A.  
Information systems (A-5001/A-4300)    
Other expenditure - Part A (specify) 
IT developments  
  
Total   
The amounts are total expend iture for twelve months. 
1 Specify the type of committee and the group to which it belongs. 
 
EN 54   EN I. Annual total (7.2 + 7.3) in 2011 
II. Duration of action III. Total cost of action (2007 to 2012)  
The needs for human and administrative resour ces shall be covered within the allocation 
granted to the managing DG in the framew ork of the annual al location procedure 
8. FOLLOW-UP  AND  EVALUATION 
8.1. Follow-up arrangements 
Many of the effects of the draft paediatric legi slation lend themselves to measurement. Others, 
including the overall objective of  improved child health will be more difficult to measure due 
to a lack of robust EU-wide data. Collection of the following data is possible. 
• The dates on which the Paediatric Committee and EU network of clinical trialists are 
established and guidelines and first inve ntory of therapeuti c needs are adopted. 
• The date on which the database of pa ediatric studies becomes operational. 
• The number of clinical trials in children initiated and completed (broken down by country 
and type of trial). 
• The number of children enrolled into clinical trials. 
• The number of draft paediatric investig ation plans submitted for assessment and the 
number of paediatric investigation plan s agreed by the Paediatric Committee. 
• The number of requests for waivers and the nu mber of waivers granted by the Paediatric 
Committee. 
• The number of requests for deferrals and the nu mber of deferrals granted by the Paediatric 
Committee. 
• The number of requests for scientific advice. 
• The numbers of marketing authorisation appl ications made and granted for adults and 
children. 
• The number of PUMA applications made and PUMAs (with their associated data 
protection) granted. 
• The number of requests for post-marketing st udies, pharmacovigilance plans and risk 
management systems and the delivery against those plans. 
• The number of existing studies in childr en submitted and the number of marketing 
authorisations updated as a result. 
 
EN 55   EN • The number of times marketing authorisations record that a paediatric investigation plan 
has been complied with. This provides a measure of the number of supplementary 
protection certificates that can be extended. 
• Impact on the budget of the EMEA. 
These data would provide a robust measure of the impact of the draft pa ediatric regulation in 
terms of stimulating research, development a nd authorisation of medicines for children and 
any collateral effect on the aut horisation of medicines for othe r populations. They would also 
provide a measure of the fi nancial impacts on the EMEA. 
Prospective measurement of the costs to industry and on the price of medicines is not 
proposed as such measurement lends itself better to a post-hoc study.  
Section 4 of the extended impact assessment poi nts out that the impact, both financial and 
social, of improved health of the children of Eu rope is very difficult to measure. Unless there 
is major investment in the central collection of indices of EU child health, this difficulty will remain when attempting to measure, in the future, the impact of the draft paediatric Regulation. 
8.2. Arrangements and schedule for the planned evaluation 
The draft paediatric regulation in cludes proposals for: a database of paediatric studies; annual 
reports from the Member States to the Commission on problems encountered with the 
implementation of the draft paediatric regulation;  annual publication of lis ts of companies that 
have benefits from the rewards / incentives or companies that have failed to comply with the 
obligations, and; within six year s of entry into force, a genera l report on experienced acquired 
as a result of the applicati on of the draft paediatric Regu lation, including in particular a 
detailed inventory of all medici nal products authorised for paedia tric use since it came into 
force. 
Through these measures, specif ically proposed in the draf t paediatric Regulation, ex post  
evaluation is already planned. The general report will likely be based on the indices listed in section 8.1. Furthermore, the need for a design ated independent study to support the general 
report should be considered. Such an independ ent study could include within its scope the 
financial and social impacts for which pr ospective data collection is problematic. 
9. ANTI-FRAUD
 MEASURES 
The European Medicines Agency has specific bu dgetary control mechanisms and procedures. 
The Management Board, which comprises repr esentatives of the Member States, the 
Commission and the European Parliament, adop ts the draft budget (Article 57.5) as well as 
the final budget (Article 57.6). The European Cour t of Auditors examines the execution of the 
budget each year (Article 57.9) and the Management  Board gives a discharge to the Director 
regarding the budget (A rticle 57.10). In addition the Agency adopted on 1 June 1999 a 
decision concerning co-operation with the Eu ropean Anti-Fraud Office (EMEA/D/15007/99). 
The Quality Management System applied by the Agency supports a continuous review with 
the intention of ensuring that the correct proc edures are followed and that these procedures 
and policies are pertinent and efficient. Several internal audits are undertaken each year as 
part of this process.