Rev. PRIORITY LIST 2010

8/7/2019 Rev. PRIORITY LIST 2010

1/8

7 Westferry Circus Canary Wharf London E14 4HB United Kingdom

Telephone +44 (0)20 7418 8400 Facsimile +44 (0)20 7523 7040

E-mail [email protected] Website www.ema.europa.eu An agency of the European Union

European Medicines Agency, 2010. Reproduction is authorised provided the source is acknowledged.

26 July 2010*EMA/480197/2010Human Medicines Development and Evaluation

Revised priority list for studies into off-patent paediatricmedicinal productsfor the 5th call 2011 of the 7th Framework Programme of the European

Commission

This priority list of off patent medicines is the basis only for the 5th Call 2011 of the 7th

Framework Programme of the European Commission.

The 6th call 2012 will be based on a separate priority list. Please refer to the website of the

European Medicines Agency.

Objective of the list

The priority list provides the basis for the 5th Call 2011 of the 7th Framework Programme of the

European Commission. It ensures that funds are directed into research of medicinal products with the

highest needs in the paediatric population.

The aim ofRegulation (EC) No1901/2006 of the European Parliament and the Council on Medicinal

Products for Paediatric Use is to increase availability of medicines authorised for children as well as to

increase the information available on the use of medicinal products in the paediatric population. The

Regulation includes provisions for funding of studies into off-patent medicinal products. This funding,

currently provided through the EU Framework Programme, should cover the development of off-patent

medicinal products with a view to the submission of a Paediatric Use Marketing Authorisation (PUMA;

Art. 30, Regulation [EC] No 1901/2006).

Agreement on the specific content of a PUMA application will eventually be through a PaediatricInvestigation Plan (PIP).

Notes

The following are always considered to be of high priority: Development of age-appropriate formulations and strengths (even if not explicitly stated for a

product);

Data in neonates for all conditions (except oncology);*This document was revised on this date. Please refer to the table of amendments on the last page.
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000092.jsp&mid=WC0b01ac05800260a4&murl=menus/regulations/regulations.jsp&jsenabled=truehttp://cordis.europa.eu/fp7/find-doc_en.html#workprogrammeshttp://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2006:378:0001:0019:en:PDFhttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000413.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025ea2http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000413.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025ea2http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2006:378:0001:0019:en:PDFhttp://cordis.europa.eu/fp7/find-doc_en.html#workprogrammeshttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000092.jsp&mid=WC0b01ac05800260a4&murl=menus/regulations/regulations.jsp&jsenabled=true


2/8

Prioritised needs, as listed in the column Priority, do not necessarily cover all needs for thetreatment of the paediatric population, nor will they automatically cover the regulatory

requirements for a Paediatric Investigation Plan (PIP). In other words, whereas not all conditions

mentioned for an active substance have to be investigated, all paediatric subsets must be covered

either by a PIP or a waiver.

Further information regarding the PIP can be obtained: as a first step from the website of the European Medicines Agency (EMA) through a pre-submission teleconference (Q&A Nr. 24.) with the EMAIt should be noted that the PIP is a 120-day procedure with a clock-stop phase, during which the

applicant can amend the PIP according to the requests and proposals of the PDCO.

Disclaimer

The list includes only products considered to be off-patent, i.e. not covered by a basic patent or a

supplementary protection certificate. Information on the authorisation status as well as on available

paediatric formulations of medicinal products is limited and not available for all European Member

States. Information on the off-patent and authorisation status is not guaranteed by the EMA. Users of

this list are therefore advised to check the patent status and the authorisation status of the medicinal

products of interest.

The methodology used to establish the list was based as much as possible on evidence-based medicine

(see p.12). It is however acknowledged that identification of priorities for research into medicinal

products for paediatric use is partly based on subjective criteria and that identified priorities may

change over time. This may also be the case should further information of which the Paediatric

Committee is not aware, become available (e.g. on pharmacokinetics, safety and efficacy, submission

of PIPs for prioritised products, etc.). Projects already funded, such as for the development of age-

appropriate formulations, should not be re-submitted. Please check the website of the European

Commission for already funded projects.

This revision was adopted by PDCO on 16/07/2010.

Revised priority list for studies into off-patent paediatric medicinal products

EMA/480197/2010 Page 2/8
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/q_and_a/q_and_a_detail_000015.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b8ehttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/q_and_a/q_and_a_detail_000015.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b8ehttp://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580025b91


3/8

Abbreviations

CF Cystic fibrosisDM II Diabetes mellitus Type IIHSCT Haematopoietic stem cell transplantation

JIA Juvenile idiopathic arthritisPK PharmacokineticsPUMA Paediatric use marketing authorisationTNF Tumour necrosis factor

Therapeutic field Product Condition(s) Specific needs

Cardiology

(refer also to 'nephrology')

amiodarone Supraventricular andventricular arrhythmia

Data on long-termsafety.

dobutamine Shock, cardiac failure Data on efficacy inneonates.*

milrinone Cardiac failure Data onpharmacokinetics (PK),efficacy and safety.*

propranolol Hypertension,supraventriculartachycardia

Data on PK, efficacy andsafety.

* Please note that there is a need for international consensus on the definition of 'shock' inneonates, and any medicine development should take this into consideration.

Child & adolescentpsychiatry

fluoxetine Major depressivedisorder with

psychotic symptoms,General anxietydisorder,obsessive compulsivedisorder

Data on short and longterm-safety.

Data on short and longterm-safety and efficacy.

Dermatology

(refer to immunology)

Endocrinology

androstanolone gel Micropenis/severehypospadias


cholestyramine Hypercholesterolaemi

a

Data on efficacy and

safety in children from 6years. Palatableformulation.

glibenclamide Diabetes mellitus typeII

Data on PK, efficacy andsafety in children from10 years.

hydrocortisone Adrenal insufficiency Age-appropriate oralformulation; age group0-2 years.


EMA/480197/2010 Page 3/8


4/8


EMA/480197/2010 Page 4/8


metformin

Impaired glucosetolerance in obese

children forprevention of type IIdiabetesSmall-for-gestational-age children withprecocious/early/rapidly progressingpubertyPolycystic OvarySyndrome

Data on PK, efficacy andsafety in children from 6years.Data on PK, efficacy andsafety.Data on PK, efficacy andsafety.

carbimazole Hyperthyroidism Data on safety.

Gastroenterology

(refer also to immunology)

bisacodyl Constipation Data on long-term

efficacy, safety, all agegroups; age-appropriateformulation.

macrogol Constipation Data on long-termefficacy, safety, all agegroups.

Haematology

unfractionatedheparin

Anticoagulation Data on PK, efficacy andsafety.

Immunology

(refer also to oncology, gastroenterology and rheumatology)

azathioprine Chronic rejection in

transplantationCrohns diseaseSevere atopicdermatitis

Data on efficacy in

infants. Age appropriateoral formulation.Data on efficacy and

safety in combinationwith biologicals such asanti tumour necrosisfactor (TNF); age-appropriate formulation.Data on efficacy andsafety.

etopophos

Before allogenic and

autologoushematopoietic stemcell transplantation(HSCT) for variousconditions.

Data on PK, short and

long term safety in allpaediatric age groups.

fludarabine Before allogenic HSCTfor various conditions.

Data on PK, short- andlong-term safety; in allpaediatric age groups.

melphalan

Before allogenic andautologous HSCT forvarious conditions.

Data on PK, efficacy,short- and long-termsafety; in all paediatricage groups.


5/8


EMA/480197/2010 Page 5/8


methotrexate Crohns disease

Juvenile idiopathicarthritis (JIA)Juveniledermatomyositis,childhoodscleroderma, JIA-related uveitis

Data on efficacy andsafety [includingcombination withbiologicals such as anti-TNF].Data on long-term safetyData on PK, efficacy andsafety.

mycophenolatemofetil

Short and long termimmunosuppressionfor prevention of graftrejection and graft

versus host diseaseafter allogenic HSCTSystemic LupusErythematosus (SLE)nephritisRenal, heart and livertransplantation


Data on PK, efficacy andsafety.Data on PK, efficacy andsafety in children from 0-2 years.

Infections

amphotericin B Mycotic infections Data on efficacy andsafety in immuno-compromised patients inall age groups, includingneonates and preterminfants.

clindamycin Osteomyelitis;infections caused bymethicillin resistantStaphylococcus

aureus and methicillinresistantStaphylococcus

epidermidis

Data on PK (unlessavailable) in all agegroups; relevant tissueand fluid levels; short-and long-term efficacyand safety.

ganciclovir Cytomegalovirusinfection

Data on PK, efficacy andsafety in immuno-compromised patients inall age groups, neonates,and preterm infants.

itraconazole Invasive mycoticinfections,aspergillosis, chronicgranulomatousdisease, febrileneutropenia.


Intensivecare/anaesthesiology

(refer also other fields such as cardiology, haematology, infections,neonatology and pain)


6/8


7/8


8/8


EMA/480197/2010 Page 8/8


dornase alfa Cystic fibrosis (CF)

Primary ciliarydyskinesia, non-CFbronchiectasis

Data on PK, efficacy andsafety; age group belowthe age of 5 years.Data on PK, efficacy andsafety; all paediatric agegroups.

Rheumatology

(refer to immunology)

Methodology

The original list 2003 had been prepared from a public health perspective prioritising in a first step

conditions based on factors such as severity of disease, non-availability of treatment alternatives,

affected paediatric age groups and paediatric prevalence data. In a second step for each condition

medicinal off-patent products were identified according to published therapeutic reviews.

For the revision in 2008 medicinal products were prioritised also taking into account the WHO list of

essential medicines for children, the FDA/NICHD list of products and further paediatric needs. Potential

collaboration with FDA/NICHD has been taken into consideration with a view to avoid duplication of

efforts.

The revision in 2009 took into account the projects which have been funded in the previous calls as

well as comments and proposals from learned scientific and paediatric Societies, following a wide call

for expression of interest. For the revision in 2010 funded products and products with sufficient data

were removed from the list.

Amendments

Date Page Product Amendment

26/07/2010 1-2 Correction of hyperlinks and references to FP-7-Health-Call 2011

26/07/2010 2 Clarification of procedure

26/07/2010 4 metformin Clarification of the condition and priority regarding type II diabetes

26/07/2010 5 methotrexate EMA was informed by the EC that methotrexate for JIA (Data on long-term

safety) has not been covered by any project funded by the EC. Therefore it has

been re-included on the priority list.

26/07/2010 7 cytarabine Removed from the priority list, as it is authorised for all age-groups and project

would not meet the requirements for a PUMA.

Documents

Rev. PRIORITY LIST 2010