Background Paper 7.1 Priority Medicines for Children

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  • Priority Medicines for Europe and the World

    "A Public Health Approach to Innovation"

    Update on 2004 Background Paper

    Written by MGP Zuidgeest, MJC Willemen and JN van den Anker

    Background Paper 7.1

    Priority Medicines for Children

    By Verica Ivanovska1,2, Aukje K. Mantel-Teeuwisse1, Liset Van Dijk3

    May 10, 2013

    1Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, the Netherlands

    2University Goce Delcev, Stip, Macedonia 3Netherlands Institute for Health Services Research (NIVEL), Utrecht, the Netherlands

    This Background Paper was commissioned by the WHO Collaborating Centre for Pharmaceutical

    Policy and Regulation (www.pharmaceuticalpolicy.nl). This work was financially supported by the

    Dutch Ministry of Health, Welfare and Sport.

    https://solismail.uu.nl/owa/redir.aspx?C=UC-4ThrLF0OVgcLLsgB07HKeOSNMIdAIF1CpEdVv9WTL0lwEC017_2qKrKavEipiPoYltgQ4e-U.&URL=http%3a%2f%2fwww.pharmaceuticalpolicy.nl

  • Update on 2004 Background Paper, BP 7.1 Priority Medicines for Children

    7.1-2

    Table of contents

    Executive summary and key research points ................................................................................................. 4

    1. Introduction ................................................................................................................................................. 8

    2. Demographics and diseases faced by children ..................................................................................... 8

    2.1 Childhood mortality ........................................................................................................................... 8

    2.2 Childhood morbidity ......................................................................................................................... 9 2.2.1 Asthma in children ....................................................................................................................... 10 2.2.2 Diabetes in children ..................................................................................................................... 10 2.2.3 Mental diseases in children ......................................................................................................... 11 2.2.4 Infectious diseases in children .................................................................................................... 11

    2.3 General use of medicines in children ............................................................................................. 12

    2.4 Conclusions ....................................................................................................................................... 13

    3. Product related issues in children ......................................................................................................... 13

    3.1 Paediatric age-appropriate dosing and formulations .................................................................. 13

    3.2 New oral paediatric formulations .................................................................................................. 15

    3.3 Novel paediatric drug devices ........................................................................................................ 17

    3.4 Taste and palatability of paediatric dosage forms ....................................................................... 18

    3.5 Use of excipients in paediatric dosage forms ............................................................................... 19

    3.6 Clinical evidence on impact of paediatric pharmaceutical development ................................. 20

    3.7 WHO activities towards better medicines for children ............................................................... 21

    3.8 Conclusions ....................................................................................................................................... 22

    4. Regulatory aspects related to children ................................................................................................. 23

    4.1 Implementation of the EU paediatric legal framework ............................................................... 23

    4.2 Achievements of the EU Paediatric Regulation ............................................................................ 25

    4.3 Patients participation in the development of paediatric medicines ......................................... 30

    4.4 Conclusions ....................................................................................................................................... 31

    5. The usage environment ........................................................................................................................... 32

    5.1 Off-label and unlicensed use of medicines in children ............................................................... 32

    5.2 Medicines use in children for specific diseases ............................................................................ 34 5.2.1 Use of antibiotics in children ...................................................................................................... 35 5.2.2 Use of psychotropic medicines in children ............................................................................... 35 5.2.3 Use of medicines in preterm newborns ..................................................................................... 36

    5.3 Use of medicines in children in hospitals ...................................................................................... 37

    5.4 Use of medicines in children in developing and transitional countries .................................... 38

    5.5 Adherence to treatment in children ............................................................................................... 39

  • Update on 2004 Background Paper, BP 7.1 Priority Medicines for Children

    7.1-3

    5.6 Availability of information on (off-label) paediatric medicines and its dissemination to health workers and patients ......................................................................................................................... 41

    6. Identified gaps and recommendations for research and policy ...................................................... 42

    References ........................................................................................................................................................... 44

    Annex 7.1.1: Infant mortality rates in Europe in 2010 .............................................................................. 55

    Annex 7.1.2: Regional causes of childhood deaths in 2010 ...................................................................... 56

    Annex 7.1.3: Developmental Changes in Physiology in Children .......................................................... 57

    Annex 7.1.4: EMA Matrix routes of administration/dosage form vs. age ........................................... 58

    Annex 7.1.5: Novel drug formulations for children .................................................................................. 59

    Annex 7.1.6: Novel drug devices for children ........................................................................................... 60

    Annex 7.1.7: Paediatric products prequalified up to 2012 ....................................................................... 60

    Annex 7.1.8: List of centrally authorised medicinal products for which the therapeutic indication

    was extended or amended to the paediatric population. ......................................................................... 62

    Annex 7.1.9: List of medicinal products and companies that have benefited from the 6-month

    extension of the supplementary protection certificate.............................................................................. 68

    Annex 7.1.10: Funded off-patent medicine projects (start up to 1 January 2010) and agreed PIPs, if

    available. ......................................................................................................................................................... 76

    Annex 7.1.11: Therapeutic needs in the paediatric population according to the survey of all

    paediatric uses (EMA/794083/2009) and projects addressing the needs ................................................ 78

    Annex 7.1.12: Projects on use of paediatric medicines funded by the Sixth and Seventh Framework

    Programme (FP6, FP7), excluding off-patent funded projects, presented in Annex 7.1.8 ................... 80

    Annex 7.1.13: WHO/INRUD prescribing indicators by health facility ownership ............................... 81

    Annex 7.1.14: WHO/INRUD prescribing indicators by prescriber type ................................................ 82

  • Update on 2004 Background Paper, BP 7.1 Priority Medicines for Children

    7.1-4

    Executive summary and key research points

    Children are entitled to safe, efficacious, and age-appropriate medicines. However, the

    provision of optimal medicines for children is limited by the lack of commercial incentives, a

    dearth of clinical trials on paediatric medicines, delays in licensing medicines for children,

    and the absence of suitable formulations for children. Children are not small adults, but

    rather a vulnerable population with specific needs resulting from their changing physiology,

    who make up a heterogeneous patient group with a scope of diseases different than those of

    adults, and for whom there is a scarcity of data on appropriate medicines delivery and use.

    Therefore, these needs are discussed in d