Ethical, Regulatory and Scientific Challenges in Paediatric Drug Development
Tóm tắt
Từ khóa
Tài liệu tham khảo
US Food and Drug Administration. Overview and history of FDA and the Center for Food Safety and Applied Nutrition [online]. Available from URL: http://www.cfsan.fda.gov/~lrd/fdahist.html [Accessed 2008 May 13]
The story of the laws behind the labels. Part III: 1962 — drug amendments [online]. Available from URL: http://www.cfsan.fda.gov/~lrd/histor1b.html [Accessed 2008 May 13]
Hilts PJ. Protecting America’s health. New York: Alfred A. Knopf, 2003
Wax P. Elixirs, diluents, and the passage of the 1938 Federal Food, Drug and Cosmetic Act. Ann Intern Med 1995; 122: 456–61
US Food and Drug Administration. The story of the laws behind the labels. Part II: 1938 — The Federal Food, Drug, and Cosmetic Act [online]. Available from URL: http://www.cfsan.fda.gov/~lrd/histor1a.html [Accessed 2008 May 13]
International Conference on Harmonisation. History and future of ICH [online]. Available from URL: http://www.ich.org/cache/compo/276-254-1.html [Accessed 2008 May 13]
International Conference on Harmonisation. ICH tripartite guideline: guideline for good clinical practice — ICH E6(R1) [online]. Available from URL: http://www.ich.org/LOB/media/MEDIA482.pdf [Accessed 2008 May 13]
International Conference on Harmonisation. ICH tripartite guideline: clinical investigation of medicinal products in the pediatric population — ICH E11 [online]. Available from URL: http://www.ich.org/LOB/media/MEDIA487.pdf [Accessed 2008 May 13]
US Food and Drug Administration. FDA/NICHD conference. Clinical pharmacology during pregnancy: addressing clinical needs through science [online]. Available from URL: http://www.fda.gov/oc/speeches/2000/nichdconference12-4.html [Accessed 2008 May 13]
Golombek SG, van den Anker J, Rose K. Clinical trials in children: ethical and practical issues. Int J Pharm Med 2007; 21 (2): 121–9
US Food and Drug Administration. The pediatric exclusivity provision: status report to congress; 2001 Jan [online]. Available from URL: http://www.fda.gov/cder/pediatric/reportcong01.pdf [Accessed 2008 May 13]
Stoetter H. Paediatric drug development: historical background of regulatory initiatives. In: Rose K, van den Anker JN, editors. Guide to paediatric clinical research. Basel: Karger, 2007: 25–32
McIntyre J, Conroy S, Avery A, et al. Unlicensed and off label prescribing of drugs in general practice. Arch Dis Child 2000; 83: 498–501
Turner S, Longworth A, Nunn AJ, et al. Unlicensed and off label drug use in paediatric wards: prospective study. BMJ 1998; 316: 343–5
Ekins-Daukes S, Helms PJ, Taylor MW, et al. Off-label prescribing to children: attitudes and experience of general practitioners. Br J Clin Pharmacol 2005; 60 (2): 145–9
Conroy S, Choonara I, Impicciatore P, et al. Survey of unlicensed and off-label drug use in paediatric wards in five European countries. BMJ 2000; 320: 79–82
’t Jong GW, Vulto AG, de Hoog M, et al. Survey of the use of off-label and unlicensed drugs in a Dutch children’s hospital. Pediatrics 2001; 108 (5): 1089–93
’t Jong GW, Eland IA, Sturkenboom MC, et al. Unlicensed and off label prescription of drugs to children: population based cohort study. BMJ 2002; 324: 1313–4
Liebenson B. ART; dressing children in a century past. New York Times 2000 Jan 9 [online]. Available from URL: http://query.nytimes.com/gst/fullpage.html?res=9402EFD6173BF93AA35752C0A9669C8B63 [Accessed 2008 May 13]
Museum of Fine Arts, Boston. Dressing up: children’s fashions 1720–1920 [online]. Available from URL: http://www.mfa.org/exhibitions/sub.asp?key=15&subkey=654 [Accessed 2008 May 13]
J Pediatr Adol Med 2008 Jan; 9395 (76): [online]. Available from URL: http://www.medical-library.org/j_ped.htm [Accessed 2008 May 13]
Children’s health and welfare. In: Encyclopaedia Britannica. Chicago (IL): Encyclopaedia Britannica, 1962
Lederer SE, Grodin MA. Historical overview: pediatric experimentation. In: Grodin MA, Glanth LH, editors. Children as research subjects: science, ethics, and law. New York: Oxford University Press, 1994: 3–25
Ross LF. Children in medical research: access versus protection. New York: Oxford University Press, 2006
Weyers W. The abuse of man: an illustrated history of dubious medical experimentation. New York: Ardor Scribendi, 2003: 161, 235
Mozes-Kor E. The Mengele twins and human experimentation. In: Annas GJ, Grodin MA, editors. The Nazi doctors and the Nuremberg Code: human rights in human experimentation. New York: Oxford University Press, 1992
National Institutes of Health. Regulations and ethical guidelines: directives for human experimentation [online]. Available from URL: http://ohsr.od.nih.gov/guidelines/nuremberg.html [Accessed 2008 May 13]
Nuremberg code [online]. Available from URL: http://www.hhs.gov/ohrp/references/nurcode.htm [Accessed 2008 May 13]
Declaration of Geneva, 1948 — the medical code of ethics [online]. Available from URL: http://www.donoharm.org.uk/gendecl.htm [Accessed 2008 May 13]
International code of medical ethics of the World Medical Association — 1949 [online]. Available from URL: http://www.cirp.org/library/ethics/intlcode/ [Accessed 2008 May 13]
United Nations. Universal declaration of human rights [online]. Available from URL: http://www.un.org/Overview/rights.html [Accessed 2008 May 13]
United Nations. Declaration of the rights of the child (1959) [online]. Available from URL: http://www.cirp.org/library/ethics/UN-declaration/ [Accessed 2008 May 13]
Human experimentation: code of ethics of the World Medical Association [online]. Available from URL: http://www.pubmedcentral.nih.gov/picrender.fcgi?artid=1816102&blobtype=pdf [Accessed 2008 May 13]
Wikipedia. Declaration of Helsinki [online]. Available from URL: http://en.wikipedia.org/wiki/Declaration_of_Helsinki [Accessed 2008 May 13]
Krugman S. The Willowbrook hepatitis studies revisited: ethical aspects. Rev Infect Dis 1986; 8: 157–62
Hornblum AM. Acres of skin: human experiments at Holmesburg Prison. New York: Routledge, 1998
Jones JH. Bad blood: the Tuskegee syphilis experiment. New York: The Free Press, 1981
PBS Online. An apology 65 years late [online]. Available from URL: http://www.pbs.org/newshour/bb/health/may97/tuskegee_5-16.html [Accessed 2008 May 13]
US Department of Health and Human Services. Part 46: protection of human subjects. Section 46.103: assuring compliance with this policy — research conducted or supported by any Federal Department or Agency [online]. Available from URL: http://www.hhs.gov/ohrp/humansubjects/guidance/45cfr46.htm#46.103 [Accessed 2008 May 13]
US Department of Health and Human Services, Office for Human Research Protections (OHRP). OHRP 45 CFR part 46 frequently asked questions (FAQs) [online]. Available from URL: http://www.hhs.gov/ohrp/45CFRpt46faq.html [Accessed 2008 May 13]
President’s Council on Bioethics. Former bioethics commissions [online]. Available from URL: http://www.bioethics.gov/reports/past_commissions/in-dex.html [Accessed 2008 May 13]
Field MJ, Behrman RE, editors. Ethical conduct of clinical research involving children. Washington, DC: Institute of Medicine of the National Academies, The National Academies Press, 2004
Regulations and ethical guidelines. The Belmont report: ethical principles and guidelines for the protection of human subjects of research [online]. Available from URL: http://ohsr.od.nih.gov/guidelines/belmont.html [Accessed 2008 May 13]
Cassell EJ. The principles of the Belmont Report: how have respect for persons, beneficence, and justice been applied in clinical medicine? In: Childress JF, Meslin EM, Shapiro HT, editors. Belmont revisited: ethical principles for research with human subjects. Washington, DC: Georgetown University Press, 2005
American Academy of Pediatrics. Guidelines for the ethical conduct of studies to evaluate drugs in pediatric populations. Pediatrics 1977; 60 (1): 91–101 [online]. Available from URL: http://pediatrics.aappublications.org/cgi/reprint/60/1/91.pdf [Accessed 2008 May 26]
American Academy of Pediatrics. Guidelines for the ethical conduct of studies to evaluate drugs in pediatric populations. Pediatrics 1995; 95 (2): 286–94 [online]. Available from URL: http://pediatrics.aappublications.org/cgi/reprint/95/2/286.pdf [Accessed 2008 May 26]
American Academy of Pediatric/Society for Pediatric Research. Statement before the Institute of Medicine Committee on Clinical Research involving Children: participation and protection of children in clinical research. Presented: 2003 Jul 9 [online]. Available from URL: http://aps-spr.org/public_policy/2003_Docs/IOM0709.htm [Accessed 2008 May 26]
National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research. Report and recommendations: research involving children [online]. Available from URL: http://bioethics.gov/reports/past_commis-sions/Research_involving_children.pdf [Accessed 2008 Jul 14]
Department of Health and Human Services. Code of Federal Regulations 45 — public welfare, part 46: protection of human subjects. Subpart D: additional protections for children involved as subjects in research [online]. Available from URL: http://www.hhs.gov/ohrp/humansubjects/guidance/45cfr46._htm#subpartd [Accessed 2008 May 26]
Department of Health and Human Services. Code of Federal Regulations 45 — public welfare, part 46: protection of human subjects. Subpart B: additional protections for pregnant women, human fetuses and neonates involved in research [online]. Available from URL: http://www.hhs.gov/ohrp/humansubjects/guidance/45cfr46.htm#subpartb [Accessed 2008 May 26]
Department of Health and Human Services. Code of Federal Regulations 45 — public welfare, part 46: protection of human subjects. Subpart C: additional protections pertaining to biomedical and behavioral research involving prisoners as subjects [online]. Available from URL: http://www.hhs.gov/ohrp/human-subjects/guidance/45cfr46.htm#subpartc [Accessed 2008 May 26]
United Nations. Convention on the rights of the child. Document A/RES/44/25, 1989 Dec 12 [online]. Available from URL: http://www.cirp.org/library/ethics/UN-convention/ [Accessed 2008 May 26]
Office for Human Research Protections. Common rule [online]. Available from URL: http://www.hhs.gov/ohrp/policy/#common [Accessed 2008 May 26]
Council of Europe. Convention on human rights and biomedicine [online]. Available from URL: http://www.worldcarecouncil.org/pdf/CoE_HRBioMed.pdf [Accessed 2008 May 26]
World Medical Association. World Medical Association declaration of Ottawa on the rights of the child to health care [online]. Available from URL: http://www.wma.net/e/policy/c4.htm [Accessed 2008 May 26]
Council for International Organizations of Medical Sciences. International ethical guidelines for biomedical research involving human subjects [online]. Available from URL: http://www.cioms.ch/frame_guidelines_nov_2002.htm [Accessed 2008 May 26]
EMEA. Ethical considerations for clinical trials performed in children. Recommendations of the Ad hoc group for the development of implementing guidelines for Directive 2001/20/EC relating to good clinical practice in the conduct of clinical trials on medicinal products for human use [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/paediatrics/docs/paeds_ethics_consultation20060929.pdf [Accessed 2008 May 26]
Ethical considerations for clinical trials on medicinal products conducted with the paediatric population: recommendations of the ad hoc group for the development of implementing guidelines for Directive 2001/20/EC relating to good clinical practice in the conduct of clinical trials on medicinal products for human use. Final 2008 [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-10/ethical_considerations.pdf [Accessed 2008 Jul 14]
EU clinical trials directive. Directive 2001/20/EC of the European Parliament and of the council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use [online]. Available from URL: http://www.eortc.be/Services/Doc/clinical-EU-directive-04-April-01.pdf [Accessed 2008 May 26]
Food And Drug Administration Moderization Act of 1997: Public Law 105-115 [online]. Available from URL: http://www.fda.gov/cder/guidance/105-115.htm#SEC.%20111 [Accessed 2008 May 26]
US Food and Drug Administration, Center for Drug Evaluation and Research. Guidance for industry qualifying for pediatric exclusivity under section 505A of the Federal Food, Drug, and Cosmetic Act [online]. Available from URL: http://www.fda.gov/cder/guidance/2891fnl.htm [Accessed 2008 May 26]
US Food and Drug Administration. Best pharmaceuticals for children act [online]. Available from URL: http://www.fda.gov/cder/pediatric/PL107-109.pdf [Accessed 2008 May 26]
US Food and Drug Administration. Pediatric research equity act of 2003 [online]. Available from URL: http://www.fda.gov/cder/pediatric/S-650-PREA.pdf [Accessed 2008 May 26]
US Food and Drug Administration. Guidance for industry: how to comply with the pediatric research equity act [online]. Available from URL: http://www.fda.gov/cder/guidance/6215dft.pdf [Accessed 2008 May 26]
US Food and Drug Administration, Department of Health and Human Services. 21 CFR Parts 201, 312, 314 and 601._Regulations requiring manufacturers to assess the safety and effectiveness of new drugs and biological products in pediatric patients: final rule [online]. Available from URL: http://www.fda.gov/ohrms/dockets/98fr/120298c.pdf [Accessed 2008 May 26]
Foley Hoag LLP. Overview of the Food and Drug Administration amendments act of 2007. Life Sciences Alert 2007 Sep 21 [online]. Available from URL: http://www.foleyhoag.com/~/media/Files/Publications/Alerts/Life%20Sciences/LifeSciences_Gvt_Strategies_Alert_092107.ashx [Accessed 2008 May 26]
US Food and Drug Administration. Drugs to which FDA has granted pediatric exclusivity for pediatric studies under section 505A of the Federal Food, Drug, and Cosmetic Act [online]. Available from URL: http://www.fda.gov/cder/pediatric/exgrant.htm [Accessed 2008 May 26]
Pasquali SK, Sanders SP, Li JS. Oral antihypertensive trial design and analysis under the pediatric exclusivity provision. Am Heart J 2002; 144 (4): 608–14
US Food and Drug Administration, Division of Metabolic and Endocrine Drug Products. sNDA application for Fosamax (alendronate sodium) [online]. Available from URL: http://www.fda.gov/cder/foi/esum/2003/20560se1-038BP-CA.pdf [Accessed 2008 Jun 3]
Birenbaum L. Labeled products with significant new information [online]. Avail- able from URL: http://www.fda.gov/cder/pediatric/presentation/dlb2-DIA-Mar2003/sld011.htm [Accessed 2008 Jun 3]
Boston BA, Drury Brown MK. McCune-Albright syndrome. eMedicine [online]. Available from URL: http://www.emedicine.com/ped/topic1386.htm [Accessed 2008 Jun 3]
US Food and Drug Administration. Approved active moieties to which FDA has issued a Written Request for Pediatric Studies under Section 505A of the Federal Food, Drug, and Cosmetic Act [online]. Available from URL: http://www.fda.gov/cder/pediatric/wrlist.htm [Accessed 2008 Jun 3]
Clinical trials in children, for children [editorial]. Lancet 2006 Jun 17; 367 (9527): 1953
European Medicines Agency. The European paediatric initiative: history of the paediatric regulation. EMEA/17967/04 Rev 1 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/paediatrics/1796704en.pdf [Accessed 2008 Jun 3]
Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004._Off J EU 2006 Dec 27 [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/reg_2006_1901/reg_2006_1901_en.pdf [Accessed 2008 Jun 3]
Regulation (EC) No 1902/2006 of the European Parliament and of the Council of 20 December 2006 amending Regulation 1901/2006 on medicinal products for paediatric use [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/reg_2006_1902/reg_2006_1902_en.pdf [Accessed 2008 Jun 3]
Wikipedia. Supplementary protection certificate [online]. Available from URL: http://en.wikipedia.org/wiki/Supplementary_protection_certificate [Accessed 2008 Jun 3]
EMEA. Paediatric Committee [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/pdco.htm [Accessed 2008 Jun 3]
EMEA. EMEA Paediatric Working Party meets for last time ahead of creation of new Paediatric Committee [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/press/pr/25919207en.pdf [Accessed 2008 Jun 3]
EMEA. New Paediatric Committee holds its first meeting [press release; online]. Available from URL: http://www.emea.europa.eu/pdfs/human/pdco/29568907en.pdf [Accessed 2008 Jun 3]
Seigneuret N. Implementation on the paediatric regulation EMEA views: 2007 EMEA — EFPIA info day [online]. Available from URL: http://www.emea.europa.eu/pdfs/conferenceflyers/EMEA-EFPIA_2Feb2007/1330-N_Seigneuret.pdf [Accessed 2008 Jun 3]
EMEA. Practical aspects on how to submit an application for paediatric investigation plan and requests for waiver and deferral. EMEA/252191/2007-rev 5 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/paed-iatrics/practical_aspects.pdf [Accessed 2008 Jun 3]
EMEA. Paediatric investigation plans (PIPs), waivers and modifications [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/pips.htm [Accessed 2008 Jun 3]
EMEA. Submission of paediatric studies [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/studies.htm [Accessed 2008 Jun 3]
EMEA. Compliance [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/compliance.htm [Accessed 2008 Jun 3]
EMEA. Scientific advice [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/sci_advice.htm [Accessed 2008 Jun 3]
EMEA. Scientific advice and protocol assistance [online]. Available from URL: http://www.emea.europa.eu/htms/human/sciadvice/Scientific.htm [Accessed 2008 Jun 3]
Committee for Medicinal Products for Human Use. Guideline on conduct of pharmacovigilance for medicines used by the paediatric population. EMEA/CHMP/PhVWP/235910/2005, rev 1 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/phvwp/23591005en.pdf [Accessed 2008 Jun 3]
EMEA. Paediatric-use marketing authorisations (PUMAs) [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/pumas.htm [Accessed 2008 Jun 3]
Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/reg_2004_726/reg_2004_726_en.pdf [Accessed 2008 Jun 3]
US Food and Drug Administration. Overview of new legislation: Food and Drug Administration Amendments Act of September 2007Amendments 2007 [online]. Available from URL: http://www.fda.gov/ohrms/dockets/ac/08/slides/2008-4344s1_10_01_Introduction%20Murphy.pdf [Accessed 2008 Jun 3]
EMEA. Commission guideline on the format and content of applications for agreement or modification of a paediatric investigation plan and requests for waivers or deferrals and concerning the operation of the compliance check and on criteria for assessing significant studies [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/paediatrics/docs/draft_guideline_pip_2007-02.pdf [Accessed 2008 Jun 3]
Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the community code relating to medicinal products for human use [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/dir_2001_83/dir_2001_83_en.pdf [Accessed 2008 Jun 3]
Commission Directive 2003/63/EC of 25 June 2003 amending Directive 2001/83/ EC of the European Parliament and of the Council on the community code relating to medicinal products for human use [online]. Available from URL: http://ec.europa.eu/enterprise/pharmaceuticals/eudralex/vol-1/dir_2003_63/dir_2003_63_en.pdf [Accessed 2008 Jun 3]
EMEA. EMEA decisions on paediatric investigation plans (PIPs), including defer- rals and waivers, and on modification of an agreed PIP [online]. Available from URL: http://www.emea.europa.eu/htms/human/paediatrics/decisions.htm [Accessed 2008 Jun 3]
Wikipedia. Gaucher’s disease [online]. Available from URL: http://en.wikipedia.org/wiki/Gaucher%27s_disease [Accessed 2008 Jun 3]
Mehta A, Hughes DA. Fabry disease. GeneReviews [online]. Available from URL: http://www.genetests.org/query?dz=fabry [Accessed 2008 Jun 3]
Aggarwal S. What’s fueling the biotech engine? Nature Biotechnol 2007; 25: 1097–104 [online]. Available from URL: http://www.nature.com/nbt/journal/v25/n10/abs/nbt1007-1097.html [Accessed 2008 Jun 3]
Cystic Fibrosis Foundation. About cystic fibrosis: what you need to know [online]. Available from URL: http://www.cff.org/AboutCF/ [Accessed 2008 Jun 3]
Pediatric exclusivity labeling changes as of May 1, 2008 [online]. Available from URL: http://www.fda.gov/cder/pediatric/labelchange.htm [Accessed 2008 Jun 3]
Department of Health and Human Services. The Orphan Drug Act: implementation and impact [online]. Available from URL: http://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf [Accessed 2008 Jun 3]
EU. Fighting rare diseases: 22 new orphan drugs in five years [press release; online]. Available from URL: http://europa.eu/rapid/pressReleasesAc-tion.do?reference=IP/06/844&type=HTML&aged=0&language=EN&guiLanguage=en [Accessed 2008 Jun 3]
Pizzo PA, Poplack DG, editors. Principles and practice of pediatric oncology. 5th ed. Philadelphia (PA): Lippincott Williams & Wilkins, 2006
Hirschfeld S, Ho PTC, Smith M, et al. Regulatory approvals of pediatric oncology drugs: previous experience and new initiatives. J Clin Oncol 2003; 21: 1066–73
Cancer Research UK. Childhood cancer survival statistics [online]. Available from URL: http://info.cancerresearchuk.org/cancerstats/childhoodcancer/survival/ [Accessed 2008 Jul 14]
Ries LAG, Smith MA, Gurney JG, et al., editors. Cancer incidence and survival among children and adolescents: United States SEER Program 1975–1995. Bethesda (MD): National Cancer Institute, 1999. NIH Pub. No. 99–4649 [online]. Available from URL: http://seer.cancer.gov/publications/childhood/ [Accessed 2008 Jun 3]
Center for Drug Evaluation and Research. Pediatric cancer therapeutics: letter of interest [online]. Available from URL: http://www.fda.gov/cder/pediatric/ped-cancerletter.htm [Accessed 2008 Jun 3]
National Cancer Institute. Targeted cancer therapies: questions and answers [online]. Available from URL: http://www.cancer.gov/cancertopics/factsheet/Therapy/targeted [Accessed 2008 Jun 3]
Smith M. Evolving molecularly targeted therapies and biotherapeuics. In: Pizzo PA, Poplack DG, editors. Principles and practice of pediatric oncology. 5th ed. Philadelphia (PA): Lippincott Williams & Wilkins, 2006: 366–404
EMEA. Note for guidance on evaluation of anticancer medicinal products in man: addendum on paediatric oncology. EMEA/CPMP/EWP/569/02 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/ewp/056902en.pdf [Accessed 2008 Jun 3]
EMEA. Note for guidance on evaluation of anticancer medicinal products in man. CPMP/EWP/205/95 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/ewp/020595en.pdf [Accessed 2008 Jun 3]
Innovative Therapies for Children with Cancer: European Consortium [online]. Available from URL: http://www.itcc-consortium.org/ [Accessed 2008 Jun 3]
SIOP Europe — the European Society for Paediatric Oncology [online]. Available from URL: http://www.ecco-org.eu/About-Ecco/Membership/SIOP-Europe/page.aspx/331 [Accessed 2008 Jun 4]
Houghton PJ, Morton CL, Tucker C, et al. The pediatric preclinical testing program: description of models and early testing results. Pediatr Blood Cancer 2006; 49 (7): 928–40 [online]. Available from URL: http://www3.interscience.wiley.com/journal/113441320/abstract [Accessed 2008 Jun 3]
National Cancer Institute. Childhood cancer resources: pediatric clinical trials cooperative groups and consortia [online]. Available from URL: http://ctep.cancer.gov/resources/child.html [Accessed 2008 Jun 3]
Children’s Oncology Group (COG) [online]. Available from URL: http://www.childrensoncologygroup.org/ [Accessed 2008 Jun 3]
EMEA/CHMP. Guideline on clinical trials in small populations. CHMP/EWP/ 83561/2005 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/ewp/8356105en.pdf [Accessed 2008 Jun 3]
EMEA. Orphan drugs and rare diseases at a glance. EMEA/290072/2007 [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/comp/29007207en.pdf [Accessed 2008 Jun 3]
National Institutes of Health. Request for proposals for clinical trials: NICHD’s approach to the study of off-patent drugs mandated by the BPCA [online]. Available from URL: http://bpca.nichd.nih.gov/clinical/rfp/index.cfm [Accessed 2008 Jun 3]
Seventh research framework programme (FP7) [online]. Available from URL: http://cordis.europa.eu/fp7/home_en.html [Accessed 2008 Jun 3]
Medicines for Children Research Network [online]. Available from URL: http://www.mcrn.org.uk/ [Accessed 2008 Jun 4]
Medicines for Children Research Network [online]. Available from URL: http://www.mcrn.nl/ [Accessed 2008 Jun 4]
PAED-Net: a German paediatric network [online]. Available from URL: http://www.paed-net.org/ [Accessed 2008 Jun 4]
Finnish Investigators Network for Pediatric Medicines — FINPEDMED [online]. Available from URL: http://www.hyksinstituutti.fi/document.asp?intSiteID=2&intDocID=350 [Accessed 2008 Jul 14]
RIPPS; le Reseau d’Investigations Pédiatriques des Produits de Santé [online]. Available from URL: http://site8.actengo.com/ripps/site/defaut/ [Accessed 2008 Jul 14]
Pediatric Rheumatology International Trials Organisation (PRINTO) [online]. Available from URL: http://www.printo.it [Accessed 2008 Jun 4]
PENTA [online]. Available from URL: http://www.pentatrials.org/ [Accessed 2008 Jul 14]
Task-force in Europe for Drug Development for the Young. [online]. Available from URL: http://www.teddyoung.org [Accessed 2008 May 25]
European Medicines Agency. The Network of paediatric networks at the EMEA: draft implementing strategy (for public consultation until 20th August 2007) [online]. Available from URL: http://www.emea.europa.eu/pdfs/human/paediatrics/29559407en.pdf [Accessed 2008 May 25]
World Health Organization. Make medicines child size [online]. Available from URL: http://www.who.int/childmedicines/en/ [Accessed 2008 May 25]
Lamprill J, Fowler T. Consent and assent in paediatric clinical trials. In: Rose K, van den Anker JN, editors. Guide to paediatric clinical research. Basel: Karger, 2007