Abstract
Introduced into pharmaceutical parlance by Margaret Hamburg, US FDA Commissioner, the term ‘regulatory science’ covers ‘the development and use of new tools, standards and approaches to more efficiently develop products and to more effectively evaluate product safety, efficacy and quality’. We looked at what is new in this area concerning paediatric medicines, and in particular how these advances could be useful for children in Low- and Middle-Income Countries (LMIC).
Keywords
- Regulatory Science
- Severe Combine Immune Deficiency
- Paediatric Medicine
- Paediatric Clinical Trial
- Paediatric Investigation Plan
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.
This is a preview of subscription content, log in via an institution.
Buying options
Tax calculation will be finalised at checkout
Purchases are for personal use only
Learn about institutional subscriptionsNotes
- 1.
‘Medicine’ (or drug) as used here includes small molecules, biological products, gene or cell therapy, tissue engineered medicines and vaccines.
References
Kearns GL, Abdel-Rahman SM, Alander SW, Blowey DL, Leeder JS, Kauffman RE. Developmental pharmacology – drug disposition, action, and therapy in infants and children. N Engl J Med. 2003;349:1157–67.
World Health Organization. Development of paediatric medicines. Points to consider in formulations. http://www.who.int/childmedicines/partners/SabineKopp_Partners.pdf.
Saint-Raymond A, Sweeney F. Clinical trials in developing countries: risk or opportunity? Future Med Chem. 2009;1:1193–5. doi:10.4155/fmc.09.103.
European Medicines Agency. Concept paper on extrapolation of efficacy and safety in medicine development. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/04/WC500142358.pdf. Accessed 27 Aug 2014.
Black S, Margarit I, Rappuoli R. Preventing newborn infection with maternal immunization. Sci Transl Med. 2013;5:195ps11. doi:10.1126/scitranslmed.3005451.
Healy CM, Rench MA, Baker CJ. Importance of timing of maternal combined tetanus, diphtheria, and acellular pertussis (Tdap) immunization and protection of young infants. Clin Infect Dis. 2013;56:539–44. doi:10.1093/cid/cis923. Epub 2012 Oct 24.
Steinhoff MC. Assessments of vaccines for prenatal immunization. Vaccine. 2013;31 Suppl 4:D27–30. doi:10.1016/j.vaccine.2013.02.031.
Ruperto N, Martini A. Juvenile idiopathic arthritis and malignancy. Rheumatology (Oxford). 2014;53:968–74.
Olini N, Kurth S, Huber R. The effects of caffeine on sleep and maturational markers in the rat. PLoS One. 2013;8:e72539. doi:10.1371/journal.pone.0072539.eCollection2013.
Clarkson A, Ingleby E, Choonara I, Bryan P, Arlett P. A novel scheme for the reporting of adverse drug reactions. Arch Dis Child. 2001;84:337–9.
Blake KV, Zaccaria C, Domergue F, La Mache E, Saint-Raymond A, Hidalgo-Simon A. Comparison between paediatric and adult suspected adverse drug reactions reported to the European medicines agency: implications for pharmacovigilance. Paediatr Drugs. 2014;16:309–19. doi:10.1007/s40272-014-0076-2.
Carleer J, Karres J. Juvenile animal studies and pediatric drug development: a European regulatory perspective. Birth Defects Res B Dev Reprod Toxicol. 2011;92:254–60.
Olsson S, Pal SN, Stergachi A, Couper M. Pharmacovigilance activities in 55 low- and middle-income countries: a questionnaire-based analysis. Drug Saf. 2010;33:698–703.
Population Reference Bureau. Datasheet 2013. http://www.prb.org/pdf13/2013-population-data-sheet_eng.pdf. Accessed 27 Aug 2014.
World Health Organization. Model list of essential medicines for children 2013. http://www.who.int/medicines/publications/essentialmedicines/4th_EMLc_FINAL_web_8Jul13.pdf. Accessed 15 Sept 2014.
Global Alliance on Vaccines Initiative. www.gavi.org. Accessed 15 Sept 2014.
International Conference on Harmonization. Guideline Q1A R2 in Q1A-Q1F stability. http://www.ich.org/products/guidelines/quality/article/quality-guidelines.html. Accessed 15 Sept 2014.
Cockburn R, Agyarko KE, Akunyili D, White NJ. The global threat of counterfeit drugs: why industry and governments must communicate the dangers. PLOS Med. 2005. doi:10.1371/journal.pmed.0020100.
O’Brien KL, Selanikio JD, Hecdivert C, Placide MF, Louis M, Barr DB, et al. Epidemic of pediatric deaths from acute renal failure caused by diethylene glycol poisoning. JAMA. 1998;279:1175–80.
Almuzaini T, Choonara I, Sammons H. A systematic review of counterfeit and substandard medicines. BMJ Open. 2013;3:e002923. doi:10.1136/bmjopen-2013-002923.
United Nations Commission on life-saving commodities for women and children. http://www.everywomaneverychild.org/resources/un-commission-on-life-saving-commodities. Accessed 15 Sept 2014.
World Health Organization. Essential medicines for children. Report on an informal consultation on missing priority medicines for children. http://www.who.int/childmedicines/en/. Accessed 27 Aug 2014.
East African Community Regulatory Harmonisation. http://www.amrh.org/participating-recs-and-organisations/east-african-community-eac. Accessed 15 Sept 2014.
WHO support for regulatory harmonisation in Africa: focus on East African Community. WHO Drug Information. 2014;28(1). http://www.who.int/medicines/publications/druginformation/DI_28-1_Africa.pdf. Accessed 15 Sept 2014.
European & Developing countries Clinical Trials Partnership (EDCTP). http://www.edctp.org/. Accessed 15 Sept 2014.
African Vaccines Regulatory Forum (AVAREF). http://www.who.int/immunization_standards/vaccine_regulation/africa_network/en/. Accessed 15 Sept 2014.
President’s Emergency Plan for AIDS Relief. http://www.pepfar.gov/. Accessed 27 Aug 2014.
European Medicines Agency. Article 58 opinions. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000312.jsp&mid=WC0b01ac058001d12c. Accessed 27 Aug 2014.
World Health Organization. Paediatric medicines Regulators’ network. http://www.who.int/childmedicines/paediatric_regulators/en/. Accessed 27 Aug 2014.
Global Research in Paediatrics Network of Excellence. http://www.grip-network.org/index.php/cms/en/home. Accessed 27 August 2014.
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Additional information
Disclaimer
The views expressed in this chapter are the personal views of the author(s) and may not be understood or quoted as being made on behalf of or reflecting the position of the EMA or one of its committees or working parties.
Rights and permissions
Copyright information
© 2015 Springer International Publishing Switzerland
About this chapter
Cite this chapter
Saint-Raymond, A., Cooke, E. (2015). Regulatory Science for Paediatric Medicines in Low- and Middle-Income Countries. In: MacLeod, S., Hill, S., Koren, G., Rane, A. (eds) Optimizing Treatment for Children in the Developing World. Adis, Cham. https://doi.org/10.1007/978-3-319-15750-4_10
Download citation
DOI: https://doi.org/10.1007/978-3-319-15750-4_10
Publisher Name: Adis, Cham
Print ISBN: 978-3-319-15749-8
Online ISBN: 978-3-319-15750-4
eBook Packages: MedicineMedicine (R0)