Abstract
Independent development of regulatory frameworks in Australia, Europe and the USA has led to differences in their regulatory approach to biologics (or biologicals). Some of these were favourable for the conduct of early clinical trials (i.e. TGA CTN and CTX). Others have been affected by external factors (i.e. UK membership in the EU) or have expanded their scope (i.e. CBER emergence within the FDA). Recently efforts have been made to harmonise the three frameworks via joint guidances to industry and researchers and memoranda of understanding and cooperation among the regulatory bodies from the regions. We present our own experience in manufacturing and use of human placenta-derived mesenchymal stromal cells (hpMSC) in phase 1 clinical trials conducted in Australia according to the new Biologicals Framework established by Therapeutic Goods Administration (TGA) as from 1 July 2011. We also present similarities and differences with some other regulatory frameworks (USA and EU) that may be of interest to us in the future.
This is a preview of subscription content, log in via an institution to check access.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Abbreviations
- ARTG:
-
Australian Register of Therapeutic Goods
- CBER:
-
Center for Biologics Evaluation and Research
- CDER:
-
Center for Drugs Evaluation and Research
- CFR:
-
Code of Federal Regulation
- CTN:
-
Clinical Trial Notification
- CTX:
-
Clinical Trial Exemption
- DDXs:
-
Doctors and Dentists Exemptions
- EMA:
-
European Medicines Agency
- FDA:
-
Food and Drug Administration
- GCP:
-
Good Clinical Practice
- GMP:
-
Good Manufacturing Practice
- HREC:
-
Human Research Ethics Committee
- ICH:
-
International Conference on Harmonisation
- IND:
-
Investigational New Drug Application
- IRB:
-
Institutional Review Board
- MHRA:
-
Medicines and Healthcare Products Regulatory Agency
- PMDA:
-
Pharmaceutical and Medical Devices Agency
- TGA:
-
Therapeutics Goods Administration
References
Australian Government, Department of Health and Ageing, Therapeutic Goods Administration. Access to Unapproved Therapeutic Goods—Clinical Trials in Australia. October 2004. http://www.tga.gov.au/docs/html/clintrials.htm
Australian Government, Department of Health and Ageing, Therapeutic Goods Administration. Human Research Ethics Committees and the Therapeutic Goods Legislation. June 2001. http://www.tga.gov.au/docs/html/hrec.htm
Therapeutic Goods Administration [Online]. http://www.tga.gov.au/docs/html/tga/tgaginfo.htm
Therapeutic Goods Administration [Online]. http://www.tga.gov.au/docs/html/clintrials.htm
Therapeutic Goods Administration [Online]. http://www.tga.gov.au/unapp/ctglance.htm
The Center for Biologics Evaluation and Research (CBER). http://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/NewDrugApplicationNDAProcess/default.htm
The Food and Drug Administration (FDA). http://www.fda.gov
The Center for Drug Evaluation and Research (CDER). http://www.fda.gov/cder/regulatory/applications/ind_page_1.htm
The Center for Drug Evaluation and Research (CDER). http://www.fda.gov/cder/regulatory/applications/ind_page_1.htm
The Center for Drug Evaluation and Research (CDER). http://www.fda.gov/cder/guidance/phase1.pdf
The Center for Drug Evaluation and Research (CDER). http://www.fda.gov/cder/handbook/ind.htm
The Center for Biologics Evaluation and Research (CBER). http://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/default.htm and http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/ComplianceActivities/Enforcement/CompliancePrograms/ucm095207.htm
The European Medicines Agency (EMA). http://www.ema.europa.eu/ema/index.jsp?curl=/pages/home/Home_Page.jsp
The European Medicines Agency (EMA). http://www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/general/general_content_000235.jsp&murl=menus/about_us/about_us.jsp&mid=
Brooke G, Rossetti T, Ilic N, Murray P, Hancock S, Hancock S, Atkinson K (2008) Points to consider in designing mesenchymal stem cell-based clinical trials. Transf Med Hemother 35:279–285
Brooke G, Rossetti T, Pelekanos R, Ilic N, Murray P, Hancock S, Antonenas V, Huang G, Gottlieb D, Bradstock K, Atkinson K (2008) Manufacturing of human placenta-derived mesenchymal stem cells for clinical trials. Br J Haematol 144:571–579
Barlow S, Brooke G, Chatterjee K, Price G, Pelekanos R, Rossetti T, Doody M, Venter D, Pain S, Gilshenan K, Atkinson K (2008) Comparison of human placenta- and bone marrow-derived multipotent mesenchymal stem cells. Stem Cells Dev 17(6):1095–1108
Brooke G, Tong H, Levesque JP, Atkinson K (2008) Molecular trafficking mechanisms of multipotent mesenchymal stem cells derived from human bone marrow and placenta. Stem Cells Dev 17:1039–41
Jones BJ, Brooke G, Atkinson K, McTaggart SJ (2007) Immunosuppression by placental indoleamine 2,3-dioxygenase: a role for mesenchymal stem cells. Placenta 28:1174–1181
Yen BL, Huang HI, Chien CC, Jui HY, Ko BS, Yao M, Shun CT, Yen ML, Lee MC, Chen YC (2005) Isolation of multipotent cells from human term placenta. Stem Cells 23:3–9
Ilic N, Brooke G, Murray P, Barlow S, Rossetti T, Pelekanos R, Hancock S, Atkinson K (2011) Manufacturing of clinical grade human placenta-derived mesenchymal stem cells (MSC). In Vemuri M, Chase L, Rao M (eds) Mesenchymal stem cell assays and applications. Methods in molecular biology, vol 698, 1st edn. Humana, Totowa. ISBN: 978-1-60761-998-7
Brooke G, Cook M, Blair C, Han R, Heazlewood C, Jones B, Kambouris M, Kollar K, McTaggart S, Pelekanos R, Rice A, Rossetti T, Atkinson K (2007) Therapeutic applications of mesenchymal stromal cells. Semin Cell Dev Biol 18:846–858
The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). http://www.ich.org
Guide to the selection of mothers and cord blood donors. AusCord Cord Blood Banks 2008
National Statement on Ethical Conduct in Human Research. Australian Government 2007. http://www.nhmrc.gov.au
Note for guidance on GCP (CPMP/ICH/35/95) annotated with TGA comments. http://www.tga.gov.au
The Australian clinical trials handbook—a simple, practical guide to the conduct of clinical trials to International standards of Good Clinical Practice (GCP) in the Australian Context, March 2006. http://www.tga.gov.au
Acknowledgments
The authors would like to acknowledge funding from the Inner Wheel Australia, Mater Foundation, Therapeutic Innovation Australia (formerly Research Infrastructure Support Services), The Australian Stem Cell Foundation and Mater Health Services for making this work possible.
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2013 Springer Science+Business Media New York
About this chapter
Cite this chapter
Ilic, N., Khalil, D., Hancock, S., Atkinson, K. (2013). Regulatory Considerations Applicable to Manufacturing of Human Placenta-Derived Mesenchymal Stromal Cells (MSC) Used in Clinical Trials in Australia and Comparison to USA and European Regulatory Frameworks. In: Chase, L., Vemuri, M. (eds) Mesenchymal Stem Cell Therapy. Stem Cell Biology and Regenerative Medicine. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-200-1_17
Download citation
DOI: https://doi.org/10.1007/978-1-62703-200-1_17
Published:
Publisher Name: Humana Press, Totowa, NJ
Print ISBN: 978-1-62703-199-8
Online ISBN: 978-1-62703-200-1
eBook Packages: Biomedical and Life SciencesBiomedical and Life Sciences (R0)