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International Harmonization for Cell and Gene Therapy Products

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Regulatory Aspects of Gene Therapy and Cell Therapy Products

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1430))

Abstract

To increase the global availability of cell and gene therapy products, international regulatory agencies engage in programs that enhance dialogue between regulators, provide opportunities for training low- and middle-income countries lacking the capacity for regulatory oversight of cell and gene therapies, and support harmonization of regulatory requirements. This chapter provides overviews of the International Pharmaceutical Regulators Programme (IPRP) Cell Therapy Working Group (CTWG) and Gene Therapy Working Group (GTWG), the International Conference on Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), and the Asia Pacific Economic Cooperation (APEC) Regulatory Harmonization Steering Committee (RHSC). Also discussed are programs between small groups of regulators referred to as “Discussion Clusters” and Parallel Scientific Advice (PSA).

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Abbreviations

APEC:

Asia Pacific Economic Cooperation

APIC:

Association for Professionals in Infection Control

BIO:

Biotechnology Innovation Organization

BD:

Biodistribution

CIOMS:

Council for International Organizations of Medical Science

COFEPRIS :

Federal Commission for the Protection against Sanitary Risk

CTWG:

Cell Therapy Working Group

CoRE:

Centers of Regulatory Excellence

EAC:

East African Community

EC:

European Commission

EDQM:

European Directorate for the Quality of Medicines

EFPIA:

European Federation of Pharmaceutical Industries and Associations

EMA:

European Medicines Agency

GHC:

Global Health Corps

GTWG:

Gene Therapy Working Group

HSA:

Health Sciences Authority

ICH:

International Conference on Harmonization of Technical Requirements for Pharmaceuticals for Human Use

IGBA:

International Generic and Biosimilar Medicines Association

IPEC:

International Pharmaceutical Excipients Council

IPRF:

International Regulators Forum

IPRP:

International Pharmaceutical Regulators Programme

JPMA:

Japan Pharmaceutical Manufacturers Association

MHRA:

Medicines Healthcare Products Regulatory Agency

PANDRH:

Pan American Network for Drug Regulatory Harmonization

PhRMA:

Pharmaceutical and Research and Manufacturers of America

PIC/S:

Pharmaceutical Inspection Convention and Pharmaceutical Inspection Cooperation Scheme

PSA:

Parallel Scientific Advice

RHSC:

Regulatory Harmonization Steering Committee

SADC:

Southern Africa Development Community

USFDA:

United States Food and Drug Administration

USP:

United States Pharmacopeia

WHO:

World Health Organization

References

  1. IPRP Working Groups, https://www.iprp.global/working-groups (accessed 1/25/23)

  2. General Principles for Address the Nature and Duration of Follow-up for Subjects of Clinical Trials Using Cell Therapy Products, 2018. https://admin.iprp.global/sites/default/files/201812/IPRP_CTWG_ReflectionPaper_2018_1017.pdf (accessed 11/17/22).

  3. Expectations for Biodistribution (BD) Assessments for Gene Therapy (GT) Products, 2018. https://admin.iprp.global/sites/default/files/2018-09/IPRP_GTWG_ReflectionPaper_BD_Final_2018_0713.pdf (accessed 11/17/22).

  4. Draft Guideline ICH S12 Non-clinical Biodistribution Consideration for Gene Therapy Products, 2021. https://database.ich.org/sites/default/files/ICH_S12_Step2_DraftGuideline_2021_0603.pdf (accessed 11/18/22).

  5. Overview of ICH, 2022. https://admin.ich.org/sites/default/files/2022-11/OverviewOfICH_2022_1129.pdf (accessed 1/25/23)

  6. Formal ICH Procedure, https://ich.org/page/formal-ich-procedure (accesses 1/25/23).

  7. General Principles to Address the Risk of Inadvertent Germline Integration of Gene Therapy Vectors”, 2006. https://admin.ich.org/sites/default/files/2019-04/ICH_Considerations_General_Principles_Risk_of_IGI_GT_Vectors.pdf (accessed 11/17/22).

  8. General Principles to Address Virus and Vector Shedding, 2009. https://admin.ich.org/sites/default/files/2019-04/ICH_Considerations_Viral-Vector_Shedding_.pdf (accessed 11/18/22).

  9. Oncolytic Viruses, 2009. https://admin.ich.org/sites/default/files/2019-04/ICH_Considerations_Oncolytic_Viruses_rev_Sep_17_09.pdf (accessed 11/17/22).

  10. Roadmap to Promote Prospective Regulatory Convergence in Cell and Tissue-based Products, 2019. https://www.apec.org/-/media/Satellite/RHSC/PWA-Documents/Advanced-Therapies/APEC-RHSCATPRoadmap-2019-Nov.pdf (assessed 11/17/22).

  11. Asia Pacific Economic Cooperation Regulatory Harmonisation Steering Committee Centers of Excellence, https://www.apec.org/RHSC/Contact-Us/Centers-of-Excellencehttps://www.apec.org/RHSC/Contact-Us/Centers-of-Excellence (accessed 1/27/23)

  12. Asia Pacific Economic Cooperation Regulatory Harmonisation Steering Committee Centers of Excellence Advanced Therapies Core Curriculum, 2019. https://www.apec.org/-/media/Satellite/RHSC/PWA-Documents/Advanced-Therapies/APEC-RHSCATPCore-Curriculum-2019-Feb.pdfhttps://www.apec.org/-/media/Satellite/RHSC/PWA-Documents/Advanced-Therapies/APEC-RHSCATPCore-Curriculum-2019-Feb.pdf (accessed 1/25/23)

  13. General Principles FDA-EMA Parallel Scientific Advice (Human Medicinal Products), 2021. https://www.fda.gov/media/105211/download (accessed 11/17/22).

  14. CBER SOPP 8001.6 Procedures for Parallel Scientific Advice with EMA and FDA, 2020. https://www.fda.gov/media/87478/download#:~:text=Parallel%20Scientific%20Advice%20(PSA)%20%2D,human%20drugs%20and%20biologic%20products (assessed 11/17/22)

  15. European Medicines Agency Guidance for Applicants Seeking scientific advice and protocol assistance, 2022. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/european-medicines-agency-guidance-applicants-seeking-scientific-advice-protocol-assistance_en.pdf (assessed 11/17/22)

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Correspondence to Judith Arcidiacono .

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Arcidiacono, J. (2023). International Harmonization for Cell and Gene Therapy Products. In: Galli, M.C. (eds) Regulatory Aspects of Gene Therapy and Cell Therapy Products. Advances in Experimental Medicine and Biology, vol 1430. Springer, Cham. https://doi.org/10.1007/978-3-031-34567-8_14

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