News Hub

Gabrielle O’Sullivan PhD MPH (Hons)
Executive Officer, 
Royal Prince Alfred Hospital Institutional Biosafety Committee, 
Royal Prince Alfred Hospital, Australia

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AUSTRALIAN AND NEW ZEALAND REGULATORY UPDATES

Australian Government response to the House of Representatives Standing Committee on Health, Aged Care and Sport report on the inquiry into approval processes for new drugs and novel medical technologies in Australia: The New Frontier – Delivering better health for all Australians [1]

On 30 November 2023, the Australian Government Department of Health and Aged Care published its Response to the House of Representatives Standing Committee on Health, Aged Care and Sport Report on the Inquiry into approval processes for new drugs and novel medical technologies in Australia. In 2020, the Inquiry was referred to the House of Representatives Standing Committee on Health, Aged Care and Sport. The Inquiry Report was tabled in 2021 and provided 31 recommendations. The Australian Government’s Response to the recommendations in the Inquiry Report is entitled “The New Frontier – Delivering better health for all Australians.” [8]  Most of the 31 recommendations have been accepted by the Government. These include (among others) recommendations to:

• Establish a Centre for Precision Medicine and Rare Diseases within the Department of Health
• Simplify the Health Technology Assessment (HTA) process for cell and gene therapies
• Establish an Office of Clinical Evaluation within the Department of Health to assess the best and most effective care for patients in the context of new and emerging health technologies
• Improve regulatory submission fee structures and processes 
• Establish a fund to support patients, clinicians and non-profit organisations to sponsor registration and reimbursement applications where there is no realistic prospect of a company serving as sponsor, and where the Department of Health is otherwise supportive of the application
• Improve the Orphan Drugs Program and HTA processes
• Integrate the patient voice into HTAs
• Reform regulatory and reimbursement processes
• Develop a national clinical trial register

Implementation of the Inquiry recommendations is expected to result in major improvements to Australians’ access to new drugs and health technologies.

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GMP
The TGA has created a new GMP clearance Sponsor Information Dashboard to provide industry with current information relating to GMP clearance. It will be updated monthly and provide information on processing timeframes, current work and priorities, key messages and updates.

In consultation with the TGA-Industry Working Group on GMP (TIWGG), the TGA has updated the GMP clearance guidance document for sponsors seeking to obtain GMP clearance for an overseas manufacturing site used in the manufacture of a medicine, or an Active Pharmaceutical Ingredient (API) used in a medicine, intended for supply in Australia.

Note: The updated guidance referred to above is not intended for sponsors of biologicals, human blood and blood components and Haematopoietic Progenitor Cells (HPCs), because Australia has its own manufacturing standard for these product types; however, where the manufacturing site performs only sterilisation of these product types, the guidance may be used. [2, 3, 4] 

The TGA has various international arrangements with other countries and regulatory authorities that allow mutual reliance on each other’s GMP inspection programs [5]. Recently TGA updated its temporary measures for processing GMP clearances via the Mutual Recognition Agreement (MRA) and Compliance Verification (CV) pathways to ensure Australian sponsors can maintain the validity of their GMP clearances and supply of goods to Australia. [6]

The Australia and New Zealand (ANZ) Region of ISCT is represented on TIWGG by members of the ANZ ISCT Legal and Regulatory Affairs (LRA) Committee. [7] 

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Gene Drive Technology

Gene drives are genetic elements that are favoured for inheritance, and which can therefore spread through populations at a greater rate than genes with standard Mendelian inheritance [8]. They can be generated in organisms that reproduce sexually (e.g., mosquitos for malaria control) or asexually (e.g., bacteria [E. coli] and viruses [Human cytomegalovirus (HCMV)]), and may have potential for infectious disease control and modifying treatment resistance [9].

The Australian Government Department of Health and Aged Care is consulting on a draft National Gene Drive Policy Guide [8]. The Guide is not designed to add additional regulatory complexity or to deregulate any of the mandatory requirements needed to manage any risks The Australian Government Department of Health and Aged Care is consulting on a draft National Gene Drive Policy Guide [8]. The Guide is not designed to add additional regulatory complexity or to deregulate any of the mandatory requirements needed to manage any risks posed by genetically modified (GM) gene drive organisms. Instead, the aim of the consultation is to improve the Guide for potential users of gene drive technology. [8] 

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Misinformation on the regulation of mRNA vaccines

The Office of the Gene Technology Regulator (OGTR) has published a statement addressing misinformation on the regulation of mRNA vaccines. [10]

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INTERNATIONAL REGULATORY UPDATES RELEVANT TO AUSTRALIA

& NEW ZEALAND

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FDA

The FDA recently published guidances on Potency Assurance (Draft) [11], Considerations For The Development of CAR T-Cell Products (Final) [12], and Human Gene Therapy Products Incorporating Human Genome Editing (Final). [13]

The FDA is Investigating Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies. [14] It has also asked a number of companies to add a serious warning on the label of their cancer therapies that use CAR-T technology [15].

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RELEVANT ARTICLES, WEBINARS & PAPERS

Opinion piece on unproven stem cells. [16]

ISCT webinar on Getting your cell therapy product approved in Australia - some Dos and Don'ts on 9 February 2024 from 12:00 to 13:00 (AEST). [17]

Analysis of conditional approval pathway in Japan. [18]

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REFERENCES

1. Australian Government Department of Health and Aged Care, Inquiry into approval processes for new drugs and novel medical technologies in Australia, Publication date 30 November 2023, https://www.health.gov.au/resources/publications/inquiry-into-approval-processes-for-new-drugs-and-novel-medical-technologies-in-australia?language=en

2. TGA, GMP clearance sponsor information dashboard and guidance updates, Date published 12 December 2023, https://www.tga.gov.au/news/notices/gmp-clearance-sponsor-information-dashboard-and-guidance-updates  

3. TGA, GMP clearance Sponsor Information Dashboard (SID), Date published 11 January 2024, https://www.tga.gov.au/how-we-regulate/manufacturing/gmp-clearance-sponsor-information-dashboard-sid

4. TGA, GMP clearance guidance, Version 18.4, November 2023, Last updated 11 December 2023, https://www.tga.gov.au/resources/resource/guidance/gmp-clearance-guidance

5. TGA, International agreements and arrangements for GMP clearance, Last updated 12 December 2023, https://www.tga.gov.au/how-we-regulate/manufacturing/manufacture-medicine/good-manufacturing-practice-gmp/international-agreements-and-arrangements-gmp-clearance

6. TGA, GMP clearance: Mutual Recognition Agreement (MRA) and Compliance Verification (CV) pathway updates, Published 12 December 2023, https://www.tga.gov.au/news/notices/gmp-clearance-mutual-recognition-agreement-mra-and-compliance-verification-cv-pathway-updates

7. TGA, TGA-Industry Working Group on Good Manufacturing Practice (GMP) (TIWGG), Last updated 10 December 2020, https://www.tga.gov.au/about-tga/advisory-bodies-and-committees/tga-industry-working-group-good-manufacturing-practice-gmp-tiwgg

8. Australian Government Department of Health and Aged Care, Consultation on a draft National Gene Drive Policy Guide, https://consultations.health.gov.au/best-practice-regulation/consultation-on-a-draft-national-gene-drives-polic/, Opened 2 Jan 2024, Closes 3 Mar 2024

9. O’Sullivan, Philips, Dornbusch, Rasko JEJ (2022) 20 Years of Legislation - How Australia Has Responded to the Challenge of Regulating Genetically Modified Organisms in the Clinic. Front. Med. 9:883434. doi: 10.3389/fmed.2022.883434, https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9127347/#:~:text=Risk%20Assessment,of%20its%20unmodified%20parent%20organism.

10. OGTR, Addressing misinformation on the regulation of mRNA vaccines, Publication date:

December 2023, https://www.ogtr.gov.au/resources/publications/addressing-misinformation-regulation-mrna-vaccines

11. FDA, Potency Assurance for Cellular and Gene Therapy Products (DRAFT), December 2023, https://www.fda.gov/regulatory-information/search-fda-guidance-documents/potency-assurance-cellular-and-gene-therapy-products

12. FDA, Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products (FINAL), January 2024, https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-development-chimeric-antigen-receptor-car-t-cell-products

13.       FDA, Human Gene Therapy Products Incorporating Human Genome Editing (FINAL), January 2024, https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing

14. FDA, FDA Investigating Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies, Published 28 November 2023, https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-serious-risk-t-cell-malignancy-following-bcma-directed-or-cd19-directed-autologous

15. G Rajan, G Mehar, Reuters, US FDA seeks 'boxed warning' for CAR-T cancer therapies, Published 24 January 2024, https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-requires-boxed-warning-car-t-cancer-therapies-2024-01-23/

16. P Foong & A Leung, Consumers should beware of stem cell treatments for Covid-19, BioEdge, Published 4 January 2024, https://bioedge.org/stem-cells/consumers-should-beware-of-stem-cell-treatments-for-covid-19/?fbclid=IwAR0orEElIlZRWix7iiimkDj0YTU81pGH7qf8-dLiYpzMFP2C8y3_-xc0WGw

17. ISCT, Getting your cell therapy product approved in Australia - some Dos and Don'ts, Feb 9, 2024 from 12:00 to 13:00 (AEST), https://www.isctglobal.org/events/event-description?CalendarEventKey=97531cb9-9ac7-4459-a0ad-018cd63bbdbf&Home=%2fhome

18. Cyranoski D, Sipp D, Mallik S, Rasko JEJ. Too little, too soon: Japan's experiment in regenerative medicine deregulation. Cell Stem Cell. 2023 Jul 6;30(7):913-916. doi: 10.1016/j.stem.2023.06.005. PMID: 37419104. https://pubmed.ncbi.nlm.nih.gov/37419104/