Regulation of advanced therapies

Advanced therapies or advanced therapy medical products is a term used to describe innovative therapies. International regulators use this term to include gene, cell and tissue therapies.

The TGA uses the following definition for advanced therapies:

1. Gene therapies

   1. the substance is used in or administered to human beings to regulate, repair, replace, add or delete a genetic sequence

      AND

   2. the substance is involved in the therapeutic, prophylactic, or diagnostic effect of the product
2. Gene modified cell therapies
3. Cell and tissue therapies that:
   1. are not devices
   2. have been classified as class 3 or 4 biologicals.
4. Either a or b in combination with a device

Regulation of advanced therapies

The TGA regulates therapeutic goods consisting of gene, cell and tissue therapies under the Therapeutic Goods Act 1989 (the Act).

Any biological or prescription medicine that involves genetic modification must also be approved by the Office of the Gene Technology Regulator (OGTR). OGTR approval is needed before TGA approval. OGTR applications should be done before or concurrently with an Australian Register of Therapeutic Goods (ARTG) applications.

Advanced therapies regulated as biologicals

We regulate gene therapies that involve ex vivo manipulation of human cells as biologicals under section 32 of the Act. For example, CAR-T cells.

Human cell and tissue-based therapeutic goods, and live animal cells, tissues and organs are also regulated as biologicals.

Information on the regulation, supply and use of biologicals is available on the Biologicals web page.

Advanced therapies regulated as prescription medicines

We regulate therapies that involve in-vivo genetic manipulation of human cells as prescription medicines under section 23 of the Act. This includes small silencing RNAs, CRISPR and other gene editing technologies, and gene therapies administered by vectors.

Information on the regulation, supply and use of prescription medicines is available on our prescription medicines web page.

Submissions for advanced therapies

There is a dedicated section for gene therapy medicines in the TGA Prescription Medicines Authorisation Branch. For more information, contact the streamlined submission team.

For more information about cell and tissue product submissions, contact the TGA biologicals section.

Pre-submission meetings

The TGA encourages pre-submission meetings for advanced therapies to help address technical questions. We can help you with the documentation required for regulatory approval but are unable to offer advice about product development.

Please use the contact details below to ask for a pre-submission meeting.

The TGA does not make decisions to fund advanced therapies. Funding approvals need a submission to the Australian Government Department of Health. However, you may ask for a joint pre-submission meeting with OGTR, Technology Assessment and Access Division and the TGA.

European Medicines Agency (EMA) guidance

Sponsors should refer to the following EMA guidance on Advanced Therapies:

• ICH Q5D (pdf,189kb)
  Derivation and characterisation of cell substrates used for production of biotechnological/biological products
• ICH Topic Q 5 B (pdf,133kb)
  Note for guidance on quality of biotechnological products: analysis of the expression construct in cell lines used for production of r-DNA derived protein products
• EMA/CAT/852602/2018 (pdf,559kb)
  Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials
• EMEA/CHMP/SWP/28367/07
  Guideline on strategies to identify and mitigate risks for first-in-human and early clinical trials with investigational medicinal products
• EMA/CAT/GTWP/671639/2008 (pdf,496kb)
  Quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells
• EMEA/CHMP/410869/2006 (pdf,400kb)
  Guideline of human cell-based medicinal products
• EMA/CHMP/BWP/271475/2006 (pdf,127kb)
  Guideline on potency testing of cell based immunotherapy medicinal products for the treatment of cancer

Clinical trials

Clinical trials performed in Australia that involve 'unapproved therapeutic goods' including gene, cell and tissue therapies, need a Clinical Trial Notification (CTN) or a Clinical Trial Approval (CTA). The CTN scheme is a notification pathway. The CTA scheme involves an evaluation of limited scientific data by the TGA, with a focus on safety.

For more information about the CTN and CTA pathways refer to the Australian clinical trial handbook.

Certain class 4 biologicals must be approved under the CTA scheme. The following guidance is to help sponsors, in collaboration with a Human Research Ethics Committee (HREC), to decide which scheme is the most appropriate:

• 'Choosing between the CTN and CTA schemes' section of the Australian clinical trial handbook
• Which clinical trial scheme should I choose?

Sponsors should contact us for pre-submission advice before submitting a CTA scheme application.

The TGA does not give scientific advice in relation to the clinical development program. However, we can provide pre-submission meetings and advice to clarify the regulatory requirements and what data you need in your submission.

Contact details for advanced therapies

For biologicals

For medicines

For clinical trials