2022 Repurposing medicines

Closed 1 Apr 2022

Opened 10 Mar 2022

Feedback updated 12 Aug 2022

We asked

The 2022 follow up consultation sought stakeholder input into four core challenges for the repurposing of medicines:

  • address commercial and intellectual property issues;
  • identify the best candidates for repurposing;
  • prioritise candidates; and
  • approach and incentivise the market.

You said

Twenty-seven (27) responses were received from four key stakeholder groups: patients and patient groups, health professionals, academia, and the pharmaceutical industry. The feedback we received can be summarised in four areas.

Overcoming commercial constraints and addressing intellectual property issues

  • Incentives will need to counter costs involved in preparing a submission, fees, the burden of post-market surveillance and supply guarantee obligations.
  • Collaborative submissions would require clear, fair, and appropriate incentives for all involved. Responsibilities and liabilities would need to be clearly determined. The Department will also need to consider anti-competition issues.
  • Clarity regarding data sharing and ownership, regulatory, manufacturing, and operational responsibilities, as well as cost sharing will be required when repurposing off-patent medicines.
  • Extension of data or market exclusivity regimes to the repurposed indications may discourage ‘game play’ and strategic timing of new indications to maximise market monopoly through ‘rolling’ periods of data exclusivities.

Identifying potential candidates

  • The Department should make patient voice a priority.
  • Rare diseases and unmet need should be focus areas.
  • Candidates may be identified in current clinical practice documentation such as clinical guidelines and Standards of Care.
  • National and state organisations such as the Council of Australian Therapeutic Advisory Groups (CATAG), as well as specialist medical colleges and compassionate programs may be able to identify potential candidates.
  • Involvement of hospital networks and pharmacies in sourcing pharmacy compounding records, retrospective medication charts and patient records may enable a demand driven model for identifying potential candidates.
  • Horizon scanning for new products that might enter the market and meet unmet patient needs should be conducted in parallel with the repurposing process.

Prioritising candidates

  • Real-world data insights should be considered early in the process of prioritisation.
  • Candidate medicines that have the largest impact on patient care and quality of life should be a priority.
  • An expert advisory panel with representation from all stakeholders and expertise in all the different facets of the repurposing process should be responsible for prioritising candidates.
  • Safety, efficacy, commercial feasibility, and other factors should be considered early in the process for efficient use of time and resources.
  • Clear guidance and criteria should be set by the Department.
  • If the Department chooses to set priority therapeutic foci, National Health Priority Areas (NHPA) should be considered, with flexibility where unexpected changes arise due to shortages, commercial decisions or delisting.
  • The TGA could publish a list of indications prioritised for repurposing to attract sponsors of off-patent product who have interest and capability to pursue a new indication even if they do not currently sponsor the candidate product in Australia.
  • The current EMA pilot of repurposing medicines could be a model for the prioritisation of candidates in Australia.
  • The Government should be transparent about how the decisions are made, which indications may be considered, and the outcomes of applications.

Encouraging sponsors to apply for regulation and reimbursement

  • Fee waivers and exclusivity periods of sufficient length (e.g., up to 5 years) may provide adequate financial returns to cover the ongoing cost of pharmacovigilance activities and financial and legal obligations for local sponsors.
  • A streamlined process with simpler data requirements would reduce the burden on sponsors of applying for the repurposed indication.
  • Detailed guidance on how the TGA and PBAC will evaluate repurposing applications would be beneficial.
  • Active negotiation such as pre-submission meetings between sponsors and government as well as parallel evaluations by the TGA and PBAC may help clarify minimum standards for approval, garner mutual commitment and expedite the process.
  • An initial feasibility assessment of an acceptable price would allow sponsors to explore commercial incentives, and consider international pricing risks.
  • PBS exclusivity rather than regulatory exclusivity may be attractive to some sponsors.
  • Sponsors should be allowed ample time (e.g. up to 3-4 months) to consider their interests and decide to take on a repurposing application.

We did

The Department is now using this and other feedback to shape regulatory reforms and policy. Consultation with internal and external stakeholders continues. Implementation of some options may require government approval (regulatory change) and/or parliamentary approval (legislative change), and the Department will present these to the Government for consideration.

Published responses

View submitted responses where consent has been given to publish the response.


Prescription medicines have been used ‘off-label’ for a given indication for many years to treat conditions or uses that have not been registered in Australia. Whilst this is often aligned with accepted clinical practice, it can lead to patient inequity as it is applied in an ad hoc manner at the discretion of individual doctors. It also can involve significant medicolegal risk in cases where the particular off-label use is not generally accepted in clinical practice. New uses are not always supported by evidence and the off-label use may not be reimbursed meaning the off-label use may be uneven.

Obtaining regulatory approval and registration on the Australian Register of Therapeutic Goods (ARTG), addresses medico-legal concerns and increases patient and medical confidence which will lead to wider and safer use, and improve the equity of access to medicines. To seek reimbursement through the Pharmaceutical Benefits Scheme (PBS) listing also requires formal registration of the indication on the ARTG. Repurposing medicines has the potential to remove some of these barries and improve the access of patients to the medicines they need.

For a sponsor to market a new therapeutic use for an existing medicine in Australia, they need to seek regulatory approval for an ‘extension of indication’. It is common to extend an indication to other populations such as from adults only to paediatric or adolescent patients. Extending indications is a relatively common and successful paradigm in expanding access to patients and the commercial value of new drugs.

In general terms, there are two types of indication expansion:

‘Cascading’ extension of indications:

  • Changes to population (e.g., expansion to include adolescents).
  • Use of a medicine for a closely related condition (e.g., use of an oncology medicine for a related tumour type or for the same tumour type in a different organ.)

Novel clinical use

  • May involve a different body system.
  • May be pharmacodynamically different.

An extension of indication typically involves a high administrative burden and significant costs for the sponsor. Therefore, most regulatory and reimbursement applications for extension of indications occur whilst the medicine remains on-patent and the original developer or licensor of a medicine can benefit from their intellectual property. The obstacles of seeking the extension of indications are countered by the potential revenue. After patent and/or data protection ceases sponsors are less likely to seek the extension as price reductions reduce the incentive for an initiating sponsor. The return-on-investment is expected to be low or even absent for medicines that are out of basic patent and regulatory protection.

The process of extending indications is also known as ‘repurposing’. For this consultation the focus is the process of marketing authorisation for novel clinical uses of existing medicines registered on the ARTG, rather than the commonly used practice with certain on-patent medicines of extension of indications to related populations. The intent of consultation is to establish options to reduce barriers and identify incentives for medicines to be repurposed for novel clinical uses. There is a particular focus on off-patent medicines, although opportunities for repurposing on-patented medicines also exist.

Targeted medicines

The Department is aiming to improve the environment to encourage the repurposing of medicines, with a primary focus on novel clinical uses of proven safe medicines.

To be considered for repurposing the medicine must:

  • Have a history of safe usage within Australia;
  • Be a prescription medicine that is registered on the ARTG or has formerly been registered on the ARTG (but not cancelled through safety concerns);
  • Have limited commercial viability – not part of a commercial planned extension of indications program.

It is recognised that different approaches for identification of candidates for repurposing may be required for on-patent (innovator) and off-patent (generic) medicine.

It will also be important that any process developed to support the repurposing of medicines for a novel indication does not disrupt the existing, active process of extension of indications (particularly of on-patent medicines). Since 2015, over 230 medicines have had their indications extended on the ARTG.

How to respond?

The Department welcomes feedback on the options presented in this paper and encourages alternative suggestions that may assist. The consultation hub poses questions to encourage feedback within the tool. Stakeholders are also welcomed to provide more specific responses and attach a separate response document if required.

The information presented in this survey can be downloaded below, and you can upload a response document (if required) on the final page.


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