2022 Repurposing medicines

Closed 1 Apr 2022

Opened 10 Mar 2022

Overview

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.

Audiences

  • Anyone from any background

Interests

  • Hospitals
  • e-Health
  • Medicare
  • Legislation
  • Pharmaceutical benefits
  • Health insurance
  • Regulatory policy