Medicines Repurposing Program – supporting new uses for existing medicines

Off-label prescribing refers to prescribing that is not concordant with the indications, doses, routes of administration or patient groups included in the Australian approved product information.¹ It is increasingly common and arises because certain patient groups are excluded from preregistration clinical trials, or the commercial benefit for pharmaceutical companies (sponsors) does not offset the high cost of seeking approval for additional indications or patient groups after initial registration of a medicine. These issues, including the lack of subsidy for off-label prescribing on the Pharmaceutical Benefits Scheme (PBS), were discussed in an Australian Prescriber article in December 2023.¹

Following several years of consultation, in March 2024, the Commonwealth Department of Health and Aged Care announced the establishment of the Medicines Repurposing Program.² The program aims to improve patient access to medicines by financially supporting sponsors to apply for Therapeutic Goods Administration (TGA) approval and PBS subsidy for new (off-label) therapeutic uses for existing medicines. New therapeutic uses may include those already approved in other countries or accepted in clinical practice, and those for less common diseases or that are less commercially profitable.

The program focuses on off-patent medicines with an existing evidence base, demonstrated treatment or access gap, and clear public health benefits. Any person or organisation can nominate a medicine for consideration, provided the medicine is registered on the Australian Register of Therapeutic Goods and the new use being proposed is a distinct indication (either a different medical condition or a new patient group).³ The nominator should provide substantial evidence supporting the medicine’s proposed new clinical use. Before applying, nominators are encouraged to contact the existing sponsors to confirm there is interest in bringing this new use to market.

Without sponsor participation a medicine cannot progress through the program. The Medicines Repurposing Program liaises with existing sponsors to establish whether it is feasible to participate. The program also considers the evidence base to support the new use, such as clinical guidelines, scientific literature and approval by overseas regulators. It seeks advice from the Advisory Committee on Medicines to help prioritise nominated medicines for repurposing.

Sponsors of those medicines selected for repurposing have their application and evaluation fees waived for TGA approval and PBS subsidy. However, selection for repurposing does not guarantee TGA approval or PBS subsidy, and standard requirements still apply for these applications.

Medicines that are not selected for repurposing may be renominated in the future if new evidence is provided.

As with any new program, refinements are likely to be required as stakeholders engage in the program. The program is currently funded until June 2027.

 

References

1. Day RO. Ongoing challenges of off-label prescribing. Aust Prescr 2023;46:86-9.
2. Therapeutic Goods Administration. Establishment of the Medicines Repurposing Program. Department of Health and Aged Care; 2024. [cited 2024 Apr 11]
3. Therapeutic Goods Administration. Medicines Repurposing Program - Eligibility criteria and prioritisation considerations. Department of Health and Aged Care; 2024. [cited 2024 Apr 11]