title: Encouraging the regulatory evaluation of off-patent repositioned medicines in Australia
Creators: name: Perkin, Katrina
Publisher: name: The Australian National University
Year: 2025

description: Australia's National Medicines Policy (NMP) aims to ensure medicines meet agreed standards of quality, safety and efficacy. This is achieved when a prescription medicine is evaluated by the Therapeutic Goods Administration (TGA) to ensure benefits outweigh risks prior to approval. The use of a prescription medicine beyond approved limits is known as off-label use. Medicines prescribed off-label for different health conditions, usually due to a lack of effective approved treatments, are known as repositioned medicines. Without regulatory approval, prescription medicines are not eligible for Commonwealth Government cost subsidisation, making access unaffordable for some patients. There is limited understanding of how repositioned medicines are used across the Australian health system and what changes might be made to improve regulation and access. Informed policy responses rely on an assessment of the best available evidence and the production of intentional strategies for reform. This thesis reviewed and synthesised multiple sources of secondary data to explore ways to encourage the regulatory evaluation of off-patent repositioned medicines in order to create a novel policy model to improve regulation and access. An initial scoping review found that the extent of off-patent repositioned medicine use across Australia is greater than previously thought. In addition, state and territory funded patient access varies because safety, efficacy and cost effectiveness are evaluated at the local level rather than nationally. Analysis of regulatory processes and policies found that while regulatory pathways in Australia and comparable international jurisdictions permit the evaluation of off-patent repositioned medicines, complexity and cost limit use of these systems. In particular, the presence or absence of three key drivers (evidence generation, application submission and revenue generation) either facilitate or impede the regulatory evaluation of repositioned medicines. This means that in practice, when patent protection expires, a medicine sponsor is unlikely to seek regulatory approval for repositioned medicines due to effort and unrecoverable costs. Evidence shows that stakeholders in Australia are advocating for better access to repositioned medicines for patients and changes to the regulatory system to promote medicine repurposing. However, without the potential for revenue generation, sponsors of off-patent repositioned medicines are unlikely to invest in evidence generation and application submission to enable regulatory evaluation. The findings of the studies conducted enabled the development of a draft policy framework which aims to facilitate affordable patient access to safe and effective medicines. Through generation of the clinical evidence necessary to enable the regulatory evaluation of off-patent repositioned medicines, the policy framework removes barriers to the submission of clinical evidence in a regulatory evaluation application and creates avenues for revenue generation post approval. The utility of the draft policy framework was tested with experts via a modified Delphi consultation. Consensus was reached on characteristics of a policy model specifically designed to facilitate evidence generation, application submission and revenue generation for off-patent repositioned medicines. The novel policy framework translates key drivers of regulatory evaluation into practice in the Australian context. This thesis highlights the drivers of regulatory evaluation in order to identify the barriers specific to off-patient repositioned medicines. Strategic reforms that concurrently incentivise evidence generation, application submission and revenue generation could potentially improve affordable patient access to safe and effective off-patent repositioned medicines in Australia. The novel policy framework presented should help to improve the quality use of medicines and alignment with broader NMP objectives.