2022 Repurposing medicines

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.

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.

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.