Australia-UK Platform Studies in Areas of Unmet Clinical Need

Funding for joint UK-Australia platform studies (ie multi-arm studies to evaluate multiple interventions) in areas of unmet medical need where there is a pipeline of technologies that justifies such an approach.

Established in 2006 and primarily funded by the Department of Health and Social Care, the National Institute for Health and Care Research (NIHR) funds health and care research in the UK.

NIHR is partnering with the Medical Research Future Fund (MRFF) and the National Health and Medical Research Council (NHMRC) in Australia to deliver this new scheme for ambitious platform studies in areas of unmet medical need. The platform must be internationally collaborative, with joint leads based in Australia and the UK.

This call is for either phase 2 or phase 3 focused platforms, where there is a pipeline of technologies that justifies a platform approach. Phase 2 platforms should focus on establishing the efficacy of multiple interventions in a defined patient group or circumstance (which may include sub-groups). The study must focus on interventions with convincing human clinical proof of concept that suggests they may be efficacious. The study should have the potential to inform future phase 3 (comparative and cost-effectiveness) trials. Phase 3 platforms should aim to efficiently generate high-quality evidence around the clinical and cost-effectiveness of multiple interventions in a defined patient group or circumstance. The focus must be on candidate therapies that have demonstrated some clinical efficacy, but which require further testing in a multi-centre, pragmatic trial to compare against current best practice.

For this call, a platform is defined as a multi-arm study employing a master protocol and shared infrastructure to simultaneously evaluate multiple interventions. Arms may be terminated early, based on interim analyses, or added as new candidates are identified and mature. A platform should realise efficiencies in conduct and management, which may include statistical efficiencies due to a shared control group and early termination of arms that do not show any positive effect. It is envisaged that the successful project(s) will set up a platform to evaluate the current most promising candidates and will have the potential to adopt additional interventions at a later date through an adaptive protocol. 

For the purposes of this call, unmet medical need arises where individuals have a serious health condition where there are limited satisfactory options for prevention, diagnosis or treatment to support improved health outcomes. Applications are welcomed across all areas of health that meet these criteria. Areas of particular interest include, but are not limited to:

• Childhood, brain and prostate cancers.
• Stroke and traumatic brain injuries.
• Neurodegenerative diseases.
• Cardiometabolic conditions, including obesity.

These areas are identified on the basis of shared strategic interest; applicants are still required to provide justification for a platform approach.

Applications to this funding opportunity must describe a platform trial seeking to evaluate a number of specified technologies. Each technology must have sufficient clinical, human proof of concept to justify a definitive efficacy study.

Within each jurisdiction (the UK and Australia), applications are encouraged that include recruitment from geographic populations with high disease burden that have been historically underserved by research activity. The involvement of consumers, including patients and the public, must be embedded throughout the research lifecycle. Applications are expected to evidence strong patient and public involvement and engagement in the development of the clinical study protocol.