Specification
This call is for either Phase 2 or Phase 3 focused platforms, where there is a pipeline of technologies which justifies a platform approach. This call will not support funding for studies aiming to run platforms seamlessly from phase 2 into phase 3.
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 which suggests they may be efficacious. By robustly evaluating efficacy, 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 which have demonstrated some clinical efficacy but which require further testing in a multi-centre, pragmatic trial to compare against current best practice.
Interventions must have an appropriate level of supporting evidence for the phase of the platform, although it is recognised that there may be some variation in the amount of evidence for the interventions across a platform.
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 which do not show any positive effect.
A platform approach may be justified where:
There are multiple technologies requiring evaluation, with others likely to arise within the anticipated lifetime of the platform.
Relevant outcomes/endpoints may be observed in the short- and medium-term, allowing the platform to adapt and focus on the most promising interventions.
A collaborative, multi-disciplinary team and multi-centre recruitment plan can support the delivery of a platform.
It is envisaged that the project(s) awarded from this call 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 which 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.
Scope
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.
Although applications must set out to evaluate predetermined interventions, applicants should describe an adaptive protocol, able to discontinue study arms as appropriate and incorporate additional interventions as candidates are identified. Applicants must demonstrate that there is a pipeline of interventions which justifies a platform approach. Specifically, that further candidates are in development and are likely to be evaluated for a ‘signal of efficacy’, such that they may be considered for evaluation through the platform. Phase 3 platform studies should aim to evaluate the clinical (and cost) effectiveness of multiple interventions, in a defined condition or area so evidence is aligned to the needs of clinicians and policy makers and will ultimately contribute to changes in practice and policy.
A clear process and Independent Scientific Advisory Group for the identification and prioritisation of future candidate interventions should be described. Applicants should consider the anticipated funding model for sustainability of the platform.
Existing platforms are not eligible to apply for further funding under this call, and should apply through other existing Medical Research Future Fund (MRFF) National Health and Medical Research Council (NHMRC)
The proposed study must be multi-centre and collaborative. Recruitment must be split across both countries but needn’t be apportioned equally, with a minimum of 20% of the total number of participants being recruited in each country. Further international recruitment involving other countries may be proposed, where justified, however the MRFF, NHMRC and NIHR are not able to pay site and recruitment costs for these countries through this call.
Within each jurisdiction, applications are encouraged which include recruitment from geographic populations with high disease burden which have been historically underserved by research activity. The involvement of consumers including patients and the public must be embedded throughout the research lifecycle from informing the research priorities and needs, the design of clinical trial plans and during the conduct of the clinical trial and dissemination of research findings. Applications are expected to evidence strong patient and public involvement and engagement in the development of the clinical study protocol particularly around the acceptability of the proposed treatment strategies and, the feasibility of recruitment. Applicants should clearly define and justify their inclusion/exclusion criteria, study design and outcome measures, and explain how the research fits within the scope of the call.
Applicants must ensure they meet eligibility requirements specified by their respective governmental funding bodies. Australian applicants must follow the MRFF and NHMRC Grant Opportunity Guidelines for this grant opportunity. These are available on GrantConnect
Research team
Applications must be jointly led, with an Australian Lead and United Kingdom Lead. For practical purposes (e.g. application portals, regulators, ethics) there must be one named ‘Lead Applicant’ (UK Lead) and one named lead collaborator (Australian Lead) listed on the NIHR UK application as a co-applicant.
The successful platforms are expected to contribute to the building of experience and capacity for research in their area. Applications should demonstrate how the study will support capacity building amongst Early and Mid-Career Researchers (EMCRs), through experience and training in the conduct and leadership of clinical trials. EMCRs will also become members of the NIHR Academy, with access to career development support such as leadership training and mentorship. Consideration should also be given to the potential of exchange visits between UK and Australian based EMCRs to facilitate knowledge and skills exchange.
Additionally, consideration must be given to a strategy that allows collaborators to join and exit the platform as appropriate, a publications plan that enables recognition of collaborators’ contribution, and the development of a succession plan to allow the leadership of the platform to change over time if sustained.
Duration and costs
The total funding available through this funding opportunity is in the region of $30 million AUD/ £15.5 million GBP with contributions from each funder.
for the Australian-based component the maximum grant amount that can be requested is up to $5 million AUD over 7 years, with a total contribution of up to $15m AUD.
for the NIHR UK-based component, there is approximately £2.6m per award over 7 years, with a total contribution of up to £7.8 million GBP.
MRFF, NHMRC and NIHR are aiming to fund 1 to 3 platforms for up to 7 Years. NHMRC and MRFF will fund the Australian research component and the NIHR will fund the UK research component of successful applications. Each national funder will support selected individual awards and there will be no shared or split funding arrangements between funders.
There is no set upper limit for the cost of applications for UK based components however, value for money will be a key consideration. The Australian-based components have a maximum amount of $5 million.
Studies are expected to incorporate predefined stop/go decision points, with measurable criteria to enable assessment of the successful completion of the stage. A reporting schedule agreed as part of the contracting process will assess completion of these critical contractual milestones during the lifetime of the award. Funders reserve the right to close studies not achieving critical milestones.
The successful platform(s) will be commissioned for up to 7 years, to evaluate the interventions set out at application. Due to the adaptive nature of platforms, it is anticipated that additional funding to support per-intervention costing may be required at a later date, to continue the platform. Applicants are encouraged to consider the sustainability of the platform beyond the initially funded work, where relevant, and how additional costs would be funded.
Applicants must separately detail the funding requested for research activities undertaken in Australia and funding requested for research activities undertaken in the UK. Costs for trials’ coordination activity may be included within costs for the country leading this activity.
The NIHR, MRFF and NHMRC wish to encourage and grow collaborations under this call. Costs relating to the exchange of personnel between international sites may be considered, where this is linked to a specific research activity, of clear benefit to the study and of a short duration.
A small proportion (up to 10%) of costs may relate to research services to be undertaken by co-applicants or collaborators in other countries, if teams can justify that suitable facilities are not available in Australia or the UK.
For more information, please refer to the funding opportunity guidance notes. Australian applicants must also review and follow the MRFF and NHMRC Grant Opportunity Guidelines to ensure that what they’re requesting funding for is eligible and the requests adhere to specific MRFF and NHMRC requirements (e.g. salaries funded through Personnel Support Packages). These are available on GrantConnect
General guidance
Governance and regulation
Applicants are asked to consult the following documents and follow them as appropriate:
Note that trials or studies involving medicinal products must comply with The Medicines for Human Use (Clinical Trials) Regulations 2006 and the amendment to the regulations. In these cases, the DHSC expects the employing institution of the chief investigator to be nominated as the sponsor. Other institutions may wish to take on this responsibility or agree co-sponsorship with the employing institution. The DHSC is prepared to accept the nomination of multiple sponsors. Applicants who are asked to submit a stage 2 proposal will need to obtain confirmation of a sponsor(s) to complete their application. The DHSC reserves the right to withdraw from funding the project if they are not satisfied with the arrangements put in place to conduct the trial. The MHRA info@mhra.gsi.gov.uk .
For Australian applicants
Australian personnel must comply with all relevant Australian laws and regulations in undertaking the project. Australian personnel must also comply with any specific legislation/policies/industry standards within the grant agreement, such as:
the MRFF Act the NHMRC Act working with Vulnerable People registration
state/territory legislation in relation to working with children
ethics and research practices
All personnel contributing to the research activity must familiarise themselves with the Australian Code for the Responsible Conduct of Research, the National Statement of the Ethical Conduct of Human Research, the Australian Code for the Care and Use of Animals for Scientific Purposes and other relevant NHMRC policies concerning the conduct of research, and agree to conduct themselves in accordance with those policies.
The Medical Research Future Fund (MRFF) Australia, the National Health and Medical Research Council (NHMRC) Australia and National Institute for Health and Care Research (NIHR) UK are inviting applications for ambitious platform studies in areas of unmet medical need. The platform must be internationally collaborative, with joint leads based in Australia and the United Kingdom. 