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Platform studies to efficiently evaluate the efficacy of multiple interventions specification document


Published: 29 November 2023

Version: 1.0 - November 2024

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MRC-NIHR funding collaboration

The MRC-NIHR Efficacy and Mechanism Evaluation (EME) programme is inviting applications for phase 2 platform studies. The study must aim to efficiently evaluate the efficacy of multiple interventions, in a defined patient group or circumstance (which may include sub-groups), where there is a pipeline which justifies a platform approach.

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 projects 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.

Through this call, the programme hopes to fund multiple platforms. Collectively, these projects should also aim to enhance learning around the potential efficiencies, challenges and conduct of platform designs.

Call Scope

Applications to this call must describe a phase 2 platform study seeking to evaluate a number of predetermined technologies. Each technology must have sufficient clinical, human proof of concept, such that the proposed trial arms are each within remit for the EME programme. The platform must aim to provide appropriate efficacy data to inform any subsequent clinical and cost effectiveness trials.

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.

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. A clear process and Independent Scientific Advisory Group for the identification and prioritisation of future candidate interventions should be described. Applicants may also wish to describe the anticipated funding model for sustainability of the platform.

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.

The proposed study must be multi-centre and collaborative. Applications are encouraged which include recruitment from geographic populations with high disease burden which have been historically underserved by research activity.

The successful platforms are expected to contribute to the building of experience and capacity for research in their area. Applications should demonstrate how the trial will enable early-career researchers to gain experience in the conduct and leadership of clinical trials. 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.

Applications with international collaboration are welcome where this will benefit the study (but are not essential), for example by enabling recruitment in rare disease or phenotypes. Applicants including an international element should note the following:

  • The application must be multi-centre and UK-led.
  • The EME Programme is typically not able to provide funding for international recruitment. Applicants will be required to demonstrate that consideration has been
    given to the funding of any recruitment outside of the UK, where appropriate.
  • The benefit of international recruitment will require justification, including whether this is in a similar health and healthcare context, generalisable to the UK.
  • For more information, please read our International Funding guidance.

In 2022, the EME programme launched an Application Acceleration funding opportunity to support the development of applications in this area. Applications developed through these awards may be submitted to this call. The call is also open to applications for other platform studies, for which development work has been completed and the team is ready to apply for the full study.

Duration and costs

Applications must detail project costs and duration according to the initial interventions proposed.

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.

Unless otherwise invited, applications to the MRC-NIHR EME Programme for additional funding to add arms will be considered as requests for a ‘Variation to Contract’, which should be limited in scale as a proportion of the overall cost. Substantial requests will be considered alongside, and in competition with, other applications by the EME Funding Committee.

There is no set upper limit for the duration or cost of applications, however value for money will be a key consideration.

Process for Assessment of Applications

Applications to this call will follow a 2 stage application process.

The deadline for Stage 1 applications will be: 23rd April 2024