Introduction
Obesity arises from a complex web of genetic, biological and environmental causes, resulting in; ill health, increased risk of developing disease such as cancer, heart disease and Type 2 Diabetes for people affected by it and leading to reduced quality of life and life expectancy.
In addition to the significant burden placed on individuals, at a system level the pressures from overweight and obesity related conditions are significant – with cost estimates of over £6bn each year to the NHS and over 1 million obesity related hospital admissions in 2021/22 alone.
This issue is recognised as one of the greatest current and future health challenges faced in UK, as articulated in the Life Sciences Vision (2021) and Tackling Obesity government strategy (2020). The launch of an obesity healthcare mission in 2022 backed by £20 million in funding, and the further announcement of an additional £40 million for implementation pilots that will explore how approved, innovative weight-loss medications can be made safely available to patients living with obesity outside of hospital settings, further demonstrates the government’s commitment to harnessing and supporting innovation in this space. It is the intention of the obesity mission to ensure promising interventions are fast-tracked towards patient benefit and that the UK is seen as the global destination of choice for Obesity research and development.
Globally, there is an active industry pipeline of Obesity innovations spanning pharmaceutical, MedTech and digital technologies, with the pharmaceutical market alone forecast to grow in worth to $54bn by 2030. These innovations have the potential to profoundly change the clinical management and treatment of obesity, but there is a need to accelerate promising products through the pipeline. A key component of this pathway, and the focus of this call therefore, is the speedy, high-quality testing of efficacy in a robust clinical trial, once there is clinical, human ‘proof of concept’.
It is the ambition of the MRC-NIHR Efficacy and Mechanism Evaluation Programme (EME) to fund a project which will establish the phase 2, pre-licensing platform architecture to test multiple pharmaceutical interventions and may provide equivalent evidence for digital interventions for obesity. The platform will be UK-led and will aim to simultaneously test multiple different technologies to identify if and how these products, and combinations of such products where intended by design, can be used in real world settings to support sustainable weight loss. The platform will also be able to adopt new technologies as they mature to the appropriate point, thereby supporting the development pipeline.
Obesity is recognised as a high priority and major healthcare challenge internationally. It is therefore important that investment and the evidence generated from clinical trials is relevant as widely as possible. The UK has a diverse and representative population, accompanied by strong clinical research and data infrastructure to deliver trials at scale. As reported in the Life science competitiveness indicators 2022, the UK has a leading reputation in producing high-quality and impactful research in medical sciences publications. It is therefore anticipated that the UK would represent a strong global location for a platform which enables industry to efficiently generate the evidence and documentation required to accelerate the progress of assets targeting obesity.
We recognise that developing an application for a phase 2 platform represents a complex bid, which will require time and the navigation of specific challenges. Of particular importance for the proposed obesity platform, it will be necessary to work closely with industry stakeholders to explore industry needs and critical issues, solutions to barriers to the inclusion of industry assets, and awareness of the efficiencies and benefits of a platform. The programme is therefore inviting applications for work which will accelerate the necessary learning and preparation to apply for the platform. This will take the form of an Application Development Award (ADA).
It is anticipated that teams may require up to £200,000 over a maximum of 12 months.
The Application Development Award must deliver the following key outputs:
- Delivery and write-up (including a slide set summary) of an Industry Symposium involving key stakeholders, details below.
- Development of a Post-Development Application to EME for an adaptive Phase 2 Platform Study, which seeks to evaluate specific interventions at the outset and may form the infrastructure for the evaluation of future interventions as these mature. Details below.
- A written summary of the project.
Please note, only the team(s) awarded an Application Development Award under this call will be invited to apply for the Platform Study.
Call scope
Applications to this call must aim to accelerate the development of an application for a platform study to evaluate pharmaceutical and/or digital interventions and technologies, individually or in combination, to support sustainable weight loss for people with overweight and obesity (applicants to define and justify specific inclusion criteria, which may include sub-groups with obesity-associated co-morbidities). Digital interventions needn’t take place at the outset of treatment; for example, digital interventions to sustain weight loss after pharmaceutical treatment may also be within scope. This platform must be positioned as a Phase 2 study, within remit of the MRC-NIHR EME Programme.
Applicants should provide details regarding the anticipated platform, including the study team, research design and governance structures where known, however full details are not required at this stage and some elements may be defined as part of the application development work.
Applications to this call must define and justify the specific challenges which will be addressed within the award and how they will ultimately enable a complete application to be made. Examples of the types of work which may be supported include, but are not limited to:
- Developing networks or collaborations to support the set-up and delivery of the platform.
- Overcoming barriers to rapid start-up of the platform
- Identifying potential technologies to be evaluated and important collaborations to establish
- Addressing challenges relating to industry assets entering the platform
- Targeted work to clarify the ‘signal of efficacy’ for technologies to be included at the outset.
- Further understanding the anticipated effectiveness of the platform study design, including learning from previous platform studies.
- Defining and agreeing a study protocol that may be followed across all collaborating centres.
- Defining and validating elements of the full study design, such as the comparator, sample size or most important health outcome measures.
- Establishing the feasibility of a study in the proposed setting, including Patient and Public Involvement (PPI).
- Work to ensure the proposed platform follows need and recognises health disparities, including reaching disproportionately affected and under-served groups.
Applicants must articulate what the valuable outputs of the award will be, including any generalisable learning around the planning, conduct and effectiveness of platform studies. These will include:
An Industry Symposium
This event should take place within the first 4 months of the award starting. The focus should be on critical issues around industry engagement with a phase 2 platform, aiming to identify solutions to barriers for platform trial entry for industry assets.
Key stakeholders are likely to include:
- life sciences industry actively developing potential candidate pharmaceutical or digital technologies, at a pre-licence stage
- regulatory and licensing experts
- academic and clinical researchers
- representatives from the NIHR Intellectual Property Unit
The symposium should conclude with a written report, which will include learning specific to the obesity platform and may include generalisable learning across platform designs or other phases of obesity research. The team will be expected to work with the NIHR Secretariat to coordinate the review, approval, and dissemination of this report.
Post-Development Application
A Stage 1 application must be submitted to the EME programme by the given deadline, approximately 10 months after commencing the award (date to be determined).
The application will be required to:
- Detail a phase 2, pre-licensing UK-led platform study within remit of the EME programme. For this call, a platform study is defined as a single award which adaptively and simultaneously evaluates more than one intervention or technology with common infrastructure.
- Include a number of defined pharmaceutical and/or digital interventions at a pre-licensing stage to be evaluated at the outset. The study design should evaluate the combination of interventions as appropriate, where this is intended by design.
- Build on industry engagement, such that the platform is positioned to support the pipeline of potential candidate interventions. The application must detail an adaptive design, allowing for interventions to be dropped and new candidates to further populate the platform as technologies mature.
- Define and justify inclusion and exclusion criteria for the platform, for example, whether individuals with overweight or multimorbidity may be included. Applicants may also wish to consider the generalisability of findings to obesity associated with specific conditions.
- Detail a process with independent oversight for the identification and prioritisation of new technologies and study arms to be incorporated, as part of the adaptive design.
- Demonstrate that the proposed research will focus on areas where obesity rates and health disparities are highest.
- Take account of further application guidance and feedback arising throughout the application development award, e.g. reflections on the industry symposium.
The research team must:
- Demonstrate experience in delivering multi-centre interventional studies in the UK. Experience of innovative adaptive designs is likely to be particularly relevant.
- Demonstrate a track record in working with members of the public, people who draw on services and in locations of greatest need to perform inclusive studies.
- Evidence ability to work effectively with industrial partners and deliver robust, independent and industrially relevant research
- Evidence ability to establish and coordinate data input from a wide variety of sites.
A written summary of the project
Given the anticipated cost and duration of the award and other key outputs, only a proportionate written report is required.
Successful ADA applicants will be expected to produce a single Research Article of the core work, two weeks after the contract end date, for publication in the Efficacy and Mechanism Evaluation journal as part of the NIHR Journals Library.
If an ADA team is successful for the next phase of the research, the ADA research article will feature in the final issue of the award via the NIHR Journals Library, Threaded Publication approach.
In addition, NIHR supports teams to produce audience-appropriate outputs, such as a variety of digital formats (e.g., podcast, video, social media) including related articles to third-party journals, but the publication of the Journals Library article cannot be delayed to accommodate this.
Out of scope
This call is for application development work only. An application for the full platform study will be invited from the successful team.
Diet and exercise interventions are not considered to be in scope for the platform. Study teams may need to undertake work as part of the ADA to define standard care as a comparator, to include diet and exercise.
Application Development Award Process
To enable quicker funding decisions, this is a straight to Stage 2 (single stage) funding opportunity.
A key deliverable within this award must be the development of a Stage 1 Post-Development Application. This will represent a milestone for the award and will likely be in September 2024. This application may recognise areas of continuing work within the Application Development Award.
Any shortlisted Post-Development Application will be invited to submit a Stage 2 application.