Creating a Phase 2 Trials Platform for Obesity Therapies in the UK

BACKGROUND

New pharmacotherapies and digital technologies are leading to radical improvements in the management of obesity, and there is a promising pipeline of candidate interventions in development. The challenge for UK science is to develop an environment for testing these therapies and technologies that are attractive to both the life sciences industry and those living with obesity.

AIM

To collaborate with industry, academia, the NHS and other stakeholders to design a UK phase 2 trials platform to test new obesity therapies and technologies in adults and children, harnessing the UK strengths and addressing current barriers. OBJECTIVES

O1) Map the key needs in organisations that commission phase 2 trials for obesity therapies and technologies, understand the unique selling points and barriers of undertaking research in the UK, and agree on optimal approaches to contracting industry-funded research through academia and the NHS.

O2) Engage people living with obesity and relevant stakeholders to understand how recruitment channels, retention, and patient experience can be optimised in obesity trials. O3) Design a trials platform incorporating advanced methods and addressing key agreed outputs from O1 and O2. METHODS Based on pre-submission engagement work, we have designed two work packages (WPs).

WP1: Engagement (Month 1-12):

There is uncertainty as to where a trials platform could be optimally positioned in the UK. Early engagement work supported several solutions, ranging from a trials platform to support streamlined contracting and master protocol with core outcomes including patient reported outcomes, through to incorporating dose finding, combination therapies and novel adaptive features through innovative trial design.

The first stage of WP1 will be to better understand an approach that best meets the needs of people with overweight/obesity, the life sciences industry, and other stakeholders. This will be addressed through workshops with: 1) patients and public; 2) industry, academic and clinical representatives; 3) regulatory and senior research and data governance representatives from academia and the NHS.

The most appropriate outline format will be agreed based on findings across the workshops, before a one-day symposium to understand stakeholder views on how to optimise and operationalise this platform. WP2: Trial design (Month 3-12):

We will develop the platform based on the findings from WP1. The initial proposed model will support a standardised approach to conducting multiple individual phase 2 randomised controlled trials. A master protocol will be developed with a core set of primary (weight change) and secondary outcomes, and an optional set of standardised secondary outcomes that allow greater therapeutic insight.

We will work closely with methodological experts and statisticians to explore the incorporation of novel adaptive features where indicated by the findings of WP1. ANTICIPATED IMPACT AND DISSEMINATION

A phase 2 weight management trial platform could increase efficiency of set up/delivery processes, accessibility of trial participation for underserved populations, and commercial appeal of conducting industry-funded phase 2 trials obesity trials in the UK.

We will publish findings from the workshops and symposium, and invite further discussion. We will also disseminate our learnings to inform wider stakeholders designing similar platforms in other conditions and clinical trial phases.