Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatment for PD through a multiarm, multistage (MAMS) platform trial approach.

Background: Funding and sustainability are most relevant in a MAMS trial given its long term, and potentially open-ended nature. Prospective funders for this type of work include charities, pharmaceutical companies, and government organisations.

Method: A Funding and Sustainability Working Group (WG) created as part of EJS ACT-PD aims to address the challenges associated with funding and sustaining a complex innovative platform trial in PD. The WG includes experts from various backgrounds (scientific, clinical, charities, Government organisations, clinical trial facilities, financial services), as well as Patient and Public Involvement and Engagement (PPIE) representatives. Apart from regular group meetings, the WG is arranging encounters with prospective funders to present the rationale for the project, understand funders’ priorities and discuss potential challenges to address before contemplating funding this type of project. The WG is supported by the Communications Subgroup, which is helping to create materials (written summary, videos, website) to inform of the need for a MAMS trial platform in PD.

Results: Provisional cost estimates have been calculated to provide ball park figures for funding requirements, and to highlight the potential cost savings in a MAMS platform approach in comparison to conventional trial designs. The best funding approach (e.g. individual treatment arms, consortium across funders) is being discussed with potential funders, and the proposed strategies stem from previous MAMS trials in other conditions. The long-term sustainability of the trial warrants an initial greater investment, for which rationale is found in the success and cost-effectiveness of previous MAMS trials.

Conclusion: Although challenging, this initiative is an opportunity to devise new and collaborative approaches to funding PD clinical research. Flexible funding that can support recruitment and follow up of one or more trial arms to Phase III, or be diverted to new trial arms to replace failed ones at the interim stage will be needed. Transparency and a continuous communication with stakeholders are key to success in this endeavour.

« Back to 2022 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/funding-and-sustainability-challenges-in-multi-arm-multi-stage-clinical-trials-in-parkinson-disease/