The European DREAMS project has recently formed two pivotal groups to advance the development of an adaptative clinical trial protocol for rare diseases. The goal is to accelerate research and improve access to innovative therapies for these rare conditions.
The European project DREAMS is dedicated to identifying new shared therapies using artificial intelligence to discover effective treatments for a specific set of five rare neuromuscular diseases (NMDs). This effort revolves around two core areas—managing cells, data, and algorithms, and validating predictions—and is complemented by a third focus, Towards Therapy, which is dedicated to developing an Adaptive Clinical Trial Protocol tailored to these conditions.
The objective of designing an innovative clinical trial is to adapt the study to the specific needs and unique circumstances of patients with rare diseases. This adaptive approach is intended to speed up the validation of new therapies and treatments, while effectively addressing the unique aspects of these conditions.
“As part of the DREAMS initiative, we aim not only to discover therapies but also to rethink the clinical trial process, striving to make it faster, more flexible, and more inclusive for patients with rare diseases,” explains Wael Khazen, Senior Clinical Project Manager at AFM-Téléthon. “This project leverages advancements in AI, drug repurposing, pharmacological screening, and stem cell technology to explore potential solutions for certain neuromuscular diseases”.
Role of the Patient Advisory Group and Expert Committee in Trial Design
A key component of this initiative is incorporating the perspectives and unmet needs of patients throughout the project. This will be accomplished through the active engagement of a newly established Patient Advisory Board (PAB) and the integration of advanced concepts into the novel clinical trial protocol.
PAB members will actively participate in reviewing essential study documents intended for submission to an ethics committee for patient sample collection. They will also select relevant Patient-Reported Outcome Measures to incorporate into the trial protocol, and evaluate both intermediate and final versions of the protocol to prioritize outcomes and endpoints that are meaningful to patients.
Furthermore, a Clinical Expert Committee has been created, coordinated by DREAMS’ partners AFM-Telethon and APHP, comprising at least 10 permanent members with the expertise necessary to develop the clinical trial protocol. This committee includes statisticians specializing in small population requirements, clinicians experienced in neuromuscular disease trials, patients and experts from other fields who bring valuable insights into basekt adaptive and seamless flexible trial designs.
“Incorporating the patient’s perspective into the DREAMS project is crucial because patients provide invaluable insights into their experience with the disease, helping to shape more effective and meaningful treatment,” explains Alexandre Mejat, Deputy Director – International Scientific Networks at AFM-Téléthon. By involving them at every step, from trial design to therapy development, we ensure that our solutions are not only scientifically sound but also aligned with the real needs and expectations of those affected by these rare diseases.”
Together, the PAB and the Clinical Expert Committee are working to advance the development of a groundbreaking clinical trial for rare diseases, with the ultimate goal of accelerating research and expanding access to new therapies for these rare conditions.
About AFM-Teléthon
AFM-Téléthon, a nonprofit patient organization focused on ensuring patient engagement, regulatory compliance, policy involvement, and public dissemination, is a key partner in the DREAMS project. Their participation brings a focus on the effectiveness and accessibility of the project’s outcomes, significantly enhancing its expected impact. AFM-Telethon’s clinical trial experts will lead the design of an innovative adaptive clinical trial model for the future validation of pharmacological therapies for rare diseases (RDs). As the coordinator of both the Patient Advisory Board (PAB) and the Expert Committee, AFM-Telethon plays a crucial role in ensuring meaningful patient involvement throughout the project.
