Developing new treatments for rare and ultra-rare neurological diseases requires more than scientific expertise. It requires listening carefully to the experiences of patients and their families. At Servier, patient advocacy groups (PAGs) play an important role in bringing this perspective into research and development. 

PAGs are non-profit organizations dedicated to representing people living with specific diseases or conditions. Many provide education and support to patients and caregivers, raise awareness about their condition, advocate for improved access to care, and fund research initiatives. Just as importantly, they create communities where patients and families can connect and share experiences, something that can be particularly valuable in rare diseases, where patients are often geographically dispersed and access to information can be limited. 

For researchers working in rare neurological conditions, these organizations can provide insights that are difficult to access through traditional scientific channels alone. PAGs often maintain patient registries and support natural history studies, helping researchers better understand how diseases evolve and which symptoms most affect patients’ quality of life. These insights can also directly inform drug development. Patient organizations can provide valuable input on the feasibility of clinical trials, helping researchers design studies that reduce the burden on patients and caregivers while ensuring the clinical endpoints (such as the most impactful symptoms) are truly relevant to those living with the condition. Because PAGs maintain close relationships with patient communities, they can also help facilitate communication around clinical trials and research opportunities. 

At Servier, engagement with patient advocacy groups begins early in the development process. Once a potential therapeutic solution is identified and a decision is made to advance it, teams may initiate partnerships with relevant organizations. These collaborations are built around shared goals and ongoing dialogue, often through regular meetings and project-specific interactions. 

“Communities living with neurological and neurodevelopmental disorders have long felt invisible in mainstream medicine, despite the devastating impact these conditions have on patients and their families. FRAXA and other patient organizations have spent decades working to advance the science needed to reach this moment, so it is deeply encouraging to see Servier’s growing commitment to neurology research and development. For families affected by Fragile X syndrome and related conditions, Servier’s engagement brings real hope that life-changing therapies are finally within reach.” said Katie Clapp, President and Executive Director of the FRAXA Research Foundation. 

“In rare neurological diseases, data alone is not enough. We have a responsibility to understand how patients experience their condition in their daily lives, and to reflect that in the way we design and conduct our studies. Close collaboration with patient advocacy groups is essential to challenge our assumptions, ask better questions, and make more informed decisions as we move therapies forward,” explains Nitza Thomasson, Servier Global Head of R&D Neurology.  

Patient perspectives can also influence concrete research decisions. One example of this is when Servier worked with caregivers from KCNT1 Foundation to review a clinical trial protocol synopsis and provided feedback that led to several modifications to the study design. Adjustments included changes to exclusion criteria and modifications to the scheduling of hospital and site visits, helping reduce participation burden while maintaining scientific rigor. Beyond the protocol itself, their insights helped shape the materials designed for patient families, ensuring the trial information is clear and easy to understand. 

As Servier expands its work in neuroscience, engagement with PAGs remains a key part of the approach: listening early, ensuring patient perspectives are reflected in the research process and collaborating throughout development.