Poster presentation at 20th World Symposium: A paradigm shift in collecting RWD for GM2 gangliosidosis.
Collecting real-world data (RWD) for rare diseases, particularly GM2 gangliosidosis, has historically posed challenges. However, a recent shift in the approach to RWD collection effectively led by Pulse Infoframe fosters a more collaborative and comprehensive approach to research. The FDA’s guidelines on RWD stress the importance of creating harmonized datasets, which aligns with Pulse’s methodology to overcome the issue of fragmented data.
Previous attempts at collecting RWD for GM2 gangliosidosis have often been unsatisfactory due to fragmented data from different stakeholders. Existing datasets also tend to focus on specific disease aspects, absent of a comprehensive view and essential information such as clinical data, genomics, biomarkers, treatment history, and patient-reported data. Recognizing this gap, a transformative approach led by Pulse was developed to address knowledge gaps and promote collaboration between researchers, industry, and advocacy groups.
To better understand the disease and the experiences of GM2 patients and their caregivers, a detailed exploration of collaborative research feasibility and requirements was conducted. Key Opinion Leaders, including those with a clinical and advocacy background, were interviewed, providing valuable insights forming the basis of a strategic RWD approach aligning with regulatory guidelines. This approach ensures harmonized data mapped to a standardized framework throughout every stage of drug development for the disease.
Central to this approach is recognizing the significance of each stakeholder group and their unique experiences. A thematic approach was employed to document value propositions for patient advocacy, clinicians, and industry, ensuring transparency of stakeholder considerations and laying the groundwork for further knowledge development.
The collection of RWD for GM2 is undergoing a significant transformation beyond traditional methods. This new approach encourages collaboration through open communication and transparency, overcoming the challenges of scattered data. It provides a glimpse into the future of research and serves as a blueprint to expedite advancements and improve outcomes in GM2 gangliosidosis, other lysosomal storage diseases and rare diseases in general.
To learn more about our transformative approach to real-world data collection email us at email@example.com