“RegistryNXT!” builds upon largest resource in the world for Gaucher disease data
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY), announced today the launch of a new web-based platform, RegistryNXT!SM, to support the International Collaborative Gaucher Group (ICGG) Gaucher Registry. The first and largest database of its kind in the world, the global ICGG Gaucher Registry was built to promote the understanding of Gaucher and to track the outcomes of treatments worldwide. Now in its 20th year, nearly 30 published papers on Gaucher disease have resulted from the wealth of observational data in the registry.
“One of the most important accomplishments of the registry to date is its direct impact on patient care through its ability to provide a solid base of evidence for best clinical practices, including patient assessments and recommendations on quantifiable treatment goals”
Since 1991, Genzyme has worked closely with the international Gaucher community to understand disease epidemiology, natural history and long-term treatment outcomes. Because of the rarity of diseases like Gaucher, having one central repository of data is crucial to understand disease progression and to help manage patient outcomes. The ICGG Gaucher Registry remains the only resource of its kind for Gaucher disease, with over 700 participating physicians representing nearly 6,000 patients and over 40,000 patient-years of follow-up data. Genzyme was the first company in the world to commercialize a treatment for Gaucher disease, and today Cerezyme® (imiglucerase for injection) is the standard of care for patients with Gaucher disease type 1.
“After twenty years of data collection and analysis, the fundamental goals of the Gaucher Registry remain the same: to advance the medical and scientific understanding of Gaucher disease and to improve the quality of care of patients around the world,” said Genzyme’s Head of Clinical Development and Medical Affairs, Richard Moscicki, MD. “RegistryNXT! will facilitate real-time and more comprehensive analysis of data, enabling publication of additional data and development of evidence-based disease management guidelines.”
The launch of the RegistryNXT! application follows three years of development, with new features that include interactive, real-time report generation for disease management, more efficient data collection and more opportunity for patient access to their information, with the involvement of their treating physician. Plans are underway to roll out this new Registry platform for Genzyme’s other lysosomal storage disorder registries: Pompe, Fabry and Mucopolysaccharidosis I (MPS I).
“One of the most important accomplishments of the registry to date is its direct impact on patient care through its ability to provide a solid base of evidence for best clinical practices, including patient assessments and recommendations on quantifiable treatment goals,” said Neal J. Weinreb, MD, Chairman of the ICGG North American Board and co-Chair of the ICGG International Board. “Over the years, patients have repeatedly sought greater direct involvement with the Gaucher Registry. Registry NXT! will now enable physicians to share real time data with patients both at the point of service and via personal computers, a major advance that should enhance patient involvement, understanding and satisfaction.”
The ICGG Gaucher Registry is overseen by international and regional boards of scientific advisors. These advisors are external physicians that form the governance body, oversee the scientific integrity and guide research, publications and policy. Genzyme has provided financial support of the operations, estimated to be over $50 million since 1991. The ICGG Gaucher Registry is operated by a team of individuals from various functional areas at Genzyme, including medical directors, data managers, statisticians, health outcomes researchers, epidemiologists and programmers.