In Europe, 30 million people live with rare diseases, for many of which currently there is no available treatment. Although pharmaceutical companies are continuously working to develop new therapies, the traditional way of conducting randomised...
Since March 2020 the EMA has been actively following a strategy named “Regulatory Science to 2025” in order to advance its engagement with regulatory science over the next five to ten years. The EMA are aware that massive amounts of data that could...
When it comes to rare diseases, Real-World Data (RWD) precision can be lost when aggregating samples across countries, and there is a strong case to create a common data model in Europe to form a single population where multiple autonomous sources...
Rare disease come with some specific considerations that affect the optimal use of Real-World Data (RWD), including factors that arise due to limited patients and limited data.
There are multiple challenges when dealing with Real-World Data (RWD) in rare diseases, from how to organize teams and plan operational capabilities, to small sample sizes introducing statistical limitations and data protection restrictions, to RWD...
Real-World Data (RWD) and Real-World Evidence (RWE) present several opportunities in application to rare diseases, including supplementing clinical trials data for drug approval applications, better understanding the disease itself, expanding the...
The healthcare industry stores over 2,300 exabytes of healthcare data, according to the World Economic Forum, with the vast majority going unused, siloed in different formats, structures, and locations.
