Developing an Integrated Evidence Strategy for Rare Disease Clinical Development: Key Takeaways
Currently there are 6,000 recognised rare diseases, affecting over 300 million people globally. Despite these staggering numbers, only 5% of
Currently there are 6,000 recognised rare diseases, affecting over 300 million people globally. Despite these staggering numbers, only 5% of
Clinical trials are by design, a scientific experiment, which require highly selective populations in a controlled setting, and with often
Real-world evidence (RWE) is increasingly being used within the regulatory and reimbursement process. RWE studies allow for a clearer understanding
Background The pharmaceutical manufacturer (Sponsor) aimed to study the real-world effectiveness and usage of their medicine in patients with
Background The pharmaceutical manufacturer (Sponsor) wanted to understand the effectiveness of their medication in the real-world. For this
Pregnancy registries gather health information from women who take prescription medicines or vaccines when pregnant and compare it to data
Patient registries are organised systems that use observational methods to collect uniform data to evaluate specified outcomes for a population
For years, the prospect of ‘virtual’ and decentralised research was discussed with hope as a potential disruptor to enable greater
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