
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
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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


Expanded Access Programs (EAPs), sometimes known as Managed Access Programmes (MAPs), allow pharmaceutical sponsors to offer patients their pre-approval investigational
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