Advancements in Real-World Data for Rare Disease Research

This technical report discusses the advancements in real-world data (RWD) and its impact on rare disease research in Europe. It outlines how RWD is transforming the drug development landscape, particularly for rare diseases, which historically lacked targeted research. The report details various initiatives across Europe aimed at leveraging RWD to enhance understanding of rare diseases, including the establishment of patient registries and collaborations among healthcare professionals, patient advocacy groups, and regulatory bodies. It describes the regulatory framework established by the European Medicines Agency (EMA) to support orphan medicinal product development and the incentives provided to pharmaceutical companies. The report also highlights the success of RWD in recent drug approvals, such as blinatumomab and avelumab, and discusses the barriers to RWD evidence generation, including data fragmentation and harmonization challenges. Finally, it presents future trends in RWD utilization and the expected growth of the real-world evidence solutions market.