ESGCT/BSGCT Annual Congress

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Academia developing medicines for rare diseases to receive free EMA scientific advice

The academic sector plays an important role in the development of innovative medicines. Their scientific research is often at the source of novel methodologies and innovative medicines with a potential to benefit patients with rare diseases. Early interaction with EU regulators is important for academia to understand the regulatory requirements and allow the generation of robust evidence needed to establish the medicines’ benefits and risks. This helps them to navigate the regulatory process and ultimately to translate their discoveries into authorised, patient-focused medicines. However, feedback from academia showed that fees for protocol assistance (scientific advice for orphan medicines) represent a hurdle to engage with EMA. More on the website https://www.ema.europa.eu/en/news/academia-developing-medicines-rare-diseases-receive-free-ema-scientific-advice