Module232025

07/05/2025

Rare Diseases

The Organisation for Professionals in Regulatory Affairs

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Rare Diseases – how much we have covered in other populations

Because of their rarity, patients are often far from centres – possibly in other countries – so you have the travel, language and cultural issues They may be seen only in children, because no-one lives long enough to grow up We often don’t know what is “normal” for patients, or if that changes as patients grow up We have little experience of treating rare diseases in utero and we often don’t know the impact of pregnancy on a rare diseas patient The basic principles apply: Understand the geographic, linguistic and cultural limitations Provide information in formats patients and parents understand

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Communication

Understand the potential for PK variability Work with advocacy groups, where possible

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