Volume 34, Issue 1 - Rare Diseases
Writing for orphan drugs: A compass to navigate document types and regional requirements
Authors: Katie Brooks, Pauline Haleux, Montserrat Cuadrado
Abstract
Medical writing for rare diseases encompasses the development of various regulatory documents that are required to obtain orphan drug designation and marketing authorisation for treatments targeting rare conditions. Effective planning and close collaboration with key stakeholders are essential to navigate regional regulatory requirements and address unmet patient needs, ultimately facilitating the approval of treatments for rare diseases.
Medical Writing. 2025;34(1):24–30. https://doi.org/10.56012/suws8001
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