This issue’s rare disease section is bursting with articles spanning a range of topics within the sector. First up, Quotient Sciences consider orphan drug development and CRO/CDMO collaboration, specifically in paediatric rare diseases (page 10), while ProPharma explores clinical trials for rare diseases and how these could be improved (page 14). Alexion then tells us about the challenges facing rare disease treatment and the importance of patient-centricity (page 20), and Veeva assesses how companies are overcoming challenges related to rare disease education, development and launch of new medicines (page 22). Finally, Synaptix Bio tells us about the problems facing the treatment of rare diseases (page 17).

Psychoactive medicine is a huge area of development within the pharma industry, as various companies look for alternative options to traditional standard-of-care treatments for many conditions – including, but not limited to, mental health conditions. In this issue, Compass Pathways explores the need for new treatments in the mental health space, as well as assessing whether psychoactives could be the way forward (page 24). Also in our psychoactives section, Beckley Psytech tells Pharmafile about the use of psychedelic medicine for the treatment of psychiatric and neurological conditions, and how this field could develop in the future (page 26).

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ISSN 0959-8758. All rights reserved. © 2024 Samedan Ltd

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Contents

6 NEWS

12 RARE DISEASES

28 PSYCHOACTIVES

34 WOMEN’S HEALTH