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Chapter 16

Possible Solutions to Accelerate Access to Rare Disease Treatments

The introduction of legislative incentives to promote rare disease research, along with changes to regulatory policy, has resulted in increases in the number or orphan drug designations. Despite this, the number of drugs reaching marketing approval across OECD countries remains frustratingly flat. Research into rare diseases faces inherent challenges throughout clinical drug development and regulatory approval. Tailor-made regulatory and access solutions are needed to overcome these problems. Here I suggest areas for consideration that could have an immediate impact in facilitating regulatory approval and access to treatments for rare diseases.

Publication details

Print publication date
14 Aug 2014
Copyright year
Print ISBN
ePub eISBN

From the book series:
Drug Discovery