What is it about?
There is an unmet medical need for orphan drug development. However, because of limited financial incentives, it remains a challenge for drug development entities until now. This paper outlines orphan drug development, and its regulatory incentives as a result of patient advocacy and public incentives. It ends with some possible means to advance orphan drug development, and with the view that it is a golden opportunity for both the pharmaceutical industry and patients of rare diseases.
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Why is it important?
Orphan drug development is an intricate dilemma, and it is important for all stakeholders to make it a sustainable commercial activity for the benefits of patients of rare diseases. On the other hand, drug development entities are obligated to find a cost-effective resolution for long-term sustainability. Some of the strategies and proposals toward this end are provided.
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This page is a summary of: A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?, The Journal of Clinical Pharmacology, September 2015, Wiley,
DOI: 10.1002/jcph.599.
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