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.
Perspectives
Since rare diseases afflict about 1/10 of the population as a whole, we have a moral obligation to find effective drugs to alleviate the pains and sufferings caused by rare diseases. In this aspect, the public, including patient advocacy groups, regulators and scientific communities need to put the heads together to face the challenge.
Dr Charles Oo
Read the Original
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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