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The Challenges in Treating Rare and Orphan Diseases

Posted on March 21st, 2016 by in Pharma R&D


Those outside of the drug and medical research communities often do not understand the complexities and challenges in developing therapeutic treatments for diseases that afflict a patient population that cannot possibly offset the cost of R&D, clinical trials, and manufacturing and distribution. This is not to say that everyone involved in the research process is not aware of the enormity of these diseases and are willing to contribute. We recognize the tremendous, and often heartbreaking, impact these diseases have, not only on the afflicted, but also to the families, friends, and caregivers. [In May 2015], I was at a leading pharmaceutical company where the head of a society addressing a rare disease had recently given a motivational and inspirational talk to the researchers. The effect of that talk was tangible: ‘What can we do to help?’

There are a number of potential ways to address this that have been considered, including potentially bringing in government-sponsored drug development. As a member of the scientific community tasked with providing and disseminating research data, it occurred to me that one critical area where we can focus our efforts is in optimizing how we can take ‘known’ data and transform it to understood data. Through enabling the understanding data we can facilitate a better level of understanding of genotypic and phenotypic data contextually with mechanisms of action, compound and/or biologic characteristics, safety, and potential adversity. If we can bring the data together in a way to make these relationships more apparent, with data that is more directly comparable, and inclusive of data from the broadest possible spectrum of sources, we can minimize the barriers to discovering and designing more tailored therapeutics.

This article from PharmaVOICE’s June ’15 issue [Rare Diseases: Research, Development and Commercialization] gives a very strong set of perspectives illustrating the research community’s thoughts and potential directions (caveat: I am proud to be included in the article). I hope to have more to say on this topic in the near future, and in the meantime encourage you to read the article. As always I am very happy for any comments or feedback.

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