In the midst

snow trees Sparked by the shock resignation of Sarepta CEO Chris Garabedian; this week I am reviewing the relationship between academia and industry and sharing my perspective as a postdoc working in the midst.

My research is focused on a ‘rare’ childhood muscle disorder called Duchenne muscular dystrophy (DMD).  Rare disease expertise typically resides in the clinic, academia and small biotech companies with Big Pharma traditionally reluctant to invest.  However, legislation such as the Orphan Drug Act of 1983 followed by the European Orphan Drug Regulation in 1999 has made drug development for rare diseases more attractive with incentives such as fast-track status and a seven year exclusivity period; not to mention the higher prices that can be charged for orphan drugs.

I have experienced that rare disease research is particularly collaborative both within academia and with industry and uniquely, I think our success depends on it.  This is largely due to the urgent, unmet medical need and the difficulties associated with low patient numbers, all of which has led to a real sense of community that is constantly being driven forward by a very strong patient and parent voice.  Industry seek our clinical and technical expertise and in academia we need the drug development and manufacturing know-how, and lets be honest, the resources of industry.  Yes transparency is essential from both parties for public trust and one can question the prohibitive cost of orphan drugs ’till the cows come home but I believe that these relationships are vital to expedite drug development and the exon skipping drug candidate, eteplirsen, for DMD is a great example.

Five years ago I was working on the first AVI BioPharma (now Sarepta) sponsored systemic clinical trial of eteplirsen which is currently on track for a New Drug Application to the FDA later this (2015) year.  I continue to work on pre-clinical and clinical projects with industry sponsors and/or collaborators to facilitate the journey of this and related drugs to the clinic.  As a postdoc I feel in the middle; valued as an expert by industry but removed from the politics and policy.  Perhaps this is a good place to be, after all I have had the pleasure of being one of the first people to see evidence of drug efficacy in patients with my own eyes down the microscope.  It is so inspiring and rewarding to know that my research may ultimately contribute to delivering a drug to Duchenne patients, and all politics aside, could we really have achieved so much already without an academia-industry relationship, no matter how perfect?

This week saw #IamAScientistBecause trending on Twitter so I’ll end with my tweet which is particularly fitting for this weeks post:

#IamAScientistBecause the implications of scientific research for the future of our children are too great to ignore

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