BIO 2011: What Fuels Rare Disease Development

BIO 2011: What Fuels Rare Disease Development

06.27.11 | By Kate Connors

Speaking at the BIO 2011 International Convention, Jeff Behrens from early-stage company Edimer Pharma discussed the many challenges that orphan drug developers confront in their quest to successfully treat a rare disease.

From difficulty in reaching the patient population (due to both low disease incidence as well as poor documentation of many rare diseases) to the problems associated with reaching physicians (as many rare disease patients see what he described as a "patchwork quilt" of specialists) the challenges are many, according to Behrens. And, in fact, we've discussed many other such challenges here before.
But Behrens also made a point that helps to explain the remarkable amount of perseverance and commitment that drives these companies: "We know the disease better than anyone else."
That's why they continue this often-frustrating work, he said - because with their knowledge of the disease, the science and the patients, biopharmaceutical company scientists are the right people for the job.

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