The Research Pulse

Canada Needs Orphan Diseases Policy

I read with much interest an article on orphan diseases in the GLOBE AND MAIL on April 5.  This article followed a CBC Radio segment of The Current addressing the ordeals patients with rare diseases have, firstly, in obtaining a diagnosis, and then in trying to get insurance coverage for extremely expensive medications to treat their disease.

We recently completed a growth disorder study for a leading pharma client.  Most growth disorders are so rare that there are no patient advocacies or charities dedicated to them in Canada.  Fortunately there is a foundation called the Magic Foundation in the US that is dedicated to advocacy for patients with rare growth disorders and they were kind enough to confidentially invite their Canadian members to participate in our study. (more…)

Rare Disease Patient Research

By: Mike Peirce

In October 2010 a top 3 pharma approached us with the daunting task of conducting patient level marketing research with individuals suffering with an ultra low incidence (less than 1 in 10,000 Canadians) genetic disorder.  This marketing research would provide valuable patient insights and experiences to support a “go/no go” decision for the launch of a product to treat the disorder.  Under normal circumstances we would draw on our patient registry to identify potential respondents or, failing that option, contact the relevant disease association or charity to request their support in inviting patients to participate.  Unfortunately the disorder is so rare that we had no patients identified with the disorder in our panel and there is no related patient advocacy group or charity in Canada.  This is the point at which the vast majority of researchers would have “bailed out” – if they hadn’t done so when they first heard “rare disorder patient study”. But we dug our heels in determined to deliver the research that our valued client needed. (more…)