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.

Obviously lack of awareness is partly a function of the rarity of these disorders but there are countries with smaller populations than Canada doing far better at supporting patients with orphan diseases. It’s ironic that we Canadians believe we are a compassionate people and, despite its well publicized challenges, we still firmly wear our universal health care system as a badge of honour, yet we allow these most vulnerable individuals to slip through the cracks.  It is essentially a perfect storm for these individuals and their families.  First they must suffer with often debilitating symptoms, then it can take months or even years for a correct diagnosis – often after enduring several misdiagnoses and finally they have to do to an exhaustive battle trying to source funding for unaffordable medicines and treatments.  I can’t imagine the shock in learning my child had a lifelong incurable disease and then subsequently learning that there is no funding for her medication that would help her lead a normal life but I couldn’t possibly afford it.  

The Globe article accurately points out that Canada is one of the few developed countries with no federal policy or funding for orphan diseases … something that continues to be lacking due to political wrangling between provinces and the federal government.   It is mind boggling the difference in orphan drug funding between provinces.

Slow progress is being made in some provinces but this has dragged on for too long.  It’s time for a federal orphan drug policy in Canada and the only way it will happen is if all stakeholders including federal and provincial governments, pharmas, patient advocacies and the medical community collaborate.

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.

With much trepidation our first step was to approach clinics and specialist physicians who treat patients with the disorder.  No surprise – the vast majority declined their support.  We recognized years ago that the days of soliciting patients for marketing research studies via their physicians and clinics were over.  Legal and privacy concerns are oft cited but physicians and clinics also want to avoid any real or perceived influence from pharmas.  Like most patient research today we knew we had to reach patients more directly.

It occurred to us to look at the larger North American population.  If there was no patient advocacy group in Canada perhaps there was one in the US with Canadian members.  Viola, there was a patient advocacy group in the US with a considerable number of Canadian patient members.  Once comfortable with our references and experience with other disease charities, and understanding the benefits of the study, the charity agreed to send a confidential “evite” to their Canadian membership.  Only the identity of those patients “opting in” would be known to us.  With the support of the charity we were able to complete the study and deliver the profound learnings to our client.

We find that patients are nearly universally supportive of studies that may benefit them or others with their disease, even those with very serious illnesses.  The challenge is finding these patients.  The internet, including social networks, is making these patients more accessible through online associations, but they must be approached in a credible and meaningful manner.

This is a very simple example of how, with a little tenacity, a very simple, low cost approach delivered extremely valuable market intelligence to support a critical marketing decision – exactly what marketing research is supposed deliver.