A blog exploring pharmaceutical relationship marketing, emarketing and innovation with a focus on rare disorders.
The convergence of rare disease, digital communications, and pharmaceutical marketing communications

Participatory Medicine in a Rare Disorder

Posted by | 11:18am on Wednesday, April 8, 2009

Especially around rare disorders, patients are driven to push harder and faster than our current healthcare system allows, and that changes everything. 

I was catching up on my reading today, and Gilles Frydman’s post, “A wonderful story of participatory medicine” pointed me to a powerful article from last week’s WSJ.  The WSJ article, written by Amy Marcus, is about a mother who goes to the mat finding and convincing a researcher to help her get treatment for her twins’ fatal rare disorder.

Chris Hempel got the fatal diagnosis of Niemann-Pick Type C in October 2007 for her 5 year old twins and immediately started looking for a treatment to save their lives.  She found a compound called cyclodextrin that extended the lives of affected mice and tracked down all kinds of leads about how this compound had been used, and once she was persuaded it might work, she was determined to find a way to get FDA approval to try it for her children.  In the process she found a pre-eminent AIDS researcher, Dr. James Hildreth, who was also interested in getting FDA approval for the same drug but to help in the prevention of HIV transmission during heterosexual sex.  With shared data and the help of Johnson and Johnson they eventually worked together to get FDA approval to infuse the twins.

Gilles points out:

“This is typical of the way innovative treatments are discovered in communities of people dealing with rare diseases.  There is a fast growing number of stories of patients/caregivers capturing scientific news and inferring that it may work for the condition that concerns them.  Because it is most often happening with rare diseases, the public at large is unaware of the sea change that is taking place right under our nose.  It is truly like watching in real time a paradigm shift in action!”

Gilles Frydman is a pioneer of medical online communities and founder, in 1995, of the Association of Cancer Online Resources (ACOR), the largest online social network for cancer patients. ACOR has served over 1/2 million cancer patients and caregivers.

This is a great story about participatory medicine and the way the internet – by providing easier access to information – is changing the relationships of patients, healthcare providers and pharma companies in healthcare.  Especially around rare disorders, patients are driven to push harder and faster than our current healthcare system allows, and that changes everything.

So, what does this mean for pharma marketing?

As pharma marketers, we have the opportunity and obligation to provide patients, their family members, healthcare providers, as well as researchers with essential and accurate information.  And as this story illustrates, pharma companies are being affected and can play a role in participatory medicine.

Because most of the connections that lead to these changes start with an internet search, one thing that pharma brands can do now is create information rich websites that share stories, resources, strategies and results.  This creates the right kind of brand experience for the patients and caregivers who are making the decisions in rare disorders. And with long-tail search, people you may not expect may be coming to your website and making connections you haven’t thought about.

Have you heard of other examples that demonstrate the role pharma can play in participatory medicine?

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(Image courtesy of nemeaux via Flickr)

About Wendy White

Since founding Siren Interactive in 1999, Wendy has been recognized as a thought leader at the intersection of niche pharma brands, patient empowerment and online marketing. Her vision for how the internet can facilitate interactions and provide crucial information that patients, caregivers and their healthcare providers previously struggled to find has propelled Siren to the forefront of relationship marketing for rare disorder therapies.

View other posts from Wendy

  • Barbara Woldin

    I was indirectly involved in participatory medicine. For 15 years I worked (in marketing)for the company that developed the oil for Lorenzo’s Oil and knew some of the chemists that worked with the Odones and Dr. Moses. We got to “go Hollywood” for awhile.

  • Wendy White


    What a fantastic story that so exemplifies the early days of participatory medicine. I vividly remember when your “hollywood” story came out. I have to believe that pioneering work helped people believe they could make a difference in their loved ones lives. And now with the availiblity on the internet of access to information and social communitites it is easier (or at least faster) to find possible treatments and make connections that used to be out of reach. Thank you for sharing your story.

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