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

The Need For A Rare Disorder Patient Registry

Posted by | 9:02am on Tuesday, June 16, 2009

everything we have learned about how the human body works we have learned from people with rare disorders.  It is at that end of the spectrum that innovation happens. 

I went to the first National Organization for Rare Disorders (NORD) Summit a few weeks ago in DC and learned that ONE of the major problems with bringing more treatments to more people with rare disorders is that there is often no natural history (database) of people with rare disorders. (There are other issues, not the least of which is not enough trained scientists to develop good assays to test and not enough scientists to follow through with the research on the drug’s overall effect on humans once some likely candidates have been identified).

Rare Disorders as a Public Health Issue

First, let’s assume this is a public health issue that America should be funding.  According to Dr. Tim Cote, head of the orphan drug division of the FDA (sitting on the dais with Janet Woodcock, director of CDER, and Francis Collins, leader of the human genome project, among others), everything we have learned about how the human body works we have learned from people with rare disorders.  It is at that end of the spectrum that innovation happens.

Also anecdotally, it is possible that a large percentage of the people living with rare disorders are children (6,000 disorders that, taken together, make up an estimated 10% of the population).  We have the scientific knowledge to save these children, but we don’t have a good process for drug approval, and the drugs are very expensive (most, but not all, of the time this is directly related to the cost of research in bringing a drug to market because the process was set up for drugs that treat small problems in large populations).

Can/Should Social Media Play a Role?

I think there is potential for social media to play a role in solving the patient recruitment/ natural history problem for bringing drugs to market. Online communities like PatientsLikeMe provide a wealth of data to the people most likely to do something about this problem — e-patients, but I think the answer may need to be more universal.  I would love to hear some ideas about how to address this issue and any thoughts you might have on the importance of rare disorders as a pubic health issue.

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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.

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