Virtual zeal: rare disease caregivers carry a powerful voice in social media

Melissa Hogan




This article explores the voice of rare disease patients and caregivers in social media and the power they wield to support or challenge the pharmaceutical companies that serve them.


The equivalent of a modern day megaphone, telephone, and printing press combined, social media showcases its power in no more passionate an arena than in rare disease.


While some think of Facebook as a fun pastime, in private rooms or sometimes publicly, rare disease sufferers and their caregivers are offering diagnoses and treatment advice, discussing side effects, and advising on every other aspect of life with a chronic or rare condition. While some follow celebrities on Twitter, those affected by rare disease might use Twitter to make sure their views are heard by pharma and their governments alike.


On the heels of Rare Disease Day, you’d be remiss not to know that rare disease advocates are often proud of their status as ‘zebras’. “If you hear hoofbeats, think horses, not zebras” is the oft-quoted tenet of medical diagnosis, the assumption being that often the simplest explanation, rather than a rare or exotic disease, is usually the best.


Zeal is not just a pack of zebras


It is that perspective that has made rare disease groups stand up and proudly claim the title of zebras, often displaying stuffed zebras at their events. But when zebras become a pack, as they often do on social media, they become a zeal, a title that is not only categorically appropriate, but descriptively appropriate as well.



Figure 1: Zebras on display at Rare Disease Day 2011 at National Institutes of Health. Photo courtesy of National Institutes of Health.

In addition to describing a pack of zebras, zeal is defined as “great energy or enthusiasm in pursuit of a cause or an objective.” More than almost any other online group, those affected by rare disease approach their cause of support, education, medical care, and advocacy with great energy and enthusiasm. In describing them and others like them as the “alpha geeks” of health care, internet geologist Susannah Fox notes: “They are in the crucible. They ‘roll their own’ by creating communities of health information exchange where none had existed.”



“On the heels of Rare Disease Day, you’d be remiss not to know that rare disease advocates are often proud of their status as ‘zebras’.”



Feeling the zeal


Not only can rare disease patients utilize social media in their diagnosis, support, and treatment efforts within their population, one should be cognizant of both the benefits patients can confer in raising awareness and getting expensive treatments reimbursed, but also the damage they can inflict via the democratizing medium of social media. An example of both can be found in the efforts of Canadian-based The ISAAC Foundation and its founder, Andrew McFadyen, a parent of a child with the rare disease Mucopolysaccharidosis (MPS) VI.


To date, Mr. McFadyen’s social media efforts have succeeded in obtaining reimbursement of high priced enzyme replacement therapies for several patients, including his own son Isaac, by their Canadian provinces after the provinces first declined or delayed reimbursement. While pharma companies are surely lobbying for reimbursement of their treatments, sometimes only the efforts of the patients they serve are viewed with collective sympathy.


In 2012, for example, the family of a young girl with MPS VI, Violet Revet, had been awaiting for approximately six months for word on reimbursement for the drug Naglazyme® by the Saskatchewan, Canada health ministry. Without an answer, and watching the disease progress in their daughter, the parents went to Mr. McFadyen for help. A Twitter campaign caught the attention of Premier Brad Wall who responded and the treatment was approved within days. The effect was as clear as the Premier’s statement: Twitter “democratizes things.”



“While pharma companies are surely lobbying for reimbursement of their treatments, sometimes only the efforts of the patients they serve are viewed with collective sympathy.”



Amplifying the voice of reason


While pharma can benefit from the efforts of patient advocates, they can be the target of such efforts as well, such as Mr. McFadyen’s latest endeavor, which I was able to view from the inside out.


Over the last several years, scientists began studying the use of a Janssen FDA-approved drug for interstitial cystitis called Elmiron® for the bone and joint problems that plague children with MPS. The ISAAC Foundation was one financial supporter of those studies. Data from small animal studies were completed in 2012 and presented at several conferences. This precipitated Mr. McFadyen’s conversations with Janssen itself to explore the research further and possibly support human trials.



Figure 2: Isaac McFadyen (front) who suffers from MPS VI, flanked by his family including his father and advocate Andrew McFadyen (right). Image courtesy of The ISAAC Foundation.



According to Mr. McFadyen, his efforts were rebuffed several times until finally in February 2012, he began a social media campaign, including a website not so subtly titled “”, Twitter and e-mail barrage, and Facebook community to encourage Janssen / Johnson &amp, Johnson to reconsider its decision. The campaign culminated around February 28, Rare Disease Day, with several Janssen executives receiving hundreds of e-mails from impassioned parents and supporters of MPS families.



“…it is clear that their behaviors and efforts cannot be ignored.”



On March 1, Johnson &amp, Johnson changed its Twitter handles that had been receiving some of the barrage, from @JNJComm and @JNJStories to the new @JNJCares, @JNJParents, and @JNJNews. Janssen also quickly began organizing a medical advisory board to both bring Janssen up to speed on a disease with which it was unfamiliar (Mucopolysaccharidosis) and to consider the scientific evidence and what next steps to take.


Some might disagree with the public pressure of social media tactics like those employed by Mr. McFadyen and his supporters, calling it public bullying. When asked what he thinks of those who might say that using such tactics make him a bully, Mr. McFadyen replied:

This is not being a bully. I’m just one man with a firm belief in the rights of those with rare disease to have treatments just like those with cancer or heart disease. One might instead call a large pharmaceutical company or a government a bully when they make decisions without considering who they affect. With social media, we simply help amplify the voice of reason.

Whether studying, interacting, advertising to, benefitting from, or even suffering from the behaviors of rare disease sufferers on social media, it is clear that their behaviors and efforts cannot be ignored.


Related articles


Angry Parents Force J&amp,J To Do Damage Control – pharmalot


When Rare Just Isn’t Enough: The Case of Elmiron –







About the author:


Melissa Hogan is a lawyer and strategic consultant by profession, but since her youngest son Case’s diagnosis with MPS II in 2009, she has also applied her experience to become an advocate, author, and speaker on behalf of rare disease families. She writes about advocacy, medical research, pharma, clinical trials, therapies, social media and special education on, a blog that is now read in over 100 countries. She also uses other social media strategies such as Twitter, Facebook, Pinterest, YouTube, and LinkedIn and serves on the Advisory Board for the Mayo Clinic Center for Social Media.


She is also the author of the e-book Calmer: Medical Events with Cognitively Impaired Children (2012) which seeks to share strategies for preventing medical trauma in children with chronic medical conditions.


For more information, visit Melissa can be reached via Twitter @savingcase or by e-mail at


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