Virtual Zeal Rare Disease Caregivers Carry A Powerful Voice In Social Media

Sticky Post By mcfadyena On 8 March, 2013

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 “ShameonJNJ.com”, Twitter and e-mail barrage, and Facebook community to encourage Janssen / Johnson &, 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.

This article highlights how rare disease patients and caregivers can harness the power of social media to support or challenge pharmaceutical companies. The equivalent of a modern day megaphone, telephone, and printing press combined, social media showcases its power in no more passionate…

Melissa Hogan – SavingCase

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.

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.

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

On March 1, Johnson &, 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.

Angry Parents Force J&J To Do Damage Control

Sticky Post By mcfadyena On 6 March, 2013

There is nothing like a bit of pressure from an angry mom or dad to generate heat. And thanks to the modern wonders of the Internet, an Ontario schoolteacher has succeeded in forcing Johnson & Johnson to scramble to contain a mushrooming controversy. At issue: a months-long refusal by the health care giant to support further research involving one of its own drugs for a debilitating disease.

The uproar emerged two weeks ago. Andrew McFadyen, whose eight-year-old son suffers from MPS, a group of rare genetic disorders caused by the absence or malfunctioning of lysosomal enzymes, grew frustrated with J&J and turned to the Internet to publicize his quest. For more than a year, he had hoped J&J would agree to work with researchers at the Mt. Sinai School of Medicine who found that a J&J drug called Elmiron may offer some hope.

His primary contact at Mt. Sinai is Calogera Simonaro, an associate professor in the Department of Genetics and Genomic Sciences, who recently co-authored a paper showing various improvements in rats given Elmiron, a J&J drug that is approved for treating interstitial cystitis, which is also known as painful bladder syndrome. The two met a few years ago when Simonaro applied for a grant from the foundation that McFadyen created to further MPS research and help his son, Isaac (see photo above).

“She met with (J&J representatives) last spring to present her data to show how the drug worked and she reported back to me that they weren’t interested,” says McFadyen, who hoped J&J would back additional studies, such as testing in larger animals. “Essentially, we were put off. The discussions were not going anywhere. Our researchers and their medical team finally held a teleconference last November, but then, there was no follow up.”

“We spent nearly a year trying to convince J&J that they should (support the MPS research at Mt. Sinai and clinical research using Elmiron). It could save the healthcare industry lots and lots of money. Right now, it can cost $500,000 to $1 million a year for enzyme replacement therapy, which is an imperfect situation,” he says. “But most important, it might increase the quality of life for the children and save a lot of lives.”

Simonaro declined to comment, other than to offer a statement in which she said “we are in discussions with potential partners who have an interest in testing (Elmiron) for MPS in a formal clinical trial setting so that the therapy can be approved and available for use by all patients.” Mt. Sinai, by the way, is obtaining intellectual property rights to use the drug for MPS treatment, according to sources.

There are actually several forms of MPS, which can cause a variety of symptoms, including mental retardation, cloudy corneas, short stature, stiff joints, incontinence, speech and hearing impairment, chronic runny nose, hernia, heart disease, hyperactivity, depression, pain and a shortened life span. The disease occurs in about one in every 25,000 births, according to theInternational MPS Network.

In other words, this is a rare disease and Fadyen has openly expressed concerns that J&J was not interested in making an investment that called for pursuing years worth of costly research for a treatment for a small patient population, even though orphan drugs are increasingly commanding price tags of $250,000 or more per year for each patient (read this and this).

So last month, McFadyen reached out once again to J&J and received a reply from Steve Silber of the R&D team at Janssen, the J&J unit that sells Elmiron. Silber offered to make the drug available to McFadyen’s son on a compassionate use basis and to work with physicians on a so-called investigator-initiated study (read his letter here). McFadyen responded two ways – he wrote a harsh response that accused J&J of stalling tactics and he created a web site calledshameonjnj.com.

The web site quickly attracted attention not only among those with MPS and their family members, but people who were upset that J&J appeared unwilling to extend its largesse to others. As McFadyen viewed it, J&J was offering only his son compassionate access, which he declined to accept because he believed the health care giant should make Elmiron available to anyone who might benefit. And since J&J released its letter publicly, he placed that and his own reply on the new web site.

“From the very beginning, we have approached Johnson & Johnson about working toward such a study and, time and time again, we were ignored, rebuffed and put off,” he responded to Silber. “Families dealing with this disease are incredibly vulnerable and being caught in the middle of weighing the risks vs. rewards of putting our children on this treatment off label without adequate safety and efficacy data.”

By last week, this very public exchange, which prompted angry parents to Tweet links to the web site and post on a Facebook page as well, was on the verge of giving J&J (JNJ) yet another image headache. The health care giant, you may recall, has suffered a series of embarrassing gaffes over the past three years – manufacturing problems that led to the recall of countless over-the-counter items such as Tylenol and Motrin; courtroom setbacks over Risperdal marketing (seethis and this) and a scandal over the safety of hip implant replacements.

And so, J&J late last week began a counterattack. In response to the sudden burst of negative publicity, Jannsen had its chief medical officer, Amrit Ray, respond to McFadyen in yet another letter. And this time, he made a point of writing that compassionate access would be available to any child and reiterated the offer to support an investigator-initiated study with any physician would be interested in doing so. Ray also maintained that J&J was convening a group of experts to explore the possiblities for supporting MPS research with Elmiron.

“We’re trying to get in the right place to where we can get the right data. It’s not one of the areas where we have a lot of experience,” Ray told us. “There has never been any data to indicate it would help patients.. but we’re eager to understand the data… and we’re certainly open to hearing a proposal. In this case, we would like to get some additional expertise to assess any proposal” from a physician willing to administer the drug. However, he disputed some of the chronology that McFadyen offered about miscues last year for substantive discussions.

Initially, McFadyen responded with skepticism and continued to express concern that experimental usage of the drug poses risks and that J&J should be willing to commit to supporting a regular clincical trial. By the start of this week, though, he had held several telephone conversations with Ray and now tells us he is willing to give J&J a chance to work with families. In fact, he is considering pulling down the shameonjnj web site. “I think the web site achieved its goal,” he says.

“Does this mean that a clinical trial is imminent? The answer is no, we aren’t there yet. But this does mean that a true review of Elmiron by Janssen will be thoroughly conducted by the some of the best minds in the field of MPS, from all over the world,” McFadyen wrote on the Isaac Foundation web site (read here). “We look forward to monitoring the progress of those discussions as we seek to find the best treatment options possible for all of our kids suffering from MPS.”

Whether J&J follows through, of course, remains to be seen. The drugmaker is not under any obligation to fund additional research, although the resources needed to explore the possibilities – with the help of scientific experts who can ballpark the odds of success – are relatively modest. For now, though, J&J has scored a win. In a hurly burly world where just about everything can – and often does – go viral quickly, the health care giant has managed to diffuse a potential crisis. And unless J&J commits yet another blunder and angers MPS families again, this is a rare accomplishment when considering the recent spate of scandals, gaffes and setbacks on so many fronts.

06 Mar 2013

Sticky Post By mcfadyena Posted in Advocacy, Media, Newspaper Permalink