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The Power of Rare Disease Patients

Posted by | 5:39pm on Wednesday, February 22, 2012
hands clasped

with more accomplishments, increased media exposure and higher awareness of rare diseases, we certainly have something to celebrate 

Thanks to the Pew Internet Project, we have a lot of data about ePatients. These empowered, engaged and educated patients (and families) are helping to bring about a transformation of healthcare in this country. Pew’s Susannah Fox has named them “healthcare superheroes.”

Through our experience at Siren Interactive, we know a lot about rare disease patients who are supercharged ePatients. These patients are hyper-empowered. Because they have to be. Leading up to Rare Disease Day on February 29, 2012 (a rare day indeed) I’d like to celebrate some of the amazing accomplishments of a few rare disease communities.

Rare disease patients start clinical trials
Team Sanfilippo Foundation is a nonprofit medical research foundation started by a group of parents with the mission of finding potential therapies that can be tested clinically in the near future. Team Sanfilippo used social media to beat out thousands of others to win $250,000 from the Pepsi Refresh Project. The grant funded a clinical trial led by Dr. Haiyan Fu of the Research Institute at Nationwide Children’s Hospital in Columbus.

Rare disease patients recruit for research studies
After Katherine Leon was diagnosed with spontaneous coronary artery dissection (SCAD), a poorly understood heart condition that affects a few thousand Americans every year, she went online. Via online communities Leon was able to connect with a few similar women. Leon approached the Mayo Clinic’s Sharonne Hayes, MD, about how she could help spur more research into SCAD. Dr. Hayes was inspired to start a research study, with a recruitment goal of 12 patients. Leon spread the word via online communities and within a week had recruited 18 volunteers. Due to the strong interest, Mayo has launched a bigger trial with 200 SCAD patients and an equal number of their close relatives.

Rare disease patients change laws
Bill and Nicole Morris successfully advocated for the passage of Greyson’s Law (HB 1795) to ensure that Texas screens newborns for 52 disorders. This effort was driven by the memory of their son, Greyson, who died of Krabbe disease, a rare inherited degenerative disorder of the central and peripheral nervous systems.

Rare disease patients start their own biotech companies
After an Israeli biotech company stopped working on a promising medication, two US families bought the drug and decided to develop it themselves as a possible treatment for their children’s Duchenne muscular dystrophy. The company, Halo Therapeutics, has been granted orphan drug status for the therapy.

This year, with more accomplishments, increased media exposure and higher awareness of rare diseases, we certainly have something to celebrate and inspire us to go further. These are only a few examples of the power of rare disease patients; please share more stories in the comments below.

(Image courtesy of Victor Bezrukov on 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

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  • http://twitter.com/SusannahFox SusannahFox

    Wendy is very kind to give me and the Pew Internet Project credit in this post. In fact, we owe a debt to all the people who have responded to our surveys over the years, without whom we would not be able to function as researchers.

    The work that I did in preparation for the release of the report “Peer-to-peer Healthcare” in 2011 was some of the most rewarding of my career so far. The surveys we conducted were the culmination of a decade of research related to how people use the internet, both in relation to health but also in relation to entertainment, commerce, civic life, and education. I knew that we’d learn a lot from both the national telephone survey as well as the special, online survey of people living with rare conditions, but I could not have predicted how it affected me personally.

    The stories shared by those health care superheroes are with me every day as I continue to study the social impact of the internet. This is the joy of my working life. I’ve been deputized by these amazing people to share their insights as broadly as possible, so that every conversation about the internet and health is infused with both data and patients’ voices.

    Here’s the 2011 report if anyone would like to read it:

    Peer-to-peer Healthcare

    It has been cited in a number of media outlets, but here are two favorites:

    NPR’s Talk of the Nation: Patients Seek Moral And Medical Support Online – includes an interview with Pat Furlong, founder of Parent Project Muscular Dystrophy

    JAMA: Patients Go Online Seeking Support, Practical Advice on Health Conditions

  • http://www.sireninteractive.com wendy White

    Thanks Susannah. It was an amazing experience seeing the hunger and speed of those rare disease stories that you collected for your p2phealthcare report.

    Since we are so near rare disease day — I hope you consider segregating rare disease patient internet usage from other chronic disease usage in your next report.

    I truly believe there is a difference between someone with type 2 diabetics who can get lots of information from their doctor (and maybe their friends and neighbors) and someone with a truly rare disorder. There is probably also a difference between caregiver behavior and patient behavior that has not yet been truly studied.

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  • Michael Dayton

    As an advocate for rare diseases, I would like to pass along some helpful information The Rare Genomics Institute, a non-profit organization, focuses on helping patients and families with rare/orphan diseases that might benefit from genome sequencing. Rare Genomics Institute helps patients with gaining access to genome sequencing services, support, and funding. Washington University School of Medicine’s Genomics and Pathology Services and the Rare Genomics Institute are awarding grants for the sequencing of 99 exomes to rare disease advocacy groups. The grants will be free of charge to the rare diseases community. Interested applicants should submit letters of interest by April 2. If you are interested, or know of someone who might benefit, visit the web site for this program at the Rare Genomics Institute site: http://www.raregenomics.org/rare99x
    Mike Dayton

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