Orphan diseases: Connecting with caregivers
Tales of love, hope, and community by rare disease caregivers
I am thrilled to announce that Siren has published Uncommon Challenges, Shared Journeys, a collection of 13 personal stories offering intimate views into the lives of families affected by rare disorders.
The narratives reveal how parents-mothers, in particular-play critical roles in obtaining a correct diagnosis and appropriate treatment for children with rare diseases.
The stories also show how caregivers in the rare disease community seek and use information differently from other groups and have many unmet needs for education and support.
My daughter has a rare disorder, and I compiled the anthology and shared my own family's story.
Dr. Timothy Cot, director of the FDA's Office of Orphan Products Development, says the book "strikes straight to the heart of the matter" by focusing on patients and their families.
"Academics talk molecules, industry talks revenues, government talks regulations, but parents' words of their heartbreaking love for their children with rare diseases are the truest words of all. I recommend this book to anyone who wants to understand the power that's driving the orphan drug movement." (For more on orphan drugs, see Forecasting for orphan drugs: The data challenge'.)
Rare disorders, also called orphan diseases, are defined as conditions that affect fewer than 200,000 people in the US.
Despite the seemingly low number, such disorders affect one in 10 Americans, 80% of them children.
The category includes cystic fibrosis, hemophilia, muscular dystrophy, and all childhood cancers as well as numerous diseases with lesser-known names. (To learn how one patient increased awareness and compliance around hemophilia, see Collaborative care: Improving information exchange and adherence'.)
Rare diseases are often difficult to diagnose, and approximately 70% of treatment therapies are prescribed off-label.
I believe the pharmaceutical industry can provide significant educational services to parents of children with rare disorders, but must realize that conventional ways of building relationships are ineffective with this group.
Mothers dominate this community, mothers who are fighting for their children's lives, in an environment offering scarce information and resources.
They are unusually driven, and the Internet has made them unusually empowered.
They are avid researchers who become experts in their children's diseases.
They are critical analysts and ardent advocates who explore all treatments and fight for their children's right to access them.
Pharma marketers must re-think how they approach this community and explore new ways of meeting their needs. (For more on patient-pharma collaboration, see The pharma connection: How to foster patient-physician collaboration'.)
Uncommon Challenges, Shared Journeys is available throughAmazon.comfor $16.00 per copy or through Siren Interactive for bulk orders.
All profits will be distributed among the rare disease groups represented in the book.
Stories were contributed by:Catherine Calhoun,Jordana Holovach,Janis Marie,Laura Buchanan,Lori and Matt Sames,Julie Loeffler,Terri Granard,Dominique Friend,Nicole and Bill Morris,Joan Bellontine,Dayna Fladhammer,andPat Furlong.
To learn more about the authors and their charities, go to the book website:http://press.sireninteractive.com.
Wendy White is founder and president of Siren Interactive.