Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale helped inspire a law designed to stimulate the creation of new treatments for rare diseases and spur economic development in North Carolina. House Bill 823, signed into law by Governor McCrory on August 6, will establish an Advisory Council on Rare Diseases within the School of Medicine of the University of North Carolina at Chapel Hill (UNC).

"Rare disease impacts more people than HIV and cancer combined—about one million people just in North Carolina," said Taylor's Tale president Sharon King, who played a key role in spearheading the new legislation after presenting to legislators and other leaders in late March. "With its strong research universities, biotechnology companies and large patient population, this state has an incredible opportunity to change the future for millions fighting a rare disease."

House Bill 823, filed in the North Carolina General Assembly on April 14 and passed unanimously on July 28, establishes an Advisory Council on Rare Diseases to provide guidance on research, diagnosis, treatment and education related to rare disease. The Council's membership will include a broad spectrum of rare disease advocates including health care professionals, representatives of the state's academic research institutions, a rare disease survivor and a rare disease foundation representative.

Supporters point to the current lack of treatments for rare diseases, many of them with tragic consequences, and spiraling health care costs as reasons why this kind of action is not only advisable, but necessary.

"The creation of the Rare Disease Advisory Council represents a unique opportunity for the UNC School of Medicine and others in North Carolina to serve the people of the state and beyond," said Bruce Cairns, director of the North Carolina Jaycee Burn Center at UNC-Chapel Hill. "We strongly believe a partnership between advocacy groups including Taylor's Tale, academic medical centers, the National Institutes of Health and others keenly interested in the diagnosis and treatment of rare diseases can be a model for the nation as we tackle some of the most challenging diseases affecting our citizens. We are grateful that the General Assembly has passed the bill and that Governor McCrory has now signed it. We are ready to get to work."

The message that progress can and should be made in developing treatments for rare diseases has resonated with legislators and other leaders since the initial filing of the bill on April 14. House Bill 823 received strong bipartisan support, and each version passed unanimously. The bill was co-sponsored by Representatives Carney, Avila, Bishop, Earle, Farmer-Butterfield, Fisher, Floyd, Glazier, Hamilton, Harrison, Insko, Jeter, R. Johnson, R. Moore, Pierce, Richardson and Wray and Senators Hartsell and Pate.

"Providing better treatments can improve quality of and possibly prolong life for rare disease patients, ease the burden on our health care system and reduce the economic burden of rare disease that affects us all," said Rep. Becky Carney, a primary sponsor of the bill. "Building partnerships and engaging the various sectors in rare disease is the quickest way to success, and North Carolina can and should be a leader in this effort."

King noted that while Taylor's Tale initiated the legislation, the Advisory Council on Rare Diseases is the result of numerous advocates driven to build a better future for the rare disease community and the state of North Carolina. "This was a joint effort that couldn't have happened without the dedication and hard work of many people," said King.

Taylor's Tale is named in honor of King's daughter, Taylor, 16, who was diagnosed with infantile Batten disease in 2006. Batten disease causes vision loss, loss of cognitive skills and seizures. Progressively, children suffer loss of memory and speech until they are mentally and physically incapacitated, wheelchair bound, and then bedridden. With no treatment or cure, Batten disease is always fatal.

According to the National Organization for Rare Disorders, one in 10 Americans (approximately one million people in North Carolina and 30 million people in the U.S.) and an estimated 350 million people worldwide have a rare disease. In the U.S., a condition is considered "rare" if it affects fewer than 200,000 persons combined in a particular rare disease group.