CRND Clinical-Translational Seminar March 9, 2012 - Mary Dunkle

March 9, 2012 - Mary Dunkle presents a seminar on "The Role of the Rare Disease Patient Community."

 

"To remain ignorant of things that happened before you were born is to remain a child."

Cicero

Ms. Mary Dunkle is the Vice-President for Communications for the National Organization for Rare Diseases (NORD), a nonprofit confederation of many voluntary health organizations, which serve rare disease patients. NORD's mission is to advance the identification, treatment, and cure of rare diseases through education, advocacy, research, and service. Acting in the critical role of VP for Communications, Dunkle has oversight for all of NORD's communications, including the website, publications, educational activities, and diverse research programs.

Dunkle's presentation to CRND's students introduced a crucial dimension to the CTSS survey of health networks in rare diseases: an historical review of the early days of NORD, including the evolution of the national rare disease coalition and its seminal role in the passage of The Orphan Drug Act. This historical account, together with a synopsis of NORD's extensive contemporary resources for patients and families, patient organizations, medical professionals, and many other communities of interest, gave students a sense of how miraculously the rare disease landscape has been transformed and the shoulders on which they stand today observing a hopeful future.

Prior to NORD’s founding in 1983, the world of rare disease patient support comprised numerous relatively disconnected family groups. Typically, these small groups were centered around individual rare diseases or small clusters of related disorders. Worse yet, in the decade before NORD came into existence, only 10 new rare disease therapies made it to market. According to Dunkle, things started to change when these disparate groups began to recognize the common issues shared by all patients with rare diseases - isolation and the need to connect with others who have the same disease, extensive and painful diagnostic delays, inadequate research and funding, very limited therapies and access to services, a paucity of medical experts, lack of understanding in the mainstream community, and the absence of a broad communication network to facilitate information gathering and dissemination.

Dunkle's undergraduate audience was engrossed as she traced a story that unfolded more than a decade before they were born. Like many amazing stories about movements that change the world, this one started with a desperate need and a loving parent.

In 1999, when Mary Dunkle arrived at NORD, she had the honor of working with the organization's founder, Mrs. Abbey Meyers. Years earlier, Mrs. Meyers had started out as was a volunteer leader of the Tourette's Syndrome Association. Her son had Tourette's Syndrome and was being treated successfully as a result of a clinical trial that made a medication available to him. Unfortunately, the clinical trial was designed to explore the efficacy of the drug in treating another condition. When the drug proved inapplicable for the more prevalent target disease, the pharmaceutical company stopped making it available. From a commercial standpoint, it didn't pay to make the drug for the small market Abbey’s son represented. At that point, Mrs. Meyers began reaching out to rare disease folks across the spectrum and across the nation. She started asking them if they had experienced some of the same problems. In the brief span of a year, this outreach and robust exchange led to the formation of the national coalition and also a change in the pharmaceutical company's stance regarding medication for Abbey's son. Throughout the period of the early ‘80’s, NORD and its confederates worked tirelessly to advocate for the passage of The Orphan Drug Act of 1983. They succeeded in this too.

This landmark Orphan Drug Act has greatly incentivized the development of new treatments for rare diseases in the past three decades. And, Mary Dunkle expects that much more is promised in the future. She cited over 300 drugs that have been brought to market for rare diseases and another 2,000 that are "in the pipeline."

Another extraordinary event of this early period is worth noting. Dunkle shared with her students the story of the wonders wrought by Mr. Jack Klugman, star of the highly popular Quincy, M.E. medical drama (1976-1983). Jack Klugman, the actor who played the medical examiner, had a brother who was dying of a rare blood disease. Klugman was attuned to the troubles of rare disease patients. The show aired a story about a patient (much like Ms. Meyer's son) who was confronted with the loss of a life-saving medication that had helped him. This dramatic episode produced such a massive response on the part of the public that the subsequent publicity attracted congression attention. Mary Dunkle summarized, "We've got clippings in our files showing Jack Klugman testifying to Congress about the need for an Orphan Drug Act and it really was this - you know people talk about the power of the popular press - [this] T.V. show that really, really helped make it happen."

Since its inception, NORD's advocacy for patients and families has been instrumental in bringing vital recognition to the condition of tens of millions of Americans. This increased attention and focused effort has born fruit in expanded congressional and legislative support, greatly enlarged funding, and significant interactions between institutional networks to produce scientific, medical, academic, commercial and community partnerships. In 2002, NORD played a key role in the passage of the Rare Diseases Act, which established the Office of Rare Diseases at the National Institutes of Health, a central hub for information, research, services and innovation for the entire rare disease community.

When Mary Dunkle started with NORD, many of the founders and trail-blazing physicians involved with the early rare disease movement were nearing retirement. Dunkle recalled there was a very real concern on the part of many that the exodus of the senior generation might make the organization vulnerable and potentially re-marginalize the community. Happily, this did not come to pass. A new generation accepted the challenge of working for rare disease patients and their organizations. That generation has moved the agenda to encompass global efforts and networks, creating new synergies in research and patient care. As she looked over the assembled CRND students, Dunkle warmly expressed her delight in a new generation preparing for the work ahead.

 

For information about NORD, please visit their website : http://www.rarediseases.org/

 

 

 

 

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