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NC Rare Disease Network

About the NC Rare Disease Advisory Council

Mission

The mission of the North Carolina Rare Disease Network is to improve the lives of millions of people with one of the 7000 devastating rare diseases by creating a unique collaborative partnership between patients, providers, scientists, agencies and industry.

Introduction

House Bill 823, An Act Establishing the Advisory Council on Rare Diseases within the School of Medicine at UNC Chapel Hill, was ratified by the General Assembly on the July 29, 2015. The Governor signed this legislation on August 5, 2015. The legislation received bi-partisan support and passed unanimously through the NC House and Senate. http://www.ncleg.net/Sessions/2015/Bills/House/PDF/H823v4.pdf

This established the Advisory Council on Rare Diseases that will be housed initially in the School of Medicine of the University of North Carolina at Chapel Hill to advise the Governor, the Secretary, and the General Assembly on research, diagnosis, treatment, and education relating to rare diseases.

For purposes of this council, rare disease is defined as one in 10 Americans or 10% of the U.S. population are living with rare disease. In the United States, a condition is considered “rare” if it affects fewer than 200,000 persons combined in a particular rare disease group. Approximately 50% of the people affected by rare disease are children and 80% of rare diseases are genetic in origin, and thus are present throughout a person’s life, even if symptoms do not immediately appear.

First and foremost, this council and the recommended outcomes will always serve the citizens of North Carolina.

Powers and Duties of the Advisory Council

The advisory council shall have the following powers and duties:

  1. Advise on coordinating statewide efforts for the study of the incidence of rare diseases within the State and the status of the rare disease community.
  2. Report to the Secretary, the Governor, and the Joint Legislative Oversight Committee on Health and Human Services on behalf of the General Assembly annually, on the activities of the advisory council and its findings and recommendations regarding rare disease research and care in North Carolina, including any recommendations for statutory changes and amendments to the structure, organization, and powers or duties of the advisory council.”

Initially, members of the Advisory Council include research and clinical faculty from University of North Carolina School of Medicine and Duke University.

Objectives

To ensure the citizens of North Carolina by investigating and recommending changes to existing structures or policies within the state that could better serve North Carolinians and their families, care givers, medical providers, researchers, therapeutics concerning rare disease.

  1. The short term goal is to bring together health care providers (clinicians, investigators), from North Carolina Universities and healthcare systems, NC Department of Health and Human Services (NC DHHs) and patient advocacy groups in a collaborative effort to discuss the best strategies to improve understanding of the diagnosis, treatment and impact of rare diseases- first for the citizens of North Carolina, then the nation.
  2. Collect data on rare diseases including the cost and economic impact for people in North Carolina.
  3. Identify technology platforms that are relevant for rare disease treatments.
  4. Report findings to the UNC SOM who will report back to DHHS and the legislature with the goal to address changes in rare disease policies that will positively impact citizens and the state on an annual basis.
  5. Coordinate rare disease collaborations across the state (including academic medical centers, investigators, relevant funding agencies, biotech companies, and advocacy groups).
  6. Highlight collaborations and synergy between clinicians and investigators from Duke, UNC, NCSU initially, academic medical centers, state and federal funding agencies, biotech and pharmaceutical companies and North Carolina Department of Health and Human Services.

The overall goal of the Council is to leverage the unique resources in North Carolina to create a state based, rare disease collaborative network that serves as a model for the nation.

Administrative Structure of the Council

Dr. Bruce Cairns, M.D., Chair of the Advisory Council will provide the leadership to the council members.

Tara J. Britt, Associate Chair of the Council, provides funding opportunities and administrative oversight including corporate partnership opportunities and economic development.