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PCORI Appoints First Members of New Advisory Panel on Rare Disease

Panel’s Expertise Will Guide Funding Priorities and Community Engagement 

Contact: Christine Stencel, Associate Director of Media Relations (202-570-9275 or cstencel@pcori.org)

Washington, D.C. (March 25, 2014) — The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved 13 people as  members of PCORI’s new Advisory Panel on Rare Disease. It is one of two new advisory panels for which the Board approved members at its meeting today; the other focuses on clinical trials.

The diverse group of panelists will apply their experience and expertise to advising PCORI on its research priorities in the area of rare disease, as well as on engaging with the rare disease research community. Rare diseases are defined in the United States as conditions that occur in fewer than 200,000 people.

“Although they’re called rare, these are conditions that collectively affect millions of Americans and their families and they merit our attention as we seek to support comparative effectiveness research on patients’ most pressing unanswered questions,” said Bryan Luce, PhD, MBA, PCORI’s Chief Science Officer. “We’re pleased that so many qualified individuals wanted to lend their experience and deep knowledge to ensure that we support high-quality research on rare conditions and we’ll benefit greatly from the insights of this stellar group of individuals.”

The new panelists represent a range of stakeholder groups and perspectives, including people with rare diseases, family caregivers, clinicians, drug and device makers, and researchers, among others. More than a third represent patients and patient advocates. A list of the new panelists, with brief biographies and conflict of interest disclosures, is available on PCORI’s website.

The panel members and three alternates were selected from 129 applicants. PCORI staff thoroughly evaluated every candidate’s personal experience and qualifications in the area of rare disease as well as their ability to contribute to the panel’s advisory tasks and responsibilities. More information about PCORI’s process for reviewing and recommending advisory panel nominees is available on its website.

The panel’s composition will enable it to carry out a variety of tasks, including providing input on the research needs of the rare disease community and advising on strategies for disseminating study findings.  However, given that the National Institutes of Health lists more than 6,800 rare conditions, the panel will assist PCORI in identifying experts to serve on ad hoc advisory panels as needed to consider research issues related to particular conditions. More details about the panel’s responsibilities and activities can be found in its charter.

PCORI’s authorizing legislation allows it to appoint permanent or ad hoc advisory panels to assist it in identifying research priorities and establishing its research agenda and specifically called on the institute to establish an expert panel on rare disease. While they are not policy-making groups, their advice and recommendations inform the work and decisions of PCORI’s staff, Board and committees.

The panel members have been notified of their appointment to the panel and are expected to respond to their invitations today. If needed, alternate members will be selected from the list also approved by the Board today. The Advisory Panel on Rare Disease will convene for its first meeting on Wednesday, April 30, in Washington, D.C.

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About PCORI

The Patient-Centered Outcomes Research Institute (PCORI) is an independent, non-profit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed healthcare decisions. PCORI is committed to continuously seeking input from a broad range of stakeholders to guide its work. More information is available at www.pcori.org.