Rare Disease Report

NORD News for December - The Voice of the Community

DECEMBER 16, 2015
Guest post by NORD
News from NORD and its Member Organizations

FDA Awards NORD $250,000 Grant to Support Development of Natural History Studies  

The U.S. Food and Drug Administration (FDA) has awarded a $250,000 grant to the National Organization for Rare Disorders (NORD) to develop natural history studies to support rare disease research. NORD will use the grant to develop studies for 20 rare diseases, to be selected with a lottery system. Patient organizations can enter the lottery by applying on the NORD website.
“NORD’s Natural History Study Project tackles one of the greatest needs and an inherent challenge for the rare disease community: having enough longitudinal data to help medical researchers better understand how these diseases develop and progress over time,” said NORD President and CEO Peter L. Saltonstall. “The FDA’s support enables us to expand this important program with our shared goal of supporting patient organizations and helping patients.”

NORD’s registry platform, which was launched last year, was developed with input from patients and patient organizations, researchers and regulators, consistent with NORD’s mission to support research on all 7,000 rare diseases.

“Natural history studies can yield vital information that is essential to clinical trial design such as biomarkers, demographics, genetic and environmental variables, disease progression, and patient perspectives on the challenges of living with the disease,” said NORD Research Program Manager Jacqueline Kraska.

In addition to the 20 natural history studies, NORD will work with patient groups and the FDA to develop a registry toolkit with best-practice tools and templates to help organizations initiate and conduct natural history studies on additional rare diseases in the future.  

Join NORD in Educating Legislators and Other State Officials with “State House Events”   

As it has done for the past several years, NORD will again work with volunteers within states to organize and host “State House Events” for Rare Disease Day 2016. These events are intended to enhance awareness and understanding of rare disease challenges among state officials.  They take place on or around Rare Disease Day, which is observed on the last day of February each year.
“Currently, we are working with volunteers in 29 states to support their efforts to organize events for Rare Disease Day 2016,” said Kristen Angell, the staff member who coordinates this service for NORD. “We’re hearing from many others who are interested, and we anticipate that there will be several other states joining the initiative.”

The events have taken on particular importance in recent years because many important healthcare decisions now are made at the state level, such as those related to newborn screening and prescription drug cost-sharing.

The events typically take place at the state capitol or legislative office building. They bring together patient advocates, researchers, clinicians and others to speak and meet with legislators. The purpose is to address key issues of importance to all and facilitate conversation on specific issues within states. 

This year, volunteers will be able to use information from NORD’s first-ever State Policy Progress Report, which was published recently. In addition, NORD’s Rare Action Network, launched in 2015, focuses on public policy at the state level and provides an additional source of support for those organizing State House Events.  

While each event is designed and drive by volunteers within the state, NORD helps assure a successful event by providing guidance and support. Anyone interested in volunteering to lead or help with an event may contact Angell (kangell@rarediseases.org) to learn what is involved and whether an event is currently being organized in their state.

In addition to the State House Events, NORD provides guidance and resources to students and others organizing Rare Disease Day events on campuses across the U.S. To request NORD’s help in arranging for patient speakers, handouts and other resources for Rare Disease Day campus events, write to bhollister@rarediseases.org.

A representative of NORD will be speaking at the 2016 Rare Disease Day event hosted by the National Institutes of Health (NIH) at the NIH Clinical Center.  Registration is now open for the NIH event.

Watch the official U.S. Rare Disease Day website hosted by NORD for additional news related to Rare Disease Day 2016, and be sure to follow Rare Disease Day US on Facebook and Twitter, using #RareDiseaseDay. 

NORD Publishes Two New Rare Disease Guides for Physicians

Two new topics have been added to NORD’s free online Physician Guide series.  The NORD Physician Guide to Paroxysmal Nocturnal Hemoglobinuria  and the NORD Physician Guide to atypical Hemolytic Uremic Syndrome were published this week and are free and available to all.

The PNH Guide was authored by Carlos M. De Castro III, MD, Professor of Medicine, Duke University School of Medicine, and Wendell Rosse, MD, Florence McAlister Professor Emeritus of Medicine, Duke University School of Medicine. The aHUS Guide was reviewed by Larry Greenbaum, MD, PhD, Marcus Professor of Pediatrics, Emory University School of Medicine and Children’s Healthcare of Atlanta. Both guides were made possible by educational grants from Alexion.

The physician guides are part of NORD’s educational outreach to medical professionals and students preparing for healthcare careers. During 2015, more than 25,000 medical professionals from 180 countries worldwide visited the website.

Join NORD in Action to Support Advancing Research for Neurological Diseases Act

Anyone interested in supporting the Advancing Research for Neurological Diseases Act may show support via an action alert on the NORD website. This legislation is intended to help the Centers for Disease Control and Prevention track the incidence and prevalence of neurological diseases to better understand certain aspects of these diseases.

NORD Rare Impact Awards Celebration to Take Place on May 17th

NORD is proud to host the 2016 Rare Impact Awards to bring together the rare disease community and to honor and celebrate the year’s outstanding achievements. This event will take place on Tuesday, May 17, at the Warner Theatre in Washington DC. Registration will open soon. Photos and information from the 2015 event may be viewed on the NORD website.

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