NORD’s Assistant Director of Public Policy, Paul Melmeyer, authored this great piece (below) in the March 2015 issue of the NCC Collaborator, the monthly publication from the National Coordinating Center for the Genetic Service Collaboratives (NCC). NCC focuses on bringing quality genetic and newborn screening services to local communities, and building bridges between public health, primary care/ Medical Home, geneticists and other specialists, and families and consumers. You can read the piece and March issue online here.
Since the passage of the Affordable Care Act in 2010 and the subsequent Supreme Court decision allowing states to decide whether to expand their Medicaid programs, decisions about healthcare coverage and quality standards for patients with rare diseases are increasingly being made at the state level. From public health initiatives, such as newborn screening programs, to insurance plan structure and coverage regulations, state governments play an integral role in facilitating the access of quality, affordable care for individuals with rare diseases.
For 31 years, the National Organization for Rare Disorders (NORD) has served as America’s leading patient advocacy organization for patients with rare diseases. Following the passage of the Orphan Drug Act in 1983, NORD has been involved in numerous Federal policy initiatives, including the Rare Diseases Act of 2002, the Affordable Care Act, and the Food and Drug Administration Safety and Innovation Act (FDASIA), among others.
Beyond our policy efforts, NORD represents over 220 organizations for individuals with rare diseases and provides education and coordination services for patients and their families. read more >
June 10th was an emotional – but very educational – day for everyone who attended the Food and Drug Administration’s public meeting on Patient-Focused Drug Development for Inborn Errors of Metabolism. read more >
Leleah Robinson, NORD’s Special Assistant to the Vice President of Public Policy, recently attended the FDA public workshops. This blog provides her insight into that event.
In July 2012, President Obama signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) which requires the FDA to hold at least one public meeting to “encourage and accelerate the development of new therapies for pediatric rare diseases”. read more >
The terms risk, benefit, effectiveness, and safety are key to the approval of all new medical treatments (and of previously approved treatments for new uses). However, there is a vast divide between the concepts of relative risk and relative benefit (for a well defined group of patients) and the concepts of effectiveness and safety. The distinctions between these concepts are particularly important in the world of rare diseases. read more >
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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