The Food and Drug Administration’s Office of Orphan Products Development (OOPD), in collaboration with the FDA Center for Drug Evaluation and Research (CDER), has launched a web-based educational tool for rare disease patients, advocacy groups, researchers and industry on various FDA-related topics. read more >
CAMBRIDGE, MA and DANBURY, CT—-Genzyme, a Sanofi company, and the National Organization for Rare Disorders (NORD) today announced the creation of a fund to pay for standard diagnostic testing for people with mysterious, undiagnosed medical conditions. The fund will help those who have applied to the National Institutes of Health (NIH) Undiagnosed Diseases Program but who cannot afford the basic medical tests needed to make them eligible to participate in the NIH program. read more >
Posted at September 18, 2013 7:50 pm by Leleah Robinson
Government agencies, industry, and patient advocacy groups all agree that a patient-centered perspective is vital during the processes of drug development, clinical trials, benefit/risk assessment, and regulatory review. However, legal barriers and proprietary constraints complicate when patients can be brought to the table and integrate their input.
At the FDA’s annual Patient Network meeting, “Demystifying FDA: An Exploration in Drug Development”, there was a concerted effort among all stakeholders to increase collaboration. read more >
Most mornings, my commute is a 12-minute drive from my home in the hills above Danbury, Connecticut, to the nearby NORD offices. But Tuesday’s commute started early – 5:30 a.m. – with a train ride into New York City and a brisk walk up 42nd Street to the NASDAQ Marketplace in Times Square. read more >