NORD has submitted the following Letter to the Editor of the Journal of the American Medical Association (JAMA):
The recent study evaluating the evidence supporting FDA approval of novel therapeutic agents has generated debate about FDA exercise of flexibility and judgment in approving new drugs. FDA Commissioner Margaret Hamburg, MD, wrote a blog explaining why FDA supports a flexible approach to drug development. read more >
Stephen C. Groft, PharmD, the Director of the NIH Office of Rare Diseases Research since its inception, will retire effective February 8. NORD honored Dr. Groft at our 30th anniversary celebration in May 2013. We reprint here the text from our gala program book. read more >
Genome editing is a process that involves cutting out mutations in DNA and replacing them with new genetic material. This is an exciting idea because if it can be refined to a level that is safe and effective, the prospect of eradicating genetic diseases is no longer science fiction. 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 >