The next several weeks are a period of critical importance for the rare disease community in the U.S. If Congress isn’t able to reach agreement on a budget deficit reduction plan by January, across-the-board funding cuts of 8.2% will go into effect for FDA, NIH and other federal agencies. This would have devastating effects on [...]
The 1st US Conference on Rare Diseases and Orphan Products, co-organised by NORD (National Organization for Rare Disorders, USA) and the DIA (Drug Information Association), took place in Washington DC from 11 – 13 October 2011. Organised in partnership with the FDA, NIH and EURORDIS, this first conference was a huge success, assembling over 250 [...]
The summer of 2010 saw remarkable activity related to the development of treatments for rare diseases. The U.S. Food and Drug Administration hosted a two-day public hearing for patient advocates, industry, academic researchers and others to voice their views on current procedures and possible ways to improve them.
The Senate HELP (Health, Education, Labor and [...]
Seriously ill patients who have exhausted all treatment options often seek access to investigational drugs by way of government-sanctioned “expanded access programs”. Recently, the U.S. Food and Drug Administration (FDA) implemented new regulations to clarify and increase patient access to investigational drugs.
However, even with the new rules, this topic is more complex than it may [...]
