Transformation is happening – FDA and rare disease!
We have gotten so accustomed to hearing criticisms of the The U.S. Food and Drug Administration which, like most criticism, is sometimes valid, sometimes not). Here are two new developments worth talking about! First, in an admirable show of cooperation, the FDA reached an agreement with the European Medicines Agency to allow sponsors of approved orphan drug and biologic products to submit a single annual report to both agencies. Read more here:–fda-and-european-medicines-agency-agree-to-accept-a-single-orphan-drug-designation-annual-report-85607477.html
And the FDA and NIH have announced too important collaborative efforts designed to speed both translational research (which is one step before clinical) and regulatory science – both of which are directly related to the speed with which new therapies reach the clinic and actual patients. Read more here:
Jonathan Jacoby