National Organization of Rare Diseases (NORD), USA

The National Organization of Rare Diseases (NORD) backs legislation introduced in the Senate and House of Representatives to modernize and expand the 'Accelerated Approval' pathway of the  Food and Drug Administration (FDA). The two bills are entitled 'Transforming the Regulatory Environment to Accelerate Access to Treatments' (TREAT), in the Senate, and 'Faster Access to Specialized Treatments' (FAST), in the House. NORD believes that TREAT and FAST will speed up the development of treatments for rare diseases, while still maintaining standards for safety and efficacy.  NORD did not support an earlier proposal, known as ULTRA, that, says NORD, went too far in reducing the requirement to demonstrate safety and efficacy. ULTRA has been replaced by FAST, which includes language suggested by NORD.