Latest Article on Breakthrough Therapy Designations in Nature Biotechnology

As of last month, 41 products have been granted breakthrough therapy designations by the US Food and Drug Administration-drugs against cancer, hepatitis C and monogenetic diseases predominate.

In 2012, the US Food and Drug Administration (FDA) created a new expedited pathway called 'breakthrough therapy designation' (BTD) to facilitate rapid approval of therapies that have shown substantial activity in early trials. As of March 7, the FDA had received 155 BTD applications, of which 41 have been granted BTDs and 3 drugs with 4 BTDs have received marketing authorizations. The new pathway promises faster approval and earlier access to therapies for unmet needs. Despite the popularity of this new process, several areas of uncertainty still exist, especially the data requirements for the designation, the definition of "substantial improvement" and "existing therapies," the scenarios for handling product failures, and the potential impact of the BTD on pricing and reimbursement.

NOTE: As of April 29, 2014, 5 drugs with 6 BTDs have been approved by the FDA. The latest BTDs to be approved are Zykadia for NSCLC and Arzerra for first-line CLL.

The latest and final guidance on BTD was published by the FDA in May 2014. The PDF can be downloaded from here.Breakthrough Therapy Guidance by FDA FINAL