Chevy Chase, MD (PRWEB) April 13, 2014
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, 2014 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.
The NOVEL team analyzed the BTDs, assessing the available data on safety and efficacy, mechanisms of action, type of molecule and indications. The analysis also included systematic review of available clinical and marketing data for the three approved products that have received BTDs.
This analysis was published in the April 2014 issue of Nature Biotechnology. The latest full list of breakthrough therapy designations is available at http://www.novelhealthstrategies.com/breakthrough.htm.
The article covers the following topics:
This study was also peer-reviewed and selected for presentations at two upcoming conferences:
Details of these presentations is available upon request.
For more details about the BTDs or to request discussion with the author, please email at info(at)novelhealthstrategies(dot)com