Breakthrough Therapy For Patients With Chronic Lymphocytic Leukemia

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Breakthrough Therapy Designation is introduced as a new designation with the passage of the FDA Safety and Innovation Act of 2012 (FDASIA) [1]. According to section 902 of the FDASIA, the general criteria according to which this new designation can be applied are (1) serious or life-threatening disease or condition and (2) the drug also demonstrates substantial improvement over existing therapies on one or more clinically significant endpoints. Once a drug is designated as a Breakthrough Therapy, the FDA and the drug sponsor work closely to determine the most efficient pathway for generating additional evidence about safety and efficacy. As of March 31, 2015, a total of 293 requests for Breakthrough Therapy designation have been submitted, out of which 82 requests are granted [2].
This document outlines the application process for Breakthrough Therapy designation for novel drugs. It then proceeds with outlining the supporting data needed to apply for a Breakthrough Therapy designation for a novel anti-cancer molecule that has evidence of efficacy to treat patients with Chronic Lymphocytic Leukemia.
When to apply for designation request
The Breakthrough Therapy designation is granted on the basis of evaluating preliminary clinical evidence obtained from clinical trial data in humans. The data from animal studies or studies conducted in vitro are insufficient to defend this Breakthrough Therapy designation [1] . Therefore, a sponsor usually submits a Breakthrough

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