Great article on FDA Breakthrough Therapy Designat

Great article on FDA Breakthrough Therapy Designation

http://www.elsevierbi.com/~/media/Supporting%...162012.pdf

Here is some of the most relevant info for CTIX:

B.

Criteria for Breakthrough Designation

A profound therapeutic breakthrough was defined by Sharma and Schilsky as one that “fundamentally alters the way oncologists think about a disease in terms of the prognosis, treatment options, and quality of life of our patients”

While future breakthroughs may be readily apparent to those familiar with the disease they aim to treat, they may be less apparent to others outside that particular field. This pathway should not be viewed as a default pathway for all oncology drugs. Defining a threshold of evidence required to obtain Breakthrough designation is necessary to provide some degree of consistency and predictability to the process. Because it may be unrealistic and restrictive to define a breakthrough exclusively in quantitative terms based on early results such as response rates relative to existing therapies, we have proposed qualitative criteria to be met for Breakthrough designation. The qualitative criteria discussed below are contextual; ultimately, the designation of a new drug as a potential Breakthrough Therapy should be determined on a case-by-case basis by those with relevant expertise.

1. The diseases under study will be serious (either debilitating or life-threatening) and no established SoC exists or the current accepted SoC yields poor clinical outcomes (such as low response rates, lack of durability, limited survival, inadequate symptom control, severe acute or chronic effects, reduced quality of life).

2. Breakthrough designation should be based on compelling early evidence suggesting major clinically meaningful improvement over existing therapies in a defined disease setting.

The potential Breakthrough Therapy under consideration could be designated on the basis of early data suggesting substantial clinical efficacy (e.g. quality or rate of response and/or duration):
i. Early clinical studies should suggest a substantial clinically meaningful improvement over a similarly defined concurrent or historical comparator.
ii. Acceptable safety in a reasonable number of treated patients (number of patients calibrated to incidence/prevalence of disease, depends on understanding of mechanism and expected toxicity).

The potential Breakthrough Therapy under consideration could be designated on the basis of early data suggesting a superior clinical therapeutic index compared to SoC in a similarly defined population.
i. Should exceed or clearly maintain comparable efficacy
ii. Superior safety or tolerability advantage is the key consideration

3. The potential Breakthrough Therapy under consideration will typically have a compelling scientific rationale and promising mechanism of action, such as targeting a molecular driver of a biologically characterized disease (e.g., ALK-positive subset of lung cancer).