• Kisqali approved under Real Time Oncology Review Program
• One of two new programs intended to make the development of cancer drugs more effecient
• Approval came less than one month after submission

The U.S. FDA annouced on 7/18/18 that it had approved Novartis' Kisqali (ribociclib) in combination with an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, as initial endocrine-based therapy. The FDA also approved Kisqali in combination with fulvestrant for the treatment of postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine based therapy or following disease progression on endocrine therapy.

This is the first approval the FDA has granted as part of two new programs launched last year. The new programs are intended to make the development and review of cancer drugs more efficient while improving the FDA's safety and efficacy standards. The two programs are, Real Time Oncology Review and templated Assessment Aid.

Under the Real Time Oncology Review program he FDA was able to start evaluating Kisqali clinical data as soon as the trial results become available. This enabled FDA to be ready to approve the new indication upon filing of a formal application with the Agency. The approval came less than one month after the June 28 submission date and several months ahead of the goal date.

The Real Time Oncology Review pathway allows the FDA to begin reviewing data after clinical trial results become available and the database is locked - before the information is formally submitted to the FDA. This allows the FDA to begin its analysis of the data earlier, and provide feedback to the sponsor on how they can most effectively analyze the data to answer key regulatory questions.

The templated Assessment Aid helps the applicant organize its submission into a structured format to facilitate FDA’s review of the application.By using a structured template, the FDA is able to layer its assessment into the same file submitted by the sponsor, allowing this annotated application to serve as the document that contains the FDA review.

In reference to the approval, FDA commissioner, Scott Gottlieb said, “With this approval, we’ve demonstrated some of the benefits of the new programs that we’re piloting for our review of cancer drugs, to improve regulatory efficiency while enhancing the process for evaluating the data submitted to us. This shows that, with smart policy approaches, we can gain efficiency while also improving the rigor of our process. These new programs were designed to reduce some of the administrative issues that can add to the time and cost of the review process, including the staffing burdens on the FDA.”

Kisqali was first approved in March 2017 for use with an AI ( aromatase inhibitor) to treat HR-positive, HER2-negative breast cancer in post-menopausal women whose cancer is advanced or has spread to other parts of the body.

“The approval adds a new treatment choice for patients with breast cancer,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “We are committed to continuing to bring more treatment options to patients.”