20 days from application to approval! FDA's new pilot program to accelerate the evaluation of anticancer drugs

Today, the U.S FDA approved kiscali (ribociclib) and aromatase inhibitor (AI) of Novartis as the initial endocrine therapy for premenopausal or postmenopausal women with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative advanced or transitional breast cancer The FDA also approved kisquali in combination with fulvestrant as an initial endocrine therapy or post progression endocrine therapy for postmenopausal women with HR positive, HER2 negative advanced or metastatic breast cancer It is worth mentioning that this is the first approval granted by FDA as part of two new pilot projects announced earlier this year The two pilot projects are real-time Oncology Review and new assessment aid The former enables the FDA to start the review before the clinical trial results are officially submitted; the latter facilitates the application company to submit the results in a structured format to speed up the review of FDA These measures are aimed at making research and development and evaluation of cancer drugs more effective, while improving FDA's strict criteria for evaluating efficacy and safety Through this real-time review, FDA can start to evaluate clinical data immediately after the test results appear, so that FDA can be ready for approval when submitting a formal application Breast cancer is the most common form of cancer in the United States The National Cancer Institute of the National Institutes of Health (NIH) estimates that about 266120 women will be diagnosed with breast cancer this year, 40920 of whom will die from the disease About 72% of breast cancer patients have HR positive and HER2 negative tumors They need effective treatment to alleviate the disease Kisquali is a selective cyclin dependent kinase inhibitor It is a kind of drug that can help slow down the progress of cancer by inhibiting two kinds of cyclin dependent kinase 4 and kinase 6 (CDK4 / 6) When these proteins are overactivated, they can cause cancer cells to grow and divide too fast Targeting CDK4 / 6 with high accuracy is expected to avoid uncontrolled replication of cancer cells The drug was first approved in March 2017 in combination with aromatase inhibitors for the treatment of postmenopausal women with HR positive, HER2 negative advanced or metastatic breast cancer The efficacy of kisquali in combination with aromatase inhibitors in premenopausal or postmenopausal women was confirmed in a clinical trial of monaleesa-7 involving 495 patients All patients in the study received ovarian suppression by goserelin The results showed that patients receiving kisquali plus aromatase inhibitors had a longer median progression free survival (PFS) than those receiving placebo plus aromatase inhibitors (13.8 months vs 27.5 months) The efficacy of kisquali in combination with fluvastatin in the treatment of advanced or metastatic breast cancer was also demonstrated in clinical trial monaleesa-3 involving 726 patients The results showed that the median PFS was longer in patients receiving kisquali plus fluvec group than in patients receiving placebo plus fluvec group (12.8 months vs 20.5 months) "With this approval, we have demonstrated some of the benefits of the new project we are trying to review cancer drugs, which can improve regulatory efficiency and enhance the process of data evaluation This shows that through the method of intelligent policy, we can improve the efficiency and rigor of the process These new programs are designed to reduce administrative issues that may increase review time and cost, including the FDA's staffing burden For example, by analyzing the data earlier before it is formally submitted to FDA, and evaluating the submitted data in a structured template, we can more easily identify the key analysis or information that may delay the review in the application earlier, "FDA director Scott Dr Gottlieb said: "with today's approval, FDA uses these new methods to enable the audit team to start analyzing the data before actually submitting the application and help guide the company's analysis of the top line data to sort out the most relevant information This allowed us to approve the treatment less than a month after it was submitted on June 28, a few months ahead of the target date " (