How is the U.S. FDA Preparing for the Era of Personalized Medicine?

Mon, 12/30/2013 - 2:43pm
Andrew S. Wiecek, Editor

In an October 2013 report, the U.S. Food and Drug Administration (FDA) highlighted the regulatory challenges of personalized therapeutics, as well as the agency’s response.1 Overall, the report demonstrated the agency’s commitment to establishing regulatory processes, policies and infrastructure to support the review of drugs that target smaller patient populations. One of the biggest challenges comes from the cornerstone of personalized medicine, which is the codevelopment of a drug and a companion diagnostic test that can identify patients who will benefit from that drug. In response, the FDA has improved its capabilities to evaluate these medical diagnostic products as well as built an infrastructure to increase collaboration between the different centers within the agency that regulate drugs and diagnostic products.

The FDA is working to leverage these new diagnostics to improve the design of clinical trials. Because many cancers are heterogeneous, which has resulted in varying patient responses for some cancer drugs, the agency believes that oncology drug development could stand to benefit significantly from incorporating molecular diagnostics into the process. One example is the ongoing I-SPY 2 trial for women with advanced breast cancer, which is designed to use molecular diagnostics to better predict which enrolled patients will benefit from the addition of investigational drugs to chemotherapy.

Although the increased safety and effectiveness of target therapeutics allows for smaller trial designs, there are some additional challenges, such as missing data, multiple endpoints, patient enrichment and adaptive designs. In response, the agency is developing new clinical trial designs and statistical methodologies that address some of those issues. In addition, rare adverse side effects of these drugs are less likely to be identified in these smaller trials, so the FDA is increasing their focus on postmarket monitoring of these drugs to ensure efficacy and safety.


1. U.S. Food and Drug Administration. Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development. Available at: Published October 29, 2013. Accessed December 9, 2013.


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