Despite advances in technology and our understanding of the genetic and molecular underpinnings of cancer, making a meaningful impact on the survival and quality of life of patients with cancer remains a significant challenge. In fact, a recent review revealed that, among 59 cancer drugs approved by the U.S. Food and Drug Administration (FDA) based...
With the passage of the Pediatric Research Equity Act (PREA) of 2003, drug manufacturers were required to submit an initial pediatric study plan (iPSP) prior to commencement of Phase 3 studies (or new drug application [NDA]/biologics license application [BLA] submission in the absence of a Phase 3 study) for studies involving a new active ingredient,...
The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a particular childhood cancer. Enacted August 18, 2017, as part of the Food...
Summary The Research to Accelerate Cures and Equity (RACE) for Children Act The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a...
Innovation in oncology drug development is being driven by “precision medicine.” Precision medicine promises a new paradigm in oncology where every patient receives truly personalized treatment. This approach to disease diagnosis, treatment and prevention utilizes a holistic view of the patient—from their genes and their environment to their lifestyle—to make more accurate decisions. Growing at...
Precision oncology promises a new standard of care where therapies are tailored to the molecular profile of a specific tumor. For the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks will need to evolve to the nuances of these novel treatments. In recent years, innovation in oncology drug development...
Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research...
Personalized oncology promises a new model of cancer care where medical decisions are based on a holistic view of the patient, including their genes, environment, and lifestyle, and tailored to the molecular profile of their tumor. To date, great strides toward the paradigm of personalized oncology have been made in the area of cancer immunotherapy,...
Of the 1.7 million new cases of cancer in the U.S. in 2018, 10,590 involved children aged 14 or younger.[1] While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for...
Risk-Based Monitoring (RBM) makes an impact. The US Food and Drug Administration (FDA) requires that clinical trial sponsors “provide oversight to ensure adequate protection of the rights, welfare, and safety of human subjects and the quality of the data submitted to FDA.” [US FDA 2019] This has traditionally been accomplished through onsite monitoring visits and...