In the field of oncology, as we got better at understanding the changes that occur in cancers at a molecular level, we have seen a shift towards precision medicine. Biomarkers are central to this change and can be leveraged to provide important insights at every stage of drug development. By advancing our understanding of cancer...
Advances in genomic and proteomic technologies have led to the identification of numerous biomarkers with potential clinical utility in oncology, including gene mutations, gene amplifications, gene expression signatures, and altered proteins. To date, more than 1,500 potential oncology biomarkers have been identified in the preclinical setting. Among these, approximately half are being studied in active...
The success of clinical trials is contingent upon finding participants who meet certain criteria, ensuring that the resulting study data are meaningful and relevant to the target patient population. Clinical trial matching is the process of identifying and connecting potential participants with studies that align with their specific medical condition and characteristics. In precision oncology,...
Individually, rare diseases by definition have a low prevalence, but collectively, the societal burden and impact of these uncommon conditions is significant. The majority of rare diseases are genetic in origin, and advances in genomic sequencing tools and technologies have driven an increase in the identification of rare disorders. Currently, one out of every 10...
Introduction Over the past decade, significant advances have improved our understanding of the genetic and molecular mechanisms that lead to cancer. And yet, a recent review of the oncology drugs approved by the U.S. Food and Drug Administration (FDA) on the basis of improvement in tumor response rates showed only 10 percent of these therapies...
Skin diseases are the fourth leading cause of health afflictions, affecting nearly 900 million people worldwide.1,2 At the same time, there has been a remarkable increase in dermatology drug development activity in recent years. For conditions such as atopic dermatitis, psoriasis, and melanoma, there are hundreds of ongoing or planned clinical trials.3 In this new...
Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. In our previous blog post, we discussed site selection, manufacturing, long-term...
Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post, we introduce a case study as a framework for exploring critical study design considerations of...
As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. To date, there is no harmonized international standard for regulating gene therapy products; however, the U.S., EU, and Japan have established regulatory frameworks with subtle variations. Understanding how gene...
Rare cancers account for 27 percent of all new cancer diagnoses in the U.S. and 22 percent of all new cancer diagnoses in the EU.1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment...