Laboratory developed tests (LDTs) and in vitro diagnostics (IVDs) play a critical role in modern medical care. According to the US Centers for Disease Control and Prevention (CDC), 14 billion laboratory tests are ordered each year and an estimated 70 percent of medical decisions are based on the results of these tests.1 Thus, diagnostic tests—and...
In today’s competitive landscape, device companies are facing increased demand for clinical evidence of product efficacy and safety. In addition to pressure from regulators prior to market approval, device companies must answer to other stakeholders, including: This increased scrutiny has prompted more device companies to perform clinical trials to meet regulatory requirements, differentiate their products,...
On June 26, 2024, the U.S. Food and Drug Administration (FDA) released its much-anticipated draft guidance on clinical trial diversity.1 This 23-page document outlines proposed requirements for racial and ethnic diversity action plans, which will soon become mandatory for studies involving drugs, devices, and other medical products. In this article, we highlight the key provisions...
Cell therapies, despite their transformative potential, face significant barriers that can hinder their development and commercialization. Safety concerns, manufacturing challenges, analytical complexities and scale-up difficulties present substantial risks. Competition from other drug modalities, coupled with the inherent complexity of clinical development, further complicates the pathway to market. Distribution and supply chain challenges add another layer...
Cell therapy development faces significant challenges on the path from concept to clinical application. From navigating the regulatory landscape to ensuring product consistency and standardization, the initial phases of developing cell therapies require careful planning and close collaboration among cross-functional teams. In this blog, we explore the complexities of early cell therapy development, with a...
Dose escalation studies are pivotal in the early phases of clinical drug development for evaluating the safety profile of a new therapeutic agent and identifying a recommended dose for further investigation. These studies lay the groundwork for understanding the pharmacokinetic and pharmacodynamic properties of novel therapies and, if designed and executed thoughtfully, can move seamlessly...
Adaptive design is a progressive approach that utilizes different statistical modeling to allow for modifications to enrollment in a clinical trial after study initiation without undermining data integrity and validity. This methodology is particularly beneficial and increasingly common in phase 1 clinical trials, which focus primarily on safety, tolerability, pharmacokinetics, and pharmacodynamics. The essence of...
Since the approval of ipilimumab in 2011 for metastatic melanoma, immune checkpoint inhibitors (ICI) have shifted the treatment paradigm in oncology. Now increasingly indicated as first-line treatment of certain metastatic cancers, ICIs can trigger deep, durable responses and offer significant survival benefit. However, the majority of patients do not respond and many epeirence immune-related adverse...
Medical device development must address some unique challenges that have grown over the last decade due to increasing medical device complexity, a more complex regulatory framework catching up with the industry, and an established and variable standard clinical practice to compete with: These factors have a major impact on the medical device development cycle, which...
Decentralized clinical trials (DCTs) have emerged as a promising solution to address the unique challenges of pediatric rare disease research. In this blog, we discuss the intricate landscape of conducting clinical research in pediatric rare diseases, identify the unique challenges, discuss the potential of decentralized trial strategies, and underscore relevant risks and considerations. Navigating the...