Clinical Research: Phase 1 - Phase 4

Operationalizing Biomarker-Guided Oncology Trials: Planning for Success

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...

Leveraging Technology and Expert Clinician Review to Enhance Risk Detection in Neuroscience Studies

Across all indications, the primary source of trial failure has been—and remains—an inability to demonstrate efficacy.1 An analysis of 640 phase 3 trials with novel therapeutics demonstrated that 54% failed in clinical development. Among those that failed, more than half—57%—failed due to inadequate efficacy.2 In neuroscience indications, an estimated 85% of late phase studies fail,...

Regulatory Considerations for Radiotherapeutics and Radiodiagnostics

Scientific innovation and advances in ligand targeting have spurred the development of many different types of radiopharmaceuticals (RPs) – radioisotopes bound to biological molecules to target specific organs, tissues, or cells within the body — with a wide range of clinical uses. As those uses have proliferated, and as research has continued to yield novel...

What Patient Population is Participating in Your Early-Phase Gene Therapy Trials? These Factors Could Influence Your Strategy

Today there are over 10,000 rare diseases affecting more than 30 million people in the U.S.1 Many of these disorders are life-threatening, and more than 90% do not have a U.S. FDA-approved treatment2. Novel gene therapies offer hope for improving the health of patients afflicted with these conditions. Securing clearance for these treatments is...

The Multi-Faceted World of Clinical Trial Matching in Precision Oncology Studies

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,...

Where Biomarkers and Gene Therapy Trials Intersect: Benefits Abound

We’re currently experiencing breakthrough times for clinical research in gene therapies. These complex treatments offer much-needed hope to more than 30 million U.S. patients affected by over 10,000 rare diseases1, many of which are life-threatening and the vast majority of which have no available treatment. Gene therapy products must meet the same key regulatory...

A Different Pace: Meeting the Demands of Liquid Biopsy Development for Early Cancer Detection

Development of liquid biopsies for early cancer detection requires careful planning. Understanding the regulatory environment and the challenges of conducting the studies needed for approval is integral to success. Here, we review the regulatory pathways for in vitro diagnostics (IVDs) in both the US and the EU and offer strategies for designing and operationalizing large-scale...

It’s Better for Everyone: The Promise of Liquid Biopsy as a Cancer Screening Tool

Cancer is the leading cause of death worldwide, accounting for nearly one in every six deaths. According to a recent paper, an estimated 15% of cancer-related deaths could be avoided by early disease detection.1 However, there are currently very few population-based screening programs and, even where a screening test does exist, compliance is relatively low....

Checklist: Developing Digital Medicines and Digital Therapeutics: Five Steps for Success

Digital medicines and digital therapeutics (DTx) are changing the future of healthcare by empowering patients to participate in—and influence—their treatment, health, and well-being. These technologies can support various stages of the healthcare journey, addressing unmet needs and bridging gaps in the market for traditional medicine. This convergence of software and healthcare creates opportunities, but the...

Food for Thought: The Power of Decentralized Clinical Trials in Accelerating Infant Formula Studies

Infant formula is an essential food product that serves as the sole source of nutrition for many babies in North America and Europe during their first year of life, supporting health, growth, and development.1,2 More than a year since the shutdown of the largest infant formula plant in the US in February 2022, the country...