It is an unfortunate reality that many diseases and conditions affect such small numbers of patients that, when a sponsor develops a drug or biological product to treat them, relatively little return on investment is generated to offset the high development costs. As a result, the pharmaceutical industry has neglected many rare diseases in the past. However,...
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,...
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...
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...
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....
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...
Rare cancers account for 25-30 percent of all new cancer diagnoses and 25 percent of cancer deaths.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 options, researchers, clinicians, and patients may be...
The drug development process is a long journey, beginning with drug discovery, moving through nonclinical and clinical studies, and ultimately culminating in regulatory approval. With many steps in between, each as important as the next, multiple factors regarding development strategy and approach must be considered at the earliest stages. This blog post offers advice for...
Progress in advanced computational methods using modeling and simulation has been enabled by advancements in computing technologies and the rise of artificial intelligence and machine learning. This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of...
Chimeric antigen receptor (CAR) T-cell therapy is becoming an increasingly important part of the cancer treatment landscape, with a growing number of clinical trials investigating its use across a range of cancer types. To date, the FDA has approved six CAR T-cell therapies, all for hematological malignancies and all primarily used as treatments for patients...