Tag: blog 125 results

Dermatology

Digital Disruption in Dermatology: The Role of mHealth Technologies in Clinical Care and Clinical Research

In a fast-paced digital world, healthcare has had to adapt to better serve consumers who increasingly prefer the convenience of web- or app-based services to in-person assistance. Mobile health (or mHealth) solutions provide increased access to care, making it easier for patients to receive care and for healthcare professionals to deliver that care. Visual assessments…

Rare Disease

In Silico Modeling Unveils a New Era in Rare Disease Drug Development

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…

Oncology & Hematology

Beyond Boundaries: Revolutionary CAR-T Therapies Pave Way to Conquer Solid Tumors

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…

Rare Disease

To Placebo or Not to Placebo? The Great Debate in Rare Disease Trials

Small patient populations. Geographic dispersion. Competing studies. Sound familiar? If you’re a sponsor of a rare disease study, you know all too well the challenges that come with recruiting patients. These challenges lead to immense pressure to design and implement studies that limit the burden of participation, while keeping patients engaged and generating high-quality data…

Rare Disease

Realizing the Full Value of Patient Centricity in Rare Disease: 5 Ways to Maximize Your Engagement Efforts

As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders, sponsors need to be proactive in demonstrating their commitment to addressing unmet therapeutic needs.    Beyond…

Rare Disease

At the Intersection of Rare Disease and Precision Medicine: A Road to Growth

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…

Rare Disease

Rare Disease Patients as Partners: Value Beyond Enrollment

In 2020, the European Joint Programme for Rare Diseases developed a guide on patient partnerships in rare disease research projects. This guide still rings true today, and we summarize in this blog the key points for sponsors of rare disease studies to consider when engaging patients and their representatives in study planning. If you’re developing…

Oncology & Hematology

9 Tips for Designing and Operationalizing Early-Stage Precision Oncology Studies

Traditionally, early-stage clinical trials focus on toxicity assessment and dose selection. Today, a growing number of Phase 1/2a trials are designed to draw inference about preliminary response rates due, in part, to the use of biomarkers and adaptive design approaches that enhance the early detection of efficacy signals. These approaches may add to study complexity…