Clinical Research: Phase 1 - Phase 4

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...

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 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...

Charting the Path: How Patient and Caregiver Journeys Guide Rare Disease Study Design

It comes as no surprise that putting patients first is imperative for achieving success in rare disease development programs. However, to what extent can sponsors integrate patient input into their study design while still securing the high-quality data they need? Although a bit of a balancing act, it is possible to design a study that...

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...

Can Your Protocol Flex? The Importance of Adaptive Trial Designs in Precision Oncology Studies

Cancer is one of the most scientifically complex and dynamic diseases. Even with significant advances in our understanding of the genetic and molecular mechanisms that lead to cancer, only 10 percent of approved oncology drugs demonstrate an overall survival benefit.1 In recent years, there has been a shift in how early-phase oncology studies are conducted...

Premier Research and CENTOGENE Launch Strategic Partnership to Accelerate and De-Risk Rare Disease Clinical Development

Leveraging Rare Disease Insights Powered by the CENTOGENE Biodatabank and Centralized Multiomic Laboratories in Clinical Trials MORRISVILLE, N.C., and CAMBRIDGE, Mass., ROSTOCK, Germany, and BERLIN, January 9, 2023 — Premier Research, whose mission is to help the most innovative biotech and medtech companies take their best ideas from concept to commercialization, and Centogene N.V. [Nasdaq:...

The Rise of Radiotherapeutics: Brief History and Best Practices for Clinical Trials

Radiation therapy was first used to treat cancer more than a century ago, and nearly half of all cancer patients still receive it at some point during their treatment.1 Historically, most radiation therapy was given by delivering ionizing beams of radiation from outside the body, but with advances in the field of nuclear medicine, radionuclides...

Premier Perspective on Biomarkers in Oncology

In this era of precision medicine, biomarkers play a critical role in the rational development of novel therapies. With advances in both our understanding of cancer pathophysiology and tumor profiling technology, biomarkers can now be used to provide important insights at every stage of drug discovery and development. In this article, we explore the common...

Adaptive Trial Designs in Early Oncology: Minimizing Risk & Accelerating Timelines

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...