Tag: biomarkers

Clinical Research: Phase 1 - Phase 4

Getting It Right from the Start: 10 Essentials for Designing Biomarker-Guided Oncology Clinical Studies

In the field of oncology, as we got better at understanding the changes that occur in cancers at a molecular level, we have seen a shift towards precision medicine. Biomarkers are central to this change and can be leveraged to provide important insights at every stage of drug development. By advancing our understanding of cancer...

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...

Clinical Research: Phase 1 - Phase 4

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...

Clinical Research: Phase 1 - Phase 4

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...

Clinical Research: Phase 1 - Phase 4

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...

Clinical Research: Phase 1 - Phase 4

Adaptive Design Methods Offer Rapid, Seamless Transition Between Study Phases in Rare Cancer Trials

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy. Adaptive...

Consulting

Biomarker Trends: Advancing the Body’s Ability to Fight Cancer

As researchers seek to harness the human immune system to fight cancer, they’re looking at several emerging opportunities to expand use of biomarkers. Among them: Human leukocyte antigen typing. Microbiome analysis for determining risk of inflammatory complications with immune therapeutics. Tumor mutation burden, measured via whole genome sequencing, whole exome sequencing, or comprehensive gene panel...

Clinical Research: Phase 1 - Phase 4

4 Phases of Biomarker Method Validation in Rare Disease Research

Rare diseases present some unique obstacles for researchers that significantly contribute to the high cost of drug development. Fortunately, innovations in biomarker use have the potential to significantly cut back on these costs while improving data quality. However, before biomarkers can be used in this research, their associated collection, measurement, and evaluation methods must be validated. The key phases of biomarker...

Clinical Research: Phase 1 - Phase 4

Fit-For-Purpose: A Strategic Approach to Biomarker Method Validation for Rare Disease

Ensuring the integrity of your surrogate endpoints Rare disease studies present unique obstacles to drug developers, including small patient populations, slow disease progression, lack of prior clinical research, and uncertainty around validating biomarker measurements intended for use as surrogate endpoints. Our white paper describes a fit-for-purpose approach to validating bioanalytical methods in orphan drug trials....

Clinical Research: Phase 1 - Phase 4

Biomarker Method Validation in Rare Disease Drug Development

While drug discovery has seen major advances over the last few years — especially in the domain of rare disease research — the number of new drug approvals has not kept pace with increasing development costs. However, the innovative use of biomarkers has the potential to change that trend. Biomarkers in Rare Disease Research Biomarkers are objectively measurable characteristics...