Tag: Rare Disease

Clinical Research: Phase 1 - Phase 4

7 Tips for Designing and Operationalizing Rare Disease Natural History Studies

Natural history studies play a crucial role in rare disease drug development, providing insights that advance discovery and shape clinical trial design. The benefits of these studies also extend to understanding patient journeys, clarifying the standard of care, identifying disease-specific centers of excellence, and surfacing opportunities to improve patient care. In this blog post, we...

Clinical Research: Phase 1 - Phase 4

How to Use Patient Registries and Natural History Studies in Rare Disease Development

Successful drug development requires a deep understanding of the disease of interest—its etiology, epidemiology, presentation, manifestations, and progression. In rare diseases, however, much of this information may be unknown. Patient populations are small and historical data are collected inconsistently and dispersed across treating physicians practicing in diverse geographies. Patient registries and natural history studies are...

Clinical Research: Phase 1 - Phase 4

Natural History Studies: Understanding and Enhancing Their Value in Rare Diseases

Successful drug development requires a comprehensive understanding of the underlying disease. To design reliable clinical trials with meaningful, measurable outcome measures, sponsors must apply thorough knowledge of disease presentation, manifestations, and progression, which may be challenging in rare diseases where this information is minimal. Thus, observational studies, encompassing both registry and natural history studies, play...

Clinical Research: Phase 1 - Phase 4

Rare Disease Recruitment: 5 Strategies for Differentiating Trials in a Competitive Research Environment

Recruitment for rare disease research has always been difficult due to myriad factors inherent to the low incidence and prevalence of these conditions. In recent years, however, recruiting for rare disease trials has become even more challenging due to increasing competition in the clinical research environment. In addition, as technologies advance and information becomes more...

Clinical Research: Phase 1 - Phase 4

Achieving the Promise of Gene Therapy: New Pathways to Overcome Patient Enrollment and Safety Challenges, Upcoming Webinar

TORONTO (PRWEB) SEPTEMBER 15, 2020 Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. This webinar will examine the evolving...

Clinical Research: Phase 1 - Phase 4

Novel Trial Approaches for Rare Cancer Patients, Upcoming Webinar

TORONTO (PRWEB) AUGUST 4, 2020 Join Rupa Doshi, Ph.D., Executive Director –Program Strategy, Oncology, Premier Research and Sameena Sharif, Ph.D., Senior Vice President, Product Strategy, Premier Research in a live webinar on Monday, August 24, 2020 at 11am EDT (4pm BST/UK). Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no...

Consulting

The State of Gene Therapy in Dermatology

When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success of gene-based therapies relies on three critical elements:1 A well-defined disease geneA therapeutic geneAn efficient...

Clinical Research: Phase 1 - Phase 4

Premier Insight 272: Delivering the Global Expertise Required to Coordinate a Pediatric Gene Therapy Trial

With their potential for long-term or even curative efficacy, gene therapies are of great interest to researchers, clinicians, patients and caregivers alike. But coordinating and conducting a global, multi-center gene therapy trial is a complex, high-risk undertaking. Beyond the usual protocols and procedures required to ensure patient safety and data quality, gene therapy studies must...

Consulting

BIO International: Discover the Latest in Innovative Rare Disease Trial Design

DURHAM, N.C., June 5, 2019 — Premier Research is sponsoring the Orphan & Rare Disease session track at this year’s BIO International Convention, June 3-6 in Philadelphia. Our own Angi Robinson, vice president for clinical development services in rare diseases and pediatrics, will participate in the panel discussion Rare Finds: Innovative Clinical Trial Design for...