Rare Disease

Clinical Research: Phase 1 - Phase 4

Pharmaceutical Outsourcing | Registries and Natural History Studies in Rare Disease Drug Development

Increasing emphasis on rare diseases has led to a greater need to accurately define the profile, characteristics, and outcomes of patients living with those conditions. The development of safe and effective treatments of rare diseases requires a deep understanding of the disease of interest, including its frequency, clinical features, risk factors, outcomes, burden, and evolution....

Clinical Trials in Rare Diseases

December 7, 2022 9:30 a.m. GMT Virtual Join Premier Research for Clinical Trials in Rare Diseases. This online event will explore the challenges when working with Orphan Drugs and Rare Diseases, and the benefits from a development viewpoint. It will explore the benefits of early planning and engagement with regulatory authorities, as well as looking...

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

International Clinical Trials: How Contingency Plans Became the New Normal

Over the last decade, there has been increasing interest in mobile health (mHealth) and wearables. The general population has been tracking their steps, checking their heart rates, and monitoring their sleep patterns. Meanwhile, the clinical trial industry has been exploring decentralized trials and remote monitoring options as a way of alleviating the travel burden that...

Medical and Regulatory Affairs

Clinical Data Trends: Transforming Data Access/Analysis

The clinical trial industry has been steadily undergoing a data evolution. Data from remote capture devices, such as wearables and other novel sources (e.g., increasingly large health data repositories and electronic healthcare data), have gained importance. Proper management and utilization of such data allows sponsors to untether from a set roster of trial sites, focus...

Medical and Regulatory Affairs

Long-Term Gene Therapy Follow-Up: Patient Considerations

When developing a long-term follow-up strategy for gene therapy trials, safety is the main concern, closely followed by the patient perspective. Digital technologies and monitoring have proven essential to this, easing the burden on the patient, and providing accurate, time-saving methods of data management. Momentum is increasing in the field of gene therapy, with over...

Patient and Stakeholder Engagement

“It Motivates Me Every Day”: Our Premier Research Team on Why Rare Disease Research Matters

There are as many as 7,000 distinct types of rare and genetic diseases, and an estimated 400 million people suffer from a rare disease globally. In addition, three of 10 children with a rare disease won’t live to see their fifth birthday. Rare Disease Day, celebrated around the world on the last day of February,...