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...
Expertise: Rare Disease 82 results
Rare Disease
The Expanding Role of RWE in Rare Studies
Real-world evidence is increasingly important in drug development as the medical and pharma communities recognize the data’s potential for predicting the benefits of treatment interventions.
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...
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...
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...
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...
Rare cancers account for 27% of all new cancer diagnoses in the US and 22% of all new cancer diagnoses in the EU. With the shift toward grouping cancer based on molecular subtypes rather than by location and tissue type,...
Comparing Orphan Drug, Rare Pediatric Disease, and Humanitarian Use Device Designations The US FDA offers sponsors a variety of special designation programs to incentivize them to develop and deliver therapies to treat unmet patient needs, such as Fast Track Designation,...
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...
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...