Both commercial trials and academically sponsored studies share questions around trial design, recruitment targets, mitigating dropout and, ultimately, challenges of regulatory approval if the bar for efficacy and safety are met. However, trial design and delivery in the rare disease arena bring ...
Rare disease clinical trials pose unique and complex challenges. Small and geographically dispersed locations of treatment centers, disease...
Clinical research for rare disease: Opportunities, challenges, and solutions Over 7000 rare diseases, each RC Griggs,M Batshaw,M Dunkle,... - 《Molecular Genetics & Metabolism》 被引量: 335发表: 2009年 Clinical trials and rare diseases: A way out of a conundrum. Argues that randomized ...
In conducting trials with pediatric patients in this space, Albireo faces four challenges common to developers of pediatric rare disease drugs. The first is the lack of a natural history of the disease. The second is determining the study design and appropriate endpoints for the trial. The next ...
The Challenges of Rare Disease Clinical Trials The first challenge of rare disease trials is the most obvious: it’s hard to find patients. Around3.5%-6% of peoplehave a disease classified as “rare.” An even smaller percentage will have the specific disease that a clinical trial is attempti...
Medpace understands the unique challenges rare disease clinical trials face because we have conducted global trials covering Phase I-IV across a broad range of rare disease indications. Explore a sampling of our experience in these therapeutic areas: ...
in ultra rare disease from 2018-2023 Access the latest in human science and data science to accelerate results Real World Evidence & Registries:Leverage real world evidence to support natural history studies and registries, addressing challenges of low patient numbers and difficult endpoints. Feeder re...
The Challenges and Opportunities of Rare Disease Drug DevelopmentDOWNLOAD THE FREE WHITE PAPER New therapeutics development in rare diseases presents both opportunities and complexities. Because of the small patient pool available in these indications, there are challenges in designing and conducting ...
Given rare disease populations are not only small, but also globally dispersed, decentralized clinical trials (DCTs) can encourage participation and increase retention of patients as data collection is achieved at least partially remotely or with fewer site visits. In addition, DCT methodologies can in...
Natalie Douglas looks at the ongoing needs and challenges for rare disease patients and their care moving forward