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Ultra-Rare Disease and Clinical Research– Different Approaches for Unique Studies
In a previous blog, we discussed ultra-rare diseases and the unique research challenges they pose to the development of new therapies. The nature of rare and ultra-rare disease means that drug development pathways and the types of studies commonly conducted can differ from the pathways and approaches used for more common diseases.
Ultra-Rare Diseases: Decentralized Solutions to Tackle Challenging Clinical Studies
Though there are few individual patients with ultra-rare diseases, the total number of patients affected runs into the millions and that number grows each year as advances in genome sequencing improves the ability to diagnose them. Whatever definition is used, the extreme rarity of such patients presents unique challenges to their diagnosis and treatment paradigms.
How Rare Diseases Were Affected by the COVID Pandemic
The latest issue of Raconteur (published as a supplement in The Times, UK) took a deep look at the rare disease landscape, including how rare diseases were affected by the COVID pandemic, and how patients who are engaged in the drug development process through clinical trials can build connections through digital platforms.
3 Tips for Applying DCT Approaches in Rare Disease Trials
Decentralized clinical trial (DCT) approaches can be extremely useful in helping to facilitate clinical research participation for rare disease patient populations. That said, many sponsors new to conducting studies that include DCT elements may need some help getting started. Following are some tips based on THREAD’s experience helping sponsors to select and execute DCT approaches for their rare disease studies.