The number of orphan drugs, i.e. drugs for rare diseases, is vastly growing. These drugs are often associated with high drug prices. Cost-effectiveness studies can be used to examine the ratio of incremental cost and effects of a new intervention. Tim Kanters and colleagues recently published a cost-effectiveness study of enzyme replacement therapy for adult patients with Pompe disease, an orphan disease, in Orphanet Journal of Rare Diseases.
Monthly Archives: December 2017
Here Dr Lana Garmire describes her new tool to analyze scRNA-seq data, published in Genome Medicine.
The ISRCTN registry has continued to change and evolve since its launch in the early 2000s and now has over 16,300 records. ISRCTN keeps adapting to evolving guidelines and expectations in order to support transparency in clinical research, reduce selective results reporting and ensure an unbiased evidence base. Find out how with our ISRCTN registry quiz!