As the global CONVID-19 pandemic rages and the death toll mounts, whole classes of patients not directly discussed often face great danger. With 30 million rare disease patients in the U.S. alone, and about 400 million worldwide, great risk has rippled through the...
Orphan Drug
Category
FDA Approves More Drugs & Faster but Are Drugs Better and Patients Safer? A Harvard Study Questions
The Food and Drug Administration’s (FDA) attempts at expediting and streamlining the approval of advanced new medicines is surely appreciated by those in the public that understand the importance of access to such therapies to address serious disease. And in fact,...
Growth in Rare Disease Impacts Drug Development and Strategy
About 33% of all drugs in active R&D pipelines are now included in the rare disease category. This categorization presents scientific and operational challenges to sponsors and clinical trial ecosystem participants, the adoption of new strategies, operating models...
Clinical Research as Care: Cut the Patient off Post Clinical Trial—A Lose-Lose in the End
Deborah Cohen of the BBC reports that patients in the UK who contributed to orphan studies only to be cut from ongoing access once the study is completed. The patient receives care from the clinical trial only to be cut once the trial is over and the drug approved....
Fibrocell Wins $1.4 Million from FDA to Advance FCX-007 In Trials for RDEB
Fibrocell received $1.4 million from the U.S. Food and Drug Administration’s Office of Orphan Products Development (OOPD) to support the company’s ongoing clinical trials testing its FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). In...