This edition of the Drug Discovery TOE depicts trends across orphan drugs in North America. Compared with standard drugs, orphan drugs involve fewer clinical trial issues. Typically, orphan drugs spend less time in regulatory review, comprise smaller phase III trial sizes, and are more likely to be approved by the US Food and Drug Administration (FDA) because they represent significant therapeutic advances for really unsolved problems. This and the higher chance of FDA approval makes many companies to penetrate the market. However, innovation is critical. Strategic partnerships and tactical regulatory planning that allow for optimizing budgets and exploiting research and clinical expertise are crucial for development success. Some companies innovating in this field and the patent scenario are depicted in this TOE.
The Drug Discovery TechVision Opportunity Engine (TOE) reports and analyzes about the latest developments in drug discovery and therapeutics, thereby providing an opportunity to acquire strategic insights into crucial developments in this industry domain. Technologies profiled include those used for biomarker and target discovery such as high throughput screening, signal transduction, micro array, RNAi, metabolomics, toxicogenomics, biosensors and nanotechnology. In addition to these, a fair focus shall be dedicated to the latest trends in therapeutic approaches across a diversity of diseases including infectious, genetic, metabolic, neurological, cardiac, and autoimmune disorders.
The Health and Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.
Keywords: Orphan drugs, rare diseases, genetic diseases, clinical trials, fast-track designation, breakthrough therapy designation, lysosomal storage disorders, post-transcriptional control processes, synthetic retinoid products