Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape
The orphan drug industry is gaining importance as a significant and rewarding market in the pharmaceutical and biotech sectors. Technological and scientific innovations in specific drug discovery pathways as well as major developments in genetics are cultivating growth in this industry. BCC Research’s objective in conducting this report is to provide detailed information on drug development in rare diseases. This report provides a comprehensive analysis and examines the future direction of these drugs as an important means for the treatment of orphan and rare diseases.
- An overview of the drug development in rare diseases with special focus on clinical trial and regulatory landscape
- Detailed description of different kinds of orphan drugs and a comparative assessment on biological and chemical or non-biological orphan drugs
- Discussion on Orphan Drug Act; insights into general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs
- Highlights of technological advances and innovations in genetic manufacturing technologies and information on terms such as generic competition, patent expirations, premium pricing and information on orphan drug designation applications
This report analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report also analyzes the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. This report covers an overview of the global orphan drugs market and its competitive landscape.
BCC Publishing Staff comprises expert analysts who are skilled in conducting primary research, secondary research and data analysis and have decades of combined experience covering a wide range of industries, including healthcare, advanced materials and emerging technologies. Collectively, the team represents a diverse set of educational achievements with individual graduate work completed in fields such as microbiology, electrical engineering, business administration and surgery, among others.