• Document: ORPHAN DRUG. ORPHAN DRUG & RARE DISEASE DEVELOPMENT: Understanding the U.S. and European Regulatory Landscape & RARE DISEASE DEVELOPMENT
  • Size: 410.87 KB
  • Uploaded: 2019-03-24 09:43:29
  • Status: Successfully converted


Some snippets from your converted document:

White Paper presented by Premier Research ORPHAN DRUG & RARE DISEASE DEVELOPMENT ORPHAN DRUG & RARE DISEASE DEVELOPMENT: Understanding the U.S. and European Regulatory Landscape Abstract Today, more than ever, researchers are focused on providing care for the approximately 7,000 rare diseases that may have been overlooked in the past due to the challenges of conducting clinical trials in small populations and limited commercialization potential. The orphan drug arena focuses on identifying and studying potential treatments to serve the significant unmet needs of patients living with rare diseases, and the current landscape offers a variety of incentives, government support and regulatory agency assistance to encourage orphan drug development. In this white paper, we will discuss key strategic considerations – for both the U.S. and the EU – that may be employed to accelerate your orphan drug and rare disease development program. We will focus on the regulatory aspects of bringing therapies for rare diseases to market, with specific emphasis on the similarities and differences between the regulatory pathways in the U.S. and Europe. White Paper presented by Premier Research US Orphan Drug and Rare Disease Landscape In the U.S., a rare disease is defined a condition that affects fewer than 200,000 people and an orphan drug is either:1 – A drug or biological product used for the prevention, diagnosis or treatment of a rare disease in the U.S., or – A drug or biological product that will not be profitable within 7 years following approval by the FDA ORPHAN DRUG & RARE DISEASE DEVELOPMENT Since 2003, there has been a dramatic increase in the number of orphan drug designations, particularly in the U.S., as sponsors better Understanding the U.S. and European understand the incentives associated with orphan drug development and the strategies that can be leveraged to streamline development Regulatory Landscape efforts. There has also been more financial commitment and a deliberate effort by both established firms and up-and-coming companies to invest more time and resources to developing more compounds and products in the orphan drug arena. Figure 1. Trends in Orphan Drug Designation2 USA, EU & Japan Designation per Year (1983-2013) 200 USA Designations per Yr EU Designations per Yr 150 Japan Designations per Yr Designations Per Year 100 Strong increase from 2003 50 0 1983 1984 1985 1986 1987 1988 1989 1990 1991 1992

Recently converted files (publicly available):