Description: The underlying principle of orphan drug development is to encourage the development of drugs for rare diseases. Orphan Drugs is vaccine, medicine, or in-vivo diagnostic agent that is intended to treat, diagnose, and prevent a rare disease (or) rare medical condition, the condition itself being as an orphan disease. Recent changes in the international regulatory landscape have started to provide a favorable environment for the development of orphan drugs. Many countries have implemented a combination of legislation, regulations and policies to promote the development of orphan drugs within the last two decades. The development of orphan drugs has been financially incentivized through US law via the Orphan Drug Act of 1983. Likewise, a number of countries such as Japan, Australia, Europe etc. use orphan drug legislation to encourage product research, development and marketing. Some countries have big patient population but does not have defined legislation in place such as China and Japan. Orphan drug qualification criteria differ among countries and, as these criteria continue to evolve, it will become even more important to verify a given geography’s requirements. It is important to understand orphan drug regulatory and policy initiatives around the globe and their differences to improve research and policy development and to guide regulatory submissions for treating rare diseases.In this presentation we will discuss the following:- Rationale and Considerations associated with rare diseases clinical Trial, Marketing Authorization, etc. Regulatory Filing Strategy based on comparative policies and incentives across regions – North Americas, Europe, EMEA, China etc. Current Trends and Case Studies for Gene Therapy and Oncology drug development
Frame the discussion with an overview of various rationale and considerations associated with rare diseases - Clinical Trial, Marketing Authorization, etc.)
Upon completion audience would be able to understand and develop Regulatory Filing Strategies based on comparative policies and incentives across regions – North Americas, Europe, APAC, EMEA, China etc.
Provide audience with real-world examples and guidelines on current trends illustrated through Case Studies for Gene Therapy and Oncology drug development