Orphan drugs are pharmaceutical products that treat rare diseases or conditions. Assignment of orphan status to a disease and any drugs developed to treat it has been a matter of public policy and has led to the development of drug products that otherwise may not exist. Now there is renewed interest in developing more high-value treatments for rare diseases with unmet medical needs.
In this webcast, you will learn the history of orphan drugs and relevant rules and regulations. You will gain an understanding of the requirements associated with orphan drug development, including challenges to and benefits of their approval. You will also learn how to write an orphan drug application to request orphan drug status.
Learning Level(s): Basic and Intermediate
Upon the webcast's conclusion, you should:
- Understand orphan drug rules and regulations in US and EU
- Be able to develop strategy for orphan drug development
- Have the ability to write an application requesting orphan drug designation
- Understand unique challenges and benefits associated with orphan drug development
Who Should Attend:
- Regulatory professionals
- Clinical professionals
Marketing departments of companies interested in developing orphan drugs
- Business development professionals working in the orphan drug area
Priya Jambhekar, president, PBS Regulatory Consulting Group
Priya Jambhekar is the president of PBS Regulatory Consulting Group. Previously, she was a global VP of regulatory and auality at Paramount BioSciences, Ethicon, a J&J company and Alkermes. Jambhekar has more than 20 years of experience in the areas of product development, product safety, quality assurance, quality compliance and US and international regulatory affairs. She has extensive experience in establishing regulatory strategy and operational excellence for optimal success during all aspects of pre- and postapproval phases of drugs, devices and biologics, and product development and marketing covering various therapeutic areas, including orphan drugs.