Mr. James E. Valentine

Hyman, Phelps & McNamara PC, Washington DC, USA

James Valentine assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine also works with clients on clinical trials operations and compliance matters.

Before joining the firm in 2014, Mr. Valentine worked in FDA’s Office of Health and Constituent Affairs (previously Office of Special Health Issues) where he facilitated patient input in benefit-risk decision-making and served as a liaison to stakeholders on a wide range of regulatory policy issues. Mr. Valentine administered the FDA Patient Representative Program, facilitated stakeholder consultations during the reauthorization of PDUFA and MDUFA, helped launch the Patient-Focused Drug Development program, and developed the FDA Patient Network.

Mr. Valentine also worked at the Center for Drug Evaluation and Research’s (CDER) Office of Regulatory Policy where he coordinated the implementation of the medical gases certification scheme that was established in FDASIA and handled a variety of postmarket safety issues including REMS and safety labeling changes.


DAY 3: September 13, 2019 | Keynote Speaker | 10:45 AM - 11:15 AM

The Foundation for Rare Disease Drug Development in the Era of Systems Medicine

James E. Valentine, JD, MHS, Hyman, Phelps & McNamara PC, Washington DC, USA

In 2018, for the first time, the U.S. Food and Drug Administration approved more novel orphan drugs than it did drugs for more prevalent conditions. This uptick in approvals of products for rare diseases is based on a 35-year foundation of incentives and experience in navigating the unique challenges that are common to rare diseases. However, with 95% of all rare diseases without an FDA-approved drug, there remains a great need to leverage new technologies and methods to improve that statistic. With 80% of rare diseases being of genetic origin, systems medicine may provide the tools to bridge this gap. This presentation will provide a foundation for understanding the environment for rare disease drug development, including providing an overview of recent trends, updates in the field of rare diseases, and providing insights into orphan drugs from various stakeholders’ perspectives.