SECTION 2
Rare disease drugs utilize multiple regulatory programs, often simultaneously, to expedite clinical development and regulatory review. In this section, Parexel experts and former regulators share insights on winning FDA Breakthrough Therapy designation, common misconceptions about developing orphan cell and gene therapies, and how to conduct an efficient and successful development program for an orphan-designated drug in the EU.
Jamie Pierson
Senior Project Leader, Parexel International
Kim MacDonnell
AD, Rare Disease Center of Excellence
Mwango Kashoki, M.D., MPH
SVP, Global Head of Regulatory Strategy
Peter Kiely, M.D.
Vice President, Technical
Rachel Smith
Executive Director, Rare Disease, Center of Excellence
Simona Stankeviciute, M.D., MSc
VP, Technical
Sinan Sirac, MD, Ph.D., MSc
VP, Technical - Regulatory Strategy
Steve Winitsky
VP, Technical - Regulatory Strategy
Jamie Pierson
Senior Project Leader, Parexel International
Kim MacDonnell
AD, Rare Disease Center of Excellence
Mwango Kashoki, M.D., MPH
SVP, Global Head of Regulatory Strategy
Peter Kiely, M.D.
Vice President, Technical
Rachel Smith
Executive Director, Rare Disease, Center of Excellence
Simona Stankeviciute, M.D., MSc
VP, Technical