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., M.P.H.
Senior Vice President, 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., M.Sc.
Vice President, Technical
Sinan Sarac, M.D., Ph.D., M.Sc.
Senior Vice President, Head, Regulatory Strategy Europe
Steve Winitsky, M.D.
Vice President, Technical - Regulatory Strategy
Jamie Pierson
Senior Project Leader, Parexel International
Kim MacDonnell
AD, Rare Disease Center of Excellence
Mwango Kashoki, M.D., M.P.H.
Senior Vice President, 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., M.Sc.
Vice President, Technical
Stay current with novel thinking and actionable insights from our experts.
At Parexel, rare disease clinical research is an integral part of what we do, and we take it personally. Our Center of Excellence for Rare Disease helps sponsors maximize success in rare disease drug development. The way we integrate clinical and regulatory consulting expertise gives customers the most efficient path to designing, planning, and executing even the most complex trials. Multidisciplinary clinical and commercial teams can help you navigate adaptive study design by guiding you toward optimal endpoints for rare disease trials. Accelerate your drug development and maximize the chances of market access success with Parexel.