Virtual: World Orphan Drug Congress USA 2020

ソートリーダーシップ（Thought Leadership）

Key Role for Patient Advocacy Groups in Rare Disease Drug Development: Making Better Use of This Valuable Resource

Monday, August 24 | 8:50 - 9:10 午前

Ray Huml, Vice President of Medical and Scientific Strategy and Head of Syneos Health’s Rare Disease Consortium

Our Experts

Raymond A. Huml, MS, DVM, RAC

Vice President of Medical and Scientific Strategy and Head of Syneos Health’s Rare Disease Consortium

Dr. Huml has over 30 years of experience in the clinical and biopharmaceutical industries and over 20 years in the Contract Research Organization (CRO) industry.  He served as Head of a Global Biosimilars Center of Excellence and as Head of Global Due Diligence where he identified risks associated with global, product-based investments (including rare disease investments), which resulted in almost $3.0b in capital committed to partnerships of all sizes.  Dr. Huml has authored or co-authored over 70 articles, 11 book chapters and 3 books on topics such as due diligence, competitive intelligence, biosimilars and muscular dystrophy.  In 2003, he was awarded North Carolina Veterinary Medical Association's Young Veterinarian of the Year Award.  In 2004, he was awarded Regulatory Affairs Professionals Society’s New Professional Award.  He has also received numerous CRO awards including the Distinguished Performance Award, Clinical Development Services President's Award and the Chairman's Award.   Dr. Huml is a Returned Peace Corps Volunteer having served in Ghana, West Africa and currently sits on North Carolina State University’s Board of Visitors.  He holds an MS in Biology from East Stroudsburg University and his DVM from North Carolina State University College of Veterinary Medicine and has earned the US Regulatory Affairs Certification (RAC).

Cinzia Dorigo, PharmD,

Vice President, Clinical Development, Rare Disease

Dr. Dorigo has over 20 years of experience in the clinical research industry, most of them dedicated to rare disease clinical development. Since 2015, Cinzia has set-up our Syneos Health Rare Disease Consortium dedicated to develop effective strategies for drug development in rare disease indications and to provide support to all Syneos Health teams working in rare disease clinical development. Cinzia has strong experience in planning, managing and delivery of rare disease studies in different therapeutic indications. Her experience covers ultra-rare and rare indications involving adult, pediatric and pediatric intensive care unit patients. Her educational background includes master neuropharmacology at the University of Milan, Italy and Masters in Executive Management at the University of Chapel Hill in North Carolina. 

Zizi Imatorbhebhe, MS, MBA, PMP

Global Vice President, Rare Diseases

Zizi brings over 30 years global commercial, consulting and clinical development experience in the Biopharmaceutical and CRO industry. She has been instrumental in working with many small to large biopharmaceutical companies to identify Rare Disease Drug Development challenges across therapeutic areas and the asset lifecycle and providing solutions to address them. She has developed and successfully led Rare Disease Centers of Excellence Rare and Global Rare Disease Initiatives.  She is also currently one the leaders in the Syneos Health Rare Disease Consortium. Zizi also provides Executive Oversight and Business Unit Leadership over clinical operations and other teams to ensure that projects meet their defined objectives and clients are delighted. Her educational background includes MS in Biotechnology from University College London, University of London; MBA from Chapman University, California. PMP® certified and lean six sigma trained – all providing a strong commercial and business perspective as well as clinical development expertise. She is passionate about supporting rare disease causes and engaging rare disease patient advocacy groups. 

Maryna Kolochavina, PharmD, PMP

Executive Director of Integrated Lifecycle Management, Rare Diseases

Maryna has over 15 years in a lifecycle management of a number of rare and complex rare and orphan conditions, including pediatric rare and ultra rare diseases. Maryna has strong experience in planning and execution of multiple studies/project phases (pre-clinical, I, IIA/IIB, IIIA/IIIB, IV) and designs, such as disease and product observational registries with prospective and retrospective data collection, pregnancy registries, observational FUP, retro chart review studies; compassionate use programs (including Named Patient Program, Expanded Access Programs, ATU, Special Access, IND); safety surveillance studies (including active and passive surveillance studies, descriptive drug utilization studies, PASS, PAES); non-interventional studies (NIS) with ePRO, HEOR and EPI modules; rare ATMP and stem cells studies, device studies, computer adaptive testing, Omics studies (tissue research). Her educational background includes PharmD in clinical pharmacology from Gdansk Medical Academy and translational work in rare skin diseases. Maryna is certified ACRP, BARQA, ASQ and PMI. She is an active patient advocate for rare and orphan patients in the Patient Voice program at Charité, Berlin, Germany.