Necessity is the Mother of Invention: The Impetus for Observational Research in Orphan Drug Development

Life Sciences, Clinical Trials, Pharmaceutical Regulation, Pharmaceutical, Patient Recruitment & Retention, Drug Discovery & Development,
  • Wednesday, December 07, 2016

The push for innovation in orphan disease research is driven by an accelerated research and development cycle, patient and family concerns for timely product access during the development process as well as following product registration, and the emergence of a regulatory and reimbursement environment facilitating innovation in program design. During this webinar, Worldwide Clinical Trial experts will be discussing these innovation drivers as well as emerging trends in clinical trial methodology for orphan disease drug development and ways to overcome challenges through the use of observational trials by:

  • Providing an impetus for further enhancements in the drug development processes in orphan disease research
  • Describing both benefits and limitations of program innovation from the perspective of patient and family engagement
  • Expanding the portfolio of observational research methods which can complement product registration programs
  • Highlighting techniques that have been considered for data analyses and methods of presentation to assist in informing healthcare decisions


Michael, Murphy, MD, PhD, Chief Medical and Scientific Officer, Worldwide Clinical Trials

Dr. Murphy’s professional career has spanned over 25 years in positions within the Pharmaceutical industry. He is board-certified in psychiatry and has a doctorate in pharmacology, with training at Tulane University, Stanford University and the Mt. Sinai School of Medicine. His contributions as Chief Medical & Scientific Officer at Worldwide encompass all therapeutic areas under Worldwide’s umbrella, and include translational research activities, scientific regulatory support, and protocol development for phases I–IV. He has been a consultant for the Duke Clinical Research Institute and is Founder and Research & Development Editor for American Health and Drug Benefits™. As a lecturer within the Center for Experimental Pharmacology and Therapeutics, Harvard-MIT Division of Health Sciences and Technology (HST) for 17 years, Dr. Murphy contributed to the curriculum for a two-year Clinical Investigator Training Program.

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Josie, Measures, Vice President Biostatistical Operations, Worldwide Clinical Trials

Josie has over 25 years’ experience as a medical statistician. She started her career analyzing nutritional surveys at the Ministry of Agriculture, Fisheries and Food. She them moved to the Medical Research Council providing expert statistical support to clinical researchers. Josie has spent the majority of her career in the Pharmaceutical industry at Pfizer and Quintiles before joining Worldwide Clinical Trials in 2014. Josie specializes in developing high performing Biostatistical Operations and enjoys working closely with teams to develop efficient and effective clinical trials. Josie has an MSc in Biometry and an MBA (with distinction).

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Lorna, Graham, BSc, MSc, Associate Director, Project Management, Evidence, Worldwide Clinical Trials

Lorna Graham has 17 years clinical research experience. Lorna has worked in both the Pharmaceutical and CRO industry in various roles including data management, site management and worked across therapeutic areas including cardiovascular, CNS, oncology, tropical disease and women’s health. Lorna has over 10 years’ experience in project management, which includes study and Late Phase senior project management. Lorna’s Late phase experience includes; Effectiveness evaluation studies, risk management plans, risk evaluations and mitigation strategies, disease registries, observational non interventional studies, healthcare provider surveys and contributions to a global economic dossier for a HIV compound.

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Barbara, Zupancic, Director, Global Patient Recruitment and Retention, Worldwide Clinical Trials

Barbara Zupancic is currently the Director, Patient Recruitment and Retention at Worldwide Clinical Trials. Barbara comes to Worldwide with over 10 year experience in clinical research, specifically focused on patient recruitment and feasibility. Prior to Worldwide, she was the Director of Operations at a large CRO and also a Director of site management at a patient recruitment vendor providing services for the industry. She also has a feasibility and site relations background, so is very cognizant of the needs of all departments. Barbara also spent time in Europe covering feasibility and patient recruitment efforts in UK, Germany, Italy, Norway, Belgium, and Greece. She is fluent in Slovenian and able to communicate in German, and has a Bachelor’s of Science in Psychology, Masters of Science in Counseling Psychology & Research, and an MBA in management.

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Who Should Attend?

Senior level professionals from pharmaceutical and biotechnology companies involved in:

  • Clinical Research
  • Clinical Development
  • Outsourcing
  • Program management
  • Project management
  • Patient Recruitment
  • Regulatory
  • Site Management
  • Outcomes
  • HEOR

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Worldwide Clinical Trials

Worldwide Clinical Trials employs more than 1,400 professionals around the world, with offices in North and South America, Eastern and Western Europe, Russia and Asia. One of the world’s leading, full-service contract research organizations (CROs), we partner with sponsors in the Pharmaceutical and biotechnology industries to deliver fully integrated clinical development and bioanalytical services, extending from first-in-human through phase IV studies. Grounded in medicine and science, we help sponsors move from medical discovery into clinical development and commercialization across a range of therapeutic areas, including neuroscience, cardiovascular diseases, immune-mediated inflammatory disorders (IMID), and rare diseases. For more information, visit

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