No Opportunity Unturned: Optimizing Orphan Drug Development

Regulations supporting orphan drug research have been evolving for more than 20 years, providing incentives and guidance for development of potentially promising drugs to treat rare indications. More recently, with global attention and advocacy focused on improving treatments in these areas of unmet need, regulators and legislators have sought meaningful changes in the design and execution of rare disease drug development programs.

Understanding existing resources, financial aid, and incentives — and how to access them — is important for any orphan product developer. This webinar will describe support offerings available through global regulatory agencies and examine pending initiatives affecting orphan drug clinical development. These include:

• Relevant guidance documents such as natural history studies and biomarkers.
• Expedited review pathways such as FDA breakthrough therapy and EMA accelerated assessment designations.
• Engaging regulators to advance development of priority medicines.
• What lawmakers are discussing

Speakers

Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research

Angi Robinson has been studying rare diseases at Premier Research for 14 years. She has provided oversight and management support for more than 30 rare disease studies in the U.S. and globally and has supported FDA pre-IND meetings, IND submissions, and NDA/BLA project directorship. Angi has worked on industry sponsored studies to support the approvals of four drugs with orphan drug designations.

Ms. Robinson helped launch the first Best Pharmaceuticals for Children Act coordinating center, collaborating with leaders at the National Institute of Child Health and Human Development. She served as Project Manager for highly complex trials, including sedation in pediatric intensive care units and pediatric bipolar disorder. Her experience includes multiple study designs including PK/PD, adaptive design, and FDA fast track and breakthrough therapy designations.

Ms. Robinson began her career in laboratory quality control and has worked in clinical trial management as a Project Director/Manager, Clinical Lead, and Clinical Monitor. She holds a bachelor’s degree in cell and molecular biology from Tulane University.

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Lisa Pitt, Vice President, Global Regulatory Affairs, Premier Research

As Premier Research’s lead regulatory official, Lisa Pitt provides strategic guidance and oversees the company’s regulatory affairs service offerings. Dr. Pitt has more than two decades of experience in the Pharmaceutical and contract research industries and extensive expertise in small-molecule and biologic Pharmaceutical product development, from early phase through registration and post-marketing.

She oversaw regulatory activity for global product development teams at MedImmune as Director of Regulatory Affairs and served as Director and Principal Regulatory Affairs Consultant at PAREXEL. She also held regulatory positions at Novartis Pharmaceuticals, and immediately prior to joining Premier Research, was a senior regulatory project manager at the U.S. Food and Drug Administration, responsible for drugs and biologic products intended for the treatment of inherited metabolism disorders.

Dr. Pitt holds a doctorate in pharmacy from the University of Maryland School of Pharmacy, a Master of Science and Jurisprudence from Seton Hall Law School, and a bachelor’s degree in biological sciences from the University of Delaware.

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Juliet M. Moritz, Executive Director, Strategic Development, Rare Diseases, Premier Research

Juliet Moritz has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare diseases, supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions.

Prior to joining Premier Research, Ms. Moritz was Associate Director of Global Project Management at PPD Inc., overseeing infectious and respiratory disease research, and prior to that was Associate Director of Clinical Research at Knopp Biosciences. She held senior positions at Wyeth, Theravance, and PPD and began her career as a clinical research associate.

Ms. Moritz holds a Master of Public Health degree from the Drexel University School of Public Health and a bachelor’s degree in biology from the University of Pennsylvania. She is a member of the National Organization for Rare Disorders and the Drug Information Association.

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