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Tuesday, June 28 • 10:30am - 11:30am
#227: Unique Global Regulatory Considerations and Drug Development Incentives in Rare Disease and Orphan Drug Development

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Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-590-L01-P; CME 1.00; IACET 1.00; RN 1.00

Early consideration of the issues unique to rare disease development allows sponsors to proactively and adequately identify/address them during development and to have productive interactions with Health Authorities (HA). Unique program elements will be compared and contrasted, focusing on regulatory aspects of rare/orphan development and early HA consult to create successful programs that address the particular challenges. We will also discuss how to strategically take advantage of the various US and EU incentives such as market exclusivity, tax reduction, and expedited review process, as well as grants to facilitate the development of orphan drugs, to enhance orphan drug development.

Learning Objectives

Discuss the regulatory requirements unique to the global development of rare disease and orphan drugs, including the regulatory implications; Explain the regulatory considerations to create efficient, feasible, and sufficient rare disease and orphan drug programs; Summarize regulations regarding orphan drug designations in the US and EU; Discuss and demonstrate how drug developers can take advantage of the regulators' incentives to optimize their investment in orphan drug development.

Chair

Maureen Smith

Speaker

Maximizing the Advantages of US and EU Orphan Drug Designation Incentives
Irene Pan

Global Regulatory Considerations in Rare Disease and Orphan Drug Development: When Patients Are Waiting and Everyday Counts
Lauren Peterson Tornetta



Chair
avatar for Maureen Smith

Maureen Smith

Patient Advocate / Secretary, Canadian Organization For Rare Disorders (CORD)
Maureen Smith’s M.Ed. (Educational Psychology) interest in patient advocacy stems from a rare disease diagnosis at the age of 8. She is the Secretary of the Canadian Organization for Rare Disorders (CORD), co-chair of the Rare/Orphan Disease program track for the Drug Information Association’s annual meeting (2014-2016), a member of the CIHR-Institute of Genetics’ GELS Priority & Planning Committee and the patient member on Ontario’s... Read More →

Speakers
IP

Irene Pan

Senior Research Scientist, UBC: An Express Scripts Company
Irene Pan, MSc, is a Senior Research Scientist with UBC: An Express Scripts Company. With over 12 years of clinical research experience, Ms. Pan has conducted numerous studies in a wide range of therapeutic indications including ophthalmology, neurology, cardiology, oncology and respiratory medicine. She has hands-on experience in designing and managing clinical trials, as well as extensive experience in non-interventional research, including... Read More →
avatar for Lauren Tornetta

Lauren Tornetta

Director, Global Regulatory Affairs, Pfizer Inc
15 years of experience across public and government (FDA) sectors with an MBA in biotechnology and an MS in biochemistry. Global experience leading reg strategy with drugs (NCE, line extensions, drug: drug codev, biologic products, and combination products [drug: device]), in all dev phases including filings, and multiple Advisory Committee Meetings. Experience in multiple TAs including Neuroscience, Pain, Inflam/Immunology, CV and Renal, GI, and... Read More →


Tuesday June 28, 2016 10:30am - 11:30am
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA