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Trk 17: Rare-Orphan Diseases [clear filter]
Monday, June 27
 

8:30am

#121: Rare Disease Clinical Trials: Coping with Unique Challenges
Limited Capacity seats available

Component Type: Symposium
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-602-L01-P; CME 1.25; IACET 1.25; RN 1.25

This symposium provides a unique opportunity to hear three interconnected but distinct perspectives on rare disease clinical trials. We will have an overview of the rare disease clinical environment in terms of therapy focus, geography and volume along with a compare and contrast of operational metrics with respect to start up and recruitment to set the stage. Next, the challenges related to ensuring robust safety and efficacy outcomes from a statistical perspective in an environment where limited patient sample sizes is a reality will be explored along with creative methods for overcoming these challenges. Finally, a rare disease in-depth case study involving Latin America and pediatric patients will showcase best practices on how to harness the unique cultural and environmental aspects of this region to meet enrollment targets.

Learning Objectives

Recall the most prolific areas in rare disease work and industry trends; Use metrics to validate start-up and enrollment challenges; Identify the operational challenges of rare disease trials in terms of recruitment as well as in conventional statistical analysis approaches; Discuss the unique cultural and environmental aspects of enrolling pediatric patients in a rare disease pediatric trial in Latin America; Share best practices on how enrollment can be met.

Chair

Maureen Smith

Speaker

Rare Disease: Understanding Operational Challenges
Linda Martin, MBA

Orphan Indications and Clinical Trials: Coping with Unique Challenges and Why Rare Diseases Warrant Special Treatment
Stephan de la Motte, DrMed, MD

Solving Enrollment Challenges for Rare Disease Global Clinical Trials in Latin America
Sara G Tylosky, MBA



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... Read More →

Speakers
LM

Linda Martin

President and Founder, KMR Group Inc.
Linda Martin is a founder and President of KMR Group, a firm specializing in biopharmaceutical R&D performance, data and analytics. Her areas of expertise include the measurement and evaluation of R&D productivity and clinical development, including subspecialties of enrollment and... Read More →
avatar for Stephan de la Motte

Stephan de la Motte

Chief Medical Advisor, Synteract HCR
Dr. Stephan de la Motte serves as Chief Medical Advisor at SynteractHCR. With nearly 30 years of experience, he is renowned for his scientific and medical expertise and clinical trial knowledge of Phase I, II and III studies. Over the course of his career, he has served as coordinating... Read More →
avatar for Stephan de la Motte

Stephan de la Motte

Chief Medical Advisor, Synteracthcr
Dr. Stephan de la Motte serves as Chief Medical Advisor at SynteractHCR. With nearly 30 years of experience, he is renowned for his scientific and medical expertise and clinical trial knowledge of Phase I, II and III studies. Over the course of his career, he has served as coordinating... Read More →
avatar for Sara Tylosky

Sara Tylosky

CEO, Farmacon
Sara Tylosky, President & CEO at Farmacon has > 20 years of experience in strategic management & global marketing in the pharmaceutical & biotech industries. Farmacon works with sponsors, from large pharmas to start-up biotechs, from market intelligence to project manage enrollment... Read More →


Monday June 27, 2016 8:30am - 9:45am
103A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

10:45am

#143: Priority Review Vouchers: Past Experiences, Legislative Reform, and Tips for Sponsors
Limited Capacity seats available

Component Type: Symposium
Level: Intermediate
CE: CME 1.25; IACET 1.25; RN 1.25

Over the past decade, several policy strategies have emerged that promise to promote drug development in areas of unmet need, such as exclusivity previsions and advanced market commitments. Recently, one of these policy strategies, the priority review voucher (PRV), has garnered new attention and has re-emerged as a possible driver in R&D for rare pediatric diseases, neglected tropical diseases, and public health emergencies. The PRV program is a regulatory incentive administered by the FDA to promote drug development for certain underserved patient populations. Under the original PRV program, signed into law in 2007, FDA awards a voucher to sponsors for the regulatory approval of a novel drug targeting a particular neglected tropical disease; sponsors that redeem a PRV are subsequently allowed to designate any one NDA/BLA, regardless of indication, as priority review, reducing the target regulatory review period by four months. Since 2007, nine vouchers have been issued by the FDA, and reported sales of the voucher have continued to increase to as much as $350 million. This panel takes a closer look at the value of the PRV as an incentive, focusing on past experiences related to the PRV program, current use, legislative trends, potential value, and its role in encouraging much needed R&D for rare pediatric and neglected tropical diseases.

Learning Objectives

Explain the history and mechanics of the FDA priority review voucher (PRV) program; Describe current policy initiatives regarding the PRV program and industry response to the PRV incentive; Assess the value of the PRV as an incentive for rare and neglected diseases.

Chair

Andrew S. Robertson, JD, PhD

Speaker

Priority Review Vouchers: A New Paradigm for Funding Research?
Patricia R Anderson, RAC

The Priority Review Voucher: The Value, the Pipeline, and the Opportunities for R&D
Andrew S. Robertson, JD, PhD

Priority Review Vouchers: Legislation and History - What You Need to Know
Alexander Varond, JD



Chair
AR

Andrew Robertson

Director, Global Regulatory Policy, Merck & Co., Inc.
Andrew S. Robertson works in Global Regulatory Affairs at Merck & Co., focusing on regulatory policies concerning vaccines, biologics, biosimilars, in vitro diagnostics, mobile medical apps and software medical devices. He has over 13 years of experience spanning academia, industry... Read More →

Speakers
avatar for Patricia Anderson

Patricia Anderson

Vice President, Regulatory Services, Mapi
Ms. Anderson has covered many aspects of drug development during over 30 years in the pharmaceutical and biotechnology industry. Ms. Anderson has held several positions which have included the areas of regulatory affairs, drug development, and due diligence for licensing. She has... Read More →
avatar for Alexander Varond

Alexander Varond

Associate, Goodwin Procter LLP
Alexander J. Varond works on drug development and medical devices, advertising and promotion, and enforcement issues. He counsels clients on drug development matters such as clinical trial design, REMS, orphan drug designation, formal dispute resolution, and patent and exclusivity... Read More →


Monday June 27, 2016 10:45am - 12:00pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA