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Trk 17: Rare-Orphan Diseases [clear filter]
Sunday, June 26
 

9:00am

#41: A Rare Disease Program Case Study: Strategies for Successful Outcomes
Limited Capacity seats available

Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-16-507-L01-P; CME 6.50; IACET 6.50; RN 6.50

An additional registration fee is required for all preconference tutorials.  Register today for this tutorial.

Working through a multifaceted case study, this one-day, intensive tutorial will address heads-on the unique challenges associated with the development of rare disease therapies. Successful outcomes are a direct result of early planning, design and engagement with patients, patient advocacy groups, treating physicians, specialists, and regulators.

This interactive case study will emphasize how “spending” time in early planning assures appropriate study outcome measures, timely enrollment of clinical trials, and successful regulatory interactions which affect the bottom line and improve the potential for developing therapies for rare disease patients faster.

Who should attend?

This tutorial is designed for professionals working with rare disease therapies including by not exclusive to researchers from academia and industry, senior managers, regulatory affairs specialists, clinical trialists, government officials and investors focused on the future of rare disease and orphan product development.

Learning Objectives

• Examine rare disease clinical and regulatory program issues and challenges;
• Discuss special considerations for study endpoint identification and development;
• Distinguish differences between working with FDA on the development of rare disease therapies as compared to more common diseases;
• Develop ideas for educating, building visibility and understanding of a rare disease programs with key stakeholders.


Speakers
avatar for Ronald Bartek

Ronald Bartek

Co-Founder/Founding President, Friedreich's Ataxia Research Alliance (FARA)
Co-founder/President, FARA, Board of Directors, NORD; NIH/NINDS National Advisory Council; partner/president, government affairs firm; 20 yrs federal service in defense, foreign policy & intelligence; U.S. Delegation to Intermediate-Range Nuclear Forces (INF) Treaty talks, Geneva... Read More →
avatar for Linda Deal

Linda Deal

Head of Patient-Centered Outcomes Measurement, Pfizer Inc.
Linda currently serves as PCOA Lead working across Pfizer’s therapeutic categories addressing efforts to develop, validate and use COAs as endpoints for providing evidence of medical treatment benefit differentiation, labeling and value. Linda has served patients and the pharmaceutical... Read More →
avatar for Jessica Foley

Jessica Foley

Head of Regulatory Services, NovusLife, LLC
Jessica is a senior regulatory professional and consultant with 10 years of experience in the global biopharmaceutical industry. She provides strategic product development and approval strategies to pharmaceutical and biotechnology companies with a focus on investigational therapies... Read More →
avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his... Read More →
avatar for David Schubert

David Schubert

Vice President of Regulatory and Quality, Stealth BioTherapeutics
David Schubert is the Vice President of Regulatory and Quality at Stealth BioTherapeutics Inc. Previously, Mr. Schubert held positions at MediVector Inc., Logical Therapeutics Inc., Coley Pharmaceutical Group, Genzyme and Cubist Pharmaceuticals. Mr. Schubert has over 30 years of experience... Read More →


Sunday June 26, 2016 9:00am - 5:00pm
112AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
 
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
 
Tuesday, June 28
 

10:30am

#227: Unique Global Regulatory Considerations and Drug Development Incentives in Rare Disease and Orphan Drug Development
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... 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... 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... Read More →


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

2:00pm

#252: Capturing Real-World Data in Rare Diseases
Limited Capacity seats available

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-591-L01-P; CME 1.00; IACET 1.00; RN 1.00

Although much can be learned from interventional-type clinical trials, a fuller picture of disease course and experiential journey can only be understood by observing patients longitudinally in real-world settings where co-morbid conditions, medication noncompliance, economic considerations, and a host of other factors come into play. However, capturing real-world data in rare diseases poses distinctive demands: fewer patients, patient inability to report on disease experience due to youth or infirmity, and need for longitudinal tracking. This panel seeks to present conventional and novel approaches to capturing real-world data in rare diseases.

Learning Objectives

Discuss the benefit of real-world data; Summarize the distinctive demands for real-world data and data collection in rare diseases; Describe ways to address real-world data demands in rare diseases.

Chair

Badri Rengarajan, MD

Speaker

Unique Paradigms to Rare Diseases Research
Donny Chen, MBA

Real-World Evidence and Rare Diseases
Derenda Nichols



Chair
avatar for Badri Rengarajan

Badri Rengarajan

Medical Affairs Lead, ASPIRE Unit, Actelion
15 years’ experience across medical affairs, product development strategy and new product planning, regulatory affairs, business development, strategy: Former Board President, International Pemphigus and Pemphigoid Foundation (rare disease foundation); Senior Medical Director, Archimedes... Read More →

Speakers
avatar for Donny Chen

Donny Chen

Senior Director, Medical Affairs Research Operations, PPD
Donny Chen, senior director of medical affairs research operations at PPD, has spent the last 18 years designing and spearheading observational research studies in a variety of therapeutic areas, with particular expertise in strategy, analysis and project management. Mr. Chen graduated... Read More →
avatar for Derenda Nichols

Derenda Nichols

Senior Director, Clinical Trial Management, Medpace
Derenda Nichols has more than 25 years experience in the CRO industry. Her diverse background includes monitoring and site management, regulatory affairs, medical writing, global project management, operations and most recently, executive management. She has successfully participated... Read More →


Tuesday June 28, 2016 2:00pm - 3:00pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
 
Wednesday, June 29
 

10:30am

#326: FDA Rare Disease Town Hall
Limited Capacity seats available

Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-608-L01-P; CME 1.25; IACET 1.25; RN 1.25

This forum will address the unique regulatory complexities and challenges specific to orphan drug development. It will provide key information about programs available to expedite the development of orphan products and will include audience Q&A.

Learning Objectives

Identify unique regulatory complexities and challenges specific to orphan drug development; Describe FDA programs available to expedite the development of orphan products.

Chair

James E. Valentine, JD

Speaker

Panelist
Jonathan C. Goldsmith, MD, FACP

Panelist
Andrew E. Mulberg, MD

Panelist
Debra Yvonne Lewis, MBA



Chair
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Speakers
avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his... Read More →
avatar for Debra Lewis

Debra Lewis

Deputy Director, Office of Orphan Products Development, Food and Drug Administration
Dr. Debra Lewis is the Deputy Director of Office of Orphan Products Development (OOPD). She facilitates incentive programs that encourage the development of promising product for rare diseases & conditions. She served as the OOPD Grants Director, Humanitarian Use Device Director... Read More →
avatar for Andrew Mulberg

Andrew Mulberg

Deputy Division Director, Gastroenterology and Inborn Errors Products, OND, CDER, FDA
Andrew E. Mulberg, MD, FAAP, CPI, is Associate Professor of Pediatrics University of Pennsylvania and Professor of Pediatrics at Unviersity of Maryland. He has principally edited a book entitled Pediatric Drug Development: Concepts and Applications published in 2009 and 2013 with... Read More →


Wednesday June 29, 2016 10:30am - 11:45am
103A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

2:00pm

#353: The Utility of Natural History Studies in Drug Development and Approval
Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: CME 1.00; IACET 1.00; RN 1.00

A well-designed natural history study can help in designing an efficient drug development program and support marketing approval. This session will discuss the utility of natural history studies and strategies for designing them.

Learning Objectives

Recognize the ways in which a natural history study can guide each stage of drug development; Identify features of natural history registries that will support drug development activities; Summarize the use of natural history controls to support important FDA drug approvals.

Chair

James E. Valentine, JD

Speaker

FDA Draft Guidance and the Utility of Natural History Studies in the Development of Drugs for Rare Diseases
Jonathan C. Goldsmith, MD, FACP

Considerations in the Use of National History Studies: Development and Registration Perspective
Camilla V. Simpson, MS

Use of Historical Controls to Support Drug Approvals
James E. Valentine, JD



Chair
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Speakers
avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his... Read More →
avatar for Camilla Simpson

Camilla Simpson

Group Vice President Regulatory Affairs, BioMarin Pharmaceutical Inc.
Camilla Simpson is a Global Regulatory Affairs executive with 20+ years in the Biopharmaceutical Industry. She is currently working for BioMarin, where she leads a team of 150 professionals. Prior to this she was with Shire Pharmaceuticals, and Abbott Laboratories. She is experienced... Read More →


Wednesday June 29, 2016 2:00pm - 3:00pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

4:00pm

#376: Pediatric Rare Disease Drug Development
Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-593-L01-P; CME 1.25; IACET 1.25; RN 1.25

This session will focus on the critical need for developing medicine for orphan diseases impacting the pediatric population, review global pediatric orphan drug development challenges, and provide information on various strategies designed to overcome these challenges.

Learning Objectives

Specify updates in regulatory policy and industry activities in pediatric orphan drug development; Discuss key clinical development challenges in demonstrating substantial evidence of effectiveness for orphan diseases in pediatrics; Review initiatives to address clinical development and regulatory approval challenges.

Chair

Kinnari Patel, PharmD, MBA, RPh

Speaker

FDA Perspective
Mary Dianne Murphy, MD

CDER Perspective
Lynne P. Yao, MD

Industry Perspective
Kinnari Patel, PharmD, MBA, RPh



Chair
avatar for Kinnari Patel

Kinnari Patel

Vice President, Head of Regulatory, Pharmacovigilance, and Compliance, Rocket Pharmaceuticals

Speakers
MM

Mary Murphy

Director, Office of Pediatric Therapeutics, Office of Special Medical Programs, FDA
Dianne Murphy, MD, FAAP, is Director of the Office of Pediatric Therapeutics (OPT) Office of the Commissioner at FDA. Dr. Murphy was a medical officer at Bethesda Naval; Dir. of the virology laboratory at the U. of Tenn., Knoxville & tenured Professor at the U. of Florida, Jacksonville... Read More →
avatar for Lynne Yao

Lynne Yao

Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA
Lynne Yao, MD, is the Director, Office of New Drugs, Division of Pediatric and Maternal Health in the Center for Drug Evaluation and Research, US Food and Drug Administration. The Division of Pediatric and Maternal Health oversees quality initiatives within the Office of New Drugs... Read More →


Wednesday June 29, 2016 4:00pm - 5:15pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA