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Sunday, June 26
 

9:00am EDT

#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 EDT
112AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
 
Monday, June 27
 

8:30am EDT

#104: Impact of Biologics, Vaccines, Oncology, and Breakthrough Therapy Designation on Traditional Global Drug Development Strategy
Limited Capacity seats available

Component Type: Forum
Level: Advanced
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-523-L01-P; CME 1.25; IACET 1.25; PDU 1.25 PMI 2166-000186; RN 1.25

This forum will provide an overview of traditional small molecule drug development, and highlight special considerations, opportunities, requirements, and risks for specialized programs, such as oncology, vaccines, biologics, or breakthrough therapies.

Learning Objectives

Explain how oncology, vaccine, and biologic drug development are similar to, or different from, a traditional small molecule program; Compare and contrast oncology, vaccine, and biologic drug development with respect to project leadership and project management strategies, approaches, and resource investment decisions; Discuss the impact of breakthrough therapy designation on each of these.

Chair

Eva M. Finney, PhD, PMP

Speaker

Panelist
Susan Uptain, PhD

Panelist
David Robinson, PhD



Chair
avatar for Eva Finney

Eva Finney

Director, Global Project & Alliance Management, Merck & Co., Inc.
Eva M. Finney, Ph.D., PMP, is Director, Global Project and Alliance Management at Merck, where she has been a Project Manager for 15 years. She has managed multiple development programs from early to late stage, including several NDA submissions leading to successful product approvals... Read More →

Speakers
DR

David Robinson

Principal Consultant, Robinson Vaccines and Biologics LLC
Independent consultant with 25 years experience in global vaccine and biologics product commercialization, process development and CMC regulatory strategy/execution (IND, BLA, PAS). Over 20 years at Merck, chaired the CMC development team that achieved approval of RotaTeq, an oral... Read More →
avatar for Susan Uptain

Susan Uptain

Head of Regulatory Affairs Operations, Baxalta
Dr. Susan Uptain is currently Head of Global Regulatory Operations at Baxalta, where she is responsible for operational excellence in execution of global regulatory strategy and tactics. Previously, she was Director of Project Management and Alliance Management at Merck & Co., Inc... Read More →
avatar for Susan Uptain

Susan Uptain

Head of Regulatory Affairs Operations, Shire
Dr. Susan Uptain is currently Head of Global Regulatory Operations at Baxalta, where she is responsible for operational excellence in execution of global regulatory strategy and tactics. Previously, she was Director of Project Management and Alliance Management at Merck & Co., Inc... Read More →


Monday June 27, 2016 8:30am - 9:45am EDT
105AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 02: Proj-Port Mgt-Strat Planning, Forum

8:30am EDT

#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, United States
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 EDT
103A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Symposium

10:45am EDT

#125: Patient Recruitment in Rare Diseases: Ideas and Framework for Out-of-the-Box Exploration
Limited Capacity seats available

Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-520-L01-P; CME 1.25; IACET 1.25; RN 1.25

Patient recruitment is a challenging activity, which becomes even more so in rare diseases where populations are small, patients are geographically dispersed, and eligible subjects may not be physically (or economically) capable of traveling to a trial site. Unfortunately, current recruiting methods (e.g., listing on trial and patient organization websites, relying on key opinion leaders and trial investigators, billboards) may be limited in reach, overly reliant on busy physicians, and too costly or cost-ineffective. It behooves clinical trialists in rare diseases to consider mechanisms beyond these standard approaches. The aim of this session is to catalyze unconventional thinking and to provide a framework to organize innovative recruitment ideas.

Learning Objectives

Discuss the challenges of patient recruitment in rare diseases; Compare conventional and out-of-the-box ideas for patient recruitment; Illustrate a framework for organizing innovative recruitment ideas.

Chair

Badri Rengarajan, MD

Speaker

Leveraging Claims Data for Targeted Recruitment
Donny Chen, MBA

Direct-to-Patient Digital Recruitment: A Targeted Approach to Recruitment Enrollment and Retention Problems
Bethany Bray

Bringing Clinical Trials to Patients: Leveraging Convergent Data Sources to Accelerate Recruitment
Scott Douglas Schliebner, MPH



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 Bethany Bray

Bethany Bray

Chief Executive Officer, Co-Founder, AutoCruitment
Bethany Bray, Oncology and Neuroscience Researcher, Entrepreneur and Technologist, founded AutoCruitment (a technology-enabled digital patient recruitment platform) with a vision to utilize the power of the internet to address recruitment, enrollment and retention problems in clinical... Read More →
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 Scott Schliebner

Scott Schliebner

Senior Vice President, Center for Rare Diseases, PRA Health Sciences
Scott Schliebner is a clinical strategist with a 20+ year background in clinical development specializing in rare diseases and orphan drug development. His experience encompasses all trial phases; a broad variety of study designs; interventional and observational studies; across a... Read More →


Monday June 27, 2016 10:45am - 12:00pm EDT
113A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 01: Clinical Operations, Session

10:45am EDT

#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 EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Symposium
 
Tuesday, June 28
 

10:30am EDT

#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 EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Session

10:30am EDT

#212: Special Populations in Clinical Pharmacology Studies
Limited Capacity seats available

Component Type: Symposium
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-524-L05-P; CME 1.25; IACET 1.25; RN 1.25

This symposium examines the complex challenges encountered in special population study design. Appropriate management of specialty populations with comorbidities on multiple medications creates challenges in protocol design and enrollment to ensure patient safety and accurate and quality data.

Learning Objectives

Discuss strategies to adapt to industry trends and changing demands of special population studies; Identify options in protocol design and the process for screening patients with comorbidities and concomitant medications; Evaluate the impact of subject population on subject safety data and on assessment of drug safety.

Chair

William B Smith

Speaker

Special Populations in Clinical Pharmacology Studies: Evolving Challenges
William B Smith

Investigational Product and Drug-Drug Interactions in Specialty Populations with Comorbidities: Options in Addressing Safety, Data, Timelines, and Costs
Harry W Alcorn, JR, PharmD

Fast to Patient: Shifting from Healthy Volunteer to Patient Population
Richard Scheyer, MD

QTcF in Special Populations
Robert Kleiman



Chair
avatar for William Smith

William Smith

CEO, Alliance for Multispecialty Research/NOCCR
William B. Smith, MD, FACC, is a Professor of Medicine at the University of Tennessee Medical Center in Knoxville, Tennessee. Dr. Smith is board certified in Nephrology, Internal Medicine, Cardiology and Critical Care. Dr. Smith is the President of New Orleans Center for Clinical... Read More →

Speakers
avatar for Harry Alcorn

Harry Alcorn

Chief Scientific Officer, DaVita Clinical Research
Chief Scientific Officer for DaVita Clinical Research a CRO, located in Minneapolis Minnesota and Lakewood Colorado. He has served as PI on over 150 protocols and Sub Investigator on over 300 protocols which have included Renal (Full and Adaptive), Hepatic and Pulmonary, along with... Read More →
RK

Robert Kleiman

Chief Medical Officer and Vice President, Global Cardiology, ERT
Dr. Kleiman is a cardiac electrophysiologist who has performed research in both basic and clinical electrophysiology and practiced clinical electrophysiology for 12 years before joining ERT in 2003. Dr. Kleiman is currently ERT’s Chief Medical Officer, and works with pharmaceutical... Read More →
avatar for Richard Scheyer

Richard Scheyer

Vice President, Medical, Medpace
Dr. Richard Scheyer is VP Medical at Medpace. Prior to Medpace, he led Experimental Medicine, BM, and PGx functions at Daiichi Sankyo. He served in leadership roles at Sanofi-Aventis and CMO at Neurotrope Bioscience. Dr. Scheyer received his BS Physics from Stanford, MD from SUNY... Read More →


Tuesday June 28, 2016 10:30am - 11:45am EDT
113A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 04: Preclin Transl-Early Clin Dev, Symposium

2:00pm EDT

#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 EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

2:00pm EDT

#236: Expanded Access: Ethical, Regulatory, and Policy Challenges and Considerations
Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-522-L04-P; CME 1.25; IACET 1.25; RN 1.25

The legislative and regulatory landscapes for expanded access to investigational therapies are evolving due in part to public attention. This session will examine strategies proposed by stakeholders to address ethical, regulatory, and policy considerations.

Learning Objectives

Recognize FDA requirements for expanded access and the current regulatory, political, and social environment; Identify challenges faced by stakeholders from ethical, regulatory, and policy perspectives; Describe industry best practices for managing expanded access programs.

Chair

Kim M. Quaintance-Lunn

Speaker

Is There a Better Way? An Industry Perspective
Sandra A. Morris, PhD, PMP

The Ethics of Compassionate Use
Alison Bateman-House



Chair
avatar for Kim Quaintance-Lunn

Kim Quaintance-Lunn

Vice President and Head, Regulatory Policy, Regulatory Affairs Americas, Bayer U.S. LLC, United States
Kim Quaintance-Lunn serves as Vice President and Head, Regulatory Policy, Regulatory Affairs Americas, at Bayer. Kim joined Bayer in February 2014 to establish and lead the US regulatory policy function for the organization. She works with colleagues to analyze regulatory policy and... Read More →

Speakers
avatar for Alison Bateman-House

Alison Bateman-House

Assistant Professor, Division of Medical Ethics, Dept of Population Health, NYU Langone Health, United States
Alison Bateman-House, PhD, MPH, MA, is an assistant professor in the Division of Medical Ethics at NYU Langone Health's School of Medicine. She is co-chair, with Arthur Caplan PhD, of the Working Group on Compassionate Use and Preapproval Access (CUPA), an academic group that studies... Read More →
avatar for Sandra Morris

Sandra Morris

Vice President, Strategy Realization, Johnson & Johnson
Dr. Morris is currently VP, Strategy Realization for the J&J CMO. Prior to joining J&J, Dr. Morris spent 18 years at Merck where she was VP, Global Project Management. Dr. Morris received her PhD in Organic Chemistry and completed postdoctoral research in biochemistry.


Tuesday June 28, 2016 2:00pm - 3:15pm EDT
108B Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 02: Proj-Port Mgt-Strat Planning, Session

4:00pm EDT

#266: Perspectives on Expanded Access to Investigational New Drugs
Limited Capacity seats available

Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-545-L01-P; CME 1.25; IACET 1.25; RN 1.25

Expanded access is the use of an investigational drug outside of a clinical trial or study. This forum will provide an opportunity to hear perspectives from regulators, industry, and patients on the use of expanded access in drug development.

Learning Objectives

Describe the different types of expanded access investigational new drugs; Explain why a sponsor might consider expanded access in their drug development programs; Recognize the patient's role and perspective on expanded access programs.

Chair

Kevin Bugin, MS, RAC

Speaker

Introduction and FDA Regulator Perspective
Jonathan P. Jarow, PhD

Industry Perspective on Expanded Access
Kenneth I. Moch, MBA

Patient Advocacy and Expanded Access
Robert Erwin

Industry Perspective on Expanded Access
Anne B. Cropp, PharmD



Chair
avatar for Kevin Bugin

Kevin Bugin

Director of Special Programs, Office of New Drugs, CDER, FDA
Kevin Bugin works for the FDA in the Office of New Drugs as the Director of Special Programs, leading various internally driven initiatives for the enhancement of the new drugs regulatory program. Prior to joining the FDA, Kevin Bugin worked in both industry and government in multiple... Read More →

Speakers
avatar for Anne Cropp

Anne Cropp

Vice President, Pfizer Inc
Anne Cropp has led the strategic planning and clinical development of several compounds in Cardiovascular and Metabolic diseases, Phases 2-4. Anne has led many business process improvements and has led several initiatives across the spectrum of clinical trial planning, design and... Read More →
avatar for Robert Erwin

Robert Erwin

President, Marti Nelson Cancer Foundation
Robert L. Erwin is co-founder and President of the Marti Nelson Cancer Foundation, a cancer patient advocacy organization with a focus on access to experimental medicine. He has served as a member of the Cancer Policy Forum of the Institute of Medicine, a member of the Research Committee... Read More →
avatar for Jonathan Jarow

Jonathan Jarow

Senior Medical Advisor, FDA
Dr. Jonathan P. Jarow is currently the senior medical advisor to the Center Director and chair of the medical policy council in CDER at FDA. Jonathan previously served as the director of CDER’s Office of Medical Policy and as deputy director of the Office of Hematology and Oncology... Read More →
avatar for Kenneth Moch

Kenneth Moch

Managing Partner, Salutramed Group, LLC
Mr. Moch is Managing Partner of The Salutramed Group, a strategic/operational advisor to life science companies. He has been co-founder or CEO of 4 firms developing therapies for life-threatening diseases and most recently was CEO of Chimerix. In August 2014, Mr. Moch and Arthur Caplan... Read More →


Tuesday June 28, 2016 4:00pm - 5:15pm EDT
108A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 08: Reg Affairs, Forum
 
Wednesday, June 29
 

10:30am EDT

#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 EDT
103A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Forum

2:00pm EDT

#333: Hearing the Patient Voice in Pharma and What Patients Want You to Know
Limited Capacity seats available

Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-517-L04-P; CME 1.25; IACET 1.25; RN 1.25

Are you considering creating a position for a director of patient engagement but have no idea where to start? Or do you just want to know more about what patients think about your drugs and what they want you to know? Learn how to do it from industry executives who have spent their time learning about patients and putting programs in place to advance advocacy efforts. They will also share real-world examples of pharma programs and experiences involving the patient voice.

Learning Objectives

Explain why it is important to have a director of patient engagement; Describe how to create the position, what the primary responsibilities should be, what the biggest challenges are in creating it, including how to overcome them; Discuss how to determine what patients think of your drug and efforts, what they are saying about it, and what they want you to know.

Chair

Ed Miseta, MBA

Speaker

Panelist
Roslyn F Schneider

Panelist
Beverly L Harrison

Panelist
Eric J. Peacock, MBA



Chair
avatar for Ed Miseta

Ed Miseta

Editor, Life Science Connect
Ed is the chief editor for the website Clinical Leader and a contributing editor to Life Science Leader magazine. He covers the latest trends, challenges, innovations, and best practices in the conduct of clinical trials. He has a BS in Business Economics and an MBA, both from The... Read More →

Speakers
avatar for Beverly Harrison

Beverly Harrison

Head, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson
Leading the Patient Support Group @ Janssen R&D (a J&J Co.), reporting directly to the Chief Medical Officer, Beverly focuses on developing strategies for patients’ needs by working with internal/external stakeholders & engaging patient advocacy organizations. She is a R&D & nonprofit... Read More →
avatar for Eric Peacock

Eric Peacock

Co-founder and Chief Executive Officer, MyHealthTeams
Eric Peacock is the CEO of MyHealthTeams. Eric cofounded MyHealthTeams with the belief that if you are diagnosed with a chronic condition, it should be easy to find the best people around to help you. To date MyHealthTeams has launched social networks in 24 different chronic conditions... Read More →
avatar for Roslyn Schneider

Roslyn Schneider

Global Patient Affairs Lead, Pfizer Inc
Roz leads Global Patient Affairs at Pfizer, a hub of patient centricity, driving integration of patient experiences and perspectives across the company and the product lifecycle. She held roles in Medical Affairs, Medical Strategy and Medicine Development for twelve years at Pfizer... Read More →


Wednesday June 29, 2016 2:00pm - 3:15pm EDT
113A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

2:00pm EDT

#335: Multi-Ethnic, Special Populations, and Patient Diversity in Clinical Trials
Limited Capacity seats available

Component Type: Symposium
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-521-L04-P; CME 1.25; IACET 1.25; RN 1.25

This symposium will cover multi-ethnic, special populations, and patient diversity in clinical trials. It will illustrate an assessment of over 10,000 trials, recently conducted by sponsor companies, to quantify the differences of pediatric trials from adult studies. It will illuminate the learning’s in site identification, site qualification, and enrollment from the DOVE study, a 14-country study representing a mix of traditional and developing regions. It will then evaluate and analyze the available data regarding the characteristics of medicines approved for older patients in European Union and the US, contributing to the information in the geriatric population in order to discuss their access to innovative drugs.

Learning Objectives

Discuss country-specific differentiation in site activation, subject recruitment, and data quality, especially around developing regions, as demonstrated by the DOVE study; Classify the difference in performance between pediatric and adult studies; Describe the challenge of addressing geriatrics adequately in the assessment of medicines including the necessity to increase global regulatory dialogue and communication.

Chair

Lisa Palladino Kim, MS

Speaker

Pediatric Studies: Experiences, Best Practices, and Trends
Conrad Hawkins

Conduct of Clinical Trials in Special Populations and Developing Regions: Challenges and Opportunities
Stephen G. Reams, MA

Medicines Development for Geriatric Patients: Unmet Needs?
Dinah Duarte, PharmD, MSc



Chair
avatar for Lisa Kim

Lisa Kim

Director of Capstone / Lecturer, Rutgers School of Health Professions, United States
Lisa Palladino Kim, is the Director of Capstone (Field Mentorship) and a Lecturer at Rutgers School of Health Professions MS in Clinical Trial Sciences Program. Lisa has 15 yrs of pharma expertise, concentrated in Clinical Ops (Pt Recruitment, Data Mgt, and Project Mgt), which allowed... Read More →

Speakers
avatar for Dinah Duarte

Dinah Duarte

Scientific Evaluation Unit, Directorate of Medicinal Products, INFARMED
Dr. Dinah Duarte is a senior assessor at the Scientific Evaluation Unit at the Directorate of Medicinal Products, in the Portuguese regulatory authority for medicines and health products (INFARMED). She is an expert member at the European Medicines Agency (EMA); the current Committee... Read More →
avatar for Conrad Hawkins

Conrad Hawkins

Associate Consultant, KMR Group Inc.
Conrad Hawkins is an associate consultant of KMR Group, a firm specializing in measuring and improving biopharmaceutical R&D performance, evaluating clinical productivity, clinical trial cycle times and enrollment processes as well as study start-up. His areas of expertise include... Read More →
avatar for Stephen Reams

Stephen Reams

Advisor, Clinical Project Management, Eli Lilly and Company
Steve has more than 27 years experience in the pharmaceutical industry, including assignments in both discovery research and clinical product development at Eli Lilly and Company. His roles have spanned multiple disciplines including clinical operations, data management, and clinical... Read More →


Wednesday June 29, 2016 2:00pm - 3:15pm EDT
113C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

4:00pm EDT

#361: Protocol Endpoints: A Clear Map to Navigate the Yellow Brick Road and the End of Endpoint-Creep
Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-533-L04-P; CME 1.00; IACET 1.00; RN 1.00

Investigators and regulatory agencies find it commonplace to receive protocols with a wide variety of primary and secondary endpoints to demonstrate efficacy for a given disease area. It is also commonplace for a medical writer or clinician to need to reinvent the wheel of endpoints for every new compound. The resulting product is that investigators and regulators receive inconsistent endpoints for similar indications, requiring the interpretive skills of a wizard. We have all gone down the ‘Yellow Brick Road’ of developing protocols and improving our endpoints for a disease area many times. Despite a plethora of research publications with clear descriptions of endpoints, it is common place to begin protocol development with no clear standards of endpoints. Clinical teams involved in protocol development often find that the long circuitous path down the ‘Yellow Brick Road’ is filled with a multitude of choices, missing bricks, potholes and wrong directions. Some of the key inefficiencies in protocol development stem from delays to endpoint finalization and resultant cascade of delays to additional protocol content such as assessments and statistical analyses. Protocol authors, whether medical directors or writers, struggle to achieve clear, consistent endpoints and all wish for ‘Dorothy shoes’ to click together for the right path. Additionally, the common occurrence of endpoint creep translates into significant increases in development costs. This session will provide insights to optimal endpoint and protocol content development required for registration protocols.

Learning Objectives

Describe the approach used to develop primary and secondary endpoints in a therapeutic area.

Chair

Anne B. Cropp, PharmD

Speaker

The ABCs of Writing Effective Clinical Study Protocols
Julia Forjanic Klapproth, PhD



Chair
avatar for Anne Cropp

Anne Cropp

Vice President, Pfizer Inc
Anne Cropp has led the strategic planning and clinical development of several compounds in Cardiovascular and Metabolic diseases, Phases 2-4. Anne has led many business process improvements and has led several initiatives across the spectrum of clinical trial planning, design and... Read More →

Speakers
avatar for Julia Forjanic-Klapproth

Julia Forjanic-Klapproth

Senior Partner, Trilogy Writing & Consulting, Germany
PhD in Developmental Neurobiology, Julia started her career as a medical writer in the pharma industry. In 2002, Julia co-founded Trilogy Writing & Consulting, a company specialized in providing medical writing of clinical regulatory documentation. In addition to company management... Read More →


Wednesday June 29, 2016 4:00pm - 5:00pm EDT
203AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 06: Med Comm-Wrtg-MSL, Session

4:00pm EDT

#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, United States
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 EDT
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
  Trk 17: Rare-Orphan Diseases, Session