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Rare Disease [clear filter]
Monday, June 24
 

11:00am

#118: Statistical Considerations for Trials Using Surrogate Endpoints for Accelerated Approval
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-531-L04-P; CME 1.00; IACET 1.00; RN 1.00

For slowly progressed diseases, it may take years, if not decades, to observe clinical outcomes, such as mortality. Owing to the regulatory agencies’ accelerated approval pathway, drug developers can now consider applying for a drug approval based on surrogate endpoints. In this session, many surrogate endpoints adopted in various diseases and statistical methods for determining the validity of the surrogacy will be discussed, including their implementation in real case examples.

Learning Objectives

Describe cases that utilize the accelerated approval and what good surrogate endpoints are for common and rare diseases; Identify how to apply appropriate statistical methods for assessing the association between surrogate endpoints and clinical outcomes.

Chair

Yeh-Fong Chen, PhD

Speaker

FDA Update
Aloka Chakravarty, PhD

Statistical Assessment of Potential Surrogate Endpoints: Is the Bar too High?
Marc E. Buyse, DrSc

Using the Biomarker Endpoint for Accelerated Approval
Stephanie O. Omokaro, MD



Speakers
avatar for Marc Buyse

Marc Buyse

Chief Scientific Officer, International Drug Development Institute (IDDI)
Marc Buyse holds master’s degrees from Brussels University (Belgium), the Cranfield School of Management (UK) and a doctorate degree from the Harvard School of Public Health (USA). He had worked for 12 years at the EORTC in Brussels and for 2 years at the Dana Farber Cancer Institute... Read More →
avatar for Aloka Chakravarty

Aloka Chakravarty

Deputy Director, Office of Biostatistics, Office of Translational Sciences, CDER, FDA
Dr. Chakravarty joined CDER in 1992. She is an internationally recognized thought leader in the area of safety evaluation, surrogate markers and biomarkers in drug development and has presented and published widely on it. Dr. Chakravarty received the FDA Award of Merit in 2008 and... Read More →
avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, FDA
Dr. Chen is the Team Leader of the Division of Biometrics IX within CDER of FDA, supervising reviewers for the Division of Non-Malignant Hematology Products. She joined FDA in 2000 after receiving her Ph.D. from the University of Iowa. She has had years' experience in reviewing drug... Read More →
avatar for Stephanie Omokaro

Stephanie Omokaro

Lead Medical Officer, Office of New Drugs, CDER, FDA
Dr. Omokaro is a Lead Medical Officer in the Division of Gastroenterology and Inborn Errors Products in the Center for Drug Evaluation and Research at FDA. She was educated in Biochemistry at Cornell University, and in medicine at Boston University. She completed a pediatric residency... Read More →


Monday June 24, 2019 11:00am - 12:00pm
Room 3 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:15pm

#136: Interpretation of New Pharmacovigilance Regulations: Key Insights
Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-539-L04-P; CME 1.00; IACET 1.00; RN 1.00

Building on the ongoing collaborative efforts of nineteen sponsor companies, a key workstream has been established on how each is addressing major new pharmacovigilance regulations and guidances. This session will provide feedback on key learnings and best practices. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Tuesday, June 25, 9:30-10:30AM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Explain some of the challenges associated with major new pharmacovigilance guidelines; Discuss how companies are interpreting and implementing the aforementioned guidelines; Share best practices across the industry and learnings from feedback from regulators.

Chair

Ajay B. Singh

Speaker

Reference Satety Information: Implementing the Recommendations Noted in the CTFG
Ajay B. Singh

Update From MHRA
Mick Foy

Combination Products: Focus on FDA Guidance on Postmarketing Safety Reporting
Jane M. Carroll, BSN, MS, RN



Speakers
avatar for Jane Carroll

Jane Carroll

Head of Global Patient Safety Region - Americas, EMD Serono
Jane Carroll is the interim Head of Global Patient Safety Regions since February 25, 2019 and Head of Global Patient Safety Region Americas for the Biopharma business of Merck KGaA Darmstadt, Germany since 2015. Jane is responsible to ensure a robust system to collect and report adverse... Read More →
avatar for Mick Foy

Mick Foy

Head of Pharmacovigilance Strategy, Vigilance Intelligence, and Research Group, Medicines and Healthcare Products Regulatory Agency (MHRA)
Mick Foy is Head of Pharmacovigilance Strategy at the MHRA. Among is responsibilities is the running of the UK ADR reporting system and signal detection activities. Mick is also leads the MHRA's work with WHO and the Bill and Melinda Gates Foundation on capacity building in low and... Read More →
AS

Ajay Singh

Team Leader, Safety Evaluation and Risk Management, GlaxoSmithKline
Ajay is a nephrologist by training and currently serves as a team leader in the Safety Evaluation and Risk Management group at GSK. The main focus of the group currently is development of oncology/immune-oncology products. Ajay represents GSK on the TransCelerate work stream focused... Read More →


Monday June 24, 2019 2:15pm - 3:15pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#154: Current Initiatives on Patient Involvement in the Medicinal Product Lifecycle: CIOMS XI
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-555-L04-P; CME 1.00; IACET 1.00; RN 1.00

The forum will cover latest developments on CIOMS XI from the perspective of patients, regulators, and industry. Current thinking in more closely involving patients throughout the medicines lifecycle will be presented and audience feedback sought. Join the Clinical Safety & Pharmacovigilance Community for a follow up Round Table discussion on Tuesday, June 25, 2:00-3:00PM in the DIA Community Zone 1, Sails Pavilion.

Learning Objectives

Discuss the latest developments in patient involvement and engagement in the medicinal product lifecycle from development to post-marketing, pharmacovigilance and risk management; Describe current progress of the CIOMS XI WG in terms of involving patients and patient groups or associations more closely with medicines addressing medical needs.

Chair

Judith Zander, MD

Speaker

An Introduction to CIOMS and the CIOMS XI Initiative on Patient Involvement in the Medicinal Product Lifecycle
Herve Le Louet, DrMed, DrSc, MD, PhD, MS

FDA Perspective
Theresa Mullin, PhD

The Role of Patients in Drug Development and Lifecycle Use
Marc M. Boutin, JD

An Overview of the CIOMS XI Initiative
Kerry Leeson-Beevers

Using Patient Data to Evaluate the Benefits and Risks of Medicines
Meredith Y. Smith, PhD, MPA



Speakers
avatar for Marc Boutin

Marc Boutin

Chief Executive Officer, National Health Council (NHC)
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum. Boutin has been actively involved in patient advocacy organization management, health advocacy... Read More →
KL

Kerry Leeson-Beevers

National Development Manager, Alström Syndrome UK
I joined Alström Syndrome UK (ASUK) in 2003 when my son was diagnosed with the condition. I am now the National Development Manager responsible for overseeing the Family Support Service and I work closely with our medical teams in Birmingham, UK to support the highly specialised... Read More →
avatar for Herve Le Louet

Herve Le Louet

President, Head of PV Coordination, CIOMS and APHP
Herve Le Louet is Professor of Clinical Pharmacology. He is currently head of Pharmacovigilance department of Paris University Hospitals (AP-HP). He is an hepatologist by training and has a PhD in Pharmacogenetic from the Paris University. He is the President of CIOMS and immediate... Read More →
avatar for Theresa Mullin

Theresa Mullin

Associate Director for Strategic Programs, CDER, FDA
Theresa Mullin, Ph.D., serves as CDER’s Associate Director for Strategic Initiatives. She leads Patient-Focused Drug Development which includes implementation of the 21st Century Cures Act. She leads the CDER International program and heads the FDA delegation to ICH, IPRP and other... Read More →
avatar for Meredith Smith

Meredith Smith

Global Risk Management Officer, Global Patient Safety, Amgen Inc.
Meredith Smith is Global Risk Management Officer at Amgen, Inc. where she leads a team of 6 scientists responsible for medicinal product benefit-risk assessment and risk management. She is a behavioral scientist and health services researcher by training with over 15 years of experience... Read More →
avatar for Judith Zander

Judith Zander

Director, Office of Pharmacovigilance and Epidemiology, OSE, CDER, FDA
Judy Zander is the Director of the Office of Pharmacovigilance and Epidemiology (OPE), in the Office of Surveillance and Epidemiology (OSE), in the Center for Drug Evaluation (CDER) at the U.S. Food and Drug Administration (FDA).She oversees the Divisions of Pharmacovigilance 1 and... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 6C San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#161: Making Trials Work for Special Populations
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-558-L04-P; CME 1.00; IACET 1.00; RN 1.00

This session discusses a variety of ways to address patient needs that help recruit and retain special populations in clinical trials.

Learning Objectives

Identify useful guidelines and regulations regarding special populations in various countries; Discuss useful models of patient engagement that focus on special populations; Discuss case studies that show how some clinical trials have attracted special groups of patients.

Chair

Deborah E. Collyar

Speaker

Therapeutic Needs of Older Patients in the Era of Mobile Health
Dinah Duarte, MSc

Implementation of a Peer Advocate Model to Optimize Trial Enrollment, Diversity, and Retention
Caroline Donovan, MPH

Site Awareness and Best Practices for Inclusion of Diverse Populations in Research
Diana Foster, BSN, PhD, MSN



Speakers
avatar for Deborah Collyar

Deborah Collyar

President, Patient Advocates In Research (PAIR)
Deborah Collyar has been a patient engagement leader since her first cancer diagnosis. She founded Patient Advocates in Research (PAIR) international communication network in 1996, "where research meets reality." Deborah infuses hundreds of patient advocates into research programs... Read More →
avatar for Caroline Donovan

Caroline Donovan

Manager of Patient Engagement, LuCIN, Lupus Therapeutics, LLC
Caroline Donovan, MPH is the Manager, Patient Engagement for Lupus Therapeutics, an affiliate of Lupus Research Alliance. She is responsible for the development and implementation of a peer education program in which prospective lupus clinical trial patients are matched with a trained... Read More →
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 Diana Foster

Diana Foster

CEO, Society For Clinical Research Sites
Dr. Foster serves as a consultant to the Society for Clinical Research Sites as the Vice President of Strategy and Development. In her role she is responsible for building relationships with industry that help amplify the voice of the clinical research site. She has been integrally... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

3:30pm

#163: Strategic Integration: Is Anyone Getting it Right?
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-19-560-L04-P; CME 1.00; IACET 1.00; PDU 1.00 PMI 2166060YI7; RN 1.00

Despite the regularity of asset or company acquisitions, integration into an existing portfolio is rarely straightforward. This forum will discuss successful (and less successful) strategies for integration across various healthcare organizations.

Learning Objectives

Describe the different components that lead to the successful integration of acquired assets or companies; Compare and contrast different strategies for strategic integration and discuss best practices from a business development, project and portfolio management perspective to promote streamlined integration of an asset or company.

Chair

Erin Mulrooney, MSc

Speaker

Industry Perspective
Vijayalakshmi Agnani, MBA, MS

Industry Perspective
Erin Mulrooney, MSc

Integration of Cultures and Teams in a Hospital Setting
Elizabeth Somers, MS



Speakers
avatar for Vijayalakshmi Agnani

Vijayalakshmi Agnani

Associate Director, Transactions, Global Business Development and Licensing, Sanofi
Vijayalaksmi (Viju) Agnani is currently Associate Director, Transactions in the Global Business Development and Licensing team at Sanofi. In this role, she engages throughout all phases of business development and licensing activities to lead and manage the execution of strategic... Read More →
avatar for Erin Mulrooney

Erin Mulrooney

Associate Director, Project Management, Merck & Co., Inc.
Erin Mulrooney joined the Global Project and Alliance Management group at Merck in 2016 as an Associate Director. She specializes in the management of large-scale, complex collaborative projects in Discovery and Early Development. Prior to her transition to Project Management, she... Read More →
avatar for Erin Mulrooney

Erin Mulrooney

Director, Portfolio Management and Operations, Biocelerate
Erin Mulrooney joined the Global Project and Alliance Management group at Merck in 2016 as an Associate Director. She specializes in the management of large-scale, complex collaborative projects in Discovery and Early Development. Prior to her transition to Project Management, she... Read More →
avatar for Elizabeth Somers

Elizabeth Somers

Executive Director of Infectious Disease, Global Project and Alliance Management, Merck & Co., Inc.
Liz is an Executive Director of Infectious Diseases in Global Project and Alliance Management at Merck. In addition to accountability for the ID TA, she manages a team focused on Discovery collaborations which leverages her experience managing complex cross functional drug development... Read More →


Monday June 24, 2019 3:30pm - 4:30pm
Room 15AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Tuesday, June 25
 

8:00am

#209: Emerging Issues in CRISPR and Gene Editing Symposium
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-577-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session will discuss emerging issues related to gene editing, including standards for off-target effects, immunogenicity issues , and DNA base editors.

Learning Objectives

Describe potential concerns related to immune responses in gene editing; Discuss the concept of on-target and off-target genome editing, and collaborative approaches to address this issue; Identify the difference between gene editing that involves making a double-stranded break in DNA versus DNA base editors that do not.

Chair

Philip (P.J.) Brooks, PhD

Speaker

Immunogenicity Issues Related to Gene Editing
Zuben Erach Sauna, PhD

Update on the Genome Editing Consortium
Samantha Maragh, PhD, MS

Base Editing: Performing Chemsitry on the Genome
Alexis C Komor, PhD



Speakers
avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), NIH
Philip John (P.J.) Brooks is a Program Director in the NCATS Office of Rare Diseases Research. He is also the Working Group Coordinator for the NIH Common Fund program on Somatic Cell Genome Editing. He earned his Ph.D. in neurobiology from the University of North Carolina at Chapel... Read More →
avatar for Zuben Sauna

Zuben Sauna

Principal Investigator, OTAT, CBER, FDA
Zuben E. Sauna is a Principal Investigator and also a CMC Reviewer at the US Food and Drug Administration. His research interests lie in understanding the pharmacogenetic basis of the immune response to proteins used in therapeutic interventions as these affect efficacy and safety... Read More →
avatar for Alexis Komor

Alexis Komor

Assistant Professor, University of California San Diego
Alexis Komor received her B. S. degree in chemistry from UC Berkeley in December 2008. She then joined the lab of Jacqueline K. Barton at Caltech where she worked on the design, synthesis, and study of DNA mismatch-binding metal complexes and received her Ph.D. in 2014. She pursued... Read More →
avatar for Samantha Maragh

Samantha Maragh

Leader, Genome Editing Program, NIST
Dr. Samantha Maragh Leads the Genome Editing Program at the National Institute of Standards and Technology (NIST). Included in this program is the newly launched NIST Genome Editing Consortium, a public-private partnership to bring together government, industry and academia to address... Read More →


Tuesday June 25, 2019 8:00am - 9:15am
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#261: Patient Experience Data: How Could this Data Enhance Decision-Making at Different Stages of Medical Product Development?
Component Type: Forum
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-613-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will provide an opportunity for key stakeholders to discuss examples of various types of patient experience data, and how this data could potentially inform decision-making at different stages of medical product development.

Learning Objectives

Define patient experience data; Describe types of patient experience data that could inform medical product development; Discuss how patient experience data can inform decision making at different stages of medical product development.

Chair

Meghana Chalasani, MHA

Speaker

FDA Perspective
Michelle Campbell, PhD

Panelist
Elizabeth Hart, MD

Panelist
Isabelle Lousada, MA

Panelist
Kristina Bowyer, LPN



Speakers
avatar for Kristina Bowyer

Kristina Bowyer

Executive Director of Patient Advocacy, Ionis Pharmaceuticals
Kristina Bowyer is the Executive Director of Patient Advocacy & Engagement at Ionis Pharmaceuticals. Kristina has worked at Ionis for over 26 years and has spent the last seven years successfully building Ionis’ internal patient engagement program to ensure that the patient perspective... Read More →
avatar for Michelle Campbell

Michelle Campbell

Senior Clinical Analyst for Stakeholder Engagement, DNP, OND, CDER, FDA
Michelle Campbell is the Sr. Clinical Analyst for Stakeholder Engagement and Clinical Outcomes for the Division of Neurology Products, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Previously, Dr. Campbell was a... Read More →
avatar for Meghana Chalasani

Meghana Chalasani

Senior Research Analyst, Office of the Center Director, CDER, FDA
Meghana Chalasani currently serves as a senior research analyst for the Patient-Focused Drug Development (PFDD) Program in FDA’s Center for Drug Evaluation and Research (CDER). She works closely on CDER’s various PFDD initiatives and provides strategic, regulatory, program, and... Read More →
avatar for Isabelle Lousada

Isabelle Lousada

Chief Executive Officer and President, Amyloidosis Research Consortium
As a former patient, Lousada has spent 20 years dedicated to patient advocacy, accelerating drug development, and promoting awareness of rare diseases. Lousada founded the Amyloidosis Research Consortium (ARC) in 2015 to address the critical needs in clinical trials and related research... Read More →
avatar for Elizabeth Hart

Elizabeth Hart

Branch Chief, General Medicine 1, OTAT, CBER, FDA
Dr. Elizabeth Hart is the Branch Chief of General Medicine 1 in the Office of Tissue and Advanced Therapies in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She completed her undergraduate medical training at the University of Pennsylvania... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 5AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#269: Driving Complex Generics to Approval: What are the Keys to Success
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-620-L04-P; CME 1.25; IACET 1.25; RN 1.25

GDUFA II provides new regulatory processes designed to accelerate access to generic versions of complex products. In thisforum, FDA and industry provide examples on how to effectively use these processes.

Learning Objectives

Explain how the FDA review of ANDAs for complex generics has been improved under GDUFA II; Describe current regulatory and scientific considerations for generic combination products; Develop strategies to communicate more effectively with FDA during development of a complex product intended for submission under an ANDA.

Chair

Robert A. Lionberger, PhD

Speaker

Industry Perspective
Michelle Lee-Bourner, MSc

FDA Update
Martha Nguyen, JD



Speakers
avatar for Martha Nguyen

Martha Nguyen

Director, Division of Policy Development, OGD, CDER, FDA
Martha Nguyen is the Director of the Division of Policy Development in FDA CDER's Office of Generic Drugs, where she provides strategic leadership and direction on broad policy issues affecting generic drugs and oversees the development and clearance of regulations, guidance documents... Read More →
avatar for Michelle Lee-Bourner

Michelle Lee-Bourner

Head Global Respiratory and Biologics Regulatory Sciences, Mylan Pharma UK Ltd
Michelle Lee-Bourner has 30 years’ experience of drug pharmaceutical research and development, focused mainly on NCE’s and more recently generics, biosimilars and biologics. At Pfizer Michelle’s career spanned drug discovery, early clinical research and Regulatory Affairs. Michelle... Read More →
avatar for Robert Lionberger

Robert Lionberger

Director, Office of Research and Standards, Office of Generic Drugs, CDER, FDA
Robert Lionberger, Ph.D. serves as Director of the Office of Research and Standards (ORS) in the Office of Generic Drugs (OGD). Dr. Lionberger leads OGD’s implementation of the GDUFA science and research commitments including internal research activities and external research grants... Read More →


Tuesday June 25, 2019 2:00pm - 3:15pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Wednesday, June 26
 

10:30am

#339: Hot Topics in Quality and Regulatory Affairs for Combination Products
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Application UAN: 0286-0000-19-670-L04-P; CME 1.00; IACET 1.00; RN 1.00

Combination products are a hot topic in healthcare. These products combine two areas of medicine: medical devices and drugs/biologics. While these products open doors to exciting new possibilities in the medical world, they also bring new challenges.

This session will discuss the importance of promoting a culture of compliance to build a compliant lifecycle management infrastructure around combination products to ensure appropriate cGXPs and Quality System regulations are enforced throughout the franchise. Open discussions, case studies and dialogue will be included.

Learning Objectives

Discuss clinical trial to launch including regulatory expectations and differences for devices and drugs, how to manage parallel development programs and minimize risks for regulatory submissions; Outline best practice of handling adverse effects, usability concerns and complaints throughout drug/ device lifecycle; Describe how to establish an on-going and rigorous post-market surveillance strategy to identify product, design, and process improvement.

Chair

Rachel SW Turow, JD, MPH

Speaker

FDA Update
Melissa B. Burns, MS

Industry Perspective
James Wabby, MHS

Industry Perspective
Jim Kershner, MSc



Speakers
MB

Melissa Burns

Senior Program Manager, Office of Combination Products, FDA
Melissa Burns is a Senior Program Manager in FDA’s Office of Combination Products (OCP). In this role, she is responsible for coordinating activities related to combination product review and regulation including development and review of guidance documents, regulations, and procedures... Read More →
avatar for Rachel Turow

Rachel Turow

Executive Counsel, Regulatory Law, Teva Pharmaceutical Industries Ltd.
Rachel Turow is Executive Counsel – Regulatory Law at Teva Pharmaceutical Industries Ltd. In this role, Rachel provides regulatory legal support to Teva’s specialty and generic pharmaceutical businesses, and supports Teva’s drug-device combination products and digital health... Read More →
avatar for James Wabby

James Wabby

Executive Director, Regulatory Affairs, Devices and Combination Products, Allergan
James Wabby is the Executive Director of Regulatory Affairs (Device and Combination Products) at Allergan in Irvine, California. He has over 15 years of experience in increasing quality compliance and regulatory affairs responsibilities within the GxP regulated environment pertaining... Read More →
avatar for Jim Kershner

Jim Kershner

Human Factors Engineering Technical Lead, Eli Lilly and Company
Jim Kershner is a senior technical lead in the Human Factors group at Eli Lilly and Company. In this role, Jim was responsible for establishing the HF group at Lilly, and continues to drive human factors technical input in accordance with regulatory guidance, consensus standards... Read More →


Wednesday June 26, 2019 10:30am - 11:30am
Room 6B San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

2:00pm

#363: Neurodegenerative Diseases: Early-Stage Challenges and Optimal Models in Drug Development
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-687-L04-P; CME 1.25; IACET 1.25; RN 1.25

Intense research on the multiple fronts advanced our understanding of neurodegenerative diseases. Many disease-specific animal models have been used to test emergent medicines in neurology. Genetics and mechanism of neuronal pathogenesis contributed greatly and has created a wealth of knowledge and became the bases for novel technologies and multiple therapeutic targets for these neurodegenerative diseases. There is however substantial difficulty in choosing/accessing an optimal model or choosing measurements which would be truly informative of the product’s efficacy in neurodegenerative rare diseases or more prevalent diseases like pediatric neurodegenerative diseases and Alzheimer’s. This session will discuss nonclinical models supporting efficacy in rare neurodegenerative conditions, looking also at the challenges in early development in pediatric neurodegenerative diseases and highlight successes and failures in the development of treatments for dyslipidemia that may be applicable to the treatment of neurodegenerative diseases like Alzheimer’s disease.

Learning Objectives

Describe the importance and value of nonclinical in vivo models in rare neurodegenerative conditions and share highlights on the challenges in early development in pediatric neurodegenerative diseases; Identify optimal strategies for testing putative Alzheimer therapies; Recognize how to select and qualify biomarkers to identify patient and measure relevant drug activity; Define how to apply lessons from development of drugs to prevent Alzheimer Dementia.

Chair

Dinah Duarte, MSc

Speaker

Rare Neurodegenerative Diseases: Early-Stage Challenges and Optimal Animal Models in Orphan Drug Development
Dinah Duarte, MSc

Alzheimers Disease Prophylaxis: Lessons from Lipid Therapeutics
Richard Scheyer, MD

The Challenges in Early Development in pediatric Neurodegenerative Diseases
Scott Demarest, MD



Speakers
SD

Scott Demarest

Assistant Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine; Children's Hospital Colorado
Scott Demarest received a Bachelor of Science in biology from the University of Texas at Austin before going on to medical school at the University of Texas Health Science Center in San Antonio. He completed his residency in Pediatrics and child neurology at Children's National Health... Read More →
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 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 →


Wednesday June 26, 2019 2:00pm - 3:15pm
Room 2 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#380: Incorporating Patient Input into the Design and Conduct of Clinical Trials
Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-712-L04-P; CME 1.25; IACET 1.25; RN 1.25

This forum will outline efforts, best practices, and key considerations to enhance the incorporation of patient input into the design and conduct of clinical trials.

Learning Objectives

Outline current efforts and opportunities to incorporate patient input into the design and conduct of clinical trials (CT); Discuss approaches, best practices, and key considerations to facilitate patient recruitment, enrollment and retention, and minimize the burden of patient participation in CTs; Identify opportunities to enhance patient engagement and ensure patient input informs CTs.

Chair

Meghana Chalasani, MHA

Speaker

Panelist
Alexis Reisin Miller, JD

Panelist
Michelle Tarver, MD, PhD

Panelist
Marilena Flouri, PhD

Panelist
Mary Elmer, BSN, MSN, RN

Panelist
T.J. Sharpe, PMP



Speakers
avatar for Meghana Chalasani

Meghana Chalasani

Senior Research Analyst, Office of the Center Director, CDER, FDA
Meghana Chalasani currently serves as a senior research analyst for the Patient-Focused Drug Development (PFDD) Program in FDA’s Center for Drug Evaluation and Research (CDER). She works closely on CDER’s various PFDD initiatives and provides strategic, regulatory, program, and... Read More →
AM

Alexis Miller

Senior Director, Regulatory Science and Policy, Sanofi
Alexis is a Senior Director for Sanofi Global Regulatory Science & Policy, helping to shape the regulatory and policy environment on topics like patient-focused drug development, digital health technology regulation, and various PDUFA initiatives and FDA program implementation. Prior... Read More →
avatar for Michelle Tarver

Michelle Tarver

Director of Patient Science and Engagement, PSE, CDRH, FDA
The Patient Science and Engagement Program fosters innovative approaches to collecting, analyzing and integrating the patient perspective in the development, evaluation and surveillance of medical devices, including digital health technologies. Her program interacts with digital health... Read More →
avatar for Mary Elmer

Mary Elmer

Executive Director, Patient Innovation Organization, Merck & Co., Inc.
Mary Elmer is the Executive Director of Patient Insights for Oncology at Merck. This group seeks to obtain patient insights and engages patients in optimizing their health, wellbeing, and healthcare outcomes. In her prior role, she led a patient engagement team that brings the voice... Read More →
MF

Marilena Flouri

Mathematical Statistician, OTS, CDER, FDA
Marilena is a statistical reviewer supporting the Division of Dermatology and Dental Products (DDDP) in the Center for Drug Evaluation and Research (CDER) at the FDA. Marilena joined the FDA in 2016 immediately after receiving her Ph.D. in Statistics from the University of Maryland... Read More →
avatar for T.J. Sharpe

T.J. Sharpe

Patient Advocate, Starfish Harbor LLC
T.J. Sharpe is a Stage IV melanoma patient who shares his journey through cancer in his Patient #1 Blog.  He was diagnosed in August 2012 with metastatic melanoma, weeks after his second child was born. He has undergone six surgeries and four immunotherapy treatments over two different... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 9 San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA

4:15pm

#388: Convergence of the Regulatory Pathways for Advanced Therapy Medicinal Products
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-19-707-L04-P; CME 1.25; IACET 1.25; RN 1.25

Advanced Therapy Medicinal Products (ATMPs) include gene therapy, somatic-cell therapy, and tissue-engineered products. Collectively, these products are being developed in an evolving landscape and are associated with unique developmental challenges because of distinct manufacturing, clinical trial, and regulatory requirements. A 75-minute session is proposed facilitating the presentation and discussion of perspectives from several experienced regulatory agencies. An objective would be to provide a global overview of the various frameworks in place for the development of ATMPs, making note of ongoing and planned harmonization and cooperation projects.

This session will include collective short talks by CBER (FDA), European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, and possibly a representative from Alliance for Regenerative Medicine (ARM) on the nature and importance of regulatory convergence with ATMPs. This would be followed by a interactive session with the speaker panel and the audience.

Learning Objectives

Describe the regulatory pathway for advanced therapy medicinal products in various global regions.

Chair

Peter W. Marks, MD, PhD

Speaker

EMA Update
Anthony Humphreys, MPharm

PMDA Update
Nobumasa Nakashima, PhD



Speakers
avatar for Peter Marks

Peter Marks

Director, Center for Biologics Evaluation and Research, FDA
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching... Read More →
avatar for Anthony Humphreys

Anthony Humphreys

Head of Scientific Committees Regulatory Science Strategy, European Medicines Agency (EMA)
He is the Head of Scientific Committees Regulatory Science Strategy Division (SciRS). He is responsible for providing leadership in the Agency in the areas of Scientific Committees Coordination and Regulatory Science Strategy in support of delivering the EMRN 2020 Strategy. He chairs... Read More →
avatar for Nobumasa Nakashima

Nobumasa Nakashima

Associate Executive Director for International Programs and Asia Training Centre, Pharmaceuticals and Medical Devices Agency (PMDA)
Dr. Nakashima holds the position of Associate Executive Director for International Programs and Asia Training Centre (ATC) at PMDA since January 2020. Before this position, he had joined MHLW in April 1992, where he worked as Director of Office of International Regulatory Affairs... Read More →


Wednesday June 26, 2019 4:15pm - 5:30pm
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
 
Thursday, June 27
 

9:00am

#410: Advancing Benefit-Risk Assessment to Support FDA’s Regulatory Review of Human Drugs and Biologics
Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-19-724-L04-P; CME 1.25; IACET 1.25; RN 1.25

FDA currently uses a structured, qualitative framework for benefit-risk assessment of human drugs and biologics. This session will cover FDA’s efforts to enhance this qualitative framework and explore more advanced benefit-risk assessment methods.

Learning Objectives

Describe FDA’s approach to benefit-risk assessment under PDUFA V and VI; Discuss efforts to continue enhancing FDA’s methodological approach to benefit-risk assessment; Summarize findings from analysis of BRFs in 2017 novel drug approvals.

Chair

Graham Thompson

Speaker

Implementation of a Structured Benefit-Risk Framework into FDA's Human Drug Review
Leila Grace Lackey, MHS, PhD

CBER’s Experience with Benefit-Risk
Hong Yang, PhD

Planning and Assessing Benefit-Risk Quantitatively in the Next Decade
Susan Duke, MSc

Best Practices for Quantitative Benefit-Risk Assessments
Tommi Tervonen



Speakers
avatar for Susan Duke

Susan Duke

Mathematical Statistician, OB, OTS, CDER, FDA
Susanis is a Mathematical Statistician reviewer in CDER, assigned to pulmonary, allergy and critical care indications. She became involved in safety related industry-wide graphics in 2009 when she joined the FDA/Industry/Academia Safety Graphics WG, which lead to subsequent interests... Read More →
avatar for Leila Lackey

Leila Lackey

Operations Research Analyst, OPSA, OSP, CDER, FDA
Dr. Lackey has a Master of Health Science from Johns Hopkins in Environmental Health Sciences and a doctorate from UCLA in Environmental Science and Engineering. Professionally, she has worked for the Environmental Protection Agency and for the Food and Drug Administration leading... Read More →
avatar for Tommi Tervonen

Tommi Tervonen

Research Scientist, Evidera, PPD
Tommi Tervonen, PhD, is an Associate Director of Patient Preferences and Research Scientist at the Patient-Centered Research team of Evidera in London, UK. Dr. Tervonen joined Evidera in September 2015 after leaving his position of Assistant Professor at the Erasmus School of Economics... Read More →
GT

Graham Thompson

Operations Research Analyst, Decision Support and Analysis Team, OSP, CDER, FDA
Graham Thompson joined FDA in 2012 and currently serves as an operations research analyst for the Decision Support and Analysis Team (DSAT) in FDA’s Center for Drug Evaluation and Research (CDER). He works on CDER projects and initiatives related to benefit-risk assessment and supporting... Read More →
avatar for Hong Yang

Hong Yang

Biologist, Office of Biostatistics and Epidemiology, CBER, FDA
Dr. Hong Yang is a senior regulatory scientist in the Office of Biostatistics and Epidemiology, CBER, FDA. She holds Ph.D. degree in Biological Engineering. Dr. Yang has expertise in benefit-risk assessment of biological products. She has been devoted in regulatory review, as well... Read More →


Thursday June 27, 2019 9:00am - 10:15am
Room 6F San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA