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Monday, June 16
 

2:15pm EDT

#124: Expanding Innovative Master Protocol Trial Designs to Cell and Gene Therapies for Rare Diseases
Monday June 16, 2025 2:15pm - 3:15pm EDT
209ABC
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-521-L04-P; CME 1.00; RN 1.00

Master Protocols, including basket trials and umbrella trials, enable efficiencies in investigating multiple conditions or multiple treatments, respectively, under a single trial design. Applying Master Protocol trial designs to Cell & Gene therapies (CGTs) can streamline clinical and regulatory processes to speed the delivery of treatments to patients with rare diseases.

Learning Objectives

Identify barriers to and solutions for leveraging Master Protocol clinical trial designs to study Cell & Gene Therapies for rare diseases.

Chair

Nancy Bradish Myers, Esq, JD

Speaker

Thought Leader Perspective
Philip (P.J.) Brooks, PhD

Innovator Perspective
Kiran Musunuru, MD, PhD, MPH

Researcher Perspective
Jeff Allen, PhD


Speakers
avatar for Jeff Allen

Jeff Allen

President and Chief Executive Officer, Friends of Cancer Research, United States
Jeff Allen, Ph.D. serves as the President and CEO of Friends of Cancer Research (Friends). During the past 25 years, Friends has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible. As... Read More →
avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Deputy Director, Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), NIH, United States
Philip J. (P.J.) Brooks is the deputy director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC... Read More →
avatar for Kiran Musunuru

Kiran Musunuru

Professor of Medicine, University of Pennsylvania, United States
An actively practicing cardiologist and committed teacher, Kiran Musunuru, MD, PhD, MPH, ML, MRA, is Professor of Cardiovascular Medicine, Genetics, and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. His research focuses on the development of novel... Read More →
avatar for Nancy Myers

Nancy Myers

CEO and Founder, Catalyst Healthcare Consulting, United States
Nancy Bradish Myers, JD, CEO & Founder, Catalyst Healthcare Consulting, is a sought-after leader with deep expertise in health law, regulation and policy. She has led a successful strategic regulatory policy consultancy for 20 years and has been active in the DC policy arena for three... Read More →
Monday June 16, 2025 2:15pm - 3:15pm EDT
209ABC Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  02: ClinTrials-Ops, Forum

3:45pm EDT

#142: Enhancing Evidence Generation Across Pharmaceutical Life Cycle: The Importance of Integrating the Patient Lived Experience
Monday June 16, 2025 3:45pm - 4:45pm EDT
144ABC
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-540-L04-P; CME 1.00; RN 1.00

Addressing data gaps in drug development, especially capturing patient experiences in both rare and non-rare diseases, is crucial. From Proof of Concept to Proof of Value, integrated evidence planning and incorporating patient voices in Target Product Profile (TPP) creation drive comprehensive evidence generation.

Learning Objectives

Discuss the utility in global drug development context, including post-marketing efforts; Identify strategies to create a patient informed comprehensive Target Product Profile; Recognize strategies outlined in the interactive guide to align PAG and industry efforts in creating a comprehensive Target Product Profile.

Chair

Agathe Le Lay, PhD, MSc

Speaker

What are we Missing: Potential Data Gaps in Drug Development and the Importance of Capturing Lived Experience in rare and non-rare Diseases
Kristin Hatcher, MEd

From Proof of Concept to Proof of Value: An Industry Approach to Integrated Evidence Planning Throughout the Product Lifecycle
Agathe Le Lay, PhD, MSc

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Patricia Roselle

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Bruce E. Miller, PhD

Panelist
Martine Zimmermann, PharmD


Speakers
avatar for Kristin Hatcher

Kristin Hatcher

Director of Pediatric and Rare Disease/ Alpha One Lived Experience Advocate, Global Liver Institute/Alpha One Patient, United States
avatar for Agathe Le Lay

Agathe Le Lay

Global Head of Value Evidence & Patient Insights, H. Lundbeck A/S, Denmark
Agathe Le Lay is the Global Head of Value Evidence and Patient Insights at H. Lundbeck A/S, and is responsible for leading a global team responsible for the strategic planning and delivery of scientific evidence supporting R&D assets as well in-line brands. Agathe is an expert in... Read More →
avatar for Bruce Miller

Bruce Miller

Chief Scientific Officer, COPD Foundation, United States
Dr. Miller is the Chief Scientific Officer of the COPD Foundation overseeing its patient-focused research programs. Inclusion of patient partners is a requirement for any research activity the foundation supports. Dr. Miller has been a participant in the Paladin Consortium since its... Read More →
avatar for Patricia Roselle

Patricia Roselle

Head of Patient Stakeholder Engagement, Sanofi, United States
Patricia leads a global team of Patient Engagement Leads within the Patient Informed Development & Health Value Translation unit within R&D at Sanofi, using patient insights to drive a patient disease strategy and to guide Research & Development decision making. Ultimately, we aim... Read More →
avatar for Martine Zimmermann

Martine Zimmermann

Senior Vice President, Head of Global Regulatory Affairs, Ipsen , France
Dr Zimmermann joined Ipsen in January 2023 as SVP, Head of Regulatory & Quality R&D, based in France and since January 2024, SVP, Head of Global Regulatory Affairs. . She has over 25 years of combined R&D and global regulatory strategy experience. She joined Alexion Pharmaceuticals... Read More →
Monday June 16, 2025 3:45pm - 4:45pm EDT
144ABC Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  05: Patient-Impact-Product-Dev, Session
 
Tuesday, June 17
 

10:30am EDT

#226: US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Tuesday June 17, 2025 10:30am - 11:30am EDT
146BC
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-573-L04-P; CME 1.00; RN 1.00

The US Orphan Drug Act and the European regulatory framework have accelerated orphan medicine development, boosting investments in rare disease treatments. This session will gather key stakeholders to discuss successes a and challenges

Learning Objectives

Discuss the impact of regulatory policies on the rare disease ecosystem; Discuss the challenges and opportunities associated with orphan drug regulations; Examine the role of regulatory frameworks in fostering innovation and access to therapies for rare diseases; Explain the importance of collaboration between regulatory agencies, industry stakeholders, and patient advocacy groups.

Chair

Imran Shah, PhD

Speaker

US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases
Anabela Marcal, PharmD

Panelist
Amy Comstock Rick, JD

Panelist
Carolyn Shore, PhD

Panelist
Victoria Gemme, MBA, MS


Speakers
avatar for Victoria Gemme

Victoria Gemme

Director, Policy and Regulatory Affairs, National Organization for Rare Disorders, United States
Victoria Gemme is a research associate at the Duke-Margolis Center for Health Policy, where she works on a range of policy topics related to medical product development and regulation. Prior to Duke-Margolis, Victoria worked at the Cystic Fibrosis Foundation where she oversaw a diverse... Read More →
avatar for Imran Shah

Imran Shah

SVP & Head, Global Regulatory, R&D Quality and Safety (RQS), EMD Serono, United States
avatar for Carolyn Shore

Carolyn Shore

Director, National Academies, United States
Carolyn Shore serves as global health lead and director of the Forum on Drug Discovery, Development, and Translation at the National Academies of Sciences, Engineering, and Medicine. Before joining the National Academies, Carolyn was an officer on Pew’s antibiotic resistance project... Read More →
SM

Segundo Mariz

European Medicines Agency, Netherlands
AC

Amy Comstock Rick

Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA, United States
https://www.fda.gov/industry/fda-rare-disease-innovation-hub/amy-comstock-rick-jd
Tuesday June 17, 2025 10:30am - 11:30am EDT
146BC Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Session

1:45pm EDT

#244: How Medical Writing and Regulatory Strategy Can Support Accelerating the Development of Rare Disease Therapies: Insights from the FDA’s START Program Pilot
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
151A
Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-582-L04-P; CME 1.00; RN 1.00

This session will explore the FDA START program for accelerating rare disease therapies. Three speakers from companies involved in the START pilot and an FDA representative will provide insights.

Learning Objectives

Explain the objectives of the FDA START Program and its role in supporting rare disease development; Discuss how the cadence of medical writing and regulatory affairs shifts for those participating in the START program.

Chair

Robin Whitsell

Speaker

Panelist
Wei Liang, PhD

Panelist
Quyen Tran, PharmD

Panelist
Paulette Robinson, PhD, RAC

Panelist
Madiha Khalid, PhD

Panelist
Jhon Cores, PhD


Speakers
avatar for Jhon Cores

Jhon Cores

Associate Director, Medical Writing, Moderna, Inc, United States
Jhon is an Associate Director of Medical Writing at Moderna Inc., where he serves as the Medical Writing lead for various rare disease an oncology programs. He has 10 years of experience in the field working in different sectors, including university startups and contract research... Read More →
avatar for Madiha Khalid

Madiha Khalid

Senior Director, Medical Writing, Larimar Therapeutics, United States
Madiha is a pharmaceutical industry professional with a background in fundamental bench research. Her experience as a regulatory medical writer working with small, mid-size, and large pharmaceutical companies over the past 10 years has given her an in-depth understanding of all phases... Read More →
avatar for Wei Liang

Wei Liang

Pharmacologist, OTAT, CBER, FDA, United States
Dr. Liang is a Pharmacology/Toxicology Reviewer in the Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). She joined OTAT in 2006. Her primary focus is the review of preclinical testing... Read More →
PR

Paulette Robinson

Senior Director, Regulatory Affairs, Neurogene Inc., United States
Paulette Robinson, PhD, RAC is the Senior Director, Regulatory Affairs at Neurogene Inc. Her regulatory affairs experience has focused on gene therapies in rare central nervous system and ophthalmic indications. She continues to lead the global regulatory strategy for the NGN-401... Read More →
QT

Quyen Tran

Science Policy Analyst, OND, CDER, FDA, United States
Dr. Quyen Tran is a Science Policy Analyst on CDER’s Rare Diseases Team. Dr. Tran joined the FDA in 2017 and has worked in the Office of New Drugs and Office of Medical Policy, CDER. She has notable experience in rare disease drug development, policy development and implementation... Read More →
avatar for Robin Whitsell

Robin Whitsell

President, Whitsell Innovations, Inc., United States
Founder and president of Whitsell Innovations, Inc., a medical writing firm headquartered in Chapel Hill, NC, Ms. Whitsell has over 25 years of experience, specializing in global submission strategy, regulatory medical writing, emerging technologies, and data visualization. Prior... Read More →
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
151A Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  04: MedAffairs-SciComm, Session
 
Wednesday, June 18
 

10:30am EDT

#323: Why Does Drug Loss Occur? Challenges to be Addressed for Patients, and Japan's Contribution to Globalized Drug Development
Wednesday June 18, 2025 10:30am - 11:30am EDT
151B
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-629-L04-P; CME 1.00; RN 1.00

This forum will address need for a global development strategy aimed at enhancing patient access to new drugs and maximizing business opportunities in Japan. The panelists will consist of members from pharma industry associations, agency and academia

Learning Objectives

Discuss maximizing value of innovative products for the global market including Japan; Apply learnings to clinical development strategy to expand target patients and market effectively; Describe need to enhance access to innovative drugs for children and patients with rare diseases; Discuss influencing regulators, industry, and trial sites to improve the local environment and reduce burdens in global simultaneous development.

Chair

Toshiharu Sano, RPh

Speaker

Initiatives of Medical Institutions - Introduction to High-Quality Trials Using Networks
Kenichi Nakamura, MD, PhD, MBA

Changes in Japan's Pharmaceutical Regulations and Clinical Environment
Akihiro Ishiguro, PhD

What is Drug Lag and Loss: Challenges for Globalization in Japan
Nobutaka Kobayashi, MPharm


Speakers
avatar for Akihiro Ishiguro

Akihiro Ishiguro

Head of PMDA, Washington D.C. Office, Pharmaceuticals and Medical Devices Agency (PMDA), Japan
Dr. Akihiro Ishiguro, PhD is the Head of Pharmaceuticals and Medical Devices Agency (PMDA) Washington D.C. Office. His work at PMDA began in 2004 when PMDA was established, allowing him to experience the contribution of post marketing drug safety, new drug review, review management... Read More →
avatar for Nobutaka Kobayashi

Nobutaka Kobayashi

Deputy Director, Mitsubishi Tanabe Pharma Corporation, Japan
Kobayashi was engaged in medicinal chemistry as a research scientist in the pharmaceutical company for 13 years. After that, he was engaged in medical policy, drug price application, and management planning for 13 years.
avatar for Kenichi Nakamura

Kenichi Nakamura

Chief, Department of International Clinical Development, National Cancer Center Hospital, Japan
Dr. Nakamura is Chief of International Clinical Development at the National Cancer Center Hospital. After earning his MD from Kyoto University in 1999 and completing surgical training, he joined NCC to manage JCOG, Japan’s largest cancer clinical trial group. Since 2015, he has... Read More →
avatar for Toshiharu Sano

Toshiharu Sano

Associate Vice President, MSD, Japan
Toshiharu Sano has over 30 years of experience in pharmaceutical industry and has broad experiences in Clinical Research, R&D planning and Business Operations for Clinical Development in Japan. He also worked as several initiative’s leads and change manager through his career in... Read More →
Wednesday June 18, 2025 10:30am - 11:30am EDT
151B Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  07: ProjectManagement-StrategicPlanning, Forum

4:00pm EDT

#366: FDA Rare Disease Town Hall
Wednesday June 18, 2025 4:00pm - 5:00pm EDT
146BC
Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-25-662-L04-P; CME 1.00; RN 1.00

This annual town hall provides a forum for senior FDA officials to reflect on key trends and discuss opportunities in rare disease drug development and review. Officials answer moderated and audience burning questions facing the work that they do.

Learning Objectives

Describe new FDA programs and initiatives intended to help facilitate development of drugs for rare diseases; Evaluate opportunities for sponsors and patient stakeholders to engage with regulators; Identify current trends in challenges and opportunities facing orphan drug development.

Chair

James Valentine, JD, MHS

Speaker

Perspectives on Rare Immunology & Inflammation Product Development
Nikolay Nikolov, MD

Perspectives on Rare Neuroscience Product Development
Teresa Buracchio, MD


Speakers
TB

Teresa Buracchio

Director, Office of Neuroscience, OND, CDER, FDA, United States
Teresa Buracchio, MD, is Director of the Office of Neuroscience in the Center for Drug Evaluation and Research, Food and Drug Administration (FDA). She oversees the review of new drug programs for neurologic and psychiatric diseases. Dr. Buracchio joined FDA in 2013. Dr. Buracchio... Read More →
NN

Nikolay Nikolov

Director, Office of Immunology and Inflammation, OND, CDER, FDA, United States
Dr. Nikolay P. Nikolov is a board-certified Internist and Rheumatologist and is currently the Director of the Office of Immunology and Inflammation, CDER. During rheumatology fellowship at NIAMS, NIH, he studied cellular and molecular mechanisms of autoimmunity. Then, at the Sjögren's... Read More →
avatar for James Valentine

James Valentine

Director, Hyman, Phelps & McNamara, PC, United States
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 →
Wednesday June 18, 2025 4:00pm - 5:00pm EDT
146BC Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Forum |   02: ClinTrials-Ops, Forum
 
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